1. BOSTON, April 07, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced that David Meeker, M.D., Chair, President and Chief Executive Officer, will participate in a fireside chat at the 20th Annual Needham & Co. Virtual Healthcare Conference on Wednesday, April 14, 2021 at 11:45 a.m. ET.

    A live audio webcast of the presentation will be available under "Events & Presentations" in the Investor Relations section of the Company's website at www.rhythmtx.com. A replay of the webcast will be available on the Rhythm website for 30 days following each presentation.

    About Rhythm Pharmaceuticals

    BOSTON, April 07, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced that David Meeker, M.D., Chair, President and Chief Executive Officer, will participate in a fireside chat at the 20th Annual Needham & Co. Virtual Healthcare Conference on Wednesday, April 14, 2021 at 11:45 a.m. ET.

    A live audio webcast of the presentation will be available under "Events & Presentations" in the Investor Relations section of the Company's website at www.rhythmtx.com. A replay of the webcast will be available on the Rhythm website for 30 days following each presentation.

    About Rhythm Pharmaceuticals

    Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity. The Company's precision medicine, IMCIVREE™ (setmelanotide), has been approved by the FDA for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing. IMCIVREE is the first-ever FDA approved therapy for these rare genetic diseases of obesity. Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity. The Company is leveraging the Rhythm Engine and the largest known obesity DNA database - now with approximately 37,500 sequencing samples - to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. The company is based in Boston, MA.

    IMCIVREE™ (setmelanotide) Indication

    IMCIVREE is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. The condition must be confirmed by genetic testing demonstrating variants in POMCPCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

    Limitations of Use

    IMCIVREE is not indicated for the treatment of patients with the following conditions as IMCIVREE would not be expected to be effective:

    • Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMCPCSK1, or LEPR variants classified as benign or likely benign;
    • Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity.

    Important Safety Information

    WARNINGS AND PRECAUTIONS

    Disturbance in Sexual Arousal: Sexual adverse reactions may occur in patients treated with IMCIVREE. Spontaneous penile erections in males and sexual adverse reactions in females occurred in clinical studies with IMCIVREE. Instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.

    Depression and Suicidal Ideation: Some drugs that target the central nervous system, such as IMCIVREE, may cause depression or suicidal ideation. Monitor patients for new onset or worsening of depression. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors.

    Skin Pigmentation and Darkening of Pre-Existing Nevi: IMCIVREE may cause generalized increased skin pigmentation and darkening of pre-existing nevi due to its pharmacologic effect. This effect is reversible upon discontinuation of the drug. Perform a full body skin examination prior to initiation and periodically during treatment with IMCIVREE to monitor pre-existing and new skin pigmentary lesions.

    Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants.

    ADVERSE REACTIONS

    • The most common adverse reactions (incidence ≥23%) were injection site reactions, skin hyperpigmentation, nausea, headache, diarrhea, abdominal pain, back pain, fatigue, vomiting, depression, upper respiratory tract infection, and spontaneous penile erection.

    USE IN SPECIFIC POPULATIONS

    Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus.

    Treatment with IMCIVREE is not recommended for use while breastfeeding.



    To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    See Full Prescribing Information for IMCIVREE.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our business strategy and plans and our participation in upcoming events and presentations. Statements using word such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881



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  2. -- Responders with HET obesity achieved mean weight loss of greater than 12 percent at nine months on setmelanotide therapy --

    -- Additional poster presentations include Phase 3 data in Bardet-Biedl and Alström syndromes and analyses of adverse events in Phase 2 and Phase 3 studies in POMC, PCSK1, or LEPR deficiency showing consistent safety results for setmelanotide --

    BOSTON, March 20, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced that three late-breaking data presentations from Phase 2 and Phase 3 studies of setmelanotide were presented during the 103rd Annual Meeting…

    -- Responders with HET obesity achieved mean weight loss of greater than 12 percent at nine months on setmelanotide therapy --

    -- Additional poster presentations include Phase 3 data in Bardet-Biedl and Alström syndromes and analyses of adverse events in Phase 2 and Phase 3 studies in POMC, PCSK1, or LEPR deficiency showing consistent safety results for setmelanotide --

    BOSTON, March 20, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced that three late-breaking data presentations from Phase 2 and Phase 3 studies of setmelanotide were presented during the 103rd Annual Meeting and Expo of the Endocrine Society (ENDO 2021) held virtually March 20-23.

    Sadaf Farooqi, M.D., Ph.D., University of Cambridge, UK, presented proof-of-concept data from Rhythm's Phase 2 study evaluating setmelanotide in individuals living with heterozygous (HET) obesity due to genetic variants in one of two alleles of the POMC, PCSK1 or LEPR gene. The oral presentation included new weight loss data that showed patients with HET obesity who were classified as setmelanotide-responsive at three months continued to lose weight as they remained on treatment, with a mean weight loss of 12.3 percent at nine months on therapy.

    In addition, two on-demand posters on setmelanotide were presented. Topline data from Rhythm's Phase 3 trial evaluating setmelanotide in patients with Bardet-Biedl syndrome (BBS) or Alström syndrome were presented by Robert Haws, M.D., of the Marshfield Clinic Research Institute. Also, Karine Clément, M.D., Ph.D., of Pitié-Salpêtrière Hospital in Paris, presented safety data from three trials evaluating setmelanotide in a total of 35 patients with severe obesity due to leptin receptor (LEPR), proopiomelanocortin (POMC), or proprotein convertase subtilisin/kexin type 1 (PCSK1) deficiency.

    "We are pleased to have world-renown physicians and scientists present these data that we believe support setmelanotide's potential as a targeted therapy to treat genetically defined patients with early-onset, severe obesity and hyperphagia. In particular, these new data in HET obesity patients demonstrate the potential for deeper, durable responses with continued therapy," said Murray Stewart, M.D., Chief Medical Officer of Rhythm. "These data give us further confidence as we advance setmelanotide through a multi-faceted clinical development program designed to address a range of rare genetic diseases of obesity caused by defects within the melanocortin-4 receptor (MC4R) pathway."

    Effects of setmelanotide observed in patients with HET obesity

    Dr. Farooqi, professor at the Wellcome-MRC Institute of Metabolic Science and NIHR Cambridge Biomedical Research Centre delivered an oral presentation entitled, "Effects of Setmelanotide in in Patients with POMC, PCSK1 or LEPR Heterozygous Deficiency Obesity in a Phase 2 Study."

    "Patients with heterozygous mutations in POMC, PCSK1 or LEPR genes that impair the MC4R pathway can suffer from severe obesity and hyperphagia, which cannot be controlled by existing therapeutic interventions, diet or exercise," Dr. Farooqi said. "These data, showing that treatment with setmelanotide achieved clinically meaningful weight loss at three months that was sustained and deepened at nine months, are quite encouraging, particularly in this refractory patient population. I look forward to learning more about the genetic causes of obesities and about setmelanotide as it is advanced into additional pivotal Phase 3 studies."

    The open-label, single-arm Phase 2 study included patients 6 years old or older with HET obesity. Participants received once daily setmelanotide at the therapeutic dose for 12 weeks. A total of 35 patients, whose mean baseline BMI was 50.3 kg/m2, were included in the analysis, which was previously reported by Rhythm in January 2021. The primary endpoint was mean percent change from baseline in body weight, and 34.3 percent of all patients in the study (12/35) responded with 5 percent or greater weight loss at three months. Mean weight loss among responders was -10.1 percent at three months.

    New data presented at ENDO 2021 included an analysis that showed that, among people who responded to treatment with setmelanotide at three months, response was maintained for up to nine months (n=9) with a mean percent change in body weight from baseline of -12.3 percent (90% CI, -16.3% to -8.4%), as of Feb. 23, 2021.

    The adverse event (AE) profile for setmelanotide continued to be consistent with what has been previously reported including skin hyperpigmentation, nausea, and injection site pruritis.

    Topline data from Phase 3 trial in Bardet-Biedl and Alström syndromes

    Dr. Haws, director of the Clinical Research Center at the Marshfield Clinic Research Institute in Marshfield, Wisc., presented a poster entitled, "A Phase 3 Trial in Participants with Obesity Due to Bardet-Biedl Syndrome or Alström Syndrome: Efficacy and Safety of the Melanocortin 4 Receptor Agonist Setmelanotide."

    As previously disclosed, data from this Phase 3 trial showed that treatment with setmelanotide was associated with significant body weight and hunger reduction in obesity due to BBS or Alström syndrome. The study met its primary and all key secondary endpoints, demonstrating statistically significant and clinically meaningful reductions in weight and hunger scores, with patients with BBS comprising all primary endpoint responders. No patients with Alström syndrome met the primary endpoint. The study included 31 patients 12 years old or older who were evaluable for the primary endpoint, including 28 patients with BBS and three patients with Alström syndrome.

    This presentation included new weight-loss data specific to adults that showed that patients 18 years old and older with BBS (n=15) had a mean weight loss from baseline of 9.4 percent. Additionally, the presentation included previously disclosed data specific to adolescents and children, which showed that patients younger than 18 years of age with BBS (n=16) had a mean reduction in BMI-Z scores of 0.8. The BMI-Z score, or BMI standard deviation score, represents the number of standard deviations from median BMI by child age and sex.

    Setmelanotide safety results from Phase 2 and Phase 3 studies in obesity due to POMC, PCSK1, or LEPR deficiency

    Dr. Clément, professor of nutrition at Pitié-Salpêtrière Hospital and Sorbonne Université in Paris presented a poster entitled, "Timing of Onset of Adverse Events with Setmelanotide, an MC4R Agonist, in Patients with Severe Obesity Due to LEPR or POMC Deficiency."

    This presentation provided data from analyses of three studies that evaluated setmelanotide in obesity due to POMC, PCSK1 or LEPR deficiency. The analysis included 35 patients (15 POMC, 2 PCSK1, 18 LEPR) across three trials. Consistent with prior clinical experience, AEs of special interest included hyperpigmentation disorders, disturbances in sexual arousal, nausea, vomiting, and injection site reactions. The onset of AEs of special interest was generally highest during the first month of treatment, with fewer events occurring during subsequent months. Apart from hyperpigmentation, all AEs resolved quickly after onset.

    The poster presentations will be available for on-demand viewing on the ENDO 2021 website, https://www.endocrine.org/endo2021, beginning at 11 a.m. on Saturday, March 20. The posters and slides from the oral presentation will be made available on Rhythm's website, https://www.rhythmtx.com/publications/, following the presentations at ENDO 2021.

    About Rhythm Pharmaceuticals

    Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity. The Company's precision medicine, IMCIVREE™ (setmelanotide), has been approved by the FDA for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing. IMCIVREE is the first-ever FDA approved therapy for these rare genetic diseases of obesity. Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity. The Company is leveraging the Rhythm Engine and the largest known obesity DNA database - now with approximately 37,500 sequencing samples - to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    IMCIVREE™ (setmelanotide) Indication

    IMCIVREE is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. The condition must be confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

    Limitations of Use

    IMCIVREE is not indicated for the treatment of patients with the following conditions as IMCIVREE would not be expected to be effective:

    • Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign;
    • Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity.

    Important Safety Information

    WARNINGS AND PRECAUTIONS

    Disturbance in Sexual Arousal: Sexual adverse reactions may occur in patients treated with IMCIVREE. Spontaneous penile erections in males and sexual adverse reactions in females occurred in clinical studies with IMCIVREE. Instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.

    Depression and Suicidal Ideation: Some drugs that target the central nervous system, such as IMCIVREE, may cause depression or suicidal ideation. Monitor patients for new onset or worsening of depression. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors.

    Skin Pigmentation and Darkening of Pre-Existing Nevi: IMCIVREE may cause generalized increased skin pigmentation and darkening of pre-existing nevi due to its pharmacologic effect. This effect is reversible upon discontinuation of the drug. Perform a full body skin examination prior to initiation and periodically during treatment with IMCIVREE to monitor pre-existing and new skin pigmentary lesions.

    Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants.

    ADVERSE REACTIONS

    • The most common adverse reactions (incidence ≥23%) were injection site reactions, skin hyperpigmentation, nausea, headache, diarrhea, abdominal pain, back pain, fatigue, vomiting, depression, upper respiratory tract infection, and spontaneous penile erection.

    USE IN SPECIFIC POPULATIONS

    Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus.

    Treatment with IMCIVREE is not recommended for use while breastfeeding.



    To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    See Full Prescribing Information for IMCIVREE.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, and our business strategy and plans, including regarding commercialization of setmelanotide. Statements using word such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881



    Primary Logo

    View Full Article Hide Full Article
  3. BOSTON, March 09, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced that three late-breaking abstracts have been accepted for presentation at the 103rd Annual Meeting and Expo of the Endocrine Society (ENDO 2021) to be held virtually March 20-23.

    In a live oral presentation, Sadaf Farooqi, M.D., Ph.D., professor at the Wellcome-MRC Institute of Metabolic Science and NIHR Cambridge Biomedical Research Centre, will present clinical data from Rhythm's Phase 2 study evaluating setmelanotide in individuals living with heterozygous (HET) obesity due to genetic variants in one…

    BOSTON, March 09, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced that three late-breaking abstracts have been accepted for presentation at the 103rd Annual Meeting and Expo of the Endocrine Society (ENDO 2021) to be held virtually March 20-23.

    In a live oral presentation, Sadaf Farooqi, M.D., Ph.D., professor at the Wellcome-MRC Institute of Metabolic Science and NIHR Cambridge Biomedical Research Centre, will present clinical data from Rhythm's Phase 2 study evaluating setmelanotide in individuals living with heterozygous (HET) obesity due to genetic variants in one of two alleles of the POMC, PCSK1 or LEPR gene.

    In addition, Robert Haws, M.D., Clinical Research Center at the Marshfield Clinic Research Institute, will present an on-demand poster detailing top-line data from a Phase 3 trial that evaluated setmelanotide in patients with Bardet-Biedl Syndrome or Alström Syndrome.

    Also, Karine Clément, M.D., Ph.D., Pitié-Salpêtrière Hospital and Sorbonne Université in Paris, will present an on-demand poster with a further analysis of safety data from two phase 3 trials evaluating setmelanotide in patients with obesity due to POMC or LEPR deficiency.

    The poster presentations will be available for on-demand viewing on the ENDO 2021 website, https://www.endocrine.org/endo2021, beginning at 11 a.m. on Saturday, March 20.

    Rhythm will also provide additional information about rare genetic diseases of obesity, genetic testing, and the company's clinical development pipeline at its virtual booth throughout the ENDO 2021 meeting.

    About Rhythm Pharmaceuticals

    Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for patients living with rare genetic diseases of obesity. Early-onset severe obesity may result from genetic variants within the melanocortin-4 receptor (MC4R) pathway, a key hypothalamic pathway that regulates hunger, caloric intake, and energy expenditure, consequently affecting body weight. Rhythm is developing setmelanotide for the treatment of rare genetic diseases of obesity that arise due to an impaired pathway, as setmelanotide has shown the potential to restore impaired pathway function.

    Setmelanotide was approved by the FDA in late 2020 for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency confirmed by genetic testing, under the brand name IMCIVREE. In addition, Rhythm is advancing a broad clinical development program with phase 2 and 3 trials evaluating setmelanotide for the treatment of obesity due to a deficiency in one of 36 additional genes associated with the MC4R pathway. Rhythm has created and is leveraging the largest known obesity DNA database - now with approximately 37,500 sequencing samples - to improve the understanding, diagnosis, and care of patients with severe obesity due to certain genetic deficiencies.

    For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    IMCIVREE™ (setmelanotide) Indication

    IMCIVREE is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. The condition must be confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

    Limitations of Use

    IMCIVREE is not indicated for the treatment of patients with the following conditions as IMCIVREE would not be expected to be effective:

    • Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign;
    • Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity.

    Important Safety Information

    WARNINGS AND PRECAUTIONS

    Disturbance in Sexual Arousal: Sexual adverse reactions may occur in patients treated with IMCIVREE. Spontaneous penile erections in males and sexual adverse reactions in females occurred in clinical studies with IMCIVREE. Instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.

    Depression and Suicidal Ideation: Some drugs that target the central nervous system, such as IMCIVREE, may cause depression or suicidal ideation. Monitor patients for new onset or worsening of depression. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors.

    Skin Pigmentation and Darkening of Pre-Existing Nevi: IMCIVREE may cause generalized increased skin pigmentation and darkening of pre-existing nevi due to its pharmacologic effect. This effect is reversible upon discontinuation of the drug. Perform a full body skin examination prior to initiation and periodically during treatment with IMCIVREE to monitor pre-existing and new skin pigmentary lesions.

    Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants.

    ADVERSE REACTIONS

    • The most common adverse reactions (incidence ≥23%) were injection site reactions, skin hyperpigmentation, nausea, headache, diarrhea, abdominal pain, back pain, fatigue, vomiting, depression, upper respiratory tract infection, and spontaneous penile erection.

    USE IN SPECIFIC POPULATIONS

    Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus.

    Treatment with IMCIVREE is not recommended for use while breastfeeding.

    To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    See Full Prescribing Information for IMCIVREE.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our business strategy and plans and our participation in upcoming events and presentations. Statements using word such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881



    Primary Logo

    View Full Article Hide Full Article
  4. -- Received FDA approval of IMCIVREE™ (setmelanotide), the first-ever therapy for chronic weight management in patients with obesity due to POMC, PCSK1 or LEPR deficiency --

    -- Announced positive topline data from Phase 3 pivotal trial evaluating setmelanotide in Bardet-Biedl and Alström syndromes; on-track to complete regulatory submissions to FDA and EMA for BBS in 2H 2021 --

    -- Announced data from five cohorts in Phase 2 Basket Study demonstrating proof-of-concept in HET POMC, PCSK1 or LEPR deficiencies, and obesity due to SRC1 and SH2B1 deficiencies --

    -- Advancing broad development program for setmelanotide, with five Phase 2 and 3 clinical studies expected to initiate in 2021 in MC4R pathway-related rare genetic diseases of obesity

    -- Received FDA approval of IMCIVREE™ (setmelanotide), the first-ever therapy for chronic weight management in patients with obesity due to POMC, PCSK1 or LEPR deficiency --

    -- Announced positive topline data from Phase 3 pivotal trial evaluating setmelanotide in Bardet-Biedl and Alström syndromes; on-track to complete regulatory submissions to FDA and EMA for BBS in 2H 2021 --

    -- Announced data from five cohorts in Phase 2 Basket Study demonstrating proof-of-concept in HET POMC, PCSK1 or LEPR deficiencies, and obesity due to SRC1 and SH2B1 deficiencies --

    -- Advancing broad development program for setmelanotide, with five Phase 2 and 3 clinical studies expected to initiate in 2021 in MC4R pathway-related rare genetic diseases of obesity --

    -- Strengthened financial position with net proceeds of $98.5M from sale of Rare Pediatric Disease PRV and $161.6M from public offering, extending cash runway through at least the second half of 2023 –

    BOSTON, March 01, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2020.

    "We enter 2021 with tremendous momentum on our journey to transform the care of people with rare genetic diseases of obesity," said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. "In recent months, we secured our first approval of IMCIVREE, validating the melanoticortin-4 receptor (MC4R) pathway as an important therapeutic target and bringing the first-ever therapy to people suffering from obesity due to POMC, PCSK1 or LEPR deficiency. The patients with defects in the MC4R pathway we have studied to date, including those with our approved indications and patients who participated in our Phase 2 Basket Study, have severe obesity and have largely failed other treatment modalities."  

    Dr. Meeker continued, "We also announced positive topline data from our pivotal, Phase 3 trial in Bardet-Biedl syndrome (BBS), and we achieved proof-of-concept in multiple additional genetic diseases of obesity, paving the way for a potentially registration-enabling trial this year. On the heels of these recent achievements, we are entering our next phase as we begin to commercialize IMCIVREE and work to expand setmelanotide's reach to address the unmet needs of many more people with a range of genetic variants in the MC4R pathway. We look forward to completing regulatory submissions to both the FDA and the EMA seeking marketing authorization for setmelanotide for the treatment of obesity in patient with BBS in the second half of 2021 and, in parallel, initiating new trials in a broad clinical program. Following the sale of our priority review voucher (PRV) and recent public offering, we are well-funded, with sufficient resources to advance the development of setmelanotide while supporting the patient community and driving awareness of genetic testing to identify and properly diagnose people with these rare conditions."

    Fourth Quarter and Recent Business Highlights:

    Pipeline and Recent Developments:

    • In January 2021, Rhythm announced new proof-of-concept interim data from its ongoing Phase 2 Basket Study across individuals with heterozygous (HET) obesity due to a genetic variant in one of the two alleles of the POMC, PCSK1, or LEPR gene, obesity due to SRC1 deficiency (SRC1) or obesity due to SH2B1 deficiency (SH2B1). Across all three populations, setmelanotide led to meaningful weight loss in approximately 30 percent to greater than 50 percent of treated patients. Rhythm believes that these data, coupled with updated sequencing results, suggest that there are approximately 100,000-200,000 potentially setmelanotide-responsive patients with HET, SRC1 or SH2B1 obesity in the United States.
    • Also in January 2021, Rhythm announced that it has leveraged its proprietary gene curation and selection strategy to identify an additional 31 genes with strong or very strong MC4R pathway relevance.
    • In December 2020, Rhythm announced positive topline results from the pivotal Phase 3 clinical trial evaluating setmelanotide in Bardet-Biedl syndrome (BBS) and Alström syndrome. The study met its primary and all key secondary endpoints, demonstrating statistically significant and clinically meaningful reductions in weight and hunger scores, with patients with BBS comprising all primary endpoint responders. No patients with Alström syndrome met the primary endpoint. Rhythm subsequently announced data from a predefined exploratory endpoint in January, demonstrating that setmelanotide was associated with statistically significant and clinically meaningful reductions in BMI-Z scores for BBS patients younger than 18 years old.
    • In November 2020, Rhythm announced that the U.S. Food and Drug Administration (FDA) approved IMCIVREE for chronic weight management in adult and pediatric patients six years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing. Visit www.IMCIVREE.com for full Prescribing Information.
    • Also in November 2020, Rhythm presented new clinical data on setmelanotide at The Obesity Society's ObesityWeek® 2020. The presentations included interim data from a Phase 2 study evaluating a once-weekly formulation of setmelanotide in healthy obese volunteers, which demonstrated safety and efficacy results comparable to the daily-dosing regimen, and additional data from the long-term extension study in obesity due to POMC deficiency, which showed durable weight loss and reductions in hunger at up to three years on setmelanotide therapy.

    Corporate:

    • In February 2021, Rhythm completed a public offering of 5,750,000 shares of its common stock at a public offering price of $30.00 per share, which included the full exercise by the underwriters of their option to purchase up to an additional 750,000 shares, for aggregate gross proceeds of approximately $172.5 million, before underwriting discounts, commissions, and offering expenses.
    • In January 2021, Rhythm announced the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for $100 million. The PRV was granted to Rhythm by the FDA with the approval of IMCIVREE for chronic weight management in adult and pediatric patients six years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency.
    • In November 2020, Rhythm announced the appointments of Camille L. Bedrosian, M.D., and Lynn Tetrault, J.D., to its Board of Directors.

    Key Upcoming Milestones:

    Rhythm expects to achieve the following milestones in 2021:

    Commercial and Regulatory Milestones:

    • Make IMCIVREE commercially available in the United States for obesity due to POMC, PCSK1 and LEPR deficiencies in the first quarter of 2021.
    • Obtain regulatory approval from the European Commission and make IMCIVREE commercially available in Europe in POMC, PCSK1 and LEPR deficiency obesities in the second half of 2021.
    • Complete regulatory submissions to both the FDA and the EMA seeking marketing authorization for setmelanotide for the treatment of obesity in patient with BBS in the second half of 2021.

    Clinical Milestones:

    • Initiate a Phase 2 clinical trial of setmelanotide in hypothalamic obesity in the first half of 2021.
    • Announce new top-line data from the ongoing exploratory Phase 2 Basket Study evaluating setmelanotide in MC4R-recusable patients in the first half of 2021.
    • Announce full data from the pivotal Phase 3 trial evaluating setmelanotide in Bardet-Biedl and Alström Syndromes in the first half of 2021.
    • Initiate a Phase 2 clinical trial of setmelanotide in pediatric patients aged two to six years old in the second half of 2021.
    • Pending FDA feedback, initiate a pivotal Phase 3 MC4R pathway trial of setmelanotide in patients with HET obesity, as well as SRC1 and SH2B1 deficiency obesities, in the second half of 2021.
    • Initiate an expanded Phase 2 Basket Study of setmelanotide in patients with variants in one of 31 additional genes with strong or very strong MC4R pathway relevance in the second half of 2021.
    • Initiate a Phase 3 potentially registration-enabling trial for the weekly formulation of setmelanotide in the second half of 2021.



    Fourth Quarter and Full Year 2020 Financial Results:

    • Cash Position: As of December 31, 2020, cash, cash equivalents and short-term investments were approximately $172.8 million, as compared to $292.5 million as of December 31, 2019. Cash, cash equivalents and short-term investments as of December 31, 2020 do not include net proceeds of $98.5 million received upon sale of Rhythm's Rare Pediatric Disease PRV in February 2021, or net proceeds of approximately $161.6 million from Rhythm's underwritten public offering of common stock, which closed in February 2021.
    • R&D Expenses: R&D expenses were $22.0 million in the fourth quarter of 2020 and $90.5 million for the year ended December 31, 2020, as compared to $24.8 million in the fourth quarter of 2019 and $109.5 million for the year ended December 31, 2019. The year-over-year decrease was primarily due to completion early in 2020 of our GO-ID genotyping study and the Phase 3 studies of setmelanotide in obesity due to POMC, PCSK1 or LEPR deficiency and a decrease in travel and conference spending due to COVID-19 restrictions. This decrease was partially offset by increases in the expanded Phase 2 Basket Study and the initiation of a new renal insufficiency pharmacokinetics study in 2020. During the year, there also was a $3.0 million payment under the licensing agreement with Ipsen related to regulatory milestones associated with the FDA approval of IMCIVREE.
    • S,G&A Expenses: S,G&A expenses were $13.1 million for the fourth quarter of 2020 and $46.1 million for the year ended December 31, 2020, as compared to $9.4 million for the fourth quarter of 2019 and $36.6 million for the year ended December 31, 2019. The year-over-year increase was primarily due to commercialization efforts focused on market access, patient engagement and disease awareness. In addition, there was an increase of approximately $0.9 million in cash related charges incurred with the separation agreements with our former chief executive officer and chief commercial officer, and $4.9 million in non-cash related stock compensation expenses related with those separation agreements as well as the hiring of the Company's current CEO in July.  
    • Net Loss: Net loss was $34.9 million for the fourth quarter of 2020 and $134.0 million for the year ended December 31, 2020, or a net loss per basic and diluted share of $0.79 and $3.04, respectively, as compared to a net loss of $33.0 million for the fourth quarter of 2019 and $140.7 million for the year ended December 31, 2019, or a net loss per basic and diluted share of $0.78 and $3.86, respectively.

    Financial Guidance: Based on its current operating plans, Rhythm expects that its existing cash, cash equivalents and short-term investments as of December 31, 2020, together with an aggregate of approximately $260.1 million in net proceeds from the February 2021 sale of its Rare Disease PRV and the February 2021 follow-on public offering, will be sufficient to fund its operating expenses and capital expenditure requirements through at least the second half of 2023.

    About Rhythm Pharmaceuticals

    Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity. The Company's precision medicine, IMCIVREE™ (setmelanotide), has been approved by the FDA for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing. IMCIVREE is the first-ever FDA approved therapy for these rare genetic diseases of obesity. Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity. The Company is leveraging the Rhythm Engine and the largest known obesity DNA database - now with approximately 37,500 sequencing samples - to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    IMCIVREE™ (setmelanotide) Indication

    IMCIVREE is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. The condition must be confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

    Limitations of Use

    IMCIVREE is not indicated for the treatment of patients with the following conditions as IMCIVREE would not be expected to be effective:

    • Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign;
    • Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity.

    Important Safety Information

    WARNINGS AND PRECAUTIONS

    Disturbance in Sexual Arousal: Sexual adverse reactions may occur in patients treated with IMCIVREE. Spontaneous penile erections in males and sexual adverse reactions in females occurred in clinical studies with IMCIVREE. Instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.

    Depression and Suicidal Ideation: Some drugs that target the central nervous system, such as IMCIVREE, may cause depression or suicidal ideation. Monitor patients for new onset or worsening of depression. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors.

    Skin Pigmentation and Darkening of Pre-Existing Nevi: IMCIVREE may cause generalized increased skin pigmentation and darkening of pre-existing nevi due to its pharmacologic effect. This effect is reversible upon discontinuation of the drug. Perform a full body skin examination prior to initiation and periodically during treatment with IMCIVREE to monitor pre-existing and new skin pigmentary lesions.

    Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants.

    ADVERSE REACTIONS

    • The most common adverse reactions (incidence ≥23%) were injection site reactions, skin hyperpigmentation, nausea, headache, diarrhea, abdominal pain, back pain, fatigue, vomiting, depression, upper respiratory tract infection, and spontaneous penile erection.

    USE IN SPECIFIC POPULATIONS

    Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus.

    Treatment with IMCIVREE is not recommended for use while breastfeeding.



    To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    See Full Prescribing Information for IMCIVREE.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, including the anticipated timing for initiation of clinical trials and release of clinical trial data and our expectations surrounding potential regulatory submissions, approvals and timing thereof, our business strategy and plans, including regarding commercialization of setmelanotide, management changes, our participation in upcoming events and presentations, and the sufficiency of our cash, cash equivalents and short-term investments to fund our operations. Statements using word such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.



    Condensed Consolidated Statements of Operations

    (in thousands, except share and per share data)

      Three months ended

    December 31, 
      Year ended December 31, 
      2020  2019   2020  2019 
    Operating expenses:             
    Research and development $21,954  $24,810   $90,450  $109,450 
    Selling, general, and administrative  13,119   9,414    46,125   36,550 
    Total operating expenses  35,073   34,224    136,575   146,000 
    Loss from operations  (35,073)  (34,224)   (136,575)  (146,000)
    Other income (expense):             
    Interest income, net  176   1,268    2,579   5,271 
    Total other income:  176   1,268    2,579   5,271 
    Net loss $(34,897) $(32,956)  $(133,996) $(140,729)
    Net loss per common share, basic and diluted $(0.79) $(0.78)  $(3.04) $(3.86)
    Weighted average common shares outstanding,

    basic and diluted
      44,216,694   42,213,180    44,127,220   36,422,450 



    RHYTHM PHARMACEUTICALS, INC.

    CONDENSED CONSOLIDATED BALANCE SHEETS

    (in thousands, except share and per share data)

           
          
      December 31, December 31, 
      2020  2019 
           
    Assets      
    Current assets:      
    Cash and cash equivalents $100,854  $62,294 
    Short-term investments  71,938   230,165 
    Prepaid expenses and other current assets  8,876   9,945 
    Total current assets  181,668   302,404 
    Property and equipment, net  3,195   3,671 
    Right-of-use asset  1,807   2,045 
    Restricted cash  403   403 
    Total assets $187,073  $308,523 
    Liabilities and stockholders' equity      
    Current liabilities:      
    Accounts payable $4,900  $10,415 
    Accrued expenses and other current liabilities  12,559   13,530 
    Lease liability  535   472 
    Total current liabilities  17,994   24,417 
    Long-term liabilities:      
    Lease liability  2,551   3,086 
    Total liabilities  20,545   27,503 
    Commitments and contingencies      
    Stockholders' equity:      
    Preferred Stock, $0.001 par value: 10,000,000 shares authorized; no shares issued and outstanding at December 31, 2020 and December 31, 2019      
    Common stock  44   44 
    Additional paid-in capital  625,762   606,307 
    Accumulated other comprehensive income  49    
    Accumulated deficit  (459,327)  (325,331)
    Total stockholders' equity  166,528   281,020 
    Total liabilities and stockholders' equity $187,073  $308,523 



    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881



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  5. BOSTON, Feb. 24, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced that David Meeker, M.D., President and Chief Executive Officer, will participate in a fireside chat at the Cowen & Co. 41st Annual Health Care Conference on Wednesday, March 3, 2021 at 2:40 p.m. ET.

    Additionally, Dr. Meeker will participate in a panel discussion, titled "New Drug Launches," on Wednesday, March 3, 2021 at 10:20 a.m. ET.

    A live audio webcast of the fireside chat will be available under "Events & Presentations" in the Investor Relations section of the Company's website at www.rhythmtx.com

    BOSTON, Feb. 24, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced that David Meeker, M.D., President and Chief Executive Officer, will participate in a fireside chat at the Cowen & Co. 41st Annual Health Care Conference on Wednesday, March 3, 2021 at 2:40 p.m. ET.

    Additionally, Dr. Meeker will participate in a panel discussion, titled "New Drug Launches," on Wednesday, March 3, 2021 at 10:20 a.m. ET.

    A live audio webcast of the fireside chat will be available under "Events & Presentations" in the Investor Relations section of the Company's website at www.rhythmtx.com. A replay of the webcast will be available on the Rhythm website for 30 days following the presentation.

    About Rhythm Pharmaceuticals

    Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity. The Company's precision medicine, IMCIVREE™ (setmelanotide), has been approved by the FDA for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing. IMCIVREE is the first-ever FDA approved therapy for these rare genetic diseases of obesity. Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity. The Company is leveraging the Rhythm Engine and the largest known obesity DNA database - now with approximately 37,500 sequencing samples - to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. The company is based in Boston, MA.

    IMCIVREE™ (setmelanotide) Indication

    IMCIVREE is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. The condition must be confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

    Limitations of Use

    IMCIVREE is not indicated for the treatment of patients with the following conditions as IMCIVREE would not be expected to be effective:

    • Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign;
    • Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity.

    Important Safety Information

    WARNINGS AND PRECAUTIONS

    Disturbance in Sexual Arousal: Sexual adverse reactions may occur in patients treated with IMCIVREE. Spontaneous penile erections in males and sexual adverse reactions in females occurred in clinical studies with IMCIVREE. Instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.

    Depression and Suicidal Ideation: Some drugs that target the central nervous system, such as IMCIVREE, may cause depression or suicidal ideation. Monitor patients for new onset or worsening of depression. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors.

    Skin Pigmentation and Darkening of Pre-Existing Nevi: IMCIVREE may cause generalized increased skin pigmentation and darkening of pre-existing nevi due to its pharmacologic effect. This effect is reversible upon discontinuation of the drug. Perform a full body skin examination prior to initiation and periodically during treatment with IMCIVREE to monitor pre-existing and new skin pigmentary lesions.

    Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants.

    ADVERSE REACTIONS

    • The most common adverse reactions (incidence ≥23%) were injection site reactions, skin hyperpigmentation, nausea, headache, diarrhea, abdominal pain, back pain, fatigue, vomiting, depression, upper respiratory tract infection, and spontaneous penile erection.

    USE IN SPECIFIC POPULATIONS

    Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus.

    Treatment with IMCIVREE is not recommended for use while breastfeeding.



    To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    See Full Prescribing Information for IMCIVREE.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our business strategy and plans and our participation in upcoming events and presentations. Statements using word such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

     

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

     

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881

     



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  6. BOSTON, Feb. 10, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced the closing of its public offering of 5,750,000 shares of its common stock at a public offering price of $30.00 per share, which includes the exercise in full by the underwriters of their option to purchase up to an additional 750,000 shares of common stock, on February 9, 2021.  The aggregate gross proceeds to Rhythm from the offering were approximately $172.5 million, before underwriting discounts, commissions, and offering expenses.  All of the shares in the offering were offered by Rhythm.

    Morgan Stanley, BofA…

    BOSTON, Feb. 10, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced the closing of its public offering of 5,750,000 shares of its common stock at a public offering price of $30.00 per share, which includes the exercise in full by the underwriters of their option to purchase up to an additional 750,000 shares of common stock, on February 9, 2021.  The aggregate gross proceeds to Rhythm from the offering were approximately $172.5 million, before underwriting discounts, commissions, and offering expenses.  All of the shares in the offering were offered by Rhythm.

    Morgan Stanley, BofA Securities, Cowen and Stifel are acting as the joint book-running managers for the offering. Canaccord Genuity is acting as lead manager for the offering.

    The offering was made pursuant to a shelf registration statement on Form S-3, including a base prospectus, that was filed by Rhythm with the Securities and Exchange Commission (SEC) and was automatically effective upon filing on November 9, 2018. A preliminary prospectus supplement relating to and describing the terms of the offering was filed with the SEC on February 3, 2021. The final prospectus supplement relating to the offering was filed with the SEC on February 5, 2021. Copies of the final prospectus supplement relating to the offering may be obtained from: Morgan Stanley, Attention: Prospectus Department, 180 Varick Street, Second Floor, New York, New York 10014, or by email at ; BofA Securities NC1-004-03-43, 200 North College Street, 3rd floor, Charlotte, NC 28255-0001, Attn: Prospectus Department, or by email at ; Cowen, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, Attention: Prospectus Department, email: , telephone: 1-833-297-2926; or Stifel, Attention: Syndicate, One Montgomery Street, Suite 3700, San Francisco, California 94104, by telephone at 415-364-2720 or by email at .

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About Rhythm Pharmaceuticals

    Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity. The Company's precision medicine, IMCIVREE™ (setmelanotide), has been approved by the FDA for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing. IMCIVREE is the first-ever FDA approved therapy for these rare genetic diseases of obesity. Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity. The Company is leveraging the Rhythm Engine and the largest known obesity DNA database - now with approximately 37,500 sequencing samples - to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. The company is based in Boston, MA.

    Company Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280



    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200



    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881





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  7. BOSTON, Feb. 04, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced the pricing of its public offering of 5,000,000 shares of its common stock at a public offering price of $30.00 per share. All of the shares are being offered by Rhythm. In addition, Rhythm has granted the underwriters a 30-day option to purchase up to an additional 750,000 shares of its common stock at the public offering price, less the underwriting discount and commission. The offering is expected to close on February 9, 2021, subject to the satisfaction of customary closing conditions.

    Morgan Stanley, BofA…

    BOSTON, Feb. 04, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced the pricing of its public offering of 5,000,000 shares of its common stock at a public offering price of $30.00 per share. All of the shares are being offered by Rhythm. In addition, Rhythm has granted the underwriters a 30-day option to purchase up to an additional 750,000 shares of its common stock at the public offering price, less the underwriting discount and commission. The offering is expected to close on February 9, 2021, subject to the satisfaction of customary closing conditions.

    Morgan Stanley, BofA Securities, Cowen and Stifel are acting as the joint book-running managers for the offering. Canaccord Genuity is acting as lead manager for the offering.

    The offering is being made pursuant to a shelf registration statement on Form S-3, including a base prospectus, that was filed by Rhythm with the Securities and Exchange Commission (SEC) and was automatically effective upon filing on November 9, 2018. A preliminary prospectus supplement relating to and describing the terms of the offering was filed with the SEC on February 3, 2021. The final prospectus supplement relating to the offering will be filed with the SEC. Copies of the final prospectus supplement relating to the offering, when available, may be obtained from: Morgan Stanley, Attention: Prospectus Department, 180 Varick Street, Second Floor, New York, New York 10014, or by email at ; BofA Securities NC1-004-03-43, 200 North College Street, 3rd floor, Charlotte, NC 28255-0001, Attn: Prospectus Department, or by email at ; Cowen, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, Attention: Prospectus Department, email: , telephone: 1-833-297-2926; or Stifel, Attention: Syndicate, One Montgomery Street, Suite 3700, San Francisco, California 94104, by telephone at 415-364-2720 or by email at .

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    Forward Looking Statement

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the completion of the public offering. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, risks associated with general economic and market conditions, the design and outcome of our clinical trials, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and the other important factors discussed under the caption "Risk Factors" in the prospectus supplement related to the offering, our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2020 and our other filings with the SEC. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this press release or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise.

    Company Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881



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  8. BOSTON, Feb. 03, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced a proposed public offering of $150 million of shares of its common stock. All shares in the offering will be offered by Rhythm. In addition, Rhythm intends to grant the underwriters a 30-day option to purchase up to an additional $22.5 million of shares of common stock at the public offering price, less the underwriting discount and commission.

    Morgan Stanley, BofA Securities, Cowen and Stifel will act as the joint book-running managers for the proposed offering. Canaccord will act as lead manager for the proposed…

    BOSTON, Feb. 03, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced a proposed public offering of $150 million of shares of its common stock. All shares in the offering will be offered by Rhythm. In addition, Rhythm intends to grant the underwriters a 30-day option to purchase up to an additional $22.5 million of shares of common stock at the public offering price, less the underwriting discount and commission.

    Morgan Stanley, BofA Securities, Cowen and Stifel will act as the joint book-running managers for the proposed offering. Canaccord will act as lead manager for the proposed offering. The offering is subject to market and other customary closing conditions, and there can be no assurance as to whether or when the offering may be completed.

    The proposed offering is being made pursuant to a shelf registration statement on Form S-3, including a base prospectus, that was filed by Rhythm with the Securities and Exchange Commission (SEC) and was automatically effective upon filing on November 9, 2018. The proposed offering will be made only by means of a preliminary prospectus supplement and the accompanying base prospectus. A copy of the preliminary prospectus relating to the offering, when available, may be obtained from: Morgan Stanley, Attention: Prospectus Department, 180 Varick Street, Second Floor, New York, New York 10014, or by email at ; BofA Securities NC1-004-03-43, 200 North College Street, 3rd floor, Charlotte, NC 28255-0001, Attn: Prospectus Department, or by email at ; Cowen, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, Attention: Prospectus Department, email: , telephone: 1-833-297-2926; or Stifel, Attention: Syndicate, One Montgomery Street, Suite 3700, San Francisco, California 94104, by telephone at 415-364-2720 or by email at   The final terms of the offering will be disclosed in a final prospectus supplement to be filed with the SEC.

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    Forward Looking Statement

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the completion, timing and size of our proposed public offering and our expectations with respect to granting the underwriters a 30-day option to purchase additional shares. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, risks associated with general economic and market conditions, the design and outcome of our clinical trials, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and the other important factors discussed under the caption "Risk Factors" in the preliminary prospectus supplement related to the offering, our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2020 and our other filings with the SEC. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this press release or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise.

    Company Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881



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  9. -- Clinical data from Phase 2 Basket Study including five cohorts totaling 65 patients demonstrated proof-of-concept in HET POMC, PCSK1 or LEPR deficiencies, SRC1 and SH2B1 deficiency obesities –

    -- Phase 2 Basket Study responder rates and updated sequencing results suggest 100,000-200,000 potentially setmelanotide-responsive HET POMC, PCSK1 or LEPR deficiencies and SRC1 or SH2B1 deficiency obesity patients in U.S. --

    -- New data from pivotal Phase 3 trial in Bardet-Biedl syndrome showed statistically significant and clinically meaningful improvements in pediatric BMI-Z scores --

    -- Plan to initiate new pivotal MC4R Pathway Study to evaluate setmelanotide for treatment of HET obesity and SRC1 or SH2B1 deficiency obesities and new exploratory

    -- Clinical data from Phase 2 Basket Study including five cohorts totaling 65 patients demonstrated proof-of-concept in HET POMC, PCSK1 or LEPR deficiencies, SRC1 and SH2B1 deficiency obesities –

    -- Phase 2 Basket Study responder rates and updated sequencing results suggest 100,000-200,000 potentially setmelanotide-responsive HET POMC, PCSK1 or LEPR deficiencies and SRC1 or SH2B1 deficiency obesity patients in U.S. --

    -- New data from pivotal Phase 3 trial in Bardet-Biedl syndrome showed statistically significant and clinically meaningful improvements in pediatric BMI-Z scores --

    -- Plan to initiate new pivotal MC4R Pathway Study to evaluate setmelanotide for treatment of HET obesity and SRC1 or SH2B1 deficiency obesities and new exploratory Basket Trial in patients with obesity defined by variants in one of 31 MC4R pathway-associated genes --

    -- Virtual R&D event today; live webcast begins at 8 a.m. ET --

    BOSTON, Jan. 26, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced positive proof-of-concept data from multiple cohorts in its Phase 2 Basket Study evaluating setmelanotide in patients with severe obesity due to genetic variants in the melanocortin-4 receptor (MC4R) pathway, and provided an update on its genetic sequencing efforts. Rhythm will review these updates at its virtual Research & Development event, beginning today at 8 a.m. ET.

    Rhythm provided a comprehensive update on its clinical development efforts with setmelanotide, including new interim data from its ongoing Phase 2 Basket Study and pivotal Phase 3 trial in Bardet-Biedl syndrome (BBS) and Alström syndrome. The Company also discussed plans to initiate a potentially registration-enabling Phase 3 trial evaluating setmelanotide in patients with MC4R pathway deficiencies due to a variant in one of the two alleles in the POMC, PCSK1, or LEPR genes (HET obesity), as well as the SRC1 and SH2B1 genes. Additionally, Rhythm provided an update on its sequencing efforts, now comprised of samples from approximately 37,500 individuals with severe obesity, and detailed an additional planned exploratory study to evaluate setmelanotide for the treatment of obesity due to a deficiency in one of 31 additional genes associated with the MC4R pathway.

    Rhythm estimates, based on the response rates observed in its Phase 2 Basket Study, together with the updated sequencing results, that there are between 100,000 and 200,000 people with HET obesity or SRC1 or SH2B1 deficiency obesity in the U.S. who could potentially benefit from setmelanotide therapy.

    "This is an exciting moment for Rhythm as we enter a new frontier in the treatment of rare genetic diseases of obesity," said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. "These new data show that setmelanotide led to meaningful weight loss in approximately 30 percent to greater than 50 percent of treated patients with genetic variants in the MC4R pathway across multiple genes. Across the setmelanotide development program we have been encouraged that patients who lost 5 percent or more weight in the first 12 to 16 weeks of therapy - our responder group - tended to go on to lose more weight over time. We believe these new data, coupled with our translational research and sequencing efforts, further validate our gene-selection strategy and support advancement into later-stage studies for certain genetic variants, as well as the expansion of our earlier-stage setmelanotide development program into as many as 31 additional MC4R pathway genes later this year. Moreover, the positive interim data in these new patient cohorts, while representing diseases which are individually rare, expand our estimates of the potential of setmelanotide to serve a far larger cumulative patient population than has been examined to date."

    Murray Stewart, M.D., Chief Medical Officer of Rhythm, added, "We have deepened our understanding of the impact of setmelanotide in specific populations, showing a statistically significant and clinically meaningful reduction in BMI-Z scores among BBS pediatric patients in our pivotal Phase 3 trial in BBS and Alström syndrome. BMI-Z scores correct for the fact a child or adolescent may be growing in height, and therefore would be expected to gain weight. These data increase our confidence in setmelanotide's potential as a precision medicine for patients with a range of MC4R pathway variants and reinforce our commitment to working with urgency to maximize setmelanotide's reach."

    Updated Interim Clinical Data from Phase 2 Basket Study

    Rhythm today announced new proof-of-concept interim data from its ongoing Phase 2 Basket Study across individuals with one of three distinct rare genetic diseases of obesity: HET obesity due to a genetic variant in one of the two alleles of the POMC, PCSK1 or LEPR gene (HETs); obesity due to SRC1 deficiency (SRC1); and obesity due to SH2B1 deficiency (SH2B1). Across five cohorts, 65 patients with severe obesity were eligible for analysis as of a cutoff date of Dec. 17, 2020.

    The primary endpoint of the study is the percent of patients in each subgroup showing at least a 5 percent loss of body weight over three months.

    HET Obesity (POMC, LEPR, PCSK1) highlights include:

    • Overall, 12 of 35 patients (34.3 percent) achieved the primary endpoint. This full analysis includes six patients who withdrew early.
    • Mean reduction from baseline in body weight across all 35 patients was -3.7 percent, which includes both clinical responders and non-responders;
    • Among the 12 patients who achieved the primary endpoint (responder group), the mean reduction from baseline in body weight was -10.1 percent;
    • Patients with HET obesity were stratified into three pre-specified cohorts by classification of their genetic variants according to American College of Medical Genetics (ACMG) guidelines:
      • Four of eight patients (50.0 percent) with a pathogenic or likely pathogenic variant achieved greater than 5 percent weight loss;
      • Four of eight patients (50.0 percent) with the N221D variant of the PCSK1 gene achieved greater than 5 percent weight loss;
      • Four of 19 patients (21.1 percent) with a variant of unknown significance (VOUS) achieved greater than 5 percent weight loss.

    Data from the SRC1 and SH2B1 cohorts were based on an interim analysis of patients who completed 12 weeks of therapy. This analysis did not include 15 patients who withdrew early due to COVID-related issues, adverse events or were lost to follow-up. Also not included were data from 12 patients who remain ongoing but have not yet reached 12 weeks of therapy.

    Obesity due to SRC1 deficiency highlights include:

    • Four of 13 patients (30.8 percent) achieved the primary endpoint;
    • Mean reduction from baseline in body weight across all 13 patients was -3.7 percent, which included both clinical responders and non-responders;
    • Among the four patients who achieved the primary endpoint (responder group) the mean reduction from baseline in body weight was -8.4 percent.

    Obesity due to SH2B1 deficiency highlights include:

    • Nine of 17 patients (52.9 percent) achieved greater than 5 percent weight loss over 12 weeks of therapy;
    • Mean reduction from baseline in body weight across all 17 patients was -3.9 percent, which included clinical responders and non-responders;
    • Among the nine patients who achieved the primary endpoint (responder group), the mean reduction from baseline in body weight was -7.1 percent.

    Consistent with prior clinical experience, setmelanotide was generally well tolerated in each of these rare genetic diseases of obesity. The most common treatment-emergent adverse events (TEAEs) included mild injection site reactions, hyperpigmentation, and nausea and vomiting, which occurred early in the treatment course. There were no serious adverse events (SAEs) related to treatment with setmelanotide.

    The Company is in discussions with the U.S. Food and Drug Administration (FDA) to define a potential path for setmelanotide towards registration for these indications. Pending the outcome of these discussions, Rhythm plans to initiate a pivotal Phase 3 trial evaluating setmelanotide in patients with HET obesity and SRC1 and SH2B1 deficiency obesities in the second half of 2021.

    Setmelanotide Showed Statistically Significant and Clinically Meaningful Improvements in BMI-Z Scores in Pediatric Patients with BBS

    In December 2020, Rhythm announced that the Phase 3 trial evaluating setmelanotide in patients with BBS and Alström syndrome met its primary endpoint and all key secondary endpoints, with statistically significant and clinically meaningful reductions in weight and hunger, with patients with BBS comprising all primary endpoint responders. No patients with Alström syndrome met the primary endpoint.

    Today, Rhythm shared data from a predefined exploratory endpoint showing the impact of setmelanotide on BMI-Z scores for patients younger than 18 years old with BBS. The BMI-Z score, or BMI standard deviation score, represents the number of standard deviations from median BMI by child age and sex. Setmelanotide was associated with statistically significant and clinically meaningful reductions in BMI-Z scores in patients with BBS:

    • In 16 patients younger than 18 with BBS, the mean BMI-Z score was reduced from 3.74 at baseline to 2.98 for a reduction of -0.76, or -24.5 percent (p=0.0006).

    Rhythm remains on track to complete regulatory submissions to both the FDA and European Medicines Agency (EMA) for BBS in the second half of 2021. The Company expects to determine next steps for Alström syndrome upon completing a full analysis of the final data from the Phase 3 trial.

    Genetic Sequencing Data from Approximately 37,500 Obese Individuals Provides Updated U.S. Prevalence Estimates for HET Obesity, SRC1 and SH2B1 Deficiency Obesities

    Rhythm, together with its collaborators, continues to expand its sequencing efforts in individuals living with early-onset, severe obesity. Since September 2019, Rhythm has increased its internal database of sequencing samples from 13,567 to approximately 37,500. The Company is using these data to support its research, patient finding and community building efforts in order to better understand rare genetic diseases of obesity.

    Rhythm's genetic sequencing results demonstrated that approximately 10 to 15 percent of obese individuals sampled as of Sept. 30, 2020, showed a relevant MC4R pathway genotype in the POMC, PCSK1, LEPR HETS, and SRC1 or SH2B1 genes. The Company estimates, based on a combination of these sequencing yields, as well as clinical data on setmelanotide response rates among patients with variants in these genes, that there are 100,000 to 200,000 potentially setmelanotide-responsive patients in the United States with these rare genetic diseases of obesity.

    Expansion of the Rhythm Basket Study into Additional 31 MC4R Pathway Genes

    Rhythm also presented its proprietary gene curation and selection strategy specifically designed to evaluate a gene's relevance to the MC4R pathway with the goal of identifying genetic patient populations with the potential to benefit from setmelanotide therapy. Using this proprietary approach, Rhythm has identified an additional 31 MC4R pathway genes with strong or very strong pathway relevance, which it plans to evaluate in its expanded basket trial.

    "We believe the new data presented today validate our proprietary approach to gene selection and efficient clinical development. Leveraging our extensive scientific expertise and years of internal research, we have developed a process that allows us to identify new genes that may be responsive to setmelanotide and to quickly advance them into our Basket Study for clinical evaluation," said Alastair Garfield, Ph.D., Vice President, Head of Translational Research of Rhythm Pharmaceuticals. "Based on our success across a range of genes with strong and very strong pathway relevance, we look forward to potentially expanding our clinical development program into patients with an additional 31 pathway genes later this year."

    Pending discussions with the FDA, Rhythm plans to initiate a new exploratory MC4R Pathway Basket Trial in patients with 31 new genes in the second half of 2021.

    Webcast Information:

    The live webcast of today's event will be available under "Events & Presentations" in the Investors & Media section of the Company's website at http://www.rhythmtx.com. A replay of the webcast will be available on the Rhythm website for 30 days following the event.

    About Rhythm Pharmaceuticals

    Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity. The Company's precision medicine, IMCIVREE™ (setmelanotide), has been approved by the FDA for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing. IMCIVREE is the first-ever FDA approved therapy for these rare genetic diseases of obesity. Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity. The Company is leveraging the Rhythm Engine and the largest known obesity DNA database - now with approximately 37,500 sequencing samples - to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    IMCIVREE™ (setmelanotide) Indication

    IMCIVREE is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. The condition must be confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

    Limitations of Use

    IMCIVREE is not indicated for the treatment of patients with the following conditions as IMCIVREE would not be expected to be effective:

    • Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign;
    • Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity.

    Important Safety Information

    WARNINGS AND PRECAUTIONS

    Disturbance in Sexual Arousal: Sexual adverse reactions may occur in patients treated with IMCIVREE. Spontaneous penile erections in males and sexual adverse reactions in females occurred in clinical studies with IMCIVREE. Instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.

    Depression and Suicidal Ideation: Some drugs that target the central nervous system, such as IMCIVREE, may cause depression or suicidal ideation. Monitor patients for new onset or worsening of depression. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors.

    Skin Pigmentation and Darkening of Pre-Existing Nevi: IMCIVREE may cause generalized increased skin pigmentation and darkening of pre-existing nevi due to its pharmacologic effect. This effect is reversible upon discontinuation of the drug. Perform a full body skin examination prior to initiation and periodically during treatment with IMCIVREE to monitor pre-existing and new skin pigmentary lesions.

    Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants.

    ADVERSE REACTIONS

    • The most common adverse reactions (incidence ≥23%) were injection site reactions, skin hyperpigmentation, nausea, headache, diarrhea, abdominal pain, back pain, fatigue, vomiting, depression, upper respiratory tract infection, and spontaneous penile erection.

    USE IN SPECIFIC POPULATIONS

    Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus.

    Treatment with IMCIVREE is not recommended for use while breastfeeding.

    To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    See Full Prescribing Information for IMCIVREE.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the timing and expansion of clinical trials, the number of U.S. patients with HET obesity or SRC1 or SH2B1 deficiency obesity who may be responsive to setmelanotide, validation of the Company's gene-selection strategy, efficient clinical development, advancement into later-stage studies and expansion of its earlier-stage development program, the expansion of the estimated number of patients who may be responsive to setmelanotide, regulatory plans, future development for the treatment of Alström syndrome, and identifying genetic patient populations that may potentially be treated with setmelanotide. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, the impact of our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    om

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881



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  10. BOSTON, Jan. 19, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced that it plans to host a virtual event on Tuesday, January 26, 2021 from 8 to 10 a.m. ET to provide an update on its ongoing exploratory Phase 2 Basket Study and genetic sequencing efforts.

    At the event, Rhythm management plans to report data for setmelanotide in individuals living with heterozygous (HET) obesity due to genetic variants in one of two alleles of the POMC, PCSK1 or LEPR gene, as well as SRC1 and SH2B1 deficiency obesities, and plans to provide an update on data from the Company's sequencing efforts…

    BOSTON, Jan. 19, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced that it plans to host a virtual event on Tuesday, January 26, 2021 from 8 to 10 a.m. ET to provide an update on its ongoing exploratory Phase 2 Basket Study and genetic sequencing efforts.

    At the event, Rhythm management plans to report data for setmelanotide in individuals living with heterozygous (HET) obesity due to genetic variants in one of two alleles of the POMC, PCSK1 or LEPR gene, as well as SRC1 and SH2B1 deficiency obesities, and plans to provide an update on data from the Company's sequencing efforts, which now comprises samples from approximately 37,500 individuals with severe obesity.

    A live webcast of the event will be available under "Events & Presentations" in the Investors & Media section of the Company's website at http://www.rhythmtx.com. A replay of the webcast will be available on the Rhythm website for 30 days following the event.

    About Rhythm Pharmaceuticals

    Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity. The company's precision medicine, IMCIVREE™ (setmelanotide), has been approved by the FDA for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing. IMCIVREE is the first-ever FDA approved therapy for these rare genetic diseases of obesity. Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity. The Company is leveraging the Rhythm Engine and the largest known obesity DNA database - now with more than 30,000 sequencing samples - to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    IMCIVREE™ (setmelanotide) Indication

    IMCIVREE is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. The condition must be confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

    Limitations of Use

    IMCIVREE is not indicated for the treatment of patients with the following conditions as IMCIVREE would not be expected to be effective:

    • Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign; and
    • Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity.

    Important Safety Information

    WARNINGS AND PRECAUTIONS

    Disturbance in Sexual Arousal: Sexual adverse reactions may occur in patients treated with IMCIVREE. Spontaneous penile erections in males and sexual adverse reactions in females occurred in clinical studies with IMCIVREE. Instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.

    Depression and Suicidal Ideation: Some drugs that target the central nervous system, such as IMCIVREE, may cause depression or suicidal ideation. Monitor patients for new onset or worsening of depression. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors.

    Skin Pigmentation and Darkening of Pre-Existing Nevi: IMCIVREE may cause generalized increased skin pigmentation and darkening of pre-existing nevi due to its pharmacologic effect. This effect is reversible upon discontinuation of the drug. Perform a full body skin examination prior to initiation and periodically during treatment with IMCIVREE to monitor pre-existing and new skin pigmentary lesions.

    Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants.

    ADVERSE REACTIONS

    • The most common adverse reactions (incidence ≥23%) were injection site reactions, skin hyperpigmentation, nausea, headache, diarrhea, abdominal pain, back pain, fatigue, vomiting, depression, upper respiratory tract infection, and spontaneous penile erection.



    USE IN SPECIFIC POPULATIONS

    Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus. Treatment with IMCIVREE is not recommended for use while breastfeeding.



    To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    See Full Prescribing Information for IMCIVREE.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, the anticipated timing for release of clinical data, data to be presented in the upcoming presentation and our business strategy and plans. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, risks associated with data analysis and reporting, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and the other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881



    Primary Logo

    View Full Article Hide Full Article
  11. BOSTON, Jan. 05, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced that it has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $100M.

    The PRV was granted to Rhythm by the U.S. Food and Drug Administration (FDA) with the approval of IMCIVREE™ (setmelanotide) for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing.

    "Rhythm…

    BOSTON, Jan. 05, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced that it has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $100M.

    The PRV was granted to Rhythm by the U.S. Food and Drug Administration (FDA) with the approval of IMCIVREE™ (setmelanotide) for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing.

    "Rhythm is focused on transforming the care of people living with rare genetic diseases of obesity," said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. "The non-dilutive capital from the sale of our PRV provides an important source of additional funding to advance the continued development of setmelanotide as a precision medicine for people whose severe obesity and insatiable hunger may be caused by genetic variants associated with the melanocortin-4 (MC4R) receptor pathway."

    According to the agreement, Rhythm will receive an upfront payment of $100M upon the closing of the transaction, which is subject to customary closing conditions and is expected to occur following expiration of the applicable U.S. antitrust clearance requirements. Jefferies LLC acted as exclusive financial advisor to Rhythm on this transaction. Latham & Watkins LLP acted as legal advisor to Rhythm.

    The non-dilutive funds expected from this transaction are in addition to the $201.8 million in cash, cash equivalents and short-term investments Rhythm reported as of September 30, 2020.

    About the Rare Pediatric Disease Priority Review Voucher Program

    The program is intended to encourage development of new drug and biological products for prevention and treatment of certain rare pediatric diseases. A PRV may be issued to the sponsor of a rare pediatric disease product application and would entitle the holder to priority review of a single New Drug Application or Biologics License Application, which reduces the target review time and could lead to an expedited approval. The sponsor receives the PRV upon approval of the rare pediatric disease product application and it can be sold without limitation, subject to applicable FDA requirements for filing and use.

    About Rhythm Pharmaceuticals

    Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity. The company's precision medicine, IMCIVREE™ (setmelanotide), has been approved by the FDA for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing. IMCIVREE is the first-ever FDA approved therapy for these rare genetic diseases of obesity. Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity. The Company is leveraging the Rhythm Engine and the largest known obesity DNA database - now with more than 30,000 sequencing samples - to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    IMCIVREE™ (setmelanotide) Indication

    IMCIVREE is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. The condition must be confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

    Limitations of Use

    IMCIVREE is not indicated for the treatment of patients with the following conditions as IMCIVREE would not be expected to be effective:

    • Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign;
    • Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity.

    Important Safety Information

    WARNINGS AND PRECAUTIONS

    Disturbance in Sexual Arousal: Sexual adverse reactions may occur in patients treated with IMCIVREE. Spontaneous penile erections in males and sexual adverse reactions in females occurred in clinical studies with IMCIVREE. Instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.

    Depression and Suicidal Ideation: Some drugs that target the central nervous system, such as IMCIVREE, may cause depression or suicidal ideation. Monitor patients for new onset or worsening of depression. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors.

    Skin Pigmentation and Darkening of Pre-Existing Nevi: IMCIVREE may cause generalized increased skin pigmentation and darkening of pre-existing nevi due to its pharmacologic effect. This effect is reversible upon discontinuation of the drug. Perform a full body skin examination prior to initiation and periodically during treatment with IMCIVREE to monitor pre-existing and new skin pigmentary lesions.

    Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants.

    ADVERSE REACTIONS

    • The most common adverse reactions (incidence ≥23%) were injection site reactions, skin hyperpigmentation, nausea, headache, diarrhea, abdominal pain, back pain, fatigue, vomiting, depression, upper respiratory tract infection, and spontaneous penile erection.

    USE IN SPECIFIC POPULATIONS

    Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus.

    Treatment with IMCIVREE is not recommended for use while breastfeeding.

    To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    See Full Prescribing Information for IMCIVREE.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the completion of the contemplated sale of the PRV (including the satisfaction of the conditions thereto), the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, and our business strategy and plans. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881



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  12. ZUG, Switzerland, Jan. 5, 2021 /PRNewswire/ -- Pharvaris, a clinical-stage company focused on the discovery and development of novel oral bradykinin-B2-receptor antagonists for the treatment of hereditary angioedema (HAE) and other bradykinin-B2-receptor-mediated indications, today announced two key appointments to its Board of Directors: David Meeker, M.D., as Chair and Robert Glassman, M.D., as Director. Dr. Meeker currently serves as the President, Chief Executive Officer and Chairman of Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM). Dr. Glassman currently serves as a Public Equity Venture Partner at OrbiMed Advisors.

    "Both David and Robert are incredibly talented, passionate and admired industry leaders," said Berndt Modig, Chief Executive Officer and co-founder of Pharvaris. "I look forward to partnering with David and Robert and leveraging their guidance in the months and years ahead as we execute on our strategy to improve treatment options for patients with HAE and other bradykinin-B2-receptor-mediated indications. David's experience in rare disease drug development and commercialization will be instrumental as we continue to progress our clinical programs. Robert will provide valuable financial insights to our team, in support of our corporate goals."

    Prior to joining Rhythm Pharmaceuticals, Dr. Meeker served as President and Chief Executive Officer of KSQ Therapeutics, Inc. from 2017 to 2020. Prior to joining KSQ, Dr. Meeker worked at Sanofi Genzyme from 2011 to 2017, in a variety of roles, including as President and Chief Executive Officer of Genzyme, a Sanofi Company, as a member of Sanofi's Executive Committee, and as Executive Vice President and Head of Sanofi Genzyme, Sanofi's specialty care unit with responsibility for rare diseases, multiple sclerosis, oncology and immunology franchises. Prior to joining Genzyme, Dr. Meeker was Director of the Pulmonary Critical Care Fellowship at the Cleveland Clinic. Dr. Meeker has served as a Director of Rhythm Pharmaceuticals, Inc. since November 2015 and as its Chairman since April 2017. Dr. Meeker has served as Chair of Trevi Therapeutics since 2017. He also served as a Director of MyoKardia, Inc. until its acquisition by Bristol Myers Squibb. Dr. Meeker holds a medical degree from the University of Vermont Medical School and completed the Advanced Management Program at Harvard Business School in 2000.

    Dr. Robert Glassman returned to OrbiMed Advisors in January 2021 as the only venture partner in public equity; he had been a private equity partner 2009-10. Dr. Glassman had been a senior investment banker for 17 years, at Merrill Lynch for the majority of his career, and as Vice Chairman at Credit Suisse since 2015. Earlier in his career, Dr. Glassman was with Merrill Lynch Global Private Equity, where he oversaw a very successful healthcare portfolio, and at McKinsey & Co, where he consulted for a wide range of clients within their Pharmaceutical and Medical Products practice. Dr. Glassman was a board-certified hematologist-oncologist who remains on the faculty of Weill Cornell as a Clinical Assistant Professor of Medicine. He has co-authored numerous articles in peer-reviewed journals and spoken widely in industry and academic forums on clinical development, reimbursement, and data interpretation. Dr. Glassman received his AB magna cum laude from Harvard College and an MD from Harvard Medical School. He completed his residency in internal medicine at the Hospital of the University of Pennsylvania, and his fellowship in hematology and oncology at Weill Cornell. He also spent several years as a basic science investigator at Rockefeller University in the laboratory of Hidesaburo Hanafusa where he received Howard Hughes Medical Institute and American Cancer Society awards.

    About Pharvaris

    Pharvaris is a clinical-stage company focused on bringing oral bradykinin-B2-receptor antagonists to patients. By targeting this clinically proven therapeutic target with novel small molecules, the Pharvaris team is advancing new alternatives to injected therapies for all sub-types of HAE and other bradykinin-mediated diseases. The company brings together executives with a breadth of expertise across pharmaceutical development and rare disorders, including HAE. For more information, visit https://pharvaris.com/.

    Investor Contact

    Chad Rubin, Solebury Trout

    Media Contact

    Maggie Beller, Russo Partners, LLC

     

    +1-646-942-5631

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/pharvaris-expands-board-of-directors-with-appointment-of-david-meeker-md-as-chair-and-robert-glassman-md-as-director-301200812.html

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  13. -- Study met primary endpoint and all key secondary endpoints with statistically significant and clinically meaningful reductions in weight and hunger --

    -- All primary endpoint responders were patients with BBS --

    -- Plan to submit sNDA to FDA for BBS in the second half of 2021 --

    -- Company to host conference call today at 8 a.m. ET --

    BOSTON, Dec. 22, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced positive topline results from a pivotal Phase 3 clinical trial evaluating setmelanotide, the company's melanocortin-4 receptor…

    -- Study met primary endpoint and all key secondary endpoints with statistically significant and clinically meaningful reductions in weight and hunger --

    -- All primary endpoint responders were patients with BBS --

    -- Plan to submit sNDA to FDA for BBS in the second half of 2021 --

    -- Company to host conference call today at 8 a.m. ET --

    BOSTON, Dec. 22, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced positive topline results from a pivotal Phase 3 clinical trial evaluating setmelanotide, the company's melanocortin-4 receptor (MC4R) agonist, for the treatment of insatiable hunger and severe obesity in individuals with Bardet-Biedl syndrome (BBS) or Alström syndrome.

    The study met its primary and all key secondary endpoints, demonstrating statistically significant and clinically meaningful reductions in weight and hunger scores. All primary endpoint responders were patients with BBS. There were three evaluable patients with Alström syndrome and none of them met the primary endpoint.

    "These Phase 3 results add to our growing understanding of setmelanotide's potential to treat people living with rare genetic diseases of obesity," said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. "We are pleased with the robust response observed in BBS patients, which supports our goal of delivering a precision medicine to this well-characterized patient population who suffer from insatiable hunger and severe, early-onset obesity. Although we are disappointed that none of the three evaluable Alström patients met the primary endpoint, we are encouraged by trends in hunger and weight reduction in some patients and look forward to evaluating the full data as we finalize our path forward in this indication."

    Rhythm enrolled 32 individuals with BBS and six individuals with Alström syndrome in the pivotal cohort for this Phase 3 trial. The primary analysis was conducted on 31 evaluable participants (28 with BBS and three with Alström syndrome) 12 years old and older. Five study participants (three with BBS and two with Alström syndrome) were younger than 12 years old at enrollment.

    The analysis of the primary endpoint shows:

    • 11 of 31 or 34.5 percent1 of participants achieved the primary endpoint of at least 10 percent reduction in body weight from baseline at approximately 52 weeks of therapy (p=0.0024);



      ◦ 11 of 28 patients with BBS achieved 10 percent reduction in body weight;

      ◦ 0 of 3 patients with Alström syndrome achieved 10 percent reduction in body weight.

    The analysis of the key secondary endpoints shows:

    • Mean reduction from baseline in body weight was -6.2 percent (p<0.0001);
    • Mean reduction from baseline in most hunger rating was -30.8 percent (p<0.0001);
    • 60.2 percent of participants achieved at least 25 percent reduction in most hunger scores from baseline at approximately 52 weeks of therapy (p<0.0001).

    Consistent with prior clinical experience, setmelanotide was generally well tolerated:

    • Treatment-emergent adverse events (TEAEs) included mild injection site reactions and nausea with infrequent vomiting;
    • There were no serious adverse events (SAEs) related to treatment with setmelanotide;
    • Eight patients discontinued from study drug treatment during the trial, five due to AEs (one on placebo at the time), and three for other reasons (one on placebo at the time).

    "Despite conducting this trial during the COVID-19 pandemic, which has been linked to weight gain across many populations, these data demonstrate that setmelanotide reduced weight and alleviated hunger in BBS patients. Overall, these results reinforce the potential value of the MC4R pathway as a therapeutic target for some rare genetic diseases of obesity and underscore our belief that obesity is a complex, multifactorial disease," said Murray Stewart, M.D., Chief Medical officer of Rhythm. "We are particularly pleased by these results given that nearly half of the evaluable patients were growing adolescents, who we would normally expect to gain weight. We look forward to completing further analyses on the full data, which will include BMI and BMI-Z scores, two measures that more accurately assess weight gain in adolescents and may further demonstrate the impact from treatment with our precision therapy."

    Rhythm plans to complete regulatory submissions to both the U.S. Food and Drug Administration (FDA) and the European Medicines Association (EMA) for BBS in the second half of 2021. The company expects to finalize a path forward for Alström syndrome upon completing a full analysis of the final data from this trial.

    About the Pivotal Phase 3 Trial in BBS and Alström Syndrome

    The combined pivotal Phase 3 trial is a multinational, open-label, single-arm study consisting of 52 weeks of treatment with setmelanotide. Participants were blinded and randomized for the first 14 weeks of the trial to receive either placebo or setmelanotide therapy. Those participants who began the trial on setmelanotide continued therapy for a total of 52 weeks, while those on placebo went on to receive 52 weeks of setmelanotide therapy after completion of the 14-week placebo period.

    Based on the statistical analysis plan, the primary analysis was completed for 28 of the 31 patients who reached or exceeded 52 weeks on setmelanotide therapy, as well as three patients who were randomized to the placebo group during the 14-week double-blind period, who have not yet reached 52 weeks on therapy. The Company expects to complete a subsequent analysis of the full data in the first quarter of 2021. Rhythm anticipates sharing the full data from this Phase 3 clinical trial in a forthcoming publication or in a presentation at an upcoming medical meeting.

    Conference Call Information

    Rhythm Pharmaceuticals will host a live conference call and webcast at 8:00 a.m. ET today to discuss these clinical data. The conference call may be accessed by dialing (844) 498-0570 (domestic) and (409) 983-9726 (international) and referring to conference ID 3794695. A webcast of the conference call will be available in the Investors section of the Rhythm website at ir.rhythmtx.com. The archived webcast will be available on Rhythm's website approximately two hours after the conference call and will be available for 90 days following the call.

    About Bardet-Biedl and Alström Syndromes

    BBS and Alström syndrome are ultra-rare genetic diseases that affect multiple organ systems. Clinical features of BBS may include cognitive impairment, polydactyly, renal dysfunction, hypogonadism, and visual impairment. Clinical features of Alström syndrome may include progressive visual and auditory impairment, insulin resistance and Type 2 diabetes, hyperlipidemia, progressive kidney dysfunction, cardiomyopathy, and short stature in adulthood. Insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life may be common in people living with either BBS or Alström syndrome. There is great variability in presentation and severity of these symptoms across individuals with BBS or Alström syndrome. In the United States, the Company estimates that BBS affects approximately 1,500 to 2,500 people and that Alström syndrome affects approximately 500 people. Currently, there are no approved therapies targeting the MC4 receptor pathway for reducing body weight and hunger in BBS or Alström syndrome.

    About Setmelanotide

    Setmelanotide is an MC4R agonist. The MC4 receptor is part of the key biological pathway that independently regulates hunger, caloric intake, and energy expenditure. Variants in genes may impair the function of the MC4R pathway, potentially leading to hyperphagia and early-onset, severe obesity. Rhythm is currently developing setmelanotide as a targeted therapy to potentially restore the function of an impaired MC4R pathway and, in so doing, potentially reduce hunger and weight in patients with rare genetic diseases of obesity. In November 2020, the FDA approved IMCIVREE™ (setmelanotide) for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing. The FDA has also granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4R pathway, which includes BBS and Alström syndrome. The EMA has granted PRIority MEdicines (PRIME) designation for setmelanotide for the treatment of obesity and the control of hunger associated with deficiency diseases of the MC4R pathway.

    About Rhythm Pharmaceuticals

    Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity. The company's precision medicine, IMCIVREE™ (setmelanotide), has been approved by the FDA for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing. IMCIVREE is the first-ever FDA approved therapy for these rare genetic diseases of obesity. Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity. The Company is leveraging the Rhythm Engine and the largest known obesity DNA database - now with more than 30,000 sequencing samples - to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    IMCIVREE™ (setmelanotide) Indication

    IMCIVREE is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. The condition must be confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

    Limitations of Use

    IMCIVREE is not indicated for the treatment of patients with the following conditions as IMCIVREE would not be expected to be effective:

    • Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign;
    • Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity.

    Important Safety Information

    WARNINGS AND PRECAUTIONS

    Disturbance in Sexual Arousal: Sexual adverse reactions may occur in patients treated with IMCIVREE. Spontaneous penile erections in males and sexual adverse reactions in females occurred in clinical studies with IMCIVREE. Instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.

    Depression and Suicidal Ideation: Some drugs that target the central nervous system, such as IMCIVREE, may cause depression or suicidal ideation. Monitor patients for new onset or worsening of depression. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors.

    Skin Pigmentation and Darkening of Pre-Existing Nevi: IMCIVREE may cause generalized increased skin pigmentation and darkening of pre-existing nevi due to its pharmacologic effect. This effect is reversible upon discontinuation of the drug. Perform a full body skin examination prior to initiation and periodically during treatment with IMCIVREE to monitor pre-existing and new skin pigmentary lesions.

    Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants.

    ADVERSE REACTIONS

    • The most common adverse reactions (incidence ≥23%) were injection site reactions, skin hyperpigmentation, nausea, headache, diarrhea, abdominal pain, back pain, fatigue, vomiting, depression, upper respiratory tract infection, and spontaneous penile erection.

    USE IN SPECIFIC POPULATIONS

    Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus.

    Treatment with IMCIVREE is not recommended for use while breastfeeding.



    To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    See Full Prescribing Information for IMCIVREE.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, including our expectations surrounding potential regulatory submissions and timing thereof, and our business strategy and plans. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Marisa Monte

    Berry & Company Public Relations

    212-253-8881

    1 Response rate is based on imputation methodology accepted by FDA. All 11 responders were BBS patients; of the 11, two were initially randomized to placebo and had not reached 52 weeks of treatment at data cut.



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  14. BOSTON, Dec. 11, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced the appointments of Camille L. Bedrosian, M.D., and Lynn Tetrault, J.D., to its Board of Directors.

    "I am thrilled to welcome both Camille and Lynn to our Board of Directors, as both leaders have tremendous experience building and leading teams that work effectively to bring new medicines to patients in need," said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. 

    "With her experience on the senior executive team of a top-10, multi-national pharmaceutical company, Lynn brings a unique…

    BOSTON, Dec. 11, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced the appointments of Camille L. Bedrosian, M.D., and Lynn Tetrault, J.D., to its Board of Directors.

    "I am thrilled to welcome both Camille and Lynn to our Board of Directors, as both leaders have tremendous experience building and leading teams that work effectively to bring new medicines to patients in need," said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. 

    "With her experience on the senior executive team of a top-10, multi-national pharmaceutical company, Lynn brings a unique perspective on developing global operations, leadership and culture," Dr. Meeker said. "And with an unwavering commitment to patients and families as both a clinician and biopharmaceutical executive, Camille has demonstrated success in developing and delivering new medicines for rare diseases."

    Dr. Bedrosian brings significant rare and ultra-disease experience to the Rhythm Board. As Executive Vice President (EVP) and Chief Medical Officer (CMO) at Ultragenyx Pharmaceutical, she provides strategic leadership for translational research, global clinical development and medical affairs for a company with three commercial products and a deep pipeline of product candidates. On joining the Rhythm Board, she said, "This is an exciting time for Rhythm as it brings the first-ever approved precision medicine to people with rare genetic diseases of obesity. I look forward to supporting the Company's unique integrated approach in leveraging the largest known genetic obesity database to advance the scientific understanding of and potential treatments for obesity due to genetic causes."

    Ms. Tetrault spent more than 20 years at AstraZeneca, including seven years as EVP for Human Resources and Corporate Affairs and a member of the company's senior executive team. She is currently Lead Independent Director of Neo Genomics Clinical Laboratories, a cancer diagnostics company. On joining the Rhythm Board, she said, "Rhythm is forging new ground in rare genetic diseases of obesity with a commitment to organizational excellence and a true partnership with the community of patients, caregivers, advocacy groups and health care providers. I look forward to joining Camille on the Board and supporting Rhythm as it transforms into a global, commercial-stage company."

    About Camille L. Bedrosian, M.D.

    Dr. Bedrosian serves as EVP and CMO at Ultragenyx Pharmaceutical, a rare disease company with a diverse portfolio of approved therapies and product candidates, where she provides strategic leadership to the clinical development and translational research programs. She oversees Medical Affairs, Global Clinical Development groups, Clinical Operations and Drug Safety/Pharmacovigilance. In addition, she is a member of the MIT Corporation Visiting Committee for the Department of Biology. Previously, Dr. Bedrosian served as SVP and CMO at Alexion Pharmaceuticals, Inc., where she provided leadership for the development of drugs and drug candidates including those designed to address devastating rare diseases such as Soliris® (eculizumab). She also had served as CMO at ARIAD Pharmaceuticals, and previously in the Clinical Research and Development Department of Genetics Institute, Inc. Before transitioning to industry, Dr. Bedrosian was an Assistant Professor of Medicine at Duke University Medical Center where she was a member of the Duke Comprehensive Cancer Center. Dr Bedrosian holds an A.B. from Harvard University, M.D. from Harvard Medical School, and M.S. in Biophysics from the Massachusetts Institute of Technology.

    About Lynn A. Tetrault, J.D.

    Ms. Tetrault is a global business leader with more than 25 years of experience in the health care industry. Ms. Tetrault spent most of her career with Astra Zeneca PLC, serving in a variety of senior roles, including as EVP of Human Resources and Corporate Affairs for seven years. She joined the Board of Neo Genomics Clinical Laboratories in 2015 and is currently Lead Independent Director. Ms. Tetrault is the founder of Anahata Leadership, an advisory firm focused on supporting the leadership and development of executive women. She is a Fellow of Simmons University's Institute for Inclusive Leadership and is a Director of Paradigm for Parity, a non-profit organization whose mission is to close the gender parity gap in corporate leadership by 2030. She began her career as a lawyer in private healthcare practice in Boston. Ms. Tetrault holds a B.A. from Princeton University and a J.D. from the University of Virginia.

    About Rhythm Pharmaceuticals

    Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity. The company's precision medicine, IMCIVREE™ (setmelanotide), has been approved by the U.S. FDA for chronic weight management in people with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing. IMCIVREE is the first-ever FDA approved therapy for these rare genetic diseases of obesity. Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity and expects to report topline pivotal Phase 3 data in Bardet-Biedl and Alström syndromes in late 2020 or early 2021. The Company is leveraging the Rhythm Engine and the largest known obesity DNA database - now with more than 30,000 sequencing samples - to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    IMCIVREE™ (setmelanotide) Indication

    IMCIVREE is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. The condition must be confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

    Limitations of Use

    IMCIVREE is not indicated for the treatment of patients with the following conditions as IMCIVREE would not be expected to be effective:

    • Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign;
    • Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity.

    Important Safety Information

    WARNINGS AND PRECAUTIONS

    Disturbance in Sexual Arousal: Sexual adverse reactions may occur in patients treated with IMCIVREE. Spontaneous penile erections in males and sexual adverse reactions in females occurred in clinical studies with IMCIVREE. Instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.

    Depression and Suicidal Ideation: Some drugs that target the central nervous system, such as IMCIVREE, may cause depression or suicidal ideation. Monitor patients for new onset or worsening of depression. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors.

    Skin Pigmentation and Darkening of Pre-Existing Nevi: IMCIVREE may cause generalized increased skin pigmentation and darkening of pre-existing nevi due to its pharmacologic effect. This effect is reversible upon discontinuation of the drug. Perform a full body skin examination prior to initiation and periodically during treatment with IMCIVREE to monitor pre-existing and new skin pigmentary lesions.

    Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants.

    ADVERSE REACTIONS

    • The most common adverse reactions (incidence ≥23%) were injection site reactions, skin hyperpigmentation, nausea, headache, diarrhea, abdominal pain, back pain, fatigue, vomiting, depression, upper respiratory tract infection, and spontaneous penile erection.

    USE IN SPECIFIC POPULATIONS

    Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus.

    Treatment with IMCIVREE is not recommended for use while breastfeeding.

    To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    See Full Prescribing Information for IMCIVREE.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our business strategy and plans, including regarding commercialization of setmelanotide, and our market opportunity. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881



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  15. -- Indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing --

    -- Approval supports company's approach to address rare genetic diseases of obesity associated with an impaired MC4 receptor pathway --

    -- People living with these rare genetic diseases of obesity struggle with insatiable hunger and excessive weight gain beginning at a young age --

    -- Approval based on data from two 52-week open-label trials that demonstrated clinically meaningful and statistically significant weight loss and reduction of hunger --

    -- Rare Pediatric Disease Priority Review Voucher issued to Rhythm by FDA --

    -- Company to host conference call

    -- Indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing --

    -- Approval supports company's approach to address rare genetic diseases of obesity associated with an impaired MC4 receptor pathway --

    -- People living with these rare genetic diseases of obesity struggle with insatiable hunger and excessive weight gain beginning at a young age --

    -- Approval based on data from two 52-week open-label trials that demonstrated clinically meaningful and statistically significant weight loss and reduction of hunger --

    -- Rare Pediatric Disease Priority Review Voucher issued to Rhythm by FDA --

    -- Company to host conference call today at 9:30 a.m. ET --

    BOSTON, Nov. 27, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. Food & Drug Administration (FDA) has approved IMCIVREE™ (setmelanotide) for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing. With this approval, IMCIVREE becomes the first-ever FDA approved therapy for these rare genetic diseases of obesity.

    "Our first new drug approval is a major milestone for Rhythm, and we look forward to delivering on the promise of IMCIVREE for patients suffering with obesity due to POMC, PCSK1 or LEPR deficiency," said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. "With IMCIVREE, we are advancing a first-in-class, precision medicine that is designed to directly address the underlying cause of obesities driven by genetic deficits in the melanocortin-4 (MC4) receptor pathway."

    Obesity due to POMC, PCSK1 or LEPR deficiency are ultra-rare diseases caused by variants in POMC, PCSK1 or LEPR genes that impair the MC4 receptor pathway, which is a pathway in the hypothalamus that is responsible for regulating hunger, energy expenditure and consequently body weight. People living with obesity due to POMC, PCSK1 or LEPR deficiency struggle with extreme, insatiable hunger beginning at a young age, resulting in early-onset, severe obesity. As an MC4 receptor agonist, IMCIVREE is designed to restore impaired MC4 receptor pathway activity arising due to genetic deficits upstream of the MC4 receptor. There have been no FDA-approved therapies specifically indicated to manage weight in obesity due to POMC, PCSK1 or LEPR deficiency prior to IMCIVREE. Rhythm expects to make IMCIVREE commercially available to patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency in the U.S. in the first quarter of 2021.

    "Many patients and families who live with these diseases face an often burdensome stigma associated with severe obesity. To manage this obesity and control disruptive food-seeking behavior, caregivers often lock cabinets and refrigerators and significantly limit social activities," said Jennifer Miller, M.D., pediatric endocrinologist at University of Florida Health. "This FDA approval marks an important turning point, providing a much needed therapy and supporting the use of genetic testing to identify and properly diagnose patients with these rare genetic diseases of obesity."

    The FDA approval of IMCIVREE is based on results from the largest studies conducted to date in obesity due to POMC, PCSK1 or LEPR deficiency. In Phase 3 clinical trials, 80 percent of patients with obesity due to POMC or PCSK1 deficiency achieved greater than ten percent weight loss and 45.5 percent of patients with obesity due to LEPR deficiency achieved greater than ten percent weight loss after one year of treatment with IMCIVREE.

    Consistent with prior clinical study experience, IMCIVREE was generally well-tolerated in both trials. The most common adverse events were injection site reaction, skin hyperpigmentation, and nausea. Warnings and precautions include disturbance in sexual arousal, depression and suicidal ideation, skin pigmentation and darkening of pre-existing nevi. There may be a risk of serious adverse reactions due to benzyl alcohol preservative in neonates and low birth weight infants. IMCIVREE is not approved for use in neonates or infants.

    "We know that not all obesity is the same, and genetic testing plays a key role in enabling physicians, patients and families to understand the underlying cause of certain severe obesities," said Murray Stewart, M.D., Chief Medical Officer of Rhythm. "In addition to POMC, PCSK1 and LEPR genes, we are continuing our efforts to identify further genes and populations to evaluate the potential for setmelanotide to address the insatiable hunger and early-onset severe obesity that characterize these diseases."

    With this approval, the FDA issued a Rare Pediatric Disease Priority Review Voucher (PRV) to Rhythm. The PRV can be redeemed to receive priority review for any subsequent marketing application or sold or transferred to other companies for their programs. The FDA previously granted Breakthrough Therapy Designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4 receptor pathway, as well as orphan drug designation for obesity due to POMC (including PCSK1) and LEPR deficiencies.

    Rhythm's Marketing Authorization Application (MAA) for setmelanotide to treat people living with obesity due to POMC, PCSK1 or LEPR deficiency is currently under review by the European Medicines Agency (EMA). The EMA has previously granted PRIority MEdicines (PRIME) designation for setmelanotide for the treatment of obesity and the control of hunger associated with deficiency diseases of the MC4 receptor pathway. Additionally, the Company is currently evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl or Alström syndrome with topline data expected late in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm also continues to enroll patients in its Phase 2 Basket Study designed to rapidly facilitate proof-of-concept in new indications.

    Conference Call Information

    Rhythm will host a live webcast beginning at 9:30 a.m. ET today to discuss the FDA approval of IMCIVREE. To access the live call, please dial (844) 498-0570 (domestic) or (409) 983-9726 (international) and refer to conference ID 8468472. A webcast of the conference call will be available under "Events and Presentations" in the Investors & Media section of Rhythm's website at http://ir.rhythmtx.com. The archived webcast will be available on Rhythm's website approximately two hours after the conference call and will be available for 90 days following the call.

    IMCIVREE™ (setmelanotide) Indication

    IMCIVREE is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. The condition must be confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

    Limitations of Use

    IMCIVREE is not indicated for the treatment of patients with the following conditions as IMCIVREE would not be expected to be effective:

    • Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign;
    • Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity.

    Important Safety Information

    WARNINGS AND PRECAUTIONS

    Disturbance in Sexual Arousal: Sexual adverse reactions may occur in patients treated with IMCIVREE. Spontaneous penile erections in males and sexual adverse reactions in females occurred in clinical studies with IMCIVREE. Instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.

    Depression and Suicidal Ideation: Some drugs that target the central nervous system, such as IMCIVREE, may cause depression or suicidal ideation. Monitor patients for new onset or worsening of depression. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors.

    Skin Pigmentation and Darkening of Pre-Existing Nevi: IMCIVREE may cause generalized increased skin pigmentation and darkening of pre-existing nevi due to its pharmacologic effect. This effect is reversible upon discontinuation of the drug. Perform a full body skin examination prior to initiation and periodically during treatment with IMCIVREE to monitor pre-existing and new skin pigmentary lesions.

    Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants.

    ADVERSE REACTIONS

    • The most common adverse reactions (incidence ≥23%) were injection site reactions, skin hyperpigmentation, nausea, headache, diarrhea, abdominal pain, back pain, fatigue, vomiting, depression, upper respiratory tract infection, and spontaneous penile erection.

    USE IN SPECIFIC POPULATIONS

    Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus.

    Treatment with IMCIVREE is not recommended for use while breastfeeding.



    To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    See Full Prescribing Information for IMCIVREE.

    About Rhythm Pharmaceuticals

    Rhythm is a biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic diseases of obesity. The company is advancing setmelanotide, its melanocortin-4 (MC4) receptor agonist, to treat a number of rare genetic diseases of obesity. The Company is leveraging the Rhythm Engine and the largest known obesity DNA database, now with more than 30,0000 sequencing samples from individuals with severe obesity, to improve the understanding, diagnosis and potentially the treatment of rare genetic diseases of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, including the anticipated timing for release of clinical trial data and our expectations surrounding potential regulatory approvals and timing thereof, and our business strategy and plans, including regarding commercialization of setmelanotide. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881

    US-SET-2000083 11/2020

    A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/c3ad0a6f-f4bf-4444-9c6d-be14f11ef6cd



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  16. -- Interim data from Phase 2 study showed that once-weekly formulation of setmelanotide achieved safety and efficacy results comparable to daily-dosing formulation --
    -- Additional data from long-term extension study in POMC deficiency obesity showed durable weight loss and reductions in hunger at up to three years on therapy --
    -- Setmelanotide was generally well tolerated --

    BOSTON, Nov. 04, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced new clinical data for setmelanotide, its investigational melanocortin-4 receptor (MC4R) agonist, being presented at The Obesity…

    -- Interim data from Phase 2 study showed that once-weekly formulation of setmelanotide achieved safety and efficacy results comparable to daily-dosing formulation --

    -- Additional data from long-term extension study in POMC deficiency obesity showed durable weight loss and reductions in hunger at up to three years on therapy --

    -- Setmelanotide was generally well tolerated --

    BOSTON, Nov. 04, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced new clinical data for setmelanotide, its investigational melanocortin-4 receptor (MC4R) agonist, being presented at The Obesity Society's ObesityWeek® 2020, held virtually from November 2-6, 2020.

    "We are excited to share new data from across our setmelanotide development program that support its potential as a new medicine for people with rare genetic disorders of obesity," said Murray Stewart, M.D., Chief Medical Officer of Rhythm Pharmaceuticals. "As we prepare for a potential FDA approval in proopiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesities later this month, we are focused on ongoing efforts to fundamentally alter the treatment paradigm for rare genetic disorders of obesity and continue advancing weekly setmelanotide as a potential option that may be more convenient and less burdensome for patients and their families."

    ObesityWeek Presentations

    A Randomized Trial of a Once-Weekly Formulation of Setmelanotide in Individuals with Obesity,' an oral presentation by Gregory Gordon, MD, JD, Vice President, Clinical at Rhythm

    Rhythm is presenting interim results from its Phase 2 study evaluating a once-weekly formulation of setmelanotide in healthy obese volunteers, including new data from a larger number of individuals, which come in addition to data announced in June 2020. The data show that healthy obese people treated with the weekly formulation of setmelanotide achieved comparable weight loss to those treated with the daily formulation and that both weekly and daily formulations of setmelanotide were observed to be generally well tolerated.

    A total of 85 individuals were included in the full data analysis: 28 individuals were treated with weekly setmelanotide without titration for 12 weeks (10mg, 20mg or 30mg doses); 20 individuals were treated with weekly setmelanotide with titration (10mg for one week, followed by 20mg for 11 weeks or 20mg for one week, followed by 30mg for 11 weeks); 13 individuals were treated with daily setmelanotide (2mg daily for one week, followed by 3mg daily for 11 weeks); and 24 individuals were treated with placebo for 12 weeks.

    As of the data cutoff of April 17, 2020, weekly setmelanotide administration was generally well tolerated, with no serious treatment-emergent adverse events (TEAE), and the safety results were similar to the daily administration and consistent with prior clinical experience. The most commonly reported TEAEs, rates of which were generally similar between individuals treated with the weekly and daily formulations, included injection site reaction, hyperpigmentation, nausea, headache and vomiting.

    Healthy obese people treated with the weekly formulation of setmelanotide achieved comparable changes in weight and hunger scores as those treated with the daily formulation:

     10mg QW20mg QW30mg QW10mg/20mg QW20mg/30mg QW2mg/3mg QDPlacebo
    Weight Change from Baseline at Week 12 (kg)-2.6-3.3-3.0-1.1-3.6-2.10.5
    Absolute Change in Most Hunger Score at Week 121-2.1-1.60.3-1.9-3.9-2.3-0.3

    Data presented as of a cutoff of April 17, 2020

    "These data demonstrate that the weight and hunger score changes with the weekly formulation were generally comparable to the daily formulation," said Dr. Gordon. "It is worth noting that the changes with both formulations in normal obese individuals were smaller relative to data we've reported separately in patients with rare genetic obesities associated with an impaired MC4R pathway. This reinforces the important role the MC4R pathway plays in regulating hunger, caloric intake, and energy expenditure and the potential for a precision medicine treatment approach in certain patients with severe obesity."

    Additionally, as previously disclosed, pharmacokinetic (PK) analyses showed similar trough drug concentrations for the daily and weekly formulations over the duration of therapy. The weekly formulation of setmelanotide demonstrated a consistent 24-hour PK range and was detected steadily over one week, with a trough concentration consistent with the trough concentration of the efficacious daily formulation.

    ‘Long-term Weight and Hunger Reduction With Setmelanotide in Individuals With POMC Deficiency Obesity,' a poster presentation by Karine Clément, MD, PhD, and Professor of Nutrition at Pitié-Salpêtrière Hospital and Sorbonne Université in Paris

    Dr. Clément is presenting updated data from the long-term extension trial evaluating setmelanotide in POMC deficiency obesity. The long-term extension trial enrolled nine individuals, all of whom previously completed Rhythm's pivotal Phase 3 clinical trial evaluating setmelanotide for the treatment of severe obesity and insatiable hunger.

    Efficacy and safety data from five individuals who reached 89 weeks on setmelanotide therapy during the long-term extension trial are being presented. As of a cutoff of April 16, 2020:

    • The mean percent reduction in body weight from pivotal trial baseline at week 89 of the extension was -30.2%, a-0.1% change from the conclusion of the pivotal trial;
    • The mean absolute reduction in body weight at week 89 of the extension was -40.2kg, a change of -0.5kg from the conclusion of the pivotal trial;
    • The mean percent reduction in body mass index at week 89 of the extension was -32.5%, a change of -0.4% from the conclusion of the pivotal trial;
    • The mean percent change in most hunger score from pivotal baseline was consistent through week 89 of the extension at -8.2%, a change of 10% from the conclusion of the pivotal trial.

    As previously reported, data showed that patients successfully maintained durable weight loss and stable hunger scores with long-term treatment with setmelanotide, for up to three years. Consistent with prior clinical experience, setmelanotide has been generally well-tolerated in the long-term extension trial and the safety results remained consistent across all patients treated for up to three years. The most common treatment-emergent adverse events included injection site reactions, nausea and vomiting, and hyperpigmentation.

    ‘Suicidality and Depression in Individuals With Genetic Obesity Treated With Setmelanotide,' a poster presentation by Peter Kühnen, MD, Institute for Experimental Pediatric Endocrinology, Charité Universitätsmedizin in Berlin

    Dr. Kühnen is presenting new data from Rhythm's pivotal Phase 3 clinical trials evaluating setmelanotide in POMC and LEPR deficiency obesities, which showed that treatment with setmelanotide was generally well-tolerated and did not induce a significant effect on depression or suicidality. Because of their severe obesity, individuals with POMC or LEPR deficiency may be at a higher risk for experiencing depression and/or suicidal ideation.

    About Setmelanotide

    Setmelanotide is an investigational MC4R agonist. The MC4R is part of the key biological pathway that independently regulates hunger, caloric intake, and energy expenditure. Variants in genes may impair the function of the MC4R pathway, potentially leading to hyperphagia and early-onset, severe obesity. Rhythm is currently developing setmelanotide as a targeted therapy to potentially restore the function of an impaired MC4R pathway and, in so doing, potentially reduce hunger and weight in patients with rare genetic disorders of obesity. Currently, no pharmacologic therapies exist to treat these conditions. The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4R pathway, which includes POMC deficiency obesity, LEPR deficiency obesity, Bardet-Biedl Syndrome and Alström Syndrome. The European Medicines Agency (EMA) has also granted PRIority MEdicines (PRIME) designation for setmelanotide for the treatment of obesity and the control of hunger associated with deficiency disorders of the MC4R pathway. Both the FDA and EMA have granted orphan drug status to setmelanotide for POMC and LEPR deficiency obesities.

    About Rhythm Pharmaceuticals

    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. The FDA has accepted for filing an NDA for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity with Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020. Rhythm also submitted a Marketing Authorization Application (MAA) for setmelanotide to treat individuals living with POMC deficiency obesity or LEPR deficiency obesity to the European Medicines Agency (EMA) in June 2020. Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, expectations regarding regulatory approval, the anticipated timing for release of clinical trial data, our ongoing efforts related to patient identification and genetic sequencing and timing thereof, and our participation in upcoming events and presentations. Statements using word such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881


    1 Score is based on 0-10 Likert scale from question, "In the last 24 hours, how hungry did you feel when you were the most hungry?," with 0 being not hungry at all and 10 being the hungriest possible.

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  17. -- NDA for setmelanotide for POMC and LEPR deficiency obesities under review, with PDUFA goal date of November 27, 2020 -- 
    -- Appointed Jennifer Chien and Yann Mazabraud to co-lead global integrated commercial strategies --
    -- On track to announce topline data from pivotal Phase 3 trial in Bardet-Biedl and Alström syndromes late in fourth quarter of 2020 or early in first quarter 2021 --
    -- Plan to announce data from multiple cohorts in ongoing Phase 2 basket study early in first quarter 2021 --

    BOSTON, Nov. 02, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today reported financial…

    -- NDA for setmelanotide for POMC and LEPR deficiency obesities under review, with PDUFA goal date of November 27, 2020 -- 

    -- Appointed Jennifer Chien and Yann Mazabraud to co-lead global integrated commercial strategies --

    -- On track to announce topline data from pivotal Phase 3 trial in Bardet-Biedl and Alström syndromes late in fourth quarter of 2020 or early in first quarter 2021 --

    -- Plan to announce data from multiple cohorts in ongoing Phase 2 basket study early in first quarter 2021 --

    BOSTON, Nov. 02, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today reported financial results and provided a business update for the third quarter ended September 30, 2020.

    "At Rhythm, we are making meaningful progress toward our goal of changing how the world thinks about obesity by focusing attention on the critical role that genetics can play in driving disease," said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. "We believe we are now on the cusp of a significant and validating inflection point with the potential approval of setmelanotide as the first-ever FDA-approved therapy for rare genetic disorders of obesity expected later this month. In parallel, we are looking forward to key readouts across our next wave of indications, with topline results from our pivotal Phase 3 trial in Bardet-Biedl and Alström syndromes expected late in the fourth quarter of 2020 or early in the first quarter 2021, and data from our Basket Study now expected early in the first quarter of 2021. We are hopeful that these data will further demonstrate setmelanotide's potential to treat the severe obesity and insatiable hunger that characterize MC4R pathway-driven rare genetic disorders of obesity and enable better care for the patients and families affected by these conditions."

    Recent Highlights:

    • In October, the Company announced that results from two pivotal Phase 3 studies evaluating setmelanotide in pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity were published in The Lancet Diabetes & Endocrinology. Rhythm believes these data validate setmelanotide's potential to be the first approved therapy for people living with POMC or LEPR deficiency obesities.
    • Rhythm recently announced the addition of two senior biopharmaceutical executives to its leadership team to lead global integrated commercial strategies in key markets. In October, the Company announced the appointment of Jennifer Chien as Executive Vice President, Head of North America, effective Nov. 9, and in September, the Company announced the appointment of Yann Mazabraud as Executive Vice President, Head of International.

    Upcoming Regulatory Milestones:

    • The Prescription Drug User Fee Act (PDUFA) target date is November 27, 2020, which is when the U.S. Food and Drug Administration (FDA) is scheduled to act on the Company's New Drug Application (NDA) for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity. As previously announced, the FDA granted rare pediatric disease designations for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity, which means Rhythm would be eligible to receive one priority review voucher, pending approval.
    • Rhythm's Marketing Authorization Application (MAA) for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity is currently under review by the European Medicines Agency (EMA). As is frequently the case with filings that initially receive accelerated assessment by the EMA, Rhythm's MAA recently has reverted from an accelerated assessment to a standard review.

    Upcoming Clinical Milestones:

    • Rhythm will present three abstracts at The Obesity Society's ObesityWeek® 2020, being held virtually from Nov. 2-6. Presentations include full data from the Phase 2 trial of a once-weekly formulation of setmelanotide, which will be presented in an oral presentation at 9:45 a.m. ET on Thursday, Nov. 5, as well as updated data from the long-term extension study of setmelanotide in POMC deficiency obesity and new data showing there is no treatment-related effect of setmelanotide on depression or suicidality, both of which will be presented in poster sessions, beginning at 12 noon ET on Nov. 3.
    • Rhythm expects to report topline data from its combined pivotal Phase 3 trial evaluating setmelanotide in BBS and Alström syndrome late in the fourth quarter of 2020 or early in the first quarter of 2021.
    • Rhythm now plans to provide an update on its ongoing exploratory Phase 2 Basket Study and genetic sequencing efforts early in the first quarter of 2021. This update will include new data from individuals living with HET obesity due to a loss-of-function variant in one of two alleles on the POMC, PCSK1 or LEPR gene, as well as SRC1 and SH2B1 deficiency obesities. The update will also include data from Company's sequencing efforts, which now includes samples from more than 30,000 individuals with severe obesity.
    • Rhythm currently is evaluating next steps for the pre-clinical development of RM-853, its ghrelin o-acyltransferase (GOAT) inhibitor.

    Third Quarter 2020 Financial Results:

    • Cash Position: As of September 30, 2020, cash, cash equivalents and short-term investments were $201.8 million, as compared to $228.6 million as of June 30, 2020. This decrease reflects $27.0 million of cash used to fund operating activities in the third quarter 2020. Based on its current clinical development plans, Rhythm expects that its existing cash, cash equivalents and short-term investments will enable it to fund its operations at least through the end of 2021.
    • R&D Expenses: R&D expenses were $23.0 million for the third quarter of 2020 as compared to $26.6 million for the third quarter of 2019. The decrease in R&D spending was primarily attributed to a $5.2 million reduction in clinical trial expenses associated with the Company's Go-ID genotyping study and the once-weekly formulation study and a decrease of $3.2 million related to translational research and genetic sequencing efforts. These decreases were partially offset by an increase of $3.6 million related to purchases of setmelanotide API and an increase of $1.0 million related to a milestone payment associated with the license agreement with Ipsen on filing the MAA for setmelanotide for the treatment of POMC and LEPR deficiency obesities.
    • S,G&A Expenses: S,G&A expenses were $11.3 million for the third quarter of 2020 as compared to $10.5 million for the third quarter of 2019. This increase was primarily due to an increase of $1.3 million in stock compensation expense associated with a grant to the Company's new chief executive officer and other new hire grants during the period as well as a modification of stock options for the Company's former chief commercial officer, partially offset by a decrease of $0.8 million in consulting services.
    • Net Loss: Net loss was $33.8 million for the third quarter of 2020, or a net loss per basic and diluted share of $0.77, as compared to a net loss of $36.0 million for the third quarter of 2019, or a net loss per basic and diluted share of $1.04.

    Year to Date Financial Results:

    • Cash Position: As of September 30, 2020, cash, cash equivalents and short-term investments were $201.8 million, as compared to $292.5 million as of December 31, 2019. This decrease reflects $92.9 million of cash used to fund operating expenses in 2020.
    • R&D Expenses: R&D expenses were $68.5 million for the nine months ended September 30, 2020, as compared to $84.6 million for the nine months ended September 30, 2019. The decrease was primarily due to a decrease of $16.0 million related to the GO-ID genotyping study, the POMC and LEPR clinical studies and the once-weekly formulation study, and a decrease of $7.5 million related to translational research, pathway validation and genetic sequencing efforts. These decreases were partially offset by an increase of $3.0 million related to a milestone expenses associated with the license agreement with Ipsen on filing the NDA with the FDA and the MAA with the EMA for setmelanotide for the treatment of POMC and LEPR deficiency obesities, an increase of $2.5 million in spending related to the Phase 2 Basket Study, and an increase of $1.2 million related to purchases of setmelanotide API.
    • S,G&A Expenses: S,G&A expenses were $33.0 million for the nine months ended September 30, 2020, as compared to $27.1 million for the nine months ended September 30, 2019. The increase was primarily due to an accounting charge of $4.0 million related to the separation agreement and modification of stock options for the Company's former chief executive officer and chief commercial officer, and an increase of $1.0 million in various consulting and professional services related to legal and IT support costs.
    • Net Loss: Net loss was $99.1 million for the nine months ended September 30, 2020, or a net loss per basic and diluted share of $2.25, as compared to a net loss of $107.8 million for the nine months ended September 30, 2019, or a net loss per basic and diluted share of $3.13.

    About Rhythm Pharmaceuticals

    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. The FDA has accepted for filing an NDA for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity with Priority Review and assigned a PDUFA goal date of November 27, 2020. Rhythm also submitted an MAA for setmelanotide to treat individuals living with POMC deficiency obesity or LEPR deficiency obesity to the EMA in June 2020. Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected late in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, including the anticipated timing for release of clinical trial data and our expectations surrounding potential regulatory approvals and timing thereof, our business strategy and plans, including regarding commercialization of setmelanotide, management changes, our participation in upcoming events and presentations, and the sufficiency of our cash, cash equivalents and short-term investments to fund our operations. Statements using word such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements.  Such statements are subject to numerous risks and uncertainties, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881

     
    Rhythm Pharmaceuticals, Inc.

    Condensed Consolidated Statements of Operations and Comprehensive Loss

    (in thousands, except share and per share data)

    (Unaudited)
        
     Three months ended September 30,  Nine months ended September 30, 
     2020

     2019

     2020

     2019

    Operating expenses:               
    Research and development$22,995  $26,572  $68,496  $84,641 
    Selling, general, and administrative 11,289   10,535   33,006   27,135 
    Total operating expenses 34,284   37,107   101,502   111,776 
    Loss from operations (34,284)  (37,107)  (101,502)  (111,776)
    Other income (expense):           
    Interest income, net 466   1,104   2,403   4,003 
    Total other income, net 466   1,104   2,403   4,003 
    Net loss$(33,818) $(36,003) $(99,099) $(107,773)
    Net loss per share, basic and diluted$(0.77) $(1.04) $(2.25) $(3.13)
    Weighted-average common shares outstanding, basic and diluted 44,142,334   34,541,765   44,097,178   34,470,995 
                
    Other comprehensive loss:           
    Net loss$(33,818) $(36,003) $(99,099) $(107,773)
    Unrealized (loss) gain on marketable securities (392)     238    
    Comprehensive loss$(34,210) $(36,003) $(98,861) $(107,773)
                    



     
    Rhythm Pharmaceuticals, Inc.

    Condensed Consolidated Balance Sheets

    (in thousands, except share data)

    (Unaudited)
        
     September 30,  December 31, 
     2020

     2019

          
    Assets     
    Current assets:     
    Cash and cash equivalents$67,670  $62,294 
    Short-term investments 134,114   230,165 
          
    Prepaid expenses and other current assets 8,130   9,945 
    Total current assets 209,914   302,404 
    Property and equipment, net 3,289   3,671 
    Right-of-use asset 1,871   2,045 
    Restricted cash 403   403 
    Total assets$215,477  $308,523 
    Liabilities and stockholders' equity     
    Current liabilities:     
    Accounts payable$3,793  $10,415 
    Accrued expenses and other current liabilities 11,536   13,530 
    Lease liability 519   472 
    Total current liabilities 15,848   24,417 
    Long-term liabilities:     
    Lease liability 2,692   3,086 
    Total liabilities 18,540   27,503 
    Commitments and contingencies     
    Stockholders' equity:     
    Preferred Stock, $0.001 par value: 10,000,000 shares authorized; no shares issued and outstanding at September 30, 2020 and December 31, 2019     
    Common stock, $0.001 par value: 120,000,000 shares authorized; 44,204,745 and 43,996,753 shares issued and outstanding at September 30, 2020 and December 31, 2019, respectively 44   44 
    Additional paid-in capital 621,085   606,307 
    Accumulated other comprehensive income 238    
    Accumulated deficit (424,430)  (325,331)
    Total stockholders' equity 196,937   281,020 
    Total liabilities and stockholders' equity$215,477  $308,523 
            

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  18. BOSTON, Oct. 30, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, announced today that results from two pivotal Phase 3 studies evaluating setmelanotide in proopiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity were published in The Lancet Diabetes & Endocrinology. As previously reported, data from the studies demonstrate that treatment with setmelanotide, the company's melanocortin-4 receptor (MC4R) agonist, led to statistically significant and clinically meaningful reductions of weight and hunger.

    "Results from Rhythm's pivotal Phase 3 studies…

    BOSTON, Oct. 30, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, announced today that results from two pivotal Phase 3 studies evaluating setmelanotide in proopiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity were published in The Lancet Diabetes & Endocrinology. As previously reported, data from the studies demonstrate that treatment with setmelanotide, the company's melanocortin-4 receptor (MC4R) agonist, led to statistically significant and clinically meaningful reductions of weight and hunger.

    "Results from Rhythm's pivotal Phase 3 studies, which are the largest studies to date in POMC and LEPR deficiency obesities, provide evidence regarding the safety and efficacy of setmelanotide and we believe they validate its potential long-term use as a novel treatment for severe obesity and hyperphagia," said co-author Peter Kühnen, M.D., Institute for Experimental Pediatric Endocrinology, Charité Universitätsmedizin Berlin, Germany. "It is important to recognize the signs of these rare genetic disorders because we may soon have a targeted treatment option available for the first time for obesity disorders caused by impairments of the MC4R pathway."

    Rhythm initially reported positive topline data from the Phase 3 studies in August 2019 and subsequently presented updated data in a late-breaking research forum during the 37th Annual Meeting of The Obesity Society at ObesityWeek® 2019.

    Eight of 10 participants with POMC deficiency obesity (80%; P<0.0001 compared with historical data) and five of 11 participants with LEPR deficiency obesity (45%; P=0.0001 compared with historical data) achieved at least 10 percent weight loss at approximately one year. The mean percent change in "most hunger" score in participants aged 12 years and older was -27.1 percent (n=7; P=0.0005) in POMC deficiency obesity and -43.7 percent (n=7; P<0.0001) in LEPR deficiency obesity. Consistent with prior clinical experience, setmelanotide was generally well-tolerated in both trials. The most common adverse events were injection site reaction, skin hyperpigmentation, and nausea.

    "These results are significant because, as we know from natural history data, individuals living with POMC or LEPR deficiency obesity consistently experience substantial weight gain each year beginning in early childhood, and we would not expect any of these patients to be able to achieve 10 percent weight loss over the course of a year without continued treatment," said co-author Karine Clément, professor of nutrition at Pitié-Salpêtrière hospital and Sorbonne University in Paris. "These data and the significant unmet need to address the obesity and hyperphagia caused by rare genetic disorders of obesity underscore the importance of testing for genetic variants that may impair MC4R activation and lead to severe obesity."

    In May 2020, Rhythm announced that the U.S. Food and Drug Administration (FDA) accepted the company's New Drug Application (NDA) for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity, granted Priority Review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020. In July 2020, the Company announced the submission of its Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the treatment of POMC deficiency obesity and LEPR deficiency obesity.

    "We are grateful to the authors and the investigators involved in our pivotal Phase 3 clinical trials for their continued partnership in advancing setmelanotide to address significant unmet needs facing people with rare genetic disorders of obesity," Murray Stewart, M.D., Chief Medical Officer of Rhythm, said.

    The article is available online here: http://www.thelancet.com/journals/landia/article/PIIS2213-8587(20)30364-8/fulltext.To request a copy of the article, email .

    About Setmelanotide

    Setmelanotide is an investigational, melanocortin-4 receptor (MC4R) agonist. The MC4R is part of the key biological pathway that independently regulates hunger, caloric intake, and energy expenditure. Variants in genes may impair the function of the MC4R pathway, potentially leading to hyperphagia and early-onset, severe obesity. Rhythm is currently developing setmelanotide as a targeted therapy to potentially restore the function of an impaired MC4R pathway and, in so doing, potentially reduce hunger and weight in patients with rare genetic disorders of obesity. Currently, no pharmacologic therapies exist to treat these conditions.

    The FDA has granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4R pathway, which includes POMC deficiency obesity, LEPR deficiency obesity, Bardet-Biedl Syndrome (BBS) and Alström syndrome. The EMA has also granted PRIority MEdicines (PRIME) designation for setmelanotide for the treatment of obesity and the control of hunger associated with deficiency disorders of the MC4R pathway. Both the FDA and EMA have granted orphan drug status to setmelanotide for POMC and LEPR deficiency obesities. Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with BBS and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021.

    About Rhythm Pharmaceuticals

    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our business strategy and plans, including regarding commercialization of setmelanotide; the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, including anticipated timing of data readouts and our expectations surrounding potential regulatory approvals and timing thereof. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881

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  19. BOSTON, Oct. 28, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that Jennifer Chien has been appointed as Executive Vice President, Head of North America, effective November 9, 2020. As EVP North America, Ms. Chien will co-lead global integrated commercial strategies in close collaboration with Yann Mazabraud, who was recently appointed EVP, Head of International.

    "Jennifer joins Rhythm at a critical inflection point as we prepare to bring setmelanotide, potentially the first approved therapy for rare genetic disorders of obesity, to patients throughout the world…

    BOSTON, Oct. 28, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that Jennifer Chien has been appointed as Executive Vice President, Head of North America, effective November 9, 2020. As EVP North America, Ms. Chien will co-lead global integrated commercial strategies in close collaboration with Yann Mazabraud, who was recently appointed EVP, Head of International.

    "Jennifer joins Rhythm at a critical inflection point as we prepare to bring setmelanotide, potentially the first approved therapy for rare genetic disorders of obesity, to patients throughout the world," said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. "With her passion for science and patients, as well as her deep experience in rare disease, Jennifer will play a crucial role in driving patient identification and delivering setmelanotide to individuals who might benefit from therapy. Jennifer understands the challenges patients, families, health care providers and the community as a whole face in trying to get the appropriate care for their devastating rare disease. We are excited to welcome her to Rhythm."

    Ms. Chien brings more than 20 years of experience in rare diseases. Most recently, she was the Chief Commercial Officer at Krystal Biotech. Prior to that, she served as Vice President, Head of Genetic Diseases at Sanofi Genzyme, where she was responsible for the U.S. commercial strategy and implementation for seven brands and launch preparation within rare diseases. During her time at Sanofi Genzyme, Ms. Chien also held leadership roles as Vice President, Head of Global Nephrological Diseases, Senior Director of Global Marketing in Fabry disease, Senior Director of Global Market Access and International Marketing for the cardiovascular business, and Director of Business Development. Ms. Chien holds a bachelor's of science degree from Massachusetts Institute of Technology and a master's in public health from Harvard University.

    "This is an exciting time to join Rhythm," said Ms. Chien. "With setmelanotide nearing its first potential approvals in the United States and Europe, I look forward to working with the Rhythm team to transform the care of individuals living with rare genetic disorders of obesity and bring them a much needed therapy."

    About Rhythm Pharmaceuticals

    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. The Company is developing setmelanotide, its investigational, melanocortin-4 receptor (MC4R) agonist, for the treatment of severe obesity and hyperphagia associated with rare genetic disorders of obesity. The U.S. Food and Drug Administration (FDA) has accepted for filing Rhythm's New Drug Application (NDA) for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity with Priority Review and a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020. Rhythm also submitted a Marketing Authorization Application (MAA) for setmelanotide to treat individuals living with POMC deficiency obesity or LEPR deficiency obesity to the European Medicines Agency (EMA) in June 2020. Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our business strategy and plans, including regarding commercialization of setmelanotide; the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, including anticipated timing of data readouts and our expectations surrounding potential regulatory approvals and timing thereof; and management changes. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881

    A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/917fe36f-5096-40b5-8c0d-de8b14bac65e.

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  20. – Company announces organizational changes designed to accelerate global strategy –

    BOSTON, Sept. 14, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today appointed Yann Mazabraud as the Company's Executive Vice President, Head of International, effective October 5, 2020. As a member of the Company's management team, Mr. Mazabraud will lead Rhythm's international operations.

    "Rhythm is building a global, integrated organization to advance setmelanotide, potentially the first approved therapy to treat individuals living with severe obesity and hyperphagia associated with rare…

    – Company announces organizational changes designed to accelerate global strategy –

    BOSTON, Sept. 14, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today appointed Yann Mazabraud as the Company's Executive Vice President, Head of International, effective October 5, 2020. As a member of the Company's management team, Mr. Mazabraud will lead Rhythm's international operations.

    "Rhythm is building a global, integrated organization to advance setmelanotide, potentially the first approved therapy to treat individuals living with severe obesity and hyperphagia associated with rare genetic disorders of obesity," said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. "Yann's appointment to this newly-created role reflects our commitment to delivering setmelanotide to individuals worldwide, and we are thrilled to have him join our team as we accelerate our strategic focus globally. Yann brings more than two decades of experience leading rare diseases commercial strategy, marketing and market access, which will prove invaluable as we continue fostering a community of health care providers, patients and families."

    With more than 20 years in the biopharmaceutical industry leading global commercial and operations teams, Mr. Mazabraud joins Rhythm from Trevi Therapeutics, where he served as Chief Commercial Officer and Head of International for the last two years. Prior to that, he held several leadership positions at Sanofi Genzyme, including, Head of Latin America, U.S. General Manager and North America Head, Rare Diseases. He also served as a member of the Sanofi Genzyme Executive Leadership Team. Mr. Mazabraud holds a master's degree in management from Ecole Supérieure de Commerce de La Rochelle.

    "I am very excited to join Rhythm at this important moment for the company," said Mr. Mazabraud. "With compelling clinical data for setmelanotide in a range of rare genetic disorders of obesity, and advanced trials ongoing in additional indications, now is the time to expand and accelerate plans to deliver setmelanotide globally. I look forward to working with the Rhythm team toward the goal of transforming the care of people living with rare genetic disorders of obesity."

    In conjunction with this change, Rhythm announced that Nithya Desikan, its Chief Commercial Officer, is leaving the Company to explore other opportunities. Rhythm is conducting a search for a Head of North American Operations, who will serve alongside Mr. Mazabraud. Both roles will report to Dr. Meeker.

    Dr. Meeker commented, "We are deeply grateful to Nithya for her many contributions to Rhythm. Under her leadership, Rhythm has grown tremendously and developed strong relationships with health care providers, advocacy groups and families, and upheld a commitment to listening to the patient community to help bolster both individual and collective understandings of rare genetic disorders of obesity. She has built the foundation of a robust, scalable commercial organization to support the potential launch of setmelanotide, and we wish her the best in her future endeavors."

    About Rhythm Pharmaceuticals

    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. The Company is developing setmelanotide, its investigational, melanocortin-4 receptor (MC4R) agonist, for the treatment of severe obesity and hyperphagia associated with rare genetic disorders of obesity. The U.S. Food and Drug Administration (FDA) has accepted for filing Rhythm's New Drug Application (NDA) for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity with Priority Review and a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020. Rhythm also submitted a Marketing Authorization Application (MAA) for setmelanotide to treat individuals living with POMC deficiency obesity or LEPR deficiency obesity to the European Medicines Agency (EMA) in June 2020. Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our business strategy and plans, including regarding commercialization of setmelanotide; the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, including anticipated timing of data readouts and our expectations surrounding potential regulatory approvals and timing thereof; and management changes. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881

    A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/e9c7c83a-19b7-4cf3-bd14-15d7391453b3

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  21. BOSTON, Sept. 09, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that company management will present at two investor conferences in September:

    Morgan Stanley 18th Annual Global Healthcare Conference on Wednesday, September 16, 2020.

    • David Meeker, M.D., Chair, President and Chief Executive Officer, will participate in a fireside chat at 3:30 p.m. ET.

    Cantor Virtual Global Healthcare Conference on Thursday, September 17, 2020.

    • Hunter Smith, Chief Financial Officer, will participate in a fireside chat at 2:00 p.m. ET.

    Live audio webcasts of both presentations will…

    BOSTON, Sept. 09, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that company management will present at two investor conferences in September:

    Morgan Stanley 18th Annual Global Healthcare Conference on Wednesday, September 16, 2020.

    • David Meeker, M.D., Chair, President and Chief Executive Officer, will participate in a fireside chat at 3:30 p.m. ET.

    Cantor Virtual Global Healthcare Conference on Thursday, September 17, 2020.

    • Hunter Smith, Chief Financial Officer, will participate in a fireside chat at 2:00 p.m. ET.

    Live audio webcasts of both presentations will be available under "Events & Presentations" in the Investor Relations section of the Company's website at www.rhythmtx.com. A replay of the webcasts will be available on the Rhythm website for 30 days following each presentation.

    About Rhythm Pharmaceuticals

    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. The Company is developing setmelanotide, its investigational, melanocortin-4 receptor (MC4R) agonist, for the treatment of severe obesity and hyperphagia associated with rare genetic disorders of obesity. The U.S. Food and Drug Administration (FDA) has accepted for filing Rhythm's New Drug Application (NDA) for setmelanotide for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity with Priority Review and a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020. Rhythm also submitted a Marketing Authorization Application (MAA) for setmelanotide to treat individuals living with POMC deficiency obesity or LEPR deficiency obesity to the European Medicines Agency (EMA) in June 2020. Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. The Company is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, expectations regarding regulatory approval, the anticipated timing for release of clinical trial data, and participation in upcoming presentations and conferences. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements.  These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881

    Primary Logo

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  22. BOSTON, Aug. 06, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm, and Hunter Smith, Chief Financial Officer, will present a corporate overview at the Canaccord Genuity 40th Annual Growth Conference on Thursday, August 13, 2020 at 9:30 a.m. ET.

    A live audio webcast of the presentation will be available under "Events & Presentations" in the Investor Relations section of the Company's website at www.rhythmtx.com. A replay of the webcast will be available on the Rhythm website for 30 days…

    BOSTON, Aug. 06, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm, and Hunter Smith, Chief Financial Officer, will present a corporate overview at the Canaccord Genuity 40th Annual Growth Conference on Thursday, August 13, 2020 at 9:30 a.m. ET.

    A live audio webcast of the presentation will be available under "Events & Presentations" in the Investor Relations section of the Company's website at www.rhythmtx.com. A replay of the webcast will be available on the Rhythm website for 30 days following each presentation.

    About Rhythm Pharmaceuticals

    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. The Company is developing setmelanotide, its investigational, melanocortin-4 receptor (MC4R) agonist, for the treatment of severe obesity and hyperphagia associated with rare genetic disorders of obesity. The U.S. Food and Drug Administration (FDA) has accepted for filing Rhythm's New Drug Application (NDA) for setmelanotide for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity with Priority Review and a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020. Rhythm also submitted a Marketing Authorization Application (MAA) for setmelanotide to treat individuals living with POMC deficiency obesity or LEPR deficiency obesity to the European Medicines Agency (EMA) in June 2020. Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, expectations regarding regulatory approval, the anticipated timing for release of clinical trial data, and participation in upcoming presentations and conferences. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements.  These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881

    Primary Logo

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  23. -- Appointed David Meeker, M.D., as President and Chief Executive Officer –

    -- FDA granted rare pediatric disease designations for setmelanotide for the treatment of POMC and LEPR deficiency obesities --

    -- Submitted MAA to EMA for setmelanotide in POMC and LEPR deficiency obesities --

    -- Received FDA acceptance of NDA for setmelanotide for POMC and LEPR deficiency obesities for filing; assigned PDUFA goal date of November 27, 2020 –

    -- On track to announce topline data from pivotal Phase 3 trial of setmelanotide in BBS and Alström syndrome in fourth quarter of 2020 or early in the first quarter of 2021 --

    BOSTON, Aug. 03, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today reported financial results and provided a business update for the second quarter ended June 30, 2020.

    "Rhythm has made tremendous regulatory and clinical progress on its path to bring the first approved therapy to individuals living with rare genetic disorders of obesity," said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. "The potential approval of setmelanotide for the treatment of pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesities will validate the melanocortin-4 receptor (MC4R) pathway as an important therapeutic target, and we look forward to furthering our ongoing efforts, with pivotal data from our Phase 3 clinical trial in Bardet-Biedl syndrome (BBS) and Alström syndromes expected in the fourth quarter or early in 2021 and the continued expansion of our ongoing Phase 2 Basket study."

    Dr. Meeker continued, "We are energized by the opportunity to help individuals living with rare genetic disorders of obesity, who currently have no meaningful treatment options for their severe obesity or insatiable hunger. Looking ahead, we are eager to complete our transformation into an integrated, patient-focused organization while continuing to advance our community building and patient engagement efforts."

    Second Quarter 2020 and Recent Business Highlights:

    Pipeline and Recent Developments:

    POMC and LEPR Deficiency Obesities

    • In July, Rhythm announced the submission of its Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity. In conjunction with this submission, Rhythm announced additional positive data from eight supplemental patients, including four pediatric patients, enrolled in its two pivotal Phase 3 clinical trials for POMC and LEPR deficiency obesities, as well as updated data from its long-term extension study of setmelanotide in patients with POMC or LEPR deficiency obesity. Rhythm included these data in its MAA submission package to the EMA.
      • As announced in July, all eight supplemental patients achieved the primary endpoint of 10 percent or greater weight loss at 52 weeks on setmelanotide therapy, as calculated under the same statistical analysis plan used in the pivotal trials.
      • Setmelanotide was well-tolerated in the long-term extension study, with continued clinical benefit and durable weight loss observed in patients at up to three years on therapy.
    • In July, Rhythm announced that the U.S. Food and Drug Administration (FDA) granted rare pediatric disease designations for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity. Subject to FDA approval, Rhythm would be eligible to receive one priority review voucher, which could then be redeemed to receive priority review for any subsequent marketing application or sold or transferred to another company.
    • In May, Rhythm announced that the FDA accepted its NDA for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity. The FDA granted Priority Review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020.

    Additional Development Pipeline Updates

    • In June, Rhythm announced positive results from a Phase 2 study evaluating a once-weekly formulation of setmelanotide in healthy obese volunteers. Healthy obese people treated with the weekly formulation of setmelanotide achieved comparable weight loss to those treated with the daily formulation, and both weekly and daily formulations of setmelanotide were observed to be safe and well-tolerated. PK analyses showed similar trough drug concentrations for the daily and weekly formulations over the duration of therapy. Rhythm plans to discuss next steps towards registration with the FDA.
    • In July, Rhythm announced the publication of results from its Phase 2 study evaluating setmelanotide in people living with BBS in the peer-reviewed journal Diabetes, Obesity and Metabolism. As previously reported, data from the study demonstrated that treatment with setmelanotide reduced body weight and hunger in individuals with BBS.
    • Rhythm today announced that it is re-evaluating potential indications for RM-853, its ghrelin o-acyltransferase (GOAT) inhibitor, and that the Company will provide an update in the fourth quarter of 2020.             

    Corporate:

    • In July, Rhythm announced the appointment of David Meeker, M.D., the Chair of Rhythm's Board of Directors, as the President and Chief Executive Officer of the Company.
    • In July, Rhythm announced the appointment of Joseph Shulman as the Company's Senior Vice President of Technical Operations.

    Upcoming Milestones:

    • Rhythm is on track to report topline data from its combined pivotal Phase 3 trial evaluating setmelanotide in BBS and Alström syndrome in the fourth quarter of 2020 or early in the first quarter of 2021.
    • Rhythm expects to announce additional data in 2020 from its ongoing Phase 2 Basket Study of setmelanotide in high-impact heterozygous (HET) obesity and additional data from one or more of the other disorders being studied in its Phase 2 Basket Study.
    • Rhythm expects to provide an update on its genetic sequencing efforts in 2020.

    Second Quarter 2020 Financial Results:

    • Cash Position: As of June 30, 2020, cash, cash equivalents and short-term investments were $228.6 million, as compared to $292.5 million as of December 31, 2019. This decrease reflects $63.9 million of cash used to fund operating activities in the first half of 2020. Based on its current clinical development plans, Rhythm expects that its existing cash and cash equivalents and short-term investments will enable it to fund its operations at least through the end of 2021.
    • R&D Expenses: R&D expenses were $22.9 million for the second quarter of 2020 as compared to $35.3 million for the second quarter of 2019. The decrease in R&D spending was primarily attributed to a $6.3 million reduction in clinical trial expenses associated with the Company's GO-ID genotyping study  and the once-weekly formulation study incurred with site initiations in 2019, a decrease of $4.0 million related to genetic sequencing with expected lower volume in the same study, and a $3.4 million decrease in expenses from purchases of setmelanotide API in the same quarter last year. These decreases were partially offset by an increase of $1.7 million in spending related to the Phase 2 Basket Study and a $2.0 million milestone payment associated with the license agreement with Ipsen on filing the NDA for setmelanotide for the treatment of POMC and LEPR deficiency obesities.
    • S,G&A Expenses: S,G&A expenses were $8.9 million for the second quarter of 2020 as compared to $8.8 million for the second quarter of 2019. This modest increase was primarily due to increased consulting and professional services fees.  
    • Net Loss: Net loss was $31.1 million for the second quarter of 2020, or a net loss per basic and diluted share of $0.71, as compared to a net loss of $42.8 million for the second quarter of 2019, or a net loss per basic and diluted share of $1.24.

    Year to Date 2020 Financial Results:

    • R&D Expenses: R&D expenses were $45.5 million for the six months ended June 30, 2020, as compared to $58.1 million for the six months ended June 30, 2019. The decrease was primarily due to a decrease of $8.7 million related to the GO-ID genotyping study, which is nearing its completion, and a decrease of $5.2 million related to genetic sequencing efforts from expected lower volumes in the same study, as well as a decrease of $2.5 million related to purchases of setmelanotide API. These decreases were partially offset by an increase of $2.0 million in spending related to the Phase 2 Basket Study and a $2.0 million milestone payment associated with the license agreement with Ipsen on filing the NDA for setmelanotide for the treatment of POMC and LEPR deficiency obesities.  
    • S,G&A Expenses: S,G&A expenses were $21.7 million for the six months ended June 30, 2020, as compared to $16.6 million for the six months ended June 30, 2019. The increase was primarily due to an accounting charge of $3.5 million related to the separation agreement and modification of stock options for the Company's former chief executive officer upon his departure on March 27, 2020, as well as an increase of $0.8 million related to patient engagement and disease awareness efforts and an increase of $0.8 million in various consulting and professional services related to legal and IT support costs.
    • Net Loss: Net loss was $65.3 million for the six months ended June 30, 2020, or a net loss per basic and diluted share of $1.48, as compared to a net loss of $71.8 million for the six months ended June 30, 2019, or a net loss per basic and diluted share of $2.08.

    About Rhythm Pharmaceuticals

    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. The FDA has accepted for filing an NDA for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity with Priority Review and assigned a PDUFA goal date of November 27, 2020. Rhythm also submitted an MAA for setmelanotide to treat individuals living with POMC deficiency obesity or LEPR deficiency obesity to the EMA in June 2020.  Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, expectations regarding regulatory approval, the anticipated timing for release of clinical trial data, our ongoing efforts related to patient identification and genetic sequencing and timing thereof, and the and the sufficiency of our cash, cash equivalents and short-term investments to fund our operations. Statements using word such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements.  Such statements are subject to numerous risks and uncertainties, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Rhythm Pharmaceuticals, Inc.

    Condensed Consolidated Statements of Operations and Comprehensive Loss

    (in thousands, except share and per share data)

     (Unaudited)

                  
       Three months ended June 30,  Six months ended June 30, 
       2020  2019  2020  2019 
    Operating expenses:             
    Research and development  $ 22,997  $ 35,308  $ 45,501  $ 58,069 
    Selling, general, and administrative    8,921    8,841    21,717    16,600 
    Total operating expenses    31,918    44,149    67,218    74,669 
    Loss from operations    (31,918)   (44,149)   (67,218)   (74,669)
    Other income (expense):             
    Interest income, net    801    1,353    1,937    2,899 
    Total other income, net    801    1,353    1,937    2,899 
    Net loss  $ (31,117) $ (42,796) $ (65,281) $ (71,770)
    Net loss per share, basic and diluted  $ (0.71) $ (1.24) $ (1.48) $ (2.08)
    Weighted-average common shares outstanding, basic and diluted    44,098,860    34,452,661    44,074,352    34,435,023 
                  
    Other comprehensive loss:             
    Net loss  $ (31,117) $ (42,796) $ (65,281) $ (71,770)
    Unrealized gain on marketable securities    567    —    630    — 
    Comprehensive loss  $ (30,550) $ (42,796) $ (64,651) $ (71,770)

    Rhythm Pharmaceuticals, Inc.

    Condensed Consolidated Balance Sheets

    (in thousands, except share data)

    (Unaudited)

          
      June 30,  December 31, 
      2020 2019
           
    Assets      
    Current assets:      
    Cash and cash equivalents $ 59,091  $ 62,294 
    Short-term investments   169,535    230,165 
    Prepaid expenses and other current assets   9,193    9,945 
    Total current assets   237,819    302,404 
    Property and equipment, net   3,331    3,671 
    Right-of-use asset   1,932    2,045 
    Restricted cash   403    403 
    Total assets $ 243,485  $ 308,523 
    Liabilities and stockholders' equity      
    Current liabilities:      
    Accounts payable $ 4,761  $ 10,415 
    Accrued expenses and other current liabilities   9,640    13,530 
    Lease liability   503    472 
    Total current liabilities   14,904    24,417 
    Long-term liabilities:      
    Lease liability   2,828    3,086 
    Total liabilities   17,732    27,503 
    Commitments and contingencies      
    Stockholders' equity:      
    Preferred Stock, $0.001 par value: 10,000,000 shares authorized; no shares issued and outstanding at June 30, 2020 and December 31, 2019   —    — 
    Common stock, $0.001 par value: 120,000,000 shares authorized; 44,115,612 and 43,996,753 shares issued and outstanding at June 30, 2020 and December 31, 2019, respectively   44    44 
    Additional paid-in capital   615,691    606,307 
    Accumulated other comprehensive income   630    — 
    Accumulated deficit   (390,612)   (325,331)
    Total stockholders' equity   225,753    281,020 
    Total liabilities and stockholders' equity $ 243,485  $ 308,523 

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  24. BOSTON, July 27, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced the appointment of Joseph Shulman as the Company's Senior Vice President, Technical Operations. Mr. Shulman brings more than 20 years of industry experience in chemistry, manufacturing and controls (CMC) technical development, operations, and program and alliance management.

    "We are pleased to welcome Joe to Rhythm, particularly as we prepare to bring setmelanotide to patients living with pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesities," said David Meeker, M.D., Chair…

    BOSTON, July 27, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced the appointment of Joseph Shulman as the Company's Senior Vice President, Technical Operations. Mr. Shulman brings more than 20 years of industry experience in chemistry, manufacturing and controls (CMC) technical development, operations, and program and alliance management.

    "We are pleased to welcome Joe to Rhythm, particularly as we prepare to bring setmelanotide to patients living with pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesities," said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. "His proven ability to develop supply chains and global manufacturing strategies will prove invaluable in enhancing and leading our efforts to bring our investigational drug setmelanotide to patients with rare genetic disorders of obesity."

    Mr. Shulman joins Rhythm from Ra Pharmaceuticals, recently acquired by UCB, where he served as senior vice president of technical operations and oversaw all aspects of CMC development and strategy as a member of the executive leadership team. Prior to that, he was senior vice president of global technical operations at Novelion Therapeutics, and earlier in his career, Mr. Shulman held similar roles at Ziopharm Oncology and Dyax Corp. He holds an M.B.A. from Boston University and earned a B.S. in chemical engineering from Miami University of Ohio.

    "Rhythm has an opportunity to deliver the first-ever therapy to individuals living with rare genetic disorders that cause severe obesity and insatiable hunger," Mr. Shulman said. "I look forward to collaborating with the team in an effort to ensure patients and caregivers around the world have a positive experience with setmelanotide. I am excited to join the Company during this transformational time."

    About Rhythm Pharmaceuticals

    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. The Company is developing setmelanotide, its investigational, melanocortin-4 receptor (MC4R) agonist, for the treatment of severe obesity and hyperphagia associated with rare genetic disorders of obesity. The U.S. Food and Drug Administration (FDA) has accepted for filing Rhythm's New Drug Application (NDA) for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity with Priority Review and a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020. Rhythm also submitted a Marketing Authorization Application (MAA) for setmelanotide to treat individuals living with POMC deficiency obesity or LEPR deficiency obesity to the European Medicines Agency (EMA) in June 2020. Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, including anticipated timing of data readouts and our expectations surrounding potential regulatory approvals and timing thereof. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881

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  25. BOSTON, July 20, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that David Meeker, M.D., the Chairman of Rhythm's Board of Directors, has been appointed as the President and Chief Executive Officer (CEO) of the company, effective immediately. Dr. Meeker succeeds Hunter Smith, the Company's Interim President and CEO and Chief Financial Officer (CFO), who will continue in his role as CFO.  

    "I am delighted to announce David's appointment as Rhythm's new CEO," said Hunter Smith, CFO of Rhythm. "Since he joined our Board in 2015, David has played a key role in shaping…

    BOSTON, July 20, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that David Meeker, M.D., the Chairman of Rhythm's Board of Directors, has been appointed as the President and Chief Executive Officer (CEO) of the company, effective immediately. Dr. Meeker succeeds Hunter Smith, the Company's Interim President and CEO and Chief Financial Officer (CFO), who will continue in his role as CFO.  

    "I am delighted to announce David's appointment as Rhythm's new CEO," said Hunter Smith, CFO of Rhythm. "Since he joined our Board in 2015, David has played a key role in shaping the clinical and commercial strategy for setmelanotide and in fostering our collaborative and patient-focused culture. As we continue to work toward the first potential approval of setmelanotide in pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesities later this year, David's extensive experience leading commercial organizations and managing the launches of new medicines for rare genetic diseases, coupled with his proven ability to build strong relationships with patient and clinician communities, will be invaluable. The Rhythm team is energized by the opportunity to work more closely with David in an effort to deliver setmelanotide and potentially transform the care of people living with rare genetic disorders of obesity."

    "Rhythm is an exciting company that I have long admired, both for its scientifically-rigorous approach to drug development and its commitment to patients with rare genetic disorders of obesity," said David Meeker, M.D. "With setmelanotide, we have the opportunity to bring one of the first meaningful therapeutic candidates to a segment of that community in dire need. Moreover, we hope our efforts will create visibility for rare genetic disorders of obesity, enabling better care for the people affected and catalyzing ongoing research efforts globally. The current management team has done a great job leading the organization through the transition and I am honored to take the CEO role."  

    Dr. Meeker has served as Chairman of Rhythm Pharmaceuticals since April 2017 and as a  member of the Board since November 2015. Most recently, he served as President and CEO of KSQ Therapeutics. Previously, Dr. Meeker was the Executive Vice President and Head of Sanofi Genzyme, the specialty-care global business unit of Sanofi that focuses on rare diseases, multiple sclerosis, oncology and immunology. Dr. Meeker joined Genzyme in 1994 as Medical Director and, over the course of his tenure, served the company as Vice President of Medical Affairs, Chief Operating Officer, and Chief Executive Officer. He led Genzyme's commercial organization and global market access functions and managed the launch of several treatments for rare genetic diseases, including Aldurazyme®, Fabrazyme® and Myozyme®. Prior to his tenure with Genzyme, Dr. Meeker was Director of the Pulmonary Critical Care Fellowship at the Cleveland Clinic and an Assistant Professor of Medicine at Ohio State University. Dr. Meeker earned his M.D. from the University of Vermont Medical School and completed the advanced management program at Harvard Business School.

    About Rhythm Pharmaceuticals

    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. The U.S. Food and Drug Administration (FDA) has accepted for filing Rhythm's New Drug Application (NDA) for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity with Priority Review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020. Rhythm also submitted a Marketing Authorization Application (MAA) for setmelanotide to treat individuals living with POMC deficiency obesity or LEPR deficiency obesity to the European Medicines Agency (EMA) in June 2020.  Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, including anticipated timing of data readouts and our expectations surrounding potential regulatory approvals and timing thereof. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881

    A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/66fae683-bf9b-4498-9dff-20f74fa14502

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  26. -- Study demonstrated that treatment with setmelanotide reduced body weight and hunger in individuals living with Bardet-Biedl syndrome --

    BOSTON, July 16, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, announced today that results from its Phase 2 study evaluating setmelanotide for the treatment of obesity and insatiable hunger, known as hyperphagia, in patients living with Bardet-Biedl Syndrome (BBS) were published in the peer-reviewed journal Diabetes, Obesity and Metabolism. As previously reported, data from the study demonstrate that treatment with setmelanotide, the…

    -- Study demonstrated that treatment with setmelanotide reduced body weight and hunger in individuals living with Bardet-Biedl syndrome --

    BOSTON, July 16, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, announced today that results from its Phase 2 study evaluating setmelanotide for the treatment of obesity and insatiable hunger, known as hyperphagia, in patients living with Bardet-Biedl Syndrome (BBS) were published in the peer-reviewed journal Diabetes, Obesity and Metabolism. As previously reported, data from the study demonstrate that treatment with setmelanotide, the company's melanocortin-4 receptor (MC4R) agonist, reduced body weight and hunger in individuals with BBS.

    "Treatment with setmelanotide demonstrated a substantial reduction in body weight and hunger, making this the first-ever Phase 2 study of any investigational pharmacologic agent to show positive results for the treatment of obesity and hunger in individuals living with BBS," said Robert M Haws, M.D., Director of the Clinical Research Center at the Marshfield Clinic Research Institute. "These findings also suggest that obesity and hyperphagia associated with BBS have ties to impaired signaling of the MC4R pathway and support further evaluation of MC4R activation as a potential treatment option."

    Rhythm initially reported topline data from its Phase 2 Basket Study of setmelanotide in BBS in June 2018 and most recently, in September 2019, Rhythm announced updated data following approximately two years on therapy. The Phase 2 study participants received a daily subcutaneous injection of setmelanotide. The dose was titrated every two weeks for up to 12 weeks to establish the individual dose. The primary outcome was mean percent change from baseline in body weight at three months. Mean percent change in body weight was also assessed after six and 12 months of treatment as additional exploratory efficacy outcomes. Change in hunger scores, metabolic parameters, and safety were secondary outcomes.

    "BBS is a complex disorder, and as the authors of this paper noted, there remains a crucial lack of clinical trials investigating obesity or hyperphagia treatment options in this population," said Murray Stewart, M.D., Chief Medical Officer of Rhythm. "There is a pressing need for a new therapy that may be able to reduce weight through hyperphagia management and potentially help manage or alleviate obesity related co-morbidities. We are encouraged by these Phase 2 study results and thank all the investigators for their  efforts in conducting this important research."  

    As announced in December 2019, Rhythm completed enrollment of the pivotal cohort in its Phase 3 clinical trial evaluating setmelanotide for the treatment of hyperphagia and severe obesity in individuals living with BBS or Alström syndrome. This Phase 3 trial enrolled 32 individuals with BBS and six individuals with Alström syndrome in the pivotal cohort, and Rhythm has continued to enroll patients in a supplemental cohort to meet demand and provide further data on the use of setmelanotide in people living with BBS or Alström syndrome. The company expects to report topline data from the pivotal Phase 3 trial evaluating setmelanotide in BBS and Alström syndrome in the fourth quarter of 2020 or early in the first quarter of 2021.

    The publication, Effect of Setmelanotide, an MC4R Agonist, on Obesity in Bardet-Biedl Syndrome, can be found online.

    About Bardet-Biedl Syndrome

    BBS is an ultra-rare genetic disorder that affects multiple organ systems. Clinical features of BBS may include Insatiable hunger, also known as hyperphagia, early-onset, severe obesity, cognitive impairment, polydactyly, renal dysfunction, hypogonadism and visual impairment. There is great variability in presentation and severity of these symptoms across individuals with BBS. In the United States, Rhythm estimates that BBS affects approximately 1,500 to 2,500 people. Currently, there are no approved therapies targeting the melanocortin-4 receptor (MC4R) pathway for reducing body weight and hunger in individuals living with BBS.

    About Setmelanotide

    Setmelanotide is an investigational, melanocortin-4 receptor (MC4R) agonist. The MC4R is part of the key biological pathway that independently regulates hunger, caloric intake, and energy expenditure. Variants in genes may impair the function of the MC4R pathway, potentially leading to hyperphagia and early-onset, severe obesity. Rhythm is currently developing setmelanotide as a targeted therapy to potentially restore the function of an impaired MC4R pathway and, in so doing, potentially reduce hunger and weight in patients with rare genetic disorders of obesity. Currently, no pharmacologic therapies exist to treat these conditions. The FDA has granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4R pathway, which includes BBS, Alström syndrome, pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity. The EMA has also granted PRIority MEdicines (PRIME) designation for setmelanotide for the treatment of obesity and the control of hunger associated with deficiency disorders of the MC4R pathway. Both the FDA and EMA have granted orphan drug status to setmelanotide for POMC and LEPR deficiency obesities. The FDA has accepted Rhythm's NDA for setmelanotide for the treatment of POMC and LEPR deficiency obesities for filing, granted Priority Review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020. Rhythm submitted an MAA for setmelanotide to treat individuals living with POMC deficiency obesity or LEPR deficiency obesity to the EMA in June 2020.

    About Rhythm Pharmaceuticals

    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. In August 2019, the company announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with POMC deficiency obesity or LEPR deficiency obesity. Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, anticipated trial enrollment and timing of data readouts, and our expectations surrounding the PDUFA goal date. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881

    Primary Logo

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  27. -- Company also announces positive data from eight supplemental patients, including four pediatric patients, enrolled in pivotal Phase 3 clinical trials; all achieved primary endpoint of 10 percent weight loss --

    -- Setmelanotide was well-tolerated in long-term extension study with continued clinical benefit and durable weight loss observed at up to three years on therapy --

    BOSTON, July 01, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that it has submitted its Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for setmelanotide…

    -- Company also announces positive data from eight supplemental patients, including four pediatric patients, enrolled in pivotal Phase 3 clinical trials; all achieved primary endpoint of 10 percent weight loss --

    -- Setmelanotide was well-tolerated in long-term extension study with continued clinical benefit and durable weight loss observed at up to three years on therapy --

    BOSTON, July 01, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that it has submitted its Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for setmelanotide, the company's melanocortin-4 receptor (MC4R) agonist, for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity. The company has been granted accelerated assessment of the MAA, which potentially shortens review time by the Committee for Medicinal Products for Human Use (CHMP).

    In conjunction with this submission, Rhythm announced additional positive data from eight supplemental patients, including four pediatric patients, enrolled in its two pivotal Phase 3 clinical trials for POMC and LEPR deficiency obesities, as well as updated data from its long-term extension study of setmelanotide in patients with POMC or LEPR deficiency obesity. Rhythm included these data in its MAA submission package to the EMA. 

    "We are pleased to have submitted our MAA for setmelanotide for the treatment of POMC and LEPR deficiency obesities, another step toward our goal of delivering setmelanotide to patients with these ultra-rare disorders," said Murray Stewart, M.D., Chief Medical Officer of Rhythm. "We are also excited to report additional data that provide further evidence regarding setmelanotide's potential impact in driving weight loss in patients with POMC or LEPR deficiency obesity. In particular, we are encouraged to see therapeutic activity in children and adolescents among our supplemental patients. These chronic disorders often arise in childhood, with patients suffering from severe obesity and insatiable hunger beginning at a very young age. By intervening earlier, we believe it may be possible to reduce body weight and hunger significantly, thereby preventing the development of debilitating comorbidities often associated with early-onset, rapid weight gain and improving quality of life for patients and their families."

    Dr. Stewart continued, "Additionally, data from our extension study show that treatment with setmelanotide remained well-tolerated for upwards of three years, with patients continuing to maintain, or even deepen, their weight loss."

    In August 2019, Rhythm announced positive topline results from the pivotal cohorts in its two Phase 3 clinical trials evaluating setmelanotide for the treatment of POMC and LEPR deficiency obesities. Both trials met their primary endpoints and all key secondary endpoints, demonstrating a statistically significant and clinically meaningful increase in weight loss and reductions in insatiable hunger, or hyperphagia, in patients with POMC and LEPR deficiency obesities.

    Data on Supplemental Patients with POMC and LEPR Deficiency Obesities

    Rhythm enrolled a total of eight patients, including four pediatric patients between the ages 6 and 12 years old, in supplemental cohorts in its Phase 3 trials evaluating setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity, with four supplemental patients enrolled in each trial. All eight supplemental patients achieved the primary endpoint of 10 percent or greater weight loss at 52 weeks on setmelanotide therapy, as calculated under the same statistical analysis plan used in the pivotal trials. All of the supplemental patients were enrolled by European investigators, as were most of the patients in the pivotal cohorts.

    The mean reduction in baseline body weight for the supplemental POMC deficiency obesity patients was -26.3 percent, and the mean reduction in body weight for the supplemental LEPR deficiency obesity patients was -13.2 percent. The estimated mean percentage reduction in most hunger score for evaluable patients in the supplemental cohorts was -57.3 percent. Hunger scores collected from children younger than 12 were calculated differently and therefore not counted in this analysis.

    Combining data from the eight supplemental patients with data from the pivotal cohorts, 12 out of 14 patients with POMC deficiency obesity and 9 out of 15 patients with LEPR deficiency obesity achieved the primary endpoints of greater than 10 percent weight loss over approximately one year. Additionally, the data for all key secondary endpoints from the supplemental cohorts were consistent with the data from the pivotal cohorts. 

    Long-term Extension Data for POMC and LEPR Deficiency Obesities

    A total of 15 patients who participated in the pivotal studies are being treated in the extension study, including nine living with POMC deficiency obesity and six living with LEPR deficiency obesity, all of whom previously completed one of Rhythm's two pivotal Phase 3 trials evaluating setmelanotide for the treatment of severe obesity and insatiable hunger. Two additional patients who were enrolled in the LEPR deficiency obesity pivotal study are expected to enroll in the extension study, pending local regulatory approval, and one patient with POMC deficiency obesity enrolled in the pivotal study did not continue in the extension study.

    As of April 16, 2020, extension study data show that patients successfully maintained durable weight loss with long-term treatment with setmelanotide for a total of up to 155 weeks. Hunger scores have typically remained stable throughout the extension study. Treatment in the extension study remains ongoing.

    Safety

    Consistent with prior clinical experience, setmelanotide has been was generally well-tolerated in the supplemental patient cohorts and in the long-term extension study. As of April 16, 2020, the safety results of setmelanotide remained consistent across all patients treated for up to three years. The most common treatment-emergent related adverse events (AEs) included injection site reactions, nausea and vomiting, and hyperpigmentation (darkening of the skin).

    Rhythm is continuing to analyze the efficacy and safety data from both the supplemental cohorts and the ongoing long-term extension study, and it plans to share more of these data at an upcoming medical meeting.

    About Setmelanotide

    Setmelanotide is an investigational, melanocortin-4 receptor (MC4R) agonist. The MC4R is part of the key biological pathway that independently regulates energy expenditure and appetite. Variants in genes may impair the function of the MC4R pathway, potentially leading to insatiable hunger and early-onset, severe obesity. Rhythm is currently developing setmelanotide as a targeted therapy to potentially restore the function of an impaired MC4R pathway and, in so doing, potentially reduce hunger and weight in patients with rare genetic disorders of obesity. Currently, no pharmacologic therapies exist to treat these conditions. The FDA has granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4R in the central melanocortin pathway, which includes pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity. The EMA has also granted PRIority MEdicines (PRIME) designation for setmelanotide for the treatment of obesity and the control of hunger associated with deficiency disorders of the MC4R pathway. Both the FDA and EMA have granted orphan drug status to setmelanotide for POMC and LEPR deficiency obesities. The FDA has accepted Rhythm's NDA for setmelanotide for the treatment of POMC and LEPR deficiency obesities for filing, granted Priority Review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020. Rhythm submitted a MAA for setmelanotide to treat individuals living with POMC deficiency obesity or LEPR deficiency obesity to the EMA in June 2020.

    About Rhythm Pharmaceuticals

    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. In August 2019, the company announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with POMC deficiency obesity or LEPR deficiency obesity. In May 2020, the FDA accepted Rhythm's NDA for setmelanotide in POMC or LEPR deficiency obesity, and in June 2020 the company submitted a MAA to the EMA. Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, anticipated trial enrollment and timing of data readouts, and our expectations surrounding the PDUFA goal date. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881

    Primary Logo

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  28. BOSTON, July 01, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designations for setmelanotide, an investigational melanocortin-4 receptor (MC4R) agonist, for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity. As previously announced, the Company's New Drug Application (NDA) for setmelanotide was accepted for filing with priority review by the FDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November…

    BOSTON, July 01, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designations for setmelanotide, an investigational melanocortin-4 receptor (MC4R) agonist, for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity. As previously announced, the Company's New Drug Application (NDA) for setmelanotide was accepted for filing with priority review by the FDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020.

    Under the FDA's rare pediatric disease designation and voucher programs, the FDA may grant a priority review voucher to a sponsor who receives a product approval for a "rare pediatric disease," which is defined as a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years and affects fewer than 200,000 people in the U.S. Subject to FDA approval of setmelanotide for the treatment of POMC and LEPR deficiency obesities, Rhythm would be eligible to receive one priority review voucher, which could then be redeemed to receive priority review for any subsequent marketing application, or sold or transferred to other companies for their programs.

    "We believe that receipt of the rare pediatric disease designation for setmelanotide, for which we have previously received Breakthrough Therapy and orphan drug status, underscores the FDA's recognition of setmelanotide's potential to treat POMC and LEPR deficiency obesities; two serious, ultra-rare conditions for which existing treatment options are woefully inadequate," said Murray Stewart, M.D., Chief Medical Officer of Rhythm. "As a result of early-onset, severe obesity and insatiable hunger, many patients experience debilitating comorbidities beginning in childhood, which worsen over time and can greatly affect their quality of life. With setmelanotide, we believe we have the potential to modify this disease trajectory by delivering a therapy that can be dosed chronically beginning in childhood. We are grateful to the FDA for this designation and look forward to continuing to work toward our goal of delivering setmelanotide to people with rare genetic disorders of obesity."

    In August 2019, Rhythm announced positive topline results from the pivotal cohorts in its two Phase 3 clinical trials evaluating setmelanotide for the treatment of POMC and LEPR deficiency obesities. Both trials met their primary endpoints and all key secondary endpoints, demonstrating a statistically significant and clinically meaningful reduction in weight loss and reductions in insatiable hunger, or hyperphagia, in patients with POMC and LEPR deficiency obesities.

    In June 2020, Rhythm announced new data from eight supplemental patients, including four pediatric patients aged 6-12, who subsequently enrolled in its two pivotal Phase 3 clinical trials for POMC and LEPR deficiency obesities. All eight supplemental patients, four of whom had POMC deficiency obesity and four of whom had LEPR deficiency obesity, achieved the primary endpoint of 10 percent or greater weight loss at 52 weeks on setmelanotide therapy, as calculated under the statistical analysis plan. 

    About POMC and LEPR Deficiency Obesities

    POMC and LEPR deficiency obesities are ultra-rare genetic disorders. Rhythm believes both disorders are underdiagnosed, and estimates there are approximately 100 to 500 patients in the U.S. with POMC deficiency obesity and approximately 500 to 2,000 patients in the U.S. with LEPR deficiency obesity. POMC deficiency obesity is a disorder caused by variants in the POMC or PCSK1 genes that can often lead to severe obesity beginning in childhood and insatiable hunger, in addition to endocrine abnormalities, and sometimes red hair and light skin pigmentation. LEPR deficiency obesity is a disorder caused by variants in the LEPR gene that can often lead to severe obesity beginning early in life and insatiable hunger, in addition to endocrine abnormalities. Most patients with POMC or LEPR deficiency obesity experience exponential weight gain in the first months of life, which continues rapidly over the course of their lives. This weight gain cannot be mitigated by diet, exercise or other lifestyle changes, or by existing therapeutic interventions.

    About Setmelanotide

    Setmelanotide is an investigational, melanocortin-4 receptor (MC4R) agonist. The MC4R is part of the key biological pathway that independently regulates energy expenditure and appetite. Variants in genes may impair the function of the MC4R pathway, potentially leading to insatiable hunger and early-onset, severe obesity. Rhythm is currently developing setmelanotide as a targeted therapy to potentially restore the function of an impaired MC4R pathway and, in so doing, potentially reduce hunger and weight in patients with rare genetic disorders of obesity. Currently, no pharmacologic therapies exist to treat these conditions. The FDA has granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4R pathway, which includes pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity. The EMA has also granted PRIority MEdicines (PRIME) designation for setmelanotide for the treatment of obesity and the control of hunger associated with deficiency disorders of the MC4R pathway. Both the FDA and EMA have granted orphan drug status to setmelanotide for POMC and LEPR deficiency obesities. The FDA has accepted Rhythm's NDA for setmelanotide for the treatment of POMC and LEPR deficiency obesities for filing, granted Priority Review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020. Rhythm submitted a MAA for setmelanotide to treat individuals living with POMC deficiency obesity or LEPR deficiency obesity to the EMA in June 2020.

    About Rhythm Pharmaceuticals

    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. In August 2019, the company announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with POMC deficiency obesity or LEPR deficiency obesity. Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, anticipated timing of data readouts, and our expectations surrounding the PDUFA goal date. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881

    Primary Logo

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  29. -- Once-weekly formulation of setmelanotide achieved weight loss efficacy comparable to daily-dosing formulation –

    -- Both weekly and daily formulations of setmelanotide were observed to be safe and well-tolerated –

    -- Pharmacokinetics analysis showed weekly formulation of setmelanotide trough drug concentration similar to daily formulation –

    BOSTON, June 24, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced interim data from a Phase 2 study evaluating a once-weekly formulation of setmelanotide, the Company's investigational melanocortin-4 receptor (MC4R) agonist…

    -- Once-weekly formulation of setmelanotide achieved weight loss efficacy comparable to daily-dosing formulation –

    -- Both weekly and daily formulations of setmelanotide were observed to be safe and well-tolerated –

    -- Pharmacokinetics analysis showed weekly formulation of setmelanotide trough drug concentration similar to daily formulation –

    BOSTON, June 24, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced interim data from a Phase 2 study evaluating a once-weekly formulation of setmelanotide, the Company's investigational melanocortin-4 receptor (MC4R) agonist. Healthy obese people treated with the weekly formulation of setmelanotide achieved comparable weight loss to those treated with the daily formulation, and both weekly and daily formulations of setmelanotide were observed to be safe and well-tolerated. Pharmacokinetics (PK) analyses showed similar trough drug concentrations for the daily and weekly formulations over the duration of therapy.

    "As a daily injection, setmelanotide has achieved statistically significant and clinically meaningful weight loss in patients with rare genetic disorders of obesity across multiple Phase 2 and Phase 3 clinical trials," said Murray Stewart, M.D., Chief Medical Officer of Rhythm. "These new data suggest that weekly setmelanotide may provide the same clinical benefit in a more convenient formulation, with the potential to reduce the burden on patients without compromising safety or efficacy. We look forward to evaluating these data further and continuing discussions with the U.S. Food and Drug Administration (FDA) about our path to registering weekly setmelanotide." 

    The Phase 2 study was designed to assess pharmacokinetics and the safety and tolerability of the weekly formulation of setmelanotide and its effect on reducing body weight in healthy individuals with a body mass index (BMI) of 40 kg/m2 or greater. A total of 75 individuals were included in this interim analysis: 42 individuals were treated with weekly setmelanotide (10mg, 20mg, or 30mg doses) for 12 weeks, 23 individuals were treated with placebo for 12 weeks, and ten individuals were treated with daily setmelanotide (2mg daily for one week, followed by3 mg daily for 11 weeks).

    The interim data analysis demonstrated that healthy obese people treated with weekly setmelanotide achieved similar weight loss to those treated with the daily formulation over 12 weeks of therapy.  The mean difference in change from baseline on weight between each weekly dose of 10mg, 20mg or 30mg and daily dose of 2mg/3mg was -0.69kg (p=0.659), -0.02kg (p=0.990), and -1.71kg (p=0.296), respectively.

    Weekly setmelanotide administration was well tolerated with no serious adverse events, and the safety profile was similar to the daily administration and consistent with prior clinical experience. Incidents of injection site reactions, hyperpigmentation and nausea or vomiting were classified as mild by investigators.

    In addition, an analysis of pharmacokinetics data measuring mean trough drug concentrations in plasma samples taken weekly for the duration of treatment showed that 20mg and 30mg doses of weekly setmelanotide were very similar to the 3mg daily dose of setmelanotide with greater trough drug concentration seen in the 30mg weekly dose.

    Rhythm is continuing to analyze these efficacy, safety and pharmacokinetics data for weekly setmelanotide, and it plans to share these data at an upcoming medical meeting. The Company also plans to discuss next steps towards registration with the FDA.

    Rhythm is developing this weekly formulation of setmelanotide to treat early-onset, severe obesity and insatiable hunger, the hallmark characteristics of rare genetic disorders of obesity. The weekly formulation leverages the extended-release FluidCrystal® injection depot technology which Rhythm licensed to develop with setmelanotide form Camurus AB in 2016. This drug delivery technology has been approved in Europe and Australia and provisionally approved by the FDA for use in weekly and monthly formulations of buprenorphine for the treatment of opioid use disorder.

    About Setmelanotide

    Setmelanotide is an investigational, melanocortin-4 receptor (MC4R) agonist. The MC4R is part of the key biological pathway that independently regulates energy expenditure and appetite. Variants in genes may impair the function of the MC4R pathway, potentially leading to insatiable hunger and early-onset, severe obesity. Rhythm is currently developing setmelanotide as a targeted therapy to restore the function of an impaired MC4R pathway and, in so doing, reduce hunger and weight in patients with rare genetic disorders of obesity. Currently, no pharmacologic therapies exist to treat these conditions. The FDA has granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4R in the central melanocortin pathway, which includes pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity. The European Medicines Agency (EMA) has also granted PRIority MEdicines (PRIME) designation for setmelanotide for the treatment of obesity and the control of hunger associated with deficiency disorders of the MC4R pathway. Both the FDA and EMA have granted orphan drug status to setmelanotide for POMC and LEPR deficiency obesities. The FDA has accepted Rhythm's New Drug Application (NDA) for setmelanotide for the treatment of POMC and LEPR deficiency obesities, granted Priority Review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020. Rhythm expects to complete submission of a Marketing Authorization Application (MAA) for setmelanotide to treat individuals living with POMC deficiency obesity or LEPR deficiency obesity to the EMA in the second quarter of 2020.

    About Rhythm Pharmaceuticals

    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. In August 2019, the company announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with POMC deficiency obesity or LEPR deficiency obesity and, in March 2020, completed its first rolling NDA submission to the FDA. Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, including the weekly formulation of setmelanotide, anticipated discussions with the FDA regarding next steps towards registration, our expectations surrounding the PDUFA goal date and the timing for submission of an MAA. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

     

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

     

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881

     

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  30. BOSTON, May 26, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that company management will participate in two investor conferences in June:

    Jefferies Virtual Healthcare Conference on Tuesday, June 2, 2020.

    • Hunter Smith, Interim Chief Executive Officer and Chief Financial Officer, and Murray Stewart, M.D., Chief Medical Officer, will present a corporate overview at 3:00 p.m. ET.

    Goldman Sachs 41st Annual Global Healthcare Conference on Tuesday, June 9, 2020. 

    • Hunter Smith, Interim Chief Executive Officer and Chief Financial Officer, and Murray Stewart, M.D…

    BOSTON, May 26, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that company management will participate in two investor conferences in June:

    Jefferies Virtual Healthcare Conference on Tuesday, June 2, 2020.

    • Hunter Smith, Interim Chief Executive Officer and Chief Financial Officer, and Murray Stewart, M.D., Chief Medical Officer, will present a corporate overview at 3:00 p.m. ET.

    Goldman Sachs 41st Annual Global Healthcare Conference on Tuesday, June 9, 2020. 

    • Hunter Smith, Interim Chief Executive Officer and Chief Financial Officer, and Murray Stewart, M.D., Chief Medical Officer, will participate in a fireside chat at 3:50 p.m. ET.

    A live audio webcast of both presentations will be available under "Events & Presentations" in the Investor Relations section of the Company's website at www.rhythmtx.com. A replay of the webcasts will be available on the Rhythm website for 30 days following the presentation.

    About Rhythm
    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. In August 2019, the company announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with POMC deficiency obesity and LEPR deficiency obesity and, in March 2020, completed its first rolling NDA submission to the FDA.  Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, the anticipated timing for release of topline clinical trial data, and participation in upcoming presentations and conferences. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements.  These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:
    David Connolly
    Head of Investor Relations and Corporate Communications
    Rhythm Pharmaceuticals, Inc.
    857-264-4280

    Investor Contact:
    Hannah Deresiewicz
    Stern Investor Relations, Inc.
    212-362-1200

    Media Contact:
    Adam Daley
    Berry & Company Public Relations
    212-253-8881

    Primary Logo

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  31. – FDA grants Priority Review of application and sets PDUFA goal date of November 27, 2020 –

    BOSTON, May 13, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that the U.S. Food and Drug Administration (FDA) has accepted the company's New Drug Application (NDA) for setmelanotide, an investigational, melanocortin-4 receptor (MC4R) agonist, for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity. The FDA granted Priority Review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November…

    – FDA grants Priority Review of application and sets PDUFA goal date of November 27, 2020 –

    BOSTON, May 13, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that the U.S. Food and Drug Administration (FDA) has accepted the company's New Drug Application (NDA) for setmelanotide, an investigational, melanocortin-4 receptor (MC4R) agonist, for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity. The FDA granted Priority Review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020. At this time, the FDA has indicated that it is not planning an advisory committee meeting as part of the NDA review.

    "The FDA's acceptance of our NDA for Priority Review signifies a critical milestone toward our mission of addressing the insatiable hunger and early-onset, severe obesity that affects individuals living with rare genetic disorders of obesity," said Murray Stewart, M.D., Chief Medical Officer of Rhythm Pharmaceuticals. "We are grateful to the Agency for its agility and dedication during these challenging times, as that has enabled continued progress and productive dialogue despite the ongoing pandemic. We look forward to working closely together throughout the review process to bring setmelanotide, if approved, to patients."

    A Priority Review designation is granted to drug candidates that may offer significant improvements in the safety or effectiveness of the treatment, prevention or diagnosis of a serious disease. Under Priority Review, the FDA aims to take action on an application within six months, compared to 10 months under standard review. The FDA previously granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4R in the central melanocortin pathway, which includes POMC deficiency obesity and LEPR deficiency obesity.

    About POMC and LEPR Deficiency Obesities
    POMC and LEPR deficiency obesities are ultra-rare genetic disorders. Rhythm estimates there are approximately 100 to 500 patients in the U.S. with POMC deficiency obesity and approximately 500 to 2,000 patients in the U.S. with LEPR deficiency obesity. POMC deficiency obesity is a disorder caused by variants in the POMC or PCSK1 genes that can often lead to severe obesity beginning early in life and insatiable hunger, in addition to endocrine abnormalities, and sometimes red hair and light skin pigmentation. LEPR deficiency obesity is a disorder caused by variants in the LEPR gene that can often lead to severe obesity beginning early in life and insatiable hunger, in addition to endocrine abnormalities. Most patients with POMC or LEPR deficiency obesity experience exponential weight gain in the first months of life, which continues rapidly over the course of their lives. This weight gain cannot be mitigated by diet, exercise or other lifestyle changes, or by existing therapeutic interventions.

    About Setmelanotide
    Setmelanotide is an investigational, melanocortin-4 receptor (MC4R) agonist. The MC4R is part of the key biological pathway that independently regulates energy expenditure and appetite. Variants in genes may impair the function of the MC4R pathway, potentially leading to insatiable hunger and early-onset, severe obesity. Rhythm is currently developing setmelanotide as a targeted therapy to restore the function of an impaired MC4R pathway and, in so doing, reduce hunger and weight in patients with rare genetic disorders of obesity. Currently, no pharmacologic therapies exist to treat these conditions. The FDA has granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4R in the central melanocortin pathway, which includes POMC deficiency obesity and LEPR deficiency obesity. The European Medicines Agency (EMA) has also granted PRIority MEdicines (PRIME) designation for setmelanotide for the treatment of obesity and the control of hunger associated with deficiency disorders of the MC4R pathway. Both the FDA and EMA have granted orphan drug status to setmelanotide for POMC and LEPR deficiency obesities. Rhythm expects to complete submission of a Marketing Authorization Application (MAA) for setmelanotide to treat individuals living with POMC deficiency obesity or LEPR deficiency obesity to the EMA in the second quarter of 2020.

    About Rhythm
    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. In August 2019, the company announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with POMC deficiency obesity or LEPR deficiency obesity and, in March 2020, completed its first rolling NDA submission to the FDA. Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, including our expectations surrounding the PDUFA goal date and the timing for submission of an MAA. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:
    David Connolly
    Head of Investor Relations and Corporate Communications
    Rhythm Pharmaceuticals, Inc.
    857-264-4280

    Investor Contact:
    Hannah Deresiewicz
    Stern Investor Relations, Inc.
    212-362-1200

    Media Contact:
    Adam Daley
    Berry & Company Public Relations
    212-253-8881

    Primary Logo

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  32. BOSTON, May 05, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that Hunter Smith, Interim Chief Executive Officer and Chief Financial Officer of Rhythm, and, Murray Stewart, M.D., Chief Medical Officer of Rhythm, will participate in a fireside chat at the 2020 BofA Securities Health Care Conference on Tuesday, May 12, 2020 at 8:20 a.m. ET.

    A live audio webcast of the presentation will be available under "Events & Presentations" in the Investor Relations section of the Company's website at www.rhythmtx.com. A replay of the webcast will be available on the Rhythm…

    BOSTON, May 05, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that Hunter Smith, Interim Chief Executive Officer and Chief Financial Officer of Rhythm, and, Murray Stewart, M.D., Chief Medical Officer of Rhythm, will participate in a fireside chat at the 2020 BofA Securities Health Care Conference on Tuesday, May 12, 2020 at 8:20 a.m. ET.

    A live audio webcast of the presentation will be available under "Events & Presentations" in the Investor Relations section of the Company's website at www.rhythmtx.com. A replay of the webcast will be available on the Rhythm website for 30 days following the presentation.

    About Rhythm
    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. In August 2019, the company announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with POMC deficiency obesity and LEPR deficiency obesity and, in March 2020, completed its first rolling NDA submission to the FDA.  Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, the anticipated timing for release of topline clinical trial data, and participation in upcoming presentations and conferences. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements.  These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:
    David Connolly
    Head of Investor Relations and Corporate Communications
    Rhythm Pharmaceuticals, Inc.
    857-264-4280

    Investor Contact:
    Hannah Deresiewicz
    Stern Investor Relations, Inc.
    212-362-1200

    Media Contact:
    Adam Daley
    Berry & Company Public Relations
    212-253-8881

    Primary Logo

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  33. -- Completed rolling NDA submission to FDA for setmelanotide in POMC and LEPR deficiency obesities; on track to submit MAA to EMA in the second quarter of 2020 –

    -- Received Orphan Drug Designation from FDA for setmelanotide for the treatment of Alström syndrome --

    -- Continues to expect topline data from pivotal Phase 3 trial of setmelanotide in Bardet-Biedl and Alström syndromes in fourth quarter of 2020 or early in the first quarter of 2021 --

    BOSTON, May 04, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today reported financial results and provided a business update for…

    -- Completed rolling NDA submission to FDA for setmelanotide in POMC and LEPR deficiency obesities; on track to submit MAA to EMA in the second quarter of 2020 –

    -- Received Orphan Drug Designation from FDA for setmelanotide for the treatment of Alström syndrome --

    -- Continues to expect topline data from pivotal Phase 3 trial of setmelanotide in Bardet-Biedl and Alström syndromes in fourth quarter of 2020 or early in the first quarter of 2021 --

    BOSTON, May 04, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today reported financial results and provided a business update for the first quarter ended March 31, 2020.

    "We are excited about the completion of our first New Drug Application (NDA) submission, which marks a critical next step towards bringing a potentially life-changing medicine to individuals living with rare genetic disorders of obesity," said Hunter Smith, interim Chief Executive Officer and Chief Financial Officer of Rhythm. "We are especially proud of how our Rhythm community, including our employees, the clinical staff at our trials sites, and the patients and families involved in our studies, has endured and met the unprecedented challenges brought on by the novel coronavirus pandemic, enabling our ongoing efforts to advance the development of setmelanotide with minimal interruption."

    Murray Stewart, M.D., Chief Medical Officer of Rhythm, said, "We are grateful for the support and dedication of the staff at the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) during these trying times, as their commitment to a consistent dialogue during the pandemic has supported the progression of our first NDA submission and our planned submission of a Marketing Authorization Application (MAA). As we move toward the potential commercialization of setmelanotide, our team is focused on identifying patients and advancing key pre-commercial activities across Rhythm. Looking ahead, we anticipate additional data from our Phase 2 Basket Study this year and remain on track to report topline data from our pivotal Phase 3 trial in Bardet-Biedl syndrome (BBS) and Alström syndrome in the fourth quarter of 2020 or early in the first quarter of 2021. As these studies progress, we are simultaneously focused on building greater awareness and understanding of melanocortin-4 receptor (MC4R) pathway obesity disorders among patients, physicians, caregivers and other key stakeholders."

    First Quarter 2020 and Recent Business Highlights:

    Pipeline and Recent Developments:

    • In March, Rhythm announced the completion of its rolling submission of an NDA to the FDA for setmelanotide for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity. Rhythm requested priority review for the application, which, if granted, could result in a six-month review period from the date of acceptance of the application.
    • In March, Rhythm announced that the FDA granted orphan drug designation to setmelanotide for the treatment of Alström syndrome.

    Corporate:

    • In March, Rhythm announced the appointment of Chief Financial Officer Hunter Smith as Interim President and Chief Executive Officer, succeeding Keith Gottesdiener, M.D., whose planned departure was announced previously in January 2020. Dr. Gottesdiener stepped down from his roles as CEO, President and member of the Board of Directors following the Company's completion of its NDA submission to the FDA. As previously disclosed, Rhythm's Board of Directors has formed a search committee and retained an executive search firm to assist in identifying a permanent successor.
    • Also in March, to help protect the health and safety of the patients, caregivers and healthcare professionals involved in its ongoing clinical trials of setmelanotide, as well as its employees, in response to the novel coronavirus (COVID-19) pandemic, Rhythm  implemented a number of precautionary clinical and operational measures to protect patient well-being and ensure consistent and appropriate clinical trial conduct. Rhythm has introduced measures designed to ensure patients already enrolled in ongoing clinical trials continue to be monitored as scheduled and receive their study drug.

    Upcoming Milestones:

    • Rhythm remains on track to submit an MAA to the EMA for setmelanotide in patients with POMC deficiency obesity and LEPR deficiency obesity in the second quarter of 2020.
    • Rhythm expects to report topline data from its combined pivotal Phase 3 trial evaluating setmelanotide in BBS and Alström syndrome in the fourth quarter of 2020 or early in the first quarter of 2021.
    • Rhythm expects to announce additional data from its ongoing Phase 2 Basket Study of setmelanotide in high-impact heterozygous (HET) obesity and additional data from one or more of its other ongoing Phase 2 Basket Study indications in 2020.
    • Rhythm expects to provide a clinical development update for its once-weekly formulation of setmelanotide in 2020.
    • Rhythm expects to submit an investigational new drug (IND) application for RM-853, its ghrelin o-acyltransferase (GOAT) inhibitor to the FDA in 2020. 
    • Rhythm expects to provide an update on its genetic sequencing efforts in 2020.

    First Quarter 2020 Financial Results:

    • Cash Position: As of March 31, 2020, cash, cash equivalents and short-term investments were $257.4 million, as compared to $292.5 million as of December 31, 2019. This decrease reflects cash used to fund operating activities in the first quarter of 2020. Based on its current clinical development plans, Rhythm expects that its existing cash and cash equivalents and short-term investments will enable it to fund its operations at least through the end of 2021.
    • R&D Expenses: R&D expenses were $22.5 million for the first quarter of 2020 as compared to $22.8 million for the first quarter of 2019. The modest decrease in R&D spending was attributed to decreased costs associated with the GO-ID genotyping study and partially offset by increased spending related to the submission of the NDA to FDA, expansion of additional studies including the Phase 2 Basket Study, long-term extension study, and the Phase 2 study of a weekly formulation of setmelanotide.
    • S,G&A Expenses: S,G&A expenses were $12.8 million for the first quarter of 2020 as compared to $7.8 million for the first quarter of 2019. The increase was primarily due to a non-cash, accounting charge of $3.5 million related to the separation agreement and modification of stock options for the former CEO and an increase of $1.2 million for efforts to drive disease awareness about rare genetic disorders of obesity and prepare for the potential commercialization of setmelanotide in the U.S.
    • Net Loss: Net loss was $34.2 million for the first quarter of 2020, or a net loss per basic and diluted share of $0.78, as compared to a net loss of $29.0 million for the first quarter of 2019, or a net loss per basic and diluted share of $0.84.

    About Rhythm Pharmaceuticals
    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. In August 2019, the company announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with POMC deficiency obesity and LEPR deficiency obesity and, in March 2020, completed its first rolling NDA submission to the FDA.  Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, the anticipated timing for release of clinical trial data, the timing for submission of an MAA, our ongoing efforts related to patient identification and genetic sequencing and timing thereof, our ongoing search for a Chief Executive Officer, the anticipated impact of the COVID-19 pandemic on our business and operations, and the and the sufficiency of our cash, cash equivalents and short-term investments to fund our operations. Statements using word such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements.  Such statements are subject to numerous risks and uncertainties, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.


    Condensed Consolidated Statements of Operations
    (in thousands, except share and per share data)
    (Unaudited)

                 
        Three months ended March 31, 
        2020
      2019
    Operating expenses:            
    Research and development   $  22,504     $  22,761  
    Selling, general, and administrative      12,796        7,759  
    Total operating expenses      35,300        30,520  
    Loss from operations      (35,300 )      (30,520 )
    Total other income, net      1,136        1,546  
    Net loss   $  (34,164 )   $  (28,974 )
    Net loss per share, basic and diluted   $  (0.78 )   $  (0.84 )
    Weighted-average common shares outstanding, basic and diluted      44,049,843        34,417,189  
                     


    Condensed Consolidated Balance Sheets
    (in thousands, except share and per share data)
    (Unaudited)

             
        March 31,    December 31, 
        2020
      2019
                 
    Assets            
    Current assets:            
    Cash and cash equivalents   $  55,776     $  62,294  
    Short-term investments      201,648        230,165  
    Prepaid expenses and other current assets      10,436        9,945  
    Total current assets      267,860        302,404  
    Property and equipment, net      3,504        3,671  
    Right-of-use asset      1,989        2,045  
    Restricted cash      403        403  
    Total assets   $  273,756     $  308,523  
    Liabilities and stockholders' equity            
    Current liabilities:            
    Accounts payable   $  5,932     $  10,415  
    Accrued expenses and other current liabilities      11,277        13,530  
    Lease liability      487        472  
    Total current liabilities      17,696        24,417  
    Long-term liabilities:            
    Lease liability      2,959        3,086  
    Total liabilities      20,655        27,503  
    Commitments and contingencies            
    Stockholders' equity:            
    Common stock      44        44  
    Additional paid-in capital      612,552        606,307  
    Accumulated deficit      (359,495 )      (325,331 )
    Total stockholders' equity      253,101        281,020  
    Total liabilities and stockholders' equity   $  273,756     $  308,523  
                     

    Corporate Contact:
    David Connolly
    Head of Investor Relations and Corporate Communications
    Rhythm Pharmaceuticals, Inc.
    857-264-4280

    Investor Contact:
    Hannah Deresiewicz
    Stern Investor Relations, Inc.
    212-362-1200

    Media Contact:
    Adam Daley
    Berry & Company Public Relations
    212-253-8881

    Primary Logo

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  34. BOSTON, April 07, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that Hunter Smith, Interim Chief Executive Officer and Chief Financial Officer of Rhythm, and Murray Stewart, M.D., Chief Medical Officer of Rhythm, will participate in a virtual fireside chat at the Needham & Co. 19th Annual Healthcare Conference on Tuesday, April 14, 2020 at 10:00 a.m. ET.

    A live audio webcast of the presentation will be available under "Events & Presentations" in the Investor Relations section of the Company's website at www.rhythmtx.com. A replay of the webcast will be available…

    BOSTON, April 07, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that Hunter Smith, Interim Chief Executive Officer and Chief Financial Officer of Rhythm, and Murray Stewart, M.D., Chief Medical Officer of Rhythm, will participate in a virtual fireside chat at the Needham & Co. 19th Annual Healthcare Conference on Tuesday, April 14, 2020 at 10:00 a.m. ET.

    A live audio webcast of the presentation will be available under "Events & Presentations" in the Investor Relations section of the Company's website at www.rhythmtx.com. A replay of the webcast will be available on the Rhythm website for 30 days following each presentation.

    About Rhythm

    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. In August 2019, the company announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with POMC deficiency obesity and LEPR deficiency obesity and, in March 2020, completed its first rolling NDA submission to the FDA.  Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Corporate Contact:

    David Connolly

    Head of Investor Relations and Corporate Communications

    Rhythm Pharmaceuticals, Inc.

    857-264-4280

    Investor Contact:

    Hannah Deresiewicz

    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:

    Adam Daley

    Berry & Company Public Relations

    212-253-8881

    Primary Logo

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  35.  Hunter Smith, Chief Financial Officer, appointed Interim Chief Executive Officer following completion of NDA submission to FDA 

     Precautionary measures in place to mitigate impact of COVID-19 on clinical programs 

    BOSTON, March 30, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage  biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that Hunter Smith, the Company's Chief Financial Officer, has been named to the additional role of Interim Chief Executive Officer, effective immediately. Mr. Smith succeeds Keith Gottesdiener, M.D. whose planned departure was announced by the Company in January 2020. Dr. Gottesdiener…

     Hunter Smith, Chief Financial Officer, appointed Interim Chief Executive Officer following completion of NDA submission to FDA 

     Precautionary measures in place to mitigate impact of COVID-19 on clinical programs 

    BOSTON, March 30, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage  biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that Hunter Smith, the Company's Chief Financial Officer, has been named to the additional role of Interim Chief Executive Officer, effective immediately. Mr. Smith succeeds Keith Gottesdiener, M.D. whose planned departure was announced by the Company in January 2020. Dr. Gottesdiener stepped down from his roles as CEO, President and member of the Board of Directors following the Company's completion of its New Drug Application (NDA) submission to the U.S. Food and Drug Administration (FDA).

    "On behalf of the Board, I am pleased that Hunter and the Rhythm management team will continue their excellent stewardship of the Company and dedication to transforming the care of people living with rare genetic disorders of obesity while we continue our search for a permanent CEO," said David Meeker, M.D., Chairman of the Board of Directors. "With Murray Stewart, Chief Medical Officer, leading our clinical development and regulatory strategy and Nithya Desikan, Chief Commercial Officer, and her team making tremendous strides in community building, we believe that Rhythm is well positioned to change the paradigm for rare genetic obesity."

    As previously disclosed, the Company's Board of Directors has formed a search committee and retained an executive search firm to assist in identifying Dr. Gottesdiener's permanent successor. The Board has made significant progress in the search process and is considering a number of highly qualified candidates.

    "I look forward to working closely with the Board, Murray and Nithya, our colleagues on the management team, and the entire Rhythm team to advance setmelanotide," Mr. Smith said. "We are committed to working with patients and the community to build greater awareness and understanding of rare genetic disorders of obesity so we can maximize our impact and transform the care of people living with these conditions."

    COVID-19 and Business Continuity
    To help protect the health and safety of the patients, caregivers and healthcare professionals involved in its ongoing clinical trials of setmelanotide, as well as its employees, in response to the novel coronavirus (COVID-19) pandemic, Rhythm has implemented a number of precautionary clinical and operational measures to protect patient well-being and ensure consistent and appropriate clinical trial conduct.

    With many clinical trial sites already closing down in response to COVID-19-related country- and state-level guidelines and more closures expected, Rhythm and study investigators and staff remain focused on the safety, treatment and monitoring of patients currently enrolled in these trials. Rhythm has introduced measures to ensure patients in ongoing clinical trials continue to be monitored as scheduled and receive their study drug.

    Mr. Smith continued, "We are acutely aware of the unprecedented crisis unfolding across the globe as a result of the COVID-19 pandemic. Over the past several  weeks, we have put measures in place to protect the health and safety of patients and staff participating in our clinical trials, as well as our employees and their families, and to seek to ensure that our ongoing studies can continue with minimal interruption."

    POMC Deficiency Obesity and LEPR Deficiency Obesity
    Today, Rhythm announced that it has completed its rolling submission of an NDA to the FDA for setmelanotide for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity. The FDA typically has a 60-day filing review period to determine whether the NDA is complete and acceptable for filing. Rhythm has requested priority review for the application which, if granted, could provide a target FDA review period of six months from the application filing date. Rhythm continues to anticipate that it will submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the second quarter of 2020. At this time, Rhythm is continuing its regular interactions with the FDA and EMA and based on current information, the Company does not anticipate COVID-19 to materially affect its timelines.

    Ongoing Clinical Trials of Setmelanotide
    Rhythm continues to expect to meet disclosed timelines for reporting data from its pivotal Phase 3 trial in Bardet-Biedl Syndrome (BBS) and Alström Syndrome and Phase 2 Basket Study. The Company anticipates announcing topline data in the fourth quarter of 2020 or early in the first quarter of 2021 from the Phase 3 BBS and Alström syndrome trial, which completed enrollment in December 2019. Rhythm also anticipates announcing additional data in high-impact heterozygous (HET) obesity and additional data from one or more of its other ongoing indications in 2020, based on current enrollment levels in the Phase 2 Basket Study.  

    Rhythm currently believes there will be no disruption of clinical supply of setmelanotide. The Company's contract manufacturers have indicated that they have appropriate plans and procedures in place to ensure uninterrupted future supply of clinical and commercial-grade setmelanotide, subject to potential limitations on their operations due to COVID-19.

    Corporate Operations

    Consistent with guidelines from the Centers for Disease Control (CDC) and the Commonwealth of Massachusetts, Rhythm has also implemented measures to help keep the Company's employees, families, and local communities healthy and safe. All employees are working remotely and all business travel has been restricted.

    About Rhythm

    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. In August 2019, the company announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with POMC deficiency obesity and LEPR deficiency obesity and, in March 2020, completed its first rolling NDA submission to the FDA.  Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, including the anticipated timing for enrollment and design of clinical trials, the timing for approval of an NDA and for submission of an MAA, the impact of COVID-19 on the Company's clinical trials, regulatory submissions and supply of setmelanotide, the ongoing CEO search, and expectations surrounding future guidance, the release of results of clinical trials and updates on patient enrollment. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements.  These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of COVID-19 and other global economic factors, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, and other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2019 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:
    David Connolly
    Head of Investor Relations and Corporate Communications
    Rhythm Pharmaceuticals, Inc.
    857-264-4280

    Investor Contact:
    Hannah Deresiewicz
    Stern Investor Relations, Inc.
    212-362-1200

    Media Contact:
    Adam Daley
    Berry & Company Public Relations
    212-253-8881

    Primary Logo

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  36. BOSTON, March 30, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that it has completed its rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for setmelanotide for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity.

    "The completion of our first NDA submission marks an integral first step in our journey to transform the care of people living with not only POMC and LEPR deficiency obesities, but also many other rare genetic disorders of obesity," said Murray…

    BOSTON, March 30, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that it has completed its rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for setmelanotide for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity.

    "The completion of our first NDA submission marks an integral first step in our journey to transform the care of people living with not only POMC and LEPR deficiency obesities, but also many other rare genetic disorders of obesity," said Murray Stewart, M.D., Chief Medical Officer of Rhythm Pharmaceuticals. "We look forward to further discussions with the FDA as we seek to obtain approval for setmelanotide, which we believe has the potential to address the insatiable hunger and early-onset, severe obesity that affects people living with POMC and LEPR deficiency obesities, who cannot be treated with lifestyle modifications alone and have no other treatment options."

    The FDA typically has a 60-day filing review period to determine whether the NDA is sufficiently complete and acceptable for filing. Rhythm has requested priority review for the application which, if granted, could provide a target FDA review period of six-months from the application filing date.   

    As first reported in August 2019, Rhythm's Phase 3 clinical trials of setmelanotide in patients with POMC deficiency obesity and LEPR deficiency obesity met their primary endpoints and all key secondary endpoints, demonstrating a statistically significant and clinically meaningful reduction in weight loss and reduction in hunger after one year of treatment with setmelanotide. Over the course of the Phase 3 trials, for patients with POMC deficiency obesity, mean weight loss was 31.9 kilograms, or 70.2 pounds, and for patients with LEPR deficiency obesity mean weight loss was 16.7 kilograms, or 36.8 pounds.

    About POMC and LEPR Deficiency Obesities
    POMC and LEPR deficiency obesities are ultra-rare disorders. Rhythm estimates there are approximately 100 to 500 patients in the U.S. with POMC deficiency obesity and approximately 500 to 2,000 patients in the U.S. with LEPR deficiency obesity. POMC deficiency obesity is a disorder caused by variants in the POMC or PCSK1 genes that can often lead to severe obesity beginning early in life and insatiable hunger, in addition to endocrine abnormalities, and sometimes red hair and light skin pigmentation. LEPR deficiency obesity is a disorder caused by variants in the LEPR gene that can often lead to severe obesity beginning early in life and insatiable hunger, in addition to endocrine abnormalities. Most patients with POMC and LEPR deficiency obesity experience exponential weight gain in the first months of life, which continues rapidly over the course of their lives. This weight gain cannot be mitigated by diet, exercise or other lifestyle changes, or by existing therapeutic interventions.

    About Setmelanotide
    Setmelanotide is an investigational, potent melanocortin-4 receptor (MC4R) agonist. The MC4R is part of the key biological pathway that independently regulates energy expenditure and appetite. Variants in genes may impair the function of the MC4R pathway, potentially leading to insatiable hunger and early-onset, severe obesity. Rhythm is currently developing setmelanotide as a targeted therapy to restore the function of an impaired MC4R pathway and, in so doing, reduce hunger and weight in patients with rare genetic disorders of obesity. Currently, no pharmacologic therapies exist to treat these conditions. The FDA has granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4R in the central melanocortin pathway, which includes POMC deficiency obesity, LEPR deficiency obesity, Bardet-Biedl syndrome (BBS) and Alström syndrome. The European Medicines Agency has also granted PRIority MEdicines (PRIME) designation for setmelanotide for the treatment of obesity and the control of hunger associated with deficiency disorders of the MC4R pathway. Both the FDA and EMA have granted orphan drug status to setmelanotide for POMC and LEPR deficiency obesities, BBS and Alström syndrome.

    About Rhythm
    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. In August 2019, the company announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with POMC deficiency obesity and LEPR deficiency obesity and, in March 2020, completed its first rolling NDA submission to the FDA.  Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, including the effectiveness of setmelanotide in patients with POMC and LEPR deficiency obesities, and the timing for approval of an NDA. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of the COVID-19 outbreak and other global economic factors, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, and other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2019 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:
    David Connolly
    Head of Investor Relations and Corporate Communications
    Rhythm Pharmaceuticals, Inc.
    857-264-4280

    Investor Contact:
    Hannah Deresiewicz
    Stern Investor Relations, Inc.
    212-362-1200

    Media Contact:
    Adam Daley
    Berry & Company Public Relations
    212-253-8881

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  37. BOSTON, March 18, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to setmelanotide for the treatment Alström syndrome. People living with Alström syndrome may experience an insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life. Rhythm is currently evaluating setmelanotide's ability to reduce hunger and affect weight loss in an ongoing pivotal Phase 3 trial in patients living with Alström and Bardet-Biedl syndromes.

    "Individuals and families living…

    BOSTON, March 18, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to setmelanotide for the treatment Alström syndrome. People living with Alström syndrome may experience an insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life. Rhythm is currently evaluating setmelanotide's ability to reduce hunger and affect weight loss in an ongoing pivotal Phase 3 trial in patients living with Alström and Bardet-Biedl syndromes.

    "Individuals and families living with Alström syndrome face a high disease burden, which often adversely affects their daily lives, and yet there are currently no treatment options available to address this serious unmet need," said Murray Stewart, M.D., Chief Medical Officer of Rhythm. "Orphan drug designation from the FDA reinforces the urgency of our work with setmelanotide in Alström syndrome, as we advance our pivotal Phase 3 trial to topline data expected by the end of this year or early next year."

    Orphan drug designation is granted by the FDA to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain incentives, which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers.

    About Alström Syndrome
    Alström syndrome is an ultra-rare genetic disorder that affects multiple organ systems. Insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life may be seen in individuals living with Alström syndrome. Clinical features of Alström syndrome may include progressive visual and auditory impairments, insulin resistance and Type 2 diabetes, hyperlipidemia, progressive kidney dysfunction, cardiomyopathy and short stature in adulthood. There is great variability in presentation and severity of these symptoms across individuals with Alström syndrome. In the United States, the Company estimates that Alström syndrome affects approximately 500 people. Currently, there are no approved therapies that restore the impaired function of the melanocortin-4 receptor (MC4R) pathway, a component of the central melanocortin pathway, for reducing body weight and hunger in Alström syndrome.

    About Setmelanotide
    Setmelanotide is a potent MC4R agonist in development for the treatment of rare genetic disorders of obesity. Setmelanotide activates MC4R, part of the key biological pathway that independently regulates energy expenditure and appetite. Variants in genes within the MC4R pathway are associated with insatiable hunger and early-onset, severe obesity. Rhythm is currently developing setmelanotide as a targeted therapy that restores function of an impaired MC4R pathway to reduce weight and hunger in patients for whom there are no effective or approved therapies. The FDA has granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4 receptor in the central melanocortin pathway, which includes POMC deficiency obesity, LEPR deficiency obesity, Bardet-Biedl syndrome and Alström syndrome. The European Medicines Agency has also granted PRIority MEdicines (PRIME) designation for setmelanotide for the treatment of obesity and the control of hunger associated with deficiency disorders of the MC4R pathway.

    About Rhythm
    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. The company recently announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with POMC deficiency obesity and LEPR deficiency obesity and plans to complete its first rolling NDA submission to the FDA in the first quarter of 2020. Rhythm is also evaluating setmelanotide in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties, including statements regarding Rhythm's anticipated timing for filing of an NDA and the release of results of clinical trials. Statements using word such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements.  Such statements are subject to numerous risks and uncertainties, including but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, and expenses, and other risks as may be detailed from time to time in our Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q and other reports we file with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:
    David Connolly
    Head of Investor Relations and Corporate Communications
    Rhythm Pharmaceuticals, Inc.
    857-264-4280

    Investor Contact:
    Hannah Deresiewicz
    Stern Investor Relations, Inc.
    212-362-1200

    Media Contact:
    Adam Daley
    Berry & Company Public Relations
    212-253-8881

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  38. -- On track to complete rolling NDA submission to the FDA for setmelanotide in POMC and LEPR deficiency obesities in the first quarter of 2020 --

    -- Granted orphan drug designation by the European Medicines Agency for setmelanotide in Alström syndrome --

    -- Completed full enrollment in Phase 3 trial of setmelanotide in Bardet-Biedl and Alström syndromes in December; topline data expected in the fourth quarter of 2020 or early in the first quarter of 2021 --

    --Rhythm Phase 2 Basket study actively enrolling multiple cohorts across five rare MC4R pathway-related disorders with potential U.S. prevalence greater than 80,000 patients; data updates expected in 2020 --

    -- Ongoing genetic sequencing efforts have sequenced more than 25,000 people with

    -- On track to complete rolling NDA submission to the FDA for setmelanotide in POMC and LEPR deficiency obesities in the first quarter of 2020 --

    -- Granted orphan drug designation by the European Medicines Agency for setmelanotide in Alström syndrome --

    -- Completed full enrollment in Phase 3 trial of setmelanotide in Bardet-Biedl and Alström syndromes in December; topline data expected in the fourth quarter of 2020 or early in the first quarter of 2021 --

    --Rhythm Phase 2 Basket study actively enrolling multiple cohorts across five rare MC4R pathway-related disorders with potential U.S. prevalence greater than 80,000 patients; data updates expected in 2020 --

    -- Ongoing genetic sequencing efforts have sequenced more than 25,000 people with early-onset, severe obesity --

    BOSTON, March 02, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2019.

    "We believe Rhythm is entering 2020 in a position of strength as we continue to make significant progress in the science and understanding of rare genetic disorders of obesity and prepare to deliver setmelanotide as the first therapeutic option for people living with these conditions," said Keith Gottesdiener, M.D., Chief Executive Officer of Rhythm.

    Murray Stewart, M.D., Chief Medical Officer, added, "We expect to submit our first New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the coming weeks. In parallel, we are focused on advancing the development of setmelanotide in five additional melanocortin-4 receptor (MC4R) pathway-related disorders in our Phase 2 Basket study. We are also working to build a robust community of patients, caregivers and healthcare providers in order to drive patient identification, increase genetic testing and improve the understanding of these conditions. These efforts are critically important to our progress in Bardet-Biedl syndrome (BBS) and Alström syndrome, for which we expect topline pivotal data in the fourth quarter of 2020 or early in the first quarter of 2021. Lastly, our ongoing sequencing efforts continue to demonstrate that a significant percentage of severely obese individuals may be suffering from MC4R-related disorders of severe obesity and unrelenting hunger, indicating a need for a therapy like setmelanotide that has the potential to treat people living with these conditions."

    Fourth Quarter and Recent Business Highlights:

    Pipeline and Recent Developments:

    • Today, Rhythm announced that its ongoing genetic sequencing programs have now sequenced more than 25,000 people with early-onset, severe obesity. Rhythm's sequencing efforts seek to uncover more rare genetic disorders of obesity and develop a better understanding of those disorders currently under study in its pivotal trials and Phase 2 Basket Study. Rhythm expects to provide an update on its genetic sequencing efforts in 2020.
    • Today, Rhythm announced that the European Commission adopted the European Medicines Agency's (EMA's) Committee for Orphan Medicinal Products' positive opinion and designated setmelanotide as an orphan medicinal product for the treatment of patients with Alström syndrome.
    • In December 2019, Rhythm announced the completion of enrollment in its pivotal Phase 3 clinical trial evaluating setmelanotide in BBS and Alström syndrome. The Company enrolled 32 individuals with BBS and six individuals with Alström syndrome in the pivotal cohort.
    • In November 2019, Rhythm presented late-breaking data from its two pivotal Phase 3 trials evaluating setmelanotide in POMC deficiency obesity and LEPR deficiency obesity at ObesityWeek 2019®, which highlighted the effect of setmelanotide on body mass index scores and certain cardiovascular parameters.

    Corporate:

    • In January 2020, Rhythm announced that Dr. Gottesdiener intends to step down as Chief Executive Officer, President and a member of the Company's Board of Directors, following the NDA submission for POMC deficiency obesity and LEPR deficiency obesity, expected in the first quarter of 2020. Rhythm's Board of Directors has initiated a search for a new chief executive officer.
    • In October 2019, Rhythm completed a public offering of 9,324,324 shares of its common stock at a public offering price of $18.50 per share, for aggregate gross proceeds of approximately $172.5 million, before underwriting discounts, commissions, and offering expenses.

    Upcoming Milestones:

    • Rhythm remains on track to complete submission of its rolling NDA to the FDA for setmelanotide in patients with POMC deficiency obesity and LEPR deficiency obesity in the first quarter of 2020.
    • Rhythm expects to submit a Marketing Authorization Application (MAA) to the EMA for setmelanotide in patients with POMC deficiency obesity and LEPR deficiency obesity in the second quarter of 2020.
    • Rhythm expects to report topline data from its combined Phase 3 trial evaluating setmelanotide in BBS and Alström syndrome in the fourth quarter of 2020 or early in the first quarter of 2021.
    • Rhythm expects to announce additional data from its ongoing Phase 2 Basket Study of setmelanotide in high-impact heterozygous (HET) obesity and additional data from one or more of its other ongoing Phase 2 Basket Study indications in 2020.
    • Rhythm expects to provide a clinical development update for its once-weekly formulation of setmelanotide in 2020.
    • Rhythm expects to submit an investigational new drug (IND) application for RM-853, its ghrelin o-acyltransferase (GOAT) inhibitor for the treatment of Prader-Willi syndrome, to the FDA in 2020. 
    • Rhythm expects to provide an update on its genetic sequencing efforts in 2020.

    Fourth Quarter and Full Year 2019 Financial Results:

    • Cash Position: As of December 31, 2019, cash, cash equivalents and short-term investments were $292.5 million, as compared to $252.1 million as of December 31, 2018. This increase reflects net proceeds of $161.4 million from Rhythm's public offering of common stock in October 2019, partially offset by cash used to fund operating activities in 2019. Based on its current clinical development plans, Rhythm expects that its existing cash and cash equivalents and short-term investments will enable it to fund operations through at least the end of 2021.
    • R&D Expenses: R&D expenses were $24.8 million in the fourth quarter of 2019 and $109.5 million for the year ended December 31, 2019, as compared to $18.8 million in the fourth quarter of 2018 and $50.3 million for the year ended December 31, 2018. The year-over-year increase was primarily due to an increase of $34.6 million related to Rhythm's clinical trials, including an expansion of enrollment and opening of new trial sites for the ongoing Phase 3 study of setmelanotide in patients with BBS and Alström syndrome, the Phase 2 Basket Study, and the GO-ID genotyping study; as well as an increase of $11.8 million related to translational research and genetic sequencing efforts designed to improve identification of patients with MC4R pathway deficiencies and pathway validation efforts; and an increase of $6.8 million due to the hiring of additional personnel in Medical Affairs as well as research and development. This increase was partially offset by a decrease of $4.4 million due to the non-cash expense related to the license acquired from Takeda for RM-853 in March 2018.    
    • S,G&A Expenses: S,G&A expenses were $9.4 million for the fourth quarter of 2019 and $36.6 million for the year ended December 31, 2019, as compared to $8.4 million for the fourth quarter of 2018 and $28.1 million for the year ended December 31, 2018. The year-over-year increase was primarily due to an increase of $6.9 million in employee-related costs in connection with the full-year impact of employees hired in 2018, as well as new personnel in 2019, to support planned commercial activities, operations and finance.
    • Net Loss: Net loss was $33.0 million for the fourth quarter of 2019 and $140.7 million for the year ended December 31, 2019, or a net loss per basic and diluted share of $0.78 and $3.86, respectively, as compared to a net loss of $25.5 million for the fourth quarter of 2018 and $74.1 million for the year ended December 31, 2018, or a net loss per basic and diluted share of $0.74 and $2.39, respectively. 

    About Rhythm Pharmaceuticals

    Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. The company recently announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with POMC deficiency obesity and LEPR deficiency obesity, and plans to complete its first rolling NDA submission to the FDA in the first quarter of 2020. Rhythm is also evaluating setmelanotide in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties, including statements regarding Rhythm's anticipated timing for enrollment and design of clinical trials, the timing for filing of an NDA, submission of an investigational new drug application and submission of an MAA, its ongoing efforts related to patient identification and genetic sequencing, the release of results of clinical trials and updates on patient enrollment, and its sufficiency of cash. Statements using word such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements.  Such statements are subject to numerous risks and uncertainties, including but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, and expenses, and other risks as may be detailed from time to time in our Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q and other reports we file with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

     
    Consolidated Statements of Operations
    (in thousands, except share and per share data)
               
        Three months ended
    December 31, 
        Year ended
    December 31, 
        2019
      2018
        2019
      2018
    Operating expenses:                          
    Research and development   $ 24,810     $ 18,763       $ 109,450     $ 50,337  
    Selling, general, and administrative     9,414       8,388         36,550       28,080  
    Total operating expenses     34,224       27,151         146,000       78,417  
    Loss from operations     (34,224 )     (27,151 )       (146,000 )     (78,417 )
    Other income (expense):                          
    Interest income, net     1,268       1,644         5,271       4,353  
    Total other income:     1,268       1,644         5,271       4,353  
    Net loss   $ (32,956 )   $ (25,507 )     $ (140,729 )   $ (74,064 )
    Net loss per common share, basic and diluted   $ (0.78 )   $ (0.74 )     $ (3.86 )   $ (2.39 )
    Weighted average common shares outstanding, basic and diluted     42,213,180       34,400,916         36,422,450       31,004,047  
                                       


     
    Consolidated Balance Sheets
    (in thousands, except share and per share data)
             
        December 31,   December 31, 
        2019
      2018
                 
    Assets            
    Current assets:            
    Cash and cash equivalents   $ 62,294     $ 49,542  
    Short-term investments     230,165       202,519  
    Prepaid expenses and other current assets     9,945       6,628  
    Total current assets     302,404       258,689  
    Property and equipment, net     3,671       1,120  
    Right-of-use asset     2,045        
    Restricted cash     403       401  
    Total assets   $ 308,523     $ 260,210  
    Liabilities and stockholders' equity            
    Current liabilities:            
    Accounts payable   $ 10,415     $ 7,640  
    Accrued expenses and other current liabilities     13,530       5,942  
    Lease liability     472        
    Total current liabilities     24,417       13,582  
    Long-term liabilities:            
    Lease liability     3,086        
    Deferred rent           372  
    Total liabilities     27,503       13,954  
                 
    Stockholders' equity:            
    Preferred Stock, $0.001 par value: 10,000,000 shares authorized; no shares issued and outstanding at December 31, 2019 and 2018            
    Common stock, $0.001 par value: 120,000,000 shares authorized; 43,996,753 and 34,410,725 shares issued and outstanding December 31, 2019 and 2018, respectively     44       34  
    Additional paid-in capital     606,307       430,824  
    Accumulated deficit     (325,331 )     (184,602 )
    Total stockholders' equity     281,020       246,256  
    Total liabilities and stockholders' equity   $ 308,523     $ 260,210  
                     

    Corporate Contact:
    David Connolly
    Head of Investor Relations and Corporate Communications
    Rhythm Pharmaceuticals, Inc.
    857-264-4280

    Investor Contact:
    Hannah Deresiewicz
    Stern Investor Relations, Inc.
    212-362-1200

    Media Contact:
    Adam Daley
    Berry & Company Public Relations
    212-253-8881

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  39. BOSTON, Feb. 25, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that Murray Stewart, M.D., Chief Medical Officer of Rhythm, and Hunter Smith, Chief Financial Officer of Rhythm, will participate in a fireside chat at the Cowen & Co. 40th Annual Health Care Conference on Tuesday, March 3, 2020 at 8:00 a.m. ET in Boston, MA.

    A live audio webcast of the presentation will be available under "Events & Presentations" in the Investor Relations section of the Company's website at www.rhythmtx.com. A replay of the webcast will be available on the Rhythm website for 30 days…

    BOSTON, Feb. 25, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that Murray Stewart, M.D., Chief Medical Officer of Rhythm, and Hunter Smith, Chief Financial Officer of Rhythm, will participate in a fireside chat at the Cowen & Co. 40th Annual Health Care Conference on Tuesday, March 3, 2020 at 8:00 a.m. ET in Boston, MA.

    A live audio webcast of the presentation will be available under "Events & Presentations" in the Investor Relations section of the Company's website at www.rhythmtx.com. A replay of the webcast will be available on the Rhythm website for 30 days following each presentation.

    About Rhythm
    Rhythm is a biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. The company recently announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with POMC deficiency obesity and LEPR deficiency obesity, and plans to complete its first rolling NDA submission to the FDA in the first quarter of 2020. Rhythm is also evaluating setmelanotide in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Corporate Contact:
    David Connolly
    Head of Investor Relations and Corporate Communications
    Rhythm Pharmaceuticals, Inc.
    857-264-4280

    Investor Contact:
    Hannah Deresiewicz
    Stern Investor Relations, Inc.
    212-362-1200

    Media Contact:
    Adam Daley
    Berry & Company Public Relations
    212-253-8881

    Primary Logo

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  40. Keith Gottesdiener, M.D. to Step Down as Chief Executive Officer Following NDA Submission for Setmelanotide in POMC and LEPR Deficiency Obesities Expected in the First Quarter of 2020

    Board Forms Search Committee to Assist in Identifying Successor

    BOSTON, Jan. 06, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that Keith Gottesdiener, M.D., intends to step down as Chief Executive Officer, President and a member of the Company's Board of Directors. The Board has formed a search committee and retained an executive search firm to assist in identifying Dr. Gottesdiener's successor…

    Keith Gottesdiener, M.D. to Step Down as Chief Executive Officer Following NDA Submission for Setmelanotide in POMC and LEPR Deficiency Obesities Expected in the First Quarter of 2020

    Board Forms Search Committee to Assist in Identifying Successor

    BOSTON, Jan. 06, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that Keith Gottesdiener, M.D., intends to step down as Chief Executive Officer, President and a member of the Company's Board of Directors. The Board has formed a search committee and retained an executive search firm to assist in identifying Dr. Gottesdiener's successor. Dr. Gottesdiener plans to serve in his current role until the completion of the Company's submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA).

    "On behalf of the entire Board of Directors, I want to thank Keith for his significant contributions to Rhythm," said David Meeker, M.D., Chairman of the Board. "Under Keith's leadership, Rhythm has become a recognized leader in rare genetic disorders of obesity, advancing setmelanotide through late-stage clinical development in four indications, identifying additional melanocortin-4 receptor (MC4R) pathway disorders that may be amenable to treatment, and building a robust community of healthcare providers, advocacy groups, patients and families to better understand, identify and treat people living with these conditions. We wish him the best in his future endeavors."

    Dr. Meeker continued, "As Rhythm prepares to enter its next chapter as a commercial company, now is an appropriate time to initiate this transition. We believe Rhythm has a strong management team, is well capitalized, and all of its major milestones are on track, including the planned submission of our first NDA to the FDA. We are confident this will be a smooth transition as we work to identify the next leader for Rhythm and continue to execute on our mission of transforming the standard of care for people with rare genetic disorders of obesity."

    "For the more than eight years that I have been CEO, it has been an honor to lead the talented Rhythm team, and I am immensely proud of our work to advance setmelanotide and bolster the understanding of rare genetic disorders of obesity," said Dr. Gottesdiener. "With the completion of our first Phase 3 trials and our pending NDA submission, it is a natural time for me to pass the baton and for Rhythm to welcome new leadership. I am confident that Rhythm is in an excellent position to deliver on the potential of setmelanotide, and I look forward to working with the Board and leadership team over the next several months to ensure a smooth transition."

    In August of 2019, Rhythm announced the achievement of positive topline results from its pivotal Phase 3 clinical trials evaluating setmelanotide, the Company's MC4R agonist, for the treatment of pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesities.

    Rhythm remains on track to complete the submission of a rolling NDA to the FDA that will cover both POMC and LEPR deficiency obesities in the first quarter of 2020. Rhythm also reiterates its previous guidance on other major milestones and continues to expect topline data from its Phase 3 trial evaluating setmelanotide in Bardet-Biedl and Alström syndromes in the fourth quarter of 2020 or early in the first quarter of 2021. Additionally, Rhythm is well capitalized, and believes it has sufficient resources to fund its operations through at least the end of 2021.

    About Rhythm
    Rhythm is a biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. The company recently announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with POMC deficiency obesity and LEPR deficiency obesity, and plans to complete its first rolling NDA submission to the FDA in the first quarter of 2020. Rhythm is also evaluating setmelanotide in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties, including statements regarding Rhythm's anticipated timing for submission of an NDA, its ability to find a successor for its Chief Executive Officer and President, its expectations regarding the timing of topline data from its clinical trials, its sufficiency of cash, and its ongoing efforts related the efficacy of setmelanotide in patients with POMC deficiency obesity, LEPR deficiency obesity, Bardet-Biedl syndrome and Alström syndrome. Statements using words such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward looking statements. Such statements are subject to numerous risks and uncertainties, including but not limited to, Rhythm's ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the impact of the management transition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, use of cash and expenses, and other risks as may be detailed from time to time in Rhythm's Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q and other reports it files with the Securities and Exchange Commission. Except as required by law, Rhythm undertakes no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:
    David Connolly
    Head of Investor Relations and Corporate Communications
    Rhythm Pharmaceuticals, Inc.
    857-264-4280

    Investor Contact:
    Hannah Deresiewicz
    Stern Investor Relations, Inc.
    212-362-1200

    Media Contact:
    Adam Daley
    Berry & Company Public Relations
    212-253-8881

    Primary Logo

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  41. -- Topline data expected in the fourth quarter of 2020 or early in the first quarter of 2021 --

    -- Supplemental cohort continues enrollment to meet patient demand --

    BOSTON, Dec. 05, 2019 (GLOBE NEWSWIRE) --  Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced it completed enrollment of the pivotal cohort in its Phase 3 clinical trial evaluating setmelanotide for the treatment of insatiable hunger and severe obesity in individuals living with Bardet-Biedl syndrome (BBS) or Alström syndrome. The Company enrolled 32 individuals with BBS and six individuals with Alström syndrome in the pivotal cohort…

    -- Topline data expected in the fourth quarter of 2020 or early in the first quarter of 2021 --

    -- Supplemental cohort continues enrollment to meet patient demand --

    BOSTON, Dec. 05, 2019 (GLOBE NEWSWIRE) --  Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced it completed enrollment of the pivotal cohort in its Phase 3 clinical trial evaluating setmelanotide for the treatment of insatiable hunger and severe obesity in individuals living with Bardet-Biedl syndrome (BBS) or Alström syndrome. The Company enrolled 32 individuals with BBS and six individuals with Alström syndrome in the pivotal cohort, and it will continue to enroll patients in a supplemental cohort to meet demand and provide further data on the use of setmelanotide in people living with these conditions.

    "With no approved treatment options available, there is a pressing need for a new therapy that addresses the insatiable hunger and severe obesity that people living with BBS or Alström syndrome may experience," said Murray Stewart, M.D., Chief Medical Officer of Rhythm. "We believe the significant demand among patients, families, caregivers and healthcare providers in the BBS community reflects this need for a therapy to address these conditions. We look forward to continuing to partner with the BBS and Alström syndrome communities as we work to identify patients, better understand the long-term burden of these disorders and advance this pivotal trial toward topline data in the fourth quarter of 2020 or early in the first quarter of 2021."

    About Bardet-Biedl and Alström Syndromes
    BBS and Alström syndrome are ultra-rare genetic disorders that affect multiple organ systems. Clinical features of BBS may include cognitive impairment, polydactyly, renal dysfunction, hypogonadism and visual impairment. Clinical features of Alström syndrome may include progressive visual and auditory impairment, insulin resistance and Type 2 diabetes, hyperlipidemia, progressive kidney dysfunction, cardiomyopathy and short stature in adulthood. Insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life may be common in people living with either BBS or Alström syndrome. There is great variability in presentation and severity of these symptoms across individuals with BBS or Alström syndrome. In the United States, the Company estimates that BBS affects approximately 2,500 people and that Alström syndrome affects approximately 500 people. Currently, there are no approved therapies targeting the melanocortin-4 receptor (MC4R) pathway for reducing body weight and hunger in BBS or Alström syndrome.

    About the Pivotal Phase 3 Trial
    The pivotal Phase 3 trial is designed to evaluate setmelanotide, a MC4R agonist, for the treatment of obesity and hyperphagia in people with BBS or Alström syndrome (NCT03746522). The trial is designed to enroll 30 patients, including at least 20 patients with BBS and at least six patients with Alström syndrome, aged six years and older. Participants were randomized to placebo or setmelanotide for 14 weeks followed by an open-label period on setmelanotide for 52 weeks. The primary endpoint of the trial is the proportion of participants (≥12 years of age) who achieve at least 10 percent reduction in body weight from baseline at approximately 52 weeks of therapy. Rhythm expects to report topline data from this trial in the fourth quarter of 2020 or early in the first quarter of 2021.

    About Rhythm
    Rhythm is a biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. The company recently announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity, and plans to complete its first rolling New Drug Application (NDA) submission to the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2019 or the first quarter of 2020. Rhythm is also evaluating setmelanotide in a pivotal Phase 3 trial in people living with BBS and Alström syndrome, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties, including statements regarding Rhythm's anticipated timing for enrollment of patients in clinical trials and submission of an NDA, its expectations regarding addressable patient populations, its expectations regarding the timing of topline data from its clinical trials, and its ongoing efforts related the efficacy of setmelanotide in patients with POMC deficiency obesity, LEPR deficiency obesity, BBS syndrome and Alström syndrome. Statements using word such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward looking statements. Such statements are subject to numerous risks and uncertainties, including but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our sufficiency of cash and our expenses, and other risks as may be detailed from time to time in our Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q and other reports we file with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:
    David Connolly
    Head of Investor Relations and Corporate Communications
    Rhythm Pharmaceuticals, Inc.
    857-254-4489

    Investor Contact:
    Hannah Deresiewicz
    Stern Investor Relations, Inc.
    212-362-1200

    Media Contact:
    Adam Daley
    Berry & Company Public Relations
    212-253-8881

    Primary Logo

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  42. BOSTON, Nov. 13, 2019 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that Hunter Smith, Chief Financial Officer of Rhythm, will present a corporate overview at the Stifel 2019 Healthcare Conference on Wednesday, November 20, 2019 at 1:50 p.m. ET in New York, NY.

    A live audio webcast of the presentation will be available under "Events & Presentations" in the Investors & Media section of the Company's website at www.rhythmtx.com. A replay of the webcast will be available on the Rhythm website for 30 days following each presentation.

    About Rhythm
    Rhythm is a biopharmaceutical company…

    BOSTON, Nov. 13, 2019 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that Hunter Smith, Chief Financial Officer of Rhythm, will present a corporate overview at the Stifel 2019 Healthcare Conference on Wednesday, November 20, 2019 at 1:50 p.m. ET in New York, NY.

    A live audio webcast of the presentation will be available under "Events & Presentations" in the Investors & Media section of the Company's website at www.rhythmtx.com. A replay of the webcast will be available on the Rhythm website for 30 days following each presentation.

    About Rhythm
    Rhythm is a biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. Rhythm is currently evaluating the efficacy and safety of setmelanotide, the company's MC4R agonist, in Phase 3 studies in patients with Pro-opiomelanocortin (POMC) deficiency obesity, Leptin receptor (LEPR) deficiency obesity, Bardet-Biedl syndrome, and Alström syndrome. The company is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Corporate Contact:
    David Connolly
    Head of Investor Relations and Corporate Communications
    Rhythm Pharmaceuticals, Inc.
    857-264-4280

    Investor Contact:
    Hannah Deresiewicz
    Stern Investor Relations, Inc.
    212-362-1200

    Media Contact:
    Adam Daley
    Berry & Company Public Relations
    212-253-8881

    Primary Logo

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  43. -- Data show treatment effect of setmelanotide on additional secondary endpoints, including body mass index -- 

    -- Additional safety analyses show no significant increases in heart rate or blood pressure --

    -- Two oral presentations delivered today during special research forum; poster presentations scheduled for Thursday --

    BOSTON, Nov. 04, 2019 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced additional data from its two pivotal Phase 3 clinical trials evaluating setmelanotide for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency…

    -- Data show treatment effect of setmelanotide on additional secondary endpoints, including body mass index -- 

    -- Additional safety analyses show no significant increases in heart rate or blood pressure --

    -- Two oral presentations delivered today during special research forum; poster presentations scheduled for Thursday --

    BOSTON, Nov. 04, 2019 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced additional data from its two pivotal Phase 3 clinical trials evaluating setmelanotide for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity showing the effect of setmelanotide on body mass index (BMI) scores and certain cardiovascular parameters. These data are being presented by study investigators in a special, late-breaking research forum during the 37th Annual Meeting of The Obesity Society at ObesityWeek® 2019, held November 3-7, 2019 in Las Vegas.

    In August 2019, Rhythm announced that both Phase 3 clinical trials of setmelanotide met their primary endpoints and all key secondary endpoints, demonstrating a statistically significant and clinically meaningful effect on weight loss and reductions in insatiable hunger, or hyperphagia, in patients with POMC deficiency obesity and LEPR deficiency obesity over the course of one year on setmelanotide treatment. Data from both trials also show that when patients withdrew from setmelanotide during a four-week placebo withdrawal period, they experienced rapid increases in weight and hunger. Setmelanotide is a melanocortin-4 receptor (MC4R) agonist designed to target impairments in the central melanocortin pathway, which is known to regulate weight and hunger.

    "Setmelanotide has demonstrated a statistically significant and clinically meaningful impact on the severe obesity and unrelenting hunger in patients living with POMC deficiency obesity or LEPR deficiency obesity," said Murray Stewart, M.D., Chief Medical Officer of Rhythm Pharmaceuticals. "With these supporting data, we are also demonstrating that setmelanotide may have additional therapeutic benefits, driving improvements in BMI and other parameters that are critical to overall health. We are excited to present these data during ObesityWeek, where we are also collaborating with obesity experts to raise awareness of the unmet needs among people living with rare genetic disorders of obesity and to attempt to change the treatment paradigm to support patient identification and earlier diagnosis."

    This week's presentations include new data showing the effect of setmelanotide on BMI scores for patients older than 19 and BMI z-scores for patients younger than 19, and its effect on vital signs including diastolic blood pressure, systolic blood pressure and heart rate. Additional analyses of safety data from the Phase 3 trials in POMC deficiency obesity and and LEPR deficiency obesity continue to support that setmelanotide is generally well-tolerated.

    ObesityWeek Oral Presentations

    Efficacy and Safety of the MC4R Agonist Setmelanotide in POMC Deficiency Obesity: A Phase 3 Trial

    Peter Kühnen, M.D., Institute for Experimental Pediatric Endocrinology, Charité Universitätsmedizin Berlin, Germany, presented data from Rhythm's Phase 3 POMC deficiency obesity trial.

    "Like many people living with rare genetic disorders of obesity, people with POMC deficiency obesity experience early-onset, rapid weight gain and severe, insatiable hunger, which are the two hallmarks of these disorders," said Dr. Kühnen, the lead investigator for this trial. "Despite significant effort to control their weight and appetite, such as supportive care or lifestyle interventions, patients often regain weight after any short-term period of weight loss. By reducing the weight gain and hunger endemic to POMC deficiency obesity, setmelanotide has the potential to shift the treatment paradigm for these patients."

    Setmelanotide was associated with reductions in BMI and BMI z-scores1 for patients with POMC deficiency obesity who were treated with setmelanotide for over one year at therapeutic dose:

    POMC deficiency obesity

      Baseline ~1 year at therapeutic dose Percent change from baseline 
    Participants aged ≥19 years, mean (SD) BMI, kg/m2 (n=4) 43.90 (8.91) 34.58 (12.42) -22.33 (14.75)
    P=0.056
    Participants aged <19 years, mean (SD) BMI z-score (n=6) 3.35 (0.61) 1.73 (1.04) -49.18 (27.20)
    P=0.007

    Consistent with prior clinical experience, setmelanotide was well tolerated in patients with POMC deficiency obesity. There were no reported cardiovascular adverse events (AEs) related to setmelanotide, and no AEs or serious AEs that led to treatment discontinuation or death. Setmelanotide was not associated with significant changes to blood pressure or heart rate:

    Mean parameter (SD)2 Diastolic blood pressure (mmHg)3 Systolic blood pressure (mmHg) Heart rate (beats/min)
    Baseline 73.13 (10.75) 111.57 (7.78) 81.03 (12.08)
    ~1 year at therapeutic dose 71.50 (9.17) 109.83 (6.12) 75.37 (7.25)
    Percent change from baseline, %

    P value
    -1.81 (6.27)

    P=0.38
    -1.36 (5.10)

    P=0.42
    -5.85 (11.44)

    P=0.14

    Efficacy and Safety of the MC4R Agonist Setmelanotide in LEPR Deficiency Obesity: A Phase 3 Trial

    Erica Van Den Akker, M.D., Ph.D., Erasmus MC-Sophia Children's Hospital University in Rotterdam, Netherlands, presented data from Rhythm's Phase 3 LEPR deficiency obesity trial.

    "Broadly, we saw clinically meaningful, significant weight loss in patients with LEPR deficiency obesity in the Phase 3 trial. Notably, after over a year on therapeutic dose of setmelanotide, two patients experienced roughly fifteen percent weight loss, while two other patients showed greater than twenty percent weight loss," said Dr. Van Den Akker, lead investigator in the LEPR deficiency obesity trial. "Weight loss of this magnitude is unprecedented in the natural history of this patient population, and strongly suggests that setmelanotide has the potential to restore MC4R pathway function and serve as a safe, effective therapy for patients with rare genetic disorders of obesity."

    Setmelanotide was associated with reductions in BMI and BMI z-scores4 for patients with LEPR deficiency obesity who were treated with setmelanotide for over one year at therapeutic dose:

    LEPR deficiency obesity

      Baseline ~1 year at therapeutic dose Percent change from baseline 
    Participants aged ≥19 years, mean (SD) BMI, kg/m2 (n=8) 51.18 (10.67) 45.82 (11.48)5 -10.59 (8.11)
    P=0.01
    Participants aged <19 years, mean (SD) BMI z-score (n=3) 3.52 (0.36) 3.03 (0.08) -13.35 (8.87)
    P=0.12

    Consistent with prior clinical experience, setmelanotide was well tolerated in patients with LEPR deficiency obesity. One participant discontinued therapy due to mild, treatment-related hypereosinophilia and one participant died from injuries as a passenger in a car accident, which was unrelated to the study drug. There were no reported cardiovascular AEs related to setmelanotide, and setmelanotide was not associated with significant changes in blood pressure or heart rate:

    Mean parameter (SD)6 Diastolic blood pressure (mmHg) Systolic blood pressure (mmHg) Heart rate (beats/min)
    Baseline 67.67 (5.83) 121.697 (8.84) 79.46 (12.60)
    ~1 year at therapeutic dose 66.48 (8.59) 115.111 (14.57) 77.89 (16.46)
    Percent change from baseline, %

    P value
    -1.58 (13.038)

    P=0.73
    -3.78 (9.94)

    P=0.29
    -1.32 (15.46)

    P=0. 80

    Poster Presentations

    Rhythm will present additional data on glucose and lipid parameters from the Phase 3 trials in POMC and LEPR deficiency obesities at ObesityWeek during the Pharmacotherapy Late Breaking Poster Session on Thursday, Nov. 7, from 12 noon - 1:30 p.m. PT (3 p.m. - 4:30 p.m. ET) in Mandalay Bay's Shoreline Exhibit Hall.

    In addition, a third poster, "Functional Characterization of Missense Variants in LEPR, POMC, & PCSK1 Genes Arising From Single Nucleotide Variant (SNV)," will be presented during the CNS Poster Session on Tuesday, Nov. 5, 12 noon - 1:30 p.m. PT (3 p.m. - 4:30 p.m. ET), in the Shoreline Exhibit Hall.

    Rhythm is on track to complete submission of a rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for POMC deficiency obesity and LEPR deficiency obesity in the fourth quarter of 2019 or the first quarter of 2020, and the Company expects to share additional data in forthcoming publications and medical meeting presentations.

    About Setmelanotide
    Setmelanotide is a potent MC4R agonist in development for the treatment of rare genetic disorders of obesity. Setmelanotide activates MC4R, part of the key biological pathway that independently regulates energy expenditure and appetite. Variants in genes within the MC4R pathway are associated with unrelenting hunger and severe, early-onset obesity. Rhythm is currently developing setmelanotide as a replacement therapy for patients with monogenic defects upstream of MC4R, for whom there are no effective or approved therapies. The FDA has granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway, which includes POMC deficiency obesity, LEPR deficiency obesity, Bardet-Biedl syndrome and Alström syndrome. The European Medicines Agency has also granted PRIority MEdicines (PRIME) designation for setmelanotide for the treatment of obesity and the control of hunger associated with deficiency disorders of the MC4R pathway.

    About Rhythm
    Rhythm is a biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. In addition to POMC deficiency obesity and LEPR deficiency obesity, Rhythm also is evaluating setmelanotide in a pivotal Phase 3 study in patients with Bardet-Biedl syndrome and Alström syndrome, and expects to complete enrollment in the second half of 2019. The company is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties, including statements regarding Rhythm's anticipated timing for enrollment of patients in clinical trials and submission of an NDA, its expectations regarding the impact of setmelanotide on BMI, heart rate, and blood pressure, its ongoing efforts related the efficacy of setmelanotide in patients with POMC deficiency obesity, LEPR deficiency obesity, Bardet-Biedl syndrome and Alström syndrome,. Statements using word such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward looking statements. Such statements are subject to numerous risks and uncertainties, including but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, and expenses, and other risks as may be detailed from time to time in our Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other reports we file with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

    Corporate Contact:
    David Connolly
    Head of Investor Relations and Corporate Communications
    Rhythm Pharmaceuticals, Inc.
    857-264-4280

    Investor Contact:
    Hannah Deresiewicz
    Stern Investor Relations, Inc.
    212-362-1200

    Media Contact:
    Adam Daley
    Berry & Company Public Relations
    212-253-8881


    1 BMI z-score, or BMI standard deviation scores, are measures of relative weight adjusted for child age and sex.
    2 N=10 for all POMC vital signs
    3 mmHG, millimeter of mecury
    4 BMI z-score, or BMI standard deviation scores, are measures of relative weight adjusted for child age and sex.
    5 N=7; one participant discontinued due to treatment-related adverse event.
    6 N=9 for all LEPR vital signs

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  44. -- Announced Positive Topline Results from Pivotal Phase 3 Clinical Trials Evaluating Setmelanotide in POMC and LEPR Deficiency Obesities --

    -- Added Four New MC4R Pathway Obesity Indications to Phase 2 Basket Study and Enrolling Patients --

    -- Announced Genetic Sequencing Data Supporting Ultra-Rarity of POMC and LEPR Deficiency Obesities and Suggesting U.S. Prevalence for Four New Basket Indications of &gt; 60,000 --

    -- Successfully Completed $172.5 Million Public Offering --

    BOSTON, Nov. 01, 2019 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company focused on the development and commercialization of therapeutics for the treatment of rare genetic disorders of obesity, today reported financial results and…

    -- Announced Positive Topline Results from Pivotal Phase 3 Clinical Trials Evaluating Setmelanotide in POMC and LEPR Deficiency Obesities --

    -- Added Four New MC4R Pathway Obesity Indications to Phase 2 Basket Study and Enrolling Patients --

    -- Announced Genetic Sequencing Data Supporting Ultra-Rarity of POMC and LEPR Deficiency Obesities and Suggesting U.S. Prevalence for Four New Basket Indications of > 60,000 --

    -- Successfully Completed $172.5 Million Public Offering --

    BOSTON, Nov. 01, 2019 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company focused on the development and commercialization of therapeutics for the treatment of rare genetic disorders of obesity, today reported financial results and provided a business update for the third quarter ended September 30, 2019.

    "We believe Rhythm is well-positioned to make a significant impact on the lives of people living with MC4R pathway-driven disorders of obesity," said Keith Gottesdiener, M.D., Chief Executive Officer of Rhythm. "In August, we announced topline data from our first pivotal studies of setmelanotide, demonstrating a statistically significant and clinically meaningful impact on weight loss and hunger in patients with pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity, and we are on track to complete our New Drug Application (NDA) submission to the U.S. Food and Drug Administration (FDA) for these two indications in the fourth quarter of this year or first quarter of 2020."

    Dr. Gottesdiener continued, "These milestones mark a critical first step toward our goal of bringing setmelanotide to many patients affected by rare genetic disorders of obesity. In parallel, we are continuing our community-building efforts to raise awareness, increase understanding of, and identify patients with these disorders, all of which are supported by the programs under our Rhythm Engine. We expect to complete enrollment in our pivotal Phase 3 trial evaluating setmelanotide for the treatment of severe obesity and hunger in patients with Bardet-Biedl syndrome (BBS) and Alström syndrome before year-end, and are enrolling patients with additional indications in our Phase 2 Basket Study, including SRC1 deficiency obesity, SH2B1 deficiency obesity, MC4R deficiency obesity and Smith-Magenis syndrome. Following our successful public offering in October, we believe that we are well-capitalized and have sufficient resources to advance our ongoing clinical and commercial efforts though at least the end of 2021."

    Third Quarter and Recent Business Highlights:

    Pipeline:

    • In September 2019, Rhythm announced the expansion of its Phase 2 Basket Study of setmelanotide to include four additional melanocortin-4 receptor (MC4R) pathway obesity disorders: SRC1 deficiency obesity, SH2B1 deficiency obesity, MC4R deficiency obesity and Smith-Magenis syndrome.
    • In September 2019, Rhythm announced results from genetic sequencing of 13,567 individuals with severe obesity yields 11.7 percent (1,584 individuals) who have a rare genetic variant within the MC4R pathway eligible for enrollment in the Phase 2 Basket Study. These sequencing data support the ultra-rarity of POMC deficiency obesity and LEPR deficiency obesity and suggest an aggregate U.S. prevalence for the four new indications of approximately greater than 60,000 patients.
    • In September 2019, Rhythm shared updated data from patients with BBS and Alström syndrome who are continuing to receive setmelanotide treatment in the Phase 2 Basket Study, demonstrating continued treatment effect in patients with BBS and Alström syndrome following nearly two years, and greater than one year, of treatment, respectively.
    • In August 2019, Rhythm announced positive topline results from its two pivotal Phase 3 clinical trials evaluating setmelanotide for the treatment of POMC and LEPR deficiency obesities. Both trials met their primary endpoints and all key secondary endpoints, demonstrating a statistically significant and clinically meaningful effect on weight loss and reductions in insatiable hunger, or hyperphagia, in patients with POMC and LEPR deficiency obesities.

    Corporate:

    • In October 2019, Rhythm completed a public offering of 9,324,324 shares of its common stock at a public offering price of $18.50 per share, for aggregate gross proceeds of approximately $172.5 million, before underwriting discounts, commissions, and offering expenses.

    Upcoming Milestones:

    • Rhythm plans to present additional data from its two pivotal Phase 3 trials of setmelanotide in POMC deficiency obesity and LEPR deficiency obesity on November 4, 2019 at The Obesity Society's ObesityWeek in Las Vegas.
    • Rhythm remains on track to complete submission of its rolling NDA to the FDA for setmelanotide in patients with POMC deficiency obesity and LEPR deficiency obesity in the fourth quarter of 2019 or the first quarter of 2020.
    • Rhythm expects to report topline data from its combined Phase 3 trial evaluating setmelanotide in BBS and Alström syndrome in 2020.
    • Rhythm expects to announce additional data from its ongoing Phase 2 Basket Study of setmelanotide in high-impact heterozygous (HET) obesity and may announce preliminary data from other Phase 2 Basket Study indications in 2020.
    • Rhythm expects to submit an investigational new drug (IND) application for RM-853, its ghrelin o-acyltransferase (GOAT) inhibitor for the treatment of Prader-Willi Syndrome, to the FDA in 2020. 

    Third Quarter 2019 Financial Results:

    • Cash Position: As of September 30, 2019, cash, cash equivalents and short-term investments were $162.4 million, as compared to $195.2 million as of June 30, 2019. This decrease reflects $33.6 million of cash used to fund operating activities in the third quarter of 2019. Cash, cash equivalents and short-term investments as of September 30, 2019 do not include aggregate gross proceeds of approximately $172.5 million from the Company's October 2019 public offering.
    • Based on its current clinical development plans and taking into account its recent public offering, Rhythm expects that its existing cash, cash equivalents and short-term investments will enable it to fund its operations through at least the end of 2021.
    • R&D Expenses: R&D expenses were $26.6 million for the third quarter of 2019, as compared to $10.7 million for the third quarter of 2018. This increase was primarily due to an increase of $10.2 million related to Rhythm's clinical trials, including an expansion of the GO-ID genotyping study and the Phase 2 Basket Study with new trial sites for both studies, as well as ongoing enrollment in the Phase 3 study of setmelanotide in patients with BBS and Alström syndrome; an increase of $3.4 million related to translational research and genetic sequencing efforts designed to improve identification of patients with MC4R pathway deficiencies and pathway validation efforts; and an increase of  $1.5 million due to the hiring of additional personnel related to community building and education efforts for physicians, care providers and patients who are facing rare genetic disorders of obesity.
    • S,G&A Expenses: S,G&A expenses were $10.5 million for the third quarter of 2019, as compared to $8.5 million for the third quarter of 2018. This is due primarily to an increase of $1.9 million in employee related costs in connection with the hiring of additional full-time employees to support planned commercial activities, operations and the continued build of finance and human resource functions.
    • Net Loss: Net loss was $36.0 million for the third quarter of 2019, or a net loss per basic and diluted share of $1.04, as compared to a net loss of $17.7 million for the third quarter of 2018, or a net loss per basic and diluted share of $0.52.

    Year to Date Financial Results:

    • Cash Position: As of September 30, 2019, cash, cash equivalents and short-term investments were $162.4 million, as compared to $252.1 million as of December 31, 2018. This decrease reflects $90.5 million of cash used to fund operating activities in 2019.
    • R&D Expenses: R&D expenses were $84.6 million for the nine months ended September 30, 2019, as compared to $31.6 million for the nine months ended September 30, 2018. The increase was primarily due to an increase of $30.0 million related to Rhythm's clinical trials associated with setmelanotide, including an expansion of the GO-ID genotyping study and the Phase 2 Basket Study with new trial sites for both studies, as well as ongoing enrollment in the Phase 3 study of setmelanotide in patients with BBS and Alström syndrome; an increase of $11.0 million related to translational research and genetic sequencing efforts designed to improve identification of patients with MC4R pathway deficiencies and pathway validation efforts; an increase of  $6.2 million due to the hiring of additional full-time employees in order to support efforts for community building and education efforts for physicians, care providers and patients who are facing rare genetic disorders of obesity, as well as to support the growth of Rhythm's research and development programs; an increase of $5.1 million primarily related to purchases of setmelanotide active pharmaceutical ingredient (API) for clinical trials, commercial scale up and pre-IND work for RM-853; and an increase of $3.0 million in consulting and professional services associated with the creation of Rhythm's EU Medical Science Liaison field force, various medical communication programs and support for Rhythm's NDA filing. These increases were partially offset by a decrease of $4.4 million due to a non-cash expense related to the license acquired from Takeda for RM-853 in March 2018.
    • S,G&A Expenses: S,G&A expenses were $27.1 million for the nine months ended September 30,  2019, as compared to $19.7 million for the nine months ended September 30, 2018. The increase was primarily due to  an increase of $5.6 million in employee related costs in connection with the hiring of additional full-time employees to support planned commercial activities, operations and the continued build of finance and human resource functions; and an increase of $2.1 million in various consulting and professional services related to efforts to drive disease awareness about rare genetic causes of obesity and prepare for the potential commercial launch of setmelanotide in the U.S.
    • Net Loss: Net loss was $107.8 million for the nine months ended September 30, 2019, or a net loss per basic and diluted share of $3.13, as compared to a net loss of $48.6 million for the nine months ended September 30, 2018, or a net loss per basic and diluted share of $1.63.

    About Rhythm Pharmaceuticals

    Rhythm is a biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. The company recently announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in patients with POMC deficiency obesity and LEPR deficiency obesity, and Rhythm expects to share additional data in forthcoming publications and medical meeting presentations. The company plans to complete its first rolling NDA submission to the FDA in the fourth quarter of 2019 or the first quarter of 2020. Rhythm is also evaluating setmelanotide in a pivotal Phase 3 study in patients with BBS and Alström syndrome and expects to complete enrollment in the second half of 2019. The company is leveraging the Rhythm Engine -- comprised of its Phase 2 Basket Study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.

    Forward-looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties, including statements regarding Rhythm's anticipated timing for enrollment and design of clinical trials, the timing for filing of a new drug application and submission of an investigational new drug application, its ongoing efforts related to patient identification, the release of results of clinical trials, and its sufficiency of cash. Statements using word such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward-looking statements.  Such statements are subject to numerous risks and uncertainties, including but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, and expenses, and other risks as may be detailed from time to time in our Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q and other reports we file with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

     
    Rhythm Pharmaceuticals, Inc.
    Condensed Consolidated Balance Sheets
    (in thousands, except share and per share data)
    (Unaudited)
               
        September 30,    December 31, 
        2019
      2018
                 
    Assets            
    Current assets:            
    Cash and cash equivalents   $ 71,680     $ 49,542  
    Short-term investments     90,755       202,519  
    Prepaid expenses and other current assets     8,442       6,628  
    Total current assets     170,877       258,689  
    Property and equipment, net     3,862       1,120  
    Right-of-use asset     2,097        
    Deferred issuance costs     295        
    Restricted cash     402       401  
    Total assets   $ 177,533     $ 260,210  
    Liabilities and stockholders' equity            
    Current liabilities:            
    Accounts payable   $ 7,745     $ 7,640  
    Accrued expenses and other current liabilities     17,005       5,942  
    Lease liability     457        
    Total current liabilities     25,207       13,582  
    Long-term liabilities:            
    Lease liability     3,211        
    Deferred rent           372  
    Total liabilities     28,418       13,954  
    Commitments and contingencies            
    Stockholders' equity:            
    Preferred Stock, $0.001 par value: 10,000,000 shares authorized; no shares issued and outstanding at September 30, 2019 and December 31, 2018, respectively            
    Common stock, $0.001 par value: 120,000,000 shares authorized; 34,578,564 and 34,410,725 shares issued and outstanding September 30, 2019 and December 31, 2018, respectively     35       34  
    Additional paid-in capital