RYTM Rhythm Pharmaceuticals Inc.

32.61
+1.66  (+5%)
Previous Close 30.95
Open 31.18
52 Week Low 12.99
52 Week High 34.2735
Market Cap $1,441,516,734
Shares 44,204,745
Float 30,822,928
Enterprise Value $1,169,563,857
Volume 926,416
Av. Daily Volume 268,301
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Upcoming Catalysts

Drug Stage Catalyst Date
Setmelanotide
Alstrom Syndrome and Bardet-Biedl Syndrome
Phase 3
Phase 3
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Setmelanotide (basket)
POMC Heterozygous Deficiency Obesity, Alstrom Syndrome, POMC Epigenetic Disorders
Phase 2
Phase 2
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Drug Pipeline

Drug Stage Notes
Setmelanotide
POMC deficiency obesity / Leptin Receptor Deficiency Obesity
Approved
Approved
FDA approval announced November 27, 2020.

Latest News

  1. -- 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

    d

    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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  2. -- 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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  3. -- 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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  4. 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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  5. 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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