MIRM Mirum Pharmaceuticals Inc.

23.87
+0.42  (+2%)
Previous Close 23.45
Open 23.98
52 Week Low 7.51
52 Week High 28.31
Market Cap $613,861,854
Shares 25,716,877
Float 12,180,929
Enterprise Value $472,715,765
Volume 68,295
Av. Daily Volume 56,898
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Upcoming Catalysts

Drug Stage Catalyst Date
Maralixibat
Alagille Syndrome (ALGS)
NDA Filing
NDA Filing
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Drug Pipeline

Drug Stage Notes
Maralixibat - MARCH
Progressive Familial Intrahepatic Cholestasis (PFIC)
Phase 3
Phase 3
Phase 3 enrollment to be completed 2Q 2021.
Maralixibat
Biliary atresia
Phase 2
Phase 2
Phase 2 study initiation planned by 1Q 2021.
Volixibat
Primary sclerosing cholangitis
Phase 2
Phase 2
Phase 2 trial planned for 1Q 2021.
Volixiba
Intrahepatic cholestasis of pregnancy
Phase 2
Phase 2
Phase 2 trial planned for 1Q 2021.

Latest News

  1. - Five-year transplant-free survival data from the Phase 2 INDIGO study used as the basis for the MAA submission

    - Maralixibat would be the first treatment labeled for patients with PFIC2 in Europe, if approved

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced that the company's Marketing Authorization Application (MAA) for its investigational medicine, maralixibat, for the treatment of patients with progressive familial intrahepatic cholestasis type 2 (PFIC2), also known as bile salt export pump (BSEP) deficiency, was accepted for review (validated) by the European Medicines Agency (EMA). The validation…

    - Five-year transplant-free survival data from the Phase 2 INDIGO study used as the basis for the MAA submission

    - Maralixibat would be the first treatment labeled for patients with PFIC2 in Europe, if approved

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced that the company's Marketing Authorization Application (MAA) for its investigational medicine, maralixibat, for the treatment of patients with progressive familial intrahepatic cholestasis type 2 (PFIC2), also known as bile salt export pump (BSEP) deficiency, was accepted for review (validated) by the European Medicines Agency (EMA). The validation of the application by the EMA confirms all essential regulatory elements are included in the submission such that the EMA can begin its review.

    "PFIC is life-altering for patients and their families as they struggle to manage the round-the-clock care and surgical decisions that many children often need," said Chris Peetz, president and chief executive officer at Mirum. "Validating our MAA is a groundbreaking step towards providing a medicine to address PFIC2. Based on the long-term transplant-free survival improvement in maralixibat responders, we believe that maralixibat could provide a treatment alternative to invasive surgeries for these patients, as well as improve quality of life. We are excited about the opportunity to make maralixibat available to patients with PFIC2 in Europe."

    Data from the Phase 2 INDIGO study evaluating maralixibat for pediatric patients with PFIC2 served as the basis of the MAA submission. Mirum recently announced data showing five-year transplant-free survival for patients who achieved serum bile acid control. The data also demonstrated improvements across multiple parameters including pruritus control, improvements of liver enzyme and bilirubin levels, and improvement in growth. These data were presented at the annual meeting of the European Association for the Study of the Liver. The MAA submission also includes data on five-year event-free survival with maralixibat compared to the NAPPED natural history cohort.

    To provide further evidence of maralixibat's potential in PFIC2 with higher doses and other PFIC subtypes, Mirum is conducting a Phase 3 study, MARCH, with completion of enrollment expected in the second quarter of 2021.

    In addition to the MAA submission for maralixibat in PFIC2, Mirum has also initiated a rolling new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for maralixibat for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS). Mirum expects to complete the submission in the first quarter of 2021, with a planned launch in the second half of the same year. The company also recently launched an Expanded Access Program making maralixibat available to eligible patients with ALGS in the United States, Canada, Australia, and certain countries in Europe.

    About Maralixibat

    Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several cholestatic liver diseases. Maralixibat inhibits the apical sodium-dependent bile acid transporter (ASBT), resulting in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thereby potentially reducing bile acid mediated liver damage and related effects and complications. More than 1,600 individuals have received maralixibat, including more than 120 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). In the ICONIC Phase 2b ALGS clinical trial, patients taking maralixibat had significant reductions in bile acids and pruritus compared to placebo, as well as reduction in xanthomas and accelerated growth long-term. In a Phase 2 PFIC study, a genetically defined subset of BSEP deficient (PFIC2), patients responded to maralixibat. The U.S. Food and Drug Administration (FDA) has granted maralixibat Breakthrough Therapy designation for treatment of pruritus associated with ALGS in patients one year of age and older and for PFIC2. Maralixibat was generally well-tolerated throughout the studies. The most frequent treatment-related adverse events were diarrhea and abdominal pain. Until maralixibat is approved by regulatory authorities and available for prescribing, the medication is available to patients with ALGS through Mirum's expanded access program. For more information, please visit ALGSEAP.com. For more information about the Phase 3 study for maralixibat in pediatric patients with PFIC, visit PFICtrial.com.

    About Mirum Pharmaceuticals

    Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. The company's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. The Company has initiated a rolling NDA submission for maralixibat in the treatment of cholestatic pruritus in patients with ALGS and expects to complete the submission in the first quarter of 2021. Additionally, Mirum's marketing authorization application for the treatment of pediatric patients with PFIC2 has been accepted for review (validated) by the European Medicines Agency.

    Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis and intrahepatic cholestasis of pregnancy. For more information, visit MirumPharma.com.

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    Forward-Looking Statements

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the results, conduct and progress of Mirum's ongoing studies for maralixibat and the regulatory approval path for maralixibat. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "plans," "will," "may," "expects," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum's business in general, the impact of the COVID-19 pandemic, and the other risks described in Mirum's filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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  2. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced that the company will participate in the following upcoming virtual investor conferences.

    Piper 32nd Annual Virtual Healthcare Conference
    Fireside Chat will be available for viewing on November 23 at 10:00am ET. Management will participate in one-on-one meetings on December 1.

    Evercore ISI 3rd Annual HealthCONx Conference
    Presentation day and time: Thursday, December 3, 2020 at 12:35pm ET

    Audio and video webcasts and archive of the presentations will be available in the Investors & Media section of the company website at ir.mirumpharma.com.

    About Mirum

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced that the company will participate in the following upcoming virtual investor conferences.

    Piper 32nd Annual Virtual Healthcare Conference

    Fireside Chat will be available for viewing on November 23 at 10:00am ET. Management will participate in one-on-one meetings on December 1.

    Evercore ISI 3rd Annual HealthCONx Conference

    Presentation day and time: Thursday, December 3, 2020 at 12:35pm ET

    Audio and video webcasts and archive of the presentations will be available in the Investors & Media section of the company website at ir.mirumpharma.com.

    About Mirum Pharmaceuticals

    Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. The company's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. The company has initiated a rolling NDA submission for maralixibat in the treatment of patients with cholestatic pruritus associated with ALGS and expects to complete the submission in the first quarter of 2021. Additionally, the company plans to submit a marketing authorization application to the European Medicines Agency for maralixibat in the treatment of patients with PFIC2 in the fourth quarter 2020.

    The company is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis and intrahepatic cholestasis of pregnancy. For more information, visit MirumPharma.com.

    Follow Mirum on Twitter, Facebook and LinkedIn.

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  3. - Results of long-term treatment from the ITCH and IMAGINE II studies presented in late-breaking oral presentation at AASLD

    - Data demonstrates significant and durable improvements in pruritus in children with Alagille syndrome through 220 weeks of maralixibat treatment

    - Results have been included as supportive data in ongoing rolling NDA submission for treatment of cholestatic pruritus in patients with Alagille syndrome

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, announced data presented at the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD) - The Liver Meeting Digital Experience™…

    - Results of long-term treatment from the ITCH and IMAGINE II studies presented in late-breaking oral presentation at AASLD

    - Data demonstrates significant and durable improvements in pruritus in children with Alagille syndrome through 220 weeks of maralixibat treatment

    - Results have been included as supportive data in ongoing rolling NDA submission for treatment of cholestatic pruritus in patients with Alagille syndrome

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, announced data presented at the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD) - The Liver Meeting Digital Experience™. The data were presented by Dr. Benjamin Shneider of Baylor College of Medicine and Texas Children's Hospital presenting on behalf of the Childhood Liver Disease Research Network (ChiLDReN) in a late-breaking oral presentation titled "Preliminary Analysis of ITCH and IMAGINE II – Outcome of Long-term Administration of Maralixibat in Children with Alagille Syndrome."

    The objective of the studies was to assess pruritus and other markers of cholestasis in patients with Alagille syndrome (ALGS) with up to 220 weeks of treatment with maralixibat. Maralixibat, an apical sodium bile acid transporter (ASBT) inhibitor, has previously been shown to interrupt the enterohepatic circulation of bile acids, reducing pruritus.

    Of the children enrolled in the ITCH and IMAGINE II studies, 28 of the 37 patients were on study at 48 weeks with 80% of those experiencing clinically meaningful reductions in pruritus (ItchRO[Obs] reduction ≥1.0 point) which were durable beyond four years (Week 220), with 90% of patients who continued on study experiencing a pruritus response at the end of treatment. The mean reduction in ItchRO(Obs) at week 48 was -1.9 points and deepened to -2.3 points at the end of treatment. Maralixibat treatment improved quality of life and led to improved growth parameters. The long-term data suggest that maralixibat has the potential to be an effective treatment and could serve as an alternative to surgery for ALGS patients, if approved.

    "Maralixibat has the potential to address the severe pruritus experienced by children with Alagille syndrome, resulting in meaningful improvement in quality of life, meeting a major unmet need for this patient population," said Benjamin L. Shneider, M.D., principal study investigator and member of the Childhood Liver Disease Research Network, funded largely by the National Institute of Diabetes and Digestive and Kidney Diseases. "Alagille syndrome often leads to progressive liver disease frequently complicated by severe pruritus, often disrupting sleep, interfering with school, and impacting the quality of life for children and their families so greatly that surgical intervention via external biliary diversion or liver transplant are required. These data show that with maralixibat, we have the potential to positively impact children's health across multiple clinical measures, without resorting to invasive surgeries which can be associated with significant complications and potential mortality."

    To view the presentation and the complete data, please visit the AASLD section within the Events page on Mirum's website.

    "These results from the study confirm the potential for maralixibat to address the consequences of cholestasis in Alagille syndrome, supporting the effects seen in the long term analysis of the pivotal ICONIC study," said Chris Peetz, president and chief executive officer of Mirum. "As we progress toward completion of our rolling NDA submission, maralixibat is being offered to eligible patients with Alagille syndrome through an expanded access program until it is available for prescribing."

    About the ITCH and IMAGINE II study

    The ITCH study is a randomized, placebo-controlled study of maralixibat in children with ALGS. The IMAGINE II study is an open-label study for participants who completed the ITCH study. ITCH and IMAGINE II were conducted by ChiLDReN in the context of a Cooperative Research and Development Agreement between Mirum Pharmaceuticals and the NIDDK. Thirty-seven children were enrolled into the ITCH study. Children receiving maralixibat in the studies also demonstrated improvements in biomarkers of disease, including reductions in cholesterol and bile acid levels. After over 4 years of treatment, maralixibat continues to be generally well tolerated. There were two serious adverse events possibly related to administration of maralixibat leading to drug withdrawal in both patients (hematochezia/anemia and autoimmune hepatitis).

    About Alagille Syndrome

    ALGS is a rare genetic disorder in which bile ducts are abnormally narrow, malformed and reduced in number, which leads to bile accumulation in the liver and ultimately progressive liver disease. The estimated incidence of ALGS is one in every 30,000 people.1 In patients with ALGS, multiple organ systems may be affected by the mutation, including the liver, heart, kidneys and central nervous system.2 The accumulation of bile acids prevents the liver from working properly to eliminate waste from the bloodstream and, according to recent reports, 60% to 75% of patients with Alagille syndrome have a liver transplant before reaching adulthood.3 Signs and symptoms arising from liver damage in ALGS may include jaundice (yellowing of the skin), xanthomas (disfiguring cholesterol deposits under the skin), and pruritus (itch)2. The pruritus experienced by patients with ALGS is among the most severe in any chronic liver disease and is present in most affected children by the third year of life.4

    About Maralixibat

    Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases. Maralixibat inhibits the apical sodium dependent bile acid transporter (ASBT), resulting in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thereby potentially reducing bile acid mediated liver damage and related effects and complications. More than 1,600 individuals have received maralixibat, including more than 120 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). In the ICONIC Phase 2b ALGS clinical trial, patients taking maralixibat had significant reductions in bile acids and pruritus compared to placebo, as well as reduction in xanthomas and accelerated growth long-term. In a Phase 2 PFIC study, a genetically defined subset of BSEP deficient (PFIC2), patients responded to maralixibat with an increase in transplant-free survival. The U.S. Food and Drug Administration has granted maralixibat Breakthrough Therapy designation for the treatment of pruritus associated with ALGS in patients one year of age and older and for PFIC2. Maralixibat was generally well-tolerated throughout the studies. The most frequent treatment-related adverse events were diarrhea and abdominal pain. Until maralixibat is approved and available for prescribing, the medication is available to patients with ALGS through Mirum's expanded access program. For more information, please visit ALGSEAP.com. For more information about the Phase 3 study for maralixibat in pediatric patients with PFIC, visit PFICtrial.com.

    About Mirum Pharmaceuticals

    Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. The company's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. The company has initiated a rolling NDA submission for maralixibat in the treatment of patients with cholestatic pruritus associated with ALGS and expects to complete the submission in the first quarter of 2021. Additionally, the company plans to submit a marketing authorization application to the European Medicines Agency for maralixibat in the treatment of patients with PFIC2 in the fourth quarter 2020.

    The company is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis and intrahepatic cholestasis of pregnancy. For more information, visit MirumPharma.com.

    Follow Mirum on Twitter, Facebook and LinkedIn.

    Forward-Looking Statements

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the results, conduct and progress of Mirum's ongoing and planned clinical studies for maralixibat and volixibat, the regulatory approval path for maralixibat, and the potential launch of maralixibat, if approved. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "plans," "expects," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum's business in general, the impact of the COVID-19 pandemic, and the other risks described in Mirum's filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

    The content in this release is the sole responsibility of the authors and does not necessarily represent the official views or imply endorsement of the National Institutes of Health.

    1Danks, et al. Archives of Disease in Childhood 1977

    2Johns Hopkins Medicine. hopkinsmedicine.org/health/conditions-and-diseases/Alagille-syndrome

    3Vandriel, et al. GALA EASL 2020; Kamath, et al. Hepatology Communications 2020

    4Elisofon, et al. Journal of Pediatric Gastroenterology and Nutrition 2010

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    • Maralixibat data highlights the relationship between the reduction of serum bile acids and pruritus intensity, improved growth, and other quality of life measures.
    • Analysis of the natural variability of pruritus in children with Alagille syndrome demonstrates persistent severity.
    • Volixibat data shows highly active dose levels selected for Phase 2 studies.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced new analyses from its maralixibat and volixibat clinical programs, as featured at the Annual Meeting of the American Association for the Study of Liver Diseases – The Liver Meeting Digital Experience™…

    • Maralixibat data highlights the relationship between the reduction of serum bile acids and pruritus intensity, improved growth, and other quality of life measures.
    • Analysis of the natural variability of pruritus in children with Alagille syndrome demonstrates persistent severity.
    • Volixibat data shows highly active dose levels selected for Phase 2 studies.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced new analyses from its maralixibat and volixibat clinical programs, as featured at the Annual Meeting of the American Association for the Study of Liver Diseases – The Liver Meeting Digital Experience™.

    A summary of findings from each of the poster presentations is below. To view the data in full, please visit the AASLD section within the Events page on our corporate website.

    "Data generated from six years of clinical evaluation of maralixibat in Alagille syndrome has offered key understandings in the severe burden of pruritus and related markers of cholestasis, helping to elucidate the potential benefit of maralixibat in this treatment setting," said Dr. Pam Vig, chief scientific officer at Mirum. "We are also excited to begin Phase 2 studies of volixibat in adult patients with cholestatic liver disease, which will evaluate dose levels shown to be effective in increasing bile acid excretion, suggesting the potential to reduce the burden of cholestasis in these settings."

    Abstract #341: Pruritus intensity is associated with cholestasis biomarkers and quality of life measures after maralixibat treatment in children with Alagille syndrome

    Alagille syndrome (ALGS)-associated pruritus is often extremely debilitating, resulting in bleeding, scarring, sleep disturbance, fatigue, and decreased quality of life, with significant impact on the patient and their family. This analysis, which utilized data from the maralixibat Phase 2b ICONIC study in pediatric patients with cholestatic pruritus associated with ALGS, evaluated how change in pruritus intensity is related to change in cholestasis markers and other clinical parameters. The study evaluated 31 patients, of which 27 were evaluated for this analysis over a 48-week period. Overall, the positive treatment effects of maralixibat in patients with ALGS demonstrate important correlations with multiple clinically relevant parameters. Pruritus, as measured by the fully validated Itch Reported Outcome Observer (ItchRO[Obs]) tool, was correlated with several parameters including the Clinician Scratch Scale, serum bile acids (sBA) and sBA subspecies, autotaxin, growth and quality of life measures, including fatigue.

    Abstract #1792: Natural variability of pruritus in Alagille syndrome; an analysis from the ICONIC study utilizing the Itch Reported Outcome Observer (ItchRO[Obs]) tool

    Pruritus is known to be one of the most burdensome symptoms associated with ALGS; however, the variability of its severity and frequency has not been fully established. Assessments rely on observer- or patient-reported outcomes measures. The ItchRO(Obs) and ItchRO(Pt) tools were used to assess the natural variability of pruritus in children with ALGS who were enrolled in the Phase 2b ICONIC, placebo-controlled, randomized study. The scores were assessed during the 28-day screening period of ICONIC, when no drug was administered. The results showed that both the morning and evening assessment of pruritus was persistent over time with minimal fluctuations in severity and frequency.

    Abstract #1221: A Phase 1 dose-ranging study assessing fecal bile acid excretion by volixibat, an apical sodium-dependent bile acid transporter inhibitor, and coadministration with loperamide

    The primary goal of this Phase 1 clinical study was to investigate the safety and efficacy of a range of dose levels and dose regimens of volixibat, to help guide dose selection for clinical trials in patients with cholestatic disease, in particular the planned Phase 2 programs in adult patients with primary sclerosing cholangitis and intrahepatic cholestasis of pregnancy. The study evaluated the effects of volixibat alone and in combination with loperamide, seeking to understand whether the addition of loperamide would reduce the mild gastrointestinal side effects that are often observed with ASBT inhibitors.

    The analysis found that volixibat was well-tolerated at all dose levels and regimens evaluated. As expected for the mechanisms of action of volixibat, fecal bile acid excretion was increased across all treatment groups. Overall, twice-daily dosing was associated with greater increases in bile acid excretion compared to once-daily dosing. Use of volixibat in adult healthy volunteers was associated with meaningful increases in fecal bile acid excretion and serum 7αC4, which are markers of bile acid synthesis. In addition, standard dosing of loperamide helped to reduce the mild and transient gastrointestinal disturbance during the initial dosing of volixibat, without a drug-drug interaction. The effects on bile acid trafficking and synthesis support the further study of volixibat in patients with cholestatic liver disease.

    About Maralixibat

    Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases. Maralixibat inhibits the apical sodium-dependent bile acid transporter (ASBT), resulting in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thereby potentially reducing bile acid mediated liver damage and related effects and complications. More than 1,600 individuals have received maralixibat, including more than 120 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). In the ICONIC Phase 2b ALGS clinical trial, patients taking maralixibat had significant reductions in bile acids and pruritus compared to placebo, as well as reduction in xanthomas and accelerated growth long-term. In a Phase 2 PFIC study, a genetically defined subset of BSEP deficient (PFIC2), patients responded to maralixibat. The U.S. Food and Drug Administration (FDA) has granted maralixibat Breakthrough Therapy designation for treatment of pruritus associated with ALGS in patients one year of age and older and for PFIC2. Maralixibat was generally well-tolerated throughout the studies. The most frequent treatment-related adverse events were diarrhea and abdominal pain. Until maralixibat is approved by the FDA and available for prescribing, the medication is available to patients with ALGS through Mirum's expanded access program. For more information, please visit ALGSEAP.com. For more information about the Phase 3 study for maralixibat in pediatric patients with PFIC, visit PFICtrial.com.

    About Volixibat

    Volixibat is an oral, minimally absorbed agent designed to selectively inhibit ASBT. Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking recycling of bile acids, through inhibition of the apical sodium dependent bile acid transporter (ASBT), thereby reducing bile acids systemically and in the liver. Phase 1 and Phase 2 clinical trials of volixibat demonstrated on-target fecal bile acid excretion, a pharmacodynamic marker of ASBT inhibition, in addition to decreases in LDL cholesterol and increases in 7αC4 which are markers of bile acid synthesis. Volixibat has been evaluated in more than 400 subjects across multiple clinical trials. The most common adverse events reported were mild to moderate gastrointestinal events observed in the volixibat groups.

    About Mirum Pharmaceuticals

    Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. The company's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. The company has initiated a rolling NDA submission for maralixibat in the treatment of patients with cholestatic pruritus associated with ALGS and expects to complete the submission in the first quarter of 2021. Additionally, the company plans to submit a marketing authorization application to the European Medicines Agency for maralixibat in the treatment of patients with PFIC2 in the fourth quarter 2020.

    The company is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis and intrahepatic cholestasis of pregnancy. For more information, visit MirumPharma.com. Follow Mirum on Twitter, Facebook and LinkedIn.

    Forward-Looking Statements

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the results, conduct and progress of Mirum's ongoing and planned studies for maralixibat and volixibat, and the regulatory approval path for maralixibat and volixibat. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "plans," "will," "may," "expects," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum's business in general, the impact of the COVID-19 pandemic, and the other risks described in Mirum's filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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  4. - Initiated rolling NDA submission and launched Expanded Access Program for maralixibat in Alagille syndrome.

    - Presented five-year transplant-free survival data for patients with PFIC2 at Digital International Liver Congress (EASL).

    - European Marketing Authorization Application submission for maralixibat in PFIC2, planned by year-end 2020.

    - Cash, cash equivalents and investments of $133.7 million.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced financial results and a corporate update for the quarter ended September 30, 2020.

    "This quarter marked several milestones toward providing better treatment…

    - Initiated rolling NDA submission and launched Expanded Access Program for maralixibat in Alagille syndrome.

    - Presented five-year transplant-free survival data for patients with PFIC2 at Digital International Liver Congress (EASL).

    - European Marketing Authorization Application submission for maralixibat in PFIC2, planned by year-end 2020.

    - Cash, cash equivalents and investments of $133.7 million.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced financial results and a corporate update for the quarter ended September 30, 2020.

    "This quarter marked several milestones toward providing better treatment options for Alagille syndrome and PFIC, with the initiation of our rolling NDA submission, the launch of an expanded access program, and presentation of five-year transplant free survival data in PFIC2," said Chris Peetz, president and chief executive officer of Mirum. "Looking forward to next year, we are planning for the U.S. launch of maralixibat in Alagille syndrome and the expansion of our programs, with upcoming study starts in biliary atresia, primary sclerosing cholangitis and intrahepatic cholestasis of pregnancy, all settings with high disease burden and no currently approved therapies."

    Key Operational Highlights

    • Presented five-year transplant-free survival data for patients with PFIC2 at Digital International Liver Congress (EASL).
    • Initiated rolling submission of New Drug Application (NDA) to U.S. Food and Drug Administration (FDA) for maralixibat for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS).
    • Launched maralixibat Expanded Access Program (EAP) for the treatment of cholestatic pruritus associated with ALGS in United States, Canada, Australia, and 10 countries in Europe.
    • Announced partnership with EVERSANA™ to support the planned launch and commercialization of maralixibat in ALGS in the United States, if approved.
    • Received Orphan Drug Designation from the U.S. FDA for maralixibat in biliary atresia; Phase 2 study initiation planned for the first quarter of 2021.
    • Received rare pediatric disease designation for maralixibat for the treatment of PFIC.

    Third Quarter 2020 Financial Results

    • Total operating expenses for the quarter ended September 30, 2020 were $21.7 million, compared to $15.9 million for the third quarter of 2019.
      • Research and development expenses were $16.0 million, compared to $12.2 million for the comparable prior-year period. This increase was primarily due to increased personnel related expenses, manufacturing activities to support Mirum's NDA, and higher consulting expenses.
      • General and administrative expenses were $5.7 million, compared to $3.7 million for the comparable prior-year period. The increase was primarily due to personnel and other compensation related expenses.
    • For the quarter ended September 30, 2020, Mirum reported a net loss of $21.5 million, or $0.86 per share, compared with a net loss of $15.1 million, or $0.84 per share for the same period in 2019.
    • As of September 30, 2020, Mirum had cash, cash equivalents and investments of $133.7 million.

    Upcoming Anticipated Milestones

    • Corporate
      • Data from the maralixibat and volixibat studies, including long-term maralixibat data (up to 220 weeks) for the treatment of patients with ALGS, to be presented at The Liver Meeting Digital Experience™ (AASLD), November 13-17, 2020.
      • Hosting inaugural Investor Day on December 9, 2020. Additional details below.
    • Regulatory
      • Complete rolling NDA submission to FDA for treatment of cholestatic pruritus in patients with ALGS in the first quarter 2021.
      • Marketing Authorization Application submission to European regulators for maralixibat in the treatment of patients with PFIC2 by the end of 2020.
    • Pipeline
      • Maralixibat:
        • Phase 2 study initiation planned for biliary atresia by first quarter 2021.
        • Completion of enrollment for MARCH PFIC study anticipated in second quarter 2021.
      • Volixibat:
        • Presenting dose-ranging data at AASLD to inform regimens for potentially pivotal studies in adult cholestasis.
        • Phase 2 study in primary sclerosing cholangitis planned for first quarter 2021.
        • Phase 2 study in intrahepatic cholestasis of pregnancy planned for first quarter 2021.

    Investor Day – December 9, 2020

    Mirum will be hosting its inaugural Investor Day to highlight Mirum's pipeline progress and commercial plans to bring potentially transformational new treatments to patients with cholestatic liver diseases. The virtual event will take place on December 9, 2020 at 11:00 a.m. ET. Additional information will be provided closer to the event date.

    AASLD – The Liver Meeting Digital Experience™ 2020

    New data from maralixibat and volixibat studies will be presented at The Liver Meeting Digital Experience, the annual meeting of the American Association for the Study of Liver Diseases, taking place November 13-17, 2020. Featured presentations to include the following abstracts:

    Late-breaker Oral Presentation

    L05: Preliminary Analysis of ITCH and IMAGINE II – Outcome of long-term administration of maralixibat in children with Alagille syndrome

    • Presented by Benjamin Shneider, M.D. on November 15, 2020 during the 5:30-7:00 p.m. ET session. View the abstract.

    Poster Presentations

    Abstract #1221: A Phase 1 dose-ranging study assessing fecal bile acid excretion by volixibat, an apical sodium-dependent bile acid transporter inhibitor, and coadministration with loperamide

    Abstract #341: Pruritus intensity is associated with cholestasis biomarkers and quality of life measures after maralixibat treatment in children with Alagille syndrome

    Abstract #1792: Natural variability of pruritus in Alagille syndrome; an analysis from the ICONIC study utilizing the Itch Reported Outcome Observer (ItchRO[Obs]) tool

    All posters will be available at the start of the congress on November 13, 2020 and available throughout the duration of the meeting. Abstracts are available via Hepatology on the AASLD website.

    About Maralixibat

    Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases. Maralixibat inhibits the apical sodium dependent bile acid transporter, resulting in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thereby potentially reducing bile acid mediated liver damage and related effects and complications. More than 1,600 individuals have received maralixibat, including more than 120 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). In the ICONIC Phase 2b ALGS clinical trial, patients taking maralixibat had significant reductions in bile acids and pruritus compared to placebo. In a Phase 2 PFIC study, a genetically defined subset of BSEP (bile salt export pump) deficient (PFIC2), patients responded to maralixibat. The FDA has granted maralixibat Breakthrough Therapy designation for pruritus associated with ALGS in patients one year of age and older and for PFIC2. Maralixibat was generally well-tolerated throughout the studies. The most frequent adverse events were diarrhea and abdominal pain. For more information about the Maralixibat Expanded Access Program please visit ALGSEAP.com. For more information about the Phase 3 study for maralixibat in pediatric patients with PFIC, visit PFICtrial.com.

    About Mirum Pharmaceuticals

    Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. The company's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. The company is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis and intrahepatic cholestasis of pregnancy. For more information, visit MirumPharma.com. Follow Mirum on Twitter, Facebook and LinkedIn.

    Forward-Looking Statements

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the results, conduct, progress and timing of Mirum's ongoing and planned studies for maralixibat and volixibat, the regulatory approval path for maralixibat and volixibat, the strength of Mirum's balance sheet and the adequacy of cash, cash equivalents and investments on hand, the impacts of the COVID-19 pandemic, and commercial readiness activities. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "plans," "will", "anticipates," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum's business in general, the impact of the COVID-19 pandemic, and the other risks described in Mirum's filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

    Mirum Pharmaceuticals, Inc.
    Condensed Consolidated Statement of Operations Data
    (in thousands, except share and per share amounts)
    (Unaudited)

     

     

     

     

     

     

     

    Three Months Ended

     

    Nine Months Ended

    September 30,

     

    September 30,

    2020

     

    2019

     

    2020

     

    2019

     
    Operating expenses:
    Research and development

    $

    15,984

     

    $

    12,159

     

    $

    51,879

     

    $

    28,611

     

    General and administrative

     

    5,732

     

     

    3,708

     

     

    15,466

     

     

    7,474

     

    Total operating expenses (1)

     

    21,716

     

     

    15,867

     

     

    67,345

     

     

    36,085

     

     
    Loss from operations

     

    (21,716

    )

     

    (15,867

    )

     

    (67,345

    )

     

    (36,085

    )

    Interest income

     

    237

     

     

    785

     

     

    1,391

     

     

    1,485

     

    Other income (expense), net

     

    (30

    )

     

    (5

    )

     

    (109

    )

     

    (1

    )

     
    Net loss before provision for income taxes

     

    (21,509

    )

     

    (15,087

    )

     

    (66,063

    )

     

    (34,601

    )

    Provision for (benefit from) income taxes

     

    (3

    )

     

    -

     

     

    4

     

     

    -

     

    Net loss

    $

    (21,506

    )

    $

    (15,087

    )

    $

    (66,067

    )

    $

    (34,601

    )

     
    Net loss per share, basic and diluted

    $

    (0.86

    )

    $

    (0.84

    )

    $

    (2.65

    )

    $

    (4.47

    )

     
    Weighted-average shares of common stock outstanding, basic and diluted

     

    25,132,916

     

     

    17,996,065

     

     

    24,965,178

     

     

    7,745,241

     

     
     

    (1) Amounts include stock-based compensation expense as follows:

     
    Research and development

    $

    1,361

     

    $

    830

     

    $

    3,662

     

    $

    1,539

     

    General and administrative

     

    2,067

     

     

    1,314

     

     

    5,313

     

     

    2,464

     

    Total stock-based compensation

    $

    3,428

     

    $

    2,144

     

    $

    8,975

     

    $

    4,003

     

     
     
     

    Mirum Pharmaceuticals, Inc.

    Selected Condensed Consolidated Balance Sheet Data

    (in thousands)

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

    September 30,

     

    December 31,

     

     

     

     

     

     

    2020

     

    2019

    (Unaudited)

     

     

     
    Cash, cash equivalents and investments

    $

    133,749

     

    $

    139,952

     

    Working capital

     

    119,359

     

     

    106,287

     

    Total assets

     

    141,865

     

     

    146,712

     

    Accumulated deficit

     

    (135,968

    )

     

    (69,901

    )

    Total stockholders' equity

     

    120,255

     

     

    130,349

     

     

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