1. Partnership leverages Takeda's leadership in rare disease, gastroenterology, and hepatology to advance maralixibat in a major market

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) and Takeda Pharmaceutical Company Limited ((TAK) announced that the companies have entered into an exclusive licensing agreement for the development and commercialization of maralixibat in Japan for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA). Maralixibat, an investigational, orally administered medication, is being evaluated globally in ALGS, PFIC, and BA.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210921005502/en/

    Under the terms of the agreement…

    Partnership leverages Takeda's leadership in rare disease, gastroenterology, and hepatology to advance maralixibat in a major market

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) and Takeda Pharmaceutical Company Limited ((TAK) announced that the companies have entered into an exclusive licensing agreement for the development and commercialization of maralixibat in Japan for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA). Maralixibat, an investigational, orally administered medication, is being evaluated globally in ALGS, PFIC, and BA.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210921005502/en/

    Under the terms of the agreement, Takeda will be responsible for regulatory approval and commercialization of maralixibat in Japan. Takeda will also be responsible for development, including conducting clinical studies in cholestatic indications.

    "Takeda is a leading global biopharmaceutical company with extensive experience in development and commercialization of novel therapies to treat rare diseases as well as gastroenterology and hepatology, making them an ideal partner as we look to accelerate the delivery of maralixibat to children living with rare liver diseases in Japan," said Chris Peetz, president and chief executive officer of Mirum. "As we approach potential commercialization in the United States and complete the recent filing for Alagille syndrome in Europe, our goal is to partner with top companies outside of North America and Europe to ensure global reach for patients with these terrible diseases. We are excited for Takeda to engage in the development of maralixibat and collaborate in our effort to advance this potentially life-changing therapy."

    "There is a significant unmet medical need for a treatment to help patients with cholestatic diseases such as ALGS and PFIC in Japan and developing novel treatment for those patients suffering from rare liver diseases is a top priority for Takeda's global R&D strategy," said Dr. Naoyoshi Hirota, general manager of Takeda development center Japan. "This agreement reinforces Takeda's commitment to developing highly differentiated medicines to improve the health and quality of life of patients."

    Mirum has submitted a 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. The NDA is currently under priority review with a PDUFA, or FDA decision date, of September 29, 2021. Mirum also recently submitted a Marketing Authorization Application to the European Medicines Agency for maralixibat for the treatment of cholestatic liver disease in patients with ALGS.

    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 effects. 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 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 shown to have a tolerable safety profile in the studies. The most frequent treatment-related adverse events were diarrhea and abdominal pain. Maralixibat has been studied extensively and its safety database represents the largest database for an ASBT inhibitor.

    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 further information about maralixibat's ongoing studies in pediatric liver disease, please visit the study websites: Phase 3 MARCH study for PFIC and Phase 2b EMBARK study for biliary atresia.

    About Mirum Pharmaceuticals, Inc.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat for the treatment of cholestatic pruritus in patients with ALGS in the U.S. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021. Additionally, Mirum has submitted a marketing authorization application for maralixibat to the European Medicines Agency for the treatment of cholestatic liver disease for patients with ALGS. Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to discover and deliver life-transforming treatments, guided by our commitment to patients, our people and the planet. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetics and Hematology, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions.

    For more information, visit https://www.takeda.com.

    Disclaimer

    The drug information contained herein is intended to disclose corporate information. Nothing contained in this document should be considered a solicitation, promotion, or indication for any prescription drug, including those currently under development.

    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 potential development and commercialization by Takeda of maralixibat in Japan for various indications, Mirum and Takeda's receipt of revenue in connection with the license agreement with Takeda, as well as the regulatory approval pathway 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 "will," "could," "would," "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. - Marketing Authorization Application for treatment of cholestatic disease in Alagille syndrome submitted.

    - Natural history analysis shows significant improvement in event-free survival and transplant-free survival.

    - PFIC regulatory strategy in Europe to focus on results of MARCH-PFIC Phase 3 study, expected in second quarter of 2022.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced the submission of a Marketing Authorization Application (MAA) for the treatment of cholestatic liver disease in patients with Alagille syndrome (ALGS) to the European Medicines Agency (EMA). In conjunction with the ALGS submission, Mirum's MAA for progressive familial intrahepatic cholestasis type 2 (PFIC2) was withdrawn with plans to re-submit after…

    - Marketing Authorization Application for treatment of cholestatic disease in Alagille syndrome submitted.

    - Natural history analysis shows significant improvement in event-free survival and transplant-free survival.

    - PFIC regulatory strategy in Europe to focus on results of MARCH-PFIC Phase 3 study, expected in second quarter of 2022.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced the submission of a Marketing Authorization Application (MAA) for the treatment of cholestatic liver disease in patients with Alagille syndrome (ALGS) to the European Medicines Agency (EMA). In conjunction with the ALGS submission, Mirum's MAA for progressive familial intrahepatic cholestasis type 2 (PFIC2) was withdrawn with plans to re-submit after availability of results from the ongoing MARCH-PFIC Phase 3 study in a broader set of PFIC sub-types and with higher doses of maralixibat.

    The submission of the ALGS MAA presents an opportunity to deliver the first ever pharmacologic treatment option for this devastating disease for which there is significant and urgent unmet need. This submission follows discussion with the EMA on the appropriate strategy for seeking marketing approval for both ALGS and PFIC broadly as soon as possible. The MAA is comprised of the long-term ICONIC study in patients with ALGS, which showed a significant improvement on pruritus (p<0.0001) and improvement on other markers of cholestatic liver disease. The ICONIC data is supported by a new analysis, which includes an aggregated cohort of maralixibat-treated patients with ALGS (n=84) compared to a natural history control cohort, demonstrating a statistically significant improvement in six-year event-free survival (p<0.0001), with events defined as biliary diversion surgery, liver transplant, hepatic decompensation (ascites requiring therapy or variceal bleeding) or death.

    "The six-year event-free survival data, coupled with the previously presented ICONIC data, provides a catalyst to accelerate our ALGS submission," said Chris Peetz, president and chief executive officer at Mirum. "We feel a tremendous urgency to advance maralixibat for patients as quickly as possible as its availability may provide a significant shift in treatment options for patients living with this unrelenting rare liver disease. The recent long-term analysis allows us to take this major step forward in ALGS today and we plan to submit for all PFIC types after completion of the MARCH-PFIC Phase 3 study."

    In the U.S., the Food and Drug Administration has accepted Mirum's new drug application for maralixibat for the treatment of cholestatic pruritus in patients with ALGS and the company is preparing for a potential launch following the PDUFA date of September 29, 2021, if approved by the FDA.

    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 effects. 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 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 shown to have a tolerable safety profile in the studies. The most frequent treatment-related adverse events were diarrhea and abdominal pain. Maralixibat has been studied extensively and its safety database represents the largest database for an ASBT inhibitor.

    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 further information about maralixibat's ongoing studies in pediatric liver disease, please visit the study websites: Phase 3 MARCH study for PFIC and Phase 2b EMBARK study for biliary atresia.

    About Mirum Pharmaceuticals, Inc.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021. Additionally, Mirum's marketing authorization application for the treatment of cholestatic liver disease in patients with ALGS has been submitted to the European Medicines Agency. Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.

    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 regulatory approval pathway for maralixibat and the safety and efficacy of 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 "will," "could," "would," "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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  3. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced that it will participate in five investor conferences taking place virtually throughout the month of September.

    Citi 16th Annual BioPharma Virtual Conference
    September 10, 2021
    Format: One-on-one meetings

    H.C. Wainwright 23rd Annual Global Investor Conference
    September 13-14, 2021
    Format: Company presentation (available September 13 at 7:00 a.m. ET) and one-one-meetings.

    Baird Global Healthcare Conference
    September 14-15, 2021
    Format: Company presentation (September 14 at 12:50 p.m. ET) and one-on-one meetings.

    Leerink CyberRX Series 1x1 Event
    September 22-23, 2021
    Format: One-on-one meetings.

    Cantor Global Healthcare Conference
    September 27-29, 2021
    Format: Company presentation

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced that it will participate in five investor conferences taking place virtually throughout the month of September.

    Citi 16th Annual BioPharma Virtual Conference

    September 10, 2021

    Format: One-on-one meetings

    H.C. Wainwright 23rd Annual Global Investor Conference

    September 13-14, 2021

    Format: Company presentation (available September 13 at 7:00 a.m. ET) and one-one-meetings.

    Baird Global Healthcare Conference

    September 14-15, 2021

    Format: Company presentation (September 14 at 12:50 p.m. ET) and one-on-one meetings.

    Leerink CyberRX Series 1x1 Event

    September 22-23, 2021

    Format: One-on-one meetings.

    Cantor Global Healthcare Conference

    September 27-29, 2021

    Format: Company presentation (September 27 at 2:40 p.m. ET) and one-on-one meetings.

    Visit the Investors and Media section of Mirum's corporate website for additional information.

    About Mirum Pharmaceuticals, Inc.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

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  4. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced the appointment of industry veteran William C. Fairey to its Board of Directors. Mr. Fairey brings to Mirum significant experience in establishing commercial organizations within public companies, building markets, and launching novel therapeutics to treat specialty and rare and orphan diseases.

    Concurrent with the appointment of Mr. Fairey, Tiba Aynechi, Ph.D., has resigned from the Company's Board of Directors and her position on the Board's Compensation and Nominating and Corporate Governance Committees, respectively, effective August 19, 2021. Dr. Aynechi has been a valued Director since Mirum's inception in 2018.

    "Bill's extensive experience in global commercial and corporate…

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced the appointment of industry veteran William C. Fairey to its Board of Directors. Mr. Fairey brings to Mirum significant experience in establishing commercial organizations within public companies, building markets, and launching novel therapeutics to treat specialty and rare and orphan diseases.

    Concurrent with the appointment of Mr. Fairey, Tiba Aynechi, Ph.D., has resigned from the Company's Board of Directors and her position on the Board's Compensation and Nominating and Corporate Governance Committees, respectively, effective August 19, 2021. Dr. Aynechi has been a valued Director since Mirum's inception in 2018.

    "Bill's extensive experience in global commercial and corporate strategy, and in rare disease, will be instrumental as we prepare for the launch of our first medication in the U.S. and continue to advance our pipeline to deliver medications for patients with rare liver diseases globally," said Chris Peetz, President and Chief Executive Officer at Mirum. "On behalf of the Board of Directors, I want to thank Tiba for her leadership and significant contributions as a founding Board member of Mirum. Her confidence in Mirum and sagacious guidance in the early days of the company helped Mirum to move from clinical-stage to a commercial-ready company, poised to make a meaningful impact for the rare liver disease community."

    Mr. Fairey brings nearly 30 years of experience building strong and scalable global commercial organizations. Most recently, Mr. Fairey served as Executive Vice President and Chief Commercial Officer at MyoKardia, where he built the commercial and portfolio strategy, established the company's European headquarters, was planning for the expansion in Latin America and Asia when the company was acquired by Bristol-Myers Squibb. Mr. Fairey held a similar role at ChemoCentryx, where, as Chief Operating Officer, he was responsible for building out the initial commercial and medical teams. Prior to ChemoCentryx, he served in a number of roles at Actelion Pharmaceuticals, including President of the U.S. division. Before taking that role, Mr. Fairey developed Actelion's Asia-Pacific business, creating new opportunities in China, Taiwan, and Southeast Asia, and chaired the Board of Directors for Actelion China. He was also President of Actelion Canada and as Vice President, Sales and Managed Markets, one of the founding members of the U.S. executive team.

    "I am excited to join Mirum's Board to champion and help to grow what is already a strong and capable organization with a proven commitment to developing life-changing medicines," said William C. Fairey. "I look forward to supporting Mirum's efforts as they prepare for the potential launch of their first medication in the U.S., and as they expand their reach to the rare disease communities globally."

    About Mirum Pharmaceuticals, Inc.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.

    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 regulatory approval pathway 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 "will," "could," "would," "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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  5. - NDA for maralixibat for cholestatic pruritus in Alagille syndrome under priority review; PDUFA date is September 29, 2021
    - Commercial preparations complete in anticipation of U.S. launch of maralixibat in ALGS
    - Conference call to provide business update today, August 5 at 1:30 p.m. PT/4:30 p.m. ET

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), today reported financial results for the quarter ended June 30, 2021 and provided a business update.

    "We are pleased with the progress we have made in the last quarter as we prepare for the potential FDA approval of maralixibat and advance our late-stage clinical development pipeline," said Chris Peetz, president and chief executive officer at Mirum. "Alagille syndrome is a terrible disease, and we…

    - NDA for maralixibat for cholestatic pruritus in Alagille syndrome under priority review; PDUFA date is September 29, 2021

    - Commercial preparations complete in anticipation of U.S. launch of maralixibat in ALGS

    - Conference call to provide business update today, August 5 at 1:30 p.m. PT/4:30 p.m. ET

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), today reported financial results for the quarter ended June 30, 2021 and provided a business update.

    "We are pleased with the progress we have made in the last quarter as we prepare for the potential FDA approval of maralixibat and advance our late-stage clinical development pipeline," said Chris Peetz, president and chief executive officer at Mirum. "Alagille syndrome is a terrible disease, and we know patients and physicians are waiting for treatment options. We are launch ready in the United States and have amplified our reach to patients globally with our expanded access program and recent partnerships to accelerate potential approvals in a number of markets."

    Recent Key Operational Highlights

    • Entered into an exclusive licensing agreement with GC Pharma to develop and commercialize maralixibat for rare liver diseases in South Korea.
    • Presented maralixibat transplant-free survival data for progressive familial intrahepatic cholestasis type 2 (PFIC2) and long-term safety analyses for Alagille syndrome (ALGS) at the 6th World Congress of Pediatric Gastroenterology, Hepatology and Nutrition (WCPGHAN) Annual Meeting 2021.
    • Appointed Carol L. Brosgart, M.D. as an independent director to the Board of Directors.
    • Presented an integrated safety analysis of maralixibat in patients with ALGS and unveiled a multi-national survey of patient reported outcomes from pregnant women with intrahepatic cholestasis of pregnancy (ICP) at the EASL International Liver Congress 2021.

    Financial Results

    • Licensing revenue for the quarter ended June 30, 2021 was $11.0 million, which was associated with our license and collaboration agreement with CANbridge Pharmaceuticals, Inc., compared to none for the second quarter of 2020.
    • Total operating expenses for the quarter ended June 30, 2021 were $48.4 million, compared to $23.6 million for the second quarter of 2020.
      • Research and development expenses for the quarter ended June 30, 2021 were $35.0 million, compared to $18.6 million for the comparable prior-year period. The increase was primarily driven by an upfront payment and funding associated with the Vivet gene therapy programs and costs associated with the initiation of volixibat clinical studies.
      • General and administrative expenses for the second quarter of 2021 were $13.4 million, compared to $5.0 million for the comparable prior-year period. The increase was primarily due to increased commercialization and headcount costs.
    • For the quarter ended June 30, 2021, Mirum reported a net loss of $43.9 million, or $1.45 per share, compared with a net loss of $23.3 million, or $0.93 per share for the same period in 2020.
    • As of June 30, 2021, Mirum had cash, cash equivalents, and short-term investments of $238.8 million.

    Upcoming Anticipated Milestones

    • Commercial and Regulatory
      • The FDA has issued a PDUFA date of September 29, 2021, and, if approved, Mirum will launch maralixibat in the U.S. representing a commercial opportunity estimated at more than $500 million.
      • Marketing authorization application (MAA) for PFIC2 decision expected in early 2022.
      • Mirum plans to submit an MAA for ALGS in Europe based on results from the ICONIC study and six-year event-free survival analyses.
    • Pipeline and New Data
      • Maralixibat
        • Topline data from the Phase 3 MARCH PFIC study is expected in the second quarter of 2022.
        • Enrollment in the Phase 2b EMBARK study of maralixibat in biliary atresia is ongoing; data expected in 2023.
      • Volixibat
        • Enrollment ongoing in the potentially registrational Phase 2b OHANA study for ICP; interim analysis expected in 2022.
        • Enrollment ongoing in the Phase 2b VISTAS study for primary sclerosing cholangitis (PSC); interim analysis expected in 2022.
        • A Phase 2b study in primary biliary cholangitis (PBC) is planned for the second half of 2021.

    Business Update Conference Call

    Mirum will host a conference call today, August 5, 2021 at 1:30 p.m. PT/4:30 p.m. ET, to provide a review of the second quarter activities and to discuss business updates. Join the call using the following details:

    Conference Call Details:

    U.S. toll-free:

    844.200.6205

    International:

    646.904.5544

    Passcode:

    097025

    You may also access the call via webcast by visiting the Investors section on Mirum's corporate website. A replay of this webcast will be available for 30 days.

    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. Maralixibat has been studied extensively and its safety database represents the largest database for an ASBT inhibitor.

    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 further information about maralixibat's ongoing studies in pediatric liver disease, please visit the study websites: Phase 3 MARCH study for PFIC and Phase 2b EMBARK study for biliary atresia.

    About Volixibat

    Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the apical sodium dependent bile acid transporter (ASBT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of ASBT, thereby reducing bile acids systemically and in the liver. Phase 1 and Phase 2 studies 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 individuals across multiple clinical trials. The most common adverse events reported were mild to moderate gastrointestinal events observed in the volixibat groups.

    Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (VISTAS study) and intrahepatic cholestasis of pregnancy (OHANA study). Mirum plans to initiate a Phase 2b study for primary biliary cholangitis later this year.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize two gene therapy programs, VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics, following preclinical evaluation and IND-enabling studies.

    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 potential regulatory approval of maralixibat as well as the benefits and expected market opportunity 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 "will," "could," "would," "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

     

    Six Months Ended

     

     

     

     

     

     

     

    June 30,

     

    June 30,

     

     

     

     

     

     

     

     

    2021

     

     

     

    2020

     

     

     

    2021

     

     

     

    2020

     

     
    License revenue

    $

    11,000

     

    $

    -

     

    $

    11,000

     

    $

    -

     

     
    Operating expenses:
    Research and development

     

    35,048

     

     

    18,555

     

     

    73,182

     

     

    35,895

     

    General and administrative

     

    13,353

     

     

    5,042

     

     

    22,832

     

     

    9,734

     

    Total operating expenses (1)

     

    48,401

     

     

    23,597

     

     

    96,014

     

     

    45,629

     

     
    Loss from operations

     

    (37,401

    )

     

    (23,597

    )

     

    (85,014

    )

     

    (45,629

    )

    Interest income

     

    80

     

     

    405

     

     

    229

     

     

    1,154

     

    Interest expense

     

    (4,776

    )

     

    -

     

     

    (8,157

    )

    Change in fair value of derivative liability

     

    (1,272

    )

     

    -

     

     

    (938

    )

    Other expense, net

     

    (514

    )

     

    (56

    )

     

    (530

    )

     

    (79

    )

     
    Net loss before provision for income taxes

     

    (43,883

    )

     

    (23,248

    )

     

    (94,410

    )

     

    (44,554

    )

    Provision for income taxes

     

    11

     

     

    3

     

     

    16

     

     

    7

     

    Net loss

    $

    (43,894

    )

    $

    (23,251

    )

    $

    (94,426

    )

    $

    (44,561

    )

     
    Net loss per share, basic and diluted

    $

    (1.45

    )

    $

    (0.93

    )

    $

    (3.13

    )

    $

    (1.79

    )

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

     

    30,274,749

     

     

    25,056,123

     

     

    30,190,352

     

     

    24,880,387

     

     
     

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

     
    Research and development

    $

    2,015

     

    $

    1,260

     

    $

    4,758

     

    $

    2,301

     

    General and administrative

     

    2,808

     

     

    1,714

     

     

    5,350

     

     

    3,246

     

    Total stock-based compensation

    $

    4,823

     

    $

    2,974

     

    $

    10,108

     

    $

    5,547

     

     
     
     

    Mirum Pharmaceuticals, Inc.

    Selected Condensed Consolidated Balance Sheet Data

    (in thousands)

     
     

    June 30,

    December 31,

     

    2021

     

     

    2020

     

    (Unaudited)

     
    Cash, cash equivalents and short-term investments

    $

    238,841

     

    $

    231,820

     

    Working capital

     

    199,338

     

     

    217,888

     

    Total assets

     

    247,668

     

     

    240,864

     

    Accumulated deficit

     

    (267,597

    )

     

    (173,171

    )

    Total stockholders' equity

     

    95,737

     

     

    172,095

     

     
     
     

     

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  6. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), today announced that the company will report financial results for the quarter ended June 30, 2021 on Thursday, August 5, 2021. That same day, Mirum will host a conference call and audio webcast at 1:30 p.m. PT/4:30 p.m. ET to provide a business update.

    Conference Call Details:

    U.S. toll-free:

    844.200.6205

    International:

    646.904.5544

    Passcode:

    097025

    You may also access the call via webcast by visiting the Investors & Media section on Mirum's corporate website. A replay of this webcast will be available for 30 days.

    About Mirum Pharmaceuticals

    Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage…

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), today announced that the company will report financial results for the quarter ended June 30, 2021 on Thursday, August 5, 2021. That same day, Mirum will host a conference call and audio webcast at 1:30 p.m. PT/4:30 p.m. ET to provide a business update.

    Conference Call Details:

    U.S. toll-free:

    844.200.6205

    International:

    646.904.5544

    Passcode:

    097025

    You may also access the call via webcast by visiting the Investors & Media section on Mirum's corporate website. A replay of this webcast will be available for 30 days.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize two gene therapy programs, VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics, following preclinical evaluation and IND-enabling studies.

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    View Full Article Hide Full Article
  7. - GC Pharma to lead development and commercialization in South Korea

    - Mirum seeks to expand global availability of maralixibat in rare liver diseases

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) and GC Pharma (KRX:006280) today announced that they have entered into an exclusive licensing agreement, pursuant to which GC Pharma has agreed to develop and commercialize maralixibat in South Korea. Maralixibat is an investigational, orally administered medication being evaluated in Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA).

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210726005832/en/

    Maralixibat targets the apical sodium…

    - GC Pharma to lead development and commercialization in South Korea

    - Mirum seeks to expand global availability of maralixibat in rare liver diseases

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) and GC Pharma (KRX:006280) today announced that they have entered into an exclusive licensing agreement, pursuant to which GC Pharma has agreed to develop and commercialize maralixibat in South Korea. Maralixibat is an investigational, orally administered medication being evaluated in Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA).

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210726005832/en/

    Maralixibat targets the apical sodium dependent bile acid transporter (ASBT) ultimately resulting in lower levels of bile acids systemically. The U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for maralixibat for the treatment of cholestatic pruritus in patients with ALGS under priority review. The European Medicines Agency is reviewing maralixibat for the treatment of PFIC2. Mirum has commenced a global Phase 2b maralixibat study (EMBARK) for the treatment of BA.

    Under the terms of the licensing agreement, GC Pharma has obtained the exclusive right to develop and commercialize maralixibat within South Korea for ALGS, PFIC, and BA. In exchange, Mirum is entitled to receive an upfront payment of $5.0 million as well payments for the achievement of future regulatory and commercial milestones, with tiered double-digit royalties based on product net sales in the region.

    "GC Pharma shares a vision for creating and delivering transformative treatments," said Chris Peetz, president and chief executive officer at Mirum. "We are excited about GC Pharma's proven track record commercializing treatments for rare diseases and the potential to pursue an expedited approval pathway for maralixibat for Alagille syndrome in South Korea."

    "This collaboration is another significant milestone in delivering our vision for rare disorders," said EC Huh, Ph.D., president of GC Pharma. "We look forward to leveraging our capabilities to explore the development and commercialization of maralixibat."

    About Mirum Pharmaceuticals, Inc.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    About GC Pharma

    GC Pharma is a biopharmaceutical company that delivers life-saving and life-sustaining protein therapeutics and vaccines. Headquartered in South Korea, GC Pharma is the largest plasma protein product manufacturer in Asia and has been dedicated to quality healthcare solutions for more than half a century. Green Cross Corporation updated its corporate brand as GC Pharma in early 2018. Green Cross Corporation remains the company's registered, legal name. For more information, visit GCcorp.com.

    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 potential development and commercialization by GC Pharma of maralixibat in within South Korea for various indications, Mirum's receipt of upfront, milestone and royalty revenue in connection with the license agreement with GC Pharma, as well as the regulatory approval pathway 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 "will," "could," "would," "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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  8. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a leader in rare liver disease, today announced that it will participate in Piper Sandler's Virtual European Association for the Study of the Liver (EASL) Takeaway Day on Monday, June 28, 2021, at 2:00 p.m. ET. Chris Peetz, president and chief executive officer, and Pam Vig, Ph.D., chief scientific officer, will participate in the fireside chat.

    Visit the event website to register. A replay will be available within the Investor Relations section of Mirum's website at https://ir.mirumpharma.com/events-and-presentations.

    About Mirum Pharmaceuticals, Inc.

    Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline…

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a leader in rare liver disease, today announced that it will participate in Piper Sandler's Virtual European Association for the Study of the Liver (EASL) Takeaway Day on Monday, June 28, 2021, at 2:00 p.m. ET. Chris Peetz, president and chief executive officer, and Pam Vig, Ph.D., chief scientific officer, will participate in the fireside chat.

    Visit the event website to register. A replay will be available within the Investor Relations section of Mirum's website at https://ir.mirumpharma.com/events-and-presentations.

    About Mirum Pharmaceuticals, Inc.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    View Full Article Hide Full Article
  9. - Data from five years of treatment with maralixibat reinforces safety and tolerability in patients with Alagille syndrome.

    - Intrahepatic cholestasis of pregnancy patient-reported data demonstrates significant burden of disease and underscores urgent need for new treatment options.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced the presentation of analyses from its rare liver disease programs during the EASL International Liver Congress™. The posters being presented feature an integrated safety analysis of maralixibat in patients with Alagille syndrome (ALGS) and the unveiling of a multi-national survey of patient reported outcomes from pregnant women with intrahepatic cholestasis of pregnancy (ICP). The posters are now available…

    - Data from five years of treatment with maralixibat reinforces safety and tolerability in patients with Alagille syndrome.

    - Intrahepatic cholestasis of pregnancy patient-reported data demonstrates significant burden of disease and underscores urgent need for new treatment options.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced the presentation of analyses from its rare liver disease programs during the EASL International Liver Congress™. The posters being presented feature an integrated safety analysis of maralixibat in patients with Alagille syndrome (ALGS) and the unveiling of a multi-national survey of patient reported outcomes from pregnant women with intrahepatic cholestasis of pregnancy (ICP). The posters are now available on the congress website.

    Abstract PO-1285: An integrated analysis of long-term clinical safety in maralixibat-treated participants with Alagille syndrome

    Data from more than five years of maralixibat treatment across three Phase 2 clinical studies (and their extension studies) were analyzed to assess the overall clinical safety of maralixibat in patients with ALGS (n=86). The analysis evaluated treatment-emergent adverse events (TEAEs) and laboratory parameters including reported rates, severity and seriousness, actions taken with maralixibat (i.e., dose reductions and discontinuations), time to onset and potential dose-response relationships. A sub-analysis of safety data was also conducted evaluating data from 13-week placebo-controlled studies.

    The analysis concluded:

    • Maralixibat was well-tolerated for more than five years.
    • The most common TEAEs were diarrhea and abdominal pain.
    • Mild to moderate gastrointestinal events (GI events) were observed in the first weeks of treatment, were transient, and lasted less than one week in duration.
      • In placebo-controlled studies, GI events occurred at similar rates between maralixibat and placebo in the background of ALGS.
      • There were no discontinuations of maralixibat due to diarrhea or abdominal pain.
    • No clinically significant trends or patterns in laboratory measures were observed.
      • ALT levels were consistent with natural history comparisons of ALGS.

    Abstract PO-2657: Patient perspectives on pruritus in intrahepatic cholestasis of pregnancy: a multinational survey

    The multinational survey was conducted in collaboration with ICP Support, a leading patient advocacy group focused on ICP and based in the United Kingdom.

    The survey evaluated the burden of cholestatic pruritus, the impact on quality of life, and the effectiveness of available treatments as reported by women who have or have had ICP (n=688). Participants in the survey were asked to assess severity of their itch, sleep disturbance, and impact of medications received, using a numerical rating scale (NRS) of 0-10, with 0 representing none and 10 representing the worst imaginable, for each symptom.

    Responses from the survey demonstrated that pruritus has debilitating effects on patients living with ICP, including a substantial impact on sleep and quality of life. The results found that:

    • The median worst itch reported was a 9 out of 10 on the NRS.
    • 94% of women reported itch-related sleep disturbances, which were associated with degree of itch severity.
    • Itch was associated with mood changes and often led to a disruption of day-to-day responsibilities and routines.
    • 71% of women reported that they were not asked about itch by their healthcare provider.
    • A majority of women reported taking ≥2 medications; however, most reported either only partial or no resolution of ICP-related pruritus.

    These responses underscore the significant impact pruritus can have on women with ICP as well as the high unmet need for the development of safe and effective therapies to treat this rare liver disease.

    "This integrated safety analysis of maralixibat with more than five years of evaluation across 86 patients with ALGS continues to underscore the safety and tolerability profile of maralixibat for use in this chronic disease setting," said Pam Vig, Ph.D., chief scientific officer at Mirum. "We are also excited to showcase the ICP survey data which demonstrates the significant burden that pruritus can have on these women, and the tremendous need for new therapies. As we continue to enroll patients in our OHANA study, we remain hopeful that volixibat's potential to reduce cholestasis and pruritus associated with ICP may improve quality of life."

    "Understanding the severity of pruritus and the impact on a woman's quality of life whilst pregnant is of critical importance to advancing research and identifying a new treatment option for ICP," said Jenny Chambers, chief executive officer, ICP Support. "We surveyed nearly 700 women globally to assess the scale of how debilitating this condition can be and to champion research that will help us better understand the effects of the condition. We hope this in turn will lead to improved outcomes for women with this uncommon and unrelenting 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 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. Maralixibat has been studied extensively and its safety database represents the largest database for an ASBT inhibitor.

    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 further information about maralixibat's ongoing studies in pediatric liver disease, please visit the study websites: Phase 3 MARCH study for PFIC and Phase 2b EMBARK study for biliary atresia.

    About Volixibat

    Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the apical sodium dependent bile acid transporter (ASBT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of ASBT, thereby reducing bile acids systemically and in the liver. Phase 1 and Phase 2 studies 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 individuals across multiple clinical trials. The most common adverse events reported were mild to moderate gastrointestinal events observed in the volixibat groups.

    Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (VISTAS study) and intrahepatic cholestasis of pregnancy (OHANA study). Mirum plans to initiate a Phase 2b study for primary biliary cholangitis later this year.

    About Mirum Pharmaceuticals, Inc.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.

    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 potential benefits and an assessment on the severity of side effects of 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 "will," "could," "would," "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.

    View Full Article Hide Full Article
  10. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a leader in rare liver disease, today announced its participation in The International Liver Congress™ taking place virtually June 23-26, 2021. The company will be presenting an integrated safety analysis of maralixibat in 86 patients with Alagille syndrome (ALGS). Mirum will also be unveiling a multi-national survey of patient reported outcomes data from pregnant women with intrahepatic cholestasis of pregnancy (ICP), conducted in collaboration with ICP Support, a leading patient advocacy group focused on ICP, based in the United Kingdom.

    Posters to be presented during the congress include:

    Abstract PO-1285:
    An integrated analysis of long-term clinical safety in maralixibat-treated participants…

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a leader in rare liver disease, today announced its participation in The International Liver Congress™ taking place virtually June 23-26, 2021. The company will be presenting an integrated safety analysis of maralixibat in 86 patients with Alagille syndrome (ALGS). Mirum will also be unveiling a multi-national survey of patient reported outcomes data from pregnant women with intrahepatic cholestasis of pregnancy (ICP), conducted in collaboration with ICP Support, a leading patient advocacy group focused on ICP, based in the United Kingdom.

    Posters to be presented during the congress include:

    Abstract PO-1285:

    An integrated analysis of long-term clinical safety in maralixibat-treated participants with Alagille syndrome

    Abstract PO-2657:

    Patient perspectives on pruritus in intrahepatic cholestasis of pregnancy: a multinational survey

    All posters will be available on The International Liver Congress website beginning Wednesday, June 23 at 8:00 a.m. CEST (2:00 a.m. ET).

    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 further information about maralixibat's ongoing studies in pediatric liver disease, please visit the study websites: Phase 3 MARCH study for PFIC and Phase 2b EMBARK study for biliary atresia.

    About Volixibat

    Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the apical sodium dependent bile acid transporter (ASBT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of ASBT, thereby reducing bile acids systemically and in the liver. Phase 1 and Phase 2 studies 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 individuals across multiple clinical trials. The most common adverse events reported were mild to moderate gastrointestinal events observed in the volixibat groups.

    Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (VISTAS study) and intrahepatic cholestasis of pregnancy (OHANA study). Mirum plans to initiate a Phase 2b study for primary biliary cholangitis later this year.

    About Mirum Pharmaceuticals, Inc.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.

    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 potential benefits of 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 "will," "could," "would," "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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  11. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced the appointment of Carol L. Brosgart, M.D., clinical professor of medicine, biostatistics and epidemiology, University of California, San Francisco, as an independent director to its Board of Directors. Dr. Brosgart currently serves as an industry consultant and scientific advisor, and as an independent director for several public and privately held biotechnology companies.

    "Dr. Brosgart is a visionary leader in development, advocacy and policy for advancing life-changing medicines," said Chris Peetz, president and chief executive officer at Mirum. "We are thrilled to welcome Carol to our board at this exciting time as we prepare for the potential launch of maralixibat and continue…

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced the appointment of Carol L. Brosgart, M.D., clinical professor of medicine, biostatistics and epidemiology, University of California, San Francisco, as an independent director to its Board of Directors. Dr. Brosgart currently serves as an industry consultant and scientific advisor, and as an independent director for several public and privately held biotechnology companies.

    "Dr. Brosgart is a visionary leader in development, advocacy and policy for advancing life-changing medicines," said Chris Peetz, president and chief executive officer at Mirum. "We are thrilled to welcome Carol to our board at this exciting time as we prepare for the potential launch of maralixibat and continue to advance our pipeline of transformative new therapies."

    Dr. Brosgart brings extensive clinical, public policy, advocacy and corporate experience to Mirum having held senior management, board and advisory positions in a number of settings. In addition to various advisory roles, Dr. Brosgart serves as an independent director on the boards of both public and privately held biotechnology companies (Galmed, Abivax, Enochian, and Intrivo Diagnostics) and previously served on the boards of Tobira and Juvaris, until their acquisitions. Dr. Brosgart's senior management experience in biotechnology and healthcare included Chief Medical Officer roles at Alios BioPharma and UCSF Benioff Children's Hospital and Research Center and Vice President, Public Health and Policy at Gilead where she was responsible for the clinical development and approval of two antiviral therapies: Viread® for the treatment of HIV, and Hepsera® for the treatment of chronic hepatitis B. Prior to Gilead, Dr. Brosgart worked for more than 20 years in public health, clinical care, research, and teaching. Dr. Brosgart also serves in public policy through advocacy, advisory and board roles for numerous organizations and foundations, including the American Liver Foundation, the Hepatitis B Foundation, the San Francisco AIDS Foundation and Forum for Collaborative Research.

    Trained as a pediatrician and in public health and preventive medicine, she was among the first physicians in the United States to recognize and treat patients with HIV/AIDS. Dr. Brosgart was the founding Medical Director of the East Bay AIDS Center at Alta Bates Medical Center in Berkeley, California. She led NIH clinical trials as a member of the Community Programs for Clinical Research on AIDS (CPCRA), chaired the CPCRA Scientific Advisory Committee, and contributed to HIV antiretroviral drug development, and to the development of prophylactic and treatment agents for opportunistic and malignant complications of HIV/AIDS.

    "I am thrilled to join Mirum at such a pivotal stage as it approaches the potential launch of maralixibat in Alagille syndrome," said Carol L. Brosgart, M.D. "Mirum's team, combined with its impressive pipeline, has tremendous potential to make a meaningful impact on the lives of patients and their families living with rare liver diseases."

    About Mirum Pharmaceuticals, Inc.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.

    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 regulatory approval pathway 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 "will," "could," "would," "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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  12. - Maralixibat-treated patients achieving serum bile acid control have improved five-year native liver survival; nominated for prestigious Alex Mowat Prize for best oral presentation in hepatology.

    - Safety data from more than five years of maralixibat treatment across three Phase 2 ALGS studies demonstrate that gastrointestinal events were generally mild to moderate and transient in nature, with no discontinuation of maralixibat treatment.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) presented analyses from its clinical studies evaluating maralixibat in patients with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis type 2 (PFIC2), two rare liver diseases affecting children.

    "The breadth of maralixibat data collected…

    - Maralixibat-treated patients achieving serum bile acid control have improved five-year native liver survival; nominated for prestigious Alex Mowat Prize for best oral presentation in hepatology.

    - Safety data from more than five years of maralixibat treatment across three Phase 2 ALGS studies demonstrate that gastrointestinal events were generally mild to moderate and transient in nature, with no discontinuation of maralixibat treatment.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) presented analyses from its clinical studies evaluating maralixibat in patients with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis type 2 (PFIC2), two rare liver diseases affecting children.

    "The breadth of maralixibat data collected for more than five years, coupled with our understanding of the safety and tolerability of long-term use, helps to underscore its importance as a potentially transformative treatment for patients with ALGS and PFIC2," said Pam Vig, Ph.D., chief scientific officer at Mirum. "These data highlight that maralixibat may be a meaningful alternative to invasive surgeries that are the current standard of care in these devastating diseases."

    Oral Presentation: H-O-012: Serum bile acid control in long-term maralixibat treated patients is associated with native liver survival in children with progressive familial intrahepatic cholestasis due to bile salt export pump deficiency.

    Presented June 3, 2021 at 3:55 p.m. CEST

    This abstract has been nominated for the Annual Alex Mowat Prize for best oral presentation in hepatology. Results to be announced on June 5, 2021 at 1:30 p.m. CEST

    The presentation is available on Mirum's website in the Publications and Presentations section.

    An analysis from the Phase 2 INDIGO open-label study evaluating long-term clinical efficacy and transplant-free survival with maralixibat in patients (n=19) with bile salt export pump (BSEP) deficiency, or progressive familial intrahepatic cholestasis type 2 (PFIC2) was presented during the congress. These data demonstrated that patients with long-term maralixibat treatment who achieved serum bile acid (sBA) response had five-year transplant-free survival. In addition, those patients who achieved sBA response also had significant improvements in pruritus, liver parameters, quality of life and growth. The study showed that maralixibat was generally well-tolerated and the most common adverse events were mild to moderate diarrhea and abdominal pain, which were transient in nature; no gastrointestinal events led to discontinuation of maralixibat.

    Poster Presentation: H-ePwP-030: Gastrointestinal tolerability of maralixibat in patients with Alagille syndrome: An integrated analysis of short- and long-term treatment

    To be presented on Saturday, June 5, 2021 at 10:40 a.m. CEST

    Poster now available via the WCPGHAN ePoster exhibition section of the website and on Mirum's website in the Publications section.

    Data from more than five years of evaluation across three maralixibat Phase 2 clinical trials and their extension studies were analyzed to assess treatment-emergent adverse events of diarrhea and abdominal pain (GI events) in patients with ALGS. In the 86 patients evaluated, the analysis included incidence, severity, seriousness, time to onset, and duration of events, irrespective of relatedness to treatment with maralixibat, as determined by the investigator.

    Findings from the integrated safety study concluded:

    • Data from the 13-week placebo group demonstrated that rates of diarrhea were similar between maralixibat and placebo, with a slight difference in abdominal pain.
    • The majority of GI events occurred within the first four weeks of treatment and lasted less than one week in duration.
    • For patients who experienced GI events, the majority of diarrhea and abdominal pain were mild to moderate in severity and transient in nature.
    • There were no GI-related discontinuations of maralixibat over the five years of evaluation.

    About Mirum Pharmaceuticals, Inc.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    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.

    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 potential of maralixibat to be a meaningful alternative to the current standard of care for ALGS and PFIC2, and the regulatory approval pathway 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 "could," "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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  13. - Integrated safety analysis on gastrointestinal tolerability of maralixibat in patients with Alagille syndrome presented in poster session.

    - Five-year analysis demonstrating improvements in transplant-free survival for patients with PFIC2 featured in oral presentation; abstract nominated for the Alex Mowat Prize for best oral presentation in hepatology.

    - Mirum to host satellite symposium on June 3, 2021.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced that data from maralixibat studies in patients with Alagille syndrome and progressive familial intrahepatic cholestasis type 2 will be presented in oral and poster sessions during the 6th Annual World Congress of Pediatric Gastroenterology, Hepatology and Nutrition (WCPGHAN) taking…

    - Integrated safety analysis on gastrointestinal tolerability of maralixibat in patients with Alagille syndrome presented in poster session.

    - Five-year analysis demonstrating improvements in transplant-free survival for patients with PFIC2 featured in oral presentation; abstract nominated for the Alex Mowat Prize for best oral presentation in hepatology.

    - Mirum to host satellite symposium on June 3, 2021.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced that data from maralixibat studies in patients with Alagille syndrome and progressive familial intrahepatic cholestasis type 2 will be presented in oral and poster sessions during the 6th Annual World Congress of Pediatric Gastroenterology, Hepatology and Nutrition (WCPGHAN) taking place virtually June 2-5, 2021.

    Data featured during the congress include:

    Oral Presentation

    Abstract number: H-O-012: Serum bile acid control in long-term maralixibat treated patients is associated with native liver survival in children with progressive familial intrahepatic cholestasis due to bile salt export pump deficiency.

    Presenter: Professor Richard Thompson, M.D., Ph.D., Professor of Molecular Hepatology at King's College London and principal investigator for the INDIGO study

    Session: Highest Scoring Abstracts

    Date and time: Thursday, June 3, 2021 – 3:55-4:05 p.m. CEST

    This abstract has been nominated for the prestigious Annual Alex Mowat Prize for best oral presentation in hepatology. Results to be announced during the congress closing ceremony on June 5, 2021 at 1:30 p.m. CEST.

    Poster Presentation

    Abstract number: H-ePwP-030: Gastrointestinal tolerability of maralixibat in patients with Alagille syndrome: An integrated analysis of short- and long-term treatment.

    Presenter: Rakesh K. Raman, M.D., Mirum Pharmaceuticals, Inc.

    Session: ePoster Session Hepatology

    Date and time: Saturday, June 5, 2021 – 10:40-11:30 a.m. CEST

    Satellite Symposium

    Pediatric Cholestasis: Itching for an alternative to liver transplantation.

    Date and time: Thursday, June 3 at 5:30 p.m. CEST

    Featured Speakers: Dr. Binita M. Kamath (The Hospital for Sick Children (SickKids) Toronto, Ontario, Canada), Dr. Emmanuel Gonzales (Hépatologie Pédiatrique, Hôpital Bicêtre, AP-HP. Université Paris-Saclay, Le Kremlin-Bicêtre, France), Dr. Richard Thompson (King's College, London, UK)

    Mirum will be sponsoring a virtual symposium featuring leading experts who will discuss the current treatment landscape for pediatric cholestasis, including the high unmet medical need and the potential for pharmacologic treatments as an alternative to surgical options. This symposium will highlight diagnostic challenges related to Alagille syndrome and progressive familial intrahepatic cholestasis as well as the burden of disease on children and their caregivers. Data supporting the potential rationale for apical sodium-dependent bile acid transporter inhibition as an alternative to surgical treatment options will also be explored.

    WCPGHAN registered attendees can view the live symposium via the WCPGHAN website within the calendar of events in the Scientific Programme At-A-Glance section.

    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, Inc.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.

    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 potential benefits, development and commercialization of 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 "will," "could," "would," "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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  14. - NDA for maralixibat in Alagille syndrome (ALGS) granted priority review by U.S. FDA; PDUFA date is September 29, 2021
    - Received additional $65.0 million in funding from Oberland Capital based on NDA acceptance
    - Commercial team hired in anticipation of U.S. launch of maralixibat in ALGS
    - Entered into two licensing agreements with Vivet Therapeutics and CANbridge Pharmaceuticals
    - Conference call to provide business update today, May 6 at 1:30 p.m. PT/4:30 p.m. ET

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), today reported financial results for the quarter ended March 31, 2021 and provided a business update.

    "We have made significant progress toward launching maralixibat for Alagille syndrome. We have a Prescription Drug User Fee Act date…

    - NDA for maralixibat in Alagille syndrome (ALGS) granted priority review by U.S. FDA; PDUFA date is September 29, 2021

    - Received additional $65.0 million in funding from Oberland Capital based on NDA acceptance

    - Commercial team hired in anticipation of U.S. launch of maralixibat in ALGS

    - Entered into two licensing agreements with Vivet Therapeutics and CANbridge Pharmaceuticals

    - Conference call to provide business update today, May 6 at 1:30 p.m. PT/4:30 p.m. ET

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), today reported financial results for the quarter ended March 31, 2021 and provided a business update.

    "We have made significant progress toward launching maralixibat for Alagille syndrome. We have a Prescription Drug User Fee Act date of September 29, 2021, we have hired our sales, payer and field-based medical teams, and designed Mirum Access Plus, our patient services program," said Chris Peetz, president and chief executive officer at Mirum. "We believe our strong balance sheet supports all of our current and planned clinical programs in multiple cholestatic indications and the addition of our gene therapy collaboration with Vivet."

    Recent Key Operational Highlights

    • Received acceptance for filing and priority review of new drug application (NDA) for maralixibat in Alagille syndrome (ALGS) by the U.S. Food and Drug Administration (FDA).
    • Announced exclusive worldwide licensing deal with Vivet Therapeutics for two gene therapy programs.
    • Entered into exclusive licensing agreement for up to $120.0 million with CANbridge Pharmaceuticals for the development and commercialization of maralixibat in Greater China.
    • Completed staffing of U.S. commercial organization through hiring of key positions including marketing, market access, and sales.
    • Received $65.0 million additional funding from Oberland Capital in April 2021, related to NDA acceptance.

    Financial Results

    • Total operating expenses for the quarter ended March 31, 2021 were $47.6 million, compared to $22.0 million for the first quarter of 2020.
      • Research and development expenses for the first quarter ended March 31, 2021 were $38.1 million, compared to $17.3 million for the comparable prior-year period. The increase was primarily driven by milestone payments to Takeda and increased headcount costs.
      • General and administrative (G&A) expenses for the first quarter of 2021 were $9.5 million, compared to $4.7 million for the comparable prior-year period. G&A investment increased in the first quarter of 2021 versus the first quarter of 2020, primarily due to increased headcount costs and investments in launch preparation, including disease state awareness campaigns.
    • For the quarter ended March 31, 2021, Mirum reported a net loss of $50.5 million, or $1.68 per share, compared with a net loss of $21.3 million, or $0.86 per share for the same period in 2020.
    • As of March 31, 2021, Mirum had cash, cash equivalents, and short-term investments of $213.1 million.

    Upcoming Anticipated Milestones

    • Commercial
      • The FDA has issued a PDUFA date of September 29, 2021 and, if approved, Mirum plans to launch maralixibat for the treatment of cholestatic pruritus in patients with ALGS one year of age and older in the United States.
      • Mirum is planning to launch maralixibat for the treatment of PFIC2 in Europe in early 2022, if approved by the European Medicines Agency.
        • Mirum plans to file for ALGS as an additional indication in Europe following the PFIC2 approval, if received, based on results from the ICONIC study and six-year event-free survival analyses.
    • Pipeline and New Data:
      • Maralixibat:
        • Topline data from the Phase 3 MARCH PFIC study expected in early 2022.
        • New data from maralixibat studies to be presented at upcoming medical congresses.
        • Continuing enrollment in EMBARK Phase 2b study of maralixibat in biliary atresia.
      • Volixibat:
        • Continuing enrollment in Phase 2b OHANA study for intrahepatic cholestasis of pregnancy (ICP) and Phase 2b VISTAS study for primary sclerosing cholangitis (PSC), two potentially registrational studies. Interim analyses expected in 2022.
        • Phase 2b study in primary biliary cholangitis (PBC) planned for the second half of 2021.
      • Vivet: VTX-803 abstracts accepted for presentation at the American Society of Gene and Cell Therapy Annual Meeting in May 2021.

    Business Update Conference Call

    Mirum will host a conference call today, May 6, 2021 at 1:30 p.m. PT/4:30 p.m. ET, to provide a review of the first quarter activities and to discuss business updates. Join the call using the following details:

    Conference Call Details:

    U.S. toll-free:

    877-261-8990

    International:

    847-619-6441

    Passcode:

    50157000

    You may also access the call via webcast by visiting the Events & Presentations section on Mirum's website. A replay of this webcast will be available for 30 days.

    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 Volixibat

    Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the apical sodium dependent bile acid transporter (ASBT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of ASBT, thereby reducing bile acids systemically and in the liver. Phase 1 and Phase 2 studies 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 individuals 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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize two gene therapy programs, VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics, following preclinical evaluation and IND-enabling studies.

    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, 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 short-term investments on hand, 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

    March 31, 2021
    Three Months

    Ended

    March 31, 2020
     
    Operating expenses:
    Research and development

    $

    38,134

     

    $

    17,340

     

    General and administrative

     

    9,479

     

     

    4,692

     

    Total operating expenses (1)

     

    47,613

     

     

    22,032

     

     
    Loss from operations

     

    (47,613

    )

     

    (22,032

    )

    Interest income

     

    149

     

     

    749

     

    Interest expense

     

    (3,381

    )

     

    -

     

    Change in fair value of derivative liability

     

    334

     

     

    -

     

    Other expense, net

     

    (16

    )

     

    (23

    )

     
    Net loss before provision for income taxes

     

    (50,527

    )

     

    (21,306

    )

    Provision for income taxes

     

    5

     

     

    4

     

    Net loss

    $

    (50,532

    )

    $

    (21,310

    )

     
    Net loss per share, basic and diluted

    $

    (1.68

    )

    $

    (0.86

    )

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

     

    30,105,017

     

     

    24,704,651

     

     
     

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

     
    Research and development

    $

    2,743

     

    $

    1,041

     

    General and administrative

     

    2,542

     

     

    1,532

     

    Total stock-based compensation

    $

    5,285

     

    $

    2,573

     

    Mirum Pharmaceuticals, Inc.
    Selected Condensed Consolidated Balance Sheet Data
    (in thousands)

    March 31,

    2021

    December 31,

    2020

    (Unaudited)
     
    Cash, cash equivalents and short-term investments

    $

    213,113

     

    $

    231,820

     

    Working capital

     

    182,733

     

     

    217,888

     

    Total assets

     

    222,241

     

     

    240,864

     

    Accumulated deficit

     

    (223,703

    )

     

    (173,171

    )

    Total stockholders' equity

     

    133,883

     

     

    172,095

     

     

    View Full Article Hide Full Article
  15. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), today announced that the company will report financial results for the quarter ended March 31, 2021 on Thursday, May 6, 2021. That same day, Mirum will host a conference call and audio webcast at 1:30 p.m. PT/4:30 p.m. ET to provide a business update.

    Conference Call Details:

    U.S. toll-free:

    877-261-8990

    International:

    847-619-6441

    Passcode:

    50157000

    You may also access the call via webcast by visiting the Events & Presentations section on Mirum's website. A replay of this webcast will be available for 30 days.

    About Mirum Pharmaceuticals

    Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage…

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), today announced that the company will report financial results for the quarter ended March 31, 2021 on Thursday, May 6, 2021. That same day, Mirum will host a conference call and audio webcast at 1:30 p.m. PT/4:30 p.m. ET to provide a business update.

    Conference Call Details:

    U.S. toll-free:

    877-261-8990

    International:

    847-619-6441

    Passcode:

    50157000

    You may also access the call via webcast by visiting the Events & Presentations section on Mirum's website. A replay of this webcast will be available for 30 days.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize two gene therapy programs, VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics, following preclinical evaluation and IND-enabling studies.

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    View Full Article Hide Full Article
  16. -          CANbridge to lead development and commercialization in China; expands rare disease pipeline

    -          Mirum is entitled to receive up to an aggregate of $120.0 million in upfront and milestone payments, and significant double-digit tiered royalties

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) and CANbridge Pharmaceuticals, Inc., announced today that they have entered into a licensing agreement, pursuant to which CANbridge has agreed to develop and commercialize maralixibat in Greater China (China, Hong Kong, Macau and Taiwan). Maralixibat, an investigational, orally administered medication, is being evaluated in Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA).

    This press release…

    -          CANbridge to lead development and commercialization in China; expands rare disease pipeline

    -          Mirum is entitled to receive up to an aggregate of $120.0 million in upfront and milestone payments, and significant double-digit tiered royalties

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) and CANbridge Pharmaceuticals, Inc., announced today that they have entered into a licensing agreement, pursuant to which CANbridge has agreed to develop and commercialize maralixibat in Greater China (China, Hong Kong, Macau and Taiwan). Maralixibat, an investigational, orally administered medication, is being evaluated in Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA).

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210429005614/en/

    Maralixibat targets the apical sodium dependent bile acid transporter (ASBT), ultimately resulting in lower levels of bile acid systemically, which could mediate liver damage. The U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for maralixibat for the treatment of cholestatic pruritus in patients with ALGS under priority review. The European Medicines Agency is reviewing maralixibat for the treatment of PFIC2. Mirum has commenced a global Phase 2b maralixibat study (EMBARK) for the treatment of BA.

    Under the terms of the licensing agreement, CANbridge has obtained the exclusive right to develop and commercialize maralixibat within the Greater China regions for ALGS, PFIC, and BA. In exchange, Mirum is entitled to receive an $11.0 million upfront payment, R&D funding, and up to $109.0 million for the achievement of future regulatory and commercial maralixibat milestones, with significant double-digit tiered royalties based on product net sales.

    In collaboration with Mirum, CANbridge has agreed to oversee Mirum's clinical study sites in China, with the goal of accelerating enrollment of the global Phase 2b EMBARK study, which was recently initiated for patients with BA. CANbridge would also have the right to manufacture maralixibat in Greater China under certain conditions.

    "Maralixibat has the potential to be a transformative medication for certain cholestatic liver diseases and our goal is to ensure its availability to patients globally," said Chris Peetz, president and chief executive officer at Mirum. "CANbridge is a leading rare disease company in China and with their track record of commercial success, we believe they will be a strategic partner to accelerate the global launch of maralixibat, if approved."

    "We are thrilled to add maralixibat to our rare disease portfolio, as we expand into liver diseases and strengthen our pipeline with a late-stage asset in indications for which there are no approved treatments," said James Xue, Ph.D., Founder, Chairman and CEO of CANbridge Pharmaceuticals, Inc. "We are looking forward to participating in the global development of maralixibat in BA by supporting the China sites for the global Phase 2b EMBARK study, in collaboration with Mirum, and to working closely with Mirum to bring this treatment to patients and families in Greater China, where the need is great."

    About Mirum Pharmaceuticals, Inc.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    About CANbridge Pharmaceuticals Inc.

    CANbridge Pharmaceuticals Inc. is a biopharmaceutical company accelerating development and commercialization of treatments for orphan diseases and rare cancers to address unmet medical needs. CANbridge has a global partnership with WuXi Biologics to develop and commercialize proprietary therapeutics for the treatment of rare genetic diseases. In greater China, where it is a recognized leader in rare diseases, CANbridge has an exclusive licensing agreement to commercialize Hunterase®, an enzyme replacement therapy for the treatment of Hunter syndrome (also known as mucopolysaccharidosis type II), developed by GC Pharma and marketed in more than 12 countries worldwide. CANbridge also has entered into a strategic collaboration and licensing agreement with LogicBio Therapeutics to develop, manufacture and commercialize gene therapy candidates for treatments for Fabry and Pompe diseases. CANbridge also has a collaborative agreement with the Horae Gene Therapy Center at UMass Medical School for the research and development of gene therapies to treat rare genetic diseases.

    For more on CANbridge Pharmaceuticals Inc., please go to: www.canbridgepharma.com.

    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.

    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 potential development and commercialization by CANbridge of maralixibat in Greater China, Mirum's receipt of milestone and royalty revenue in connection with the license agreement with CANbridge, and the regulatory approval pathway 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 "will," "could," "would," "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.

    View Full Article Hide Full Article
  17. - Methods of use patent expected to provide patent protection through 2040.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced that it has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for a patent application No. 16/994,368. The allowed patent application covers methods of treating Alagille syndrome using maralixibat, an investigational treatment being evaluated for patients with Alagille syndrome, a rare and life-threatening cholestatic liver disease. The patent application is expected to issue in 2021 and will provide patent protection in the United States of the claimed methods of use of maralixibat into 2040.

    Mirum has submitted a new drug application (NDA) to the U.S. Food and Drug…

    - Methods of use patent expected to provide patent protection through 2040.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced that it has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for a patent application No. 16/994,368. The allowed patent application covers methods of treating Alagille syndrome using maralixibat, an investigational treatment being evaluated for patients with Alagille syndrome, a rare and life-threatening cholestatic liver disease. The patent application is expected to issue in 2021 and will provide patent protection in the United States of the claimed methods of use of maralixibat into 2040.

    Mirum has submitted a 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 one year of age and older. In March 2021, the NDA was accepted for priority review by the FDA and issued a PDUFA date of September 29, 2021. Maralixibat is also being evaluated in clinical studies for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC) and biliary atresia, both of which are also cholestatic liver diseases.

    A notice of allowance is a written notification issued after the USPTO makes a determination that a patent can be granted from an application. The majority of patent applications that receive a notice of allowance will proceed to issue as a U.S. patent. However, a notice of allowance is not a guarantee of patent issuance.

    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, Inc.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize two gene therapy programs, VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics, following preclinical evaluation and IND/CTA-enabling studies.

    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 date Mirum's patent application is expected to issue and the duration of such patent protection in the United States. 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 "will," "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.

    View Full Article Hide Full Article
  18. - Mirum expands potential opportunities across rare disease pipeline

    - Vivet to lead pre-clinical development of its two gene therapy programs for PFIC3 and PFIC2

    - Mirum gains option to license VTX-803 and VTX-802

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) and Vivet Therapeutics ("Vivet") announced today an agreement whereby Mirum has the exclusive option to develop and subsequently commercialize Vivet's two proprietary AAV gene therapy programs for progressive familial intrahepatic cholestasis (PFIC), subtypes 3 and 2. The two programs, VTX-803 and VTX-802, are currently being evaluated in preclinical studies by Vivet, a privately-held gene therapy biotechnology company.

    This press release features multimedia. View the full release here…

    - Mirum expands potential opportunities across rare disease pipeline

    - Vivet to lead pre-clinical development of its two gene therapy programs for PFIC3 and PFIC2

    - Mirum gains option to license VTX-803 and VTX-802

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) and Vivet Therapeutics ("Vivet") announced today an agreement whereby Mirum has the exclusive option to develop and subsequently commercialize Vivet's two proprietary AAV gene therapy programs for progressive familial intrahepatic cholestasis (PFIC), subtypes 3 and 2. The two programs, VTX-803 and VTX-802, are currently being evaluated in preclinical studies by Vivet, a privately-held gene therapy biotechnology company.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210412005354/en/

    Under the terms of the agreement, Vivet will continue to advance the preclinical studies for VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively. Mirum has the exclusive option to license the programs after which Mirum would lead the clinical development and any future commercialization of the programs. Until that time, Mirum will provide funding to support the continued research and development costs associated with the two gene therapy programs.

    "This gene therapy collaboration will work to address the root cause of PFIC3 and PFIC2 and provide an option for patients who do not respond to ASBT inhibition," said Chris Peetz, president and chief executive officer at Mirum. "We are encouraged by the expertise and dedication of the Vivet team to develop next-generation gene therapies that have the potential to transform the lives of patients and their families. There are clear synergies in our combined missions to help the people who need it the most."

    "Mirum's dedication to the treatment of cholestatic liver diseases and those rare diseases for which there are limited medications make them a great partner to potentially develop and bring to market VTX-803 and VTX-802," said Jean-Philippe Combal, chief executive officer and co-founder of Vivet. "Their leadership in rare liver disease and in particular PFIC, is important as they not only understand the urgent need for patients but also that today's treatment options will never be enough. Gene therapy is a transformative approach, potentially benefiting more patients."

    VTX-803 and VTX-802 are two proprietary AAV gene therapy programs of Vivet currently being evaluated in preclinical studies for progressive familial intrahepatic cholestasis (PFIC), subtypes 3 and 2. It is thought that successful correction by gene therapy of the defective MDR3 transporter and bile salt export pump (BSEP) functions for PFIC3 and PFIC2, respectively, may ultimately provide a cure for patients living with these rare liver diseases. Such an approach carries the potential to overcome the main limitations of current standard of care for PFIC3 and PFIC2. It may provide long-lasting benefits by restoring physiological bile secretion and preventing severe hepatic complications of the diseases and outweighing its related significant costs.

    Vivet has received Orphan Drug Designation for VTX-803 by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Additionally, preclinical proof-of-concept studies highlighting VTX-803 were published in Nature Communications in 2019. The data demonstrated sustained and significant reversal of PFIC3 disease biomarkers in a model of PFIC3.

    About Mirum Pharmaceuticals, Inc.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics, following preclinical evaluation and IND/CTA-enabling studies.

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    About Vivet Therapeutics

    Vivet Therapeutics is a clinical stage emerging biotechnology company developing novel gene therapy treatments for rare, inherited metabolic diseases.

    Vivet is building a diversified gene therapy pipeline based on novel recombinant adeno-associated virus (rAAV) technologies developed through its partnerships with, and exclusive licenses from, the Fundación para la Investigación Médica Aplicada (FIMA), a not-for-profit foundation at the Centro de Investigación Medica Aplicada (CIMA), University of Navarra based in Pamplona, Spain.

    Vivet's lead program, VTX-801, currently under IND clinical development with the GATEWAY clinical trial, is a novel investigational gene therapy for Wilson disease which has been granted Orphan Drug Designation (ODD) by the Food and Drug Administration (FDA) and the European Commission (EC). This rare genetic disorder is caused by mutations in the gene encoding the ATP7B protein, which reduces the ability of the liver and other tissues to regulate copper levels causing severe hepatic damages, neurologic symptoms and potentially death.

    Vivet's second gene therapy product, VTX-803 for PFIC3, received US and European Orphan Drug Designation in May 2020.

    Vivet is supported by international life science investors including Novartis Venture Fund, Roche Venture Fund, HealthCap, Pfizer Inc., Columbus Venture Partners, Ysios Capital, Kurma Partners and Idinvest Partners.

    Please visit us on www.vivet-therapeutics.com and follow us on Twitter at @Vivet_tx and LinkedIn.

    About PFIC

    Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder that causes progressive liver disease typically leading to liver failure. In people with PFIC, liver cells are less able to secrete bile. The resulting buildup of bile causes liver disease in affected individuals. Signs and symptoms of PFIC typically begin in infancy. Patients experience severe itching, jaundice, failure to grow at the expected rate (failure to thrive), and an increasing inability of the liver to function (liver failure). The disease is estimated to affect one in every 50,000 to 100,000 births in the United States and Europe. Six types of PFIC have been genetically identified, all of which are similarly characterized by impaired bile flow and progressive liver disease The PFIC2 patient population accounts for approximately 60% of the PFIC patient population. PFIC2 is caused by a mutation in the ABCB11 gene, which normally encodes a bile salt export pump protein that moves bile acids out of the liver.

    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 option and license agreement between Mirum and Vivet and the potential development of VTX-802 and VTX-803. 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," "anticipates," "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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  19. - PDUFA action date is September 29, 2021.

    - Priority review and Rare Pediatric Disease Designation granted.

    - FDA has indicated that advisory committee is not currently planned.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) announced today that its New Drug Application (NDA) for maralixibat, an oral apical sodium dependent bile acid transporter (ASBT) inhibitor for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older, has been accepted for filing and priority review by the U.S. Food and Drug Administration (FDA). ALGS is a rare liver disease for which there are currently no approved therapies. Mirum completed the rolling NDA submission in January 2021 and the Prescription Drug User Fee Act…

    - PDUFA action date is September 29, 2021.

    - Priority review and Rare Pediatric Disease Designation granted.

    - FDA has indicated that advisory committee is not currently planned.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) announced today that its New Drug Application (NDA) for maralixibat, an oral apical sodium dependent bile acid transporter (ASBT) inhibitor for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older, has been accepted for filing and priority review by the U.S. Food and Drug Administration (FDA). ALGS is a rare liver disease for which there are currently no approved therapies. Mirum completed the rolling NDA submission in January 2021 and the Prescription Drug User Fee Act (PDUFA) date, or FDA decision date, is September 29, 2021. Priority review is granted by the FDA for potential drugs that, if approved, would provide significant improvements in the effectiveness and safety of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. The FDA is not currently planning to hold an advisory committee.

    The maralixibat NDA submission is based on data from the Phase 2b ICONIC study. Data on reduction of pruritus, cholestasis, xanthomas, and improvements in quality of life have been previously presented and are available within the Publications and Presentations section on Mirum's website.

    "We are extremely pleased that our NDA will move forward in the regulatory review process, bringing maralixibat one step closer to being available for patients with Alagille syndrome," said Chris Peetz, president and chief executive officer at Mirum. "With more than six years of follow-up data showing durability of response as well as safety, we believe that maralixibat, if approved, would provide a meaningful treatment option that will ultimately reduce the need for liver transplantation."

    About Alagille syndrome

    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 ALGS 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, Inc.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. 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 regulatory approval path for maralixibat in the United States. 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," "anticipates," "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.

    References

    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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  20. Oversubscribed fund to continue investing in high-impact biopharma and medical device innovation

    RiverVest Venture Partners, a leading venture capital firm, announced today the closing of its RiverVest Venture Fund V, L.P. ("Fund V"), with $275 million of capital commitments in an oversubscribed fundraise. Fund V brings the firm's total assets under management to more than $1.6 billion.

    RiverVest invests in early-stage biopharma and medical device companies addressing significant unmet medical needs and has delivered consistently strong results to investors. Fund V is RiverVest's largest fund to date, reflecting commitments from a wide range of institutional investors, as well as family offices and individual investors. Fund V's limited partners…

    Oversubscribed fund to continue investing in high-impact biopharma and medical device innovation

    RiverVest Venture Partners, a leading venture capital firm, announced today the closing of its RiverVest Venture Fund V, L.P. ("Fund V"), with $275 million of capital commitments in an oversubscribed fundraise. Fund V brings the firm's total assets under management to more than $1.6 billion.

    RiverVest invests in early-stage biopharma and medical device companies addressing significant unmet medical needs and has delivered consistently strong results to investors. Fund V is RiverVest's largest fund to date, reflecting commitments from a wide range of institutional investors, as well as family offices and individual investors. Fund V's limited partners include most major investors from earlier RiverVest funds and several new institutional investors, enabled by the larger fund size.

    "With RiverVest Venture Fund V, we will continue our investment strategy grounded in close collaboration with entrepreneurs and academic investigators to develop products for the most pressing challenges patients face today," said Jay Schmelter, RiverVest's co-founder and managing director. "Fund V's larger size will enable RiverVest to participate more fully in later equity rounds of portfolio companies which have the greatest potential."

    RiverVest has a 20-year track record of success. Of the 55 companies in which RiverVest has invested, 18 have been successfully sold and eight have gone public, including Allakos (NASDAQ:ALLK), Mirum Pharmaceuticals (NASDAQ:MIRM) and most recently Spruce Biosciences (NASDAQ:SPRB) in October 2020. Having founded 15 companies to date, RiverVest is adept at founding companies with technology that originates from academic labs or that has been spun out from larger companies. RiverVest joins syndicates with peer venture firms globally to propel early-stage companies.

    Today, there are at least 27 commercial products treating patients and many in development from companies in which RiverVest has invested. They include drugs such as Lokelma, a treatment for hyperkalemia, a life-threatening condition caused by elevated potassium levels, developed by ZS Pharma and commercialized by AstraZeneca in 2018, and medical devices such as the Supera™ stent, used to treat peripheral artery disease, developed by IDEV Technologies and acquired by Abbott in 2013.

    "RiverVest approaches each investment in our concentrated portfolio with high conviction," said Schmelter. "Working as a team, we aggressively identify and create investment opportunities, we are thorough in our due diligence, and we aim to provide our portfolio companies collaborative scientific, operational, financial and business development expertise."

    With its disciplined investment approach, RiverVest will continue to create value for patients, entrepreneurs and investors. Headquartered in St. Louis and with offices in San Diego and Cleveland, RiverVest is at the intersection of outstanding medical research universities and flourishing innovation ecosystems, reviewing a diverse opportunity set and investing nationally.

    About RiverVest

    RiverVest is a leading venture capital firm building life science companies to address significant unmet needs of patients and deliver consistently strong returns to investors. With headquarters in St. Louis and offices in San Diego and Cleveland, RiverVest accesses forward-thinking research and clinical expertise at leading institutions across the country to found and fund biopharma and medical device companies.

    Select Commercial Products Developed by RiverVest's Portfolio Companies:

    • Lokelma – Treatment for adults with elevated potassium levels (ZS Pharma/AstraZeneca)
    • Vfend – Treatment for fungal infections (CyDex Pharmaceuticals/Pfizer)
    • Quinsair – Treatment for cystic fibrosis (Mpex Pharmaceuticals/Horizon)
    • Margenza – Treatment for breast cancer (MacroGenics)
    • Lutonix – Drug-coated angioplasty balloon (Lutonix/Bard)
    • Supera – Peripheral Stent System (IDEV Technologies/Abbott)
    • Emblem – Leadless Implantable Cardioverter Defibrillator (Cameron Health/Boston Scientific)

     

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  21. - U.S. launch of maralixibat for Alagille syndrome (ALGS) planned for second half of 2021, if approved by FDA

    - Phase 2b programs initiated in Primary Sclerosing Cholangitis, Intrahepatic Cholestasis of Pregnancy and Biliary Atresia

    - Financial runway expected to support maralixibat launch in U.S. and Europe and pipeline expansion over the next three years

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), today announced financial results for the fourth quarter and year-end 2020 and provided business updates.

    "In 2020, Mirum achieved several critical milestones, further advancing maralixibat for Alagille syndrome and PFIC2, and continuing to build value in our business by initiating the volixibat programs in adult cholestasis," said Chris Peetz…

    - U.S. launch of maralixibat for Alagille syndrome (ALGS) planned for second half of 2021, if approved by FDA

    - Phase 2b programs initiated in Primary Sclerosing Cholangitis, Intrahepatic Cholestasis of Pregnancy and Biliary Atresia

    - Financial runway expected to support maralixibat launch in U.S. and Europe and pipeline expansion over the next three years

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), today announced financial results for the fourth quarter and year-end 2020 and provided business updates.

    "In 2020, Mirum achieved several critical milestones, further advancing maralixibat for Alagille syndrome and PFIC2, and continuing to build value in our business by initiating the volixibat programs in adult cholestasis," said Chris Peetz, president and chief executive officer at Mirum. "We move into 2021 poised to launch maralixibat for Alagille syndrome in the United States, if approved, and with a strong cash runway expected to support three years of growth and expansion into additional orphan liver diseases."

    Key Operational Highlights and Business Updates

    • Completed rolling NDA submission to FDA for maralixibat for the treatment of pruritus in patients with Alagille syndrome (ALGS) one year of age and older in January 2021.
    • Received validation (acceptance) of Mirum's marketing authorization application (MAA) to the European Medicines Agency (EMA) for maralixibat for the treatment of patients with Progressive Familial Intrahepatic Cholestasis Type 2 (PFIC2).
    • Announced new maralixibat data highlighting more than five years of durable improvements in pruritus and quality of life in children with ALGS treated with maralixibat during AASLD.
    • Along with existing balance sheet, secured access to approximately $400 million in capital, with cash expected to support operations for the next three years including the planned launches of maralixibat and the advancement of Mirum's development pipeline.
    • Initiated Phase 2b VISTAS study evaluating volixibat in patients with primary sclerosing cholangitis, and the Phase 2b OHANA study evaluating volixibat in patients with intrahepatic cholestasis of pregnancy.
    • Initiated EMBARK Phase 2b study evaluating maralixibat in children with biliary atresia.
    • Launched "Unbearable ALGS" in January 2021, a disease education program intended to increase awareness of Alagille syndrome with the goal of improving time and rate of diagnosis.

    Financial Results

    • Total operating expenses for the quarter ended December 31, 2020 were $37.0 million, compared to $18.7 million for the fourth quarter of 2019. For the years ended December 31, 2020 and 2019, total operating expenses were $104.3 million and $54.7 million, respectively.
      • Research and development expenses for the fourth quarter were $29.7 million, compared to $14.4 million for the comparable prior-year period. For the years ended December 31, 2020 and 2019, research and development expenses were $81.6 million and $43.0 million, respectively. The increase in full year 2020 was primarily due to increased personnel related expenses, manufacturing activities to support Mirum's NDA, and higher consulting expenses.
      • General and administrative expenses for the fourth quarter of 2020 were $7.2 million, compared to $4.3 million for the comparable prior-year period. For the years ended December 31, 2020 and 2019, general and administrative expenses were $22.7 million and $11.8 million, respectively. The increase was primarily due to personnel and other compensation-related expenses.
    • For the quarter ended December 31, 2020, Mirum reported a net loss of $37.2 million, or $1.43 per share, compared with a net loss of $18.0 million, or $0.79 per share for the same period in 2019. For the year ended December 31, 2020, Mirum reported a net loss of $103.3 million, or $4.09 per share, compared to a net loss of $52.6 million, or $4.58 per share, for the same period in 2019.
    • As of December 31, 2020, Mirum had cash, cash equivalents, and investments of $231.8 million.

    Upcoming Anticipated Milestones

    • Commercial
      • Anticipated launch of maralixibat for the treatment of cholestatic pruritus in patients with ALGS one year of age and older in second half of 2021, if approved by the FDA.
      • Launch of maralixibat for the treatment of PFIC2 in Europe planned for early 2022, if approved by the EMA.
    • Pipeline
      • Maralixibat: Enrollment completion for Phase 3 MARCH PFIC study expected in the second quarter 2021.
      • Volixibat: Phase 2b study in primary biliary cholangitis planned for the second half of 2021.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. 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, another oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. 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, 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 per share amounts)

     

     

     

    Three Months Ended December 31,

     

    Year Ended December 31,

     

     

    2020

     

    2019

     

    2020

     

    2019

     

     

    (Unaudited)

     

     

     

    Operating expenses:

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

    Research and development

     

    $

    29,726

     

     

    $

    14,380

     

     

    $

    81,605

     

     

    $

    42,991

     

    General and administrative

     

     

    7,225

     

     

     

    4,278

     

     

     

    22,691

     

     

     

    11,752

     

    Total operating expenses (1)

     

     

    36,951

     

     

     

    18,658

     

     

     

    104,296

     

     

     

    54,743

     

    Loss from operations

     

     

    (36,951

    )

     

     

    (18,658

    )

     

     

    (104,296

    )

     

     

    (54,743

    )

    Interest income

     

     

    168

     

     

     

    747

     

     

     

    1,559

     

     

     

    2,232

     

    Interest expense

     

     

    (335

    )

     

     

     

     

     

    (335

    )

     

     

     

    Other expense, net

     

     

    (83

    )

     

     

    (20

    )

     

     

    (192

    )

     

     

    (21

    )

    Net loss before provision for income taxes

     

     

    (37,201

    )

     

     

    (17,931

    )

     

     

    (103,264

    )

     

     

    (52,532

    )

    Provision for income taxes

     

     

    2

     

     

     

    21

     

     

     

    6

     

     

     

    21

     

    Net Loss

     

    $

    (37,203

    )

     

    $

    (17,952

    )

     

    $

    (103,270

    )

     

    $

    (52,553

    )

    Net loss per share, basic and diluted

     

    $

    (1.43

    )

     

    $

    (0.79

    )

     

    $

    (4.09

    )

     

    $

    (4.58

    )

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

     

     

    26,106,102

     

     

     

    22,587,752

     

     

     

    25,251,968

     

     

     

    11,486,367

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

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

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

    Research and development

     

    $

    1,467

     

     

    $

    820

     

     

    $

    5,129

     

     

    $

    2,359

     

    General and administrative

     

     

    2,112

     

     

     

    1,247

     

     

     

    7,425

     

     

     

    3,711

     

    Total stock-based compensation

     

    $

    3,579

     

     

    $

    2,067

     

     

    $

    12,554

     

     

    $

    6,070

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

    Mirum Pharmaceuticals, Inc.

    Selected Consolidated Balance Sheet Data

    (in thousands)

     

     

     

    December 31,

    2020

     

    December 31,

    2019

     

     

     

     

     

     

     

     

     

    Cash, cash equivalents and investments

     

    $

    231,820

     

     

    $

    139,952

     

    Working capital

     

     

    217,888

     

     

     

    106,287

     

    Total assets

     

     

    240,864

     

     

     

    146,712

     

    Accumulated deficit

     

     

    (173,171

    )

     

     

    (69,901

    )

    Total stockholders' equity

     

     

    172,095

     

     

     

    130,349

     

     

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  22. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced that the company will participate in the following upcoming virtual investor conferences:

    SVB Leerink 10th Annual Global Healthcare Conference
    Friday, February 26, 2021 at 3:40 p.m. ET

    Raymond James Virtual Institutional Investors Conference
    Wednesday, March 3, 2021 at 3:00 p.m. ET

    H.C. Wainwright Global Life Sciences Conference
    The presentation will be available for viewing on Tuesday, March 9, 2021 at 7:00 a.m. ET.

    Oppenheimer 31st Annual Healthcare Conference
    Wednesday, March 17, 2021 at 4:30 p.m. ET

    The audio webcasts, on-demand presentation, and an archive of all presentations will be available in the Investors & Media section of Mirum's website at ir.mirumpharma.com.

    About

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced that the company will participate in the following upcoming virtual investor conferences:

    SVB Leerink 10th Annual Global Healthcare Conference

    Friday, February 26, 2021 at 3:40 p.m. ET

    Raymond James Virtual Institutional Investors Conference

    Wednesday, March 3, 2021 at 3:00 p.m. ET

    H.C. Wainwright Global Life Sciences Conference

    The presentation will be available for viewing on Tuesday, March 9, 2021 at 7:00 a.m. ET.

    Oppenheimer 31st Annual Healthcare Conference

    Wednesday, March 17, 2021 at 4:30 p.m. ET

    The audio webcasts, on-demand presentation, and an archive of all presentations will be available in the Investors & Media section of Mirum's 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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

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  23. - Maralixibat U.S. launch expected in second half of 2021, if approved

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced it has completed submission of its 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) one year of age and older. ALGS is a rare liver disease for which there are currently no approved therapies. The company is planning for the U.S. launch of maralixibat in the second half of 2021, should the medication be approved by the FDA for this indication.

    The NDA submission included the results of the ICONIC study and additional supportive studies totaling 86 patients with ALGS with up to six-year…

    - Maralixibat U.S. launch expected in second half of 2021, if approved

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced it has completed submission of its 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) one year of age and older. ALGS is a rare liver disease for which there are currently no approved therapies. The company is planning for the U.S. launch of maralixibat in the second half of 2021, should the medication be approved by the FDA for this indication.

    The NDA submission included the results of the ICONIC study and additional supportive studies totaling 86 patients with ALGS with up to six-year follow-up. The clinical safety data submitted for maralixibat includes over 1,600 individuals treated.

    "Patients, families and physicians have an urgent need for effective medications to address the severe and life-altering symptoms associated with ALGS," said Chris Peetz, president and chief executive officer at Mirum. "This submission brings us one step closer to a potential approval of maralixibat and providing a much-needed treatment option for the patients and their families suffering with ALGS."

    Until maralixibat is approved by the FDA for prescription, maralixibat is available to eligible patients with ALGS through Mirum's Expanded Access Program (EAP). The EAP is available in the United States, Canada, Australia and in regions throughout Europe. Requests for expanded access to maralixibat must be made by a licensed physician. Physicians and patients can learn more about the maralixibat EAP by visiting the program website at www.ALGSEAP.com or via Clinicaltrials.gov. Physicians in the United States and Canada who would like to request access for their patients can email MirumALGS@clinigengroup.com. Physicians in Europe and Australia can contact medicineaccess@clinigengroup.com with a reference line of "Mirum ALGS EAP request".

    Maralixibat was granted Rare Pediatric Disease Designation for ALGS and, as such, may qualify for receipt of a priority review voucher, if approved by the FDA. Maralixibat was also granted Breakthrough Therapy Designation for the treatment of pruritus associated with ALGS in patients one year of age and older. Maralixibat was granted Orphan Drug Designation by the FDA for the treatment of patients with PFIC and ALGS.

    About Alagille syndrome

    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 ALGS 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

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. 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 regulatory approval path for maralixibat in the United States. 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," "anticipates," "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.

    References

    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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  24. - The national campaign seeks to emphasize the unbearable burden ALGS has on patients and their families.

    - Mirum underscores its commitment to continued understanding of the rare liver disease through research and support of ALGS community.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced the launch of a disease awareness campaign for Alagille syndrome (ALGS), a rare cholestatic liver disease affecting one in every 30,000 births worldwide. The campaign seeks to broaden awareness and education of ALGS so that healthcare providers can more readily identify symptoms and diagnose patients with the goal to improve time to diagnosis and treatment.

    Children with ALGS live with severe and unrelenting symptoms that can impact their overall…

    - The national campaign seeks to emphasize the unbearable burden ALGS has on patients and their families.

    - Mirum underscores its commitment to continued understanding of the rare liver disease through research and support of ALGS community.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced the launch of a disease awareness campaign for Alagille syndrome (ALGS), a rare cholestatic liver disease affecting one in every 30,000 births worldwide. The campaign seeks to broaden awareness and education of ALGS so that healthcare providers can more readily identify symptoms and diagnose patients with the goal to improve time to diagnosis and treatment.

    Children with ALGS live with severe and unrelenting symptoms that can impact their overall quality of life. Identifying these symptoms early on can lead to a diagnosis and an effective treatment plan.

    "Many patients live for months or years before being diagnosed and some patients are misdiagnosed, leading to unnecessary and invasive surgeries that do not address the underlying cause of their liver disease," said Chris Peetz, president and chief executive officer at Mirum. "Our objective is to provide a platform that helps healthcare providers understand the specific signs and symptoms of ALGS so they can be better equipped to identify and diagnose this rare disease and help their patients get the treatment they need."

    "This disease awareness campaign has the potential to help healthcare providers recognize Alagille syndrome in their patients in order to properly address this rare disease," said Roberta Smith, president of the Alagille syndrome Alliance and parent of a child with ALGS. "The impacts of Alagille syndrome on families are many. We have only begun to scratch the surface in understanding disease burden and specifically, how different regions in the world are affected differently. As we celebrate International Alagille Syndrome Awareness Day, we will focus on sharing Alagille syndrome family stories that emphasize the critical need for continued research and treatment options for patients worldwide. The patient voice is the Alagille syndrome community's greatest vehicle for change."

    Mirum's goal is to advance the understanding and diagnosis of ALGS so that patients and their families will get the medical care and support they need to improve their quality of life. The company and its founders have been dedicated to understanding ALGS through the support of resources like the Global Alagille Alliance (GALA) for continued research, as well as genetic testing platforms that help effectively identify ALGS in patients. These resources assist in advancing research and the healthcare community's understanding of this devastating rare disease.

    Visit the Unbearable ALGS website to learn more about Alagille syndrome and the significant impact it has on patients and their families.

    Alagille syndrome Awareness Day – January 24, 2021

    Mirum is also proud to champion the Alagille syndrome Alliance and their ongoing work to support ALGS families through education, outreach, and community. This year, in celebration of International ALGS Awareness Day, they are hosting a 24-hour Aware-a-Thon that will include family stories, messages, expert talks, and educational resources aimed at increasing the public's awareness of ALGS, a crucial element in their mission. To learn more about how to get involved and support the ALGS Alliance and the ALGS community, visit their Awareness Day website.

    About Alagille syndrome

    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 ALGS 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 Mirum

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum 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, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. 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 regulatory approval path for maralixibat in the United States and the European Union, and Mirum's ALGS disease awareness campaign. 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," "goal", "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.

    References

    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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  25. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced that the first patient has been enrolled in Mirum's Phase 2b VISTAS study evaluating volixibat in adult patients with primary sclerosing cholangitis (PSC), an idiopathic chronic cholestatic liver disease characterized by progressive inflammation and destruction of bile ducts often leading to serious liver disease, cancer, and ultimately liver failure. Volixibat, an oral, minimally absorbed medication, is designed to offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids through inhibition of the apical sodium dependent bile acid transporter (ASBT), thereby reducing bile acids systemically and in the liver.

    Patients meeting eligibility…

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced that the first patient has been enrolled in Mirum's Phase 2b VISTAS study evaluating volixibat in adult patients with primary sclerosing cholangitis (PSC), an idiopathic chronic cholestatic liver disease characterized by progressive inflammation and destruction of bile ducts often leading to serious liver disease, cancer, and ultimately liver failure. Volixibat, an oral, minimally absorbed medication, is designed to offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids through inhibition of the apical sodium dependent bile acid transporter (ASBT), thereby reducing bile acids systemically and in the liver.

    Patients meeting eligibility criteria for the study are randomized to receive volixibat 20 mg twice daily (BID) or 80 mg BID, or placebo for 28 weeks, after which time all patients will receive volixibat through the open-label extension phase of the study.

    The primary endpoint will evaluate the change in pruritus from baseline for volixibat versus placebo using the Adult ItchRO tool. Secondary endpoints evaluated will include safety and tolerability, quality of life measures, and serum bile acids. Exploratory endpoints will include assessments of liver fibrosis and other markers of PSC progression.

    "This study is an important step forward in evaluating the potential clinical benefit of volixibat in adult patients with primary sclerosing cholangitis," said Christopher Bowlus, MD, chief of gastroenterology and hepatology at UC Davis Health. "There are currently no approved therapies indicated for the treatment of patients with PSC suffering from pruritus, a common and often debilitating symptom of the disease."

    "We are excited to initiate the volixibat Phase 2 VISTAS study for patients with primary sclerosing cholangitis," said Ed Tucker, MD, chief medical officer at Mirum. "Our learnings from the maralixibat development program have informed the design of our volixibat study with the hope of alleviating the burdensome effects experienced by patients with PSC."

    Mirum has ongoing studies evaluating cholestatic liver diseases in pediatric patients with maralixibat, also an ASBT inhibitor. The company has initiated a rolling NDA submission to the U.S. Food and Drug Administration (FDA) for maralixibat for the treatment of cholestatic pruritus associated with Alagille syndrome, which Mirum expects to complete this quarter. Mirum is planning for a U.S. launch in the second half of 2021, should Mirum receive FDA approval for this indication. Until that time, maralixibat is available to eligible patients in the United States, Canada, Australia and in regions throughout Europe through an Expanded Access Program. Additionally, Mirum submitted a marketing authorization application which was accepted (validated) by the European Medicines Agency for maralixibat for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC), type 2.

    About Primary Sclerosing Cholangitis

    Primary sclerosing cholangitis (PSC) is a rare, serious, idiopathic chronic cholestatic liver disease characterized by cholestasis, progressive inflammation, and destruction of bile ducts, which may lead to fibrosis, cirrhosis, portal hypertension, cancer, and ultimately liver failure. It is estimated that approximately 29,000 people in the United States and approximately 50,000 people in Europe suffer from PSC with no approved therapies. The underlying etiology of PSC is not completely understood, but it is thought to arise from a combination of genetic and environmental factors. The median age at diagnosis is approximately 35 years, and approximately 70% of PSC patients have inflammatory bowel disease, principally ulcerative colitis. Signs and symptoms of PSC may include, but are not limited to, pruritus, extreme fatigue, jaundice, and abdominal discomfort. The eventual buildup of bile acids from continued bile duct injury and obstruction damages liver cells and is thought to contribute to the progression of liver failure. Complications involving the biliary tree are common and include cholangitis, as well as ductal strictures and gallstones, the latter of which frequently require endoscopic or surgical interventions. PSC also increases the risk of development of malignancies, with cholangiocarcinoma being the most common. PSC is the fifth leading indication for liver transplantation; however, the post-transplant recurrence rate of PSC is up to 25%.

    About Volixibat

    Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the apical sodium dependent bile acid transporter (ASBT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of ASBT, thereby reducing bile acids systemically and in the liver. Phase 1 and Phase 2 studies 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 individuals across multiple clinical trials. The most common adverse events reported were mild to moderate gastrointestinal events observed in the volixibat groups.

    About Mirum

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has initiated a rolling NDA submission for maralixibat for 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, to evaluate its potential in treating primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. 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 conduct and progress of Mirum's ongoing and planned studies for 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," "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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  26. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced that it will present at the annual J.P. Morgan Healthcare Conference taking place virtually January 11-14, 2021.

    Chris Peetz, Mirum's president and chief executive officer, will provide an overview of Mirum's strategy, as well as the company's commercialization plans for maralixibat and its ongoing development of both maralixibat and volixibat. Mr. Peetz and members of Mirum's leadership team will be available for one-on-one meetings during the conference.

    Presentation and Q&A Session

    Date: Tuesday, January 12, 2021
    Time: 5:20 – 6:00 p.m. ET

    A live audio webcast and archive of Mirum's presentation along with accompanying slides will be available in the Investors & Media section of…

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced that it will present at the annual J.P. Morgan Healthcare Conference taking place virtually January 11-14, 2021.

    Chris Peetz, Mirum's president and chief executive officer, will provide an overview of Mirum's strategy, as well as the company's commercialization plans for maralixibat and its ongoing development of both maralixibat and volixibat. Mr. Peetz and members of Mirum's leadership team will be available for one-on-one meetings during the conference.

    Presentation and Q&A Session

    Date: Tuesday, January 12, 2021

    Time: 5:20 – 6:00 p.m. ET

    A live audio webcast and archive of Mirum's presentation along with accompanying slides will be available in the Investors & Media section of the company's website at ir.mirumpharma.com.

    Additionally, the company will present during a fireside chat at the upcoming H.C. Wainwright Virtual BIOCONNECT 2021 Conference. A webcast of the presentation will be accessible from the Investors & Media section of Mirum's website at ir.mirumpharma.com beginning January 11, 2021.

    About Mirum

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum 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.

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  27. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases, today announced the pricing of its previously announced underwritten public offering of 3,750,000 shares of its common stock at a price to the public of $20.00 per share. The gross proceeds to Mirum from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be $75.0 million.

    In addition, Mirum has granted the underwriters a 30-day option to purchase up to an additional 562,500 shares of common stock at the public offering price less underwriting discounts and commissions. The offering is expected…

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases, today announced the pricing of its previously announced underwritten public offering of 3,750,000 shares of its common stock at a price to the public of $20.00 per share. The gross proceeds to Mirum from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be $75.0 million.

    In addition, Mirum has granted the underwriters a 30-day option to purchase up to an additional 562,500 shares of common stock at the public offering price less underwriting discounts and commissions. The offering is expected to close on December 17, 2020, subject to satisfaction of customary closing conditions.

    J.P. Morgan, SVB Leerink and Evercore ISI are acting as joint book-running managers for the offering. H.C. Wainwright & Co. is acting as lead manager for the offering.

    The offering is being made pursuant to a shelf registration statement on Form S-3, including a base prospectus, that was previously filed with the Securities and Exchange Commission (the "SEC") and was declared effective by the SEC on August 12, 2020. A preliminary prospectus supplement and accompanying prospectus relating to the offering were filed with the SEC and are available for free on the SEC's website located at http://www.sec.gov. A final prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available for free on the SEC's website located at http://www.sec.gov. Copies of the final prospectus supplement and the accompanying prospectus relating to the offering, when available, may be obtained from: J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by email at prospectus-eq_fi@jpmchase.com or by phone at (866) 803-9204; SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by email at syndicate@svbleerink.com or by phone at (800) 808-7525, ext. 6132; or Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, New York, NY 10055, by email at ecm.prospectus@evercore.com or by phone at (888) 474-0200.

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

    About Mirum Pharmaceuticals, Inc.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has initiated a rolling New Drug Application 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.

    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 regulatory approval path for maralixibat and Mirum's expectations regarding the completion and timing of the offering. 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 SEC. 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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  28. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases, today announced that it has commenced an underwritten public offering, subject to market and other conditions, to issue and sell $75.0 million of shares of its common stock. In connection with the proposed offering, Mirum expects to grant the underwriters a 30-day option to purchase up to an additional approximately $11.3 million of shares of common stock. There can be no assurance as to whether or when the proposed offering may be completed or as to the actual size or terms of the proposed offering.

    J.P. Morgan, SVB Leerink and Evercore ISI are…

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases, today announced that it has commenced an underwritten public offering, subject to market and other conditions, to issue and sell $75.0 million of shares of its common stock. In connection with the proposed offering, Mirum expects to grant the underwriters a 30-day option to purchase up to an additional approximately $11.3 million of shares of common stock. There can be no assurance as to whether or when the proposed offering may be completed or as to the actual size or terms of the proposed offering.

    J.P. Morgan, SVB Leerink and Evercore ISI are acting as joint book-running managers for the proposed offering. H.C. Wainwright & Co. is acting as lead manager for the proposed offering.

    The proposed offering is being made pursuant to a shelf registration statement on Form S-3, including a base prospectus, that was previously filed with the Securities and Exchange Commission (the "SEC") and was declared effective by the SEC on August 12, 2020. A preliminary prospectus supplement and accompanying prospectus relating to the proposed offering will be filed with the SEC and will be available for free on the SEC's website located at http://www.sec.gov. Copies of the preliminary prospectus supplement and the accompanying prospectus relating to the proposed offering, when available, may be obtained from:

    J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by email at prospectus-eq_fi@jpmchase.com or by phone at (866) 803-9204; SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by email at syndicate@svbleerink.com or by phone at (800) 808-7525, ext. 6132; or Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, New York, NY 10055, by email at ecm.prospectus@evercore.com or by phone at (888) 474-0200.

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

    About Mirum Pharmaceuticals, Inc.

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has initiated a rolling New Drug Application 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.

    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 regulatory approval path for maralixibat, Mirum's expectations regarding the completion, timing and size of the proposed offering and Mirum's expectations with respect to granting the underwriters a 30-day option to purchase additional shares. 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 SEC. 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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  29. - $60 million upfront, including a $10 million equity investment, $65 million at FDA's acceptance of NDA for maralixibat for ALGS, $35 million upon FDA approval of maralixibat in ALGS, and up to $50 million to fund in-licensing and acquisitions

    - Funding agreement to support the commercialization of maralixibat for the treatment of cholestatic pruritus in patients with ALGS in the United States and for patients with PFIC2 in Europe, and for continued development of Mirum's pipeline of treatments to address cholestatic liver diseases in children and adults

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) and Oberland Capital Management LLC (Oberland Capital) today announced a $200 million capped, tiered, revenue-based funding agreement based on…

    - $60 million upfront, including a $10 million equity investment, $65 million at FDA's acceptance of NDA for maralixibat for ALGS, $35 million upon FDA approval of maralixibat in ALGS, and up to $50 million to fund in-licensing and acquisitions

    - Funding agreement to support the commercialization of maralixibat for the treatment of cholestatic pruritus in patients with ALGS in the United States and for patients with PFIC2 in Europe, and for continued development of Mirum's pipeline of treatments to address cholestatic liver diseases in children and adults

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) and Oberland Capital Management LLC (Oberland Capital) today announced a $200 million capped, tiered, revenue-based funding agreement based on net revenues of maralixibat, as well as a $10 million equity investment agreement. Under the agreements, Mirum will receive:

    • $60 million by December 31, 2020, including a $10 million equity investment;
    • $65 million upon announcement of the U.S. Food and Drug Administration's (FDA) acceptance of Mirum's New Drug Application (NDA) for maralixibat for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS);
    • $35 million upon announcement of FDA approval of maralixibat for cholestatic pruritus in patients with ALGS; and
    • $50 million, at the option of Oberland Capital, which may be funded to support in-licensing or acquisition of assets to further build upon Mirum's development and commercialization pipeline in the pursuit of treatments for rare liver diseases.

    Mirum is submitting a rolling NDA to the FDA for maralixibat for the treatment of cholestatic pruritus in patients with ALGS, which it expects to complete in the first quarter of 2021, with launch planned in the second half of 2021. Mirum has also submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for maralixibat for the treatment of patients with progressive familial intrahepatic cholestasis type 2 (PFIC2), or bile salt export pump (BSEP) deficiency. The MAA was recently validated by the EMA and Mirum is preparing for launch in the European Union in the first quarter of 2022.

    "The funding from Oberland Capital will enable us to invest in the launch and commercialization of maralixibat in the United States and Europe," said Ian Clements, PhD, chief financial officer at Mirum. "The agreement with Oberland Capital provides us the support and flexibility to advance our pipeline focused on rare liver diseases for children and adults, an area where there is tremendous need."

    "We are pleased to support Mirum as they seek to commercialize new medicines for diseases with high unmet need," said Andrew Rubinstein, managing partner of Oberland Capital. "The Mirum team has a clear vision and the potential to bring a transformative medication to the ALGS and PFIC2 patient communities and to establish itself as the leader in the treatment of cholestatic liver diseases."

    Citi acted as Financial Advisor to Mirum on the transaction.

    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

    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. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum 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.

    About Oberland Capital

    Oberland Capital, a private investment firm with over $2.25 billion in capital commitments since inception, is focused exclusively on investing in the global healthcare industry and specializes in flexible investment structures customized to meet the specific capital requirements and strategic objectives of transaction partners. Oberland Capital's broad suite of financing solutions includes monetization of royalty streams, acquisition of future product revenues, creation of project-based financing structures, and investments in traditional debt and equity. With a combination of deep industry knowledge and extensive structured finance experience, the Oberland Capital team has a history of creating value for its transaction partners. The firm was founded in 2013 by Jean-Pierre Naegeli and Andrew Rubinstein.

    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 regulatory approval path for maralixibat in the United States and the European Union, and Mirum's ability to meet funding conditions under its financing arrangements and access to capital thereunder and consummate acquisitions of assets or in-licenses. 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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  30. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRUM) today announced that it will host its inaugural Virtual Investor Day on December 9, 2020 from 11:00 a.m. – 1:30 p.m. ET.

    The webcast will include an overview of Mirum's strategy and commercial plans for its lead investigational medication, maralixibat, as well as development progress for maralixibat and volixibat, both of which are being evaluated for the treatment of pediatric and adult patients with cholestatic liver diseases.

    Chris Peetz, Mirum's President and Chief Executive Officer, will be joined by members of the company's management team, as well as industry experts, Philip Rosenthal, MD, Director of Pediatric Hepatology, University of California, San Francisco, and Catherine Williamson…

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRUM) today announced that it will host its inaugural Virtual Investor Day on December 9, 2020 from 11:00 a.m. – 1:30 p.m. ET.

    The webcast will include an overview of Mirum's strategy and commercial plans for its lead investigational medication, maralixibat, as well as development progress for maralixibat and volixibat, both of which are being evaluated for the treatment of pediatric and adult patients with cholestatic liver diseases.

    Chris Peetz, Mirum's President and Chief Executive Officer, will be joined by members of the company's management team, as well as industry experts, Philip Rosenthal, MD, Director of Pediatric Hepatology, University of California, San Francisco, and Catherine Williamson, MD, FRCP, FMedSci, Professor of Women's Health at King's College London.

    WEBCAST AND DIAL-IN DETAILS

    Webcast link: https://edge.media-server.com/mmc/p/cphus5mg

    Toll-free dial-in: (844) 877-4019

    International toll: (602) 563-8495

    Conference ID: 1191826

    A replay of the webcast will be available in the Investor Relations section of Mirum's website following the live event.

    About Mirum

    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.

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  31. - 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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  32. 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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  33. - 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

    View Full Article Hide Full Article
    • 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.

    View Full Article Hide Full Article
  34. - 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

     

     

    View Full Article Hide Full Article
    • Maralixibat Expanded Access Program now available for patients with pruritus associated with Alagille syndrome in Australia and 10 countries in Europe.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced it has broadened its Expanded Access Program (EAP) for maralixibat for the treatment of patients with cholestatic pruritus associated with Alagille syndrome (ALGS) to Australia and countries in Europe. The announcement follows the Company's recent initiation of an EAP in the United States and Canada for maralixibat in the same indication.

    "Maralixibat has the potential to make a meaningful difference in…

    • Maralixibat Expanded Access Program now available for patients with pruritus associated with Alagille syndrome in Australia and 10 countries in Europe.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced it has broadened its Expanded Access Program (EAP) for maralixibat for the treatment of patients with cholestatic pruritus associated with Alagille syndrome (ALGS) to Australia and countries in Europe. The announcement follows the Company's recent initiation of an EAP in the United States and Canada for maralixibat in the same indication.

    "Maralixibat has the potential to make a meaningful difference in the treatment of Alagille syndrome and it is our goal to help ensure that eligible patients have access to maralixibat until it is available for prescribing in Europe and Australia," said Chris Peetz, president and chief executive officer at Mirum. "Continuity of treatment is critically important for children with rare diseases, like Alagille syndrome, and we hope that early access to investigational treatments such as maralixibat will help to reduce many of the debilitating symptoms associated with the disease as well as the need for liver transplantation."

    Maralixibat is a novel, minimally absorbed, orally administered apical sodium dependent bile acid transporter inhibitor being evaluated for the treatment of cholestatic pruritus in patients with ALGS. Mirum initiated its rolling New Drug Application (NDA) for the treatment of cholestatic pruritus in patients with ALGS to the U.S. Food and Drug Administration (FDA) in September 2020, expects to complete the submission in the first quarter of 2021, and is preparing for a U.S. commercial launch in the second half of 2021.

    About the Maralixibat Expanded Access Program

    The EAP, sometimes referred to as "compassionate use," provides a potential pathway for a patient with an immediately life-threatening condition or serious disease to gain access to an investigational medicine for the treatment of that disease outside of a clinical trial when no comparable or satisfactory alternative therapy options are available.

    The goal of Mirum's EAP is to provide access to maralixibat for the treatment of cholestatic pruritus in eligible patients with ALGS prior to regulatory approval of the medication and until maralixibat is available by prescription.

    The EAP will be open to eligible patients in Australia, Austria, Belgium, Canada, France, Germany, Italy, Netherlands, Poland, Spain, Sweden, the United States, and the United Kingdom.

    Requests for expanded access to maralixibat must be made by a licensed physician. Physicians and patients can learn more about the maralixibat EAP for ALGS by visiting the program website at www.ALGSEAP.com or via https://clinicaltrials.gov/ct2/show/NCT04530994. Physicians in select European countries and Australia who would like to request access for their patients can contact medicineaccess@clinigengroup.com with a reference line of "Mirum ALGS EAP request". Physicians in the United States and Canada can email MirumALGS@clinigengroup.com.

    For patients with progressive familial intrahepatic cholestasis (PFIC), access to maralixibat is possible through our MARCH Phase 3 study, which is currently open to enrollment. More information can be found at https://pfictrial.com. Mirum plans to continually evaluate the need for expanded access to maralixibat as studies reach enrollment milestones.

    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 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 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 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.

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    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, Mirum's Expanded Access Program for maralixibat, 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," "will", "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.

    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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    • Late-breaker oral presentation features long-term data from Phase 2 ITCH and IMAGINE II studies evaluating maralixibat in pediatric 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 today announced that new data from the company's 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 (AASLD), taking place November 13-16, 2020.

    Late-breaker Oral Presentation

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

    • Late-breaker oral presentation features long-term data from Phase 2 ITCH and IMAGINE II studies evaluating maralixibat in pediatric 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 today announced that new data from the company's 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 (AASLD), taking place November 13-16, 2020.

    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.

    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 inves