1. - 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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  2. 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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  3. - 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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  4. 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.

    View Full Article Hide Full Article
  5. - 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 . Physicians in Europe and Australia can contact 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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  6. - 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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  7. 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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  8. 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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  9. 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 or by phone at (866) 803-9204; SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by email at 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 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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  10. 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 or by phone at (866) 803-9204; SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by email at 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 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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  11. - $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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  12. 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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  13. - 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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  14. 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.

    View Full Article Hide Full Article
  15. - Results of long-term treatment from the ITCH and IMAGINE II studies presented in late-breaking oral presentation at AASLD

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

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

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

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

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

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

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

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

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

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

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

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

    About the ITCH and IMAGINE II study

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

    About Alagille Syndrome

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

    About Maralixibat

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

    About Mirum Pharmaceuticals

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

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

    Follow Mirum on Twitter, Facebook and LinkedIn.

    Forward-Looking Statements

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

    About Maralixibat

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

    About Volixibat

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

    About Mirum Pharmaceuticals

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

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

    Forward-Looking Statements

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

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  16. - 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 with a reference line of "Mirum ALGS EAP request". Physicians in the United States and Canada can email .

    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.

    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, 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 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 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 C4 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", "believes," "anticipates," "expects," "intends," "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
  17. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases today announced that data from the Company's maralixibat studies will be presented at the North American Society for Pediatric, Gastroenterology, Hepatology and Nutrition (NASPGHAN) Annual Meeting 2020, being held November 1-7, 2020.

    Maralixibat presentations are as follows:

    Oral Presentation

    Abstract 243: Durability of treatment effect with long-term maralixibat use in children with Alagille syndrome: 4-year safety and efficacy results from the ICONIC study
    November 2, 2020 from 3:00-4:30 p.m. ET (oral presentation at 4:02 p.m. ET)

    Poster Presentation

    Abstract 646: Genotype

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases today announced that data from the Company's maralixibat studies will be presented at the North American Society for Pediatric, Gastroenterology, Hepatology and Nutrition (NASPGHAN) Annual Meeting 2020, being held November 1-7, 2020.

    Maralixibat presentations are as follows:

    Oral Presentation

    Abstract 243: Durability of treatment effect with long-term maralixibat use in children with Alagille syndrome: 4-year safety and efficacy results from the ICONIC study

    November 2, 2020 from 3:00-4:30 p.m. ET (oral presentation at 4:02 p.m. ET)

    Poster Presentation

    Abstract 646: Genotype and dose-dependent response to maralixibat in patients with bile salt export pump deficiency

    November 6, 2020 from 11:00 a.m. – 1:00 p.m. ET

    The abstracts from the meeting are available on the Journal of Pediatric Gastroenterology and Nutrition website.

    Virtual Symposium

    "Solving the Puzzle of Pediatric Cholestasis"

    November 2, 2020 – 4:30-6:00 p.m. ET

    Mirum will be sponsoring a virtual symposium led by Dr. Philip Rosenthal with presentations on Alagille syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC) and Biliary Atresia (BA), by Dr. Binita Kamath and Dr. Richard Thompson. To attend the symposium, you must be registered for NASPGHAN. Once registered, the congress will email an invitation to the event.

    "We are pleased to share data from our maralixibat studies at the NASPGHAN meeting as it provides a forum for scientific exchange with leaders in the pediatric gastroenterology and hepatology community who treat children with rare diseases like ALGS and PFIC," said Chris Peetz, president and chief executive officer of Mirum. "The data demonstrate long-term treatment success of more than five years for patients with pruritus associated with ALGS, based on findings from the ICONIC study. Also being shared are data highlighting five-year transplant-free survival in patients with PFIC2 who responded to maralixibat. These data underscore our confidence in maralixibat to be a medicine that has the potential to change the treatment paradigms for ALGS and PFIC2."

    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 eligible 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 of 2020.

    The company is also developing volixibat, 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 and progress of Mirum's studies for maralixibat, as well as 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", "believes," "anticipates," "expects," "intends," "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
  18. FOSTER CITY, Calif., Oct. 15, 2020 /PRNewswire/ -- 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 partnered with EVERSANA™, a leading provider of commercial services to the life science industry, to lead the U.S. market access, distribution and patient services for maralixibat.

    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 Alagille syndrome (ALGS). Mirum initiated its rolling New Drug Application (NDA) to the U.S. Food and Drug Administration in August 2020, expects to complete the NDA submission in the first quarter of 2021, and is preparing for launch in the second half of 2021.

    Mirum will utilize EVERSANA's commercialization platform to provide integrated nationwide distribution, specialty pharmacy, patient services and Hub support for maralixibat, if approved.

    "We are working diligently to prepare for the potential launch of maralixibat in the United States and one of the most important components is ensuring comprehensive support for patients and their families," said Chris Peetz, president and chief executive officer at Mirum. "We are pleased to partner with EVERSANA and are confident in their ability to provide end-to-end services to ensure maralixibat will reach patients and their families efficiently and effectively once the medication is available for prescribing. In parallel, we are preparing to file a marketing authorization application for maralixibat in PFIC2 this year, and these programs will help to refine our strategies as we finalize launch plans in Europe."

    "Rare diseases like ALGS demand more than traditional patient support or distribution models. From day one, we knew that our partnership would focus entirely on individual patient's needs at each step of their therapeutic and treatment journey. The platform is designed so that at launch, EVERSANA'S infrastructure and experts will be ready with the right services, and each action will be continually optimized through real-time analytics," said Jim Lang, chief executive officer of EVERSANA. "Mirum's patients will have access to comprehensive support delivered by Mirum and EVERSANA, two companies equally commited to the rare disease space."

    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 1,700 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 FDA 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, abdominal pain, and vomiting. 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.In patients with ALGS, multiple organ systems may be affected by the mutation, including the liver, heart, kidneys and central nervous system. 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. 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). 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.

    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 for 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 TwitterFacebook 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 regulatory approval path for maralixibat, and the potential launch of maralixibat, if approved, the market access, distribution and patient services of maralixibat to be provided by Eversana, and Mirum's Expanded Access Program 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", "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.

    About EVERSANA™

    EVERSANA™ is the leading provider of global services to the life sciences industry. The company's integrated solutions are rooted in the patient experience and span all stages of the product lifecycle to deliver long-term, sustainable value for patients, prescribers, channel partners and payers. The company serves more than 500 organizations, including innovative start-ups and established pharmaceutical companies, to advance life science solutions for a healthier world. To learn more about EVERSANA, visit eversana.com or connect through LinkedIn and Twitter.

    MIRUM Contacts:

    Investor Contact:

    Ian Clements, Ph.D.

    Media Contact:

    Erin Murphy

    EVERSANA Contact:

    Sarah Zwicky

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/mirum-pharmaceuticals-announces-partnership-with-eversana-to-support-launch-and-commercialization-of-maralixibat-for-alagille-syndrome-in-the-united-states-301153053.html

    SOURCE EVERSANA

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  19. Agreement outlines commercialization and distribution services to support patients diagnosed 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 it has partnered with EVERSANA™, a leading provider of commercial services to the life science industry, to lead the U.S. market access, distribution and patient services for maralixibat.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201015005342/en/

    Maralixibat is a novel, minimally absorbed, orally administered apical sodium dependent bile acid transporter inhibitor being evaluated…

    Agreement outlines commercialization and distribution services to support patients diagnosed 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 it has partnered with EVERSANA™, a leading provider of commercial services to the life science industry, to lead the U.S. market access, distribution and patient services for maralixibat.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201015005342/en/

    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 Alagille syndrome (ALGS). Mirum initiated its rolling New Drug Application (NDA) to the U.S. Food and Drug Administration in August 2020, expects to complete the NDA submission in the first quarter of 2021, and is preparing for launch in the second half of 2021.

    Mirum will utilize EVERSANA's commercialization platform to provide integrated nationwide distribution, specialty pharmacy, patient services and Hub support for maralixibat, if approved.

    "We are working diligently to prepare for the potential launch of maralixibat in the United States and one of the most important components is ensuring comprehensive support for patients and their families," said Chris Peetz, president and chief executive officer at Mirum. "We are pleased to partner with EVERSANA and are confident in their ability to provide end-to-end services to ensure maralixibat will reach patients and their families efficiently and effectively once the medication is available for prescribing. In parallel, we are preparing to file a marketing authorization application for maralixibat in PFIC2 this year, and these programs will help to refine our strategies as we finalize launch plans in Europe."

    "Rare diseases like ALGS demand more than traditional patient support or distribution models. From day one, we knew that our partnership would focus entirely on individual patient's needs at each step of their therapeutic and treatment journey. The platform is designed so that at launch, EVERSANA's infrastructure and experts will be ready with the right services, and each action will be continually optimized through real-time analytics," said Jim Lang, chief executive officer of EVERSANA. "Mirum's patients will have access to comprehensive support delivered by Mirum and EVERSANA, two companies equally committed to the rare disease space."

    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 1,700 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 FDA 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, abdominal pain, and vomiting. 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. In patients with ALGS, multiple organ systems may be affected by the mutation, including the liver, heart, kidneys and central nervous system. 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. 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). 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.

    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 for 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 regulatory approval path for maralixibat, and the potential launch of maralixibat, if approved, the market access, distribution and patient services of maralixibat to be provided by Eversana, and Mirum's Expanded Access Program 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", "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.

    About EVERSANA™

    EVERSANA™ is the leading provider of global services to the life sciences industry. The company's integrated solutions are rooted in the patient experience and span all stages of the product lifecycle to deliver long-term, sustainable value for patients, prescribers, channel partners and payers. The company serves more than 500 organizations, including innovative start-ups and established pharmaceutical companies, to advance life science solutions for a healthier world. To learn more about EVERSANA, visit eversana.com or connect through LinkedIn and Twitter.

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  20. 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 present at the following virtual investor conferences in September.

    H.C. Wainwright & Co. 22nd Annual Global Investment Conference
    September 14-15, 2020
    Presentation day and time: Monday, September 14, 2020 at 9:30am ET

    Cantor Fitzgerald Virtual Global Healthcare Conference
    September 15-17, 2020
    Presentation day and time: Thursday, September 17, 2020 at 9:20am ET

    A live audio webcast 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. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced that the company will present at the following virtual investor conferences in September.

    H.C. Wainwright & Co. 22nd Annual Global Investment Conference

    September 14-15, 2020

    Presentation day and time: Monday, September 14, 2020 at 9:30am ET

    Cantor Fitzgerald Virtual Global Healthcare Conference

    September 15-17, 2020

    Presentation day and time: Thursday, September 17, 2020 at 9:20am ET

    A live audio webcast 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 TwitterFacebook and LinkedIn.

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  21. - Maralixibat would be the first treatment available for use in Alagille syndrome, if approved
    - Completion of the NDA submission expected in the first quarter of 2021
    - Expanded Access Program for maralixibat now open

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases today announced that it has submitted the first portion of its rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for maralixibat. Maralixibat is a novel, minimally absorbed, orally administered apical sodium dependent bile acid transporter (ASBT) inhibitor being evaluated for the treatment of cholestatic pruritus in patients…

    - Maralixibat would be the first treatment available for use in Alagille syndrome, if approved

    - Completion of the NDA submission expected in the first quarter of 2021

    - Expanded Access Program for maralixibat now open

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases today announced that it has submitted the first portion of its rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for maralixibat. Maralixibat is a novel, minimally absorbed, orally administered apical sodium dependent bile acid transporter (ASBT) inhibitor being evaluated for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS). Mirum expects to complete the NDA submission in the first quarter of 2021.

    Mirum also launched its Expanded Access Program (EAP) for maralixibat for the treatment of cholestatic pruritus in patients with ALGS one year of age and older. The EAP is open for registration in the United States and Canada. Through this program, physicians can request access to maralixibat for eligible patients who are not part of an ongoing clinical trial.

    "We are thrilled to initiate the rolling NDA submission for maralixibat, taking us one step closer to making this medicine widely available for patients with ALGS," said Chris Peetz, president and chief executive officer at Mirum. "We believe the results of our clinical program demonstrate the potential of maralixibat to transform the treatment of this life-threatening disease. We are also pleased to launch our Expanded Access Program for patients with ALGS in the United States and Canada and are evaluating ways in which we can make maralixibat available for patients with ALGS in other countries. Additionally, we are planning to broaden access to maralixibat through our anticipated Marketing Authorization Application submission for patients with PFIC2 in Europe later this year."

    About the NDA Submission

    Maralixibat was previously granted Rare Pediatric Disease Designation for ALGS and, as such, may qualify for receipt of a priority review voucher if the NDA is 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 in the United States.

    Data from the maralixibat Phase 2 ICONIC study evaluating patients with ALGS serves as the basis of efficacy for the submission. Previously presented data from this study in November 2019 are available within the Publications and Presentations section on Mirum's website.

    Mirum expects to complete the rolling NDA submission in the first quarter of 2021 and is planning for a potential launch of maralixibat in ALGS in the second half of 2021.

    About the 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 FDA approval of the medication and until maralixibat is available by prescription.

    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 https://clinicaltrials.gov/ct2/show/NCT04530994. Physicians who would like to request access for their patient can contact .

    For patients with 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, abdominal pain, and vomiting. 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. 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, as well as Mirum's Expanded Access Program for maralixibat, the regulatory approval path for maralixibat and volixibat, 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", "believes," "anticipates," "expects," "intends," "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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    • Maralixibat demonstrates significant transplant-free survival for patients with progressive familial intrahepatic cholestasis (PFIC2) who achieved serum bile acid (sBA) control in five-year analysis (p=0.0006).
    • sBA responders remain on maralixibat for more than five years with improvements across multiple parameters including normalization of liver enzyme and bilirubin levels, decreased pruritus and improvement in growth.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, presented an analysis from its Phase 2 INDIGO study in an oral late-breaker session at the Digital International Liver Congress™ 2020. The five-year…

    • Maralixibat demonstrates significant transplant-free survival for patients with progressive familial intrahepatic cholestasis (PFIC2) who achieved serum bile acid (sBA) control in five-year analysis (p=0.0006).
    • sBA responders remain on maralixibat for more than five years with improvements across multiple parameters including normalization of liver enzyme and bilirubin levels, decreased pruritus and improvement in growth.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, presented an analysis from its Phase 2 INDIGO study in an oral late-breaker session at the Digital International Liver Congress™ 2020. The five-year analysis showed that patients with PFIC2, also known as bile salt export pump (BSEP) deficiency, who achieved sBA control on long-term maralixibat treatment have a significant improvement in transplant-free survival.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200829005005/en/

    (Graphic: Business Wire)

    (Graphic: Business Wire)

    "The maralixibat data demonstrate a well-tolerated, long-term treatment option to address PFIC2, which may provide patients with not only a viable treatment alternative to liver transplantation, but also a possible path to addressing many of the quality of life challenges that can make PFIC a burdensome disease for patients and their families," said Richard Thompson, professor of molecular hepatology at King's College London, who presented the data.

    Late-breaker oral presentation

    August 29, 2020 – 2:45-3:00pm CET

    LB008:
    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.

    In February 2020, the NAPPED consortium published an analysis showing that surgical interruption of the enterohepatic circulation of bile acids can lead to transplant-free survival when sBAs are reduced by 75% or below 102 µmol/L. Data presented today demonstrate that, similar to the findings of NAPPED, patients exhibiting sBA control with pharmacological interruption of the enterohepatic circulation with maralixibat have significantly improved transplant-free survival (p=0.0006). Seven of the 19 non-truncating patients with PFIC2 achieved this response threshold. In addition, the data showed that maralixibat responders achieved improvement across numerous parameters including quality of life scores, normalization of liver enzyme and bilirubin levels if abnormal at baseline, decreased pruritus and accelerated growth.

    "These data further underscore the potential for maralixibat as an alternative to liver transplantation in children with PFIC2, addressing a major unmet medical need for a very burdensome disease," said Chris Peetz, president and chief executive officer at Mirum. "These children are in dire need of an effective pharmacologic treatment option, and we want to ensure patients have appropriate treatments beyond surgery. Given these efficacy data and the encouraging safety profile, we are planning to file a marketing authorization application in Europe this year with the hope of bringing maralixibat to patients with PFIC2 as quickly as possible."

    Long-term treatment with maralixibat was well-tolerated. The most frequent treatment-emergent adverse events (TEAEs) were nasopharyngitis, vomiting, cough, diarrhea, pyrexia and abdominal pain. The majority of TEAEs were mild to moderate in severity and transient.

    To view the presentation as well as other maralixibat posters presented during the meeting, please visit the Publications and Presentations section of our corporate website.

    About the Maralixibat Phase 2 INDIGO Study

    The INDIGO Phase 2 study was an open-label study evaluating the long-term treatment effects of pharmacological interruption of enterohepatic circulation with maralixibat in children with PFIC. INDIGO included 19 PFIC2 patients with non-truncating BSEP mutations who received maralixibat 280 µg/kg once or twice daily. Study endpoints were sBA, pruritus, quality of life, safety and tolerability.

    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, abdominal pain, and vomiting. The North American Expanded Access Program for ALGS is planned to open for registration in September 2020. 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 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, as well as Mirum's Expanded Access Program for maralixibat 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", "believes," "anticipates," "expects," "intends," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum's business in general, the impact of the COVID-19 pandemic, and the other risks described in Mirum's filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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  22. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced that Chris Peetz, president and chief executive officer at Mirum will present at the virtual Canaccord Genuity 40th Annual Growth Conference on August 13, 2020 at 3:00pm ET.

    A live audio webcast and an archive of the presentation 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…

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced that Chris Peetz, president and chief executive officer at Mirum will present at the virtual Canaccord Genuity 40th Annual Growth Conference on August 13, 2020 at 3:00pm ET.

    A live audio webcast and an archive of the presentation 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) and progressive familial intrahepatic cholestasis (PFIC). For more information, visit mirumpharma.com. Follow Mirum on Twitter, Facebook and LinkedIn.

    View Full Article Hide Full Article
    • Rolling NDA submission for maralixibat in Alagille syndrome (ALGS) to be initiated in third quarter 2020; planning for potential launch in second half of 2021.
    • Submission of European marketing authorization application for maralixibat in PFIC2 planned for fourth quarter of 2020.
    • Expanded Access Program for maralixibat in ALGS planned to open for registration in September 2020.
    • Orphan Drug Designation received by European Medicines Agency for maralixibat in biliary atresia.
    • Cash, cash equivalents and investments of $149.3 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…

    • Rolling NDA submission for maralixibat in Alagille syndrome (ALGS) to be initiated in third quarter 2020; planning for potential launch in second half of 2021.
    • Submission of European marketing authorization application for maralixibat in PFIC2 planned for fourth quarter of 2020.
    • Expanded Access Program for maralixibat in ALGS planned to open for registration in September 2020.
    • Orphan Drug Designation received by European Medicines Agency for maralixibat in biliary atresia.
    • Cash, cash equivalents and investments of $149.3 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 business updates for the quarter ended June 30, 2020.

    "We made significant progress toward making maralixibat available as an alternative to liver transplantation in devastating pediatric cholestatic settings in the United States and Europe," said Chris Peetz, president and chief executive officer at Mirum. "In the United States, we are initiating a rolling NDA submission for maralixibat in ALGS and are planning to start an expanded access program broadening availability to this important medicine for eligible patients. By the end of this year, we intend to submit a marketing authorization application for maralixibat in PFIC2 in Europe, dramatically shifting our timeline for a potential launch in this region."

    Second Quarter Highlights

    • Released maralixibat data demonstrating five-year transplant-free survival for pediatric patients with PFIC2. Data will be presented at the upcoming Digital International Liver Congress, August 27-29, 2020.
    • Completed pre-submission meeting with European regulators resulting in feedback enabling submission of a marketing authorization application (MAA) for maralixibat in the treatment of patients with PFIC2 in Europe in the fourth quarter of 2020.
    • Investigational new drug (IND) application cleared by FDA for the initiation of a Phase 2 study evaluating volixibat in adult patients with pruritus associated with primary sclerosing cholangitis.
    • Received orphan drug designation in Europe for maralixibat in the study of patients with biliary atresia; planning for a Phase 2 study is underway.
    • As of June 30, 2020, Mirum had cash, cash equivalents and investments of $149.3 million.

    Upcoming Anticipated Program Milestones

    • Maralixibat in ALGS:
      • Initiation of rolling new drug application (NDA) submission for maralixibat in ALGS on track for third quarter of 2020.
        • Expect to complete NDA submission in the first quarter of 2021.
        • Planning for launch in ALGS in the second half of 2021.
      • Maralixibat ALGS Expanded Access Program planned to open for registration in the U.S. and Canada in September 2020.
    • Maralixibat in PFIC:
      • Submission of MAA to European regulators for maralixibat in the treatment of patients with PFIC2 on track for fourth quarter of 2020.
      • Enrollment ongoing in Phase 3 MARCH clinical trial in PFIC.
    • Maralixibat in biliary atresia:
      • Phase 2 study initiation planned.
    • Volixibat:
      • Planned expansion into adult patients with studies in primary sclerosing cholangitis and intrahepatic cholestasis of pregnancy.

    Digital International Liver Congress™ 2020

    New findings from maralixibat studies will be presented in a late-breaker and poster sessions at the Digital International Liver Congress 2020, August 27-29, 2020. Featured presentations to include the following abstracts:

    Late-breaker Oral Presentation

    August 29, 2020 – 1:45-2:00pm CET

    Oral Presentation LB008: 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.

    Poster Presentations

    August 28, 2020

    Poster FRI-131: Psychometric evaluation of the adult itch reported outcome tool, a worst-itch numeric rating scale in adults with cholestatic liver disease.

    August 28, 2020

    Poster FRI-296: Differential expression of bile acid subspecies with maralixibat treatment in pruritus responders.

    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, abdominal pain, and vomiting. For more information about the North American 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, as well as Mirum's Expanded Access Program for maralixibat, 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", "believes," "anticipates," "expects," "intends," "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

     

    Six Months Ended

    June 30,

     

    June 30,

    2020

     

    2019

     

    2020

     

    2019

     
    Operating expenses:
    Research and development

    $

    18,555

     

    $

    11,589

     

    $

    35,895

     

    $

    16,452

     

    General and administrative

     

    5,042

     

     

    2,445

     

     

    9,734

     

     

    3,766

     

    Total operating expenses (1)

     

    23,597

     

     

    14,034

     

     

    45,629

     

     

    20,218

     

     
    Loss from operations

     

    (23,597

    )

     

    (14,034

    )

     

    (45,629

    )

     

    (20,218

    )

    Interest income

     

    405

     

     

    468

     

     

    1,154

     

     

    700

     

    Other income (expense), net

     

    (56

    )

     

    9

     

     

    (79

    )

     

    4

     

     
    Net loss before provision for income taxes

     

    (23,248

    )

     

    (13,557

    )

     

    (44,554

    )

     

    (19,514

    )

    Provision for income taxes

     

    3

     

     

    -

     

     

    7

     

     

    -

     

    Net loss

    $

    (23,251

    )

    $

    (13,557

    )

    $

    (44,561

    )

    $

    (19,514

    )

     
    Net loss per share, basic and diluted

    $

    (0.93

    )

    $

    (5.31

    )

    $

    (1.79

    )

    $

    (7.70

    )

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

     

    25,056,123

     

     

    2,551,822

     

     

    24,880,387

     

     

    2,534,877

     

     

    (1)

    Amounts include stock-based compensation expense as follows:

     
    Research and development

    $

    1,260

     

    $

    638

     

    $

    2,301

     

    $

    709

     

    General and administrative

     

    1,714

     

     

    1,003

     

     

    3,246

     

     

    1,150

     

    Total stock-based compensation

    $

    2,974

     

    $

    1,641

     

    $

    5,547

     

    $

    1,859

     

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

    June 30,

     

    December 31,

    2020

     

    2019

    (Unaudited)

     

     

     
    Cash, cash equivalents and investments

    $

    149,336

     

    $

    139,952

     

    Working capital

     

    135,495

     

     

    106,287

     

    Total assets

     

    155,766

     

     

    146,712

     

    Accumulated deficit

     

    (114,462

    )

     

    (69,901

    )

    Total stockholders' equity

     

    136,208

     

     

    130,349

     

     

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  23. 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 the Compensation Committee of Mirum's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 70,900 shares of common stock to four new employees under Mirum's 2020 Inducement Plan. The Compensation Committee of Mirum's Board of Directors approved the awards as an inducement material to the new employees' employment in accordance with Nasdaq Listing Rule 5635(c)(4).

    Each stock option has an exercise price per share equal to $19.03 per share, Mirum's closing trading price on July 10, 2020, and…

    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 the Compensation Committee of Mirum's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 70,900 shares of common stock to four new employees under Mirum's 2020 Inducement Plan. The Compensation Committee of Mirum's Board of Directors approved the awards as an inducement material to the new employees' employment in accordance with Nasdaq Listing Rule 5635(c)(4).

    Each stock option has an exercise price per share equal to $19.03 per share, Mirum's closing trading price on July 10, 2020, and will vest over four years, with 25% of the underlying shares vesting on the one-year anniversary of the applicable vesting commencement date and the balance of the underlying shares vesting monthly thereafter over 36 months, subject to the new employee's continued service relationship with Mirum through the applicable vesting dates. The awards are subject to the terms and conditions of Mirum's 2020 Inducement Plan and the terms and conditions of an applicable award agreement covering the grant.

    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) and progressive familial intrahepatic cholestasis (PFIC). For more information, visit MirumPharma.com. Follow Mirum on Twitter, Facebook and LinkedIn.

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  24. 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 the Compensation Committee of Mirum's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 60,000 shares of common stock to two new employees under Mirum's 2020 Inducement Plan. The Compensation Committee of Mirum's Board of Directors approved the awards as an inducement material to the new employees' employment in accordance with Nasdaq Listing Rule 5635(c)(4).

    Each stock option has an exercise price per share equal to $18.46 per share, Mirum's closing trading price on June 10, 2020, and…

    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 the Compensation Committee of Mirum's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 60,000 shares of common stock to two new employees under Mirum's 2020 Inducement Plan. The Compensation Committee of Mirum's Board of Directors approved the awards as an inducement material to the new employees' employment in accordance with Nasdaq Listing Rule 5635(c)(4).

    Each stock option has an exercise price per share equal to $18.46 per share, Mirum's closing trading price on June 10, 2020, and will vest over four years, with 25% of the underlying shares vesting on the one-year anniversary of the applicable vesting commencement date and the balance of the underlying shares vesting monthly thereafter over 36 months, subject to the new employee's continued service relationship with Mirum through the applicable vesting dates. The awards are subject to the terms and conditions of Mirum's 2020 Inducement Plan and the terms and conditions of an applicable award agreement covering the grant.

    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) and progressive familial intrahepatic cholestasis (PFIC). For more information, visit MirumPharma.com. Follow Mirum on Twitter, Facebook and LinkedIn.

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  25. - Maralixibat-treated patients achieving serum bile acid control have five-year native liver survival after treatment.

    - Data demonstrate normalization and reductions of liver enzymes, reduced pruritus, and improved growth.

    - Data selected for oral presentation at the Digital International Liver Congress 2020 being held August 27-29, 2020.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today reported an analysis from its Phase 2 INDIGO open-label study evaluating maralixibat in patients with bile acid salt export pump (BSEP) deficiency, or PFIC2. The data demonstrated that patients with long-term maralixibat treatment who achieved serum bile acid (sBA) control, have five-year transplant-free survival after treatment.

    "Patients need alternatives…

    - Maralixibat-treated patients achieving serum bile acid control have five-year native liver survival after treatment.

    - Data demonstrate normalization and reductions of liver enzymes, reduced pruritus, and improved growth.

    - Data selected for oral presentation at the Digital International Liver Congress 2020 being held August 27-29, 2020.

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today reported an analysis from its Phase 2 INDIGO open-label study evaluating maralixibat in patients with bile acid salt export pump (BSEP) deficiency, or PFIC2. The data demonstrated that patients with long-term maralixibat treatment who achieved serum bile acid (sBA) control, have five-year transplant-free survival after treatment.

    "Patients need alternatives to surgery or transplant to treat PFIC2, otherwise known as BSEP deficiency, and the long-term maralixibat data demonstrate the therapy's potential to provide an efficacious and well-tolerated treatment for this condition," said Richard Thompson, Professor of Molecular Hepatology at King's College London. "The long-term control of serum bile acids underscores the potential for maralixibat to transform the way PFIC2 is treated, as well as provide additional benefit across many of the quality of life measures that can be debilitating and significantly impact patients' lives."

    A study recently published by the NAPPED Consortium established that sBA control (<102µmol/L or a 75% reduction) after interruption of the enterohepatic circulation by biliary diversion surgery was associated with native liver survival of up to 15 years. Similarly, maralixibat pharmacologically interrupts the enterohepatic circulation, and maralixibat responders from the INDIGO study surpassed the sBA thresholds described in the NAPPED study, which were statistically significant compared to baseline (p<0.05). In addition, maralixibat responders achieved normalization of liver enzyme and bilirubin levels, decreased pruritus, and improved z-scores in both height and weight.

    The data and analysis have been selected by the European Association for the Study of the Liver (EASL) as a late-breaker and oral presentation at the Digital International Liver Congress 2020 being held August 27-29, 2020.

    "We are thrilled with these data as they confirm our belief that maralixibat has the potential to treat PFIC2, preventing transplant and surgical diversion and alleviating many of the symptoms associated with the condition," said Chris Peetz, president and chief executive officer at Mirum. "Supporting this, we have cases of maralixibat-treated patients being removed from the liver transplant waiting list. There is a tremendous need for new medicines for the patients and families suffering from this disease, and it is our goal to develop a treatment that can address this rare and serious cholestatic liver disease."

    About the Maralixibat Phase 2 INDIGO Study

    The INDIGO Phase 2 study is an ongoing, open-label study evaluating the long-term treatment effects of interruption of enterohepatic circulation with maralixibat in children with PFIC. INDIGO included 19 PFIC2 patients with non-truncating BSEP mutations who received maralixibat 280 µg/kg once daily. Patients without response or with partial treatment response could escalate to 280 µg/kg twice daily in the optional extension period. Study endpoints were sBA, pruritus, quality of life, safety and tolerability.

    Week 237 (n=7)

    Mean Value

    Mean Reduction

    from Baseline

    P value

    Serum bile acid (SE) µmol/L

    44.2 (38.8)

    -234.4 (80.5)

    <0.05

    ALT (SE) U/L

    16.7

    -41.1 (14.3)

    <0.05

    AST (SE) U/L

    26.7

    -35.4 (11.5)

    <0.05

    Total bilirubin (SE) mg/dL

    0.7

    -0.1 (0.3)

    0.08

    Direct bilirubin (SE) mg/dL

    0.1

    -0.4 (0.2)

    0.13

    Patients treated with maralixibat exhibited a clinically meaningful and statistically significant acceleration in height growth as measured by height z-score (p<0.01), as well as a clinically meaningful and statistically significant reduction in pruritus (p<0.001).

    Of the enrolled patients, seven achieved sBA control and remained on study as of Week 237. Long-term treatment with maralixibat was well-tolerated. The most frequent treatment-emergent adverse events (TEAEs) were pyrexia, diarrhea, cough, abdominal pain, and vomiting. TEAEs were mild to moderate in severity.

    Patients with an sBA response continue to be treated with maralixibat for more than five years with improvements demonstrated across multiple parameters.

    About Maralixibat

    Maralixibat is a novel, minimally-absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases for pediatric populations. 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 1,500 individuals have received maralixibat, including more than 100 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 Alagille syndrome 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, abdominal pain, and vomiting. For more information about the Phase 3 study for maralixibat in pediatric patients with PFIC, visit PFICtrial.com. For more information about the North American Expanded Access Program please visit ALGSEAP.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) and progressive familial intrahepatic cholestasis (PFIC). 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 clinical trials 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", "believes," "anticipates," "expects," "intends," "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. In addition, the COVID-19 pandemic continues to rapidly evolve and actual results are highly uncertain and cannot be predicted with confidence. 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), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced that Chris Peetz, president and chief executive officer at Mirum will present at the virtual Jefferies 2020 Healthcare Conference on June 2, 2020 at 10:00 a.m. Pacific Time/1:00 p.m. Eastern Time.

    A live audio webcast and archive of the presentation 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…

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced that Chris Peetz, president and chief executive officer at Mirum will present at the virtual Jefferies 2020 Healthcare Conference on June 2, 2020 at 10:00 a.m. Pacific Time/1:00 p.m. Eastern Time.

    A live audio webcast and archive of the presentation 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) and progressive familial intrahepatic cholestasis (PFIC). For more information, visit mirumpharma.com. Follow Mirum on Twitter, Facebook and LinkedIn.

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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 that the Compensation Committee of Mirum's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 17,500 shares of common stock to two new employees under Mirum's 2020 Inducement Plan. The Compensation Committee of Mirum's Board of Directors approved the awards as an inducement material to the new employees' employment in accordance with Nasdaq Listing Rule 5635(c)(4).

    Each stock option has an exercise price per share equal to $18.23 per share, Mirum's closing trading price on May 11, 2020, and will…

    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 the Compensation Committee of Mirum's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 17,500 shares of common stock to two new employees under Mirum's 2020 Inducement Plan. The Compensation Committee of Mirum's Board of Directors approved the awards as an inducement material to the new employees' employment in accordance with Nasdaq Listing Rule 5635(c)(4).

    Each stock option has an exercise price per share equal to $18.23 per share, Mirum's closing trading price on May 11, 2020, and will vest over four years, with 25% of the underlying shares vesting on the one-year anniversary of the applicable vesting commencement date and the balance of the underlying shares vesting monthly thereafter over 36 months, subject to the new employee's continued service relationship with Mirum through the applicable vesting dates. The awards are subject to the terms and conditions of Mirum's 2020 Inducement Plan and the terms and conditions of an applicable award agreement covering the grant.

    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) and progressive familial intrahepatic cholestasis (PFIC). For more information, visit MirumPharma.com. Follow Mirum on Twitter, Facebook and LinkedIn.

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  28. - On track to begin rolling submission of NDA for maralixibat in Alagille Syndrome (ALGS) in third quarter 2020

    - Cash, cash equivalents and investments balance of $166.1 million

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today reported financial results and provided business updates for the quarter ended March 31, 2020.

    "Despite the COVID-19 pandemic, we remain on track to begin the rolling submission of our new drug application to the U.S. Food and Drug Administration in the third quarter of this year with the goal of launching maralixibat for patients with Alagille syndrome in the second half of 2021," said…

    - On track to begin rolling submission of NDA for maralixibat in Alagille Syndrome (ALGS) in third quarter 2020

    - Cash, cash equivalents and investments balance of $166.1 million

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today reported financial results and provided business updates for the quarter ended March 31, 2020.

    "Despite the COVID-19 pandemic, we remain on track to begin the rolling submission of our new drug application to the U.S. Food and Drug Administration in the third quarter of this year with the goal of launching maralixibat for patients with Alagille syndrome in the second half of 2021," said Chris Peetz, president and chief executive officer at Mirum. "Further, we are exploring the potential to accelerate our regulatory filings for maralixibat in PFIC2 and will be discussing the Phase 2 INDIGO study 5-year outcomes versus a natural history cohort with regulators this year."

    First Quarter 2020 Highlights

    • Bolstered balance sheet with $48.0 million gross proceeds from a follow on public offering in January 2020.
    • As of March 31, 2020, Mirum had cash, cash equivalents and investments of $166.1 million; projected to fund current operations through the anticipated FDA approval of maralixibat in Alagille syndrome (ALGS) in the second half of 2021.
    • Completed Phase 1 dose-ranging and drug-drug interaction study for volixibat to inform dose regimens for planned registrational program.

    Upcoming Anticipated Milestones

    • Initiate rolling submission of NDA for maralixibat in ALGS in the third quarter of 2020.
    • Presentations highlighting data from maralixibat and volixibat studies planned for upcoming medical congresses including European Association for the Study of the Liver and the American Association for the Study of Liver Diseases.
    • Initiate ALGS expanded access program in the United States and Canada in the fourth quarter of 2020.
    • Discuss with regulators the suitability of maralixibat INDIGO study 5-year outcomes data in PFIC2 patients compared to natural history for potential regulatory submission.
    • US launch for maralixibat in ALGS remains on track for 2021.

    COVID-19 Pandemic Impact

    The COVID-19 pandemic has had a significant economic and social impact across the globe, presenting challenges to maintaining business continuity. In order to reduce the risk of spreading the COVID-19 virus, the company has mandated that all employees work from home. At the same time, Mirum is working diligently to ensure the advancement of all of its clinical development programs in the safest manner possible. However, due to health and safety concerns for patients, their caregivers, and healthcare personnel, there have been, and may continue to be delays in the advancement of some of the company's programs, including the completion of enrollment for the Phase 3 MARCH study in PFIC2 patients. Mirum is unable to predict with confidence the duration of such delays. As such, Mirum is withdrawing previous guidance regarding the timing of the initiation and enrollment of the ongoing and planned maralixibat and volixibat studies. Mirum's planned timeline for submission of its NDA for maralixibat in ALGS has not been impacted. Mirum continues to collect data in the INDIGO study, in which PFIC2 patients have been treated with maralixibat for more than five years.

    During this time, ensuring that patients have access to treatment is paramount. Mirum is working closely with the study sites to develop methods in which patients can safely continue on treatment, including adopting new protocols to have study medication shipped to patients' homes and to conduct virtual check-ins, as opposed to attending standard hospital visits, providing a safer alternative for all persons involved in the program.

    About Maralixibat

    Maralixibat is a novel, minimally-absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases for pediatric populations. 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 1,500 individuals have received maralixibat, including more than 100 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 Alagille syndrome 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, abdominal pain, and vomiting. For more information about the Phase 3 study for maralixibat in pediatric patients with PFIC, visit PFICtrial.com. For more information about the North American Expanded Access Program please visit ALGSEAP.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) and progressive familial intrahepatic cholestasis (PFIC). 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 clinical trials for maralixibat and volixibat, the regulatory approval path for maralixibat, the strength of Mirum's balance sheet and the adequacy of cash, cash equivalents and investments on hand, and the impacts of COVID-19, including, but not limited to, delays or difficulties in enrolling patients in its ongoing and planned clinical trials, delays or difficulties in clinical site initiation, including difficulties in recruiting clinical site investigators and clinical site staff, delays in clinical sites receiving the supplies and materials needed to conduct its clinical trials, including interruption in global shipping that may affect the transport of clinical trial materials, and effects of the executive order, the shelter-in-place order and Mirum's work-from-home policies and related effects on productivity. 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", "believes," "anticipates," "expects," "intends," "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. In addition, the COVID-19 pandemic continues to rapidly evolve and actual results are highly uncertain and cannot be predicted with confidence. 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   Three Months
      Ended   Ended
      March 31, 2020   March 31, 2019
         
    Operating expenses:    
    Research and development  

    $

    17,340

     

     

    $

    4,863

     

    General and administrative  

     

    4,692

     

     

     

    1,321

     

    Total operating expenses (1)  

     

    22,032

     

     

     

    6,184

     

         
    Loss from operations  

     

    (22,032

    )

     

     

    (6,184

    )

    Interest income  

     

    749

     

     

     

    232

     

    Other income (expense), net  

     

    (23

    )

     

     

    (5

    )

         
    Net loss before provision for income taxes  

     

    (21,306

    )

     

     

    (5,957

    )

    Provision for income taxes  

     

    4

     

     

     

    -

     

    Net loss  

    $

    (21,310

    )

     

    $

    (5,957

    )

         
    Net loss per share, basic and diluted  

    $

    (0.86

    )

     

    $

    (2.37

    )

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

     

    24,704,651

     

     

     

    2,517,743

     

         
         

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

       
         
    Research and development  

    $

    1,041

     

     

    $

    71

     

    General and administrative  

     

    1,532

     

     

     

    147

     

    Total stock-based compensation  

    $

    2,573

     

     

    $

    218

     

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

    March 31,

     

    December 31,

     

    2020

     

    2019

     

    (Unaudited)

     
         
    Cash, cash equivalents and investments  

    $

    166,061

     

     

    $

    139,952

     

    Working capital  

     

    148,888

     

     

     

    106,287

     

    Total assets  

     

    172,803

     

     

     

    146,712

     

    Accumulated deficit  

     

    (91,211

    )

     

     

    (69,901

    )

    Total stockholders' equity  

     

    156,118

     

     

     

    130,349

     

     

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  29. 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 appointment of Peter Radovich as its chief operating officer with oversight of commercial operations, technical operations, supply chain and corporate development. Mr. Radovich joins Mirum with nearly 20 years leading biopharmaceutical development and commercialization programs, having served most recently as executive vice president, operations for South San Francisco-based Global Blood Therapeutics.

    "I am thrilled to welcome Peter to Mirum and look forward to working together to deliver new treatments to patients with rare…

    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 appointment of Peter Radovich as its chief operating officer with oversight of commercial operations, technical operations, supply chain and corporate development. Mr. Radovich joins Mirum with nearly 20 years leading biopharmaceutical development and commercialization programs, having served most recently as executive vice president, operations for South San Francisco-based Global Blood Therapeutics.

    "I am thrilled to welcome Peter to Mirum and look forward to working together to deliver new treatments to patients with rare liver diseases," said Chris Peetz, president and chief executive officer, Mirum. "Peter is an effective leader and brings a patient-first mentality fitting with the Mirum strategy. His extensive experience launching life-changing medicines and recent NDA experience make him an impactful addition to our leadership team as we prepare to initiate a rolling submission of a new drug application with the FDA for maralixibat for the treatment of cholestatic pruritus associated with Alagille syndrome in the third quarter of 2020."

    In connection with his employment, the Compensation Committee of Mirum's Board of Directors granted Mr. Radovich an inducement award consisting of a non-qualified stock option to purchase 185,000 shares of common stock under Mirum's 2020 Inducement Plan. The Compensation Committee of Mirum's Board of Directors approved the award as an inducement material to Mr. Radovich's employment in accordance with Nasdaq Listing Rule 5635(c)(4).

    The stock option has an exercise price per share equal to $16.26 per share, Mirum's closing trading price on April 28, 2020, and will vest over four years, with 25% of the underlying shares vesting on the one-year anniversary of the vesting commencement date and the balance of the underlying shares vesting monthly thereafter over 36 months, subject to Mr. Radovich's continued service relationship with Mirum through the applicable vesting dates. The award is subject to the terms and conditions of Mirum's 2020 Inducement Plan and the terms and conditions of an award agreement covering the grant.

    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) and progressive familial intrahepatic cholestasis (PFIC). 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 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", "believes," "anticipates," "expects," "intends," "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 the COVID-19 pandemic, including, but not limited to, related federal and state regulations, the duration of the pandemic, disruptions at the FDA and other government agencies, and the resulting health and economic implications, Mirum's business in general, 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. New Drug Application Rolling Submission for Alagille Syndrome Planned to Begin in Third Quarter 2020

    Cash, Cash Equivalents and Investments Balance of $140.0 million before gross proceeds of $48.0 million from a follow-on public offering in January 2020

    On Track for PFIC Phase 3 Top-Line Data and Long-term Outcomes Natural History Comparisons by Year End

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today reported financial and business results for the quarter and year ended December 31, 2019.

    "2019 was a year of ground-breaking achievements for Mirum," said Chris Peetz, president and chief executive officer…

    New Drug Application Rolling Submission for Alagille Syndrome Planned to Begin in Third Quarter 2020

    Cash, Cash Equivalents and Investments Balance of $140.0 million before gross proceeds of $48.0 million from a follow-on public offering in January 2020

    On Track for PFIC Phase 3 Top-Line Data and Long-term Outcomes Natural History Comparisons by Year End

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today reported financial and business results for the quarter and year ended December 31, 2019.

    "2019 was a year of ground-breaking achievements for Mirum," said Chris Peetz, president and chief executive officer of Mirum. "We established Mirum as a leading company in cholestatic liver diseases and have positioned ourselves to deliver our first NDA submission, planned to begin in the third quarter of this year. Maralixibat has potential to alter the course of disease in pediatric cholestasis and we are excited to be one step closer to delivering medicines to children with debilitating liver diseases. With the additional capital raised in January 2020 we are well positioned to grow our pipeline including executing on the upcoming NDA, maralixibat launch preparation, completing our ongoing studies and launching potentially registrational studies for volixibat."

    Key Operational Highlights in Fourth Quarter 2019

    • Successful completion of a pre-NDA meeting with the Food and Drug Administration (FDA) for maralixibat with alignment on submission of rolling NDA starting Q3 2020.
    • Breakthrough Therapy Designation granted by the FDA for maralixibat for the treatment of pruritus associated with Alagille syndrome in patients 1 year of age and older.
    • Rare Pediatric Disease Designation granted by the FDA for Alagille syndrome. If maralixibat's NDA is approved prior to October 1, 2022, the company may be eligible to receive a priority review voucher, which can be redeemed to obtain priority review for any subsequent marketing application and may be sold or transferred.
    • Presented data demonstrating the long-term durability of maralixibat in children with cholestatic liver disease. At the Liver Meeting in November 2019, data were presented showing statistically and clinically significant effects on pruritus, xanthomas and growth maintained for up to four years of treatment in Alagille syndrome and durable multi-parameter treatment response and correlation of response with increased bile acid synthesis in progressive familial intrahepatic cholestasis.

    Fourth Quarter 2019 Financial Results

    • For the quarter ended December 31, 2019 Mirum reported a net loss of $18.0 million.
    • Total operating expenses for the quarter ended December 31, 2019 were $18.7 million.
      • Research and development expenses for the quarter ended December 31, 2019 were $14.4 million.
      • General and administrative expenses for the quarter ended December 31, 2019 were $4.3 million.

    As of December 31, 2019 Mirum had cash, cash equivalents and investments of $140.0 million, before gross proceeds of $48.0 million from Mirum's follow-on public offering of common stock in January 2020.

    Upcoming Milestones and Expectations

    • Announce results from Phase 1 study of volixibat in the second quarter of 2020, and subsequently select dose for primary sclerosing cholangitis and intrahepatic cholestasis of pregnancy studies.
    • Complete enrollment of the Phase 3 MARCH clinical trial investigating the safety and efficacy of maralixibat in patients with PFIC2 in the second quarter of 2020 and announce topline results by the end of 2020.
    • Initiate Alagille syndrome NDA rolling submission in the third quarter of 2020.
    • Initiate Alagille syndrome expanded access program in the fourth quarter of 2020.
    • Initiate maralixibat study in biliary atresia initiation by year end.
    • Initiate potentially registrational study of volixibat in primary sclerosing cholangitis in fourth quarter of 2020.

    About Maralixibat

    Maralixibat is a novel, minimally-absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases for pediatric populations. 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 1,500 individuals have received maralixibat, including more than 100 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 Alagille syndrome 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, abdominal pain and vomiting. For more information about the currently enrolling Phase 3 study maralixibat in pediatric patients with PFIC, visit PFICtrial.com. For more information about the North American Expanded Access Program please visit ALGSEAP.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) and progressive familial intrahepatic cholestasis (PFIC). 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 clinical trials for maralixibat and volixibat, 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", "believes," "anticipates," "expects," "intends," "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, 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 Three Months Period from
    Ended Ended Year Ended May 2, 2018 to
    December 31, 2019 December 31, 2018 December 31, 2019 December 31, 2018
    (Unaudited) (Unaudited)
    Operating expenses:
    Research and development

    $

    14,380

     

    $

    2,328

     

    $

    42,991

     

    $

    2,331

     

    In process research and development

     

    -

     

     

    14,490

     

     

    -

     

     

    14,490

     

    General and administrative

     

    4,278

     

     

    513

     

     

    11,752

     

     

    585

     

    Total operating expenses (1)

     

    18,658

     

     

    17,331

     

     

    54,743

     

     

    17,406

     

     
    Loss from operations

     

    (18,658

    )

     

    (17,331

    )

     

    (54,743

    )

     

    (17,406

    )

    Interest income

     

    747

     

     

    72

     

    $

    2,232

     

     

    72

     

    Other income (expense), net

     

    (20

    )

     

    (12

    )

    $

    (21

    )

     

    (14

    )

     
    Net loss before provision for income taxes

     

    (17,931

    )

     

    (17,271

    )

     

    (52,532

    )

     

    (17,348

    )

    Provision for income taxes

     

    21

     

     

    -

     

     

    21

     

     

    -

     

    Net loss

    $

    (17,952

    )

    $

    (17,271

    )

    $

    (52,553

    )

    $

    (17,348

    )

     
    Net loss per common share, basic and diluted

    $

    (0.79

    )

    $

    (9.10

    )

    $

    (4.58

    )

    $

    (19.29

    )

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

     

    22,587,752

     

     

    1,898,515

     

     

    11,486,367

     

     

    899,331

     

     
     

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

     
    Research and development

    $

    820

     

    $

    8

     

    $

    2,359

     

    $

    8

     

    General and administrative

     

    1,247

     

     

    26

     

     

    3,711

     

     

    26

     

    Total stock-based compensation

    $

    2,067

     

    $

    34

     

    $

    6,070

     

    $

    34

     

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

    December 31,

    December 31,

    2019

    2018

     
    Cash, cash equivalents and investments

    $

    139,952

     

    $

    51,963

     

    Working capital

     

    106,287

     

     

    49,526

     

    Total assets

     

    146,712

     

     

    51,975

     

    Accumulated deficit

     

    (69,901

    )

     

    (17,348

    )

    Total stockholders' equity (deficit)

     

    130,349

     

     

    (17,313

    )

     

     

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  31. 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 company management will participate at the following conferences in February and March.

    • SVB Leerink 9th Annual Global Healthcare Conference
      • Location: New York, NY
      • Presentation date: Wednesday February 26, 2020
      • Presentation time: 9:30 a.m. ET
    • Cowen 40th Annual Healthcare Conference
      • Location: Boston, MA
      • Presentation date: Monday March 2, 2020
      • Presentation time: 2:10 p.m. ET
    • Raymond James 41st Annual Institutional Investors Conference
      • Location: Orlando, FL
      • Presentation date: Wednesday March 4, 2020
      • Presentation…

    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 company management will participate at the following conferences in February and March.

    • SVB Leerink 9th Annual Global Healthcare Conference
      • Location: New York, NY
      • Presentation date: Wednesday February 26, 2020
      • Presentation time: 9:30 a.m. ET
    • Cowen 40th Annual Healthcare Conference
      • Location: Boston, MA
      • Presentation date: Monday March 2, 2020
      • Presentation time: 2:10 p.m. ET
    • Raymond James 41st Annual Institutional Investors Conference
      • Location: Orlando, FL
      • Presentation date: Wednesday March 4, 2020
      • Presentation time: 9:15 a.m. ET

    A live audio webcast and archive of the presentations outline above will be available at http://ir.mirumpharma.com

    Additionally, the company will be participating the 32nd Annual ROTH Conference, and will be on a panel on Monday March 16 at 11:00 a.m. discussing Orphan Therapeutics.

    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) and progressive familial intrahepatic cholestasis (PFIC). For more information, visit MirumPharma.com. Follow Mirum on Twitter, Facebook and LinkedIn.

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  32. MENLO PARK, Calif., Jan. 16, 2020 /PRNewswire/ -- Frazier Healthcare Partners announced today the closing of Frazier Life Sciences X, L.P., exceeding its target and closing on more than $617 million in capital commitments in an oversubscribed fundraise. Investing in life sciences has been a core strategy since the firm's inception 29 years ago, and Frazier Life Sciences X marks the third dedicated Life Sciences fund, bringing the firm's total committed capital raised since inception to nearly $4.8 billion.

    Led by General Partners Patrick Heron, James Topper and Dan Estes, the Frazier Life Sciences team invests primarily in therapeutics-focused companies developing and commercializing innovative biopharmaceuticals that address important unmet…

    MENLO PARK, Calif., Jan. 16, 2020 /PRNewswire/ -- Frazier Healthcare Partners announced today the closing of Frazier Life Sciences X, L.P., exceeding its target and closing on more than $617 million in capital commitments in an oversubscribed fundraise. Investing in life sciences has been a core strategy since the firm's inception 29 years ago, and Frazier Life Sciences X marks the third dedicated Life Sciences fund, bringing the firm's total committed capital raised since inception to nearly $4.8 billion.

    Led by General Partners Patrick Heron, James Topper and Dan Estes, the Frazier Life Sciences team invests primarily in therapeutics-focused companies developing and commercializing innovative biopharmaceuticals that address important unmet medical needs. Frazier Life Sciences X will continue its three-pronged strategy of investing in company creation, early-stage venture and late private/public opportunities, with two-thirds of its investments expected to be in Seed and Series A rounds.

    "We are extremely grateful to our limited partners for their continued support of our Life Sciences strategy, which includes creating innovative therapeutics through company creation, early-stage venture, and late private and public investing," said Patrick Heron. "We were also happy to welcome a small number of new, high-quality institutional investors to our fund."

    Recent Frazier Life Sciences IPOs and exits include Acerta (acquired by AstraZeneca), Mirum Pharmaceuticals (NASDAQ:MIRM), and Ignyta (acquired by Roche), as well as Frazier-founded companies Mavupharma (acquired by AbbVie), Phathom Pharmaceuticals (IPO: PHAT), and Silvergate Pharmaceuticals (acquired by CutisPharma). The Frazier Life Sciences team has created or seeded 23 companies since 2005.

    About Frazier Healthcare Partners

    Founded in 1991, Frazier Healthcare Partners is a leading provider of growth and venture capital to healthcare companies. With nearly $4.8 billion total capital raised, Frazier has invested in over 200 companies, with investment types ranging from company creation and venture capital to buyouts of profitable lower-middle market companies. The firm's Growth Buyout team invests in healthcare and pharmaceutical services, medical products and related sectors. The Life Sciences team invests in therapeutics and related areas that are addressing unmet medical needs through innovation. Frazier has offices in Seattle, WA, and Menlo Park, CA, and invests broadly across the U.S., Canada and Europe.

    For more information about Frazier Healthcare Partners, visit the company's website at http://www.frazierhealthcare.com.

     

    Cision View original content:http://www.prnewswire.com/news-releases/frazier-healthcare-partners-closes-oversubscribed-617-million-life-sciences-fund-300988095.html

    SOURCE Frazier Healthcare Partners

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  33. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced the promotion of Ian Clements to Chief Financial Officer. Dr. Clements joined Mirum in March 2019 and most recently served as Senior Vice President, Finance and Communications.

    "Since joining Mirum, Ian has played a pivotal role in the growth and success of the company," said Chris Peetz, president and CEO. "Ian's financial leadership through our IPO and recent financing has positioned us well for the upcoming planned NDA filing of maralixibat for pruritus due to Alagille syndrome, providing us with the financial resources expected to last the company through…

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced the promotion of Ian Clements to Chief Financial Officer. Dr. Clements joined Mirum in March 2019 and most recently served as Senior Vice President, Finance and Communications.

    "Since joining Mirum, Ian has played a pivotal role in the growth and success of the company," said Chris Peetz, president and CEO. "Ian's financial leadership through our IPO and recent financing has positioned us well for the upcoming planned NDA filing of maralixibat for pruritus due to Alagille syndrome, providing us with the financial resources expected to last the company through the potential approval."

    Prior to joining Mirum, Dr. Clements served as Vice President of Investor Relations at Dermira, Inc. Previously, Dr. Clements served as Vice President, Investor Relations and Corporate Communications at Tobira. Prior to that, he was head of investor relations and corporate communications at Avanir Pharmaceuticals. Dr. Clements has also held leadership positions at Sequenom, a genetic analysis and diagnostics company, and The Trout Group, where he managed west coast operations for life sciences investor relations. Dr. Clements received a Ph.D. in chemistry from the University of Manchester, an MBA from the Open University in the United Kingdom and a B.Sc. in chemistry from Staffordshire University.

    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 progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS). 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 planned NDA filing and potential approval for maralixibat and the adequacy of the company's financial resources. 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," "believes," "anticipates," "expects," "intends," "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, and the other risks described in Mirum's filings with the Securities and Exchange Commission , including without limitation in its Quarterly Report on Form 10-Q for the quarter ended September 30, 2019. All forward-looking statements contained in this press release speak only as of the date on which they were made. 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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  34. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced the pricing of its public offering of 2,400,000 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 estimated offering expenses, are expected to be $48.0 million.

    In addition, Mirum has granted the underwriters a 30-day option to purchase up to an additional 360,000 shares of common stock at the public offering price less underwriting discounts and commissions. The offering is expected to close on January 13, 2020, subject to satisfaction…

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced the pricing of its public offering of 2,400,000 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 estimated offering expenses, are expected to be $48.0 million.

    In addition, Mirum has granted the underwriters a 30-day option to purchase up to an additional 360,000 shares of common stock at the public offering price less underwriting discounts and commissions. The offering is expected to close on January 13, 2020, subject to satisfaction of customary closing conditions.

    Citigroup, Evercore ISI and Guggenheim Securities are acting as joint book-running managers for the offering. Raymond James is acting as lead manager. Roth Capital Partners is acting as co-manager.

    The offering is being made only by means of a prospectus. Copies of the final prospectus relating to the offering may be obtained, when available, from: Citigroup Global Markets Inc., c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by phone at (800) 831-9146; or Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, New York, NY 10055, by email at or by phone at (888) 474-0200.

    A registration statement relating to these securities has been filed with the Securities and Exchange Commission and was declared effective on January 8, 2020. 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. The company's lead product candidate, maralixibat, is an investigational oral drug in development for progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS).

     

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  35. FDA Requested Change from Type-C Meeting to pre-NDA Meeting

    Company Plans to Initiate Rolling NDA Submission in Third Quarter 2020

    Rare Pediatric Disease Designation Granted for Maralixibat for Alagille Syndrome

    Conference Call and Webcast today at 8:30 a.m. ET / 5:30 a.m. PT

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced the successful completion of its clinical pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) for maralixibat for the treatment of pruritus associated with Alagille syndrome. In addition, the company had a chemistry, manufacturing and controls…

    FDA Requested Change from Type-C Meeting to pre-NDA Meeting

    Company Plans to Initiate Rolling NDA Submission in Third Quarter 2020

    Rare Pediatric Disease Designation Granted for Maralixibat for Alagille Syndrome

    Conference Call and Webcast today at 8:30 a.m. ET / 5:30 a.m. PT

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced the successful completion of its clinical pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) for maralixibat for the treatment of pruritus associated with Alagille syndrome. In addition, the company had a chemistry, manufacturing and controls (CMC) meeting with the FDA. The purpose of the meetings was to discuss and confirm the clinical, non-clinical and CMC requirements for the company's proposed NDA submission. The company also today announced that Rare Pediatric Disease Designation has been granted for maralixibat for Alagille syndrome, and thus may qualify for the receipt of a priority review voucher if the NDA is approved by the FDA. Maralixibat was recently granted Breakthrough Therapy Designation by the FDA for Alagille syndrome.

    The company plans to pursue full approval for the indication of pruritus associated with Alagille syndrome based on feedback from the FDA and the results from the ICONIC study of maralixibat in children with Alagille syndrome. As a result of the discussions with the FDA, the company is planning a rolling NDA submission, which will be initiated in the third quarter of 2020, with full CMC sections submitted in the first quarter of 2021.

    "We are pleased that the FDA recommended that we change the classification of our requested Type C meeting to a pre-NDA meeting and that we have a path to a rolling NDA submission for maralixibat beginning in the third quarter of 2020," said Chris Peetz, president and CEO of Mirum. "We look forward to working with the FDA to bring this much-needed treatment option to children living with Alagille syndrome as rapidly as possible. As part of our commitment to patients and their families we also plan to initiate an expanded access program in the second half of 2020."

    Commenting on the recent progress, Emmanuel Gonzales M.D., Ph.D., Professor of Pediatrics, and Emmanuel Jacquemin M.D., Ph.D., Professor of Pediatrics and the study's principal investigator from the Hôpital Bicêtre, Paris, France jointly stated, "The potential that maralixibat shows is incredibly exciting. The data generated both from the ICONIC study and the long-term data presented at the Liver Meeting in November 2019 demonstrated encouraging results on cholestasis and quality of life of patients with this severe disease. New therapeutic options are desperately needed to manage cholestasis and the associated manifestations in children with Alagille syndrome."

    About the ICONIC Study in Alagille Syndrome

    The ICONIC study is a Phase 2b placebo-controlled randomized drug withdrawal study. Analyses include pruritus measures using the ItchRO (Observer) score, Clinician Xanthoma Severity Scale, Pediatric Quality of Life Inventory (PedsQL) and safety parameters.

    A total of 31 children (mean age 5.4 years) were enrolled in the study. All patients in the study were treated for 18 weeks with maralixibat (up to 400 mg/kg/day) before they were randomized to receive either placebo (n=16) or continued maralixibat (n=13) for 4 weeks. After the randomized withdrawal period, all patients were treated with maralixibat for the remainder of the 48 weeks. 29 of the 31 patients enrolled in the study were included in the Week 48 analysis.

    Overall, treatment with maralixibat led to clinically relevant and statistically significant reductions of serum bile acid (sBA) levels and pruritus compared to baseline at Weeks 18 and 48 (sBA: week18/48: p<0.001/p<0.01; pruritus week 18/48: p<0.0001). During the 4-week randomized withdrawal period, there was a significant difference in sBA levels and pruritus between those participants on placebo and those who remained on maralixibat, respectively (p<0.05 and p<0.0001). Xanthoma severity was also significantly improved (p<0.01) over the course of the study.

    Consistent with results reported after 48 weeks of treatment with maralixibat, reductions in sBA and pruritus, were statistically significant and further improved in the participants who remained on maralixibat through 191 weeks of treatment compared to baseline (p<0.005 and p<0.0001, respectively). In addition, clinician scratch scale scores continued to improve (p<0.0001) and xanthomas continued to diminish (p<0.05) with long-term treatment. Improvements were also seen in the PedsQL Multidimensional Fatigue Scale score (p<0.01). Children taking maralixibat exhibited a clinically meaningful and statistically significant acceleration in height growth as measured by height z-score (p≤0.01).

    Maralixibat was generally well tolerated. Treatment Emergent Adverse Events (TEAEs) were reported in 30 patients, most of which were mild or moderate in severity. Four patients had serious TEAEs (all considered unrelated to maralixibat), with two TEAEs leading to discontinuation (both considered unrelated to maralixibat). The most frequent TEAEs were diarrhea and abdominal pain.

    About Alagille Syndrome (ALGS)

    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 to 50,000 births in the United States and Europe. In patients with ALGS, multiple organ systems may be affected by the mutation, including the liver, heart, kidneys and central nervous system. The accumulation of bile acids prevents the liver from working properly to eliminate waste from the bloodstream and leads to progressive liver disease. Signs and symptoms arising from liver damage in ALGS may include jaundice, pruritus and xanthomas (disfiguring cholesterol deposits under the skin). 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. The progressive liver damage, pruritus, xanthomas or quality of life impacts lead to liver transplant in approximately 44% of children with ALGS.

    About Maralixibat

    Maralixibat is a novel, minimally-absorbed, orally administered investigational drug being evaluated in s