MIRM Mirum Pharmaceuticals Inc.

13.41
-0.91  -6%
Previous Close 14.32
Open 13.98
52 Week Low 13.42
52 Week High 26.31
Market Cap $410,858,182
Shares 30,638,194
Float 8,375,911
Enterprise Value $251,482,938
Volume 64,871
Av. Daily Volume 258,040
Stock charts supplied by TradingView

Upcoming Catalysts

Drug Stage Catalyst Date
Maralixibat - MARCH
Progressive Familial Intrahepatic Cholestasis (PFIC)
Phase 3
Phase 3
Premium membership is required to view catalyst dates, analyst ratings, earnings dates and cash burn data. Click here to unlock and sign up to a 14-day FREE TRIAL.
Volixibat - OHANA
Intrahepatic cholestasis of pregnancy
Phase 2b
Phase 2b
Lorem ipsum dolor sit amet, consectetur adipiscing elit. Quisque sapien.
Volixibat (VISTAS)
Primary sclerosing cholangitis
Phase 2b
Phase 2b
Lorem ipsum dolor sit amet, consectetur adipiscing elit. Quisque sapien.
Maralixibat (EMBARK)
Biliary atresia
Phase 2b
Phase 2b
Lorem ipsum dolor sit amet, consectetur adipiscing elit. Quisque sapien.

Drug Pipeline

Drug Stage Notes
Volixibat
Pruritus associated with primary biliary cholangitis
Phase 2b
Phase 2b
Phase 2b trial to be initiated 2H 2021.
LIVMARLI (Maralixibat)
Alagille Syndrome (ALGS)
Approved
Approved
Approved September 29, 2021.

Latest News

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

    Piper Sandler 33rd Annual Virtual Healthcare Conference
    November 30, 2021
    Format: Fireside Chat (available today, November 22, at 10:00 a.m. ET via Mirum's IR site) and one-on-one-meetings.

    4th Annual Evercore ISI HealthconX Conference
    December 2, 2021
    Format: Fireside Chat (December 2 at 3:30 p.m. ET) and one-on-one meetings.

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

    About Mirum Pharmaceuticals, Inc.

    Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases. Mirum's approved medication…

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

    Piper Sandler 33rd Annual Virtual Healthcare Conference

    November 30, 2021

    Format: Fireside Chat (available today, November 22, at 10:00 a.m. ET via Mirum's IR site) and one-on-one-meetings.

    4th Annual Evercore ISI HealthconX Conference

    December 2, 2021

    Format: Fireside Chat (December 2 at 3:30 p.m. ET) and one-on-one meetings.

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

    About Mirum Pharmaceuticals, Inc.

    Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases. Mirum's approved medication is LIVMARLI™ (maralixibat) oral solution, which is approved in the U.S. for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older.

    Mirum's late-stage pipeline includes two investigational treatments for debilitating liver diseases affecting children and adults. Maralixibat (LIVMARLI), an oral ileal bile acid transporter (IBAT) inhibitor, is currently being evaluated in clinical trials for pediatric liver diseases and includes the MARCH Phase 3 study for progressive familial intrahepatic cholestasis (PFIC) and the EMBARK Phase 2b study for patients with biliary atresia. In addition, Mirum has an expanded access program open in Canada, Australia, the UK and several countries in Europe for eligible patients with Alagille syndrome.

    Mirum has submitted a Marketing Authorization Application to the European Medicines Agency for maralixibat for the treatment of cholestatic liver disease in patients with Alagille syndrome.

    Mirum's second investigational treatment, volixibat, also an oral IBAT inhibitor, is being evaluated in two registrational studies including the OHANA Phase 2b study for pregnant women with intrahepatic cholestasis of pregnancy and the VISTAS Phase 2b study for adults with primary sclerosing cholangitis. Mirum is planning to launch a Phase 2b study in primary biliary cholangitis later this year.

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

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

     

    View Full Article Hide Full Article
  2. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a leader in rare liver disease, today announced that it has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher ("PRV") for $110 million.

    The PRV was granted by the U.S. Food and Drug Administration in September 2021 with the approval of LIVMARLI™ (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older.

    Pursuant to the agreement, Mirum will receive a payment of $110 million upon the closing of the transaction, which is subject to customary closing conditions and is expected to occur following the expiration of the applicable U.S. antitrust clearance requirements.

    Jefferies LLC acted…

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a leader in rare liver disease, today announced that it has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher ("PRV") for $110 million.

    The PRV was granted by the U.S. Food and Drug Administration in September 2021 with the approval of LIVMARLI™ (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older.

    Pursuant to the agreement, Mirum will receive a payment of $110 million upon the closing of the transaction, which is subject to customary closing conditions and is expected to occur following the expiration of the applicable U.S. antitrust clearance requirements.

    Jefferies LLC acted as the exclusive financial advisor to Mirum for this transaction.

    About LIVMARLI™ (maralixibat) oral solution

    LIVMARLI™ (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older and is the only FDA-approved medication to treat cholestatic pruritus associated with Alagille syndrome. For more information, please visit LIVMARLI.com.

    LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including progressive familial intrahepatic cholestasis and biliary atresia. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum's clinical trials section on the company's 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

    IMPORTANT SAFETY INFORMATION

    LIVMARLI can cause serious side effects, including:

    Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your healthcare provider should do blood tests before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen) or loss of appetite.

    Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you.

    A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat. FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment.

    Other common side effects reported during treatment were bone fractures and gastrointestinal bleeding.

    Prescribing information

    About Mirum Pharmaceuticals, Inc.

    Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases. Mirum's approved medication is LIVMARLI™ (maralixibat) oral solution which is approved in the U.S. for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older.

    Mirum's late-stage pipeline includes two investigational treatments for debilitating liver diseases affecting children and adults. Maralixibat (LIVMARLI), an oral ileal bile acid transporter (IBAT) inhibitor, is currently being evaluated in clinical trials for pediatric liver diseases and includes the MARCH Phase 3 study for progressive familial intrahepatic cholestasis (PFIC) and the EMBARK Phase 2b study for patients with biliary atresia. In addition, Mirum has an expanded access program open in Canada, Australia, the UK and several countries in Europe for eligible patients with Alagille syndrome.

    Mirum has submitted a Marketing Authorization Application to the European Medicines Agency for maralixibat for the treatment of cholestatic liver disease in patients with Alagille syndrome.

    Mirum's second investigational treatment, volixibat, also an oral IBAT inhibitor, is being evaluated in two registrational studies including the OHANA Phase 2b study for pregnant women with intrahepatic cholestasis of pregnancy and the VISTAS Phase 2b study for adults with primary sclerosing cholangitis. Mirum is planning to launch a Phase 2b study in primary biliary cholangitis later this year.

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

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    Forward-Looking Statements

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

    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

    View Full Article Hide Full Article
  3. - U.S. commercial launch underway following U.S. FDA approval of LIVMARLI™ (maralixibat) oral solution

    - The Lancet published data from the pivotal LIVMARLI ICONIC study demonstrating significant and durable responses in pruritus and other clinically meaningful improvements in patients with ALGS

    - Conference call to provide business updates and discuss data presented at AASLD today, November 15 at 1:30 p.m. PT/4:30 p.m. ET

    Mirum Pharmaceuticals, Inc…

    - U.S. commercial launch underway following U.S. FDA approval of LIVMARLI™ (maralixibat) oral solution

    - Six-year analysis showing significant improvement in event-free survival with LIVMARLI compared to natural history cohort (p<0.0001) presented as late-breaking, Best of The Liver Meeting presentation at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting® 2021

    - The Lancet published data from the pivotal LIVMARLI ICONIC study demonstrating significant and durable responses in pruritus and other clinically meaningful improvements in patients with ALGS

    - Conference call to provide business updates and discuss data presented at AASLD today, November 15 at 1:30 p.m. PT/4:30 p.m. ET

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

    "The FDA approval and launch of LIVMARLI capped a transformational third quarter for Mirum and the Alagille syndrome patient community. The launch of LIVMARLI, the first and only approved medication for the treatment of cholestatic pruritus in patients one year of age and older with Alagille syndrome, is off to a strong start," said Chris Peetz, president and chief executive officer at Mirum. "We believe the launch of LIVMARLI, coupled with a strong balance sheet and an advancing late-stage clinical pipeline, positions Mirum for sustained growth as we lead the way in rare liver disease worldwide."

    Recent Key Operational Highlights

    • Received U.S. Food and Drug Administration (FDA) approval for and launched LIVMARLI for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older.
    • Presented LIVMARLI six-year event-free survival late breaker data at AASLD, showing significant improvement in event-free and transplant-free survival (p<0.0001).
    • Presented clinical prognostic markers that are predictive of event-free and transplant-free survival in patients treated with LIVMARLI, furthering the understanding of clinical management of ALGS.
    • Published four-year safety and efficacy data from the ICONIC pivotal study in The Lancet, highlighting that LIVMARLI provides durable and clinically meaningful improvements across multiple disease parameters in patients with ALGS.
    • Submitted a Marketing Authorization Application (MAA) for LIVMARLI for the treatment of cholestatic liver disease in patients with ALGS to the European Medicines Agency (EMA).
    • Entered into an exclusive licensing agreement to develop and commercialize LIVMARLI for rare pediatric liver diseases in Japan.
    • Pamela Vig, Ph.D., chief scientific officer at Mirum, promoted to Head of R&D.
    • Appointed William C. Fairey, a global commercial and corporate strategy leader in rare disease, as an independent director to the Board of Directors.
    • Initiated screening in VANTAGE Phase 2b study of volixibat in primary biliary cholangitis.

    Financial Results

    • Licensing revenue for the quarter ended September 30, 2021 was $5.0 million, which was associated with Mirum's license and collaboration agreement with GC Pharma, compared to none for the third quarter of 2020.
    • Total operating expenses for the quarter ended September 30, 2021 were $47.8 million, compared to $21.7 million for the third quarter of 2020.
      • Research and development expenses for the third quarter ended September 30, 2021 were $30.5 million, compared to $16.0 million for the comparable prior-year period. The increase was primarily due to Vivet Collaboration Agreement program development funding, increases related to volixibat clinical trial expenses for primary sclerosing cholangitis (PSC), primary biliary cholangitis (PBC) and intrahepatic cholestasis of pregnancy (ICP) and related manufacturing activities supporting clinical supply, increases in personnel and other compensation-related expenses and increases for outside consulting services, regulatory fees and other general development expenses.
      • General and administrative (G&A) expenses for the third quarter of 2021 were $17.4 million, compared to $5.7 million for the comparable prior-year period. G&A investment increase in the third quarter of 2021 versus the third quarter of 2020 was primarily due to increased personnel and operational costs associated with the launch of LIVMARLI, as well as expenses related to general legal and public relation activities.
    • For the quarter ended September 30, 2021, Mirum reported a net loss of $47.1 million, or $1.55 per share, compared with a net loss of $21.5 million, or $0.86 per share for the same period in 2020.
    • As of September 30, 2021, Mirum had cash, cash equivalents, and short-term investments of $205.0 million.

    Upcoming Anticipated Milestones

    • Pipeline
      • LIVMARLI (maralixibat)
        • Phase 3 MARCH-PFIC topline data expected in the second quarter of 2022.
        • Potential EMA approval of LIVMARLI for cholestatic liver disease in patients with ALGS in second half of 2022.
        • Phase 2b EMBARK study for biliary atresia enrolling; topline data expected in 2023.
      • Volixibat:
        • Interim analyses expected in 2022 for the Phase 2b OHANA study for ICP and Phase 2b VISTAS study for PSC, two potentially registrational studies.

    Business Update Conference Call

    Mirum will host a conference call today, November 15, 2021 at 1:30 p.m. PT/4:30 p.m. ET, to provide business updates and discuss data presented today at AASLD. Join the call using the following details:

    Conference Call Details:

    U.S. toll-free:

    844-200-6205

    International:

    646-904-5544

    Passcode:

    588077

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

    About LIVMARLI™ (maralixibat) oral solution

    LIVMARLI™ (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older and is the only FDA-approved medication to treat cholestatic pruritus associated with Alagille syndrome. For more information, please visit LIVMARLI.com.

    LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including progressive familial intrahepatic cholestasis and biliary atresia. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum's clinical trials section on the company's website.

    IMPORTANT SAFETY INFORMATION

    LIVMARLI can cause serious side effects, including:

    Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your healthcare provider should do blood tests before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen) or loss of appetite.

    Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you.

    A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat. FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment.

    Other common side effects reported during treatment were bone fractures and gastrointestinal bleeding.

    Prescribing information

    About Volixibat

    Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, 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 IBAT inhibition, in addition to decreases in LDL cholesterol and increases in 7αC4 which are markers of bile acid synthesis. Volixibat has been evaluated in more than 400 individuals across multiple clinical trials. The most common adverse events reported were mild to moderate gastrointestinal events observed in the volixibat groups.

    Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (VISTAS study), intrahepatic cholestasis of pregnancy (OHANA study) and primary biliary cholangitis (VANTAGE study).

    About Mirum Pharmaceuticals, Inc.

    Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases. Mirum's approved medication is LIVMARLI™ (maralixibat) oral solution which is approved in the U.S. for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older.

    Mirum's late-stage pipeline includes two investigational treatments for debilitating liver diseases affecting children and adults. Maralixibat (LIVMARLI), an oral ileal bile acid transporter (IBAT) inhibitor, is currently being evaluated in clinical trials for pediatric liver diseases and includes the MARCH Phase 3 study for progressive familial intrahepatic cholestasis (PFIC) and the EMBARK Phase 2b study for patients with biliary atresia. In addition, Mirum has an expanded access program open in Canada, Australia, the UK and several countries in Europe for eligible patients with Alagille syndrome.

    Mirum has submitted a Marketing Authorization Application to the European Medicines Agency for maralixibat for the treatment of cholestatic liver disease in patients with Alagille syndrome.

    Mirum's second investigational treatment, volixibat, also an oral IBAT inhibitor, is being evaluated in three registrational studies including the OHANA Phase 2b study for pregnant women with intrahepatic cholestasis of pregnancy, the VISTAS Phase 2b study for adults with primary sclerosing cholangitis, and the VANTAGE Phase 2b study for primary biliary cholangitis.

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

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    Forward-Looking Statements

    This press release includes forward-looking statements pertaining to the Company's planned participation at a scientific conference, which may include discussion of the Company's revenue, business, and operations, including the discovery, development and commercialization of our product candidates and technologies, and the therapeutic potential thereof, the continuation of our clinical trials, and the success of our collaborations with partners and any potential future collaborations. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Applicable risks and uncertainties include those relating to our preclinical research and clinical programs and other risks identified under the heading "Risk Factors" included in our most recent Form 10-Q and Form 10-K filings and in other future filings with the SEC. The forward-looking statements contained in this press release reflect Mirum's current views with respect to future events, and Mirum does not undertake and specifically disclaims any obligation to update any forward-looking statements.

     
    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,

     

    2021

     

     

     

    2020

     

     

     

    2021

     

     

     

    2020

     

     
    License revenue

    $

    5,000

     

    $

    -

     

    $

    16,000

     

    $

    -

     

     
    Operating expenses:
    Research and development

     

    30,471

     

     

    15,984

     

     

    103,653

     

     

    51,879

     

    General and administrative

     

    17,353

     

     

    5,732

     

     

    40,185

     

     

    15,466

     

    Total operating expenses (1)

     

    47,824

     

     

    21,716

     

     

    143,838

     

     

    67,345

     

     
    Loss from operations

     

    (42,824

    )

     

    (21,716

    )

     

    (127,838

    )

     

    (67,345

    )

    Other income (expense):
    Interest income

     

    72

     

     

    237

     

     

    301

     

     

    1,391

     

    Interest expense

     

    (5,667

    )

     

    -

     

     

    (13,824

    )

     

    -

     

    Change in fair value of derivative liability

     

    1,355

     

     

    -

     

     

    417

     

     

    -

     

    Other expense, net

     

    (35

    )

     

    (30

    )

     

    (565

    )

     

    (109

    )

     
    Net loss before provision for income taxes

     

    (47,099

    )

     

    (21,509

    )

     

    (141,509

    )

     

    (66,063

    )

    Provision for (benefit from) income taxes

     

    9

     

     

    (3

    )

     

    25

     

     

    4

     

    Net loss

    $

    (47,108

    )

    $

    (21,506

    )

    $

    (141,534

    )

    $

    (66,067

    )

     
    Net loss per share, basic and diluted

    $

    (1.55

    )

    $

    (0.86

    )

    $

    (4.68

    )

    $

    (2.65

    )

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

     

    30,367,727

     

     

    25,132,916

     

     

    30,250,127

     

     

    24,965,178

     

     
     

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

     
    Research and development

    $

    3,035

     

    $

    1,361

     

    $

    7,792

     

    $

    3,662

     

    General and administrative

     

    4,380

     

     

    2,067

     

     

    9,731

     

     

    5,313

     

    Total stock-based compensation

    $

    7,415

     

    $

    3,428

     

    $

    17,523

     

    $

    8,975

     

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

     

     

     

    September 30,

     

    December 31,

     

    2021

     

     

     

    2020

     

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

    $

    205,031

     

    $

    231,820

     

    Working capital

     

    146,418

     

     

    217,888

     

    Total assets

     

    235,167

     

     

    240,864

     

    Accumulated deficit

     

    (314,705

    )

     

    (173,171

    )

    Total stockholders' equity

     

    56,396

     

     

    172,095

     

     

    View Full Article Hide Full Article
  4. -

    - Study presented by Global Alagille Alliance (GALA) at The Liver Meeting® (AASLD) in a late-breaking oral presentation; selected to be featured in Best of Liver Meeting series

    - Data underscore LIVMARLI's potential to delay liver transplant and improve disease outcomes associated with Alagille syndrome

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced that a new analysis comparing pooled LIVMARLI™ (maralixibat) oral solution clinical trial data with a natural history cohort was presented at the American Association for the Study of Liver Diseases annual congress, The Liver Meeting®. LIVMARLI…

    - Results demonstrate statistically significant improvement in six-year event-free survival (p<0.0001) and transplant-free survival (p<0.0001)

    - Study presented by Global Alagille Alliance (GALA) at The Liver Meeting® (AASLD) in a late-breaking oral presentation; selected to be featured in Best of Liver Meeting series

    - Data underscore LIVMARLI's potential to delay liver transplant and improve disease outcomes associated with Alagille syndrome

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced that a new analysis comparing pooled LIVMARLI™ (maralixibat) oral solution clinical trial data with a natural history cohort was presented at the American Association for the Study of Liver Diseases annual congress, The Liver Meeting®. LIVMARLI is an oral ileal bile acid transporter (IBAT) inhibitor recently approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211115006047/en/

    Significant Improvement in Event-Free Survival in Patients with ALGS with LIVMARLI Compared to Untreated Cohort​ (Graphic: Business Wire)

    Significant Improvement in Event-Free Survival in Patients with ALGS with LIVMARLI Compared to Untreated Cohort​ (Graphic: Business Wire)

    The oral, late-breaking session, which was selected as part of the "Best of the Liver Meeting" series, reported on an analysis independently conducted and presented by Dr. Bettina Hansen on behalf of Dr. Binita Kamath and the Global Alagille Alliance (GALA) Study Group, which has aggregated the largest global natural history clinical database established for ALGS. The analysis evaluated six years of follow-up from pooled LIVMARLI studies (n=84) in ALGS and compared it against an external natural history control cohort from the GALA database. The objective of the analysis was to compare the time to first clinical event in LIVMARLI-treated patients with ALGS versus a natural history cohort, with events defined as liver transplantation, biliary diversion surgery, decompensation events (ascites requiring therapy or variceal bleeding), or death. Additional analyses included transplant-free survival as well as several sensitivity and subgroup analyses to ensure robustness of the findings. The GALA control group was selected based on alignment with eligibility criteria from the LIVMARLI clinical studies.

    The analysis demonstrated a highly significant improvement in six-year event-free survival with a p-value of <0.0001 (HR: 0.305, 95% CI: 0.189-0.491) translating to a 70% overall reduction for clinical outcomes with LIVMARLI. The analysis also showed statistically significant improvements in transplant-free survival with a p-value of <0.0001 (HR: 0.332, 95% CI: 0.197-0.559). These data underscore LIVMARLI's potential to have long-term and sustained impact for patients with ALGS.

    "The extensive real-world analysis conducted by GALA validates the statistical robustness of the findings and suggest that patients who are treated with LIVMARLI may experience statistically significant event-free survival and transplant-free survival versus the natural history cohort," said Bettina Hansen, Ph.D., Associate Professor of Biostatistics at the Toronto Centre for Liver Disease, Toronto General Hospital, Toronto General Hospital & University of Toronto, Canada, on behalf of the GALA Study Group.

    "Cholestatic pruritus associated with Alagille syndrome has a dramatic and debilitating impact on the lives of patients and is a leading indication for liver transplantation," said Pam Vig, Ph.D., Chief Scientific Officer and Head of R&D at Mirum. "The six-year analysis demonstrates that LIVMARLI significantly improves event-free survival and transplant-free survival, further supporting the impact of LIVMARLI in this patient population."

    Also at the meeting, a second late-breaker presentation was given by Dr. Ron Sokol. These complementary data analyzed predictors of event-free survival and transplant-free survival in 76 patients treated with LIVMARLI. Variables that were predictive of event-free survival included specific thresholds of week 48 total bilirubin, and week 48 serum bile acids. Change in baseline to week 48 in pruritus was also predictive of event-free survival and transplant-free survival. Sixty out of 76 patients remained event-free at the time of analysis, with up to six years of treatment with LIVMARLI. These data highlighted potential prognostic markers that could help to inform medical management for patients receiving LIVMARLI treatment.

    To learn more about the data presented during the meeting, please visit the Alagille syndrome section of our Publications & Presentations page on Mirum's website.

    The results of the natural history comparison analysis were included in Mirum's recent marketing authorization application submission (MAA) to the European Medicines Agency (EMA) for LIVMARLI in the treatment of cholestatic liver disease in patients with ALGS. The MAA is currently under review and Mirum is preparing for a launch of LIVMARLI in Europe in the second half of 2022, if approved by the EMA.

    About LIVMARLI™ (maralixibat) oral solution

    LIVMARLI™ (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older and is the only FDA-approved medication to treat cholestatic pruritus associated with Alagille syndrome. For more information, please visit LIVMARLI.com.

    LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including progressive familial intrahepatic cholestasis and biliary atresia. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum's clinical trials section on the company's 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

    IMPORTANT SAFETY INFORMATION

    LIVMARLI can cause serious side effects, including:

    Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your healthcare provider should do blood tests before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen) or loss of appetite.

    Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you.

    A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat. FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment.

    Other common side effects reported during treatment were bone fractures and gastrointestinal bleeding.

    Prescribing information

    About the Global Alagille Alliance (GALA)

    Launched in October 2017, the Global ALagille Alliance (GALA) Study is advancing research and changing lives. By pooling together medical records from around the world to create a vast one-stop database of patient information, this three-year effort will be the bedrock of future research projects that will unravel the mysteries of Alagille Syndrome (ALGS). The cornerstone of this effort is a comprehensive medical record review of patients with ALGS up to 30 years of age from 1997 to present. GALA's overarching aim is to conduct a comprehensive analysis of an international group of individuals with ALGS which will increase understanding of the disease and identify the challenges and needs of this unique population.

    About Mirum Pharmaceuticals, Inc.

    Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases. Mirum's approved medication is LIVMARLI™ (maralixibat) oral solution which is approved in the U.S. for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older.

    Mirum's late-stage pipeline includes two investigational treatments for debilitating liver diseases affecting children and adults. Maralixibat (LIVMARLI), an oral ileal bile acid transporter (IBAT) inhibitor, is currently being evaluated in clinical trials for pediatric liver diseases and includes the MARCH Phase 3 study for progressive familial intrahepatic cholestasis (PFIC) and the EMBARK Phase 2b study for patients with biliary atresia. In addition, Mirum has an expanded access program open in Canada, Australia, the UK and several countries in Europe for eligible patients with Alagille syndrome.

    Mirum has submitted a Marketing Authorization Application to the European Medicines Agency for LIVMARLI for the treatment of cholestatic liver disease in patients with Alagille syndrome.

    Mirum's second investigational treatment, volixibat, also an oral IBAT inhibitor, is being evaluated in two registrational studies including the OHANA Phase 2b study for pregnant women with intrahepatic cholestasis of pregnancy and the VISTAS Phase 2b study for adults with primary sclerosing cholangitis. Mirum is planning to launch a Phase 2b study in primary biliary cholangitis later this year.

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

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    Forward-Looking Statements

    This press release includes forward-looking statements pertaining to the Company's planned participation at a scientific conference, which may include discussion of the Company's product candidates and technologies, and the therapeutic potential thereof. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Applicable risks and uncertainties include those relating to our preclinical research and clinical programs and other risks identified under the heading "Risk Factors" included in our most recent Form 10-Q and Form 10-K filings and in other future filings with the SEC. The forward-looking statements contained in this press release reflect Mirum's current views with respect to future events, and Mirum does not undertake and specifically disclaims any obligation to update any forward-looking statements.

    The Liver Meeting® is a registered trademark of the American Association for the Study of Liver Diseases.

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

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

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

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

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

    Conference Call Details:

    U.S. toll-free: 844.200.6205
    International: 646.904.5544
    Passcode: 588077

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

    About Mirum Pharmaceuticals, Inc.

    Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases. Mirum's approved medication…

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

    Conference Call Details:

    U.S. toll-free: 844.200.6205

    International: 646.904.5544

    Passcode: 588077

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

    About Mirum Pharmaceuticals, Inc.

    Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases. Mirum's approved medication is LIVMARLI™ (maralixibat) oral solution which is approved in the U.S. for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older.

    Mirum's late-stage pipeline includes two investigational treatments for debilitating liver diseases affecting children and adults. Maralixibat (LIVMARLI), an oral ileal bile acid transporter (IBAT) inhibitor, is currently being evaluated in clinical trials for pediatric liver diseases and includes the MARCH Phase 3 study for progressive familial intrahepatic cholestasis (PFIC) and the EMBARK Phase 2b study for patients with biliary atresia. In addition, Mirum has an expanded access program open in Canada, Australia, the UK and several countries in Europe for eligible patients with Alagille syndrome.

    Mirum has submitted a Marketing Authorization Application to the European Medicines Agency for maralixibat for the treatment of cholestatic liver disease in patients with Alagille syndrome.

    Mirum's second investigational treatment, volixibat, also an oral IBAT inhibitor, is being evaluated in two registrational studies including the OHANA Phase 2b study for pregnant women with intrahepatic cholestasis of pregnancy and the VISTAS Phase 2b study for adults with primary sclerosing cholangitis. Mirum is planning to launch a Phase 2b study in primary biliary cholangitis later this year.

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

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    View Full Article Hide Full Article
View All Mirum Pharmaceuticals Inc. News