MIRM Mirum Pharmaceuticals Inc.

17.23
+0.03  (+0%)
Previous Close 17.2
Open 17.4
52 Week Low 14.94
52 Week High 27.43
Market Cap $524,711,634
Shares 30,453,374
Float 8,191,091
Enterprise Value $313,790,032
Volume 61,040
Av. Daily Volume 109,561
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Upcoming Catalysts

Drug Stage Catalyst Date
Maralixibat
Alagille Syndrome (ALGS)
PDUFA priority review
PDUFA priority review
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Volixibat (VISTAS)
Primary sclerosing cholangitis
Phase 2b
Phase 2b
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Maralixibat - MARCH
Progressive Familial Intrahepatic Cholestasis (PFIC)
Phase 3
Phase 3
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Volixiba - OHANA
Intrahepatic cholestasis of pregnancy
Phase 2b
Phase 2b
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Drug Pipeline

Drug Stage Notes
Volixibat
Pruritus associated with primary biliary cholangitis
Phase 2b
Phase 2b
Phase 2b trial to be initiated 2H 2021.
Maralixibat (EMBARK)
Biliary atresia
Phase 2
Phase 2
Phase 2 trial initiated 1Q 2021.

Latest News

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

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

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

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

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

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

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

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

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

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

    Recent Key Operational Highlights

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

    Financial Results

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

    Upcoming Anticipated Milestones

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

    Business Update Conference Call

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

    Conference Call Details:

    U.S. toll-free:

    877-261-8990

    International:

    847-619-6441

    Passcode:

    50157000

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

    About Maralixibat

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

    About Volixibat

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

    About Mirum Pharmaceuticals

    Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021. Additionally, Mirum's marketing authorization application for the treatment of pediatric patients with PFIC2 has been accepted for review (validated) by the European Medicines Agency. Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

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

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    Forward-Looking Statements

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

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

    Ended

    March 31, 2021
    Three Months

    Ended

    March 31, 2020
     
    Operating expenses:
    Research and development

    $

    38,134

     

    $

    17,340

     

    General and administrative

     

    9,479

     

     

    4,692

     

    Total operating expenses (1)

     

    47,613

     

     

    22,032

     

     
    Loss from operations

     

    (47,613

    )

     

    (22,032

    )

    Interest income

     

    149

     

     

    749

     

    Interest expense

     

    (3,381

    )

     

    -

     

    Change in fair value of derivative liability

     

    334

     

     

    -

     

    Other expense, net

     

    (16

    )

     

    (23

    )

     
    Net loss before provision for income taxes

     

    (50,527

    )

     

    (21,306

    )

    Provision for income taxes

     

    5

     

     

    4

     

    Net loss

    $

    (50,532

    )

    $

    (21,310

    )

     
    Net loss per share, basic and diluted

    $

    (1.68

    )

    $

    (0.86

    )

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

     

    30,105,017

     

     

    24,704,651

     

     
     

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

     
    Research and development

    $

    2,743

     

    $

    1,041

     

    General and administrative

     

    2,542

     

     

    1,532

     

    Total stock-based compensation

    $

    5,285

     

    $

    2,573

     

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

    March 31,

    2021

    December 31,

    2020

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

    $

    213,113

     

    $

    231,820

     

    Working capital

     

    182,733

     

     

    217,888

     

    Total assets

     

    222,241

     

     

    240,864

     

    Accumulated deficit

     

    (223,703

    )

     

    (173,171

    )

    Total stockholders' equity

     

    133,883

     

     

    172,095

     

     

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

    Conference Call Details:

    U.S. toll-free:

    877-261-8990

    International:

    847-619-6441

    Passcode:

    50157000

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

    About Mirum Pharmaceuticals

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

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

    Conference Call Details:

    U.S. toll-free:

    877-261-8990

    International:

    847-619-6441

    Passcode:

    50157000

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

    About Mirum Pharmaceuticals

    Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021. Additionally, Mirum's marketing authorization application for the treatment of pediatric patients with PFIC2 has been accepted for review (validated) by the European Medicines Agency. Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

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

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

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

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

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

    This press release…

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

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

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

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

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

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

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

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

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

    About Mirum Pharmaceuticals, Inc.

    Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021. Additionally, Mirum's marketing authorization application for the treatment of pediatric patients with PFIC2 has been accepted for review (validated) by the European Medicines Agency. Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

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

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    About CANbridge Pharmaceuticals Inc.

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

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

    About Maralixibat

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

    Forward-Looking Statements

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

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

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

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

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

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

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

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

    About Maralixibat

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

    About Mirum Pharmaceuticals, Inc.

    Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021. Additionally, Mirum's marketing authorization application for the treatment of pediatric patients with PFIC2 has been accepted for review (validated) by the European Medicines Agency. Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

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

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    Forward-Looking Statements

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

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  5. - Mirum expands potential opportunities across rare disease pipeline

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

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

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

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

    - Mirum expands potential opportunities across rare disease pipeline

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

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

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

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

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

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

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

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

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

    About Mirum Pharmaceuticals, Inc.

    Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021. Additionally, Mirum's marketing authorization application for the treatment of pediatric patients with PFIC2 has been accepted for review (validated) by the European Medicines Agency. Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

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

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    About Vivet Therapeutics

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

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

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

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

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

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

    About PFIC

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

    Forward-Looking Statements

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the option and license agreement between Mirum and Vivet and the potential development of VTX-802 and VTX-803. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "plans," "will," "may," "anticipates," "expects," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum's business in general, the impact of the COVID-19 pandemic, and the other risks described in Mirum's filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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