US Market News
1日前
Mirum Pharmaceuticals and Incyte Announce Pivotal Late-Breaking Results for Zilurgisertib in Fibrodysplasia Ossificans Progressiva Accepted for Presentation at ENDO 2026June 4, 2026 8:05 AM
Business Wire - Late-breaking rapid-fire presentation to include results from Cohort 1 of the placebo-controlled PROGRESS study evaluating zilurgisertib in fibrodysplasia ossificans progressiva (FOP) Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) and Incyte (Nasdaq: INCY) today announced that pivotal Phase 2 results from the PROGRESS study evaluating zilurgisertib, an investigational ALK2 inhibitor, in patients with fibrodysplasia ossificans progressiva (“FOP”) will be presented at ENDO 2026, the Endocrine Society’s annual meeting, taking place June 13-16, 2026, in Chicago, Illinois. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260604863826/en/ The late-breaking rapid-fire presentation will include results from Cohort 1 of the placebo-controlled PROGRESS study, which enrolled patients 12 years of age and older and formed the basis of the New Drug Application (NDA) for zilurgisertib to the U.S. Food and Drug Administration (FDA). In April 2026, Mirum entered into an exclusive license agreement with Incyte for worldwide rights to zilurgisertib. “ENDO 2026 is an important milestone for the zilurgisertib FOP program as we share pivotal results from the PROGRESS study in patients living with this debilitating disease,” said Steven Stein, M.D., Executive Vice President, Chief Medical Officer and Head of Late-stage Development at Incyte. “These data add to the growing clinical understanding of zilurgisertib’s potential in FOP as Incyte and Mirum continue to advance toward the FDA’s Priority Review PDUFA date of September 26, 2026.” “FOP is a devastating, progressive disease that profoundly impacts patients and families,” said Joanne Quan, M.D., Chief Medical Officer at Mirum Pharmaceuticals. “At Mirum, in partnership with Incyte, we are committed to advancing zilurgisertib with urgency as we work toward potentially bringing a needed new treatment option to people living with FOP.” Congress Presentation Additional details regarding the presentation are as follows: Abstract # ORF37-04 Title Zilurgisertib in Patients with Fibrodysplasia Ossificans Progressiva: Interim Results from the PROGRESS Study Date/Time Sunday, June 14, 3:30-4:15 pm CT Abstracts accepted for presentation at ENDO 2026 will be available once published through the Endocrine Society’s ENDO 2026 website. About Zilurgisertib Zilurgisertib is an investigational, oral, small molecule, activin receptor-like kinase 2 (ALK2) inhibitor in development for the treatment of Fibrodysplasia Ossificans Progressiva (FOP). Zilurgisertib is designed to inhibit the ALK2 receptor, which is abnormally active in most patients with FOP and leads to bone formation in soft tissues, a process known as heterotopic ossification (HO). FOP is an ultra-rare genetic disease that affects approximately 300 patients in the U.S. and 900 worldwide, with diagnosis typically occurring in early childhood. Zilurgisertib was evaluated in the PROGRESS pivotal Phase 2 study, which formed the basis of a new drug application (NDA). The FDA has accepted the NDA for zilurgisertib in FOP under Priority Review with a Prescription Drug User Fee Act (PDUFA) date of September 26, 2026. Mirum licensed zilurgisertib from Incyte for development and commercialization globally. About the PROGRESS Study PROGRESS is a global, randomized, double-blind, placebo-controlled Phase 2 study evaluating the efficacy and safety of zilurgisertib in patients with fibrodysplasia ossificans progressiva (FOP). PROGRESS Cohort 1 enrolled patients 12 years of age and older who were randomized 1:1 to receive zilurgisertib 100 mg once daily or placebo during a 24-week double-blind treatment period, followed by an open-label extension. Additional PROGRESS cohorts will evaluate the efficacy and safety of zilurgisertib in patients ages 6 to
US Market News
2週前
Mirum Pharmaceuticals to Present Data Showcasing Leadership in Rare Liver Diseases at the EASL International Liver Congress 2026May 21, 2026 8:05 AM
Business Wire - Late-breaking VISTAS results demonstrate rapid, sustained, and clinically meaningful reductions in pruritus with volixibat in primary sclerosing cholangitis (PSC) - Late-breaking Phase 2b AZURE-1 results demonstrate efficacy and safety of brelovitug for the treatment of chronic hepatitis delta virus (HDV) - Additional presentations highlight long-term outcomes and real-world experience with LIVMARLI® (maralixibat) in progressive familial intrahepatic cholestasis (PFIC) Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that it will present new data at the European Association for the Study of the Liver (EASL) International Liver Congress 2026, taking place May 27-30, 2026, in Barcelona, Spain. Presentations span Mirum’s rare liver disease programs, including late-breaking data from the company’s Phase 2b VISTAS study of volixibat in PSC and Phase 2b AZURE-1 study of brelovitug in HDV, alongside data featuring its established therapy, LIVMARLI® (maralixibat) in PFIC. Together, these data highlight the breadth of Mirum’s rare liver disease portfolio. “We’ve built our rare liver disease business by staying close to patients and focusing on the needs and outcomes that most affect their daily lives,” said Chris Peetz, Chief Executive Officer at Mirum. “At the EASL Congress 2026, we’re sharing data that expand on our experience in rare cholestatic liver diseases while also extending into new areas like HDV, where there remains a significant unmet need.” Congress Presentations Mirum’s presentations at the EASL Congress 2026 reflect continued advancement across approved and investigational programs. All presentations were titled by their respective authors. Volixibat Presentations include results from the Phase 2b VISTAS study of volixibat in PSC, alongside data characterizing the PSC disease burden and patient experience. ID Type Title Presenter Date/Time Location LBO-006 Oral (Late Breaker; Best of EASL) Efficacy and safety of volixibat, an IBAT inhibitor, in patients with primary sclerosing cholangitis and moderate-to-severe pruritus: Results of the VISTAS trial Cynthia Levy, MD, FAASLD, AGAF (University of Miami, USA) Saturday, May 30, 2026, 2:15–2:30 pm CEST Summerfield – Hall 8.0 SAT-301 Poster Investigating the burden of illness in primary sclerosing cholangitis (PSC): A multinational study Deepak Joshi, MBBS, PhD, FRCP (King’s College Hospital, UK) Saturday, May 30, 2026, 11:30 am–12:30 pm CEST Poster Area – Hall 7 SAT-365 Poster Real-world survey assessing pruritus in PSC in France, Germany, Italy and the United States: Results from a matched patient and their treating physician cohort Marlyn Mayo, MD (UT Southwestern Medical Center, USA) Saturday, May 30, 2026, 11:30 am–12:30 pm CEST Poster Area – Hall 7 SAT-403 Poster Analysis of structured and unstructured data from a large electronic health record system identifies frequent pruritus and fatigue in patients with PSC Sarah Sidhu, MD (Mirum Pharmaceuticals, Inc., USA) Saturday, May 30, 2026, 11:30 am–12:30 pm CEST Poster Area – Hall 7 Brelovitug Featured late-breaking presentation of the Phase 2b AZURE-1 study evaluating brelovitug in patients with HDV. ID Type Title Presenter Date/Time Location LBP-021 Poster (Late Breaker) 24-week safety and efficacy of brelovitug monotherapy for the treatment of chronic hepatitis D: Data from phase 2b of AZURE-1 Tatyana Kushner, MD, MSCE (Weill Cornell Medicine, USA) Friday, May 29, 2026, 12:30–1:30 pm CEST Poster Area – Hall 7 Maralixibat Presentations will highlight clinical and real-world outcomes with maralixibat in PFIC, along with emerging data in intrahepatic cholestasis of pregnancy (ICP). ID Type Title Presenter Date/Time Location OS-086 Oral; Best of EASL Event-free survival of maralixibat-treated patients with progressive familial intrahepatic cholestasis compared with aligned real-world data of untreated patients from NAPPED Bettina E. Hansen, Professor, PhD (Toronto General Hospital Research Institute, Canada) Saturday, May 30, 2026, 10:15–10:30 am CEST Rizzetto – Hall 8.0 SAT-366 Poster Use of maralixibat for the treatment of cholestatic pruritus in late-onset PFIC Marlyn Mayo, MD (UT Southwestern Medical Center, USA) Saturday, May 30, 2026, 11:30 am–12:30 pm CEST Poster Area – Hall 7 SAT-434 Poster (Exploratory Case Series) Utilization of maralixibat, an ileal bile acid transporter inhibitor, for the treatment of intrahepatic cholestasis of pregnancy: A case series Catherine Williamson, Professor, MD (Imperial College, UK) Saturday, May 30, 2026, 11:30 am–12:30 pm CEST Poster Area – Hall 7 Mirum’s presence at the EASL Congress 2026 underscores the company’s commitment to advancing therapies across rare liver diseases. Full abstracts are available on the EASL Congress website. Following the congress, detailed analyses will be posted on the Publications & Presentations section of Mirum’s website. About LIVMARLI® (maralixibat) oral solution and tablets LIVMARLI® (maralixibat) is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI.com. LIVMARLI has received orphan designation for ALGS and PFIC. LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website. IMPORTANT SAFETY INFORMATION Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein. LIVMARLI can cause side effects, including Liver injury. Changes in certain liver tests are common in patients with ALGS and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams 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), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal. Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. 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 is common in patients with Alagille syndrome and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects. US Prescribing Information
EU SmPC
Canadian Product Monograph About Volixibat Volixibat is an investigational 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. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study), and primary biliary cholangitis (PBC) (VANTAGE study). In 2026, Mirum shared that the Phase 2b VISTAS study of volixibat in PSC met its primary endpoint, with statistically significant and clinically meaningful reductions in pruritus observed in patients treated with volixibat. Volixibat’s safety profile in the study was generally consistent with the known effects of IBAT inhibition. In 2024, Mirum announced positive interim results from the Phase 2b VANTAGE study of volixibat in PBC. No new safety signals were observed in the study. Volixibat has been granted FDA Breakthrough Therapy designation for the treatment of PBC. About Brelovitug Brelovitug is an investigational, highly potent, pan-genotypic, fully human immunoglobulin G1 (IgG1) monoclonal antibody (mAb) that targets the surface antigen (anti-HBsAg) on both the hepatitis delta virus (HDV) and the hepatitis B virus (HBV). Brelovitug is designed to neutralize and remove hepatitis B and hepatitis D virions and deplete HBsAg-containing subviral particles. Brelovitug has FDA Breakthrough Therapy designation for the treatment of chronic HDV infection and PRIME and Orphan designations from the European Medicines Agency. In 2026, Mirum announced that in the Phase 2b portion of the AZURE-1 study in HDV, treatment with brelovitug demonstrated strong antiviral activity in HDV and achieved the primary composite endpoint of virologic response and alanine aminotransferase (ALT) normalization at Week 24 in both brelovitug dose arms as compared to the delayed treatment arm. Favorable safety and tolerability profiles were observed. Brelovitug is currently being evaluated in the global Phase 3 AZURE clinical program. Mirum owns worldwide rights to brelovitug. About Mirum Pharmaceuticals Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum focuses on rare liver and rare genetic diseases, where it has built deep expertise and strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM® (cholic acid) for bile-acid synthesis disorders, and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX). Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV), zilurgisertib, an ALK2 inhibitor under regulatory review with the FDA for fibrodysplasia ossificans progressiva (FOP), and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS). Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at www.mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and X. 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 Company’s planned participation at a scientific congress, Mirum’s continued or advancing leadership in rare liver disease and the potential benefit of Mirum products and candidates in real world settings versus scientific presentations of data at the EASL Congress 2026. 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 “expected,” “will,” “could,” “would,” “guidance,” “potential,” “continue” 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 geopolitical and macroeconomic events, and the other risks described in Mirum’s Annual Report for the year ended December 31, 2024, filed with the Securities and Exchange Commission on February 26, 2025, and subsequent filings with the Securities and Exchange Commission, which are available at www.sec.gov. 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 and the Mirum logo are trademarks of Mirum Pharmaceuticals, Inc. View source version on businesswire.com: https://www.businesswire.com/news/home/20260521728786/en/ Investor Contact:
Andrew McKibben
ir@mirumpharma.com Media Contact:
Meredith Kiernan
media@mirumpharma.com Original: Mirum Pharmaceuticals to Present Data Showcasing Leadership in Rare Liver Diseases at the EASL International Liver Congress 2026
US Market News
3週前
Mirum Pharmaceuticals Prices $600.0 Million Convertible Senior Notes Offering; Refinances a Portion of 2029 Convertible NotesMay 12, 2026 11:56 PM
Business Wire Mirum Pharmaceuticals, Inc. (“Mirum”) (Nasdaq: MIRM), a leading rare disease company, today announced the pricing of its offering of $600.0 million aggregate principal amount of 0.00% convertible senior notes due 2032 (the “notes”) in a private placement (the “offering”) to persons reasonably believed to be qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended (the “Securities Act”). The issuance and sale of the notes are scheduled to settle on May 15, 2026, subject to customary closing conditions. Mirum also granted the initial purchasers of the notes an option to purchase, for settlement within a period of 13 days from, and including, the date the notes are first issued, up to an additional $90.0 million aggregate principal amount of notes. The notes will be senior, unsecured obligations of Mirum. The notes will not bear regular interest and the principal amount of the notes will not accrete. The notes will mature on June 1, 2032, unless earlier converted, redeemed or repurchased. Before March 1, 2032, noteholders will have the right to convert their notes only in certain circumstances and during specified periods. From and after March 1, 2032, noteholders may convert all or any portion of their notes at any time at their election until the close of business on the second scheduled trading day immediately before the maturity date. Mirum will settle conversions by paying or delivering, as applicable, cash, shares of its common stock or a combination of cash and shares of its common stock, at Mirum’s election. The initial conversion rate is 7.1971 shares of common stock per $1,000 principal amount of notes, which represents an initial conversion price of approximately $138.94 per share of common stock. The initial conversion price represents a premium of approximately 30.0% over the last reported sale price of $106.88 per share of Mirum’s common stock on May 12, 2026. The conversion rate will be subject to adjustment upon the occurrence of certain events. If a “make-whole fundamental change” (as defined in the indenture for the notes) occurs, then Mirum will in certain circumstances increase the conversion rate for a specified period of time. Mirum may not redeem the notes at its election at any time before June 6, 2029. The notes will be redeemable, in whole or in part (subject to a partial redemption limitation), for cash at Mirum’s option at any time, and from time to time, on a redemption date on or after June 6, 2029 and, in the case of any partial redemption, on or before the 30th scheduled trading day immediately before the maturity date, at a cash redemption price equal to the principal amount of the notes to be redeemed, plus accrued and unpaid special interest and additional interest, if any, to, but excluding, the redemption date, but only if (i) the notes are “freely tradable” (as defined in the indenture for the notes) as of the date Mirum sends the related redemption notice and all accrued and unpaid additional interest, if any, has been paid in full as of the first interest payment date occurring on or before the date Mirum sends such notice; and (ii) the last reported sale price per share of Mirum’s common stock exceeds 130% of the conversion price on (1) each of at least 20 trading days, whether or not consecutive, during the 30 consecutive trading days ending on, and including, the trading day immediately before the date Mirum sends the related redemption notice; and (2) the trading day immediately before the date Mirum sends such notice. In addition, calling any note for redemption will constitute a make-whole fundamental change with respect to that note, in which case the conversion rate applicable to the conversion of that note will be increased in certain circumstances if it is converted after it is called for redemption. If a “fundamental change” (as defined in the indenture for the notes) occurs, then, subject to a limited exception, noteholders may require Mirum to repurchase their notes at a cash repurchase price equal to the principal amount of the notes to be repurchased, plus accrued and unpaid special interest and additional interest, if any, to, but excluding, the fundamental change repurchase date. Mirum estimates that the net proceeds from the offering will be approximately $583.8 million (or approximately $671.6 million if the initial purchasers fully exercise their option to purchase additional notes), after deducting the initial purchasers’ discounts and commissions and Mirum’s estimated offering expenses. Mirum expects to use a portion of the net proceeds from the offering to pay the cash portion of the consideration in the note exchange transactions as described below. Mirum expects to use the remainder of the net proceeds of this offering for general corporate purposes, which may include the acquisition of complementary products, technologies, intellectual property or businesses as part of its growth strategy. Mirum expects to use approximately $475.0 million of the net proceeds from the offering and to issue approximately 3.2 million shares of its common stock in exchange for approximately $237.2 million aggregate principal amount of the 4.00% convertible senior notes due 2029 (the “2029 notes”) in privately negotiated transactions (each, a “note exchange transaction”) entered into concurrently with the pricing of the offering. This press release is not an offer to exchange the 2029 notes, and the offering of the notes is not contingent upon the note exchange transactions. In connection with any note exchange transaction, Mirum expects that holders of the 2029 notes who agree to have their 2029 notes exchanged and who have hedged their equity price risk with respect to such 2029 notes (the “hedged holders”) will, concurrently with, or shortly after, the pricing of the notes, unwind all or part of their hedge positions by buying Mirum’s common stock and/or entering into or unwinding various derivative transactions with respect to its common stock. The amount of Mirum’s common stock to be purchased by the hedged holders or the notional number of shares of Mirum’s common stock underlying such derivative transactions may be substantial in relation to the historical average daily trading volume of Mirum’s common stock. This activity by the hedged holders could increase (or reduce the size of any decrease in) the market price of Mirum’s common stock. Mirum cannot predict the magnitude of such market activity or the overall effect it will have on the price of the notes or its common stock. The offer and sale of the notes, any shares of common stock issuable upon conversion of the notes and any shares of common stock issuable in connection with any note exchange transaction have not been, and will not be, registered under the Securities Act, any state securities laws or the securities laws of any other jurisdiction, and unless so registered, may not be offered or sold except pursuant to an exemption from, or in a transaction not subject to, the registration requirements of the Securities Act and any other applicable securities laws. This press release does not constitute an offer to sell, or the solicitation of an offer to buy, any of these securities nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, sale or solicitation would be unlawful. About Mirum Pharmaceuticals, Inc. Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum focuses on rare liver and rare genetic diseases, where it has built deep expertise and strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM® (cholic acid) for bile-acid synthesis disorders, and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX). Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV), zilurgisertib, an ALK2 inhibitor under regulatory review with the FDA for fibrodysplasia ossificans progressiva (FOP), and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS). Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. 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 completion of the proposed offering and note exchange transactions, the intended use of the proceeds and the potential impact of the foregoing or related transactions on the market price of Mirum’s common stock or the price of the notes. 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 “anticipate,” “expected,” “will,” “could,” “would,” “guidance,” “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 related to associated with market conditions, including market interest rates, the trading price and volatility of Mirum’s common stock, Mirum’s business in general, the impact of geopolitical and macroeconomic events, and the other risks described in Mirum’s Quarterly Report on Form 10-Q for the period ended March 31, 2026 and subsequent filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. View source version on businesswire.com: https://www.businesswire.com/news/home/20260512532949/en/ Investor Contact:
Andrew McKibben
ir@mirumpharma.com Media Contact:
Meredith Kiernan
media@mirumpharma.com Original: Mirum Pharmaceuticals Prices $600.0 Million Convertible Senior Notes Offering; Refinances a Portion of 2029 Convertible Notes
US Market News
2月前
Mirum Pharmaceuticals Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)April 13, 2026 4:01 PM
Business Wire
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that on April 10, 2026, the Compensation Committee of Mirum’s Board of Directors granted inducement awards consisting of non-qualified stock options to purchase 13,700 shares of common stock and 18,200 restricted stock units (“RSUs”) to 12 new employees under Mirum’s 2020 Inducement Plan. The Compensation Committee of Mirum’s Board of Directors approved the awards as an inducement material to the new employees’ employment in accordance with Nasdaq Listing Rule 5635(c)(4).
Each stock option has an exercise price of $95.20 per share, which is equal to the closing price of Mirum’s common stock on April 10, 2026, and will vest over four years, with 25% of the underlying shares vesting on the one-year anniversary of the applicable vesting commencement date, and the balance of the underlying shares vesting monthly thereafter over 36 months, subject to the new employees’ continued service relationship with Mirum through the applicable vesting dates. The RSUs will vest over three years, with 33% of the underlying shares vesting on each anniversary of the applicable initial vesting date, subject to the new employees’ continued service relationship with Mirum through the applicable vesting dates. The awards are subject to the terms and conditions of Mirum’s 2020 Inducement Plan and the terms and conditions of an applicable award agreement covering the grant.
About Mirum Pharmaceuticals
Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum combines deep rare disease expertise with strong connections to patient communities.
The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM® (cholic acid) for bile-acid synthesis disorders, and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX). Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV) and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS).
Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at www.mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and X.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260413572080/en/
Investor Contact:
Andrew McKibben
ir@mirumpharma.com
Media Contact:
Meredith Kiernan
media@mirumpharma.com
Original: Mirum Pharmaceuticals Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
US Market News
3月前
Mirum Pharmaceuticals Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)March 12, 2026 5:30 PM
Business Wire
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that on March 11, 2026, the Compensation Committee of Mirum’s Board of Directors granted inducement awards consisting of non-qualified stock options to purchase 5,400 shares of common stock and 6,800 restricted stock units (“RSUs”) to five new employees under Mirum’s 2020 Inducement Plan. The Compensation Committee of Mirum’s Board of Directors approved the awards as an inducement material to the new employees’ employment in accordance with Nasdaq Listing Rule 5635(c)(4).
Each stock option has an exercise price of $91.75 per share, which is equal to Mirum’s closing trading price on March 11, 2026, and will vest over four years, with 25% of the underlying shares vesting on the one-year anniversary of the applicable vesting commencement date, and the balance of the underlying shares vesting monthly thereafter over 36 months, subject to the new employees’ continued service relationship with Mirum through the applicable vesting dates. The RSUs will vest over three years, with 33% of the underlying shares vesting on each anniversary of the applicable initial vesting date, subject to the new employees’ continued service relationship with Mirum through the applicable vesting dates. The awards are subject to the terms and conditions of Mirum’s 2020 Inducement Plan and the terms and conditions of an applicable award agreement covering the grant.
About Mirum Pharmaceuticals, Inc.
Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum combines deep rare disease expertise with strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM® (cholic acid) for bile-acid synthesis disorders, and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX).
Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV), and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS).
Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at www.mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and X.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260312816557/en/
Investor Contact:
Andrew McKibben
ir@mirumpharma.com
Media Contact:
Meredith Kiernan
media@mirumpharma.com
Original: Mirum Pharmaceuticals Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
US Market News
3月前
Mirum Pharmaceuticals to Participate in Upcoming Investor ConferencesFebruary 23, 2026 4:01 PM
Business Wire
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that it will participate in the following upcoming investor conferences:
TD Cowen’s 46th Annual Health Care Conference: Monday, March 2, 2026
Company fireside chat starting at 1:10 p.m. ET
The Citizens Life Sciences Conference: Tuesday, March 10, 2026
Company fireside chat starting at 10:10 a.m. ET
Barclays 28th Annual Global Healthcare Conference: Tuesday, March 10, 2026
2026 Leerink Partners Global Healthcare Conference: Wednesday, March 11, 2026
Company fireside chat starting at 10:00 a.m. ET
The fireside chats will be webcast live and can be accessed by visiting the Investors section of Mirum’s corporate website. The archived webcasts will be available for replay.
About Mirum Pharmaceuticals, Inc.
Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum combines deep rare disease expertise with strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM® (cholic acid) for bile-acid synthesis disorders, and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX).
Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV), and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS).
Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at www.mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and X.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260223143357/en/
Investor Contact:
Andrew McKibben
ir@mirumpharma.com
Media Contact:
Meredith Kiernan
media@mirumpharma.com
Original: Mirum Pharmaceuticals to Participate in Upcoming Investor Conferences
US Market News
4月前
Mirum Pharmaceuticals Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)February 10, 2026 10:45 PM
Business Wire
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that on February 10, 2026, the Compensation Committee of Mirum’s Board of Directors granted inducement awards consisting of non-qualified stock options to purchase 163,040 shares of common stock, 219,090 restricted stock units (“RSUs”), and 48,880 performance stock units (“PSUs”) to 37 new employees under Mirum’s 2020 Inducement Plan. The Compensation Committee of Mirum’s Board of Directors approved the awards as an inducement material to the new employees’ employment in accordance with Nasdaq Listing Rule 5635(c)(4).
Each stock option has an exercise price per share equal to $100.91 per share, Mirum’s closing trading price on February 10, 2026, and will vest over four years, with 25% of the underlying shares vesting on the one-year anniversary of the applicable vesting commencement date and the balance of the underlying shares vesting monthly thereafter over 36 months, subject to the new employees’ continued service relationship with Mirum through the applicable vesting dates. The RSUs will vest over three years, with 33% of the underlying shares vesting on each anniversary of the applicable initial vesting date, subject to the new employees’ continued service relationship with Mirum through the applicable vesting dates. The PSUs are subject to two performance conditions of achieving certain net product sales levels during the years ending December 31, 2027 and 2028 and, if the performance conditions are met, the applicable tranche of the awards will vest on March 15, 2028 and March 15, 2029, subject to continued service with Mirum through the applicable vesting dates. The number of PSUs to be vested in the first tranche of the PSU awards is calculated by multiplying 50% of the shares subject to the PSU award by a percentage calculated based on attained net sales metrics in the year ended December 31, 2027, as certified by Mirum’s Board of Directors or its Compensation Committee. The number of PSUs to be vested in the second tranche of the PSU awards is calculated by multiplying 50% of the shares subject to the PSU award by a percentage calculated based on attained net sales metrics in the year ended December 31, 2028, as certified by Mirum’s Board of Directors or its Compensation Committee. The awards are subject to the terms and conditions of Mirum’s 2020 Inducement Plan and the terms and conditions of an applicable award agreement covering the grant.
About Mirum Pharmaceuticals, Inc.
Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum combines deep rare disease expertise with strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM® (cholic acid) for bile-acid synthesis disorders, and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX).
Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV), and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS).
Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at www.mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and X.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260210382264/en/
Investor Contact:
Andrew McKibben
ir@mirumpharma.com
Media Contact:
Meredith Kiernan
media@mirumpharma.com
Original: Mirum Pharmaceuticals Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
US Market News
4月前
Mirum Pharmaceuticals Announces Health Canada Authorization of LIVMARLI® Tablet Formulation for the Treatment of Cholestatic Pruritus in Patients with Alagille SyndromeFebruary 5, 2026 8:51 AM
Business Wire
- Tablet authorization builds upon established use of LIVMARLI oral solution in Canada
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that Health Canada has authorized the tablet formulation of LIVMARLI® (maralixibat) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS).
LIVMARLI had previously been authorized in Canada as an oral solution for the treatment of cholestatic pruritus in patients with ALGS aged 12 months or older. The tablet formulation complements the existing 9.5 mg/ml oral solution, offering an additional dosing option for patients with ALGS weighing 22 kg or more who are able to swallow tablets.
“LIVMARLI’s tablet formulation provides an important new option for patients with ALGS,” said Jamie Twiselton, General Manager, Mirum Pharmaceuticals Canada. “With a liquid formulation for younger patients and a convenient one tablet per dose option available for older patients, LIVMARLI now provides greater dosing flexibility, supporting continuity of care as patients with ALGS age.”
ALGS is a rare genetic disorder that affects multiple organ systems, including the liver, where bile duct abnormalities can lead to cholestasis and pruritus. Chronic itching associated with ALGS can significantly disrupt sleep, growth and daily life for patients and their families.
“As children with Alagille syndrome grow, treatment needs can change,” said Dr. Or Steg Saban, Division of Gastroenterology, Hepatology and Nutrition, The Hospital for Sick Children, University of Toronto. “The availability of a tablet formulation of LIVMARLI in Canada provides an additional option for appropriate patients, which may support treatment adherence and continuity of care while continuing to address cholestatic pruritus.”
Earlier today, Mirum announced that Health Canada has authorized LIVMARLI® for the treatment of cholestatic pruritus in patients aged 12 months and older with progressive familial intrahepatic cholestasis (PFIC), including both the 19 mg/mL oral solution and tablet formulations to support flexible dosing. Link
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. In patients with ALGS, multiple organ systems may be affected by the mutation, including the liver, heart, kidneys and central nervous system. The accumulation of bile acids prevents the liver from working properly to eliminate waste from the bloodstream and, according to recent reports, 60% to 75% of patients with ALGS have a liver transplant before reaching adulthood. Signs and symptoms arising from liver damage in ALGS may include jaundice (yellowing of the skin), xanthomas (disfiguring cholesterol deposits under the skin) and pruritus (itch). The pruritus experienced by patients with ALGS is among the most severe in any chronic liver disease and is present in most affected children by the third year of life.
About LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets
LIVMARLI® (maralixibat) is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. LIVMARLI is authorized in Canada for the treatment of cholestatic pruritus in patients 12 months of age and older with ALGS.
LIVMARLI is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. In Canada, LIVMARLI is authorized for the treatment of cholestatic pruritus in patients aged 12 months or older with progressive familial intrahepatic cholestasis (PFIC).
LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website.
IMPORTANT SAFETY INFORMATION
Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein.
LIVMARLI can cause side effects, including:
Liver injury. Changes in certain liver tests are common in patients with ALGS and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams 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), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal. LIVMARLI is contraindicated in patients with prior or active hepatic decompensation events.
Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. 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 is common in patients with ALGS and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects.
Canadian Product Monograph
US Prescribing Information
EU SmPC
About Mirum Pharmaceuticals
Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum combines deep rare disease expertise with strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM® (cholic acid) for bile-acid synthesis disorders, and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX). Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV) and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS). Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at www.mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and X.
Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the potential benefits of a tablet formulation of LIVMARLI versus a liquid formulation, ability of patients to remain on therapy as they age, the availability of a tablet formulation for all patients taking LIVMARLI and the real life usage patterns of patients on LIVMARLI. 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,” “can,” “would,” “potential,” “hope,” “opportunity,” 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 macroeconomic and geopolitical developments, 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’s Quarterly Report on Form 10-K for the quarter ended December 31, 2024 and subsequent filings with the U.S. Securities and Exchange Commission and available at www.sec.gov.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260204062678/en/
Investor Contact:
Andrew McKibben
ir@mirumpharma.com
Media Contact:
Meredith Kiernan
Media@mirumpharma.com
Canadian Media Contact (English):
Olivia Simmonds
energi PR
Olivia.simmonds@energipr.com
724-524-8108
Original: Mirum Pharmaceuticals Announces Health Canada Authorization of LIVMARLI® Tablet Formulation for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome
US Market News
4月前
Mirum Pharmaceuticals annonce l’homologation par Santé Canada de LIVMARLI® sous forme de comprimés pour le traitement du prurit cholestatique chez les patients atteints du syndrome d’AlagilleFebruary 5, 2026 8:52 AM
Business Wire
LIVMARLI en comprimés vient s’ajouter à la solution orale déjà approuvée au Canada
Mirum Pharmaceuticals, Inc. (Nasdaq : MIRM), une entreprise à l’avant-garde dans le domaine des maladies rares, vient d’annoncer que Santé Canada a autorisé la mise en marché de LIVMARLI® (maralixibat) sous forme de comprimés pour le traitement du prurit cholestatique chez les patients atteints du syndrome d’Alagille.
LIVMARLI était déjà approuvé au Canada sous forme de solution orale pour le traitement du prurit cholestatique chez les patients âgés de 12 mois ou plus atteints du syndrome d’Alagille. La présentation sous forme de comprimés vient s’ajouter à la solution orale à 9,5 mg/mL déjà sur le marché, offrant une autre option aux patients atteints du syndrome d’Alagille qui pèsent au moins 22 kg et qui sont capables d’avaler des comprimés.
« La présentation de LIVMARLI en comprimés fournit aux patients atteints du syndrome d’Alagille une nouvelle option importante », de dire Jamie Twiselton, directeur général chez Mirum Pharmaceuticals Canada. « Grâce à la préparation liquide pour les patients très jeunes et à la posologie pratique où les enfants un peu plus vieux peuvent prendre un comprimé par dose, LIVMARLI offre une plus grande flexibilité posologique, favorisant la continuité des soins au fur et à mesure que l’enfant atteint du syndrome d’Alagille vieillit. »
Le syndrome d’Alagille est une maladie génétique rare qui touche divers systèmes organiques, notamment le foie, où des anomalies au niveau des voies biliaires peuvent entraîner une cholestase et un prurit. Les démangeaisons chroniques associées au syndrome d’Alagille peuvent perturber considérablement le sommeil et la croissance, ainsi que la vie quotidienne de la personne atteinte et de sa famille.
« Les besoins thérapeutiques peuvent changer au fur et à mesure que l’enfant atteint du syndrome grandit », déclare Dr Or Steg Saban, de la Division de gastro-entérologie, d’hépatologie et de nutrition, The Hospital for Sick Children, Université de Toronto. « L’accès à LIVMARLI en comprimés au Canada offre une option supplémentaire aux patients appropriés, ce qui pourrait favoriser l’observance thérapeutique et la continuité des soins tout en assurant la prestation soutenue d’une option qui vise le prurit cholestatique. »
Mirum a annoncé plus tôt aujourd’hui que Santé Canada vient d’approuver l’utilisation de LIVMARLI® pour le traitement du prurit cholestatique chez les patients âgés de 12 mois ou plus atteints d’une cholestase intrahépatique familiale progressive (CIFP). La solution orale à 19 mg/mL et les comprimés sont les deux formes posologiques approuvées pour cette indication, favorisant une flexibilité posologique. Link
À propos du syndrome d’Alagille
Le syndrome d’Alagille est une maladie génétique rare où les voies biliaires sont anormalement étroites, mal formées et clairsemées, entraînant une accumulation de bile dans le foie qui finit par causer une hépatopathie progressive. L’incidence estimative du syndrome d’Alagille est de un cas sur 30 000 personnes. Chez la personne atteinte du syndrome d’Alagille, la mutation génétique peut avoir un effet sur divers systèmes organiques, notamment sur le foie, le cœur, les reins et le système nerveux central. L’accumulation d’acides biliaires empêche le foie de fonctionner correctement et d’éliminer les déchets de la circulation sanguine. D’après des rapports publiés récemment, 60 à 75 % des patients atteints du syndrome d’Alagille subissent une greffe du foie avant l’âge adulte. Les signes et symptômes résultant des dommages au foie associés au syndrome d’Alagille comprennent un ictère (jaunissement de la peau), des xanthomes (dépôts de cholestérol défigurants sous la peau) et un prurit (démangeaisons). Le prurit dont sont affligés les patients atteints du syndrome d’Alagille figure parmi les plus sévères associés à toute maladie chronique du foie et il se présente avant l’âge de trois ans chez la majorité des enfants atteints.
À propos de LIVMARLI® (maralixibat) en solution orale et de LIVMARLI® (maralixibat) en comprimés
LIVMARLI® (maralixibat), un inhibiteur du transporteur iléal des acides biliaires (TIAB) qui s’administre par voie orale, a été approuvé par la Food and Drug Administration des États-Unis pour le traitement de deux hépatopathies cholestatiques pédiatriques. Le produit est approuvé aux États-Unis pour le traitement du prurit cholestatique chez les patients atteints du syndrome d’Alagille qui sont âgés de trois mois ou plus, et à partir de l’âge de deux mois en Europe. LIVMARLI est approuvé au Canada pour le traitement du prurit cholestatique chez les patients âgés de 12 mois ou plus atteints du syndrome d’Alagille.
LIVMARLI est également approuvé aux États-Unis pour le traitement du prurit cholestatique chez les patients âgés de 12 mois ou plus atteints d’une cholestase intrahépatique familiale progressive (CIFP), alors qu’en Europe, il est approuvé pour le traitement de la CIFP à partir de l’âge de trois mois. Au Canada, LIVMARLI est approuvé pour le traitement du prurit cholestatique chez les patients âgés de 12 mois ou plus atteints d’une cholestase intrahépatique familiale progressive (CIFP).
L’étude de phase III EXPAND actuellement en cours porte sur l’utilisation possible de LIVMARLI pour traiter le prurit cholestatique dans d’autres contextes. Pour en savoir davantage sur les essais cliniques de LIVMARLI en cours, veuillez visiter la section des essais cliniques du site web de Mirum.
RENSEIGNEMENTS IMPORTANTS SUR L’INNOCUITÉ
Limites d’utilisation : LIVMARLI n’est pas destiné aux patients atteints d’une CIFP de type 2 dont la protéine d’exportation des sels biliaires (BSEP) est sévèrement défectueuse.
LIVMARLI peut causer des effets secondaires, y compris les suivants :
Atteinte hépatique. Il arrive souvent que les patients atteints du syndrome d’Alagille ou d’une CIFP présentent des changements dans les résultats de certains tests hépatiques, mais ces changements peuvent s’aggraver pendant le traitement et pourraient signaler une lésion hépatique. En présence d’une CIFP, cette atteinte peut être grave, pouvant nécessiter une greffe du foie ou entraîner le décès du patient. Votre fournisseur de soins de santé devrait procéder à un examen physique et vous faire passer des tests sanguins avant et pendant le traitement, pour vérifier votre fonction hépatique. Signalez immédiatement à votre fournisseur de soins de santé la survenue de tout signe ou symptôme de problèmes hépatiques, tels que nausées ou vomissements, jaunissement de la peau ou du blanc des yeux, urines foncées ou brunes, douleur du côté droit de l’abdomen, ballonnement de l’abdomen, perte d’appétit ou saignement ou formation d’ecchymoses plus facilement provoqués qu’en temps normal. LIVMARLI est contre-indiqué chez les patients ayant déjà présenté ou présentant actuellement des événements associés à une décompensation hépatique.
Problèmes gastro-intestinaux. LIVMARLI peut causer des problèmes d’estomac et d’intestin, notamment la diarrhée et des douleurs gastriques. Votre fournisseur de soins de santé pourrait vous conseiller de surveiller l’apparition de nouveaux problèmes gastriques ou d’une aggravation de problèmes gastriques existants, y compris les suivants : douleurs gastriques, diarrhée, présence de sang dans les selles ou vomissements. Avisez votre fournisseur de soins de santé immédiatement si vous présentez l’un ou l’autre de ces symptômes plus souvent ou avec plus d’intensité que ce qui est normal dans votre cas.
Un état appelé carence en vitamines liposolubles, causé par des réserves peu élevées de certaines vitamines (vitamines A, D, E et K) dans le gras du corps, se présente fréquemment chez les patients atteints du syndrome d’Alagille ou d’une CIFP mais pourrait s’aggraver pendant le traitement. Votre fournisseur de soins de santé devrait vous faire passer des tests sanguins avant et pendant le traitement et pourrait surveiller chez vous la survenue de fractures et de saignements, qui ont été signalés comme étant des effets secondaires courants.
Monographie de produit canadienne
Renseignements thérapeutiques pour les États-Unis
Résumé des caractéristiques du produit pour l’Europe
À propos de Mirum Pharmaceuticals
Mirum Pharmaceuticals (NASDAQ : MIRM), une entreprise à l’avant-garde dans le domaine des maladies rares, possède des produits qui sont approuvés à l’échelle mondiale ainsi qu’un vaste portefeuille de médicaments encore à l’étude. Mise sur pied pour fournir des médicaments novateurs aux personnes présentant des états pathologiques méconnus, Mirum joint ses connaissances approfondies dans le domaine des maladies rares à ses liens étroits avec les communautés de patients. Le portefeuille de produits commercialisés de l’entreprise comprend LIVMARLI® (maralixibat) pour le syndrome d’Alagille et la cholestase intrahépatique familiale progressive (CIFP), CHOLBAM® (acide cholique) pour les déficits de synthèse des acides biliaires et CTEXLI® (chénodiol) pour la xanthomatose cérébrotendineuse (XCT). Les produits de Mirum faisant l’objet d’essais cliniques comprennent le volixibat, un inhibiteur du TIAB en phase avancée de développement pour le traitement de la cholangite sclérosante primitive (CSP) et de la cholangite biliaire primitive (CBP), le brelovitug, un anticorps monoclonal entièrement humain dont le développement est également avancé pour le traitement du virus delta de l’hépatite chronique (VDH) et MRM-3379, un inhibiteur de la PDE4D en cours d’évaluation pour traiter le syndrome de l’X fragile (SXF). Le succès de Mirum repose sur une équipe qui se consacre au développement stratégique de médicaments dont l’impact est élevé, à la réalisation disciplinée de ses objectifs et à la collaboration engagée avec les entités œuvrant dans le domaine des maladies rares. Pour en savoir davantage, visitez le site www.mirumpharma.com et suivez Mirum dans Facebook, LinkedIn, Instagram et X.
Énoncés prospectifs
Les déclarations contenues dans ce communiqué de presse concernant des sujets qui ne sont pas des faits historiques sont des « énoncés prospectifs » au sens de la Private Securities Litigation Reform Act de 1995. Ces énoncés prospectifs comprennent des énoncés concernant, entre autres, les avantages possibles de LIVMARLI sous forme de comprimés par rapport à la préparation liquide, la capacité des patients de poursuivre le traitement au fil des années, l’accès à la présentation en comprimés pour tous les patients prenant LIVMARLI et les tendances d’utilisation en situation réelle chez les patients sous LIVMARLI. Ces énoncés étant associés à des risques et incertitudes, les résultats réels pourraient différer matériellement de ceux exprimés ous sous-entendus dans ces énoncés. Des mots comme « sera », « pourrait », « peut », « serait », « possible », « espoir », « occasion » et autres expressions semblables ont pour but d’identifier les énoncés prospectifs. Ces énoncés prospectifs sont fondés sur les attentes actuelles de Mirum et comportent des hypothèses qui pourraient s’avérer incorrectes ou ne jamais se matérialiser. Les résultats réels pourraient différer matériellement de ceux anticipés dans ces énoncés prospectifs en raison de divers risques et incertitudes qui comprennent, sans s’y limiter, les risques et incertitudes associés aux activités de Mirum en général, l’impact de la situation macroéconomique et géopolitique, et les autres risques décrits dans les documents déposés par Mirum auprès de la Securities and Exchange Commission. Tous les énoncés prospectifs contenus dans ce communiqué de presse ne s’appliquent qu’à la date à laquelle ils ont été faits et sont basés sur les hypothèses et les estimations de la direction à cette date. Mirum décline toute obligation de mettre à jour ces énoncés en vue de refléter les événements se produisant ou les circonstances survenant après la date à laquelle ils ont été faits, sauf si la loi l’exige. Le rapport trimestriel de Mirum (Form 10-K) pour le trimestre se terminant le 31 décembre 2024 et les documents déposés par la suite auprès de la Securities and Exchange Commission des États-Unis sont accessibles à l’adresse www.sec.gov.
Consultez la version source sur businesswire.com : https://www.businesswire.com/news/home/20260204770874/fr/
Personne-ressource pour les investisseurs :
Andrew McKibben
ir@mirumpharma.com
Personne-ressource pour les médias :
Meredith Kiernan
Media@mirumpharma.com
Personne-ressource pour les médias canadiens (de langue française) :
Camille Turbide
energi PR
Camille.turbide@energipr.com
(514) 755-5354
Original: Mirum Pharmaceuticals annonce l’homologation par Santé Canada de LIVMARLI® sous forme de comprimés pour le traitement du prurit cholestatique chez les patients atteints du syndrome d’Alagille
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