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Yahoo
a day ago
- Health
- Yahoo
IBAT Inhibitors Market Witnesses Strong Growth During the Forecast Period (2025-2034) Owing to the Rising Demand for Rare Cholestatic Liver Disease Therapies
The market for IBAT inhibitors is projected to experience significant growth in the near future, fueled by the rising incidence of genetic disorders, a strong pipeline of clinical trials, and growing regulatory approvals. Leading companies such as GlaxoSmithKline, Mirum Pharmaceuticals, Ipsen Pharma, Albireo, Takeda, and others are actively developing IBAT inhibitor therapies targeting conditions like PBC, PSC, ALGS, PFIC, and related diseases. LAS VEGAS, Aug 11, 2025 /PRNewswire/ -- DelveInsight's IBAT Inhibitors Market Size, Target Population, Competitive Landscape & Market Forecast report includes a comprehensive understanding of current treatment practices, addressable patient population, which includes top indications such as primary sclerosing cholangitis (PSC), primary biliary cholangitis (PBC), metabolic dysfunction-associated steatohepatitis (MASH), chronic constipation, biliary atresia, Alagille syndrome (ALGS), and others. The selected indications are based on approved therapies and ongoing pipeline activity. The report also provides insights into the emerging IBAT inhibitors, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM. Key Takeaways from the IBAT Inhibitors Market Report As per DelveInsight's analysis, the total market size of IBAT inhibitors in the 7MM is expected to surge significantly by 2034. The report provides the total potential number of patients in the indications, such as primary sclerosing cholangitis (PSC), primary biliary cholangitis (PBC), metabolic dysfunction-associated steatohepatitis (MASH), chronic constipation, biliary atresia, Alagille syndrome (ALGS), and others. Leading IBAT inhibitor companies, such as GlaxoSmithKline, Mirum Pharmaceuticals, Ipsen, Albireo, and others, are developing novel IBAT inhibitors that can be available in the IBAT inhibitors market in the coming years. Some of the key IBAT inhibitors in the pipeline include Linerixibat, Volixibat, Ritivixibat, and others. In March 2025, Takeda announced that Japan's MHLW approved LIVMARLI Oral Solution 10 mg/mL for the treatment of pruritus associated with cholestasis in ALGS and PFIC. In November 2024, Ipsen announced data at the AASLD assessing the long-term efficacy and safety of patients treated with BYLVAY from two Phase III open-label extension studies in PFIC and ALGS. In October 2024, Mirum Pharmaceuticals announced that the FDA granted a breakthrough therapy designation (BTD) to volixibat as a possible treatment for cholestatic pruritus in patients with PBC. Volixibat also received FTD from the FDA in 2016 for the treatment of NASH in patients with liver fibrosis. Discover which indication is expected to grab the major IBAT inhibitors market share @ IBAT Inhibitors Market Report IBAT Inhibitors Market Dynamics The market dynamics for IBAT inhibitors are shaped by a confluence of factors, including rising prevalence of rare cholestatic liver diseases, growing awareness of pediatric liver disorders, and the increasing regulatory support for rare disease therapeutics. The approval of two key drugs, LIVMARLI and BYLVAY, by the US FDA and European regulatory authorities has not only validated the therapeutic mechanism but also catalyzed investment and R&D activity in this niche segment. The market is expected to witness significant growth due to the unmet medical need in pediatric hepatology and the relatively limited competition within this orphan drug space. Both LIVMARLI and BYLVAY have demonstrated clinically meaningful reductions in pruritus and improvements in liver function biomarkers, supporting their adoption. Furthermore, these drugs benefit from orphan drug designation, which grants market exclusivity, reduced regulatory fees, and extended patent life, enhancing the commercial attractiveness for developers. Their use is currently being explored in additional indications, including biliary atresia and intrahepatic cholestasis of pregnancy, which could further expand their market potential. Despite the momentum, there are key challenges that could moderate growth. One is the high cost of therapy, which may limit access in markets with less robust healthcare reimbursement systems. Additionally, the rarity of target diseases necessitates specialized diagnostic capabilities and referral networks, which are underdeveloped in many regions. Market penetration in emerging economies remains limited, creating a disparity in treatment availability. Moreover, competition is expected to intensify as more players enter the space with either me-too IBAT inhibitors or gene therapy-based solutions aiming at disease modification. IBAT Inhibitors Treatment Market As of now, two IBAT inhibitors, LIVMARLI and BYLVAY (also marketed as KAYFANDA), have received regulatory approvals. LIVMARLI, an oral IBAT inhibitor available in both liquid and tablet forms, has been approved by the U.S. FDA for use in pediatric patients with cholestatic liver diseases. In the U.S., it is indicated for treating cholestatic pruritus in patients with Alagille syndrome (ALGS) aged three months and older, and in Europe for those aged two months and older. Additionally, it is approved in the U.S. for patients aged 12 months and older with progressive familial intrahepatic cholestasis (PFIC), and in Europe for patients aged three months and above. The drug is currently being studied in the Phase III EXPAND trial to assess its potential in broader settings of cholestatic pruritus, with enrollment expected to conclude by 2026. LIVMARLI has received Breakthrough Therapy designation in the U.S. for ALGS and PFIC2, and Orphan Drug Designation (ODD) for both ALGS and PFIC in the U.S. and Europe. Japan's Ministry of Health, Labour and Welfare (MHLW) granted it ODD for anticipated indications in ALGS and PFIC in December 2022. In April 2025, Mirum Pharmaceuticals announced FDA approval of a new tablet formulation of LIVMARLI for use in ALGS and PFIC, with commercial availability expected in June 2025 via the Mirum Access Plus program. Additionally, in November 2024, the company presented new clinical data on LIVMARLI at the AASLD Liver Meeting. BYLVAY is a potent, once-daily IBAT inhibitor that works locally in the small intestine with limited systemic absorption. It is approved in the U.S. for treating pruritus in PFIC patients aged three months and older, where it benefits from orphan drug exclusivity. First launched in the U.S. in 2021 for PFIC, BYLVAY is supported by programs to facilitate patient access and support. In the EU, it is approved for PFIC in children aged six months and above and is available in over nine countries, with reimbursement in key markets such as Germany, France, Italy, the UK, and Belgium. In June 2023, the U.S. FDA approved BYLVAY for treating cholestatic pruritus in ALGS patients aged 12 months and older. Subsequently, in September 2024, the European Commission approved the drug, under the brand name KAYFANDA, under exceptional circumstances for treating cholestatic pruritus in ALGS in children aged six months and above. Learn more about the IBAT inhibitors @ IBAT Inhibitors Analysis Key Emerging IBAT Inhibitors and Companies Key IBAT inhibitors in the pipeline include Volixibat (Mirum Pharmaceuticals), Linerixibat (GlaxoSmithKline), Ritivixibat (Ipsen/Albireo), and others. Volixibat is an orally administered, minimally absorbed drug designed to specifically block the ileal bile acid transporter (IBAT). By inhibiting IBAT, it disrupts the enterohepatic circulation of bile acids, potentially lowering bile acid levels in the liver and bloodstream—offering a novel treatment strategy for adult cholestatic liver diseases. The drug is currently being tested in Phase IIb trials: the VISTAS study for primary sclerosing cholangitis (PSC) and the VANTAGE study for primary biliary cholangitis (PBC). According to Mirum Pharmaceuticals' 2024 annual report, enrollment in the VISTAS study for PSC is expected to conclude in the second half of 2025, with top-line results anticipated in 2026. Enrollment for the VANTAGE study in PBC is projected to be completed in 2026. In November 2024, Mirum presented data on volixibat at the AASLD meeting. Linerixibat, another IBAT inhibitor, is an orally administered therapy under development for managing cholestatic pruritus linked to PBC, a rare autoimmune liver disorder. By blocking the reabsorption of bile acids, linerixibat aims to tackle the underlying mechanism of itch in these patients. Both the U.S. FDA and the European Medicines Agency (EMA) have granted orphan drug designation (ODD) to linerixibat for this indication. In November 2024, GSK reported positive top-line results from GLISTEN, its global Phase III trial assessing linerixibat in adults with PBC-related cholestatic pruritus. According to GSK's Q4 update, regulatory submissions in the U.S., Europe, and China are expected in the first half of 2025, followed by a U.S. regulatory decision and a Japan submission in the second half of the year. Regulatory decisions in Europe, Japan, and China are projected for 2026. The anticipated launch of these emerging therapies are poised to transform the IBAT inhibitors market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the IBAT inhibitors market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. To know more about IBAT inhibitors clinical trials, visit @ IBAT Inhibitors Treatment IBAT Inhibitors Overview IBAT inhibitors are typically taken orally, with most designed to remain in the gut and exhibit minimal absorption into the bloodstream. As a result, plasma levels often remain below detectable limits after either single or repeated dosing within the approved range. Nonetheless, newer IBAT inhibitors that are systemically absorbed are currently under development. A key drawback of gut-restricted IBAT inhibitors is their reduced effectiveness in patients with complete or near-total biliary obstruction, where bile acids do not adequately reach the small intestine. This is especially a concern in children, as biliary atresia is the leading cause of cholestasis in this population. To address this, systemically active ASBT inhibitors have been created. ASBTs are also found in the proximal tubules of the kidneys, where they help reabsorb small amounts of bile acids filtered through the glomerulus. IBAT Inhibitors Epidemiology Segmentation The IBAT inhibitors market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into: Total Cases in Selected Indications for IBAT Inhibitors Total Eligible Patient Pool in Selected Indications for IBAT Inhibitors Total Treated Cases in Selected Indications for IBAT Inhibitors IBAT Inhibitors Report Metrics Details Study Period 2020–2034 IBAT Inhibitors Report Coverage 7MM [The United States, the EU-4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan] Key Indications Covered in the Report Primary sclerosing cholangitis (PSC), primary biliary cholangitis (PBC), metabolic dysfunction-associated steatohepatitis (MASH), chronic constipation, biliary atresia, Alagille syndrome (ALGS), and others Key IBAT Inhibitors Companies GlaxoSmithKline, Mirum Pharmaceuticals, Ipsen, Albireo, Takeda, Ipsen Pharma, and others Key IBAT Inhibitors Linerixibat, Volixibat, Ritivixibat, LIVMARLI, BYLVAY/KAYFANDA, and others Scope of the IBAT Inhibitors Market Report IBAT Inhibitors Therapeutic Assessment: IBAT Inhibitors' current marketed and emerging therapies IBAT Inhibitors Market Dynamics: Conjoint Analysis of Emerging IBAT Inhibitor Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, IBAT Inhibitors Market Access and Reimbursement Discover more about IBAT inhibitors in development @ IBAT Inhibitors Clinical Trials Table of Contents 1 Key Insights 2 Report Introduction 3 Executive Summary 4 Key Events 5 Epidemiology Market Forecast Methodology of IBAT Inhibitors 6 IBAT Inhibitors Market Overview at a Glance in the 7MM 6.1 Market Share (%) Distribution by Therapies in 2025 6.2 Market Share (%) Distribution by Therapies in 2034 6.3 Market Share (%) Distribution by Indications in 2025 6.4 Market Share (%) Distribution by Indications in 2034 7 IBAT Inhibitor: Background and Overview 7.1 Introduction 7.2 The potential of IBAT inhibitors in Different Indications 7.3 Clinical Applications of IBAT Inhibitors 8 Target Patient Pool 8.1 Key Findings 8.2 Assumptions and Rationale: 7MM 8.3 Epidemiology Scenario in the 7MM 8.3.1 Total Cases in Selected Indications for IBAT Inhibitor in the 7MM 8.3.2 Total Eligible Patient Pool in Selected Indications for IBAT Inhibitor in the 7MM 8.3.3 Total Treated Cases in Selected Indications for IBAT Inhibitor in the 7MM 8.4 The US 8.5 EU4 and the UK 8.6 Japan 9 Marketed Drugs 9.1 Key Competitors 9.2 LIVMARLI (maralixibat chloride): Mirum Pharmaceuticals 9.2.1 Product Description 9.2.2 Regulatory Milestones 9.2.3 Other Developmental Activities 9.2.4 Clinical Development 9.2.5 Safety and Efficacy 9.2.6 Analyst Views 10 Emerging Therapies 10.1 Key Cross Competition 10.2 Volixibat: Mirum Pharmaceuticals 10.2.1 Drug Description 10.2.2 Others Developmental Activities 10.2.3 Clinical Trials Information 10.2.4 Safety and Efficacy 10.2.5 Analyst's View 10.3 Linerixibat: GlaxoSmithKline List of drugs to be continued in the final report... 11 IBAT Inhibitor: the 7MM Analysis 11.1 Key Findings 11.2 Key Market Forecast Assumptions 11.2.1 Cost Assumptions and Rebates 11.2.2 Pricing Trends 11.2.3 Analogue Assessment 11.2.4 Launch Year and Therapy Uptakes 11.3 Market Outlook 11.4 Attribute Analysis 11.5 Total Market Size of IBAT Inhibitor in the 7MM 11.6 The US Market Size 11.6.1 Total Market Size of IBAT Inhibitor in the US 11.6.2 Market Size of IBAT Inhibitors by Indication in the United States 11.6.3 Market Size of IBAT Inhibitor by Therapies in the US 11.7 EU4 and the UK Market Size 11.8 Japan Market Size 11 Unmet Needs 12 SWOT Analysis 13 KOL Views 14 Market Access and Reimbursement 14.1 The US 14.2 EU4 and the UK 14.3 Japan 15 Acronyms and Abbreviations 16 Bibliography 17 Report Methodology Related Reports Metabolic Dysfunction-associated Steatohepatitis Market Metabolic Dysfunction-associated Steatohepatitis Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key MASH companies, including Inventiva Pharma, Novo Nordisk A/S, Cirius Therapeutics, Akero Therapeutics, 89bio, Boehringer Ingelheim, Zealand Pharma, Galectin Therapeutics, Lipocine, Viking Therapeutics, Eli Lilly and Company, Boston Pharmaceuticals, Pfizer, HighTide Biopharma, CytoDyn, Merck & Co., Hanmi Pharmaceutical, Hepagene (Shanghai), Hepion Pharmaceuticals, Enyo Pharmaceuticals, Gilead Sciences, Poxel SA, Zydus Therapeutics, Sagimet Biosciences, Ionis Pharmaceuticals, Corcept Therapeutics, among others. Primary Sclerosing Cholangitis Market Primary Sclerosing Cholangitis Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key PSC companies, including Gilead Sciences, Phenex Pharmaceuticals, HighTide Therapeutics, Dr. Falk Pharma, Mirum Pharmaceuticals, Pliant Therapeutics, Inc., NGM Biopharmaceuticals, CymaBay Therapeutics, Chemomab Therapeutics, among others. Primary Biliary Cholangitis Market Primary Biliary Cholangitis Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key PBC companies, including CymaBay Therapeutics, Zydus Therapeutics, Genfit, GlaxoSmithKline, Calliditas Therapeutics AB (Calliditas Therapeutics Suisse SA), Intercept Pharmaceuticals, Mirum Pharmaceuticals, Escient Pharmaceuticals, Gannex Pharma, Nanjing Chia-tai Tianqing Pharmaceutical, among others. Biliary Atresia Market Biliary Atresia Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key biliary atresia companies, including Mirium Pharmaceuticals, Albireo, Intercept Pharmaceuticals, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. Contact UsShruti Thakur info@ +14699457679 Logo: View original content: SOURCE DelveInsight Business Research, LLP Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
6 days ago
- Business
- Business Wire
Mirum Pharmaceuticals Reports Second Quarter 2025 Financial Results and Provides Business Update
FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for the second quarter 2025 and provided a business update. 'Our second quarter results once again underscore the strength of our commercial programs with notable outperformance from our International business and U.S. PFIC launch,' said Chris Peetz, chief executive officer of Mirum. 'The momentum we are seeing with Livmarli globally reinforces our belief that the medicine will reach and help more patients than we initially projected, allowing us to raise guidance with confidence.' Peetz added, 'We are carrying that same momentum into our pipeline, where we are on course for three important late stage clinical milestones in 2026. Notably, the VISTAS study of volixibat in PSC will complete enrollment this quarter, and we plan to report topline data in the second quarter of next year.' Commercial: Raising full year revenue guidance to $490 to $510 million Second quarter 2025 global net product sales of $127.8 million. Second quarter 2025 LIVMARLI net product sales were $88.2 million, representing 87% growth over second quarter 2024 net product sales. LIVMARLI single oral tablet dose U.S. launch in June. Bile Acid Medicines second quarter 2025 net product sales were $39.6 million, representing 30% growth over second quarter 2024 net product sales. Pipeline: Advancing toward multiple 2026 milestones Volixibat VISTAS study in primary sclerosing cholangitis (PSC) topline data expected in the second quarter of 2026. Volixibat VANTAGE study in primary biliary cholangitis (PBC) expected to complete enrollment in 2026. LIVMARLI EXPAND Phase 3 study for pruritus in rare cholestatic conditions expected to complete enrollment in 2026. Expect to initiate Phase 2 study for MRM-3379 in Fragile X Syndrome (FXS) in the fourth quarter of 2025. Corporate and Financial: Strong balance sheet and financial independence Total revenue for the quarter ended June 30, 2025, was $127.8 million compared to $77.9 million for the quarter ended June 30, 2024. Total operating expenses were $132.8 million for the quarter ended June 30, 2025, compared to $102.1 million for the quarter ended June 30, 2024. Total operating expenses for the quarter ended June 30, 2025, included $24.5 million of non-cash stock-based compensation, intangible amortization, and other non-cash expenses compared to $17.7 million for the quarter ended June 30, 2024. As of June 30, 2025, Mirum had unrestricted cash, cash equivalents, and investments of $321.7 million compared to $292.8 million as of December 31, 2024. Business Update Conference Call Mirum will host a conference call today, August 6 th at 1:30 p.m. PT/4:30 p.m. ET, to provide business updates. Join the call using the following details: Conference Call Details: U.S./Toll-Free: +1 833 470 1428 International: +1 404 975 4839 Access Code: 448461 You may also access the call via webcast by visiting the Events & Presentations section on Mirum's website. A replay of this webcast will be available for 30 days. About LIVMARLI® (maralixibat) oral solution 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. 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 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. EU SmPC Canadian Product Monograph About Volixibat Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study), and primary biliary cholangitis (PBC) (VANTAGE study). In 2024, Mirum announced positive interim results from the Phase 2b VANTAGE study showing statistically significant improvement in pruritus as well as meaningful reductions in serum bile acids and improvements in fatigue for patients treated with volixibat. No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate. Volixibat has been granted breakthrough therapy designation for the treatment of PBC. About CHOLBAM® (cholic acid) capsules The FDA approved CHOLBAM (cholic acid) capsules in March 2015, the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for adjunctive treatment of patients with peroxisome biogenesis disorder-Zellweger spectrum disorder. The effectiveness of CHOLBAM has been demonstrated in clinical trials for bile acid synthesis disorders and the adjunctive treatment of peroxisomal disorders. An estimated 200 to 300 patients are current candidates for therapy. CHOLBAM® (cholic acid) Indication CHOLBAM is a bile acid indicated for Treatment of bile acid synthesis disorders due to single enzyme defects. Adjunctive treatment of peroxisomal disorders, including Zellweger spectrum disorders, in patients who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption. LIMITATIONS OF USE The safety and effectiveness of CHOLBAM on extrahepatic manifestations of bile acid synthesis disorders due to single enzyme defects or peroxisomal disorders, including Zellweger spectrum disorders, have not been established. IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS – Exacerbation of liver impairment Monitor liver function and discontinue CHOLBAM in patients who develop worsening of liver function while on treatment. Concurrent elevations of serum gamma glutamyltransferase (GGT) and alanine aminotransferase (ALT) may indicate CHOLBAM overdose. Discontinue treatment with CHOLBAM at any time if there are clinical or laboratory indicators of worsening liver function or cholestasis. ADVERSE REACTIONS The most common adverse reactions (≥1%) are diarrhea, reflux esophagitis, malaise, jaundice, skin lesion, nausea, abdominal pain, intestinal polyp, urinary tract infection, and peripheral neuropathy. Please see full Prescribing Information for additional Important Safety Information. About CTEXLI™ (chenodiol) tablets CTEXLI™ (chenodiol) tablets is FDA-approved for the treatment of adults with cerebrotendinous xanthomatosis (CTX). Chenodiol is another name for chenodeoxycholic acid (CDCA). CDCA is a naturally occurring bile acid that was originally approved for the treatment of people with radiolucent stones in the gallbladder. CTEXLI was evaluated as part of the Phase 3 RESTORE study, the first and only clinical trial for CTX. CTX is a rare progressive disease that can affect the brain, spinal cord, tendons, eyes and arteries. IMPORTANT SAFETY INFORMATION CTEXLI can cause side effects, including: Liver Injury: You will need to undergo laboratory testing before starting and while taking CTEXLI to check your liver function. Changes in certain liver tests may occur during treatment and may be a sign of liver injury. This can be serious. Stop taking CTEXLI immediately and tell your healthcare provider right away if you get any signs or symptoms of liver problems, including, stomach (abdomen) pain, bruising, dark-colored urine, feeling tired (fatigue), bleeding, yellowing of the skin and eyes, nausea, and itching. Most Common Side Effects: Diarrhea, headache, stomach pain, constipation, high blood pressure, muscular weakness, and upper respiratory tract infection. Tell your healthcare provider about all the medications that you take, as CTEXLI may interact with other medicines. US Prescribing Information About Mirum Pharmaceuticals, Inc. Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution/LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum has initiated the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (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, continued positive commercial results for our approved medicines, including continued financial growth, the potential achievement of our yearly financial guidance, continued strong execution across our approved medicines and pipeline, the initiation, results, enrollment, conduct and progress of our ongoing and planned studies for our product candidates, the timing and results of interim analyses of, and topline data for, our ongoing studies and the regulatory approval path for our product candidates in any indication or any specific territory. 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 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 Balance Sheet Data (in thousands) (Unaudited) December 31, 2024 Assets Current assets: Cash and cash equivalents $ 228,122 $ 222,503 Short-term investments 76,430 57,812 Accounts receivable 106,836 78,286 Inventory 22,941 22,403 Prepaid expenses and other current assets 19,069 11,784 Total current assets 453,398 392,788 Restricted cash 480 425 Long-term investments 17,112 12,526 Intangible assets, net 237,870 249,819 Other noncurrent assets 16,965 15,196 Total assets $ 725,825 $ 670,754 Liabilities and Stockholders' Equity Current liabilities: Accounts payable $ 17,657 $ 14,618 Accrued expenses and other current liabilities 127,139 111,933 Total current liabilities 144,796 126,551 Operating lease liabilities, noncurrent 7,884 7,972 Convertible notes payable, net, noncurrent 308,933 308,082 Other liabilities 9,054 2,509 Total liabilities 470,667 445,114 Commitments and contingencies Stockholders' equity: Preferred stock — — Common stock 5 5 Additional paid-in capital 919,520 870,189 Accumulated deficit (664,719 ) (644,181 ) Accumulated other comprehensive income (loss) 352 (373 ) Total stockholders' equity 255,158 225,640 Total liabilities and stockholders' equity $ 725,825 $ 670,754 Expand
Globe and Mail
16-07-2025
- Business
- Globe and Mail
Liver Cirrhosis Clinical Trials 2025: EMA, PDMA, FDA Approvals, Medication, Therapies, Treatment Market, Mechanism of Action, Route of Administration, and Companies by DelveInsight
"Liver Cirrhosis Clinical Trials" Liver cirrhosis companies such as Versantis AG, Mirum Pharmaceuticals, Sagimet Biosciences, TenNor Therapeutics, Prism Pharma, Vedanta Biosciences, Lipocine, Gwo Xi Stem Cell Applied Technology, Galectin Therapeutics, Resolution Therapeutics, Ipsen, AstraZeneca, and others. (Albany, USA) DelveInsight's 'Liver Cirrhosis Pipeline Insight 2025' report provides comprehensive global coverage of pipeline liver cirrhosis therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the liver cirrhosis pipeline domain. The liver cirrhosis treatment market is poised for significant growth, driven by the rising global prevalence of liver diseases, particularly NAFLD, fueled by lifestyle factors like obesity, alcohol consumption, and metabolic syndrome. Technological advancements in antiviral therapies and regenerative medicine are enhancing treatment outcomes, while an aging population further increases demand. These factors collectively highlight a strong growth trajectory for the market. Key Takeaways from the Liver Cirrhosis Pipeline Report DelveInsight's liver cirrhosis pipeline report depicts a robust space with 30+ active players working to develop 30+ pipeline liver cirrhosis drugs. Key liver cirrhosis companies such as Versantis AG, Mirum Pharmaceuticals, Sagimet Biosciences, TenNor Therapeutics, Prism Pharma, Vedanta Biosciences, Lipocine, Gwo Xi Stem Cell Applied Technology, Galectin Therapeutics, Resolution Therapeutics, Ipsen, AstraZeneca, and others are evaluating new liver cirrhosis drugs to improve the treatment landscape. Promising pipeline liver cirrhosis therapies such as Volixibat, VS-01, TVB-2640, TNP-2092, PRI-724, VE303, LPCN 1148, GXHPC1, Belapectin, RTX001, Elafibranor, Zibotentan, and others are under different phases of liver cirrhosis clinical trials. In February 2025, Sagimet announced lipidomic data from the Phase IIb FASCINATE-2 trial, focusing on triglycerides and LDL cholesterol in advanced fibrosis, which will be presented at the MASH Pathogenesis and Therapeutic Approaches Keystone Symposium. The US FDA granted denifanstat Breakthrough Therapy (BTD) and Fast Track Designation (FTD) for non-cirrhotic MASH with moderate to advanced fibrosis. Madrigal Pharmaceuticals aims to roll out REZDIFFRA across Europe starting with Germany in the second half of 2025, pending EMA approval for REZDIFFRA, making it the first authorized therapy for MASH-related liver fibrosis in the region. Furthermore, updated two-year data from the MAESTRO-NAFLD-1 trial, released in February 2025, indicate possible benefits for patients with compensated MASH cirrhosis, suggesting an expanded scope of clinical effectiveness. In January 2025, Akero Therapeutics announced the completion of patient enrollment in the Phase III SYNCHRONY Real-World study for MASH or MASLD (F1-F4), with results expected in the first half of 2026. In January 2025, the company emphasized its strong position for the year, with ongoing Phase III trials in MASH. It expects to release topline data from its first Phase III trial in late 2025. The company is also conducting two Phase III trials—ENLIGHTEN-Fibrosis for non-cirrhotic MASH (F2-F3) patients and ENLIGHTEN-Cirrhosis for compensated cirrhotic MASH (F4) patients—both of which are actively enrolling global patients. In December 2024, Galectin Therapeutics announced results from its global clinical trial NAVIGATE evaluating belapectin in patients with Metabolic Dysfunction-Associated Steatohepatitis (MASH) cirrhosis and portal hypertension. In October 2024, PharmaIN Corporation announced the company will present interim results from its ongoing Phase I clinical trial of PHIN-214, the company's lead candidate, for the prevention and treatment of decompensated cirrhosis. In June 2024, Resolution Therapeutics Limited announced key data presentations of RTX001 with the University of Edinburgh at the EASL Congress 2024, held in Milan, Italy which demonstrate the significant potential of macrophage cell therapy as a treatment for advanced liver cirrhosis. In June 2024, Lipocine announced that Phase II results on LPCN 1148 in cirrhosis were featured in a late breaking oral presentation at the European Association for the Study of Liver (EASL) Congress in Milan, Italy. In April 2024, LyGenesis announced that the first patient had been dosed in their Phase IIa clinical trial evaluating their first-in-class allogenic regenerative cell therapy transplanted into patients' lymph nodes as a potential treatment for end-stage liver disease (ESLD). In March 2024, Lipocine announced positive topline results from a Phase II clinical study of LPCN 1148. LPCN 1148 is an oral candidate under development for the clinical management of liver cirrhosis. Liver Cirrhosis Overview Liver cirrhosis is a condition where the liver becomes scarred and permanently damaged, with healthy liver tissue replaced by scar tissue, impairing its normal function. It often results from long-term liver damage caused by conditions like alcohol-related liver disease, nonalcoholic fatty liver disease, chronic hepatitis C, and chronic hepatitis B. Symptoms may not be noticeable until significant liver damage occurs and can include fatigue, severe itching, and swelling in the legs and abdomen. Doctors diagnose cirrhosis through a combination of medical history, physical examination, and tests such as blood work, imaging, and liver biopsy. While there is no definitive cure, addressing the underlying causes can slow disease progression and reduce the risk of liver failure. Complications may include portal hypertension, infections, and liver cancer. Managing cirrhosis involves eating a healthy diet, avoiding alcohol and liver-damaging foods like raw shellfish, and, in severe cases, considering a liver transplant. The symptoms of liver cirrhosis can vary based on its severity. Early signs include fatigue, poor appetite, weight loss, nausea, abdominal pain, and spider-like red blood vessels on the skin. As the condition worsens, symptoms may include fluid buildup in the legs and abdomen, yellowing of the skin and eyes (jaundice), redness on the palms, easy bruising, abnormal bleeding, confusion or difficulty thinking, pale stools, and gastrointestinal bleeding. Diagnosing liver cirrhosis involves blood tests, imaging studies, and, sometimes, a liver biopsy. Blood tests can identify elevated liver enzymes, abnormal liver function, or signs of inflammation or infection. Imaging techniques such as ultrasound, CT scans, or MRIs can reveal liver abnormalities, while a biopsy can confirm the diagnosis and evaluate the extent of damage. Treatment focuses on slowing scar tissue formation, managing symptoms, and preventing complications. Lifestyle changes may include a healthy diet, stopping alcohol consumption, weight loss for obese individuals, regular exercise, and maintaining good hygiene to lower infection risks. Medications may include antivirals for hepatitis, diuretics to reduce fluid retention, beta-blockers to prevent bleeding from enlarged veins, and creams to relieve itching. Liver Cirrhosis Therapeutics Assessment The liver cirrhosis pipeline report proffers an integral view of the emerging liver cirrhosis therapies segmented by stage, product type, molecule type, route of administration, and mechanism of action. Elafibranor: Ipsen Volixibat: Mirum Pharmaceuticals GXHPC1: Gwo Xi Stem Cell Applied Technology RTX-001: Resolution Therapeutics PHIN-214: PharmaIN Scope of the Liver Cirrhosis Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Intra-articular, Intraocular, Intrathecal, Intravenous, Oral, Parenteral, Subcutaneous, Topical, Transdermal Therapeutics Assessment By Molecule Type: Oligonucleotide, Peptide, Small molecule Therapeutics Assessment By Mechanism of Action: Sodium-bile acid cotransporter inhibitors, Regulatory T-lymphocyte stimulants, Ammonia scavengers, DNA gyrase inhibitors, DNA topoisomerase inhibitors, DNA-directed RNA polymerase inhibitors, Beta-catenin inhibitors, CREB-binding protein inhibitors, Wnt signalling pathway inhibitors, Bacteria replacements, Microbiome modulators Key Liver Cirrhosis Companies: Versantis AG, Mirum Pharmaceuticals, Sagimet Biosciences, TenNor Therapeutics, Prism Pharma, Vedanta Biosciences, Intercept Pharmaceuticals, Boehringer Ingelheim, Ohara Pharmaceutical, Ocelot Bio, Calliditas Therapeutics, Galecto Biotech, Pharmicell, and others. Key Liver Cirrhosis Pipeline Therapies: Volixibat, VS-01, TVB-2640, TNP-2092, PRI-724, VE303, Obeticholic Acid (OCA), BI 685509, OP-724, OCE-205, GKT137831, GB1211, Cellgram-LC, and others. Dive deep into rich insights for new liver cirrhosis treatments, visit @ Liver Cirrhosis Drugs and Therapies Table of Contents 1. Liver Cirrhosis Pipeline Report Introduction 2. Liver Cirrhosis Pipeline Report Executive Summary 3. Liver Cirrhosis Pipeline: Overview 4. Analytical Perspective In-depth Commercial Assessment 5. Liver Cirrhosis Clinical Trial Therapeutics 6. Liver Cirrhosis Pipeline: Late-Stage Products (Pre-registration) 7. Liver Cirrhosis Pipeline: Late-Stage Products (Phase III) 8. Liver Cirrhosis Pipeline: Mid-Stage Products (Phase II) 9. Liver Cirrhosis Pipeline: Early-Stage Products (Phase I) 10. Liver Cirrhosis Pipeline Therapeutics Assessment 11. Inactive Products in the Liver Cirrhosis Pipeline 12. Company-University Collaborations (Licensing/Partnering) Analysis 13. Key Companies 14. Key Products in the Liver Cirrhosis Pipeline 15. Unmet Needs 16. Market Drivers and Barriers 17. Future Perspectives and Conclusion 18. Analyst Views 19. Appendix About DelveInsight DelveInsight is a leading Business Consultant, and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Media Contact Company Name: DelveInsight Business Research LLP Contact Person: Ankit Nigam Email: Send Email Phone: +14699457679 Address: 304 S. Jones Blvd #2432 City: Albany State: New York Country: United States Website:
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11-06-2025
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Mirum Pharmaceuticals Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
FOSTER CITY, Calif., June 11, 2025--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that on June 10, 2025, the Compensation Committee of Mirum's Board of Directors granted inducement awards consisting of non-qualified stock options to purchase 115,620 shares of common stock and 57,740 restricted stock units ("RSUs") to 10 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 $48.98 per share, Mirum's closing trading price on June 10, 2025, 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 vesting commencement 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, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat), CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum is also initiating the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis (PBC). Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit and follow Mirum on Facebook, LinkedIn, Instagram and X. View source version on Contacts Investor Contact: Andrew McKibbenir@ Media Contact: Erin Murphymedia@ Sign in to access your portfolio
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11-06-2025
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Mirum Pharmaceuticals Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
FOSTER CITY, Calif., June 11, 2025--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that on June 10, 2025, the Compensation Committee of Mirum's Board of Directors granted inducement awards consisting of non-qualified stock options to purchase 115,620 shares of common stock and 57,740 restricted stock units ("RSUs") to 10 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 $48.98 per share, Mirum's closing trading price on June 10, 2025, 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 vesting commencement 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, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat), CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum is also initiating the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis (PBC). Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit and follow Mirum on Facebook, LinkedIn, Instagram and X. View source version on Contacts Investor Contact: Andrew McKibbenir@ Media Contact: Erin Murphymedia@
