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Globe and Mail
7 days ago
- 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:
Yahoo
11-06-2025
- Business
- Yahoo
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
Yahoo
11-06-2025
- Business
- Yahoo
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@
Yahoo
06-06-2025
- Business
- Yahoo
Why Is Mirum Pharmaceuticals (MIRM) Down 1.3% Since Last Earnings Report?
It has been about a month since the last earnings report for Mirum Pharmaceuticals, Inc. (MIRM). Shares have lost about 1.3% in that time frame, underperforming the S&P 500. Will the recent negative trend continue leading up to its next earnings release, or is Mirum Pharmaceuticals due for a breakout? Before we dive into how investors and analysts have reacted as of late, let's take a quick look at the most recent earnings report in order to get a better handle on the important catalysts. It turns out, estimates revision have trended upward during the past month. The consensus estimate has shifted 12.93% due to these changes. At this time, Mirum Pharmaceuticals has a great Growth Score of A, a grade with the same score on the momentum front. However, the stock was allocated a grade of D on the value side, putting it in the bottom 40% for this investment strategy. Overall, the stock has an aggregate VGM Score of A. If you aren't focused on one strategy, this score is the one you should be interested in. Estimates have been trending upward for the stock, and the magnitude of these revisions looks promising. Notably, Mirum Pharmaceuticals has a Zacks Rank #3 (Hold). We expect an in-line return from the stock in the next few months. Mirum Pharmaceuticals is part of the Zacks Medical - Biomedical and Genetics industry. Over the past month, Moderna (MRNA), a stock from the same industry, has gained 7.1%. The company reported its results for the quarter ended March 2025 more than a month ago. Moderna reported revenues of $108 million in the last reported quarter, representing a year-over-year change of -35.3%. EPS of -$2.52 for the same period compares with -$3.07 a year ago. Moderna is expected to post a loss of $2.97 per share for the current quarter, representing a year-over-year change of +10.8%. Over the last 30 days, the Zacks Consensus Estimate has changed -3.9%. Moderna has a Zacks Rank #3 (Hold) based on the overall direction and magnitude of estimate revisions. Additionally, the stock has a VGM Score of D. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Mirum Pharmaceuticals, Inc. (MIRM) : Free Stock Analysis Report Moderna, Inc. (MRNA) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research 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
19-05-2025
- Business
- Business Wire
Mirum Pharmaceuticals Appoints Doug Sheehy, JD, as Chief Legal Officer
FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced the appointment of Doug Sheehy, JD, as chief legal officer. Mr. Sheehy brings nearly two decades of experience leading global legal and compliance operations for biopharmaceutical companies. Mr. Sheehy was most recently chief legal officer and secretary at Sonoma Biotherapeutics, Inc., which is developing regulatory T cell therapies for autoimmune and inflammatory diseases. Prior to Sonoma, from 2016 to 2020, he served as general counsel and secretary for Aimmune Therapeutics, Inc., a biopharmaceutical company that specialized in the development and commercialization of treatments for life-threatening food allergies. Mr. Sheehy served at Aimmune until its acquisition by Nestle Health Science in 2020. Before Aimmune, from 2007-2016, he was executive vice president, chief administrative officer, general counsel, and secretary of Codexis, Inc., a company focused on the development of synthetic enzymes for the pharmaceutical and food industries. Earlier in his career, he served in several roles within the legal department at CV Therapeutics. Mr. Sheehy began his career as a corporate and securities lawyer in Silicon Valley representing emerging growth companies and venture funds. Mr. Sheehy received an A.B. in History from Dartmouth College and a J.D. from American University's Washington College of Law. 'Doug brings extensive global legal experience that will strengthen Mirum's capabilities as we continue to grow our commercial business and advance our rare disease pipeline,' said Chris Peetz, chief executive officer at Mirum. 'Doug will be a great addition to Mirum's leadership team, and I look forward to leveraging his experience in guiding transformative growth for the company.' 'Mirum is an impressive company that has established its place as a leader in rare disease, and I am excited to have the opportunity to join the leadership team and contribute to the exciting work underway,' said Doug Sheehy. 'The company has a focused growth strategy and is poised for continued success as it works to make a meaningful difference in the lives of people impacted by rare disease.' 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).
