Cholic Acid

- Topline data announcement expected second quarter of 2026 FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the completion of enrollment in the Phase 2b VISTAS study of volixibat, an investigational oral IBAT inhibitor, for the treatment of cholestatic pruritus in patients with primary sclerosing cholangitis (PSC). The trial previously met its pre-specified efficacy and safety thresholds at the blinded interim analysis for dose selection conducted in 2024 and topline results are expected to be announced in the second quarter of 2026. “Completing enrollment in VISTAS is a major milestone for the PSC community and for Mirum,” said Joanne Quan, M.D., Chief Medical Officer. “This progress reflects the dedication of patients, investigators, and our team working together toward the shared goal of addressing the daily challenges of PSC, including persistent itch and fatigue. We believe the data generated from VISTAS will be an important step toward bringing meaningful medicines to patients who currently have no approved therapies.” Ricky Safer, CEO and Founder of PSC Partners Seeking a Cure, a patient-led advocacy organization dedicated to supporting those affected by PSC, added “PSC Partners is thrilled to see Mirum reach this important milestone with the VISTAS study. Completing enrollment marks a significant step forward in advancing a possible treatment for itch in PSC, bringing tremendous hope to our community. We are grateful to Mirum for their commitment and to all the patients and families who made this kind of progress possible. Together, we are moving closer to new possibilities and a brighter future for those impacted by PSC.” Volixibat is also being evaluated in the Phase 2b VANTAGE study for primary biliary cholangitis (PBC), which is expected to complete enrollment in 2026 and announce topline results in the first half of 2027. About the VISTAS Phase 2b Study The VISTAS study (NCT04663308) is a global, randomized, double-blind, placebo-controlled Phase 2b trial evaluating the efficacy and safety of volixibat in patients with primary sclerosing cholangitis and cholestatic pruritus over a 28-week period. The primary endpoint is a reduction in pruritus as measured by the Adult ItchRO, a pruritus numerical rating scale ranging from 0 (no itch) to 10 (worst possible itch). Secondary endpoints include assessments of fatigue, serum bile acids, and safety. About Primary Sclerosing Cholangitis (PSC) Primary sclerosing cholangitis (PSC) is a rare, chronic, progressive liver disease in which the bile ducts inside and outside the liver become inflamed, scarred, and narrowed over time. This bile duct damage leads to bile accumulation, liver injury, and eventually liver failure. Patients with PSC experience a heavy symptom burden, with pruritus (severe itching), fatigue, and abdominal pain that may be debilitating and life-altering. Beyond symptom burden, PSC is associated with increased risk of bile duct cancer and need for liver transplantation. There are currently no approved therapies for PSC, leaving patients with limited treatment options focused on symptom management and supportive care. 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 to 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. The Phase 2 VISTAS study met its pre-specified efficacy and safety thresholds at the blinded interim analysis for dose selection conducted in 2024. Volixibat has been granted breakthrough therapy designation for the treatment of cholestatic pruritus in patients with PBC. 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. for 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 conducting 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 2b 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 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 results and progress of our ongoing and planned studies for our product candidates, the timing and results of 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 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. Investor Contact: Andrew McKibben ir@mirumpharma.com Media Contact: Bryan Blatstein Spectrum Science Communications BBlatstein@spectrumscience.com

PARIS, Aug. 14, 2025 /PRNewswire/ -- Norgine today announced the successful completion of the acquisition of Theravia, an international pharmaceutical company specialising in therapies for patients with rare and debilitating conditions. The closure of this acquisition marks a major step forward for Norgine to deliver on its ambition to bring life-changing medicines to patients with high unmet medical needs. With the transaction now complete, Norgine will begin a structured integration process to align operations, systems, and teams. "We are pleased about the successful closing of this acquisition as the addition of Theravia to our company further enhances our expertise in, and growing portfolio of rare disease medicines," said Janneke van der Kamp, Chief Executive Officer, Norgine. "This acquisition marks a critical step forward in our growth strategy, and with our strong focus on expanding the reach of products to patients in areas of high unmet medical need in Europe and ANZ, we are well positioned to ensure Theravia's medicines will reach more patients." As a result of the acquisition, Theravia has become a wholly owned subsidiary of Norgine. Notes About Norgine Norgine is a uniquely positioned, specialty pharmaceutical and consumer healthcare company, with over €550 million of annual revenues and a 120-year track record of bringing life-changing products to patients and consumers across our core markets of Western Europe, Australia and New Zealand. Norgine's integrated approach – strong commercial capabilities, as well as deep medical, regulatory and clinical expertise, in-house manufacturing, robust supply networks, and best-in-class enabling functions – ensures that Norgine can deliver high-quality, transformative medicines quickly and effectively to over 25 million patients annually. Norgine is a nimble, innovative, and high-performing company that has been transformed by a relentless focus on operational excellence. This focus will enable us to secure the legacy of more than a century of innovation and doing the right thing by our patients, as we push the boundaries and take strides into therapeutic innovation. NORGINE and the sail logo are trademarks of the Norgine group of companies. Logo: Media contact: Annabel Cowper [email protected] WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED