Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), today announced the decision to discontinue the OHANA study of volixibat in intrahepatic cholestasis of pregnancy (ICP) due to enrollment feasibility. ICP is a setting of cholestasis during pregnancy with pruritus and elevated bile acid levels that can lead to fetal complications. In an interim analysis, patients treated with volixibat saw meaningful reductions in serum bile acids and pruritus. However, due to the challenges in enrollment in this high-risk pregnancy setting, Mirum has decided to discontinue OHANA, which allows the company to prioritize other programs.
“We are disappointed that the ICP program will not continue due to enrollment feasibility, but we are grateful to the patients and the study personnel who were involved in OHANA. Additionally, we are excited about the clear response seen in reduction of serum bile acids and pruritus in the OHANA study, which builds confidence in volixibat for our PSC and PBC programs,” said Chris Peetz, president and chief executive officer at Mirum. “We are approaching the end of a very successful year for LIVMARLI in Alagille syndrome and look forward to the potential approval in Europe before the end of the year, as well as a number of important program milestones in 2023.”
Mirum is continuing to advance its broad pipeline of indications where LIVMARLI and volixibat have the potential for life-changing impact for patients. Key upcoming milestones include:
- LIVMARLI anticipated approval in Europe for cholestatic pruritus in patients with Alagille syndrome two months of age and older, by year end 2022.
- LIVMARLI sNDA application for pruritus in patients with progressive familial intrahepatic cholestasis (PFIC), expected in first quarter 2023.
- Volixibat interim analysis in PSC, expected mid-year 2023.
- Volixibat interim analysis in PBC, expected second half 2023.
- LIVMARLI Phase 2b data in biliary atresia expected second half 2023.
Mirum reiterates revenue expectation of $70 million net product revenue, $72 million total revenue for 2022, and current balance sheet supporting growth through cash flow positive operations.
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. Phase 1 and Phase 2 studies of volixibat demonstrated on-target fecal bile acid excretion, a pharmacodynamic marker of IBAT inhibition, in addition to decreases in LDL cholesterol and increases in 7αC4 which are markers of bile acid synthesis. Volixibat has been evaluated in more than 400 individuals across multiple clinical trials. The most common adverse events reported were mild to moderate gastrointestinal events observed in the volixibat groups.
Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (VISTAS clinical trial) and primary biliary cholangitis (VANTAGE clinical trial).
About Mirum Pharmaceuticals
Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases. Mirum’s approved medication is LIVMARLI® (maralixibat) oral solution which is approved in the U.S. for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older. In Europe, the European Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for LIVMARLI for the treatment of cholestatic pruritus in patients with Alagille syndrome two months of age and older. A decision by the European Commission is expected by year-end 2022.
Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver diseases affecting children and adults. LIVMARLI, an oral ileal bile acid transporter (IBAT) inhibitor, is currently being evaluated in clinical trials for pediatric liver diseases and includes the MARCH Phase 3 clinical trial for progressive familial intrahepatic cholestasis (PFIC) and the EMBARK Phase 2b clinical trial for patients with biliary atresia. In addition, Mirum has an expanded access program open across multiple countries for eligible patients with ALGS and PFIC.
Mirum’s second investigational treatment, volixibat, an oral IBAT inhibitor, is being evaluated in two potentially registrational studies including the VISTAS Phase 2b clinical trial for adults with primary sclerosing cholangitis and the VANTAGE Phase 2b clinical trial for adults with primary biliary cholangitis.
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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, Mirum’s potential value generating milestones, Mirum’s revenue expectations, and the regulatory approval pathway for volixibat. 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 “look,” “ahead,” “will,” “may,” “expect” 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 filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.
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Ian Clements, Ph.D.