Mirum Pharmaceuticals Stock Price, News & Analysis

The European Commission has granted marketing authorization for Mirum Pharmaceuticals' LIVMARLI (maralixibat) oral solution to treat progressive familial intrahepatic cholestasis (PFIC) in patients aged three months and older. This approval follows the positive opinion from the CHMP, noting LIVMARLI's significant clinical benefits over existing PFIC treatments. The decision also aligns with a positive COMP opinion recommending the continuation of Orphan Drug Designation for LIVMARLI in PFIC. Approval is based on data from the Phase 3 MARCH study, the largest randomized trial in PFIC, showing substantial reductions in pruritus and serum bile acids with LIVMARLI compared to placebo. The study included 93 patients with various genetic types of PFIC. Common adverse events were mild and transient diarrhea. LIVMARLI is also approved in the U.S. for treating cholestatic pruritus in PFIC patients aged five years and older.

Mirum Pharmaceuticals has submitted a New Drug Application (NDA) to the FDA for chenodiol, aiming to treat cerebrotendinous xanthomatosis (CTX), a rare genetic disorder of cholesterol metabolism. The submission is based on positive results from the Phase 3 RESTORE study, which showed a significant reduction in bile alcohols and improved serum cholestanol in patients treated with chenodiol. The study's primary endpoint was met with high statistical significance (p<0.0001), and chenodiol demonstrated a 20-fold difference in efficacy compared to placebo. Diarrhea and headache were the most common adverse events, generally mild or moderate. If approved, chenodiol would be the first and only therapy available in the US for CTX, offering hope for early diagnosis and treatment of this debilitating condition.

Mirum Pharmaceuticals (Nasdaq: MIRM) announced positive interim results from two Phase 2b studies evaluating volixibat in patients with primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC).

In the VANTAGE PBC study, volixibat demonstrated a statistically significant improvement in pruritus with a 3.82 point reduction (p<0.0001) and a 2.32 point placebo-adjusted difference (p=0.0026). 75% of patients on volixibat achieved over 50% reduction in serum bile acids. Fatigue also improved significantly at week 16, with no new safety signals. The most common adverse event was mild to moderate diarrhea (77%).

The VISTAS PSC study exceeded the efficacy threshold for continuation. The independent data review committee recommended continuing the study with the current dose of 20 mg twice daily. Mirum will discuss these findings in a conference call on June 17 at 8:30 a.m. ET.

Mirum Pharmaceuticals (NASDAQ: MIRM) will host an investor call on June 17, 2024, at 8:30 a.m. ET to present interim results from two Phase 2b studies: the Volixibat VANTAGE study for primary biliary cholangitis (PBC) and the Volixibat VISTAS study for primary sclerosing cholangitis (PSC). Dial-in options are available, and a webcast will be accessible on Mirum's website. The replay of the call will be available for 30 days.

Mirum Pharmaceuticals (NASDAQ: MIRM) has announced the grant of inducement awards to 14 new employees under its 2020 Inducement Plan. On June 10, 2024, the Compensation Committee of Mirum’s Board of Directors approved stock options for 56,600 shares and 28,300 restricted stock units (RSUs). The stock options have an exercise price of $25.55 per share and will vest over four years, with 25% vesting after one year and the remainder vesting monthly over the next three years. The RSUs will vest over three years, with 33% vesting annually. These awards are in accordance with Nasdaq Listing Rule 5635(c)(4).

Mirum Pharmaceuticals presented data at the EASL Congress showing long-term benefits of LIVMARLI (maralixibat) in treating Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). The MERGE study demonstrated sustained clinical benefits for ALGS patients over seven years, including reductions in pruritus and serum bile acids, and improvements in height.

The MARCH/MARCH-ON trials indicated statistically significant growth improvements in PFIC patients over 82 weeks, with better height and weight z-scores. Another analysis from these trials showed that reductions in serum bile acids correlated with improved liver health markers like bilirubin, suggesting potential disease-modifying effects of LIVMARLI.

Mirum Pharmaceuticals (Nasdaq: MIRM) announced its participation in the Goldman Sachs 45th Annual Global Healthcare Conference in Miami on June 12th at 10:00 AM EDT.

The company, known for developing therapies for rare and orphan diseases, will provide a presentation accessible via the Investors and Media section on Mirum's corporate website.

For more details, investors can visit the company's site for webcast links and additional information.

Mirum Pharmaceuticals has received a positive opinion from the European Committee for Medicinal Products for Human Use (CHMP) for LIVMARLI® (maralixibat) oral solution for treating Progressive Familial Intrahepatic Cholestasis (PFIC) in patients aged three months and older. This decision is based on the Phase 3 MARCH study, which showed a highly significant reduction in pruritus severity (p<0.0001) compared to placebo across all PFIC types. Furthermore, the Committee for Orphan Medicinal Products (COMP) recommended maintaining the Orphan Drug Designation for LIVMARLI in PFIC. A final decision by the European Commission is expected in Q3 2024. Currently, LIVMARLI is approved for treating cholestatic pruritus in Alagille syndrome (ALGS) in Europe and the U.S., and for PFIC in U.S. patients aged five years and older. Mirum also plans to submit a supplemental new drug application (sNDA) to expand the label for younger PFIC patients in the U.S.

Mirum Pharmaceuticals announced the presentation of new long-term data from LIVMARLI studies at the upcoming EASL Annual Congress in Milan, Italy (June 5-8, 2024). The data highlights sustained clinical benefits of LIVMARLI for Alagille syndrome (ALGS) over seven years and improvements in liver markers and growth for Progressive Familial Intrahepatic Cholestasis (PFIC) patients. Key presentations include:

- THU-094: Clinical benefits of Maralixibat for ALGS patients through 7 years (MERGE Study).

- THU-129: Maralixibat improves growth in PFIC patients (MARCH/MARCH-ON Trials).

- THU-158: Serum bile acid levels and liver health improvements in PFIC children after Maralixibat treatment (MARCH/MARCH-ON Trials).

Additionally, a Mirum-sponsored symposium will discuss the role of IBAT inhibitors in adult hepatology and unmet needs in primary sclerosing cholangitis (PSC).