Mirum's Livmarli Cuts Itch Versus Placebo In 52-Patient Phase 3 Trial, Sets 2027 Filing Plan
The drug met the trial's main goal and all five secondary measures, and Mirum plans a U.S. supplemental application in the first half of 2027.
Mirum Pharmaceuticals said its drug Livmarli met the main goal of the 52-patient EXPAND trial and was statistically significant on all five prespecified secondary measures, according to an 8-K filed Tuesday. The company announced the topline results Monday.
What the 8-K says
The late-stage trial tested Livmarli (maralixibat) for itching in patients aged 6 months or older with ultra-rare cholestatic liver diseases, including biliary atresia. It enrolled 46 pediatric and six adult patients, randomized to the drug or placebo for a 20-week double-blind period.
In the pediatric group, itch severity improved a mean 1.81 points on maralixibat versus 0.83 on placebo, a placebo-adjusted 0.98 points (p=0.0004). Serum bile acids in the overall population fell 70.1% versus 3.5% on placebo.
In patients with biliary atresia and a native liver, itch scores improved 2.16 points versus 1.01 on placebo. Pruritus (itching) improved on 96.4% of days versus 52.2%.
Diarrhea occurred in 64.0% of drug patients versus 11.1% on placebo. Abdominal pain was 20.0% versus 14.8%. The company said safety matched prior experience with maralixibat and no new signals appeared; five serious events in drug-treated patients were all judged unrelated.
What it changes
Mirum plans to file a supplemental New Drug Application in the United States in the first half of 2027, based on these results. Livmarli is already FDA-approved for itching tied to Alagille syndrome and progressive familial intrahepatic cholestasis, Fierce Pharma reported.
The company estimates about 2,000 biliary atresia patients in the United States and Europe are addressable for Livmarli. MIRM closed at $86.06 on Monday, up 0.06% from the prior session.
What to watch
The next dated items are the supplemental filing, planned for the first half of 2027, and topline results from a mid-stage volixibat trial, expected in the first quarter of 2027.
The filing's safety table puts diarrhea at 16 of 25 drug patients versus 3 of 27 on placebo, the largest gap among the adverse events it lists.
Sources
- 8-K Filing — Mirum Pharmaceuticals, Inc. (MIRM) — SEC EDGAR
- Mirum on its way to 3rd rare liver indication for Livmarli | Fierce Pharma — fiercepharma.com
- Mirum Pharma Shares Gain After Liver-Disease Treatment Meets Primary Endpoint in Phase 3 Trial — MarketWatch
- Why Is Mirum Pharmaceuticals Stock Gaining Monday? — benzinga.com
- Mirum Pharmaceuticals to Host Investor Call to Share Topline Results from the Phase 3 EXPAND Study of LIVMARLI® (maralixibat) in Ultra-Rare Cholestatic Liver Diseases — Yahoo Finance
- Mirum Pharmaceuticals to report Livmarli trial results Monday (MIRM:NASDAQ) | Seeking Alpha — seekingalpha.com
