Mirum reports Phase 3 EXPAND trial meets primary and secondary endpoints for LIVMARLI
Mirum Pharmaceuticals announced that its Phase 3 EXPAND study of LIVMARLI (maralixibat) achieved its primary endpoint and all key secondary measures in patients with ultra‑rare cholestatic liver diseases. The trial showed a statistically significant reduction in itch severity and serum bile acids versus placebo. The company said it will file a supplemental New Drug Application in the first half of 2027 to seek label expansion.
Why it matters
The company expects the sNDA filing to broaden LIVMARLI’s approved use, which could increase its addressable market and drive revenue growth (Baird note says the stock rating was reiterated and price targets raised). The positive data also lifted the share price, which rose about 5.7% after the announcement.
Key facts
- 1The EXPAND trial enrolled a total of 52 participants, of which 46 were children and six were adults. stocktitan.net
- 2In the pediatric cohort, maralixibat improved the ItchRO(Obs) severity score by 1.81 points versus 0.83 points for placebo, a placebo‑adjusted difference of 0.98 points (p=0.0004). stocktitan.net
- 3Serum bile acid levels fell 70.1% from baseline in the maralixibat arm compared with a 3.5% decline on placebo, yielding a 69% treatment effect (p<0.0001). rttnews.com
- 4Treatment‑emergent adverse events occurred in 96% of patients in both the drug and placebo groups; grade 3 or higher events were reported in 24% of maralixibat patients and 22.2% of placebo patients. stocktitan.net
- 5Diarrhea was reported in 64% of maralixibat‑treated participants versus 11.1% of those receiving placebo. stocktitan.net
- 6Mirum plans to submit a supplemental New Drug Application in the first half of 2027 to seek expanded indication for LIVMARLI. tradingview.com
Summary written by AlphAI AI Desk from 12 of 12 sources. Not investment advice. Figures are as stated by the linked sources.