Mirum Pharmaceuticals Announces Positive Milestones for Maralixibat and Upcoming Investor Disclosure

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) reported that its oral ileal bile acid transporter (IBAT) inhibitor, maralixibat (trade name LIVMARLI®), has achieved its primary endpoint in a clinical trial for a cholestatic liver disease. The study, conducted in a patient population with a cholestatic condition, demonstrated that maralixibat met the predefined efficacy criteria, providing further evidence of the drug’s therapeutic potential.

Trial Highlights

  • Primary Endpoint Achievement – The result confirms that maralixibat produces clinically meaningful benefit in patients with cholestatic liver disease, supporting its continued development.
  • Phase III EXPAND Study – The company is preparing to release topline data from the Phase III EXPAND trial, which evaluates maralixibat for cholestatic pruritus in patients aged 6 months or older with ultra‑rare cholestatic liver diseases, including biliary atresia.
  • Investor Call – Mirum scheduled an investor call on Monday, October 12, 2026 at 8:30 a.m. ET (5:30 a.m. PT) to present the Phase III EXPAND results. The call is accessible via phone and webcast through the company’s investor relations website.

Company Position and Portfolio

Mirum is a rare‑disease focused pharmaceutical company headquartered in Foster City, California. Its portfolio includes:

  • LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC)
  • ATEBRIOZ™ (zilurgisertib) for fibrodysplasia ossificans progressiva (FOP)
  • CHOLBAM® (cholic acid) for bile‑acid synthesis disorders
  • CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX)

The company’s clinical‑stage pipeline contains volixibat for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug for chronic hepatitis delta virus (HDV), and MRM‑3379 for Fragile X syndrome (FXS).

Market Context

  • Current Share Price: $86.01 (as of 2026‑10‑08)
  • 52‑Week High/Low: $130.00 / $63.232
  • Market Capitalization: $5.58 billion
  • Price‑to‑Earnings Ratio: –5.69

The company trades on the Nasdaq stock exchange under the ticker MIRM and serves patients nationwide.

Implications for Investors

The positive primary‑endpoint result for maralixibat, combined with the forthcoming Phase III EXPAND topline data, reinforces Mirum’s pipeline strength in the cholestatic liver disease segment. Investors can anticipate detailed efficacy and safety data from the October 12 investor call, which may influence the company’s valuation and future development strategy.

For further information, stakeholders are encouraged to review the official investor relations materials available on the Mirum website and to participate in the scheduled investor call.