Mirum Pharmaceuticals (MIRM) presented its rare-disease strategy and pipeline milestones at the 12th Annual Cantor Fitzgerald Global Healthcare Conference on September 9, 2026, highlighting a growing revenue base and several upcoming data readouts across its hepatology-focused drug portfolio.
Chief Executive Officer Chris Peetz described the company's approach as centered on acquiring overlooked rare-disease assets and developing them into high-impact medicines that larger pharmaceutical firms tend to bypass. "Probably only 5%" of rare diseases have an approved treatment, he noted.
On financials, Mirum reported trailing revenue of $618 million over the past twelve months, representing 44% year-over-year growth. The company said it is running at an annualized revenue rate of approximately $680 million to $700 million across three approved medicines. Gross profit margin came in at 81.7%, and management guided that LIVMARLI (maralixibat) could eventually reach peak sales above $1 billion.
Mirum's market capitalization stood at roughly $6.46 billion at the time of the conference. Its stock closed regular trading at $98.32, down 1.22%, and traded near $98.30 in after-hours activity; it was later listed around $99.53, down 1.36%.
Peetz signaled continued interest in acquisitions, saying, "I'm more interested in what can we buy rather than the other."
The company's pipeline includes several late-stage programs targeting rare liver diseases. Volixibat, being developed for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), is a central focus. Mirum held a pre-NDA meeting with the FDA in early 2024 and targets an NDA submission for PSC in the first half of 2025. For PBC, Phase III data is expected in the first quarter of 2025.
Peetz expressed confidence that existing VISTAS data supports a full NDA filing without further Phase III trials. "We don't see what would be gained by a phase III," he said, adding that the results are "clear and definitive" and sufficient to support labeling.
For hepatitis delta virus (HDV), brelovitug is being studied in the AZURE-1 and AZURE-4 programs. Readouts from the Phase III portions of both studies are expected in the fourth quarter of 2024, measuring week 24 endpoints. Week 48 follow-up data from the Phase II-B portion of AZURE-1 is anticipated in the fall of 2024. Only about 10% of hepatitis B-positive patients are currently tested for HDV, Peetz noted.
Zilurgisertib, an acquired program for fibrodysplasia ossificans progressiva (FOP), has a PDUFA date expected later in 2024.
LIVMARLI's EXPAND Phase III trial is also expected to read out in the fourth quarter of 2024. The drug is approved for progressive familial intrahepatic cholestasis (PFIC), with an estimated addressable adult population of about 2,000 patients in the U.S. Coverage currently reaches the top decile of adult hepatology and gastroenterology prescribers, with plans to expand into community GI clinics. Literature suggests about 20% of intrahepatic cholestasis of pregnancy (ICP) patients may carry PFIC genetics, opening a potential additional indication.
The PBC class pricing benchmark sits at approximately $150,000 per patient annually.
