Mirum Pharmaceuticals (MIRM) raised its full-year 2024 revenue guidance to $680 million–$700 million at the H.C. Wainwright 28th Annual Global Investment Conference on Tuesday, citing continued momentum behind its approved liver-disease drug LIVMARLI.
Second-quarter revenue totaled $176.2 million across approved medicines, with LIVMARLI alone contributing $129 million, up 13% sequentially. Trailing twelve-month revenue as of Q2 reached $618 million, representing 44% year-over-year growth, and the company reported a gross profit margin of 81.7%.
Mirum said it expects 2024 cash flow to be roughly breakeven to slightly negative as it scales manufacturing capacity, with a return to operational cash-flow positivity targeted for 2025.
CEO Chris Peetz described the company's strategy of focusing on underserved rare-disease indications that receive less attention from larger pharma companies. "Across rare disease, probably only about 5% of those rare diseases have approved medicines," he said.
Pipeline Catalysts
The conference provided an updated timeline for Mirum's development programs:
Zilurgisertib, an oral ALK-2 inhibitor for fibrodysplasia ossificans progressiva (FOP), co-developed with Incyte, has a PDUFA decision date of September 26, 2024. The target label covers patients aged 12 and older.
Brelovitug, a bulevirtide derivative for hepatitis delta, is set to report AZURE-1 topline data later in September 2024, followed by AZURE-4 topline data in the fourth quarter. In Phase IIb, brelovitug achieved a 100% virologic response rate at the 300 mg weekly dose at the 24-week time point.
Volixibat, developed for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), will report PSC data and support a planned New Drug Application in the first half of 2025. The VANTAGE study in PBC is expected to read out in the first quarter of 2027. On the interim results, Peetz said the observed reduction in pruritus was "strikingly strong" and could be "a game changer for PBC patients."
Commercial Expansion
LIVMARLI is approved for cholestatic pruritus in patients with Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC). The EXPAND basket trial, which includes patients with biliary atresia and other cholestatic diseases, should complete unblinding in Q4.
Mirum noted approximately 2,000 underdiagnosed adult PFIC patients in the U.S. The company currently targets only the top 10% of adult hepatology and gastroenterology specialists, with an expanded sales force expected to deploy fully by the end of 2024 or early 2025. Peetz highlighted Latin America as a region with notable growth potential, while noting the Middle East remains in earlier stages of commercial development.
