On Thursday, H.C. Wainwright adjusted its outlook on Mirum Pharmaceuticals (NASDAQ:MIRM), increasing the price target to $58 from the previous $45 while retaining a Buy rating on the stock. This change follows the recent approval by the U.S. Food and Drug Administration (FDA) for an additional use of Mirum's drug LIVMARLI (maralixibat).
The FDA has sanctioned LIVMARLI for the treatment of cholestatic pruritus in patients aged 5 and above with progressive familial intrahepatic cholestasis (PFIC), expanding its indications beyond the initial September 2021 approval for Alagille syndrome (ALGS).
The endorsement of LIVMARLI for PFIC is grounded in the outcomes of the Phase 3 MARCH study, which showed significant reductions in pruritus severity score and serum bile acid response. The study was notable for its inclusion of various PFIC genetic subtypes, offering a broader assessment compared to another market competitor, Ipsen's Bylvay, which was tested on a narrower range of subtypes.
With the immediate availability of LIVMARLI for PFIC patients in the U.S., Mirum Pharmaceuticals is set to prioritize broad payor coverage in 2024. Approximately 20 patients, nearly all over 5 years old, are anticipated to transition from the ongoing Phase 3 MARCH-ON study to commercial drug use by the end of 2024. This expectation has led to a projection of modest U.S. sales for LIVMARLI in PFIC for 2024, estimated at $10.4 million.
The revenue growth from PFIC is expected to be significant in the future, despite modest market penetration, due to higher pricing compared to ALGS. This anticipated growth has been a key factor in the raised price target for Mirum's shares. The company's strategic focus on securing extensive payor coverage is seen as a foundational step toward achieving these revenue targets in the coming years.
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