Mirum Pharmaceuticals Inc. (NASDAQ: MIRM) and Incyte Corp. (NASDAQ: INCY) announced that results from a Phase 2 study of zilurgisertib for fibrodysplasia ossificans progressiva will be presented at the Endocrine Society's annual meeting in June 2026.

The presentation will include data from Cohort 1 of the placebo-controlled PROGRESS study, which evaluated zilurgisertib in patients 12 years and older with the rare genetic disorder. These results formed the basis for the New Drug Application submitted to the U.S. Food and Drug Administration.

Zilurgisertib is an investigational oral ALK2 inhibitor designed to treat fibrodysplasia ossificans progressiva, a condition where soft tissues gradually turn into bone. The disease affects approximately 300 patients in the United States and 900 worldwide.

The FDA accepted the drug application under Priority Review with a target decision date of September 26, 2026. In April 2026, Mirum entered into an exclusive worldwide licensing agreement with Incyte for zilurgisertib.

The PROGRESS study is a randomized, double-blind, placebo-controlled trial that enrolled patients in a 1:1 ratio to receive either zilurgisertib 100 mg daily or placebo for 24 weeks, followed by an open-label extension. The primary endpoint measures the proportion of patients developing new heterotopic ossification lesions at week 24.

The presentation is scheduled for June 14, 2026, during the ENDO 2026 conference in Chicago. Additional study cohorts are evaluating the drug in younger patients aged 6 to 12 years and 2 to 12 years.