WILMINGTON, DE — Mirum Pharmaceuticals (Nasdaq: MIRM) and Incyte (Nasdaq: INCY) are set to present pivotal Phase 2 clinical data for investigational fibrodysplasia ossificans progressiva (FOP) treatment zilurgisertib this week, as the companies move toward a key U.S. regulatory decision expected later this year.
The results, which will be presented at ENDO 2026 in Chicago, include findings from Cohort 1 of the placebo-controlled PROGRESS study and formed the basis of the New Drug Application submitted to the U.S. Food and Drug Administration for the rare disease therapy.
The presentation comes as the FDA reviews the application under a Priority Review designation, with a Prescription Drug User Fee Act (PDUFA) target action date of Sept. 26, 2026.
FOP is a rare genetic disorder that causes bone to form in muscles, tendons, and other connective tissues, progressively restricting mobility and leading to severe disability.
Zilurgisertib is an investigational ALK2 inhibitor designed to target the biological pathway responsible for abnormal bone growth in patients with the disease.
In April, Mirum acquired exclusive worldwide rights to zilurgisertib from Incyte under a licensing agreement, positioning the therapy as a potential addition to its portfolio of treatments for rare diseases.
“These data add to the growing clinical understanding of zilurgisertib’s potential in FOP,” Steven Stein, Incyte’s executive vice president, chief medical officer and head of late-stage development, said in discussing the upcoming presentation.
The late-breaking presentation, titled Zilurgisertib in Patients with Fibrodysplasia Ossificans Progressiva: Interim Results from the PROGRESS Study, is scheduled for June 14 at the Endocrine Society’s annual meeting.
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