Mirum Pharmaceuticals and Incyte Announce Pivotal Late-Breaking Results for Zilurgisertib in Fibrodysplasia Ossificans Progressiva Accepted for Presentation at ENDO 2026

About this update from Mirum Pharmaceuticals, Inc.

- Late-breaking rapid-fire presentation to include results from Cohort 1 of the placebo-controlled PROGRESS study evaluating zilurgisertib in fibrodysplasia ossificans progressiva (FOP) FOSTER CITY, Calif. & WILMINGTON, Del. --(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) and Incyte (Nasdaq: INCY) today announced that pivotal Phase 2 results from the PROGRESS study evaluating zilurgisertib, an investigational ALK2 inhibitor, in patients with fibrodysplasia ossificans progressiva (“FOP”) will be presented at ENDO 2026, the Endocrine Society’s annual meeting, taking place June 13-16, 2026 , in Chicago, Illinois . This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260604863826/en/ The late-breaking rapid-fire presentation will include results from Cohort 1 of the placebo-controlled PROGRESS study, which enrolled patients 12 years of age and older and formed the basis of the New Drug Application (NDA) for zilurgisertib to the U.S. Food and Drug Administration (FDA). In April 2026 , Mirum entered into an exclusive license agreement with Incyte for worldwide rights to zilurgisertib. “ENDO 2026 is an important milestone for the zilurgisertib FOP program as we share pivotal results from the PROGRESS study in patients living with this debilitating disease,” said Steven Stein , M.D., Executive Vice President, Chief Medical Officer and Head of Late-stage Development at Incyte . “These data add to the growing clinical understanding of zilurgisertib’s potential in FOP as Incyte and Mirum continue to advance toward the FDA’s Priority Review PDUFA date of September 26 , 2026.” “FOP is a devastating, progressive disease that profoundly impacts patients and families,” said Joanne Quan , M.D., Chief Medical Officer at Mirum Pharmaceuticals . “At Mirum, in partnership with Incyte , we are committed to advancing zilurgisertib with urgency as we work toward potentially bringing a needed new treatment option to people living with FOP.” Congress Presentation Additional details regarding the presentation are as follows: Abstract # ORF37-04 Title Zilurgisertib in Patients with Fibrodysplasia Ossificans Progressiva: Interim Results from the PROGRESS Study Date/Time Sunday, June 14 , 3:30-4:15 pm CT Abstracts accepted for presentation at ENDO 2026 will be available once published through the Endocrine Society’s ENDO 2026 website . About Zilurgisertib Zilurgisertib is an investigational, oral, small molecule, activin receptor-like kinase 2 (ALK2) inhibitor in development for the treatment of Fibrodysplasia Ossificans Progressiva (FOP). Zilurgisertib is designed to inhibit the ALK2 receptor, which is abnormally active in most patients with FOP and leads to bone formation in soft tissues, a process known as heterotopic ossification (HO). FOP is an ultra-rare genetic disease that affects approximately 300 patients in the U.S . and 900 worldwide, with diagnosis typically occurring in early childhood. Zilurgisertib was evaluated in the PROGRESS pivotal Phase 2 study, which formed the basis of a new drug application (NDA). The FDA has accepted the NDA for zilurgisertib in FOP under Priority Review with a Prescription Drug User Fee Act (PDUFA) date of September 26, 2026 . Mirum licensed zilurgisertib from Incyte for development and commercialization globally. About the PROGRESS Study PROGRESS is a global, randomized, double-blind, placebo-controlled Phase 2 study evaluating the efficacy and safety of zilurgisertib in patients with fibrodysplasia ossificans progressiva (FOP). PROGRESS Cohort 1 enrolled patients 12 years of age and older who were randomized 1:1 to receive zilurgisertib 100 mg once daily or placebo during a 24-week double-blind treatment period, followed by an open-label extension. Additional PROGRESS cohorts will evaluate the efficacy and safety of zilurgisertib in patients ages 6 to

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