Mirum Pharmaceuticals: ENDO 2026: Positive FOP Data For Zilurgisertib Sets Up September PDUFA

研报英文原文证据摘录

Mirum Pharmaceuticals: ENDO 2026: Positive FOP Data For Zilurgisertib Sets Up September PDUFA

Update

June 15, 2026 01:44 AM GMT

Morgan Stanley & Co. LLCMMirum Pharmaceuticals | North America Michael E Ulz

Equity Analyst

ENDO 2026: Positive FOP Data Mike.Ulz@morganstanley.comRohit Bhasin +1 212 761-4650

Research Associate

Rohit.Bhasin@morganstanley.com +1 212 761-0534

For Zilurgisertib Sets Up Avraham Novick

Avi.Novick@morganstanley.com +1 212 761-2231

September PDUFA

Mirum Pharmaceuticals (MIRM.O, MIRM US)

Biotechnology | United States of America

Key Takeaways Stock Rating Overweight

Industry View Attractive

Pivotal Ph2 PROGRESS Cohort 1 data showed reductions in total HO lesion Price target $150.00

volume, new HO lesions, and flare activity during 24-week period. Shr price, close (Jun 12, 2026) $99.35

Mkt cap, curr (mm) $5,847

No new HO lesions were observed in OLE among patients continuing zilurgisertib 52-Week Range $114.99-47.79

or crossing over from placebo at Week 24, based on 48-week data.

Most AEs were mild/moderate, none led to treatment discontinuation , and

serious AEs (including Grade ≥3) occurred at low rates across both treatment

groups.

Data from PROGRESS supported an accepted NDA for zilurgisertib in FOP under

Priority Review, with a PDUFA date set for Sep 26.

Overall, we believe the FOP data position Mirum well for commercialization in

another rare disease indication, further expanding its commercial footprint.

FOP PDUFA on track for late-September. In a late-breaking presentation at ENDO

2026 (Chicago, June 13-16), Mirum and Incyte (covered by Judah Frommer) reported

pivotal Ph2 results from Cohort 1 of the PROGRESS study evaluating zilurgisertib in

patients with fibrodysplasia ossificans progressiva (FOP; press release; presentation;

poster).