USFDAJul 29, 2026, 04:33 PM
Dyne Therapeutics BLA for z-rostudirsen in DMD Accepted by FDA with Priority Review
AI Summary
Dyne Therapeutics announced that the FDA accepted its Biologics License Application (BLA) for z-rostudirsen in exon 51 DMD, granting Priority Review with a PDUFA target action date of January 21, 2027. The company also completed enrollment for the registrational expansion cohort of its Phase 1/2 ACHIEVE trial for z-basivarsen in DM1, with topline data expected in Q1 2027, and received FDA clearance for its IND application for DYNE-302 in FSHD. Financially, Dyne reported a net loss of $178.6 million for Q2 2026 and expects its cash runway to extend into Q2 2028, bolstered by a recent $405.0 million public offering.
Key Highlights
- FDA accepted BLA for z-rostudirsen in DMD with Priority Review.
- PDUFA target action date for z-rostudirsen is January 21, 2027.
- Completed enrollment of 71 participants in ACHIEVE trial for z-basivarsen (DM1).
- Topline data for z-basivarsen (DM1) planned for Q1 2027.
- FDA cleared IND application for DYNE-302 in FSHD.
- Cash, cash equivalents, and marketable securities were $898.5 million as of June 30, 2026.
- Net proceeds from July 2026 public offering were approximately $405.0 million.
- Expected cash runway into Q2 2028.
- Net loss for Q2 2026 was $178.6 million, or $1.08 per share.
- R&D expenses for Q2 2026 increased to $152.2 million from $99.2 million YoY.
Price Impact
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