Sarepta Therapeutics, Inc. SRPT
Sarepta Therapeutics, Inc. (Nasdaq: SRPT) is a rare genetic drug developer. Its lead program, ELEVIDYS (delandistrogene moxeparvovec-rokl) and PMO products EXONDYS 51, VYONDYS 53, AMONDYS 45, is in Phase 3 for Duchenne muscular dystrophy. Its next catalyst on record is SRP-1003: Further Phase 1/2 data (H2 2026).
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Next further out: SRP-1003 — Further Phase 1/2 data (H2 2026). 2 catalysts on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| ELEVIDYS | Duchenne (ambulatory patients; non-ambulatory removed from label Nov 2025, boxed warning for ALI/ALF) | gene therapy | Approved |
| EXONDYS 51 / VYONDYS 53 / AMONDYS 45 | Duchenne exon 51/53/45 skipping (accelerated approvals); sNDAs for VYONDYS 53 and AMONDYS 45 accepted for filing June 2026 | RNA (siRNA/ASO/mRNA) | Approved |
| SRP-9003 (bidridistrogene xeboparvovec) | LGMD2E/R4 (EMERGENE Phase 3 fully enrolled and dosed; FDA clinical hold since July 2025; FDA requires sirolimus data before accepting BLA) | gene therapy | Phase 3 |
| SRP-1003 | myotonic dystrophy type 1 (Phase 1/2, Arrowhead-conducted) | RNA (siRNA/ASO/mRNA) | Phase 1 |
| SRP-1001 | FSHD (Phase 1/2, Arrowhead-conducted) | RNA (siRNA/ASO/mRNA) | Phase 1 |
| SRP-1005 | Huntington's disease (Phase 1 INSIGHTT, first patient enrolled Q2 2026) | antibody | Phase 1 |
Recent results and decisions
- May 6, 2026submitted Sarepta completed sNDA submissions to FDA seeking conversion of AMONDYS 45 and VYONDYS 53 from accelerated to traditional approval.
Completed submission of sNDA for AMONDYS 45 and VYONDYS 53 to FDA seeking conversion to traditional approval
Filing - May 6, 2026positive First Phase 1/2 data for SRP-1001 showed dose-dependent drug exposure, early biomarker effects and favorable tolerability.
siRNA pipeline delivers first Phase 1/2 data for SRP-1001 (FSHD1) and SRP-1003 (DM1) showing dose-dependent drug exposure, early biomarker effects, and favorable tolerability
Filing - May 6, 2026positive First Phase 1/2 data for SRP-1003 showed dose-dependent drug exposure, early biomarker effects and favorable tolerability.
siRNA pipeline delivers first Phase 1/2 data for SRP-1001 (FSHD1) and SRP-1003 (DM1) showing dose-dependent drug exposure, early biomarker effects, and favorable tolerability
Filing
3 more on record.
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 3 rare genetic companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.