Scholar Rock Holding Corp SRRK
Scholar Rock Holding Corp (Nasdaq: SRRK) is a rare genetic drug developer. Its lead program, apitegromab, is in Phase 3 for Spinal muscular atrophy (SMA), non-ambulatory Type 2/3. Its next catalyst on record is SRK-439: Topline data from Phase 1 healthy-volunteer study (H2 2026).
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Next further out: SRK-439 — Topline data from Phase 1 healthy-volunteer study (H2 2026). 1 catalyst on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| apitegromab (IV) | SMA (BLA resubmitted, under FDA review; EMA MAA validated) | antibody | Phase 3 |
| apitegromab | Facioscapulohumeral muscular dystrophy (Phase 2 FORGE) | antibody | Phase 2 |
| apitegromab (OPAL) | SMA patients under age two (Phase 2) | antibody | Phase 2 |
| apitegromab + tirzepatide (EMBRAZE) | Preservation of lean mass during GLP-1 weight loss (Phase 2 completed, seeking partner) | antibody | Phase 2 |
| apitegromab SC formulation | SMA | antibody | Phase 1 |
| SRK-439 | Rare neuromuscular diseases (Phase 1 healthy volunteers) | antibody | Phase 1 |
| SRK-181 | Anti-PD-(L)1-resistant solid tumors (Phase 2-ready, seeking partner) | antibody | Phase 1 |
| SRK-373 | Fibrotic diseases | antibody | Pre-clinical |
| SRK-256 | Iron-restricted anemias | antibody | Pre-clinical |
Recent results and decisions
- Sep 11, 2026approval FDA approved ISEMBYLD (apitegromab-mstn) for SMA in adults and children 2 years and older on SMN2-targeted therapy, ahead of the Sept 30 PDUFA date; Rare Pediatric Disease priority review voucher awarded.
today announced that the FDA has approved ISEMBYLD (apitegromab-mstn) for the treatment of spinal muscular atrophy (SMA) in adults and children two years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment.
Filing - Aug 6, 2026started Phase 2 FORGE trial of apitegromab in FSHD initiated (about 60 patients, 1:1 vs placebo, primary endpoint lean muscle volume change at 12 months).
Phase 2 FORGE trial initiated.
Filing - May 7, 2026accepted FDA accepted the apitegromab BLA resubmission with a September 30, 2026 PDUFA action date; FDA completed reinspection of Catalent Indiana.
FDA accepted apitegromab Biologics License Application (BLA) for treatment of children and adults with spinal muscular atrophy (SMA) with September 30, 2026 Prescription Drug User Fee Act (PDUFA) action date
Filing
2 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.