PTC Therapeutics, Inc. PTCT
PTC Therapeutics, Inc. (Nasdaq: PTCT) is a rare genetic drug developer. Its lead program, Sephience (sepiapterin), is in Phase 3 for Phenylketonuria. Its next catalyst on record is ST-920 (isaralgagene civaparvovec): PTC completed the ST-920 acquisition and expects to complete the rolling BLA submission for accelerated approval in Q4 2026. (Q4 2026).
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Next further out: ST-920 (isaralgagene civaparvovec) — PTC completed the ST-920 acquisition and expects to complete the rolling BLA submission for accelerated approval in Q4 2026. (Q4 2026). 1 catalyst on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| Sephience (sepiapterin) | PKU | small molecule | Approved |
| Emflaza | Duchenne muscular dystrophy | small molecule | Approved |
| Kebilidi | AADC deficiency | gene therapy | Approved |
| Vatiquinone | Friedreich ataxia | small molecule | Phase 3 |
| PTC844 | not captured | small molecule | Phase 1 |
| PTC612 | not captured | small molecule | Phase 1 |
Recent results and decisions
- Aug 12, 2026submitted PTC to acquire BLA-stage ST-920 from Sangamo; rolling BLA for accelerated approval has nonclinical and clinical modules submitted, CMC package and completion expected Q4 2026.
A rolling BLA submission to FDA for accelerated approval of ST-920 is expected to be completed in Q4 2026.
Filing - Aug 12, 2026positive Phase 1/2 STAAR showed a positive mean annualized eGFR slope at 52 weeks after one-time ST-920 gene therapy, with alpha-Gal A activity sustained up to 4.5 years.
The Phase 1/2 STAAR study demonstrated positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks following ST-920 administration
Filing - Apr 28, 2026positive 24-month interim of PIVOT-HD extension showed dose-dependent slowing of cUHDRS progression vs natural history in Stage 2 HD patients: 52% at 10 mg and 28% at 5 mg, no treatment-related NfL increases.
there was evidence of dose-dependent benefit in slowing progression on the Composite Unified Huntington's Disease Rating Scale (cUHDRS) relative to a propensity weighted natural history cohort in Stage 2 participants, with 52% and 28% slowing for 10 mg and 5 mg participants, respectively.
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.