PTC Therapeutics, Inc.
PTCTPTC Therapeutics, Inc. is a US pharmaceutical company focused on the development of orally administered small molecule drugs and gene therapy which regulate gene expression by targeting post-transcriptional control (PTC) mechanisms in orphan diseases.
Drugs in Pipeline
11
Phase 3 Programs
5
Upcoming Catalysts
0
Next Catalyst
None scheduled
Market Overview
Stock performance and key metrics
0 upcoming, 0 past
No catalysts found.
How PTCT's past catalysts played out
PTC923
BH4 Deficiency
Ataluren (PTC124®)
Cystic Fibrosis
Vatiquinone
Friedreich Ataxia
Sepiapterin
Phenylketonuria
Ataluren
Duchenne Muscular Dystrophy
CNSA-001
Gastroparesis
PTC124
Cystic Fibrosis
EPI-743
Leigh Syndrome
EPI-589
Amyotrophic Lateral Sclerosis
EPI-743 400 mg
Friedreich's Ataxia
EPI-743 15 mg/kg
Leigh Syndrome
| Drug Name | Phase | Indication | Designations | Patent |
|---|---|---|---|---|
PTC923 | Phase 3 | BH4 Deficiency | - | - |
Ataluren (PTC124®) | Phase 3 | Cystic Fibrosis | - | - |
Vatiquinone | Phase 3 | Friedreich Ataxia | - | - |
Sepiapterin | Phase 3 | Phenylketonuria | - | - |
Ataluren | Phase 3 | Duchenne Muscular Dystrophy | - | - |
CNSA-001 | Phase 2 | Gastroparesis | - | - |
PTC124 | Phase 2 | Cystic Fibrosis | - | - |
EPI-743 | Phase 2 | Leigh Syndrome | - | - |
EPI-589 | Phase 2 | Amyotrophic Lateral Sclerosis | - | - |
EPI-743 400 mg | Phase 2 | Friedreich's Ataxia | - | - |
EPI-743 15 mg/kg | Phase 2 | Leigh Syndrome | - | - |
Frequently asked questions
What is in PTC Therapeutics, Inc.'s drug pipeline?→
CatalystAlert tracks 11 drug candidates in PTC Therapeutics, Inc.'s (PTCT) pipeline across Phase 3, Phase 2. Its lead program is PTC923 (Phase 3). Each drug's phase, indication and upcoming catalysts are listed on this page.
When is PTC Therapeutics, Inc.'s next FDA catalyst?→
CatalystAlert tracks every upcoming catalyst for PTC Therapeutics, Inc. (PTCT) — PDUFA decision dates, Phase 1/2/3 trial readouts, FDA advisory committee meetings and NDA/BLA filings — each confidence-rated as confirmed, estimated or AI-detected.
How do I track PTCT biotech catalysts?→
Add PTCT to your CatalystAlert watchlist for alerts ahead of each catalyst, and follow PTC Therapeutics, Inc.'s live catalyst calendar, drug pipeline and clinical-trial timeline on this page — sourced from ClinicalTrials.gov, FDA and SEC filings.