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
12
Phase 3 Programs
5
Upcoming Catalysts
1
Next Catalyst
Aug 6, 2026
6wMarket Overview
Stock performance and key metrics
1 upcoming, 1 past
PTC923
BH4 Deficiency
Ataluren (PTC124®)
Cystic Fibrosis
Vatiquinone
Friedreich Ataxia
Sepiapterin
Phenylketonuria
Ataluren
Duchenne Muscular Dystrophy
CNSA-001
Gastroparesis
PTC518
Huntington Disease
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 | - | - |
PTC518 | Phase 2 | Huntington Disease | - | - |
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 | - | - |