PTC Therapeutics
74
2
6
46
Key Highlights
Risk & Performance
Pipeline Risk Assessment
Pipeline Risk Assessment
Based on historical performance
High Risk
Score: 60/100
25.7%
19 terminated/withdrawn out of 74 trials
70.8%
-15.7% vs industry average
23%
17 trials in Phase 3/4
78%
36 of 46 completed trials have results
Key Signals
Enrollment Performance
Analytics
Activity Timeline
Global Presence
Clinical Trials (74)
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Role: lead
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
Role: lead
A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
Role: lead
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
Role: lead
Vatiquinone Expanded Access Protocol
Role: collaborator
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
Role: lead
A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
Role: collaborator
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
Role: lead
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
Role: lead
Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Role: lead
A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
Role: lead
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
Role: lead
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
Role: lead
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
Role: lead
Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS
Role: lead
A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)
Role: lead
Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
Role: lead
Unesbulin in Women With Ovarian Cancer Receiving Neoadjuvant Chemotherapy
Role: collaborator
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Role: lead
A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)
Role: lead