PTC Therapeutics

Biotech

Pipeline Programs↗

Distinct drugs in development (Preclinical–Phase 3)

Indications↗

Distinct diseases across all programs

Clinical Trials↗

All sponsored trials (all statuses)

Orange Book Patents

Across 1 drug with upcoming expirations

Pipeline

Pipeline Snapshot

By Phase

3 drugs in development

Phase 33

By Therapeutic Area

top 2

Rare Disease

240%

Neurology

120%

R&D Momentum

35/100

Pipeline Velocity

6/100

Trial Completion Rate

68/100

Pipeline Concentration

36/100

Pipeline Freshness

74/100

Strategic Signals

↗Mature portfolio may be looking to in-license early assets
↗Active in Rare Disease but thin pipeline — potential licensing opportunity

Activity

Key Events

Jun 24, 2026TrialA Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria

Jul 28, 2025FDASephience — SEPHIENCE

Mar 4, 2024TrialA Study of Sepiapterin in Participants With Phenylketonuria (PKU)

Dec 8, 2022TrialA Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

Oct 12, 2022TrialA Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

Jun 22, 2022TrialA Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

Jun 3, 2022TrialA Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)

May 23, 2022TrialA Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)

3 more events

May 15, 2022TrialStudy to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS

Dec 29, 2021TrialA 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)

Sep 30, 2021TrialA Study of PTC923 in Participants With Phenylketonuria

Details

Company Info

Company Overview

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Mkt Cap

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Revenue

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Cash

Patent Expirations

2 patent groups across drugs · Source: FDA Orange Book

DrugExpires

Sephience3 patentsApr 16, 2038 SephienceMar 6, 2042

Pipeline

Drug Pipeline

Indication

Preclinical

Phase 1

Phase 2

Phase 3

Approved

PTC923Phase 3

SephiencePhase 3

Unknown / Other

Hyperphenylalaninemia

PTC923Phase 2

Mitochondrial Disease

VatiquinonePhase 3

Regulatory

Regulatory Timeline

20172 events

FDAFeb 9

Emflaza — EMFLAZA

Application NDA208685 (NDA)

FDAFeb 9

Emflaza — EMFLAZA

Application NDA208684 (NDA)

20251 event

FDAJul 28

Sephience — SEPHIENCE

Application NDA219666 (NDA)

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)