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Intelligence · Clinical Pipeline

R&D Pipeline Programs

ClinicalTrials.gov programs linked to pharma sponsors — phase, modality, and trial IDs for BD and competitive intelligence.

Programs indexed
18,156
Companies
1,062
Drugs tracked
3,385

Quick answer

NovaPharmaNews tracks 18,156 pharmaceutical R&D pipeline programs sourced from ClinicalTrials.gov, linked to sponsor companies. Each profile includes development phase, modality, trial status, NCT identifiers, and indication text — searchable for business development and competitive intelligence.

52 results for “PTC THERAPEUTICS, INC.” Clear

  1. Ataluren

    PTC THERAPEUTICS, INC.

    Duchenne Muscular Dystrophy

    phase 3 small molecule terminated

    9 trials

  2. Ataluren

    PTC THERAPEUTICS, INC.

    Cystic Fibrosis

    phase 3 small molecule completed

    6 trials

  3. Ataluren

    PTC THERAPEUTICS, INC.

    Muscular Dystrophy, Duchenne

    phase 3 small molecule terminated

    3 trials

  4. Ataluren (PTC124®)

    PTC THERAPEUTICS, INC.

    Cystic Fibrosis

    phase 3 small molecule completed

    1 trial

  5. Deflazacort

    PTC THERAPEUTICS, INC.

    Duchenne Muscular Dystrophy

    phase 3 small molecule completed

    3 trials

  6. Deflazacort

    PTC THERAPEUTICS, INC.

    Limb-Girdle Muscular Dystrophy

    phase 3 small molecule terminated

    1 trial

  7. PTC299

    PTC THERAPEUTICS, INC.

    Pneumonia

    phase 3 small molecule terminated

    1 trial

  8. PTC923

    PTC THERAPEUTICS, INC.

    Phenylketonuria

    phase 3 small molecule active

    2 trials

  9. PTC923, Kuvan 100 mg soluble tablets, PTC923

    PTC THERAPEUTICS, INC.

    Phenylketonuria

    phase 3 small molecule active

    1 trial

  10. PTC923, PTC923

    PTC THERAPEUTICS, INC.

    Metabolic Disorders - Phenylketonuria

    phase 3 small molecule active

    1 trial

  11. Sephience 250 mg oral powder in sachet, Sephience 1 000 mg oral powder in sachet

    PTC THERAPEUTICS, INC.

    Phenylketonuria

    phase 3 small molecule active

    1 trial

  12. Sepiapterin

    PTC THERAPEUTICS, INC.

    Phenylketonuria

    phase 3 small molecule active

    1 trial

  13. Unesbulin

    PTC THERAPEUTICS, INC.

    Leiomyosarcoma

    phase 3 small molecule terminated

    2 trials

  14. Vatiquinone

    PTC THERAPEUTICS, INC.

    Friedreich ataxia

    phase 3 small molecule active

    1 trial

  15. Vatiquinone

    PTC THERAPEUTICS, INC.

    Friedreich Ataxia

    phase 3 small molecule active

    3 trials

  16. Vatiquinone

    PTC THERAPEUTICS, INC.

    Inherited Mitochondrial Disease

    phase 3 small molecule completed

    1 trial

  17. Vatiquinone

    PTC THERAPEUTICS, INC.

    Mitochondrial Diseases

    phase 3 small molecule terminated

    1 trial

  18. AAV2-hAADC

    PTC THERAPEUTICS, INC.

    Aromatic Amino Acid Decarboxylase Deficiency

    phase 2 small molecule completed

    1 trial

  19. Ataluren

    PTC THERAPEUTICS, INC.

    Hemophilia A

    phase 2 small molecule terminated

    1 trial

  20. Ataluren

    PTC THERAPEUTICS, INC.

    Aniridia

    phase 2 small molecule terminated

    2 trials

  21. Ataluren

    PTC THERAPEUTICS, INC.

    Nonsene Mutation Duchenne Muscular Dystrophy

    phase 2 small molecule completed

    1 trial

  22. Ataluren

    PTC THERAPEUTICS, INC.

    Amino Acid Metabolism, Inborn Errors

    phase 2 small molecule terminated

    1 trial

  23. Ataluren + Pembrolizumab

    PTC THERAPEUTICS, INC.

    Colorectal Cancer

    phase 2 small molecule active

    1 trial

  24. CNSA-001

    PTC THERAPEUTICS, INC.

    Gastroparesis

    phase 2 small molecule completed

    1 trial

Frequently asked questions

What is the NovaPharmaNews pipeline directory?

It is a searchable index of 18,156 clinical development programs grouped by sponsor company, with phase (Phase 1–3), modality, trial status, and linked NCT IDs from ClinicalTrials.gov.

Where does pipeline data come from?

Program records are derived from ClinicalTrials.gov and linked to NovaPharmaNews company profiles. Trial status and phase reflect registry data at last sync — verify on ClinicalTrials.gov before investment or partnering decisions. See our data sources page for attribution.

How do I find pipeline programs by company or phase?

Use the search box to match program name, indication, company name, or slug. Results are ordered with late-stage (Phase 3) programs first. Open any row to view the full program profile under its sponsor company.

Data attribution and licensing: Data sources