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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. EPI-589

    PTC THERAPEUTICS, INC.

    Amyotrophic Lateral Sclerosis

    phase 2 small molecule completed

    1 trial

  2. EPI-743

    PTC THERAPEUTICS, INC.

    Leigh Syndrome

    phase 2 small molecule completed

    1 trial

  3. Eladocagene Exuparvovec

    PTC THERAPEUTICS, INC.

    AADC Deficiency

    phase 2 gene therapy active

    1 trial

  4. PTC124

    PTC THERAPEUTICS, INC.

    Cystic Fibrosis

    phase 2 small molecule completed

    2 trials

  5. PTC124

    PTC THERAPEUTICS, INC.

    Duchenne Muscular Dystrophy

    phase 2 small molecule completed

    1 trial

  6. PTC299

    PTC THERAPEUTICS, INC.

    Neurofibromatosis 2

    phase 2 small molecule terminated

    1 trial

  7. PTC518

    PTC THERAPEUTICS, INC.

    Huntington Disease

    phase 2 small molecule completed

    1 trial

  8. PTC857

    PTC THERAPEUTICS, INC.

    Amyotrophic Lateral Sclerosis

    phase 2 small molecule terminated

    1 trial

  9. PTC857, Placebo to match PTC857 60 mg/mL oral solution, Utreloxastat 62.5

    PTC THERAPEUTICS, INC.

    Amyotrophic Lateral Sclerosis

    phase 2 small molecule active

    1 trial

  10. PTC923

    PTC THERAPEUTICS, INC.

    BH4 Deficiency

    phase 2 small molecule completed

    1 trial

  11. Placebo

    PTC THERAPEUTICS, INC.

    Leigh Syndrome

    phase 2 small molecule completed

    1 trial

  12. Placebo

    PTC THERAPEUTICS, INC.

    Friedreich's Ataxia

    phase 2 small molecule completed

    1 trial

  13. Placebo to match 5-mg PTC518 tablets, Placebo to match 20-mg PTC518 tablets, PTC518, PTC518

    PTC THERAPEUTICS, INC.

    Huntington’s Disease

    phase 2 small molecule active

    1 trial

  14. VEGF inhibitor PTC299

    PTC THERAPEUTICS, INC.

    Kaposi's Sarcoma

    phase 2 small molecule terminated

    1 trial

  15. ataluren

    PTC THERAPEUTICS, INC.

    Epilepsy

    phase 2 small molecule completed

    1 trial

  16. gene therapy

    PTC THERAPEUTICS, INC.

    Aromatic L-amino Acid Decarboxylase (AADC) Deficiency

    phase 2 small molecule completed

    1 trial

  17. Ataluren

    PTC THERAPEUTICS, INC.

    Healthy

    phase 1 small molecule completed

    1 trial

  18. Deflazacort

    PTC THERAPEUTICS, INC.

    Renal Impairment

    phase 1 small molecule completed

    1 trial

  19. Deflazacort

    PTC THERAPEUTICS, INC.

    Hepatic Impairment

    phase 1 small molecule completed

    1 trial

  20. Deflazacort

    PTC THERAPEUTICS, INC.

    Healthy Volunteers

    phase 1 small molecule completed

    1 trial

  21. Deflazacort and rifampin

    PTC THERAPEUTICS, INC.

    Healthy Volunteers

    phase 1 small molecule completed

    1 trial

  22. PTC299

    PTC THERAPEUTICS, INC.

    Leukemia, Myeloid, Acute

    phase 1 small molecule terminated

    1 trial

  23. PTC299

    PTC THERAPEUTICS, INC.

    Advanced Cancer

    phase 1 small molecule terminated

    1 trial

  24. PTC299

    PTC THERAPEUTICS, INC.

    Metastatic Breast Cancer

    phase 1 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