Wednesday, June 10, 2026
🇺🇸 FDA

pharma · Phenylketonuria · Amyotrophic Lateral Sclerosis · PTCT

PTC THERAPEUTICS

PTC THERAPEUTICS, INC.

PTC THERAPEUTICS clinical programs — 52 development programs from ClinicalTrials.gov with phase, modality, indication, and trial identifiers.

Website Investor relations
500 Warren Corporate Center Drive, Warren, NJ 07059, US HQ
1998 Founded
1,153 Employees
Public company Type
PTCT · NYSE Ticker
Company details
Status
Public
HQ
500 Warren Corporate Center Drive, Warren, NJ 07059, US
Founded
1998
Employees
1,153
Programs
52
Drugs
21
Patents
149
Official website ↗ All projects Drug pipeline Patents News & analysis
Intelligence · Clinical Programs

PTC THERAPEUTICS projects

52 ClinicalTrials.gov programs sponsored by PTC THERAPEUTICS.

PTC THERAPEUTICS has 52 clinical development programs indexed from ClinicalTrials.gov, including phase, modality, indication text, and linked NCT identifiers.

All phases Phase III Phase II Phase I NDA / BLA filed Approved programs

52 programs

Clinical programs

Page 1 of 2

  1. Ataluren

    PTC THERAPEUTICS, INC.

    Muscular Dystrophy, Duchenne

    phase 3 small molecule terminated

    3 trials

  2. Ataluren

    PTC THERAPEUTICS, INC.

    Duchenne Muscular Dystrophy

    phase 3 small molecule terminated

    9 trials

  3. Ataluren

    PTC THERAPEUTICS, INC.

    Cystic Fibrosis

    phase 3 small molecule completed

    6 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, PTC923

    PTC THERAPEUTICS, INC.

    Metabolic Disorders - Phenylketonuria

    phase 3 small molecule active

    1 trial

  10. PTC923-PKU-301

    PTC THERAPEUTICS, INC.

    Phenylketonuria

    phase 3 small molecule active

    1 trial

  11. PTC923-PKU-401

    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.

    Mitochondrial Diseases

    phase 3 small molecule terminated

    1 trial

  15. Vatiquinone

    PTC THERAPEUTICS, INC.

    Friedreich ataxia

    phase 3 small molecule active

    1 trial

  16. Vatiquinone

    PTC THERAPEUTICS, INC.

    Friedreich Ataxia

    phase 3 small molecule active

    3 trials

  17. Vatiquinone

    PTC THERAPEUTICS, INC.

    Inherited Mitochondrial Disease

    phase 3 small molecule completed

    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.

    Amino Acid Metabolism, Inborn Errors

    phase 2 small molecule terminated

    1 trial

  21. Ataluren

    PTC THERAPEUTICS, INC.

    Nonsene Mutation Duchenne Muscular Dystrophy

    phase 2 small molecule completed

    1 trial

  22. Ataluren

    PTC THERAPEUTICS, INC.

    Aniridia

    phase 2 small molecule terminated

    2 trials

  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

  25. EPI-589

    PTC THERAPEUTICS, INC.

    Amyotrophic Lateral Sclerosis

    phase 2 small molecule completed

    1 trial

  26. EPI-743

    PTC THERAPEUTICS, INC.

    Leigh Syndrome

    phase 2 small molecule completed

    1 trial

  27. Eladocagene Exuparvovec

    PTC THERAPEUTICS, INC.

    AADC Deficiency

    phase 2 gene therapy active

    1 trial

  28. PTC124

    PTC THERAPEUTICS, INC.

    Duchenne Muscular Dystrophy

    phase 2 small molecule completed

    1 trial

  29. PTC124

    PTC THERAPEUTICS, INC.

    Cystic Fibrosis

    phase 2 small molecule completed

    2 trials

  30. PTC299

    PTC THERAPEUTICS, INC.

    Neurofibromatosis 2

    phase 2 small molecule terminated

    1 trial

  31. PTC518

    PTC THERAPEUTICS, INC.

    Huntington Disease

    phase 2 small molecule completed

    1 trial

  32. PTC518-CNS-002-HD

    PTC THERAPEUTICS, INC.

    Huntington’s Disease

    phase 2 small molecule active

    1 trial

  33. PTC857

    PTC THERAPEUTICS, INC.

    Amyotrophic Lateral Sclerosis

    phase 2 small molecule terminated

    1 trial

  34. PTC857-CNS-001-ALS

    PTC THERAPEUTICS, INC.

    Amyotrophic Lateral Sclerosis

    phase 2 small molecule active

    1 trial

  35. PTC923

    PTC THERAPEUTICS, INC.

    BH4 Deficiency

    phase 2 small molecule completed

    1 trial

  36. Placebo

    PTC THERAPEUTICS, INC.

    Leigh Syndrome

    phase 2 small molecule completed

    1 trial

  37. Placebo

    PTC THERAPEUTICS, INC.

    Friedreich's Ataxia

    phase 2 small molecule completed

    1 trial

  38. VEGF inhibitor PTC299

    PTC THERAPEUTICS, INC.

    Kaposi's Sarcoma

    phase 2 small molecule terminated

    1 trial

  39. ataluren

    PTC THERAPEUTICS, INC.

    Epilepsy

    phase 2 small molecule completed

    1 trial

  40. gene therapy

    PTC THERAPEUTICS, INC.

    Aromatic L-amino Acid Decarboxylase (AADC) Deficiency

    phase 2 small molecule completed

    1 trial

  41. Ataluren

    PTC THERAPEUTICS, INC.

    Healthy

    phase 1 small molecule completed

    1 trial

  42. Deflazacort

    PTC THERAPEUTICS, INC.

    Renal Impairment

    phase 1 small molecule completed

    1 trial

  43. Deflazacort

    PTC THERAPEUTICS, INC.

    Hepatic Impairment

    phase 1 small molecule completed

    1 trial

  44. Deflazacort

    PTC THERAPEUTICS, INC.

    Healthy Volunteers

    phase 1 small molecule completed

    1 trial

  45. Deflazacort and rifampin

    PTC THERAPEUTICS, INC.

    Healthy Volunteers

    phase 1 small molecule completed

    1 trial

  46. PTC299

    PTC THERAPEUTICS, INC.

    Leukemia, Myeloid, Acute

    phase 1 small molecule terminated

    1 trial

  47. PTC299

    PTC THERAPEUTICS, INC.

    Metastatic Breast Cancer

    phase 1 small molecule completed

    1 trial

  48. PTC299

    PTC THERAPEUTICS, INC.

    Advanced Cancer

    phase 1 small molecule terminated

    1 trial

Frequently asked questions

How many clinical projects does PTC THERAPEUTICS have?

52 published programs are linked to this sponsor in our ClinicalTrials.gov index.

Is this the complete company pipeline?

This directory lists published programs for the sponsor in our database. Partnered or out-licensed assets may appear under multiple companies.

Drug approvals, clinical trials, M&A, event intelligence, and company updates — weekly.

Customize topics & region
Topics
Region

No spam. Unsubscribe anytime. See our privacy policy.