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PTC124 · 3 trials · 2 indications
| Arm | Type | Description |
|---|---|---|
| PTC124 PO | EXPERIMENTAL | 4-, 4-, and 8-mg/kg TID first 14 days of cycle 10-, 10-, and 20-mg/kg TID second 14 days of cycle 28 day study |
| Name | Type | Description |
|---|---|---|
| PTC124 | DRUG | 4-,4-, and 8-mg/kg PO first 14 days 10-, 10-, and 20-mg/kg TID second 14 days |
Inclusion Criteria: Patients must meet all of the following conditions to be eligible for enrollment into the study: * Completion of ≥ 1 cycle of PTC124 treatment in the previous Phase 2a study protocol (Protocol Number PTC124-GD-005-CF). * Abnormal nasal epithelial TEPD total chloride conductance...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | VX-121/TEZ/D-IVA |
| Sionna Therapeutics, Inc. | SION | 2 | PHASE2 | SION-719 |
| BiomX Inc. | PHGE | 1 | PHASE2 | BX004 |
| 4D Molecular Therapeutics, Inc. | FDMT | 1 | PHASE2 | 4D-710 |
| Arcturus Therapeutics Holdings, Inc. | ARCT | 1 | PHASE2 | ARCT-032 |
| Krystal Biotech, Inc. | KRYS | 1 | PHASE1 | KB407 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
PTC124 is an investigational small molecule being studied for the treatment of Duchenne Muscular Dystrophy and Cystic Fibrosis. It is being developed by PTC Therapeutics, Inc. (NASDAQ: PTCT). The drug has completed Phase 2 clinical trials for both indications, but it is not yet approved and remains in clinical development.
PTC124 is a small molecule that targets the underlying genetic defects in Duchenne Muscular Dystrophy and Cystic Fibrosis. It is designed to promote readthrough of premature stop codons, allowing the production of full-length, functional proteins. This mechanism is intended to address the root cause of these diseases.
PTC124 is being developed by PTC Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol PTCT. The company is conducting clinical trials to evaluate the safety and efficacy of PTC124 in patients with Duchenne Muscular Dystrophy and Cystic Fibrosis.
PTC124 is in Phase 2 clinical development. It has completed two Phase 2 trials for Cystic Fibrosis and one Phase 2 trial for Duchenne Muscular Dystrophy. All trials are completed, and the drug is not yet approved by regulatory authorities. It remains an investigational agent.
PTC124 has been studied in three completed Phase 2 trials. NCT00234663 and NCT00351078 evaluated PTC124 in Cystic Fibrosis, while NCT00264888 assessed its safety and efficacy in Duchenne Muscular Dystrophy. These trials enrolled a total of 43 participants across the United States and Israel.
PTC124 is also known as ataluren. The drug is being developed by PTC Therapeutics for the treatment of Duchenne Muscular Dystrophy and Cystic Fibrosis. It has completed Phase 2 trials, but it is not yet approved for these indications.