Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
PTC518 · 1 trial · 1 indication
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. A TEAE was defined as an AE that had an onset date or date of worsening on or after the first dose of study drug. A summary of other non-serious AEs and all serious adverse events (SAEs), regardless of causality is located in the 'Reported AE section'.
Least square (LS) mean and 95% confidence interval (CI) were calculated using mixed-model repeated measures (MMRM). The blood tHTT protein value was log-transformed before fitting the MMRM model. The LS means from MMRM model were back-transformed to present geometric mean percent changes.
| Arm | Type | Description |
|---|---|---|
| PTC518 5 mg | EXPERIMENTAL | Participants will receive PTC518 5 milligrams (mg) tablets once daily orally for 12 months. |
| PTC518 10 mg | EXPERIMENTAL | Participants will receive PTC518 10 mg tablets once daily orally for 12 months. |
| PTC518 20 mg | EXPERIMENTAL | Participants will receive PTC518 20 mg tablets once daily orally for 12 months. |
| Placebo | PLACEBO_COMPARATOR | Participants will receive placebo matching to PTC518 tablets once daily orally for 12 months. |
| Name | Type | Description |
|---|---|---|
| PTC518 | DRUG | PTC518 will be administered per dose and schedule specified in the arm. |
| Placebo | DRUG | Placebo matching to PTC518 will be administered per schedule specified in the arm. |
Key Inclusion Criteria: * Genetically confirmed HD diagnosis with a cytosine-adenine-guanine (CAG) repeat length from 40 to 50, inclusive Eligibility for HD-ISS Stage 2 Group (Parts A, B, and C): * A Unified Huntington's Disease Rating Scale (UHDRS)-Independence Scale (IS) score of 100 * A UHDRS ...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novartis AG Sponsored ADR | NVS | 2 | PHASE3 | Votoplam |
| Neurocrine Biosciences, Inc. | NBIX | 1 | PHASE3 | Valbenazine |
| Hoffmann-La Roche | RHHBY | 3 | PHASE2 | Tominersen, RG6496, SPK-10001 |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE1 | ALN-HTT02 |
| uniQure N.V. | QURE | 2 | PHASE1 | AMT-130 |
| Sarepta Therapeutics, Inc. | SRPT | 1 | PHASE1 | SRP-1005 |
PTC518 is an investigational oral small molecule being developed for the treatment of Huntington disease, a progressive inherited neurological disorder. It is designed to reduce production of the mutant huntingtin protein that causes the disease. PTC518 has completed a Phase 2 clinical trial in adults with Huntington disease and is not approved for any indication.
PTC518 targets huntingtin, or HTT, the protein encoded by the gene mutated in Huntington disease. By acting on HTT, the small molecule is intended to lower levels of the disease-causing protein. HTT is a protein target, and PTC518 is an orally administered small molecule developed by PTC Therapeutics.
PTC518 is being developed by PTC Therapeutics, Inc., a biopharmaceutical company that trades on the Nasdaq under the ticker symbol PTCT. PTC Therapeutics is the sponsor of the clinical program evaluating PTC518 in Huntington disease.
PTC518 is in Phase 2 development for Huntington disease. A Phase 2 trial has been completed, and the program has received Fast Track designation from the U.S. Food and Drug Administration. PTC518 is investigational and has not been approved by the FDA for any indication.
PTC518 has been studied in one Phase 2 clinical trial, NCT05358717, titled A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease. The trial was randomized, double-blind, placebo-controlled, and enrolled 159 participants across the United States, Australia, Austria, Canada, France, Germany, Italy, Netherlands, New Zealand, Spain, and the United Kingdom. It is completed.