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 |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE1 | ALN-HTT02 |
| uniQure N.V. | QURE | 2 | PHASE1 | intra-striatal rAAV5-miHTT |
| Sarepta Therapeutics, Inc. | SRPT | 1 | PHASE1 | SRP-1005 |
PTC518 is an investigational small molecule being developed for Huntington's disease, a neurodegenerative disorder. It is currently in Phase 2 clinical development and has not been approved by the FDA. The drug is being studied to evaluate its safety and efficacy in participants with Huntington's disease.
PTC518 is a small molecule designed to target the underlying cause of Huntington's disease by modulating the expression of the huntingtin protein. It is being developed to potentially reduce the levels of this protein, which is central to the disease's pathology. The drug is in Phase 2 clinical trials.
PTC518 is being developed by PTC Therapeutics, Inc., a biopharmaceutical company listed on NASDAQ under the ticker symbol PTCT. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Huntington's disease.
PTC518 is in Phase 2 clinical development. It has completed one Phase 2 trial (NCT05358717) with 159 participants. The drug is not FDA approved and remains investigational. It has received Fast Track designation from the FDA for the treatment of Huntington's disease.
PTC518 has been studied in a Phase 2 clinical trial (NCT05358717) titled 'A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)'. This randomized, double-blind, placebo-controlled trial enrolled 159 participants across multiple countries including the United States, Australia, and several European nations.
PTC518 is a distinct investigational small molecule being developed by PTC Therapeutics for Huntington's disease. It is not known to be the same as any other approved or investigational treatment. Its mechanism involves targeting the huntingtin protein, which differentiates it from other therapies in development.