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Adjusted LOA
ML Risk
Verdiperstat · 1 trial · 1 indication
Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score using a Bayesian repeated measures model that accounts for loss to follow-up due to mortality. Each of 12 questions assessing distinct functional ability is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times.
| Arm | Type | Description |
|---|---|---|
| Verdiperstat | EXPERIMENTAL | Verdiperstat is administered twice daily p.o. for 24 weeks. |
| Matching Placebo | PLACEBO_COMPARATOR | Matching placebo is administered twice daily p.o. for 24 weeks. |
| Name | Type | Description |
|---|---|---|
| Matching Placebo | DRUG | Drug: Matching Placebo Administration: Oral Dose: two tablets twice daily |
| Verdiperstat | DRUG | Drug: Verdiperstat Administration: Oral Dose: 600mg twice daily |
Inclusion Criteria: * No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683). Exclusion Criteria: * The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683). 1. Parti...
Verdiperstat is an investigational small molecule being studied for the treatment of Amyotrophic Lateral Sclerosis (ALS). It is a -stat enzyme inhibitor, though the specific molecular target has not been disclosed. The drug is currently in Phase 2 clinical development and is not yet approved by regulatory authorities.
Verdiperstat is being developed by Merit Medical Systems, Inc., a company traded on the Nasdaq under the ticker symbol MMSI. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Amyotrophic Lateral Sclerosis.
Verdiperstat is in Phase 2 clinical development. It has completed one Phase 2 trial, the HEALEY ALS Platform Trial, which enrolled 167 participants. The drug remains investigational and has not received FDA approval for any indication.
Verdiperstat has been studied in one completed clinical trial, NCT04436510, titled "HEALEY ALS Platform Trial - Regimen B Verdiperstat." This Phase 2, randomized, double-blind, placebo-controlled trial enrolled 167 participants with Amyotrophic Lateral Sclerosis in the United States.
No, Verdiperstat is not FDA approved. It is an investigational drug currently in Phase 2 clinical development for Amyotrophic Lateral Sclerosis. The completed trial NCT04436510 was a randomized, double-blind, placebo-controlled study, but the drug has not yet received regulatory approval.