Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Verdiperstat · 2 trials · 2 indications
UMSARS - clinician-rated scale comprised of 4 parts: Part I (Historical Review), Part II (Motor Examination), Part III (Autonomic Examination), Part IV (Global Disability Scale). Modified UMSARS is composed of subset of 9 items from original UMSARS Part I and Part II. Responses are measured on 4-point scale ranged from 0-3, where 0= no/mild impairment, 1= moderate impairment, 2= severe impairment, 3=complete impairment. Total modified UMSARS score is sum of these 9 items, score range from 0 to 27. Higher scores indicate greater impairment.
An adverse event (AE) is defined as any new untoward medical occurrence or worsening of a pre-existing medical condition in participants or clinical investigation participants administered an investigational (medicinal) product and that does not necessarily have a causal relationship with this treatment. A serious AE (SAE) is defined as any event that met any of the following criteria at any dose: death; life-threatening; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant disability/incapacity; congenital anomaly/birth defect in the offspring of a participant who received study drug; other important medical events that may not have resulted in death, be life-threatening, or required hospitalization, or, based upon appropriate medical judgment, they may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the other serious outcomes.
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 | Participants received verdiperstat 300 mg tablet orally once daily for 1 week, followed by 300 mg twice daily for 1 week, and then 600 mg twice daily for the remaining 46 weeks of the double-blind phase. Participants who completed the double-blind phase were offered the opportunity to enroll in an open-label extension (OLE) phase to continue verdiperstat 600 mg twice daily for 48 weeks. |
| Placebo | PLACEBO_COMPARATOR | Participants received placebo matching with verdiperstat for 48 weeks. Participants who completed the double-blind phase were offered the opportunity to enroll in an OLE phase to receive verdiperstat 600 mg tablet orally twice daily for 48 weeks. |
| Matching Placebo | PLACEBO_COMPARATOR | Matching placebo is administered twice daily p.o. for 24 weeks. |
| Name | Type | Description |
|---|---|---|
| Verdiperstat | DRUG | 300mg 2 oral tablets, twice daily |
| Placebo | DRUG | Matching placebo |
| Matching Placebo | DRUG | Drug: Matching Placebo Administration: Oral Dose: two tablets twice daily |
Inclusion Criteria: 1. Diagnosis of probable or possible MSA according to consensus clinical criteria (Gilman et al 2008), including participants with MSA of either subtype (MSA-P or MSA-C). 2. Able to ambulate without the assistance of another person, defined as the ability to take at least 10 ste...
Verdiperstat is an investigational small molecule drug being developed by Biohaven Ltd. for neurological conditions. It has been studied in amyotrophic lateral sclerosis (ALS) and multiple system atrophy (MSA). It is a myeloperoxidase (MPO) inhibitor and has been evaluated in placebo-controlled clinical trials.
Verdiperstat has been studied as a treatment for amyotrophic lateral sclerosis (ALS) and multiple system atrophy (MSA). It was tested in the HEALEY ALS Platform Trial and in a Phase 3 study in MSA. It remains investigational and is not approved for these indications.
Verdiperstat targets myeloperoxidase, also known as MPO. It is an MPO inhibitor, meaning it is designed to block the activity of this enzyme. Myeloperoxidase is an enzyme involved in inflammatory and oxidative processes, and inhibiting it is the mechanism under investigation in ALS and multiple system atrophy.
Verdiperstat is being developed by Biohaven Ltd., which trades under the ticker BHVN. Biohaven sponsored the clinical studies of Verdiperstat, including the Phase 3 trial in multiple system atrophy and the regimen tested within the HEALEY ALS Platform Trial.
Verdiperstat has completed a Phase 3 trial in multiple system atrophy and a Phase 2 regimen in the HEALEY ALS Platform Trial. It is an investigational drug and is not FDA approved. No active trials of Verdiperstat are currently listed.
Verdiperstat has been studied in two completed trials. NCT03952806 was a Phase 3 study of BHV-3241 in participants with multiple system atrophy, enrolling 421 participants across the United States and Europe. NCT04436510 was the Phase 2 HEALEY ALS Platform Trial Regimen B, which enrolled 167 participants with ALS in the United States.