Recent Updates
Recently added Catalysts

Verdiperstat

Phase 3

Multiple System Atrophy | Small molecule | Neurology |Biohaven Ltd.|Last Updated: Sep 29, 2023

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment421

FDA Designations

No designations recorded

Clinical trial landscape

Verdiperstat · 1 trial · 1 indication

Phase 3 1
NCT03952806Study of BHV-3241 in Participants With Multiple System AtrophyMultiple System Atrophy
COMPLETED421 Analytics
PHASE3COMPLETED
Study of BHV-3241 in Participants With Multiple System Atrophy
Multiple System AtrophyUnlock trial analytics

Study Endpoints

Primary Endpoints

Change From Baseline in the Modified UMSARS Score at Week 48
Baseline and Week 48

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.

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
Up to 100 weeks

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.

Secondary Endpoints

Clinical Global Impression of Improvement (CGI-I) Score at Week 48
Week 48
Change From Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Motor Subscale at Week 48
Baseline and Week 48
Change From Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Non-motor Subscale at Week 48
Baseline and Week 48
Unlock Study Endpoints

Study Design & Arms

AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
VerdiperstatEXPERIMENTALParticipants 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.
PlaceboPLACEBO_COMPARATORParticipants 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.

Interventions

NameTypeDescription
VerdiperstatDRUG300mg 2 oral tablets, twice daily
PlaceboDRUGMatching placebo
Unlock Study Design Details

Eligibility Criteria

Age Range40 Years to 80 Years
SexALL
Healthy VolunteersNo
Study Sites48

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...

Countries:United StatesAustriaFranceGermanyItalyUnited Kingdom
Unlock Eligibility Criteria

Frequently asked questions about Verdiperstat

What is Verdiperstat used for?

Verdiperstat is an investigational small molecule being developed for the treatment of Multiple System Atrophy (MSA), a rare neurodegenerative disorder. It is currently in Phase 3 clinical development and is not yet approved by regulatory authorities.

What does Verdiperstat target?

Verdiperstat targets myeloperoxidase, an enzyme involved in oxidative stress and inflammation. By inhibiting this enzyme, it aims to reduce neuronal damage in patients with Multiple System Atrophy. This mechanism is being evaluated in clinical trials to assess its potential therapeutic benefit.

Who makes Verdiperstat?

Verdiperstat is being developed by Biohaven Ltd., a biopharmaceutical company focused on neuroscience. The company is listed on the stock exchange under the ticker symbol BHVN. Biohaven is conducting clinical trials to evaluate the drug's safety and efficacy in Multiple System Atrophy.

What phase is Verdiperstat in?

Verdiperstat is in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory agency. The Phase 3 trial has been completed, and the results will determine whether further development or regulatory submission is pursued.

What clinical trials is Verdiperstat in?

Verdiperstat has been studied in one Phase 3 clinical trial with the identifier NCT03952806. This randomized, double-blind, placebo-controlled study enrolled 421 participants with Multiple System Atrophy across multiple countries, including the United States, Austria, France, Germany, Italy, and the United Kingdom. The trial has been completed.

Is Verdiperstat the same as BHV-3241?

Yes, Verdiperstat is also known as BHV-3241. The clinical trial NCT03952806, which evaluated the drug in Multiple System Atrophy, used the name BHV-3241 in its title. Both names refer to the same investigational compound being developed by Biohaven Ltd.