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Verdiperstat

Also known as BHV-3241, AZD3241

Bottom line

One placebo-controlled trial found verdiperstat did not slow ALS, help muscle strength or breathing. No further ALS trials have been announced.

Verdiperstat was a pill designed to calm a type of inflammation in the brain and spinal cord. It was tested in the HEALEY ALS Platform Trial, which runs several drugs side by side against a shared placebo group. In 2022 it showed no benefit on ALS progression, muscle strength or breathing, and Biohaven did not continue it in ALS.

Information checked 5 Nov 2026

Evidence: Not shown to work

Studies: 1 against

Controlled trials found no benefit.

No benefit vs placebo

About the same decline as placebo, 1.02 vs 1.05 points a month on the 48-point ALSFRS-R

How we decide the overall verdict: How to read this site.

At a glance

CompanyBiohavenExplanation and sources

Biohaven is a US biotech company focused on brain and nerve diseases. It provided verdiperstat to the HEALEY ALS Platform Trial, which is run by the Healey & AMG Center for ALS at Massachusetts General Hospital.

Sources

  1. Biohaven Provides Update From Pivotal Phase 2/3 Trial With Verdiperstat In ALS (HEALEY ALS Platform Trial)Biohaven (via PR Newswire) · 29 Sept 2022 · Company press release
  2. Verdiperstat in ALS - HEALEY ALS Platform Trial results (Regimen B)JAMA Neurology · 17 Feb 2025 · Peer-reviewed paper
How it worksBlocks an enzyme linked to inflammation and oxidative stressMyeloperoxidase (MPO) inhibitorExplanation and sources

Myeloperoxidase is an enzyme made by immune cells that produces harmful oxidising chemicals. In ALS, inflammation in the nervous system may speed up motor neuron damage. Verdiperstat blocks this enzyme in the hope of reducing that inflammation and oxidative stress.

Sources

  1. Verdiperstat in ALS - HEALEY ALS Platform Trial results (Regimen B)JAMA Neurology · 17 Feb 2025 · Peer-reviewed paper
  2. Biohaven Provides Update From Pivotal Phase 2/3 Trial With Verdiperstat In ALS (HEALEY ALS Platform Trial)Biohaven (via PR Newswire) · 29 Sept 2022 · Company press release
StageDiscontinuedFailed in HEALEY Platform Trial (Phase 2/3) in 2022Explanation and sources

Verdiperstat was Regimen B of the HEALEY ALS Platform Trial. Biohaven announced in September 2022 that it did not meet its main or key secondary goals. It had also failed a Phase 3 trial in multiple system atrophy, a different brain disease, in 2021.

Sources

  1. Biohaven Provides Update From Pivotal Phase 2/3 Trial With Verdiperstat In ALS (HEALEY ALS Platform Trial)Biohaven (via PR Newswire) · 29 Sept 2022 · Company press release
  2. Biohaven's verdiperstat fails in HEALEY ALS trialBioPharma Dive · News
What happenedMissed primary and key secondary goals in HEALEY Platform Trial29 Sept 2022Explanation and sources

Biohaven announced that verdiperstat did not slow ALS compared with placebo. The full peer-reviewed results in 2025 confirmed no effect, and no further ALS trials of verdiperstat have been announced.

Sources

  1. Biohaven Provides Update From Pivotal Phase 2/3 Trial With Verdiperstat In ALS (HEALEY ALS Platform Trial)Biohaven (via PR Newswire) · 29 Sept 2022 · Company press release
  2. Verdiperstat in ALS - HEALEY ALS Platform Trial results (Regimen B)JAMA Neurology · 17 Feb 2025 · Peer-reviewed paper
Best reported effectNo benefit vs placeboAbout the same decline as placebo, 1.02 vs 1.05 points a month on the 48-point ALSFRS-RExplanation and sources

In the published results (167 people given verdiperstat or its matched placebo), the estimated rate of decline was about the same as placebo (disease rate ratio 0.98, where 1.0 means no difference). Muscle strength and breathing, the key secondary goals, did not improve either. This is a clear negative result.

This is the most favourable result reported. See all key studies for the full picture.

Sources

  1. Verdiperstat in ALS - HEALEY ALS Platform Trial results (Regimen B)JAMA Neurology · 17 Feb 2025 · Peer-reviewed paper
  2. Biohaven Provides Update From Pivotal Phase 2/3 Trial With Verdiperstat In ALS (HEALEY ALS Platform Trial)Biohaven (via PR Newswire) · 29 Sept 2022 · Company press release
  3. Verdiperstat fails to differentiate from placebo in critical HEALEY ALS Platform TrialNeurologyLive · News
Who it's forAdults with ALSAll ALS · Oral, twice dailyExplanation and sources

The HEALEY trial enrolled adults with ALS at sites across the USA. People were randomly assigned to one of several drugs being tested, and then to the drug or placebo, taken by mouth twice a day for 24 weeks.

Sources

  1. HEALEY ALS Platform Trial - Regimen B VerdiperstatClinicalTrials.gov · Trial registry
  2. HEALEY platform trialMND Association · Patient organisation
Can I join?Not available; development in ALS endedExplanation and sources

Verdiperstat is not available and there are no ALS trials recruiting. The HEALEY Platform Trial continues to test other drugs.

Sources

  1. HEALEY ALS Platform Trial - Regimen B VerdiperstatClinicalTrials.gov · Trial registry
  2. HEALEY platform trialMND Association · Patient organisation

Timeline

4 events, newest first. Company updates are marked; independent news and regulator decisions are shown too.

2025

  1. PublicationJAMA Neurology

    Full results published in JAMA Neurology

    Peer-reviewed results confirmed verdiperstat did not slow ALS progression compared with placebo.

2022

  1. Trial registryClinicalTrials.gov

    Verdiperstat regimen completed

    The registry records completion of the 167-person verdiperstat regimen.

  2. Press releaseFrom the companyBiohaven

    Verdiperstat fails in HEALEY trial

    Biohaven reported that verdiperstat did not meet its main or key secondary goals in ALS.

2020

  1. Trial registryClinicalTrials.gov

    Verdiperstat arm of HEALEY Platform Trial starts

    Verdiperstat began testing as Regimen B of the HEALEY ALS Platform Trial, run from Massachusetts General Hospital at US sites.

All sources for this page

  1. Biohaven Provides Update From Pivotal Phase 2/3 Trial With Verdiperstat In ALS (HEALEY ALS Platform Trial)Biohaven (via PR Newswire) · 29 Sept 2022 · Company press release
  2. Verdiperstat in ALS - HEALEY ALS Platform Trial results (Regimen B)JAMA Neurology · 17 Feb 2025 · Peer-reviewed paper
  3. HEALEY ALS Platform Trial - Regimen B VerdiperstatClinicalTrials.gov · Trial registry
  4. Biohaven's verdiperstat fails in HEALEY ALS trialBioPharma Dive · News
  5. Verdiperstat fails to differentiate from placebo in critical HEALEY ALS Platform TrialNeurologyLive · News
  6. HEALEY platform trialMND Association · Patient organisation