dm+d

Unassigned

New Medicines

Amyotrophic lateral sclerosis (motor neurone disease)

Information

New molecular entity
Biohaven Pharmaceuticals
Biohaven Pharmaceuticals

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials

Category

Irreversible inhibitor of myeloperoxidase that penetrates brain
MND is relatively uncommon with an annual incidence of about 2 cases per 100,000 population. Prevalence is about 5-7 per 100,000 [2].
Amyotrophic lateral sclerosis (motor neurone disease)
Oral

Multiple system atrophy

Information

New molecular entity
Biohaven Pharmaceuticals
Biohaven Pharmaceuticals

Development and Regulatory status

Discontinued
Discontinued
Discontinued
Nov 21Although the mechanism of action for verdiperstat, myeloperoxidase inhibition, was shown not to be effective for MSA, Biohaven maintains there remains a strong rationale of targeting brain inflammation in other disease states such as amyotrophic lateral sclerosis. MSA no longer listed in company pipeline; presume development discontinued [6,7].

Category

Irreversible inhibitor of myeloperoxidase (MPO) that penetrates brain
Multiple system atrophy (MSA) is a neurodegenerative disorder characterised by autonomic failure (cardiovascular and/or urinary), parkinsonism, cerebellar impairment and corticospinal signs with a median survival of 6-9 years. Prevalence ranges from 1 per 50,000-1 per 20,000. MSA-parkinsonian type (MSA-p) predominates in the Western Hemisphere and MSA-cerebellar type (MSA-c) predominates in the Eastern Hemisphere. Genders are equally distributed [1].
Multiple system atrophy
Oral

Trial or other data

Sep 21Company announces verdiperstat for Multiple System Atrophy did not meet the expected outcomes. No statistical difference was found for primary and secondary measures between verdiperstat vs placebo [5].