Biohaven’s latest failure is far from systemic

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Biohaven investors yesterday shrugged off the failure of verdiperstat in multiple system atrophy. But a look at the pipeline shows that there are not many other projects in development for the disease, which shares some similarities with Parkinson’s. Indeed, it is notable that several efforts are being spearheaded by academic centres, some involving drugs approved for other uses. Earlier-stage projects from the likes of Biogen/Ionis and Bayer look more interesting, as do assets from Modag and Alterity targeting alpha-synuclein; however, questions remain about this target after recent setbacks with similarly acting agents in Parkinson’s. For now, Modag's Anle138b is only in the clinic for Parkinson’s, although the private group also has plans in MSA. As for verdiperstat, that project will have another chance soon, with the academic Healey platform trial in amyotrophic lateral sclerosis set to complete enrolment this year. Leerink analysts believe that verdiperstat, which targets neuroinflammation, might still have legs in ALS and other neurodegenerative diseases, but hopes cannot now be high. Astrazeneca’s decision to offload the asset after disappointing phase 2 data looks wise. Still, Biohaven investors seem more focused on the group’s migraine franchise, which is off to a good launch.

Selected projects in development for multiple system atrophy
Project Decsription Trial sponsor Status
Verdiperstat Myeloperoxidase inhibitor Biohaven Failed ph3 M-Star in Sep 2021
Tllsh2910 NMDA modulator National Taiwan University Hospital Ph3 completes Nov 2022
Rituximab Anti-CD20 MAb (sold by Roche as Rituxan) First Affiliated Hospital of Fujian Medical University Ph2 completed Jul 2021
Exenatide  GLP1 agonist (sold by Astrazeneca as Bydureon) University College, London Ph2 completes Apr 2022
Mesenchymal stem cells Intrathecal autologous MSC therapy  Mayo Clinic, FDA, NINDS Ph1/2 completes Mar 2024
ATH434 (PBT434) Tau & alpha-synuclein aggregation inhibitor Alterity Therapeutics Ph1 in Australia/New Zealand completed; ph2 to start by YE 2021
Anle138b Small molecule binding toxic alpha-synuclein Modag Ph1 in Parkinson's completes Jun 2021
BIIB101 (ION464) Alpha-synuclein antisense Biogen/Ionis Ph1 Horizon completes Jul 2022
AAV2-GDNF GDNF gene therapy Bayer (via Askbio/Brain Neurotherapy Bio) Ph1 completes Nov 2022
GDNF=glial cell line-derived neurotrophic factor; NINDS=National Institute of Neurological Disorders and Stroke. Source: Evaluate Pharma, clinicaltrials.gov & Leerink note Sep 14, 2021.

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