Nova Mentis is the latest biotech to enter the psychedelic space, initiating a Phase IIa trial testing psilocybin in fragile X syndrome.

Canada-based Nova Mentis is now recruiting patients for a 28-day study testing oral microdoses of psilocybin in 10 patients with fragile X syndrome, the leading genetic cause of autism spectrum disorder. The study will take place in a facility of the CRO KGK Sciences in London, Ontario, Canada.

The Phase IIa study will test the safety and efficacy of the psilocybin formulation NM-1001 administered every other day for 28 days. Nova Mentis will explore the effects of NM-1001 on behavioural symptoms using diagnostic tests based on mRNA and serotonin biomarker technology, combined with artificial intelligence (AI).

The Phase IIa trial design has approval from Health Canada, a regulatory body that has shown willingness to approve studies testing psychedelic therapies. Nova Mentis and KGK plan to treat the first patient in Q2 2023 and expect preliminary data later this year.

Psilocybin in fragile X syndrome

Fragile X syndrome causes mutations in the gene Fragile X Messenger Ribonucleoprotein 1 (FMR1), leading to a life-long disorder associated with a spectrum of developmental, intellectual, and behavioural challenges. There are no cures for fragile X syndrome, though are many treatments available for symptoms and a robust drug development pipeline.

Psilocybin, which is the main psychoactive ingredient found in psychedelic fungi known as magic mushrooms, is a type of serotonin agonist. The Nova Mentis website states that psilocybin could mimic serotonergic signalling to help compensate for low serotonin levels common in development disorders like fragile X syndrome and autism spectrum disorder.

Nova Mentis’s Phase IIa trial will use oral microdoses of psilocybin that are approximately one-tenth of a normal dose. Shifting regulatory and clinical perspectives have led to a recent surge in clinical trials testing psychedelics, though most data is from early-stage clinical trials.