Trial of psilocybin microdose therapy in fragile X cleared in Canada

KGK Science will launch a Phase 2a trial in the coming months to test NM-1001, a psilocybin microdose therapy — meaning one that contains low doses of the hallucinogenic compound — in people with fragile X syndrome.

The company has received a needed exemption from the Canadian government to conduct the trial, expected to begin sometime early this year. In late December, KGK received approval from Health Canada to start the study, after requesting clearance earlier that month.

The trial will enroll up to 10 people with fragile X at KGK’s dedicated research facility in Ontario. All participants will be treated with NM-1001.

“KGK Science is thrilled to have received all of the regulatory approvals required to conduct this cutting-edge clinical trial,” Najla Guthrie, CEO of KGK, said in a company press release.

KGK, a wholly owned subsidiary of Wellbeing Digital Sciences, will be conducting the trial in partnership with NM-1001’s developer, Nova Mentis Life Science, as per an agreement between the two companies that was announced last year.

“Securing this latest regulatory approval from the Canadian government is a critical step in Nova Mentis’ research and drug development program and allows us to begin our groundbreaking clinical trial,” said William Rascan, president and CEO of Nova Mentis.

“We are eager to begin recruiting participants as we seek to better understand the therapeutic potential of psilocybin in the treatment of fragile X syndrome,” Rascan added.

Nova Mentis previously announced that it had completed the manufacturing of a large supply of 1.5-mg psilocybin microdose capsules — a dosage of 1.5 mg — to use in the study. The company also secured regulatory permissions to ship the medication to Canada from the U.S.

We are eager to begin recruiting participants as we seek to better understand the therapeutic potential of psilocybin in the treatment of fragile X syndrome.

Psilocybin is the main psychoactive compound found in so-called magic mushrooms, and is responsible for the hallucination-inducing effects of these fungi. NM-1001 contains microdoses of psilocybin — meaning the dose is low enough that it’s not expected to induce hallucinations or other psychedelic effects.

The low dose is instead expected to have subtler effects on brain chemistry, which may help to ease common symptoms of fragile X like unstable or depressive mood problems, according to Nova.

Experiments done by the company in animal models of fragile X have suggested that the psilocybin microdose can improve memory and normalize behavior.

Nova’s formulation of psilocybin has been granted orphan drug designation in the U.S. and Europe for the treatment of fragile X.

This status is meant to provide several incentives intended to speed the therapy’s clinical development and regulatory review. Among them are assistance with trial protocols, reduced regulatory fees, and a market exclusivity period, if approved, of seven years in the U.S. and 10 years in Europe.

“We believe that this study will be an impactful assessment of the potential of psilocybin in a disorder that truly affects the lives of many families and that has not yet been studied,” Guthrie said, adding, “We look forward to working with Nova Mentis and contributing to the science.”