Psilocybin Therapy for Treatment-Resistant Depression is Being Taken Seriously by Regulators as FDA Gives Go-Ahead to Clinical Trial

Psilocybin, the psychoactive compound in hallucinogenic “magic mushrooms,” is emerging as a potential treatment for mental health disorders, however researchers have limited data on the safety and effectiveness of the drug for this indication. Now, London, England-based life sciences company COMPASS Pathways has announced it will be initiating a clinical trial of psilocybin in patients with treatment-resistant depression.

“We are excited to be starting this landmark trial which has the potential to transform lives,” said George Goldsmith, Chairman and Co-founder of COMPASS Pathways. “Depression is the leading cause of ill-health and disability worldwide, and treatment-resistant depression affects more than 100 million people. It is a huge unmet need and the trial will teach us more about how this new approach might address it.”

This week, the US Food and Drug Administration (FDA) approved COMPASS Pathways’ application to conduct a Phase IIb trial, with the company already getting the go-ahead from regulators in Canada, the UK and the Netherlands. According to COMPASS Pathways, their dose-ranging study has an enrollment target of about 216 patients diagnosed with treatment-resistant depression who will participate in the trial hosted at up to 15 international research sites.

COMPASS Pathways is set to start the trial before the end of August, with sites in London, Manchester and Newcastle expected to begin the study. While the company already has sites in Finland, Norway and the Netherlands lined up, the study won’t begin there, or in other countries, for the next few months.

“The design of this study has been a truly collaborative effort, with scientists, clinicians, patient representatives and regulators from Europe and North America working together with the goal of helping patients suffering with treatment-resistant depression,” said Ekaterina Malievskaia, Chief Medical Officer and Co-founder of COMPASS.

According to COMPASS Pathways, multiple academic studies conducted in the US and the UK have implicated psilocybin as a potentially safe and effective treatment for patients suffering from treatment-resistant depression. However, the psilocybin therapy tested in the upcoming trial will involve more than just the psychoactive drug; the study investigators plan to pair psilocybin with psychological support to determine its effect on depression.

If all goes to plan, COMPASS Pathways could be following this trial with a Phase III study of psilocybin therapy. There are four studies investigating the effects of psilocybin on depression currently listed on ClinicalTrials.gov, two of which are actively recruiting. The studies – which are being conducted at educational institutions, including Imperial College London, Yale University and Johns Hopkins University – are largely investigating psilocybin use in patients with major depressive disorder, potentially making COMPASS Pathways the only company currently studying the psychoactive drug in treatment-resistant depression.