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The first mental health treatment using schedule-1 drug MDMA faces a significant regulatory hurdle as the US Food and Drug Administration is poised to ask outside experts to scrutinise the treatment for post-traumatic stress disorder.
Lykos Therapeutics’ MDMA-assisted therapy for PTSD is set to be reviewed by an FDA advisory committee in the coming months, which would vote on whether the controversial treatment should be approved, according to two people familiar with the matter.
The decision to convene the panel, which could be announced as early as this week, shows the high degree of caution with which the FDA is approaching a new class of drugs that have historically come with harsh criminal penalties in combination with therapy to treat mental health disorders.
An interim review of the two clinical studies associated with Lykos’ PTSD treatment by the Institute for Clinical and Economic Review raised concerns about the design and conduct of the trials. The influential non-profit group said they raised “many uncertainties about the balance of benefits and harms” of the treatment.
By the conclusion of Lykos’ second late-stage study into PTSD treatment, during which patients take MDMA while receiving psychotherapy, 71 per cent of people in the MDMA group improved sufficiently to no longer meet the criteria for PTSD diagnosis, compared with 48 per cent in the placebo group.
But the ICER expressed concern about the challenges inherent with “double-blinding” patients in a study of a psychedelic drug, saying it was nearly impossible to ensure that neither patients nor investigators knew who was undergoing the treatment because of the medicine’s obvious hallucinatory effects.
People close to Lykos said they had been expecting an advisory committee to be called and were still hopeful that the decision to convene the panel would not delay the expected August 11 approval date for the treatment. The results of the panel’s votes are not binding but are typically followed by the agency.
The advisory panel is likely to opine on which specific patients should be able to access the treatment. The FDA was also handling the decision with caution as it rarely reviews treatments involving psychotherapy as well as drugs, the people said.
“Certainly, there’s a labelling issue there that the FDA will be interested in: who has PTSD that matches what the therapy is approved for,” said David Rind, ICER’s chief medical officer. “They need to really understand what the therapy is . . . to move forward with this as there were many therapy options in these trials, and I actually think that causes problems for how you disseminate this.”
The approval decision over Lykos has big implications for the nascent category of psychedelic-based treatments for mental health problems — as a number of other biotechs conduct late-stage trials into psychedelic-based mental health treatments.
The psychedelic drug sector has garnered billions of dollars of investment in recent years. Lykos was spun out from the Multidisciplinary Association for Psychedelic Studies, a non-profit group which has been a forthright advocate for psychedelic drugs since it was founded by Rick Doblin in 1986.
Earlier this year, Lykos raised $100mn in a funding round from 10 investors including the charitable foundation run by hedge fund billionaire Steven Cohen and his wife Alexandra.
If the drug is approved by the FDA, the Drug Enforcement Administration will be given 90 days to reclassify MDMA as a less harmful drug as the schedule-1 category restricts the use of drugs for medical purposes.
London-based Compass Pathways is expected to publish data later this year from a phase-three trial of 800 human subjects who have taken synthetic psilocybin for treatment-resistant depression, while Nasdaq-listed Cybin will also initiate its late-stage trial looking at a psilocybin analogue as a treatment for major depressive disorder.
Lykos did not immediately respond to multiple requests for comment. The FDA said the agency “cannot comment on possible or pending product applications or approvals”.
