Lykos Therapeutics’ groundbreaking MDMA-assisted therapy for post-traumatic stress disorder (PTSD) is encountering a significant regulatory hurdle as the US Food and Drug Administration (FDA) prepares to engage external experts for a thorough evaluation.
Scheduled for review by an FDA advisory committee next month, the treatment’s fate hinges on the committee’s vote regarding its potential approval, as confirmed by two individuals familiar with the matter.
This development holds profound implications for the emerging field of psychedelic-based mental health treatments, where several biotech firms are advancing late-stage trials for similar therapies.
The decision to convene the panel underscores the FDA’s cautious approach toward a new class of drugs historically associated with severe legal consequences. Combining MDMA with therapy to address mental health disorders represents a departure from traditional treatment methods.
An interim assessment conducted by the Institute for Clinical and Economic Review (ICER) on the two clinical trials linked to Lykos’ PTSD therapy highlighted concerns regarding trial design and execution. The influential non-profit organization pointed out numerous uncertainties regarding the treatment’s overall risk-benefit profile.
According to findings from Lykos’ second late-stage study, where patients underwent MDMA-assisted psychotherapy, 71 percent of those in the MDMA group exhibited significant improvement, no longer meeting the diagnostic criteria for PTSD. This compared with 48 percent in the placebo group.
However, the ICER raised apprehensions about the inherent challenges of “double-blinding” patients in studies involving psychedelic drugs, given the unmistakable hallucinatory effects of the medication. Ensuring that neither patients nor investigators were aware of who received the treatment posed significant challenges.
Upon inquiry from the Financial Times, Amy Emerson, Lykos’ CEO, expressed the company’s anticipation of discussing its treatment application during the FDA panel review.
Insiders at Lykos indicated their expectation of an advisory committee review and remained hopeful that this process would not impede the anticipated approval date of August 11 for the treatment. While the panel’s votes are not binding, they typically carry weight with the FDA.
The advisory panel is expected to provide recommendations on which patient demographics should have access to the treatment. Notably, the FDA is handling this decision with care, as it rarely evaluates treatments combining psychotherapy and drugs.
David Rind, ICER’s Chief Medical Officer, emphasized the importance of understanding the therapy’s nuances before advancing. He highlighted the complexity of identifying suitable patients for the treatment amidst various therapy options explored during the trials.
The burgeoning psychedelic drug sector has attracted substantial investments in recent years. Lykos, spun out from the Multidisciplinary Association for Psychedelic Studies, secured $100 million in funding earlier this year from notable investors, including the charitable foundation of hedge fund billionaire Steven Cohen and his spouse Alexandra.
Should the FDA approve the treatment, the Drug Enforcement Administration (DEA) will have 90 days to reconsider the classification of MDMA, potentially reclassifying it to allow broader medical usage beyond the current restrictive schedule-1 categorization.
Meanwhile, London-based Compass Pathways is poised to release data later this year from a phase-three trial involving 800 subjects treated with synthetic psilocybin for depression resistant to conventional therapies. Nasdaq-listed Cybin is also commencing late-stage trials examining a psilocybin analogue’s efficacy in treating major depressive disorder.
The FDA declined to comment on pending product applications or approvals, maintaining its standard policy.
