FDA to review Lykos' PTSD psychedelic therapy

The psychedelic drug midomafetamine (MDMA) is set to undergo regulatory scrutiny next month as the FDA convenes an advisory panel to evaluate its potential as a treatment for post-traumatic stress disorder (PTSD) when used in combination with psychological therapy. This significant meeting, scheduled for June 4, marks the first time in 25 years that the panel will review a new potential treatment for PTSD.

Developed by Lykos Therapeutics, formerly known as MAPS Public Benefit Corporation, the MDMA-assisted therapy has garnered substantial interest, especially after the company secured $100 million in an oversubscribed series A funding round earlier this year. The application for this therapy has been granted a priority review, with a target action date set for August 11.

The submission for approval is backed by data from the Phase III MAPP1 and MAPP2 trials, both of which achieved their primary and secondary endpoints. Notably, results from the MAPP2 trial, published in Nature in September, demonstrated that MDMA-assisted therapy significantly alleviated PTSD symptoms compared to a placebo, corroborating the findings from the MAPP1 trial. Importantly, no serious adverse events were reported in either trial.

Despite these promising results, there are concerns regarding the design of these trials. A draft evidence report by the Institute for Clinical and Economic Review (ICER) called attention to potential biases in the trials' design and the adequacy of blinding participants. The report highlighted "many uncertainties about the balance of benefits and harms," suggesting that these aspects require thorough examination.

One complicating factor in the FDA's assessment is the hybrid nature of MDMA-assisted therapy, which involves administering the drug in capsule form alongside psychotherapy. David Rind, the chief medical officer at ICER, pointed out that the FDA would need to address labelling issues to determine which patients with PTSD are suitable for this therapy. He emphasized the necessity of understanding the therapy's specifics to move forward, noting that the variety of therapy options used in the trials could pose challenges for widespread implementation.

The outcome of the FDA panel's review is anticipated to have significant implications for the burgeoning field of psychedelic medicine. Other companies are also exploring the therapeutic potential of psychedelics; for instance, Compass Pathways is expected to release data from a Phase III trial involving 800 patients treated with its investigational COMP360 psilocybin for treatment-resistant depression later this year. Similarly, Cybin is set to initiate a late-stage study of its psilocybin analogue, CYB003, aimed at treating major depressive disorder.

In summary, the upcoming FDA advisory panel meeting represents a critical juncture for MDMA-assisted therapy as a treatment for PTSD. While the therapy has shown substantial promise in clinical trials, concerns about trial design and the hybrid nature of the treatment must be carefully addressed. The panel's conclusions could influence not only the future of MDMA-assisted therapy but also the broader landscape of psychedelic medicine.