FDA denies Lykos MDMA PTSD therapy

On Friday, the FDA issued a complete response letter (CRL) to Lykos Therapeutics, concerning their midomafetamine capsules intended for treating PTSD. This communication arrived two days earlier than expected and outlined that the New Drug Application (NDA) could not be approved based on the current data. The FDA has requested that Lykos undertake an additional phase 3 clinical trial to further investigate the safety and efficacy of midomafetamine.

The concerns detailed in the CRL align closely with those discussed during the FDA's Psychopharmacologic Drugs Advisory Committee (AdComm) meeting in June. At that time, the committee voted against the approval of the treatment due to concerns about its efficacy (with a vote of 2-9 against) and its risk-benefit profile (1-10 against). This decision highlighted significant skepticism regarding the treatment's potential benefits compared to its risks.

There were, however, some issues raised about the composition and conduct of the AdComm meeting. Lykos Therapeutics pointed out that the panel included a limited number of subject matter experts. Additionally, the nature of the discussions reportedly extended beyond the scientific content presented in the briefing documents. Lykos has previously addressed these issues in a published response, noting that the clinical data might not have convincingly demonstrated the durability of the treatment's effects. They also pointed out concerns about expectancy bias, which seemed to be influenced primarily by participants with prior MDMA usage.

The approval of midomafetamine capsules would have marked a significant milestone in PTSD treatment. It would have been the first psychedelic-assisted therapy approved for this condition and the first new treatment for PTSD in over 20 years. The treatment protocol involves the use of MDMA capsules in conjunction with psychological interventions.

In light of the FDA’s decision, Lykos Therapeutics has announced its intention to request a meeting with the agency. The aim of this meeting would be to ask for a reconsideration of the decision and to discuss in detail the FDA's recommendations for resubmission. The company remains committed to addressing the concerns raised and to demonstrating the treatment's efficacy and safety through additional clinical trials as required by the FDA.