Adam JacksonLykos Therapeutics hit a major roadblock Friday in its bid to bring the first psychedelic-based therapy for post-traumatic stress disorder to market, as U.S. regulators rejected its application and called for additional research.
The Food and Drug Administration issued a complete response letter for Lykos’ new drug application for its midomafetamine capsules, an MDMA-based treatment for PTSD in adults. According to an afternoon news release on Friday, the agency requested another Phase 3 trial to further study the drug’s safety and efficacy.
In a statement, Lykos CEO Amy Emerson called the decision “deeply disappointing,” particularly for “the millions of Americans with PTSD, along with their loved ones, who have not seen any new treatment options in over two decades.”
Emerson added, “While conducting another Phase 3 study would take several years, we still maintain that many of the requests that had been previously discussed with the FDA and raised at the Advisory Committee meeting can be addressed with existing data, post-approval requirements or through reference to the scientific literature.”
The rejection comes just days before the FDA’s August 11 deadline to rule on the application. It marks a significant setback for Lykos, which had received Priority Review status in February and Breakthrough Therapy designation in 2017.
Midomafetamine, also known as MDMA, is being studied as a catalyst for psychotherapy in treating PTSD, a condition affecting millions Americans annually. Lykos has used the drug in combination with psychological intervention.
The FDA’s concerns echoed those raised during a contentious June 4 advisory committee meeting, where panelists overwhelmingly recommended against approval. Issues included questions about the treatment’s durability and potential bias from participants with prior MDMA use.
Lykos has maintained its data from two Phase 3 trials, in which 69% of participants no longer met the diagnostic criteria for PTSD after treatment, provide sufficient evidence of efficacy and durability in line with FDA guidance.
The setback also comes despite recent support from prominent researchers. Last month, a group of 23 medical doctors and researchers from institutions including Harvard Medical School and the University of California, Berkeley, urged the FDA to accept MDMA therapy for PTSD, citing an urgent need for more effective treatments.
The decision could have a broader impact on the psychedelic medicine industry. Several companies are developing treatments based on substances like psilocybin and ketamine for various mental health conditions.
Current PTSD treatments include trauma-focused talk therapy and two FDA-approved antidepressants, but many patients don’t respond adequately or quit treatment early.
Lykos plans to meet with the FDA to discuss recommendations and explore regulatory pathways for approval. The company did not provide a timeline for the additional study or potential resubmission.
