Lykos' MDMA PTSD Treatment Rejected by FDA Panel

The U.S. Food and Drug Administration (FDA) has declined the approval of midomafetamine (MDMA) for treating post-traumatic stress disorder (PTSD) following a panel recommendation. Lykos Therapeutics, based in the United States, had proposed an MDMA-assisted therapy that combines MDMA capsules with psychological intervention. This marked the first formal discussion by FDA advisers regarding a psychedelic drug for medical use, highlighting the agency's cautious approach.

MDMA, known for its empathogenic and entactogenic properties, has both stimulatory and hallucinogenic effects. It alters time perception, enhances sensory experiences, and provides an energizing sensation. Despite the promising aspects of MDMA, the FDA’s Psychopharmacologic Drugs Advisory Committee raised concerns over its effectiveness and safety profile.

In the initial vote, nine out of 11 experts concluded that current data does not validate midomafetamine’s efficacy for PTSD patients. A subsequent vote saw 10 out of 11 members expressing that the drug's benefits did not outweigh its risks. Although the committee's decision is not binding, it generally provides a strong indicator of the FDA's eventual ruling. The final decision is anticipated by August 11, aligning with the Prescription Drug User Fee Act (PDUFA) target action date.

Lykos Therapeutics’s CEO, Amy Emerson, expressed disappointment but emphasized the company’s commitment to collaborating with the FDA to resolve outstanding issues. Emerson highlighted the significant unmet needs in PTSD treatment, stressing the potential importance of MDMA-assisted therapy if approved.

At the meeting, Lykos presented data from two Phase III trials, MAPP1 (NCT03537014) and MAPP2 (NCT04077437), involving about 200 PTSD patients. These trials achieved their primary and secondary goals, showing improvements in PTSD symptoms and disability scores. The therapy involved administering MDMA in three eight-hour sessions conducted four weeks apart, compared against a placebo group.

While patient improvements were noted, the FDA raised questions about the trial design. Despite being double-blinded, which is considered the gold standard for placebo-controlled studies, the profound effects of MDMA made it apparent to patients which group they were in. This recognition potentially compromised the blinding integrity of the trials.

PTSD impacts roughly 5% of U.S. adults annually, equating to about 13 million individuals, according to the National Center for PTSD. Current treatments, including antidepressants and cognitive-behavioral therapy, often fall short, leaving patients with limited options. The pursuit of alternative therapies has thus gained momentum in recent years.

Interest in psychedelic drugs for medical purposes has surged, with GlobalData reporting a 500% increase in partnership deals within the pharmaceutical industry from 2019 to 2023. Recently, AbbVie joined this trend by partnering with Gilgamesh, a psychedelic specialist, in a deal potentially worth $1.95 billion. The collaboration aims to develop neuroplastogens, which induce rapid and sustained neuroplasticity with fewer side effects compared to existing psychedelic compounds.

The FDA's decision on Lykos Therapeutics's MDMA-assisted therapy remains pending, but the cautious stance reflects broader considerations of efficacy and safety in the emerging field of psychedelic medicine. The growing investment and interest in this area underscore the potential for significant advancements, provided that these therapies can meet stringent regulatory standards.