Following FDA’s refusal to approve Lykos Therapeutics’ MDMA-assisted therapy for PTSD New Drug Application (NDA), a key question is what the company might do next.
In its Complete Response Level (CRL), the agency requested that Lykos conduct a fresh Phase 3 study in order to clarify the safety and efficacy of the intervention. Questions of whether the company would be able to raise that money aside, many have asked what the FDA might expect that Phase 3 study to look like, especially given the fact that Lykos’ prior Phase 3 program was supposedly designed in collaboration with—and signed off by, via a Special Protocol Assessment (SPA)—the FDA.
Might the agency be comfortable with a Phase 3 trial that’s very similar in design to the existing two trials, but conducted to a higher standard (e.g., systematic reporting of positive drug effects, a more standardised long-term follow-up study, and so on)? Or, might the agency insist on new study design elements entirely, reflecting both the shortcomings in Lykos’ submitted NDA as well as learnings from the agency’s own journey over the past half-dozen or so years?
It’s likely to be the latter, we understand. A source familiar with the high-level recommendations in the FDA’s response told Psychedelic Alpha that the FDA has outlined at least half a dozen study design elements that it expects to see in any future Phase 3 study from Lykos.
In a two-part series, we look at each of these in turn. This first part looks at active placebos and evidence-based assisted-psychotherapy.