Pictured: A prescription bottle over a groovy neuro background/ Nicole Bean for BioSpaceOn Tuesday, an FDA advisory committee will meet to discuss the relative merits of Lykos Therapeutics’ psychedelic-assisted therapy for post-traumatic stress disorder—the first therapeutic for PTSD to undergo FDA scrutiny in this century.The recommendation of the FDA’s Psychopharmacologic Drugs Advisory Committee is non-binding and will be considered by the FDA on August 11, when it completes its New Drug Application evaluation.An approval of MDMA (3,4-methylenedioxymethamphetamine)— perhaps better known as ecstasy—to treat PTSD is expected to send major vibrations through the mental health community. The repercussions will affect not only treatment but research and are likely to spill over to other emerging psychedelic-assisted therapies.“MDMA was developed in 1912 but didn’t really start to gain use in psychiatric practice until the 1970s and 1980s,” Sam Clark, CEO of Terran Biosciences, which is also developing psychedelic therapies, told BioSpace. “Then [in 1985] it was placed on a list of Schedule I drugs,” defined by the Drug Enforcement Administration as “drugs with no currently accepted medical use and the high potential for abuse.”Research all but stopped until the 1990s when the FDA approved a clinical trial for pain relief in terminal patients. Lykos, then called MAPS Public Benefit Company, began investigating MDMA and “pushed it forward with a number of groundbreaking studies,” Clark said.After completing Lykos’ Phase III trial, he said, “Around 70% of the patients no longer met the criteria for PTSD. That’s a big impact for patients!” Independent studies reported meaningful benefits and durations of up to 12 months.“Given the data, I think [FDA approval] is highly likely,” Doug Drysdale, CEO of Cybin, another player in the psychedelics space, told BioSpace.A High Unmet NeedThe impact is so significant that the Veterans Administration is funding psychedelic studies throughout the U.S. for the first time since the 1960s

Pictured: Concept art showing a broken head figure/iStock, designer491Due to concerns of study bias, the Institute for Clinical and Economic Review on Tuesday said in a draft report that it is unable to accurately assess the net benefit of Lykos Therapeutics’ investigational MDMA-assisted therapy in post-traumatic stress disorder.The drug price watchdog said that investigators, study therapists and patients held “very strong prior beliefs” about MDMA, which could have affected the recording of harms and benefits of the investigational treatment. The trial therapists and some of its participants were “pulled heavily” from a community interested in the use of psychedelics for psychological benefits, according to the Institute for Clinical and Economic Review (ICER) draft report.These sentiments about psychedelics “lead the community to engage with them more like a religious movement than pharmaceutical products,” ICER’s draft report concluded, noting that these feelings were not only common among participants but were also sometimes “inculcated in patients participating in the trials.”ICER said it also had been informed of “pressures” to keep the result of Lykos’ trials “favorable.” Some participants felt that they were pushed to report only the good outcomes while suppressing bad results. ICER cited first-hand and second-hand accounts of “extremely severe negative outcomes” which some trial therapists said were proof of appropriate treatment response.“Some patients were prevented from entering the long-term follow-up study and felt this was done to keep these negative outcomes out of the data set,” according to ICER.The watchdog also pointed out that Lykos’ trials were “essentially, unblinded” because the psychedelic effects of MDMA allowed the patients to determine if they were given the active treatment or the control. The MDMA treatment was correctly identified by “nearly all patients” who received it.In response to ICER’s findings, Lykos said in a public comment folio that it was “disappointed” by the draft report, noting that the watchdog “discounts the significant unmet medical need for patients living with” post-traumatic stress disorder (PTSD).ICER “relies on a limited number of stakeholder perspectives, and focuses on areas squarely within the FDA’s purview on the evaluation of safety and efficacy of drugs,” according to Lykos.ICER’s draft report comes as Lykos gears up for an FDA Psychopharmacologic Drugs Advisory Committee meeting set for June 4, 2024. The panel of external experts will discuss Lykos’ application to use the psychedelic compound MDMA as a treatment for adults with PTSD, when used in conjunction with psychotherapy and other supportive mental health interventions.Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at [email protected] or [email protected]

Pictured: Gene therapy concept: iStock, Ilya Lukichev2023 was a breakthrough year for cell and gene therapies, with seven FDA approvals in the U.S. and one in the European Union, according to the Alliance for Regenerative Medicine. Looking to 2024, the Washington, D.C.–based advocacy organization predicted on Monday in its annual Cell Gene State of the Industry Briefing that the sector could see up to 17 approvals in the U.S. and EU.Regulators in the U.S. gave the greenlight in 2023 to five gene therapies for rare genetic diseases and in 2024 could see an additional five approved by the FDA, as well as the first-ever approval of adoptive cell therapy for solid tumors and the first U.S. approval of an allogeneic T-cell therapy.Other notable milestones expected in 2024 include additional therapies to treat hemophilia A and hemophilia B and dystrophic epidermolysis bullosa