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Lykos Statement Regarding ICER Final Evidence Report

Earlier today, the Institute for Clinical and Economic Review (ICER) shared its Final Evidence Report on Midomafetamine-Assisted Psychotherapy for Post-Traumatic Stress Disorder (PTSD).1 Lykos Therapeutics is disappointed in the contents and overall findings of this report. ICER’s focus and expertise is in the value assessment and cost-effectiveness evaluation of potential new treatments, and Lykos is encouraged by ICER’s exploratory finding that – based on the available data – midomafetamine (MDMA) assisted therapy may be a cost-effective treatment for PTSD (if approved). However, like the interim report before it, the final report unfortunately goes beyond ICER’s traditional scope. It cites trial design concerns that, while important, have been and are being extensively discussed with FDA through the course of Lykos’ development program and New Drug Application (NDA) review. Further, it draws on select anonymous reports, without adequate and proper documentation, and uses these to cast unfair doubt on the integrity of Lykos, dozens of dedicated clinicians and researchers, and data from hundreds of clinical trial participants. It does so without adequate regard for the high unmet need for new treatments for PTSD and the unique complexity of developing treatments for this patient population.

Lykos has completed two multi-site, randomized, double-blinded, placebo-controlled, Phase 3 clinical trials, both of which met their primary and secondary endpoints. Participants in the midomafetamine group showed clinically meaningful improvement in their symptoms relative to participants in the control group.2,3,4 Approximately 40% of Phase 3 participants treated with midomafetamine-assisted therapy achieved remission at long-term follow-up (as measured by CAPS-5).2 The adverse events experienced were as expected for midomafetamine and were mostly mild and self-limited.2 Yet ICER diminishes these findings based on a set of common and known issues (including functional unblinding and expectancy bias) discussed with FDA at the time of trial design.

ICER has also focused on a deeply unfortunate case of therapist sexual misconduct stemming from a 2015 Phase 2 clinical trial. Lykos reported that incident in a timely manner to the appropriate authorities and banned the therapists from working with Lykos in any capacity. The company is committed to working with regulators, licensing boards, centers of care, and other stakeholders to ensure robust systems are in place to help protect patients and mitigate the risk of misconduct of any kind. If midomafetamine is approved, Lykos will continue to work with the FDA on a post-approval plan to address the full range of identified risks.

Ultimately, this final report relies on subjective analysis in a manner that seems misaligned to the data-driven approach ICER is meant to provide, and in doing so creates a concerning precedent that could negatively impact innovative treatments. The company has requested time to discuss its concerns with ICER and looks forward to a constructive dialogue.

In the meantime, Lykos is focused on continuing to advance improved treatment options for the millions of Americans suffering from PTSD, including countless military veterans, first responders, and survivors of sexual and interpersonal violence who deserve better choices.

Midomafetamine (MDMA) capsules have not been approved by any regulatory agency. The safety and efficacy of midomafetamine has not been established for the treatment of PTSD. Investigational midomafetamine is also being studied in other indications.

1 ICER. Accessed June 27, 2024. June 27, 2024: Midomafetamine-Assisted Psychotherapy for Post-Traumatic Stress Disorder, Final Evidence Report
2 FDA. Accessed June 26, 2024. June 4, 2024: Meeting of the Psychopharmacologic Drugs Advisory Committee Meeting Announcement, Lykos Briefing Document | FDA
3 Mitchell JM, Bogenschutz M, Lilienstein A, et al. MDMA-assisted therapy for severe PTSD: a randomized, double-blind, placebo-controlled phase 3 study. Nat Med. 2021Jun;27(6):1025-1033. doi: 10.1038/s41591-021-01336-3
4 Mitchell JM, Ot'alora MG et al. MDMA-assisted therapy for moderate to severe PTSD: a randomized, placebo-controlled phase 3 trial. Nat Med. 2023 Sept 14 doi: 10.1038/s41591-023-02565-4

SOURCE Lykos Therapeutics

For further information: MEDIA CONTACT: Lykos Therapeutics, E. Blair Clark-Schoeb, Chief Communications Officer,