A week after an FDA committee voted against recommending midomafetamine (MDMA) treatment for post-traumatic stress disorder (PTSD), Lykos Therapeutics is working to clarify the situation and address concerns that have emerged in the broader conversation. This effort comes in the wake of significant public discourse surrounding the potential benefits and risks associated with the psychoactive treatment.
Understanding the FDA Committee’s Decision
The Psychopharmacologic Drugs Advisory Committee (PDAC) recently voted against the approval of MDMA capsules on two critical fronts: efficacy and safety. While the FDA often aligns with the committee’s guidance, it is not bound to do so. The committee’s decision sparked widespread debate, with discussions about psychoactive drugs reaching beyond the biotech community.
Lykos Therapeutics’ Commitment to Clarity
In a letter to shareholders, Lykos CEO Amy Emerson addressed the need to clarify misconceptions and present the company’s stance on midomafetamine. Emerson emphasized the importance of discussing the unique aspects of this treatment and the company’s responses to the committee’s concerns.
Evidence of Clinical Efficacy
Emerson reiterated that Lykos’ application for MDMA is supported by robust clinical evidence. Two randomized, placebo-controlled phase 3 trials demonstrated significant improvements in PTSD symptoms among participants. These results, which met primary endpoints, showed statistically significant and durable outcomes, reinforcing the potential of MDMA as a treatment for PTSD.
Addressing Functional Unblinding Concerns
The committee expressed concerns about functional unblinding in the trials, a known challenge in psychiatric drug research. Emerson acknowledged this issue but highlighted the steps taken to mitigate its impact. These included the use of independent, blinded third-party clinicians to assess outcomes, aiming to ensure the integrity of the data.
Bias and Prior Illicit MDMA Use
Another concern was whether prior illicit use of MDMA by some participants could bias the results. Emerson noted that the trials found no meaningful differences in outcomes between those who had previously used MDMA and those who had not. This suggests that the therapeutic effects observed were not influenced by past use.
MDMA-Assisted Therapy: Strengths and Liabilities
The integration of therapy with MDMA administration, known as MDMA-assisted therapy, was identified as both a strength and a potential liability. A committee member highlighted that while the therapy component is a critical aspect of the treatment’s effectiveness, it falls outside the FDA’s regulatory purview.
Ensuring Standardization in Therapy
To address this, Emerson detailed the measures taken to standardize and ensure consistency in the therapy component across trials. She emphasized that psychotherapy is already a regulated field, with oversight provided by licensing boards and professional societies. If approved, MDMA-assisted therapy will be delivered by healthcare professionals under these established guidelines.
Risk Evaluation and Mitigation Strategy (REMS)
Lykos’ MDMA therapy will include a Risk Evaluation and Mitigation Strategy (REMS) program, a requirement for certain medications to ensure their benefits outweigh potential risks. While the specifics of this program are still being finalized, Emerson stressed that it will play a crucial role in the safe administration of MDMA therapy.
Addressing Historical Misconduct
The committee also raised concerns about a case of therapist misconduct during a phase 2 trial in 2015. Emerson condemned the incident, which involved sexual abuse by a therapist, and detailed the steps Lykos has taken to prevent such occurrences in the future. These include independent reporting channels and stringent policies to detect and address any unethical behavior.
Tackling Sponsor and Investigator Bias
Concerns about potential bias among investigators, including the failure to report feelings of euphoria, were also addressed. Emerson refuted these claims, asserting that Lykos is committed to transparency and rigorous data collection.
Potential Cardiovascular and Hepatotoxicity Risks
The committee highlighted potential cardiovascular and hepatotoxicity risks associated with MDMA. Emerson pledged to work with the FDA to further characterize these risks and provide additional data if required. Lykos is confident that these risks can be managed effectively in a post-marketing environment.
Collaboration with the FDA
In conclusion, Emerson reiterated Lykos’ commitment to collaborating with the FDA. She emphasized that all relevant data and documentation have been provided to the agency, and the company remains dedicated to addressing any concerns that may arise.
Frequently Asked Questions
What is midomafetamine (MDMA) therapy?
Midomafetamine (MDMA) therapy involves the use of MDMA, a psychoactive drug, in combination with psychotherapy to treat PTSD. It aims to help patients process traumatic experiences more effectively.
Why did the FDA committee vote against MDMA therapy for PTSD?
The FDA committee voted against recommending MDMA therapy due to concerns about its efficacy and safety. However, the FDA is not bound by this vote and will make its own decision.
How does Lykos Therapeutics address concerns about functional unblinding in clinical trials?
Lykos took several measures to minimize functional unblinding, including using independent, blinded third-party clinicians to assess outcomes, ensuring the integrity of the data.
What steps has Lykos taken to prevent therapist misconduct?
Lykos has implemented strict policies and independent reporting channels to prevent and detect any unethical behavior. These measures are designed to ensure the safety and well-being of patients.
Is psychotherapy regulated in the context of MDMA-assisted therapy?
Yes, psychotherapy is a regulated field with oversight provided by licensing boards and professional societies. MDMA-assisted therapy will be delivered by qualified professionals under these established guidelines.
What are the potential risks associated with MDMA therapy?
Potential risks include cardiovascular and hepatotoxicity concerns. Lykos is working with the FDA to further characterize these risks and ensure they can be managed effectively.
Lykos Therapeutics remains committed to addressing the concerns raised by the FDA committee and providing a clear understanding of the potential benefits and risks of midomafetamine (MDMA) therapy for PTSD. With robust clinical evidence and stringent safety measures in place, the company continues to advocate for the approval of this innovative treatment, emphasizing its potential to significantly improve the lives of individuals suffering from PTSD.