PRESS RELEASE: FDA Grants Breakthrough Therapy Designation for MDMA-Assisted Psychotherapy for PTSD, Agrees on Special Protocol Assessment for Phase 3 Trials

Key highlights

Breakthrough Therapy Designation ensures that FDA will work closely with MAPS to complete Phase 3 trials as efficiently as possible

MAPS and FDA have also reached agreement on design, primary endpoint, and statistical approach for Phase 3 trials

Posttraumatic stress disorder (PTSD) is a serious and life-threatening psychiatric condition with unmet medical need despite available treatments

MAPS is sponsoring two Phase 3 clinical trials of MDMA-assisted psychotherapy in patients with severe PTSD starting in 2018

MAPS, a non-profit research organization, has raised or pledged half of the $25 million estimated cost of these trials, with $12.5 million still needed

CONTACT:

Brad Burge, Director of Strategic Communications, MAPS

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The non-profit Multidisciplinary Association for Psychedelic Studies (MAPS) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to MDMA for the treatment of posttraumatic stress disorder (PTSD). MAPS and the FDA have also reached agreement under the Special Protocol Assessment Process (SPA) for the design of two upcoming Phase 3 trials (MAPP1 and MAPP2) of MDMA-assisted psychotherapy for patients with severe PTSD.

MDMA-assisted psychotherapy is a novel treatment package that combines psychotherapeutic techniques with three administrations of MDMA as a pharmacological adjunct.

By granting Breakthrough Therapy Designation, the FDA has agreed that this treatment may have a meaningful advantage and greater compliance over available medications for PTSD.

The first Phase 3 trial (MAPP1), "A Randomized, Double-Blind, Placebo-Controlled, Multi-Site Phase 3 Study of the Efficacy and Safety of Manualized MDMA-Assisted Psychotherapy for the Treatment of Severe Posttraumatic Stress Disorder," will begin enrolling subjects in Spring 2018, after the completion of an open-label lead-in training study at Phase 3 sites starting this fall.

"Reaching agreement with FDA on the design of our Phase 3 program and having the ability to work closely with the agency has been a major priority for our team," said Amy Emerson, Executive Director of the MAPS Public Benefit Corporation (MPBC). "Our Phase 2 data was extremely promising with a large effect size, and we are ready to move forward quickly. With breakthrough designation, we can now move even more efficiently through the development process in collaboration with the FDA to complete Phase 3."

"For the first time ever, psychedelic-assisted psychotherapy will be evaluated in Phase 3 trials for possible prescription use, with MDMA-assisted psychotherapy for PTSD leading the way," said Rick Doblin, Founder and Executive Director of MAPS. "Now that we have agreement with FDA, we are ready to start negotiations with the European Medicines Agency."

The Phase 3 trials will assess the efficacy and safety of MDMA-assisted psychotherapy in 200-300 participants with PTSD, aged 18 and older, at sites in the U.S., Canada, and Israel. Participants will be randomized to receive three day-long sessions of either MDMA or placebo in conjunction with psychotherapy over a 12-week treatment period, along with 12 associated 90-minute non-drug preparatory and integration sessions. The primary endpoint will be the Clinician Administered PTSD Scale (CAPS-5), as assessed by a blinded pool of independent raters.

In MAPS’ completed Phase 2 trials with 107 participants, 56% no longer qualified for PTSD after treatment with MDMA-assisted psychotherapy, measured two months following treatment. At the 12-month follow-up, 68% no longer had PTSD. Most subjects received just 2-3 sessions of MDMA-assisted psychotherapy. All participants had chronic, treatment-resistant PTSD, and had suffered from PTSD for an average of 17.8 years.***

MDMA transiently increases heart rate, blood pressure, and body temperature in a dose-dependent manner that is generally not problematic for physically healthy individuals. Serious Adverse Events involving administration of MDMA in MAPS studies have been uncommon and non-life threatening. Phase 2 trial results are currently being prepared for publication.

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About Breakthrough Therapies

The FDA grants Breakthrough Therapy Designation to treatments that (1) are intended alone or in combination with one or more other drugs to treat a serious or life-threatening disease or condition; and (2) preliminary clinical evidence indicates may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. Since 2012, the FDA has implemented this program to expedite the development and review of any treatment designated as a breakthrough therapy. For more information, see the FDA's Fact Sheet.

About Special Protocol Assessments

The Special Protocol Assessment (SPA) process is an agreement resulting from official evaluation and written guidance from FDA on the protocol design, clinical endpoints, planned conduct, and statistical analyses for the Phase 3 trial to ensure they are acceptable to support regulatory approval. Final marketing approval depends on the efficacy results, adverse event profile, and an evaluation of the ratio of risks and treatment benefits demonstrated in the Phase 3 clinical program. The SPA agreement may only be changed through a written agreement between the sponsor and the FDA, or if the FDA becomes aware of a substantial scientific issue essential to product efficacy or safety. For more information, see the FDA's Guidance for Industry.

About MDMA-Assisted Psychotherapy

MDMA-assisted psychotherapy is a novel treatment package that combines psychotherapy with the administration of MDMA (approved generic name: midomafetamine) to achieve significant and lasting reductions in PTSD symptoms. MDMA is administered in three single-dose psychotherapy sessions administered in a clinic setting spaced three to five weeks apart, along with preparatory and integration sessions. Phase 2 clinical trials have shown that MDMA can reduce fear and defensiveness, enhance communication and introspection, and increase empathy and compassion. These combined neurological effects can enhance the therapeutic process for people suffering from PTSD.

About PTSD

PTSD is a serious, long-lasting, and life-threatening condition when not adequately treated, highlighting the need for expedited approval of new therapies. PTSD sufferers may relive their traumatic experiences through nightmares and flashbacks, have difficulty sleeping, and feel detached from daily life. Approximately 7% of the U.S. population, and 11-17% of U.S. military veterans, will have PTSD sometime in their life. As of June 2016, more than 868,000 veterans were receiving disability compensation for PTSD, with an estimated taxpayer cost of $17 billion per year. PTSD is a stress-related condition associated with reduced cognitive and psychosocial functioning, fractured relationships, inability to maintain employment, substance abuse, and increased risk of depression and suicide. In the general population, 27% of suicides are associated with PTSD.

About MAPS and MPBC

Founded in 1986, MAPS is a 501(c)(3) non-profit research and educational organization that develops medical, legal, and cultural contexts for people to benefit from the careful uses of psychedelics and marijuana. Since its founding, MAPS has raised over $43.7 million for psychedelic therapy and medical marijuana research and education. For more information, visit maps.org.

MAPS-sponsored clinical trials are conducted by the MAPS Public Benefit Corporation (MPBC), a wholly owned subsidiary of MAPS formed in 2015 for the special purpose of balancing social benefits with income from legal sales of MDMA, other psychedelics, and marijuana. For more information, visit mapsbcorp.com.

Donations are needed now to complete this vital research. No funding for these trials is yet available from governments, for-profit pharmaceutical companies, or major foundations. MAPS has raised or pledged half of the approximately $25 million needed to complete these trials, with $12.5 million still needed. To make a contribution, visit maps.org/donate.

***NOTE: This paragraph has been updated from the original version, which reported that 61% of participants no longer qualified for PTSD after 2 or 3 sessions. This percentage incorrectly included participants whose data was not included in the final report due to study drop-outs and other reasons. The correct number, 56%, is reported above. There is no change to the long-term (12-month) follow-up data.

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