MAPS Public Benefit Corporation has shown off phase 3 data it hopes will secure a landmark approval for MDMA in the U.S. next year. The second late-stage study success is the result of almost 40 years of work to show that psychedelics can treat post-traumatic stress disorder (PTSD) when used to enhance therapy.
The corporation, which the Multidisciplinary Association for Psychedelic Studies (MAPS) founded in 2014 in anticipation of FDA approval, reported the top-line success of its second phase 3 study at the start of the year. MAPS PBC ran the study to confirm the results of its first phase 3 trial, which found MDMA can reduce PTSD symptoms and functional impairment when used as an adjunct to supportive therapy.
Now, more than two years after the publication of the first set of phase 3 data, MAPS PBC has reported the results of its second pivotal trial in Nature Medicine. The key results are in line with the findings of the earlier study.
Investigators randomized around 100 people with moderate or severe PTSD to receive MDMA or placebo with supportive therapy. Participants had three eight-hour dosing sessions, in conjunction with therapy, spaced approximately one month apart. After 18 weeks, scores on CAPS-5, a PTSD scale, had decreased by 23.7 in the MDMA arm and 14.8 in the placebo group, causing the trial to hit its primary endpoint.
MAPS PBS linked the drug candidate to improvements on secondary endpoints. Scores on a measure of functional impairment fell 3.3 points in the MDMA arm, versus a 2.1 decline in the placebo group. Again, the results were similar to the findings of the earlier study and statistically significant.
On the safety front, five patients on MDMA had severe treatment-emergent adverse events, compared to two of their peers on placebo. Muscle tightness, nausea, decreased appetite and excessive sweating were more common in the MDMA arm than in the placebo group, but most cases were transient and of mild to moderate severity.
MAPS PBC aims to use the results of the two phase 3 trials to file for FDA approval by the end of the year. Hitting the target would put the organization on track to win approval next year. As a breakthrough drug, MDMA could benefit from priority review, but MAPS PBC may face questions about its trials, notably because blinding may have been undermined by the fact most subjects guessed which arm they were in.