FDA Approves MDMA for Clinical Trial Use Investigating Schizophrenia

The FDA has approved a clinical trial investigating an ± 3,4-methylenedioxymethamphetamine (MDMA) investigational medical product (IMP; LaNeo MDMA, PharmAla) in 40 mg capsules. The trial (NCT05770375) will be conducted at the University of California, Los Angeles and will examine the use of this drug to assess its tolerability in patients with schizophrenia.^1,2

Specifically, the investigators will be assessing the use of the drug to treat impaired social motivation, or asociality, which is a symptom of schizophrenia that can cause significant functional impairment. Although schizophrenia symptoms can be treated with antipsychotics, there is currently no effective treatment for asociality.^1,2

MDMA is known to have pronounced pro-social effects, which can increase the motivation for individuals to engage socially. In prior research conducted among healthy volunteers, MDMA was shown to produce feelings of empathy and closeness with others, as well as increase attention paid to positive social cues. The study investigators hypothesize that this increased level of empathy and perceived closeness may partly be due to MDMA’s effects on the social bonding hormone oxytocin.²

To date, MDMA has also shown promise in investigations assessing other psychiatric conditions such as PTSD. In this open-label, ascending-dose, within-subject trial, the investigators plan to first assess the tolerability of the drug in patients with schizophrenia in doses of 40 mg, 80 mg, or 120 mg. The doses will be given in ascending order, and doses will be stopped if subjects experience moderate or greater levels of psychotic symptoms at 24 hours. The trial will assess the tolerability and guide in the selection of a maximum well-tolerated dose for future studies.²

Additionally, the study investigators noted that the primary tolerability measure will be clinician-rated psychotic symptoms (disorganized speech, delusions, hallucinations) observed at 24 hours after MDMA administration. Further, the results will support further investigations into the use of MDMA and other psychoactive compounds in the treatment of debilitating and difficult-to-treat social deficits of schizophrenia. Future studies will look to examine interactions between the psychoactive effects of the drug and nonpharmacologic psychosocial interventions targeting social symptoms.²

"While we’ve had great success with many regulators around the world, it’s still a major milestone to receive a stamp of approval from FDA for our investigational drugs,” said Harpreet Kaur, PhD, vice president of research at PharmAla Biotech, in a press release. “As with any IMP supplier, there are always questions about Chemistry, Manufacturing and Control—even for well-known molecules. We believe that this announcement should confirm that our IMP conforms to the high levels of quality and documentation that one of the preeminent regulators in the world requires.”¹

Reference

1. USFDA Approves LaNeo MDMA for Clinical Trial Use in the United States [in email]. Vancouver, Canada: PharmAla; June 9, 2023.
2. NIH. Tolerability of MDMA in Schizophrenia (TMS). ClinicalTrials.gov. Updated May 10, 2023. Accessed June 9, 2023. https://clinicaltrials.gov/ct2/show/NCT05770375