June in Review: Updates on the Psychiatric Treatment Pipeline

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Take a look at this month’s developments in the psychiatric treatment pipeline. We compiled a recap of the latest news here, just in case you missed any of the updates.

FDA Officially Eliminates Clozapine REMS

On June 13, 2025, the US Food and Drug Administration (FDA) officially eliminated the clozapine REMS, and all REMS operations ceased. Although clozapine still carries a risk of severe neutropenia, the FDA has determined that the clozapine REMS is no longer necessary, and by eliminating the REMS, they expect to improve access to clozapine. In the wake of this decision, the Schizophrenia & Psychosis Action Alliance, in collaboration with the American Association of Psychiatric Pharmacists, has announced the upcoming development of a set of clinical, evidence-based courses designed to provide education and guidance for clozapine prescribing.

New Positive 12-Month Extension Data: Ecopipam for Tourette Syndrome in Pediatric Patients

Ecopipam for Tourette syndrome (TS) was found to be safe and effective in a 12-month, open-label extension of a phase 2b study. Ecopipam 1.8 mg/kg/day reduced tic severity and improved quality of life in children and adolescents with TS, with no evidence of tachyphylaxis. This is the largest study to date that examines the long-term safety and effectiveness of ecopipam in a pediatric population with TS.

NRx Pharmaceuticals Applies for FDA National Priority Voucher for Intravenous Ketamine (NRX-100)

NRx Pharmaceuticals is pursuing FDA approval for NRX-100, a preservative-free ketamine targeting suicidal depression and PTSD, via the new Commissioner’s National Priority Voucher pathway, which expedites review for public health priority drugs. The company anticipates an FDA decision on NRX-100 by the end of 2025. Additionally, the company plans to file a citizen petition with the FDA to withdraw preservative-containing forms of ketamine, citing concerns about benzethonium chloride toxicity. A recently filed patent on its preservative-free manufacturing process may further support market exclusivity.

COMP360 Psilocybin for Treatment-Resistant Depression: Positive Phase 3 Efficacy Data

Compass Pathways announced the successful achievement of the primary endpoint in the ongoing phase 3 COMP005 trial, the first of 2 phase 3 trials evaluating their synthetic, proprietary formulation of psilocybin for treatment-resistant depression, COMP360. COMP360 showed a statistically significant reduction in TRD symptom severity compared to placebo, with a clinically meaningful MADRS score difference. The COMP005 trial is the first phase 3 study of synthetic psilocybin, marking a milestone in psychedelic research for mental health.

ALTO-203 Fails to Primary Efficacy Endpoint in Phase 2 Major Depressive Disorder Trial

ALTO-203, a phase 2 investigational oral H3 receptor blocker, failed to significantly improve mood in patients with MDD, missing the primary efficacy endpoint in the phase 2 trial. The phase 2 trial enrolled 69 participants with MDD and elevated levels of anhedonia. Of these 69, 63 patients completed the first part of the study, which assessed the effect of single 25 µg or 75 µg doses of ALTO-203 on a measure of alertness and mood. The study’s primary outcome was the change in positive emotion as measured by the Bond-Lader Visual Analog Scale (BL-VAS). Approximately 5 hours after a single dose of ALTO-203, participants reported significant improvements in alertness and mood; however, ALTO-203 failed to statistically separate itself from placebo.

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