May 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 Filing Fee Waiver for New Drug Application of NRX-100 for Suicidal Depression

NRx Pharmaceuticals received a US Food and Drug Administration (FDA) filing fee waiver for NRX-100, a preservative-free ketamine for suicidal depression. The preservative-free formulation addresses safety concerns linked to benzethonium chloride in existing ketamine products. The company will seek to prove that NRX-100 is superior to placebo, active comparators, and electroshock therapy in clinical trials. FDA approval could improve accessibility and insurance coverage for NRX-100, with an new drug application filing anticipated by mid-year.

FDA Has Granted Request for End of Phase 2 Meeting to Discuss Alcohol Use Disorder Treatment, AD04

Adial Pharmaceuticals announced that the FDA has granted Adial’s request for an end of phase 2 meeting to discuss a proposed clinical development plan and FDA guidance on the phase 3 adaptive with enrichment design of the upcoming clinical trial for AD04. The meeting will take place on July 25, 2025. AD04 is Adial’s lead investigational treatment, a genetically targeted selective serotonin-3 receptor (5-HT3) antagonist and therapeutic agent for the treatment of alcohol use disorder in patients who engage in heavy drinking (defined as < 8 drinks/drinking day). Recent FDA confirmation of Adial's in vitro bridging strategy and a patent for genetic marker identification bolster AD04's development.

Phase 3 Development Plan for Evenamide as Add-On Therapy for Treatment-Resistant Schizophrenia

Newron Pharmaceuticals announced the approval of the pivotal ENIGMA-TRS phase 3 development program evaluating evenamide as an add-on therapy to current antipsychotics, including clozapine, in patients with treatment-resistant schizophrenia (TRS). This phase 3 development program consists of 2 pivotal studies: ENIGMA-TRS 1 and ENIGMA-TRS 2. ENIGMA-TRS 1 is a 52-week, international, randomized, double-blind, placebo-controlled study involving 600 patients, assessing evenamide's efficacy, tolerability, and safety. ENIGMA-TRS 2 is a 12-week, randomized, double-blind, placebo-controlled study with 400 participants, focusing on the 15 mg BID dose of evenamide. Previous studies demonstrated evenamide's significant efficacy and favorable safety profile, indicating its potential to address unmet needs in treatment-resistant schizophrenia.

New Ingrezza Data Demonstrates Functional and Quality of Life Improvements in Patients With Tardive Dyskinesia

Neurocrine Biosciences announced new analyses from a phase 4 randomized withdrawal study (NCT03891862) showing participants with tardive dyskinesia who received continued treatment with valbenazine (Ingrezza) capsules demonstrated functional and health-related quality of life improvements. Treatment with Ingrezza enhanced mobility, improved self-care, and reduced pain and anxiety. Additionally, significant enhancements were seen in work/school, social life, and family/home life with Ingrezza treatment, supporting its efficacy.

Novel PDE10A Inhibitor for Acute Schizophrenia Exacerbation Presented at APA Annual Meeting

Researchers presented promising data for CPL’36, a phosphodiesterase 10A (PDE10A) inhibitor intended to treat acute schizophrenia exacerbation at the 2025 American Psychiatric Association (APA) Annual Meeting in Los Angeles, California. In a double-blind, randomized, placebo controlled, parallel group phase 2 study involving 189 participants, CPL’36, a PDE10A inhibitor, showed significant improvements in PANSS scores in a phase 2 study for acute schizophrenia exacerbation. Does of 20 mg and 40 mg of CPL’36 showed notable efficacy compared with placebo.

Positive Phase 2 Results on NBI-1117568 for Schizophrenia Presented at 2025 ASCP Annual Meeting

Neurocrine Biosciences recently shared data at the American Society of Clinical Psychopharmacology 2025 Annual Meeting from the phase 2 study of NBI-1117568 in adults with schizophrenia, which showed a significant improvement in symptoms and overall severity and highlighted new data on the safety and tolerability of the treatment. NBI-1117568 is the first and only investigational oral muscarinic M4 selective orthosteric agonist in clinical development as a potential treatment for schizophrenia. The study demonstrated statistically significant improvements in PANSS and CGI-S scores by week 3 and week 2, respectively. A phase 3 registrational program has been initiated to further evaluate NBI-1117568's efficacy, safety, and tolerability in schizophrenia treatment.

New Data: Cariprazine Adjunctive to Antidepressant Therapy for Anhedonia Symptoms

Cariprazine, a dopamine D3-preferring receptor partial agonist, showed potential in reducing anhedonia symptoms in patients with major depressive disorder when used adjunctively with antidepressants in a recent study. The 26-week, phase 3, open-label study demonstrated significant reductions in anhedonia subscale scores from baseline as early as week 4, sustained through week 26. Cariprazine was found to be safe and well tolerated, with the most common dose being 3 mg/day, followed by 1.5 mg/day and 4.5 mg/day.

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