January 2026 in Review: Updates on the Psychiatric Treatment Pipeline

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.

Phase 2 Proof-Of-Concept Study Evaluating BHV-7000 for Major Depressive Disorder Fails to Meet Primary Endpoint

Biohaven reported results from a phase 2 proof-of-concept study evaluating BHV-7000 for the treatment of major depressive disorder (MDD): BHV-7000 did not demonstrate a reduction of depressive symptoms as measured by change in the Montgomery Åsberg Depression Rating Scale (MADRS) over 6 weeks compared with placebo, thus failing to meet the primary endpoint. However, trends favoring BHV-7000 were observed in some clinically relevant subgroups, including participants with more severe depression at screening and baseline, on primary and secondary outcome measures.

Fast Track Designation Granted: ML-007C-MA for Alzheimer Disease Psychosis

MapLight Therapeutic announced that the US Food and Drug Administration (FDA) has granted Fast Track designation to ML-007C-MA, an investigational novel M1/M4 muscarinic agonist, for the treatment of hallucinations and delusions associated with Alzheimer disease psychosis. ML-007C-MA—also known as ML-007C/PAC—is an oral, extended-release, fixed-dose combination of the investigational M1/M4 muscarinic agonist ML-007, coformulated with a peripherally acting anticholinergic. Developers designed ML-007C-MA to activate both M1 and M4 muscarinic receptors in the central nervous system to drive efficacy, while synchronizing the pharmacokinetics of the agonist and antagonist components to mitigate peripheral cholinergic adverse effects.

Zervimesine May Slow Progression of Dementia With Lewy Bodies, Phase 2 Results Show

Cognition Therapeutics announced positive phase 2 results of the SHIMMER study, with zervimesine meeting primary endpoints and showing favorable results in clinical features of dementia with Lewy bodies. The phase 2 SHIMMER study was a randomized, placebo-controlled trial including 130 adults aged 50 to 85 years with mild to moderate dementia with Lewy bodies. After 6 months, patients who received zervimesine showed improved symptoms as measured by the neuropsychiatric inventory. Participants receiving zervimesine also showed an improvement in fluctuations, which were defined as unpredictable lapses in attention or consciousness for minutes to days. Individuals in the treatment group also showed improvement in activities of daily living such as dressing, bathing, and writing.

FDA Grants Breakthrough Therapy Designation to Alixorexton for the Treatment of Narcolepsy Type 1

The FDA has granted Breakthrough Therapy designation to Alkermes’ alixorexton for the treatment of narcolepsy type 1 (NT1). Alixorexton is a novel, investigational, oral, selective orexin 2 receptor agonist in development for the treatment of NT1, narcolepsy type 2 (NT2), and idiopathic hypersomnia. This designation is based on phase 1 and phase 2 clinical data, including positive results from Vibrance-1, a large phase 2 study evaluating alixorexton in 92 participants with NT1.

FDA Accepts Investigational New Drug Application for COMP360 for PTSD

The FDA has accepted the Investigational New Drug Application for COMP360 in the treatment of patients with posttraumatic stress disorder. The acceptance allows Compass Pathways to initiate a phase 2b/3 clinical trial with patients with PTSD. The multicenter, randomized double blind controlled study will be comprised of a blinded arm and an open label arm and will investigate the efficacy, safety, and tolerability of the agent in patients with PTSD. The blinded arm will be 12-weeks in duration and will be the double-blinded and controlled portion of the study. It will assess the efficacy of 2 administrations of 25 mg versus 2 doses of 1 mg of COMP360, with the second administrations scheduled for approximately 4 weeks after the first. Investigators will look at the change in Clinician-Administered PTSD Scale for DSM-5 total severity score at week 8 as the primary efficacy endpoint.

Neurosterix Announces Phase 1 Study of NTX-253 for Schizophrenia

A phase 1 study has commenced for NTX-253 for treatment of schizophrenia. The positive allosteric modulator of muscarinic M4 receptor will be evaluated for safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy volunteers. Advancement of NTX-253 into phase 1 first-in-human clinical studies is supported by preclinical studies showing antipsychotic-like activity and a favorable safety profile.

New Phase 3 Clinical Vocal Biomarker Data on Brilaroxazine to Treat Negative Symptoms in Schizophrenia

Reviva Pharmaceuticals announced the publication of clinical vocal and speech biomarker data from the RECOVER phase 3 clinical trial and the therapeutic potential of brilaroxazine for the treatment of schizophrenia. Brilaroxazine is a new chemical entity with potent affinity and selectivity against key serotonin and dopamine receptors implicated in the pathophysiology of several conditions including schizophrenia. The published findings reinforce the treatment effect of brilaroxazine on negative symptoms and other symptom domains in schizophrenia and support clinician-assessed efficacy outcomes, as well as support the use of speech latency as an enrichment tool that can reduce sample-size and enhance outcomes in clinical trials for schizophrenia.

Progress Report: Second Interim Safety Review of Phase 3 Trial of LPCN 1154 in Postpartum Depression

Lipocine has announced the completion of a scheduled independent Data Safety Monitoring Board (DSMB) review of its ongoing phase 3 clinical trial evaluating oral brexanolone (LPCN 1154) for the rapid relief treatment of postpartum depression (PPD). This was the second of 2 DSMB reviews planned during the study; the DSMB recommended that the trial continue as planned with no modifications. LPCN 1154 is an oral formulation of brexanolone that is being developed to provide rapid relief of PPD in a convenient, at-home setting. It has potential to be the first line treatment choice for women with PPD while presenting no significant risk of adverse reactions from exposure to breastfed infants.

FDA Approves ProlivRX: First Prescription, Physician-Directed, At-Home Brain Neuromodulation Therapy for MDD

The FDA has approved Neurolief Inc’s ProlivRx, the first prescription, physician-directed, at-home brain neuromodulation therapy as an adjunctive treatment for adults with MDD who failed to achieve satisfactory improvement from at least 1 previous antidepressant medication. The approval under the Class III Premarket Approval pathway was supported by clinical evidence from the MOOD Study, a randomized, controlled, multicenter clinical trial, evaluating ProlivRx in MDD with inadequate response to antidepressant medications.

New Useful Insights: Analysis of Data From Phase 3 Clinical Trials Evaluating Simufilam for Mild-to-Moderate Alzheimer Disease

Cassava Sciences announced the publication of a detailed analysis of data from 2 phase 3 randomized clinical trials, RETHINK-ALZ and REFOCUS-ALZ, examining the use of simufilam to treat mild-to-moderate Alzheimer disease. While these 2 studies did not meet their prespecified coprimary, secondary, or exploratory biomarker endpoints, exploratory post-hoc analysis of the studies offers informative insights for future research. Simufilam is a proprietary, investigational, oral small molecule that is believed to modulate activity of the filamin A protein.

FDA Issues Removal of Suicidal Behavior and Ideation Warning From GLP-1 RAs

The FDA has requested that drug application holders remove information regarding the risk of suicidal ideation and behavior from the labeling of glucagon-like peptide-1 receptor agonist (GLP-1 RA) medications. The affected GLP-1 RAs are liraglutide (Saxenda), semaglutide (Wegovy), and tirzepatide (Zepbound). This news comes at the heels of a comprehensive FDA review that found no increased risk of SI/B associated with the use of GLP-1 RA medications.

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