The Psychiatric Pipeline in Review: Quarter 1, 2026

The first quarter of 2026 had several prominent developments in the psychiatric treatment pipeline. Here, learn more about what percentage of that news was positive vs negative, what disease states were most prominently featured, and what treatments you should keep an eye on.

Check out all the specifics of our coverage from the from the first quarter below.

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.

Data Supports Potential of Negative Allosteric Modulators for Anxiety and Fear-Related Disorders

Newly published data shows negative allosteric modulator targeting metabotropic glutamate receptor 7 (mGlu7) may be transformative in treating anxiety and fear-related disorders such as posttraumatic stress disorder. Preclinical data from Addex suggests mGlu7 modulation with ADX71743 can alter reconsolidation of fear memories, a potential intervention point in anxiety and trauma-related disorders. ADX71743 disrupted fear memory reconsolidation in rats, requiring recall and a defined post-recall window, and produced conditioned stimulus–specific weakening with reduced fear reinstatement.

Extension of Expanded Access Program for Zervimesine to Treat Dementia With Lewy Bodies

An extension has been announced for the expanded access program of zervimesine to treat dementia with Lewy bodies. The original expanded access program was planned for 12 months, and the extension will now add several more months of treatment for patients. Zervimesine (CT1812) is a sigma-2 receptor modulator that blocks binding and displaces A-beta and alpha-synuclein oligomers from receptor sites to preserve neuronal synapses and normalize neural functioning. In previous preclinical studies, zervimesine was shown to antagonize binding of A-beta oligomers to neuronal synapses, protect neuronal synapses, and restore membrane trafficking deficits cause by A-beta oligomers.

NDA Accepted and Priority Review Granted: Oveporexton for Narcolepsy Type 1

The FDA has accepted Takeda’s New Drug Application (NDA) and granted Priority Review for oveporexton (TAK-861) for the treatment of narcolepsy type 1 (NT1). Oveporexton is an investigational oral orexin receptor 2-selective agonist specifically designed to address orexin deficiency, an underlying issue that causes NT1, by restoring orexin signaling. If successful, this would be the first approved orexin agonist treatment to individuals living with NT1.

Denovo Biopharma Partners With Orygen for Phase 2 Study of Pomaglumetad Methionil in Treating Psychosis

Denovo Biopharma is partnering with Orygen and plans to initiate a phase 2 investigator-initiated-trial studying pomaglumetad methionil (DB103) for first-episode psychosis (FEP) or clinical high risk (CHR) of psychosis. Orygen is Australia’s leading nonprofit youth mental health organization. Pomaglumetad methionil, a first-in-class psychiatric drug that selectively acts on the glutamic acid mGlu2/3 receptor, has no cross-reaction with other receptors in the central nervous system and therefore can avoid some usual adverse effects of currently-available psychiatric drugs.

Lisdexamfetamine Dimesylate Oral Solution for ADHD to Be Available Mid-2026

Azurity Pharmaceuticals has announced that lisdexamfetamine dimesylate (Arynta) oral solution will be available mid-2026 for treating attention-deficit/hyperactivity disorder (ADHD) in adults and pediatric patients aged 6 years and older. Arynta gained US Food and Drug Administration approval back on June 16, 2025, for the treatment of ADHD in adults and pediatric patients 6 years and older, and moderate to severe binge eating disorder in adults.

Buntanetap for Neurodegenerative Diseases: Positive Recommendation From Data and Safety Monitoring Board

An independent Data and Safety Monitoring Board (DSMB) issued a positive recommendation regarding the safety of Annovis Bio’s buntanetap—an investigational oral therapy for neurodegenerative diseases such as Alzheimer disease (AD) and Parkinson disease—at 6 months. This supports the continuation of the ongoing buntanetap pivotal phase 3 AD clinical trial (NCT06709014) without any modification.

Strategy Confirmed With FDA for Phase 2b/3 Study of XPro1595 to Treat Early Alzheimer Disease

INmune Bio confirmed receipt of notes from the FDA on continuing the proposed integrated phase 2b/3 clinical development of XPro1595 for early Alzheimer disease. The company aligned regulatory strategy with FDA recommendations and will continue its drug development strategy. The phase 2b study will include an integrated design, with approximately 300 participants over a 9-month evaluation period to validate efficacy and biomarkers assumptions before the phase 3 element continues. The complete phase 3 program is expected to enroll over 1000 participants, evaluating over 18 months. For a primary efficacy endpoint, the study will use the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB). Patients will be enrolled based on 2 or more biomarkers associated with peripheral inflammation and immune-mediated disease risk (high sensitivity C-reactive protein, erythrocyte sedimentation rate, hemoglobin A1c, and apolipoprotein E4), which are known to be linked with soluble TNF signaling.

COMP360 Psilocybin for Treatment-Resistant Depression Achieves Primary Endpoint in Phase 3 Trial

Compass Pathways recently announced the successful achievement of the primary endpoint in the ongoing phase 3 COMP006 trial evaluating 2 fixed doses of COMP360—a synthetic, proprietary formulation of psilocybin for managing treatment-resistant depression. The primary endpoint was the difference in change from baseline in the Montgomery-Åsberg Depression Rating Scale scores between the 25 mg and 1 mg groups at week 6. Two fixed doses—administered 3 weeks apart—of COMP360 25 mg vs 1 mg demonstrated a highly statistically significant reduction in symptom severity (P<0.001) and a clinically meaningful difference of -3.8 points in change at the primary endpoint.

Phase 2a Trial Meets Endpoints for Psychedelic SPL026 to Treat Major Depressive Disorder

Helus Pharma’s SPL026 met its primary endpoint in a phase 2a trial, showing clinically significant reduction in depression symptoms. The drug, a form of intravenous dimethyltryptamine psychedelic, showed antidepressant effects within a week of treatment, and sustained effects for up to 3 months. Helus Pharma does not plan to advance the drug in its current form, but stated that the mechanistic and clinical insights from the trial will continue to inform the company’s drug development programs.

Phase 2 Study of CYC-126 for Treatment Resistant Depression Receives FDA Feedback, Will Proceed

Cyclerion Therapeutics, developers of CYC-126, received positive feedback from the FDA on the drug’s phase 2 study and path forward to potential regulatory approval. The investigational drug is an anesthetic-based therapy incorporating live electroencephalogram feedback to address treatment-resistant depression. The intended study is a randomized, double-blind, 2-part clinical study evaluating the drug in adults with treatment resistant depression who are also candidates for monitored anesthesia. The trial will use FDA-accepted clinical endpoints, including the Montgomery-Asberg Depression Rating Scale, and will assign participants randomly to treatment or placebo groups. Assessments of safety, efficacy, and durability of response will be the main focus.

NDA Accepted: Olanzapine Extended-Release Injectable Suspension for Treatment of Schizophrenia

Teva Pharmaceuticals recently announced that the FDA accepted its New Drug Application (NDA) for olanzapine extended-release injectable suspension (TEV-'749) for the treatment of adults with schizophrenia. The NDA was submitted to the FDA in December 2025. TEV-'749 is an investigational once-monthly subcutaneous long-acting injectable (LAI) of the second-generation atypical antipsychotic olanzapine. The NDA for TEV-'749 is based on results from the phase 3 SOLARIS trial, including week 56 results studying its efficacy, safety and tolerability in participants aged 18 to 64 living with schizophrenia. The results demonstrated an efficacy and safety profile consistent with currently available olanzapine formulations.

FDA Approves Bysanti for Treatment of Bipolar 1 Disorder and Schizophrenia

The FDA has approved Vanda Pharmaceuticals’ Bysanti (milsaperidone) for treatment of acute bipolar disorder and schizophrenia. Bysanti is a new chemical entity in the atypical antipsychotic class, providing a novel therapeutic option. Bysanti is expected to be available in the US for schizophrenia and bipolar 1 disorder later in 2026.

BXCL501 for Treatment of Opioid Withdrawal: Positive Phase 2 Topline Results

BioXcel Therapeutics announced positive topline results from a phase 2 investigator-sponsored trial evaluating BXCL501 for the treatment of opioid withdrawal symptoms in adults with opioid use disorder (OUD) undergoing a methadone taper. Study data suggest that BXCL501 may be as effective as or superior to lofexidine (Lucemyra) for reducing the symptoms of opioid withdrawal during a methadone taper, while also possessing a more convenient dosing regimen and a favorable tolerability profile. In this study, BXCL501 240 µg twice daily reduced opioid withdrawal symptoms compared with placebo during a 7-day methadone taper.

New Phase 2 Signal Detection Results: HLP004 for Moderate-to-Severe Generalized Anxiety Disorder

Helus Pharma announced topline results from a phase 2 signal detection study evaluating HLP004 as a potential treatment for adults with moderate-to-severe generalized anxiety disorder (GAD) who remained symptomatic despite ongoing standard of care antidepressant therapy, including selective serotonin reuptake inhibitors and related agents. As no adjunctive pharmacologic treatments for GAD have ever been approved, and the last new monotherapy was approved almost 2 decades ago, any new treatment in this area has potential to transform the GAD treatment space.

Phase 2 Clinical Study of AL001 Initiated for Bipolar Disorder Type 1

Alzamend Neuro has announced the initiation of a phase 2 clinical study of AL001 in patients diagnosed with bipolar disorder (BD) type 1. AL001 is a novel lithium-delivery system that has the potential to provide the benefits of marketed lithium salts while mitigating or avoiding currently experienced toxicities associated with lithium. Investigators of the study will utilize a crossover design intended for multiple 6-subject cohorts. Following screening, participants will be randomized into 1 of 2 treatment sequences: AB (AL001 followed by lithium carbonate) or BA (lithium carbonate followed by AL001). Each treatment period consists of 14 days of 3-times daily dosing. A washout period of 14 days is planned between treatment periods. During days 14 and 15 of each treatment period, participants will undergo intensive 24-hour lithium pharmacokinetic blood sampling along with advanced magnetic resonance imaging and magnetic resonance spectroscopy neuroimaging.

BPL-003 Nasal Spray Shows Quick and Enduring Effect in Treatment-Resistant Depression

BPL-003, mebufotenin benzoate nasal spray, showed reduced depressive symptoms and sustained response rates in patients with moderate to severe treatment-resistant depression. Further cohort data from the 4-part phase 2a trial have been published showing efficacy and safety of the medication in this unique form. Recently published data were gathered from cohort 1 of the phase 2a study, which included 12 participants with treatment-resistant depression in a 12-week, open-label trial. Participants included 2 women and 10 men of ages 31 to 55, and all completed the trial. Participants were not taking any concomitant antidepressants. A 10 mg dose of intranasal BPL-003 was administered, then change in Montgomery-Asberg Depression Rating Scale (MADRS) score and remission were measured at days 2, 8, 29, 57, and 85 post-dose.

Updates on Blarcamesine and Lecanemab for Alzheimer Disease From AD/PD 2026

Anavex Life Sciences shared new data on blarcamesine and Esai presented new analysis of intravenous lecanemab at the International Conference on Alzheimer’s and Parkinson’s Diseases in Copenhagen, Denmark, March 17-21, 2026. In the the AD-004 phase 2b/3 trial, the long-term data on blarcamesine showed 77.4 weeks (nearly 18 months) time saved with oral blarcamesine treatment, compared with controls, after 144 weeks of treatment. Additionally, analysis of long-term treatment persistence with IV lecanemab in patients with early Alzheimer disease showed most patients continued the medication past 18 months. Throughout the long-term study, 78.4% of patients continued treatment at 18 months, 71.7% continued at 20 months, and 67.3% continued at 24 months. This finding is consistent with the previous phase 3 Clarity AD study, in which 94% of patients completing 18 months of lecanemab treatment chose to continue maintenance treatment in the open-label extension. Patients in the Clarity AD study who continued lecanemab showed benefits from 4 years of treatment, compared with the natural course of Alzheimer disease.

New Pivotal Phase 3 Trial: LB-102 for Treatment of Schizophrenia

LB Pharmaceuticals has initiated the pivotal phase 3 NOVA-2 trial evaluating the efficacy and safety of LB-102 as a treatment for schizophrenia. LB-102, a novel, once-daily, oral investigational small molecule, is a selective antagonist of D2, D3, and 5HT-7 receptors. It is the first potential benzamide in the US for the treatment of neuropsychiatric disorders.

Tazbentetol for Schizophrenia Shows Symptom Improvement in Phase 2 Trial

Interim results from a phase 2 trial of tazbentetol for treatment of schizophrenia showed trends of improving positive and negative symptoms in patients via synaptic regeneration. The safety profile was overall favorable, and biomarker signals indicated improvements in cortical abnormalities associated with schizophrenia. The interim analysis was presented at the Schizophrenia International Research Society 2026 Annual Congress.

First Expert Consensus Recommendations for Tardive Dyskinesia in Long-Term Care Settings

Alongside consensus recommendations focused on the screening, diagnosis, and treatment of tardive dyskinesia (TD), Neurocrine Biosciences presented a first-of-its-kind post-hoc analysis from the KINECT‑PRO study at the Society for Post-Acute and Long-Term Care Medical Association PALTC26 Annual Conference. Valbenazine (Ingrezza) capsules demonstrated meaningful improvements in patient‑reported TD impact among adults aged 65 years and older. In adults aged 65 and older, once-daily Ingrezza was associated with robust patient-reported improvements in TD symptoms, quality of life and functional impairment at week 24. Patient-reported outcomes also demonstrated reduced social and emotional burden, with improvements in total TDIS scores exceeding the established threshold for clinically meaningful change.

Phase 2 Trial of LB-102 for Schizophrenia Shows Positive Impact on Cognitive Performance

LB Pharmaceuticals presented a phase 2 post hoc analysis of the NOVA-1 clinical trial investigating LB-102 for the treatment of schizophrenia at the 2026 Schizophrenia International Research Society meeting in Florence, Italy. The analysis confirmed that cognitive performance improvement was statistically significant and directly related to LB-102. Based on measurement of the Global Cognition composite score, analysis showed improved cognitive performance was a direct effect of LB-102. Generally, the phase 2 trial showed statistically significant effects of LB-102 compared with placebo at all doses tested.

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