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The Psychiatric Pipeline in Review: Quarter 3, 2025
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Key Takeaways
- Lumateperone showed significant efficacy in preventing schizophrenia relapse, leading to an sNDA submission to the FDA.
- TSND-201 received Breakthrough Therapy designation for PTSD, showing early and sustained symptom reduction.
What disease states are most prominently featured in recent research? Learn more in this exclusive article.
The third quarter of 2025 saw some huge successes and fails 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.
What percentage of 2025 Q3 psychiatric pipeline news was positive vs negative? The news this quarter was overwhelmingly positive, with approximately 94.7% good news.
What disease states are most prominently featured in recent research? The top 4 disease states this quarter were schizophrenia (36.8%), Alzheimer disease (31.6%), major depressive disorder (15.8%), and anxiety (15.8%).
Check out all the specifics of our coverage from the from the third quarter below.
sNDA Submitted: Lumateperone for the Prevention of Relapse in Schizophrenia
Johnson & Johnson announced the submission of a supplemental New Drug Application (sNDA) to the US Food and Drug Administration (FDA) based upon long-term data evaluating the safety and efficacy of lumateperone (Caplyta) for the prevention of relapse in schizophrenia. The sNDA submission is based upon positive results from a phase 3, double-blind, multicenter, placebo-controlled, randomized withdrawal trial. Investigators found time to relapse during the 26-week double-blind treatment phase was significantly longer in patients receiving Caplyta compared with those receiving placebo (P=0.0002), the primary endpoint of the study. Treatment with Caplyta was also associated with a 63% reduction in risk of relapse vs placebo (hazard ratio [95% CI] = 0.37, [0.22, 0.65]).
TSND-201 Receives Breakthrough Therapy Designation From FDA for PTSD
The FDA granted Transcend Therapeutics with the Breakthrough Therapy designation for their compound TSND-201 for (methylone) for the treatment of posttraumatic stress disorder (PTSD) following positive results from its IMPACT-1 study. TSND-201 showed significant efficacy in reducing PTSD symptoms, with improvements seen as early as Day 10 and sustained through Day 64.
Seaport Therapeutics announced that the first patient has been dosed in the phase 2b BUOY-1 study of GlyphAllo (Glyph Allopregnanolone, or SPT-300) in MDD with or without anxious distress. If successful, GlyphAllo could be a first-in-class treatment for patients with MDD, with or without anxious distress. GlyphAllo is a novel, “glyphed” oral prodrug of allopregnanolone. In the global, randomized, double-blind, placebo-controlled BUOY-1 study, investigators will evaluate the efficacy, safety, and tolerability of GlyphAllo in adults with MDD, with or without anxious distress.
FDA Advisory Committee Votes Against Recommending Brexpiprazole Plus Sertraline for PTSD
The FDA Advisory Committee voted not to recommend brexpiprazole (Rexulti) in combination with sertraline for PTSD. The panel believed the efficacy was not well-enough established to recommend its approval. The vote was 10-1. With only 2 approved psychopharmacological treatments in the past 20 years for PTSD, both with modest and inconsistent efficacy in managing PTSD symptoms, investigators hoped to meet this severe unmet need with brexpiprazole. This is the second PTSD treatment to receive a negative advisory committee vote. In August 2024, the FDA issued a complete response letter for MDMA-AT for PTSD, requesting an additional phase 3 trial to examine safety and efficacy, rather than the approving the psychedelic.
BioXcel Therapeutics has submitted a pre-supplemental New Drug Application (pre-sNDA) meeting package to the FDA for Igalmi in support of a potential label expansion for outpatient use for the acute treatment of agitation associated with bipolar disorders or schizophrenia. Igalmi is an investigational, orally dissolving film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist. Igalmi is the first and only FDA-approved orally dissolving sublingual film for mild, moderate, or severe agitation in patients with schizophrenia or bipolar I or II disorder, administered under the supervision of a health care provider
Positive Phase 2 Data: Alixorexton for the Treatment of Adults With Narcolepsy Type 1
Alkermes announced positive topline results from the randomized double-blind treatment period of the Vibrance-1 phase 2 study evaluating alixorexton in patients with narcolepsy type 1 (NT1). Treatment with alixorexton led to statistically significant and dose-dependent improvements in sleep latency among patients with NT1. Alixorexton, formerly referred to as ALKS 2680, is a novel, investigational, oral orexin 2 receptor agonist in phase 2 development as a once-daily treatment for NT1, narcolepsy type 2, and idiopathic hypersomnia.
Positive Topline Results From Phase 3 Trial of AD109, First Oral Pill for Obstructive Sleep Apnea
Apnimed announced positive topline results from the LunAIRo study, its second pivotal phase 3 clinical trial of their lead candidate, AD109 (aroxybutynin 2.5 mg/atomoxetine 75 mg) for obstructive sleep apnea. AD109 is a first-in-class, anti-apneic neuromuscular modulator that targets the root cause of OSA by increasing upper airway muscle tone during sleep. It is designed to be a once-daily pill taken at bedtime.
Roche presented new data on potential Alzheimer disease (AD) treatments and diagnostic tools at the Alzheimer’s Association International Conference (AAIC) in Toronto, Canada. This includes new insights on the phase 1b/2a Brainshuttle AD study of trontinemab, the design of the phase 3 TRONTIER 1 and 2 studies of trontinemab in early symptomatic AD, plans for a phase 3 trial investigating trontinemab in preclinical AD, and data on the Elecsys pTau217 as a standalone blood test for identification of amyloid pathology. Trontinemab demonstrated significant amyloid plaque reduction and a favorable safety profile in the phase 1b/2a Brainshuttle AD study. Elecsys pTau217 blood test, comparable to PET scans, received FDA Breakthrough Device Designation, enhancing AD diagnostic accessibility.
Cingulate Receives $4.3 Million PDUFA Fee Waiver for ADHD Treatment, CTx-1301
Cingulate announced that it received a fiscal year 2025 Prescription Drug User Fee Act (PDUFA) fee waiver from the FDA for its new drug application (NDA) for dexmethylphenidate HCI (CTx-1301) for the treatment of attention-deficit/hyperactivity disorder (ADHD) in patients aged 6 years and older. The waiver, granted through the small business waiver provision of the Federal Food, Drug, and Cosmetic Act, will save Cingulate approximately $4.3 million as it prepares to submit its NDA at the end of this month.
New Data: Zervimesine for the Treatment of Dementia With Lewy Bodies and Alzheimer Disease
Cognition Therapeutics presented new data on zervimesine (CT1812) in dementia with Lewy bodies (DLB) and Alzheimer disease at the Alzheimer’s Association International Conference (AAIC) in Toronto, Canada. This included results from the phase 2 COG1201 SHIMMER study (NCT05225415) of zervimesine (CT1812) in DLB, results from the phase 2 COG0201 SHINE study (NCT03507790) of zervimesine in Alzheimer disease, and 2 additional posters supporting zervimesine’s efficacy.
FDA Clearance of IND for Ketamir-2, Novel Oral NMDA Receptor Antagonist for Neuropathic Pain
MIRA Pharmaceuticals recently announced that the FDA cleared its Investigational New Drug (IND) application for Ketamir-2, a novel oral NMDA receptor antagonist for the treatment of neuropathic pain. Ketamir-2 shows superior efficacy and safety in preclinical neuropathic pain models compared with gabapentin and pregabalin. Ketamir-2's pharmacological profile positions it as a promising nonopioid, noncontrolled treatment for neuropathic pain, with potential for broader therapeutic applications.
Anavex Life Sciences shared promising results from a phase 2b/3 open-label extension trial of extended oral blarcamesine for the treatment of early Alzheimer disease (AD) at the 2025 Alzheimer's Association International Conference (AAIC) in Toronto. Blarcamesine demonstrated significant cognitive (ADAS-Cog13: -5.43, P=0.0035) and functional (ADCS-ADL: +9.50, P<0.0001) improvements in patients with early AD.
European Medicine Agency Committee Recommends Zuranolone for Postpartum Depression
Biogen announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recently adopted a positive opinion recommending marketing authorization for zuranolone (Zurzuvae) to treat postpartum depression (PPD). If approved, this would be the first medication authorized in the European Union specifically to treat PPD. The European Commission will proceed to review the CHMP’s recommendation.
Boehringer Ingelheim and Click Therapeutics announced that the pivotal phase 3 CONVOKE study of CT-155 (BI 3972080), an investigational prescription digital therapeutic, met its primary end point. Investigators compared the effectiveness and safety of CT-155 vs a digital control app as an adjunct to standard of care antipsychotic therapy in individuals with symptoms of schizophrenia and found that CT-155 showed a reduction in experiential negative symptoms.
FDA Grants Type C Meeting for Aversa Fentanyl Abuse Deterrent Patch
The FDA has granted a Type C Meeting for Nutriband’s lead product, Aversa fentanyl, or the abuse deterrent fentanyl transdermal system. In the meeting, the FDA will specifically provide feedback on the chemistry, manufacturing, and controls plans for Aversa fentanyl from the submission of an Investigational New Drug Application through approval of a 505(b)(2) New Drug Application and subsequent commercialization. Aversa fentanyl has the potential to be the world’s first abuse-deterrent opioid patch designed to deter the abuse and misuse, and reduce the risk of accidental exposure of transdermal fentanyl patches.
NRx Pharmaceuticals today announced that the FDA has granted Fast Track designation to NRX-100 for the treatment of suicidal ideation in patients with depression, including bipolar depression. NRX-100 is the first preservative-free formulation of intravenous ketamine for acute suicidal crises in depression. Currently available forms of ketamine contain the preservative benzethonium chloride, but its safety for repeated use has never been demonstrated.
FDA Approves KETARx for Management of Surgical Pain
The FDA has approved PharmaTher Holdings’ ketamine product, KETARx, for its indicated uses in surgical pain management. PharmaTher believes this approval provides a strong foundation for the expansion of ketamine’s development across varied therapeutic areas, including in mental health conditions like depression, neurological disorders such as Parkinson disease and amyotrophic lateral sclerosis, and the management of rare or chronic pain, such as complex regional pain syndrome.
FDA Approves Tonmya, First Fibromyalgia Medication in 15 Years
The FDA has approved Tonix Pharmaceuticals' Tonmya (cyclobenzaprine HCl, formerly known as TNX-102 SL), a novel treatment form for fibromyalgia.1 The drug is now the first in a new category of non-opioid analgesics for fibromyalgia and the first new medication for this disorder in 15 years. The FDA’s approval cited efficacy from 2 double-blind, randomized, placebo-controlled, phase 3 clinical trials of almost 1000 patients that evaluated Tonmya as treatment for fibromyalgia. Across both phase 3 trials, Tonmya significantly reduced daily pain scores compared with placebo at the primary endpoint of 14 weeks.
BioXcel Therapeutics announced that it has received positive pre sNDA meeting responses from the FDA on its candidate BXCL501 for agitation associated with bipolar disorders or schizophrenia. Outside of the FDA-approved indication, dexmedetomidine (Igalmi) sublingual film, BXCL501 is an investigational proprietary, orally dissolving film formulation of dexmedetomidine, which is a selective alpha-2 adrenergic receptor agonist.
FDA Grants Fast Track Designation to Pemvidutide for Treatment of Alcohol Use Disorder
Altimmune announced that the FDA has granted Fast Track designation to pemvidutide for the treatment of alcohol use disorder (AUD). Pemvidutide is a novel, investigational, peptide-based 1:1 GLP-1/glucagon dual receptor agonist. The phase 2 trial RECLAIM (NCT06987513) evaluating the safety and efficacy of pemvidutide in AUD is currently enrolling.
Leqembi for Alzheimer Disease Launches in First European Union Countries
Eisai and Biogen today announced that the anti-amyloid beta (Aβ) monoclonal antibody lecanemab-irmb (Leqembi) for the treatment of Alzheimer disease has been launched in Austria on August 25, 2025, and will be launched in Germany on September 1, 2025. Leqembi received approval from the European Commission in April 2025 as the first therapy that targets an underlying cause of AD. Germany and Austria will mark the first launches in the EU.
Incannex Healthcare shared positive data from its phase 2 clinical trial of PSX-001 (formerly known as Psi-GAD), a psilocybin-assisted psychotherapy treatment for generalized anxiety disorder (GAD). Participants treated with PSX-001 achieved statistically significant and clinically meaningful improvements across every key endpoint assessed in the study, reinforcing its potential as a treatment for patients with moderate to severe GAD.
FDA Approves Leqembi for At-Home Maintenance Treatment of Alzheimer Disease
The FDA has approved Leqembi IQLIK (lecanemab), for self-administered injection for maintenance treatment of Alzheimer disease in patients with mild cognitive impairment or mild dementia. Leqembi, developed by Esai and Biogen, is the only treatment for Alzheimer disease that can be administered subcutaneously at home using an autoinjector. The currently available Leqembi is administered intravenously, which requires patients to visit an infusion center every 2 weeks, but this new at-home autoinjector is designed to simplify this process and allow maintenance dosing self-administered by patients.
FDA Accepts New Drug Application for Cytisinicline for Smoking Cessation
Achieve Life Sciences announced the FDA’s acceptance of an NDA for cytisinicline for smoking cessation in adults.1 A Prescription Drug User Fee (PDUFA) date has been assigned for June 20, 2026. The NDA submission provided data from the company’s ORCA-2 and ORCA-3 phase 3 trials, showing that cytisinicline treatment resulted in significantly greater rates of smoking abstinence as compared to placebo.
Target Enrollment Completed for Phase 3 AFFIRM-1 Trial of BNC-210 in Social Anxiety Disorder
Neuphoria Therapeutics announced the achievement of target enrollment of 332 participants in the AFFIRM-1 phase 3 trial that will evaluate the lead candidate BNC-210 as a first-in-class, acute, as-needed, fast-acting treatment for social anxiety disorder. BNC-210 is an oral, proprietary, selective negative allosteric modulator of the α7 nicotinic acetylcholine receptor under development for the treatment of SAD and PTSD.
Study on Zervimesine for Alzheimer Disease Reaches Enrollment Target
Cognition Therapeutics announced this week that their phase 2 START study for zervimesine (CT1812) has reached 75% enrollment. The study will measure efficacy of once-daily, oral zervimesine in treating individuals with elevated Ab plaques from mild cognitive impairment or early Alzheimer disease. The study currently has approximately 540 participants, which marked the goal of 75% enrollment completion.
Alixorexton for Narcolepsy Type 1: New Detailed Positive Phase 2 Results From Vibrance-1 Study
Alkermes announced positive results from the Vibrance-1 phase 2 study evaluating alixorexton in participants with narcolepsy type 1 (NT1). According to these results, alixorexton is the first orexin 2 receptor agonist to demonstrate clinically meaningful and statistically significant impact on wakefulness, cognition, and fatigue with once-daily dosing across a range of doses.
FDA Approves Risperidone Extended-Release Injectable Suspension
The FDA has approved Amneal Pharmaceuticals' risperidone extended-release injectable suspension, an atypical antipsychotic for treatment of schizophrenia and maintenance of bipolar 1 disorder. The approval was based on phase 3 trial results, which included a double-blind placebo-controlled trial, along with a 52-week open-label study of the medication. Over the 12 months of exposure in the open-label study, mean Positive and Negative Syndrome Scale scores improved in patients continuing from the first double-blind study, and new participants scores remained stable.
Alto Neuroscience today announced positive results from an independent, prospective replication study evaluating electroencephalography biomarkers in individuals with schizophrenia. The study successfully replicated previous findings, demonstrating that event-related theta-band responses, particularly theta-band inter-trial coherence, robustly differentiate individuals with schizophrenia from individuals without schizophrenia.
New Preclinical Data on ART12.11 for the Treatment of Mood and Anxiety Disorders
Artelo Biosciences announced the publication of new preclinical data on ART12.11, its proprietary cannabidiol:tetramethylpyrazine (CBD:TMP) cocrystal. ART12.11 significantly outperformed cannabidiol (CBD) alone in reducing stress-induced depression and anxiety symptoms, and also achieved superior oral bioavailability. Investigators found that ART12.11 reversed stress-induced behavioral deficits and produced robust antidepressant and anxiolytic-like effects.
Johnson & Johnson reported post hoc analysis focused on the effect of esketamine (Spravato) monotherapy on emotional blunting in patients with treatment-resistant depression. The analysis measured an emotional blunting composite score, ultimately finding these scores to significantly improve from baseline in patients receiving esketamine. Esketamine nasal spray monotherapy significantly reduced emotional blunting vs placebo through day 28 and as early as day 2 as measured by MADRS items 7 and 8 and PHQ-9 item 1.
Investigators presented new phase 3 data on seltorexant compared with quetiapine extended release (XR) as adjunctive treatment for MDD with insomnia symptoms at the 2025 Psych Congress in San Diego, CA. While seltorexant did not meet the primary end point of a recent phase 3 study, it may still effectively address an important unmet medical need in MDD.
BPL-003 for Treatment-Resistant Depression: New Positive Phase 2a Data
atai Life Sciences and Beckley Psytech shared positive data from a proof-of-concept study investigating a 2-dose induction regimen of intranasal mebufotenin benzoate (BPL-003), in patients with treatment-resistant depression. In the open-label phase 2a study (NCT05660642), investigators enrolled 13 patients with TRD who were not on concurrent antidepressants, and 12 met the criteria for per-protocol analysis. Participants were given an 8 mg dose of BPL-003, followed by a 12 mg dose 2 weeks later. The results show that a second dose of BPL-003 at week 2 has the potential to induce greater antidepressant effects without impact on the safety and tolerability profile of the treatment.
Neurocrine Biosciences today announced new data from a post-hoc analysis of the phase 4 KINECT-PRO open-label study that confirms robust rates of symptomatic remission of tardive dyskinesia were achieved with once-daily valbenazine (Ingrezza) capsules. The analysis also showed sustained improvements in patient-reported outcomes among participants who achieved symptomatic remission. KINECT-PRO is the first and only study to evaluate and demonstrate patient-reported improvement with vesicular monoamine transporter 2 inhibitor treatment, specifically Ingrezza, on TD using multiple clinically validated scales.
New Positive Phase 2 Data on Osavampator for Major Depressive Disorder
A new phase 2 study from Neurocrine Biosciences showed statistically significant and clinically meaningful improvement in depression severity with daily administration of 1 mg osavampator (NBI-1065845).1 The study met its primary and secondary endpoints in showing greater than 50% reduction in depression severity from baseline to day 28 and from baseline to day 56, as measured by the Montgomery-Asberg Depression Rating Scale.
SYT-510: First-in-Class Inhibitor Targets Endocannabinoid System to Improve Mental Health Outcomes
Synendos Therapeutics announced topline results for its lead asset, SYT-510, a first-in-class inhibitor that modulates a newly identified drug target in the endocannabinoid system (ECS) that helps restore healthy brain functions. SYT-510 belongs to a novel class of ECS modulators called selective endocannabinoid reuptake inhibitors, which represent first-in-class, new chemical entities that modulate the ECS through a self-limiting mode of action. This novel mode of action has the potential to combine treatment of a range of symptoms with sustained efficacy in large patient populations alongside the potential to address chronic tolerability, a key unmet need; this represents an innovative and potentially safer therapeutic approach to multiple CNS disorders such as anxiety, mood, and stress-related conditions, and more.
Phase 3 Results: Hetlioz Improves Sleep in Patients With Primary Insomnia
Vanda Pharmaceuticals today shared new positive data from a phase 3 study on tasimelteon (Hetlioz) for the treatment of patients with insomnia. Hetlioz showed significant improvement in sleep latency with effects persisting throughout the study duration. Hetlioz was well-tolerated, with no cognitive, mood changes, rebound, or withdrawal effects, and no adverse next-day residual effects observed. The study supports tasimelteon as a safe and effective treatment for chronic sleep onset insomnia, offering an alternative to current insomnia medications with fewer adverse effects.
First Patients Receive IV Selonabant for Acute Cannabinoid-Induced Toxicity in Phase 1 Trial
Anebulo Pharmaceuticals announced that patients have begun receiving intravenous selonabant in the phase 1 singe ascending dose study investigating the drug for acute cannabis-induced toxicity. The IV formulation of selonabant is being developed as a potential treatment for pediatric patients with acute cannabis-induced toxicity. The phase 1 clinical trial is a randomized, double-blind, placebo-controlled, single ascending dose study of IV administration of selonabant. Healthy adult participants aged 18 to 25 will receive the drug, testing safety, tolerability, and pharmacokinetics.
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