Core Insights - Reviva Pharmaceuticals announced positive results from the Phase 3 RECOVER open-label extension study of brilaroxazine for schizophrenia, demonstrating sustained efficacy and a well-tolerated safety profile over one year [1][2][5] Efficacy and Safety - Brilaroxazine showed robust broad-spectrum efficacy across all major symptom domains of schizophrenia, with a statistically significant reduction in PANSS total score of -18.1 at 12 months [3][4] - The treatment was generally well-tolerated, with a discontinuation rate of 35%, primarily due to withdrawal of consent and loss to follow-up [1][9] - Improvements in multiple neuroinflammatory markers were observed, suggesting enhanced efficacy and reduced side effects [1][2][9] Clinical Data - The study included a pooled analysis of 446 patients receiving doses of 15 mg, 30 mg, and 50 mg, showing dose-dependent efficacy [2][4] - Key findings included a reduction in negative symptoms by -4.4 at 12 months and significant improvements in personal and social performance scores [3][4] - The treatment also demonstrated a low incidence of treatment-emergent adverse events, with most being mild or moderate in severity [9][10] Future Development - Reviva aims to advance brilaroxazine towards regulatory submission, with plans to explore its use in other neuropsychiatric conditions such as bipolar disorder and major depressive disorder [10][11] - The company has received Orphan Drug Designation from the FDA for brilaroxazine in treating pulmonary arterial hypertension and idiopathic pulmonary fibrosis [11][12]

Core Insights - Neurocrine Biosciences announced significant improvements in symptoms and overall severity of schizophrenia in adults from the Phase 2 study of NBI-1117568, the first oral muscarinic M4 selective orthosteric agonist in clinical development for this condition [1][2][6] Company Overview - Neurocrine Biosciences is focused on developing treatments for neurological, neuroendocrine, and neuropsychiatric disorders, with a diverse portfolio that includes FDA-approved treatments and a robust pipeline of compounds in clinical development [13] Study Details - The Phase 2 study involved 210 adults aged 18 to 55 with schizophrenia, randomized to receive either NBI-1117568 or placebo over a six-week period, followed by a two-week safety follow-up [2][9] - The primary endpoint was the change in total Positive and Negative Syndrome Scale (PANSS) score from baseline to Week 6, showing statistically significant improvements with the 20 mg dose of NBI-1117568 by Week 3 [4][6] Safety and Tolerability - NBI-1117568 was generally safe and well tolerated, with treatment discontinuation rates due to adverse events similar to placebo; common adverse events included somnolence (10.7% vs 2.9%) and dizziness (9.3% vs 1.4%) [3][6] Future Development - Based on positive Phase 2 results, a Phase 3 registrational program has been initiated to further evaluate the efficacy, safety, and tolerability of NBI-1117568, expected to enroll approximately 280 patients [6][9]

Core Insights - Anavex Life Sciences Corp. has successfully completed enrollment for its Phase 2 clinical study of ANAVEX3-71, targeting schizophrenia, with a total of 71 participants [2][3][4] - The study consists of two parts: Part A with 16 participants focusing on multiple ascending doses, and Part B with 55 participants aimed at providing comprehensive clinical and biomarker data [3][4] - Top-line data from the study is expected to be reported in the second half of 2025 [5] Company Overview - Anavex Life Sciences Corp. is a clinical-stage biopharmaceutical company focused on developing treatments for various CNS disorders, including Alzheimer's disease, Parkinson's disease, and schizophrenia [2][7] - The lead drug candidate, ANAVEX2-73, has shown promise in multiple clinical trials for Alzheimer's disease and other CNS disorders [7][8] - ANAVEX3-71 is a dual SIGMAR1 receptor agonist and M1 positive allosteric modulator, which may address all symptom domains of schizophrenia without the side effects associated with standard antipsychotics [4][8] Industry Context - Schizophrenia affects nearly 24 million people globally, with significant unmet medical needs due to limitations of current treatments [6] - Approximately 34% of individuals with schizophrenia do not respond to existing therapies, and 50-60% experience only partial improvement or unacceptable side effects [6]

Core Insights - Neurocrine Biosciences has initiated a Phase 3 registrational program for NBI-1117568, an investigational oral muscarinic M4 selective orthosteric agonist aimed at treating schizophrenia, following positive Phase 2 data reported in August 2024 [1][2][3] Company Overview - Neurocrine Biosciences is a neuroscience-focused biopharmaceutical company dedicated to developing treatments for neurological, neuroendocrine, and neuropsychiatric disorders, with a diverse portfolio including FDA-approved treatments and a robust pipeline [8] Clinical Development - The Phase 3 study is a global double-blind, placebo-controlled trial targeting approximately 280 patients with schizophrenia experiencing acute exacerbation or relapse of symptoms, with primary and secondary endpoints focused on PANSS and CGI-S scale improvements [2][3] - NBI-1117568 is the first oral muscarinic M4 selective orthosteric agonist in clinical development for schizophrenia, potentially offering a novel mechanism with an improved safety profile [3][4] Market Need - Schizophrenia affects approximately 24 million people globally, with annual costs exceeding $150 billion in the U.S., highlighting the significant need for innovative treatment options [6][7] Clinical Results - The Phase 2 study demonstrated a statistically significant reduction in PANSS total score with a placebo-adjusted mean reduction of 7.5 points and an 18.2-point reduction from baseline, indicating the efficacy of NBI-1117568 [6]