SAN FRANCISCO & BOSTON, May 1, 2026
MapLight Therapeutics, Inc. announced the completion of enrollment in its Phase 2 ZEPHYR trial evaluating ML-007C-MA for schizophrenia, alongside the final patient visit completion in the Phase 2 IRIS trial assessing ML-004 for autism spectrum disorder (ASD). The company confirmed that topline results from both trials are expected by mid-August 2026, marking a significant milestone in its mission to develop next-generation therapies targeting central nervous system (CNS) disorders. With both schizophrenia and autism representing areas of high unmet medical need, these updates position MapLight at a critical inflection point in its clinical development strategy.
Advancing Schizophrenia Treatment with ZEPHYR Trial
The ZEPHYR Phase 2 trial is a randomized, double-blind, placebo-controlled study designed to evaluate the efficacy, safety, and tolerability of ML-007C-MA in adults experiencing acute exacerbation of psychosis associated with schizophrenia. A total of 307 participants were randomized into three groups receiving placebo or two different dosing regimens of ML-007C-MA. The primary endpoint focuses on change in Positive and Negative Syndrome Scale (PANSS) total score from baseline to Week 5, a widely accepted measure of schizophrenia symptom severity. Secondary endpoints include improvements in Clinical Global Impression-Severity (CGI-S) and PANSS subscales targeting both positive and negative symptoms. Additionally, exploratory endpoints aim to evaluate cognitive function improvements, addressing one of the most challenging aspects of schizophrenia management. This comprehensive design reflects a multi-dimensional approach to psychiatric treatment, going beyond symptom control to potentially improve cognitive deficits and functional outcomes.
IRIS Trial Targets Core Symptoms of Autism Spectrum Disorder
In parallel, the IRIS Phase 2 trial evaluates ML-004 in adults and adolescents with autism spectrum disorder, enrolling 161 participants, including a significant adolescent cohort. The study is also randomized, double-blind, and placebo-controlled, with the primary endpoint assessing change in Autism Behavioral Inventory (ABI) – Social Communication Domain Score over 12 weeks. Secondary endpoints include Clinical Global Impression-Improvement (CGI-I), ABI composite scores, and Aberrant Behavior Checklist-Irritability (ABC-I), which collectively measure behavioral and social function improvements. Autism remains a complex neurodevelopmental condition with limited pharmacological treatment options targeting core symptoms, making ML-004 a potentially important candidate. By focusing on social communication and behavioral regulation, the IRIS trial aims to address critical aspects of patient quality of life that are often underserved by current therapies.
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Strategic Positioning in CNS Drug Development
MapLight Therapeutics is leveraging its circuit-based neuroscience platform, which aims to identify and modulate specific neural circuits causally linked to disease pathology. This approach differentiates the company from traditional drug development strategies that often rely on broader neurotransmitter modulation. By targeting precision neurocircuitry, MapLight seeks to develop more effective and better-tolerated therapies for complex CNS disorders. The simultaneous progress of both ZEPHYR and IRIS trials underscores the company’s commitment to building a diversified pipeline across psychiatric and neurodevelopmental conditions. The expected release of topline data in August 2026 represents a key catalyst event, with the potential to validate MapLight’s scientific platform and inform future late-stage clinical development.
The completion of enrollment and trial milestones highlights the operational efficiency and strong clinical execution achieved by MapLight, supported by robust patient and investigator participation. As the company prepares to deliver critical data readouts, stakeholders across the biopharma industry will be closely monitoring outcomes that could reshape treatment paradigms in schizophrenia and autism. If successful, these programs could pave the way for innovative, circuit-targeted therapies that address both symptoms and underlying disease mechanisms, offering new hope for patients and caregivers affected by debilitating CNS disorders.
Source: MapLight Therapeutics press release
