Clinical TrialJun 22, 2026, 07:02 AM
MapLight ML-004 Phase 2 misses primary endpoint; shows irritability benefit
AI Summary
MapLight Therapeutics announced topline results from its Phase 2 IRIS trial for ML-004 in autism spectrum disorder (ASD), which did not meet its primary endpoint for social communication deficits. However, a prespecified analysis in adolescents with moderate to severe baseline irritability demonstrated a clinically meaningful improvement over placebo (effect size 1.33, nominal p=0.013). ML-004 was generally well-tolerated with no severe adverse events. The company plans to engage with the FDA for an End-of-Phase 2 meeting and remains well-capitalized ahead of ML-007C-MA Phase 2 results in mid-August 2026.
Key Highlights
- ML-004 Phase 2 IRIS trial did not meet primary endpoint in social communication deficits.
- Prespecified analysis showed 1.33 effect size (nominal p=0.013) for irritability in adolescents.
- Clinician-rated CGI-I showed 1.08 effect size (nominal p=0.036) in same subpopulation.
- ML-004 was generally well-tolerated with no severe or serious adverse events.
- Mean weight gain was lower with ML-004 than with placebo.
- Company plans End-of-Phase 2 meeting with FDA for ML-004.
- Topline results for ML-007C-MA in schizophrenia expected by mid-August 2026.
- Company to host live webcast today at 8:00 AM ET to discuss results.
Price Impact
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