MapLight Therapeutics Stock Falls Despite Schizophrenia Drug Meeting Main Trial Goal

MapLight Therapeutics Inc. (NASDAQ:MPLT) stock is trading lower on Monday after the company reported topline data from its Phase 2 ZEPHYR trial of ML-007C-MA, an oral M1/M4 muscarinic agonist (betovumeline) co-formulated with a peripherally acting anticholinergic (fesoterodine), in adults with an acute exacerbation of schizophrenia.

The study successfully met its primary endpoint at Week 5, as the 210/3 mg twice-daily dose achieved a statistically significant and clinically meaningful reduction in Positive and Negative Syndrome Scale (PANSS) total scores versus placebo.

PANSS measures the severity of symptoms in schizophrenia patients. It evaluates positive symptoms (excess functions like hallucinations), negative symptoms (loss of normal functions), and general psychopathology.

In contrast, a 330/6 mg once-daily dose delivered numerical improvements and secondary separation, but did not achieve statistical significance on the primary endpoint.

The company said the 330/6 mg once-daily dose results in lower daily exposure than twice-daily dosing.

Secondary Endpoints And Cognitive Benefits

In addition to reaching the primary endpoint, the twice-daily treatment arm generated statistically significant improvements across key secondary metrics.

These included reduced disease severity and lower scores on the Marder positive symptom factor.

Furthermore, participants with baseline cognitive impairment demonstrated meaningful cognitive gains on the Cogstate testing battery. This cognitive benefit was independent of reductions in overall psychotic symptoms.

Safety Findings and Regulatory Strategy

ML-007C-MA demonstrated a favorable safety profile across studied regimens, recording an overall all-cause discontinuation rate of 19.9%.

Treatment-emergent side effects were mostly mild and cholinergic in nature, with no drug-related serious adverse events reported.

Looking ahead, the biopharmaceutical company plans to meet with the FDA for an End-of-Phase 2 discussion to outline Phase 3 trial designs.

MapLight is also preparing site identification for confirmatory studies, continuing its VISTA trial for Alzheimer’s disease psychosis, and arranging regulatory meetings for its autism spectrum disorder asset, ML-004.

In June, MapLight Therapeutics reported mixed topline results from its Phase 2 IRIS study of ML-004 in patients with autism spectrum disorder.

The trial missed the trial’s primary endpoint while demonstrating clinically meaningful improvements in irritability among a subgroup of adolescents.

MPLT Stock Price Activity: MapLight Therapeutics shares were down 65.75% at $12.52 at the time of publication on Monday, according to Benzinga Pro data.

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