MapLight Therapeutics Announces Completion Of Phase 1 Clinical Trial For ML-007, A Novel Therapy For The Treatment Of Schizophrenia And Dyskinesias

First in human study demonstrates positive safety and tolerability profile for MapLight’s M1/M4 muscarinic agonist therapy

SAN FRANCISCO, August 18, 2022 – MapLight Therapeutics today announced it has completed its Phase 1 clinical trial evaluating ML-007, the second clinical compound developed using the MapLight platform. ML-007 is an M1/M4 muscarinic agonist that targets circuits thought to be disrupted in neurologic conditions such as schizophrenia and dyskinesia. ML-007 was studied in a randomized, double blind, placebo-controlled single ascending dose trial to evaluate its safety, tolerability, pharmacokinetic, and pharmacodynamic profile.

ML-007 was well-tolerated with no serious adverse events. The compound produced the predicted peripheral effects of muscarinic activation, demonstrating on-target activity, and these effects were dose limiting as expected. Co-treatment with a peripherally-restricted anticholinergic was effective in blocking objective measures of peripheral muscarinic symptoms. The pharmacokinetics of ML-007 were favorable with fast absorption, dose-proportional exposure, and low inter-subject variability. Target CSF concentrations were achieved at well tolerated doses.

“The successful completion of our ML-007 Phase 1 clinical trial is a major milestone for MapLight,” stated Christopher Kroeger, M.D., MBA, Chief Executive Officer and Founder. “This study is a significant step forward for our M1/M4 muscarinic agonist program, a program that we believe will yield a best in class therapy for patients suffering with schizophrenia and dyskinesia.” 

The Phase 1 single ascending dose clinical trial for ML-007 enrolled 58 healthy adult volunteers aged 18 to 45 across 7 cohorts. Six cohorts received escalating oral doses of ML-007, successfully providing data to identify a maximum tolerated dose of the single agent. Subjects in a seventh cohort received ML-007 in combination with a peripherally restricted muscarinic antagonist, which was well tolerated and without severe or serious adverse events.