MapLight Therapeutics (MPLT) Stifel 2026 Virtual CNS Forum summary

Company overview and platform

• Focuses on targeted circuit-specific therapeutics for CNS disorders, leveraging optogenetics, STARmap, and single-cell transcriptomics to identify and develop therapies.

• Pipeline includes ML-007 (muscarinic program), ML-004 (5-HT1B/1D agonist for autism), ML-009 (GPR52 PAM for hyperactivity/impulsivity), and 021 (M4 antagonist for Parkinson’s motor symptoms).

• ML-004 phase II top-line data expected Q3 2024; ML-009 and 021 to enter clinic next year.

ML-007 program and differentiation

• ML-007 combines an M1/M4 muscarinic agonist with a peripheral antagonist, aiming to improve tolerability by precisely matching PK exposures.

• Four phase I studies (275 patients, ~1,500 exposures) established a tight exposure window, resulting in an attractive safety and tolerability profile.

• Potential for once-daily dosing in schizophrenia and twice-daily in Alzheimer’s, with no fasting requirement and short titration periods.

• Preclinical data suggest superiority in cognition compared to COBENFY.

Clinical trial design and progress

• Phase II schizophrenia study: 3-arm (once daily, twice daily, placebo), 300 patients, endpoint is PANSS score change at 5 weeks; robust enrollment, top-line data expected Q3 2024.

• Dose selection based on achieving consistent CSF concentrations above ED50 from animal models; gastroretentive formulation extends half-life to 5-6 hours.

• Confident exposures for efficacy with both once-daily and twice-daily dosing.