Fast Track Designation Granted: ML-007C-MA for Alzheimer Disease Psychosis

MapLight Therapeutic today announced that the US Food and Drug Administration (FDA) has granted Fast Track designation to ML-007C-MA, an investigational novel M1/M4 muscarinic agonist, for the treatment of hallucinations and delusions associated with Alzheimer disease psychosis (ADP).¹

ML-007C-MA—also known as ML-007C/PAC—is an oral, extended-release, fixed-dose combination of the investigational M1/M4 muscarinic agonist ML-007, coformulated with a peripherally acting anticholinergic. Developers designed ML-007C-MA to activate both M1 and M4 muscarinic receptors in the central nervous system to drive efficacy, while synchronizing the pharmacokinetics of the agonist and antagonist components to mitigate peripheral cholinergic adverse effects. As to safety and tolerability, ML-007C-MA is a well-tolerated treatment option that offers convenient dosing, while also achieving or exceeding CSF exposures that are expected to result in improvement across key symptom domains for patients with ADP.

“FDA’s Fast Track designation underscores the significant unmet need of the millions of people with ADP with no currently approved treatment options,” said Erin Foff, MD, PhD, the chief medical officer of MapLight. “This designation is an important milestone for the ML-007C-MA program that recognizes its potential to address the psychotic symptoms that frequently accompany the cognitive decline in people living with Alzheimer disease. We remain committed to working closely with the FDA to advance this program expeditiously through our ongoing phase 2 VISTA study.”

Approximately 40% of individuals with Alzheimer disease develop psychotic symptoms at some point throughout the course of their illness. As the disease progresses, the likelihood of developing these symptoms increases. Furthermore, ADP is associated with significantly poorer outcomes, including faster cognitive and functional decline, higher rates of institutionalization, and increased mortality.²

In a phase 1 clinical trial, ML-007C-MA demonstrated a generally favorable safety and tolerability profile with twice daily dosing in 270 healthy older adult participants, including 42 healthy elderly volunteers who received active drug.³

“ML-007C-MA is well positioned as a potential treatment for ADP based on the favorable safety and tolerability profile observed in healthy elderly volunteers in phase 1 whose CNS exposures achieved or exceeded levels that we anticipate being clinically relevant,” said Foff.³

Enrollment is currently ongoing for the phase 2 VISTA study, which is a randomized, double-blind, placebo-controlled trial evaluating ML-007C-MA for the treatment of hallucinations and delusions associated with ADP. MapLight expects to enroll 300 participants in this trial. Topline results are expected in the latter half of 2027.

“There are no FDA-approved treatments for ADP and off-label use of antipsychotics with marginal efficacy and significant safety concerns can lead to poor patient outcomes,” said Christopher Kroeger, MD, MBA, the chief executive officer and founder of MapLight. “Muscarinic receptor agonism has shown promising results as a therapeutic strategy for ADP, and the initiation of the VISTA study marks an important milestone as we pursue parallel development of ML-007C-MA in schizophrenia, ADP, and potentially other neuropsychiatric conditions.”³

References

1. MapLight Therapeutics receives fast track designation for ML-007C-MA for Alzheimer’s disease psychosis. News release. January 5, 2025. Accessed January 5, 2025. https://www.globenewswire.com/news-release/2026/01/05/3212597/0/en/MapLight-Therapeutics-Receives-Fast-Track-Designation-for-ML-007C-MA-for-Alzheimer-s-Disease-Psychosis.html

2. Connors MH, Ames D, Woodward M, Brodaty H. Psychosis and clinical outcomes in Alzheimer disease: a longitudinal study. Am J Geriatr Psychiatry. 2018;26(3):304-313.

3. MapLight Therapeutics announces initiation of phase 2 trial of novel M1/M4 muscarinic agonist ML-007C-MA for the treatment of alzheimer’s disease psychosis. News release. September 17, 2025. Accessed January 5, 2025. https://maplightrx.com/maplight-therapeutics-announces-ml-007c-ma-phase-2-initiation-alzheimers-disease-psychosis/