Phase 2 Clinical Study of AL001 Initiated for Bipolar Disorder Type 1

Alzamend Neuro has announced the initiation of a phase 2 clinical study of AL001 in patients diagnosed with bipolar disorder (BD) type 1.¹ AL001 is a novel lithium-delivery system that has the potential to provide the benefits of marketed lithium salts while mitigating or avoiding currently experienced toxicities associated with lithium.

"The initiation of this phase 2 trial in patients with BD marks a pivotal milestone for Alzamend," said Stephan Jackman, the chief executive officer of Alzamend. "Our goal with AL001 is to optimize brain lithium delivery while reducing systemic exposure and potentially eliminating the need for burdensome TDM. If successful, AL001 could meaningfully improve treatment outcomes for over 43 million Americans living with BD, other neuropsychiatric conditions, and neurodegenerative diseases."¹

Investigators of the study will utilize a crossover design intended for multiple 6-subject cohorts. Following screening, participants will be randomized into 1 of 2 treatment sequences: AB (AL001 followed by lithium carbonate) or BA (lithium carbonate followed by AL001). Each treatment period consists of 14 days of 3-times daily dosing. A washout period of 14 days is planned between treatment periods. During days 14 and 15 of each treatment period, participants will undergo intensive 24-hour lithium pharmacokinetic blood sampling along with advanced magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS) neuroimaging.

The MRI and MRS neuroimaging methods were developed by the lab of Ovidiu Andronesi, MD, PhD, the study's principal investigator, an associate professor of radiology at Harvard University, and the director of multinuclear metabolic imaging at Martinos Center for Biomedical Imaging at Harvard Medical School. The study also incorporates a specialized, engineered head coil developed by Tesla Dynamic Coils BV, designed to enable high-resolution, whole-brain lithium imaging. This approach enables investigators to simultaneously quantify lithium concentrations in blood and brain, including individual structures, as well as assessment of brain and individual brain structure chemistry, metabolism, and biomarker effects.

This phase 2 study builds upon Alzamend's prior "Lithium in Brain" clinical trial in healthy subjects,² which completed its clinical portion back in November 2025; Topline data are expected by the end of this month, March 2026. The current BD study evaluates AL001's ability to enhance lithium delivery to targeted brain regions while reducing systemic exposure—an approach designed to address the long-standing safety and tolerability limitations associated with traditional lithium salts.

Lithium, renowned for its efficacy as a first-line therapy for manic episodes and maintenance in BD, has long been underutilized due to the complexities of therapeutic drug monitoring (TDM). Currently FDA-approved lithium salts are limited; they have a narrow therapeutic window that requires regular TDM of plasma lithium levels and blood chemistry by a clinician to mitigate potential adverse events. Additionally, conventional lithium salts are eliminated relatively quickly, so multiple administrations throughout the day are required to safely reach therapeutic plasma concentrations. AL001 could thus improve treatment outcomes by offering a treatment that potentially eliminates the need for lithium TDM.

In prior nonclinical studies, AL001 demonstrated higher lithium concentrations in brain tissue at lower doses compared to lithium carbonate. Alzamend's phase 2A multiple-ascending dose study further evaluated safety and tolerability under steady-state conditions in participants with mild to moderate Alzheimer disease and in healthy adult and older adult subjects with adequate renal function. AL001 was well tolerated across all dose levels, and the selected maximum tolerated dose for further development was determined based on maintaining plasma lithium levels below those associated in the medical literature with potential toxicity.

The maximum tolerated dose, as assessed by an independent safety review committee, provided AL001's lithium at a lithium carbonate equivalent dose of 240 mg 3-times daily. This dose was selected to maintain plasma lithium concentrations below levels commonly associated with toxicity and is risk-mitigated for use in fragile populations. It is also designed to be unlikely to require routine lithium TDM.

There are also plans to expand the program to further trials in major depressive disorder, Alzheimer disease, and posttraumatic stress disorder.

References

1. Alzamend Neuro initiates phase II clinical trial of AL001 "lithium in brain" study in patients with bipolar disorder in collaboration with Massachusetts General Hospital. News release. March 16, 2026. Accessed March 17, 2026. https://ir.alzamend.com/news-events/press-releases/detail/98/alzamend-neuro-initiates-phase-ii-clinical-trial-of-al001

2. Alzamend Neuro initiates first phase II clinical trial of AL001 “lithium in brain” study taking place at Massachusetts General Hospital. News release. May 13, 2025. Accessed March 17, 2026. https://ir.alzamend.com/news-events/press-releases/detail/91/alzamend-neuro-initiates-first-phase-ii-clinical-trial-of