Phase 2 Clinical Trial of AL001 for Major Depressive Disorder: Initiation Date Set for Late 2025

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Alzamend announced its plans to initiate a highly anticipated phase 2 clinical study of AL001, a novel lithium-delivery system for the treatment of patients with major depressive disorder (MDD), in late 2025. Alzamend aims to explore the unique properties of AL001, namely its potential to provide the benefits of marketed lithium salts while mitigating or avoiding currently experienced toxicities associated with lithium.^1,2

AL001 is designed to favorably distribute lithium in the brain, resulting in lower exposure of other organs and an improved safety profile compared with currently marketed lithium salts. Lithium salts currently approved by the US Food and Drug Administration (FDA), carbonate and citrate, are limited by a narrow therapeutic window that requires regular therapeutic drug monitoring of plasma lithium levels and blood chemistry by a clinician in order to mitigate adverse events. Although lithium does not have an FDA approved indication for antidepressant augmentation in MDD, it has been prescribed off-label for this purpose for many years. Historically, a wide variety of medications have been used in this capacity, but lithium is one of the few agents that has demonstrated efficacy in multiple randomized controlled trials. Although the ideal role for lithium augmentation has yet to be established, there is evidence to support the clinical practice of adding lithium to antidepressants for the treatment of major depressive disorder.

“With AL001, we can potentially introduce a next-generation lithium treatment that offers enhanced safety, better brain targeting, and no need for therapeutic drug monitoring, promising a leap forward from the current, burdensome options,” sais Stephan Jackman, chief executive officer of Alzamend. “This advancement stands to potentially enhance the lives of over 21 million Americans suffering from MDD by providing a more effective and user-friendly therapeutic option, potentially reshaping current treatment paradigms and improving patient quality of life substantially.”

The current study, done in collaboration with Massachusetts General Hospital (Alzamend’s contract research organization), could demonstrate AL001’s targeted effectiveness and reduced systemic adverse effects, thus providing another treatment option for patients with MDD. Notably, previous research in mice has shown that AL001 ensures better brain absorption while maintaining lower levels of lithium in the blood. Additionally, results from Alzamend’s completed phase 2A multiple-ascending dose study of AL001 in patients with Alzheimer disease and healthy subjects identified a maximum tolerated dose, as assessed by an independent safety review committee. This maximum tolerated dose is designed to be unlikely to require therapeutic drug monitoring, while providing lithium at a relatively modest but effective dose.

References

1. Alzamend Neuro Announces Initiation Date of Phase II Clinical Trial of AL001 for Treatment of Major Depressive Disorder to take Place at Massachusetts General Hospital. News release. March 18, 2025. Accessed March 19, 2025. https://www.globenewswire.com/news-release/2025/03/18/3044409/0/en/Alzamend-Neuro-Announces-Initiation-Date-of-Phase-II-Clinical-Trial-of-AL001-for-Treatment-of-Major-Depressive-Disorder-to-take-Place-at-Massachusetts-General-Hospital.html

2. Alzamend Neuro announces initiation date of first phase II clinical trial of AL001 to take place at Massachusetts General Hospital. News release. February 25, 2025. Accessed March 19, 2025. https://www.globenewswire.com/news-release/2025/02/25/3031992/0/en/Alzamend-Neuro-Announces-Initiation-Date-of-First-Phase-II-Clinical-Trial-of-AL001-to-take-Place-at-Massachusetts-General-Hospital.html