Alzamend Neuro, Inc. (Nasdaq: ALZN) has received a "Study May Proceed" notification from the U.S. Food and Drug Administration (FDA) to initiate a Phase IIA clinical trial of AL001, its next-generation lithium therapeutic drug candidate, in patients with bipolar disorder (BD) type 1.
The company plans to begin dosing patients in the first quarter of 2024 for the AL001-BD01 study. This advancement follows promising results from a recently completed Phase IIA multiple-ascending dose study of AL001 in Alzheimer's patients and healthy subjects, which identified a maximum tolerated dose (MTD) that may eliminate the need for therapeutic drug monitoring (TDM).
"Lithium was the first mood stabilizer approved by the FDA and is still a first-line treatment option considered the 'gold standard' for BD type 1," said Stephan Jackman, Chief Executive Officer of Alzamend. "If we are able to develop a next-generation lithium product that would not routinely require therapeutic drug monitoring, it would constitute a major improvement over current lithium-based treatments and positively impact the 7 million Americans afflicted with BD."
Novel Lithium Delivery System
AL001 represents a significant innovation in lithium therapy, utilizing a patented ionic cocrystal technology that delivers lithium via a therapeutic combination of lithium, proline, and salicylate. This formulation is designed to provide the established benefits of lithium while mitigating or avoiding the toxicities commonly associated with conventional lithium salts.
The drug candidate is engineered to favorably distribute lithium in the brain while reducing exposure to other body organs, potentially offering an improved safety profile compared to currently marketed lithium products. This approach addresses the "disadvantageously low ceiling for toxicity" that has historically limited the usefulness of lithium treatments.
Based on the favorable safety profile observed in completed studies and extensive safety data on the drug's constituent components, the AL001 development program may qualify for a Section 505(b)(2) New Drug Application (NDA) pathway for FDA approval, which could accelerate the regulatory process for this new formulation of an approved drug.
Addressing a Significant Mental Health Burden
Bipolar disorder, previously known as manic depression, affects approximately 3% of the U.S. population at some point in their lives. The condition is characterized by alternating periods of depression and abnormally elevated mood states that can each last from days to weeks.
BD is among the top 20 causes of disability worldwide and carries a high risk of suicide—6% of those with BD die by suicide over a 20-year period, while 30-40% engage in self-harm. The condition is also frequently accompanied by other mental health issues, including anxiety disorders and substance use disorders.
The development of improved lithium-based treatments could significantly impact patient care, as current lithium therapies require careful monitoring due to their narrow therapeutic window and potential for toxicity.
Expanding Therapeutic Pipeline
Alzamend Neuro is a clinical-stage biopharmaceutical company focused on developing novel treatments for neuropsychiatric conditions including Alzheimer's disease, bipolar disorder, major depressive disorder, and post-traumatic stress disorder.
In addition to AL001, the company's pipeline includes AL002, a patented method using a mutant-peptide sensitized cell as a cell-based therapeutic vaccine designed to restore a patient's immunological system's ability to combat Alzheimer's disease.
Both product candidates are licensed from the University of South Florida Research Foundation, Inc. under royalty-bearing exclusive worldwide licenses, reflecting the company's commitment to developing innovative approaches to addressing significant unmet needs in neuropsychiatric medicine.
With the FDA's clearance to proceed with the Phase IIA trial for bipolar disorder, Alzamend moves closer to potentially delivering a meaningful advancement in lithium therapy that could improve treatment options for millions of patients.
