Alzamend Neuro, Inc. announced that it has submitted an investigational new drug application to the U.S. Food and Drug Administration ("FDA") for the initiation of study AL001-MDD01, a Phase IIA plasma/brain pharmacokinetics clinical study of AL001 for adjunctive treatment of patients with MDD. Although lithium does not have an FDA approved indication for augmentation of an antidepressant in MDD, it has been prescribed off-label for this purpose for decades. While a wide variety of medications have been used historically in this capacity, 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 conventional antidepressants in pursuit of MDD removal. Lithium augmentation has been cited as a strategy for depressed patients not responding to an antidepressant, lithium prophylaxis for recurrent unipolar depression as an alternative to prophylaxis with an antidepressant, and for lithium's anti-suicidal properties, where appropriate. Lithium was the first mood stabilizer approved by the FDA and is still a first-line treatment option (cons considered the "gold standard") for BD but is underutilized perhaps because of the need for therapeutic drug monitoring ("TDM").

Lithium was the first drug that required TDM by regulatory authorities in product labelling because the effective and safe range of therapeutic drug blood concentrations is narrow and well-defined for treatment of BD when using lithium salts. Excursions above this range can be toxic, and below can impair effectiveness. AL001 is a novel lithium-delivery system that has the potential to provide benefits of marketed lithium salts while mitigating or avoiding currently experienced toxicities associated with lithium.

Results from Alzamend's recently completed Phase IIA multiple-ascending dose study of AL001 in Alzheimer's patients and healthy subjects identified a maximum tolerated dose ("MTD") that was vetted by an independent safety review committee. This MTD, which is designed to distribute more lithium to the brain but at lower systemic exposure, resulting in an improved safety profile compared to currently marketed lithium salts, was assessed to be unlikely to require TDM. After receipt of a " study may proceed" communication from the FDA, Alzamend plans to initiate a Phase IIA study to characterize AL001 improvements of lithium levels in the brain compared to a marketed lithium salt in MDD patients.

Alzamend anticipates that this program may, based on safety, qualify for a 505(b)(2)NDA pathway to FDA approval, which is available to new formulations of an approved drug.