Encouraging Pharmacodynamic Data From Phase 2 Clinical Trial of AL001

Alzamend Neuro today announced encouraging pharmacodynamic findings from a brain magnetic resonance spectroscopy (MRS) analysis conducted in healthy participants (N=6) in a clinical trial of AL001 conducted at Massachusetts General Hospital.¹

AL001 is a patented ionic cocrystal formulation of lithium combined for delivery with L-proline and salicylate, designed to deliver a full therapeutic amount of lithium to the brain with less systemic exposure than lithium carbonate. It is currently being studied as a treatment for Alzheimer disease, bipolar disorder type 1 (BD), major depressive disorder (MDD), and posttraumatic stress disorder (PTSD).

The study assessed changes in 5 key brain metabolites across 18 brain regions when participants received 2-weeks of blood bioequivalent and lithium-dose equivalent AL001 or lithium carbonate relative to baseline. The early data suggest that AL001 may work like lithium carbonate by selectively impacting brain chemicals where needed; however, AL001 seems to leave other, healthy brain chemicals more undisturbed than lithium carbonate, which could meaningfully impact tolerability. These results need to be further statistically confirmed in additional patient populations, the first of which is currently underway.

As to the preliminary pharmacodynamic MRS findings, investigators believe AL001 has a distinct neurochemical footprint. Participants taking AL001 has multiple brain chemicals that trended downward, while the same chemicals trended upward in these participants when they took lithium carbonate. This finding suggests that AL001 may interact with the brain in a distinct manner and could have a less disruptive effect on healthy brain tissue than lithium carbonate.

Additionally, both AL001 and lithium carbonate reduced levels of a key brain chemical called myo-inositol, as lithium-based treatments are intended to do. Notably, AL001 affected this target in nearly twice as many brain regions (17 out of 18) as lithium carbonate (8 out of 18), meaning AL001 may deliver the intended lithium benefits more broadly throughout the brain.

Furthermore, glutamate was largely undisturbed in 10 of the 18 brain regions of participants after 2 weeks of treatment AL001; oppositely, 2 weeks of lithium carbonate seemingly caused disruptions to glutamate levels across every brain region measured. This suggests AL001 may have fewer adverse effects than lithium carbonate over time, but the finding needs to be confirmed over a longer duration of exposure.

“Lithium carbonate has been a cornerstone of psychiatric treatment for over 55 years, but its harsh [adverse] effect profile has always limited how widely and how long it can be used,” said Stephan Jackman, the chief executive officer of Alzamend. “These findings suggest AL001 may finally change that equation, delivering what lithium does best, without much of what makes it difficult to tolerate. That is a potential game changer for 43+ million Americans living with BD, Alzheimer's, MDD, and PTSD. We are seeking to confirm these findings in larger, adequately powered studies.”

Based on these initial findings, Alzamend has identified a set of pharmacodynamic hypotheses to be tested in future confirmatory studies of participants with Alzheimer disease, BD, MDD, and PTSD. They are:

• “AL001 causes less disruption to healthy brain tissue than lithium carbonate, potentially resulting in fewer [adverse] effects and better long-term tolerability.”
• “AL001 produces the same beneficial brain response that makes lithium an effective treatment, by reducing a key brain chemical, myo-inositol, suggesting it works through the same proven mechanism of action as lithium carbonate, just with a potentially better safety profile.”
• “AL001 appears to leave glutamate levels in healthy brain tissue largely undisturbed, a potentially important advantage over lithium carbonate, which appears to disrupt glutamate broadly. Stable glutamate levels in healthy tissue may mean fewer cognitive [adverse] effects and better long-term tolerability for patients.”
• “Unlike lithium carbonate, AL001 may help preserve the health of brain cell membranes in healthy tissue, the protective outer layer of brain cells that play a critical role in how they function and communicate. If confirmed, this could mean AL001 is better tolerated by patients over the long-term than lithium carbonate.”

This news comes on the heels of Alzamend’s recent announcement of the initiation of a phase 2 clinical study of AL001 in patients diagnosed with BD type 1, in which investigators will evaluate 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.²

References

1. Alzamend Neuro reports encouraging pharmacodynamic data from phase II clinical trial of AL001 "Lithium in Brain" study in a trial conducted at Massachusetts General Hospital. News release. April 7, 2026. Accessed April 7, 2026. https://www.prnewswire.com/news-releases/alzamend-neuro-reports-encouraging-pharmacodynamic-data-from-phase-ii-clinical-trial-of-al001-lithium-in-brain-study-in-a-trial-conducted-at-massachusetts-general-hospital-302735711.html

2. Kuntz L. Phase 2 clinical study of AL001 initiated for bipolar disorder type 1. Psychiatric Times. March 17, 2026. https://www.psychiatrictimes.com/view/phase-2-clinical-study-of-al001-initiated-for-bipolar-disorder-type-1