AL001 · program
AL001 (lithium–salicylate–L-proline) for Bipolar disorder
Indications for AL001: Bipolar disorder · Phase 1/2 Alzheimer's disease · Phase 1/2
Alzamend Neuro's AL001 — an ionic-cocrystal lithium-delivery system (lithium with salicylate and L-proline) — in bipolar I disorder, the first patient population of its 'Lithium in Brain' program at Massachusetts General Hospital. The recruiting Phase 1/2 crossover study (NCT07540338, AL001-BD01; registry Phase 1|2, sponsor-branded Phase II) randomizes bipolar I patients in six-subject cohorts to AL001 then lithium carbonate or the reverse — 14 days of three-times-daily dosing per period with a 14-day washout — comparing 24-hour lithium blood PK paired with MRI/MRS brain imaging on days 14–15 of each period. It builds directly on the healthy-subjects study (NCT06921590, clinical phase completed November 2025), whose qualitative pharmacodynamic data (April 2026) showed AL001 reduced myo-inositol in 17 of 18 brain regions versus 8 of 18 for lithium carbonate and left glutamate largely unperturbed where lithium carbonate reduced it across all 18 regions. The sponsor guided bipolar topline data to Q3 2026 at initiation (March 2026); registry first-participant date 2026-05-11, estimated primary completion December 2026. MDD, PTSD and Alzheimer's studies are announced to follow (not yet registered).
Development timeline
- UpcomingTopline lithium blood/brain PK and MRS data from the bipolar I 'Lithium in Brain' crossover study (AL001 vs lithium carbonate) at MGH.↗
- Bipolar I 'Lithium in Brain' study (NCT07540338, AL001-BD01) reached its registry actual start date and is recruiting at MGH. Alzamend announced initiation on 2026-03-16 with topline guided to Q3 2026; the registry's actual start (first participant) is 2026-05-11, the date used here.
- Trial startedAlzamend's AL001 bipolar I 'Lithium in Brain' study starts at Massachusetts General Hospital
- pendingHealthy-subjects 'Lithium in Brain' study: qualitative MRS pharmacodynamics show AL001 reduced myo-inositol in 17 of 18 brain regions (vs 8 of 18 for lithium carbonate) with minimal glutamate effects, where lithium carbonate reduced glutamate across all 18 regions.↗
Readouts
- Q3 2026AnticipatedTopline dataNCT07540338
Topline lithium blood/brain PK and MRS data from the bipolar I 'Lithium in Brain' crossover study (AL001 vs lithium carbonate) at MGH. ↗
- 2026-04-07ReportedTopline datapendingNCT06921590
Healthy-subjects 'Lithium in Brain' study: qualitative MRS pharmacodynamics show AL001 reduced myo-inositol in 17 of 18 brain regions (vs 8 of 18 for lithium carbonate) with minimal glutamate effects, where lithium carbonate reduced glutamate across all 18 regions. ↗
Clinical trials in Bipolar disorder
NCT07540338AL001-BD01Phase 1/2Recruitingn=20
A Study to Investigate Lithium Brain/Plasma Pharmacokinetics and Safety of an AL001 Oral Capsule Compared to a Marketed Immediate-release Lithium Carbonate Capsule in Subjects With Bipolar I Disorder
NCT06921590AL001-ALZ03Phase 1Completedn=15
A Study to Investigate Lithium Brain/Plasma Pharmacokinetics and Safety of an AL001 Oral Capsule Compared to a Marketed Immediate-release Lithium Carbonate Capsule (Healthy Subjects)
Formulations
| Formulation | Route | Regimen | Pharmacokinetics |
|---|---|---|---|
| AL001 oral capsule (three times daily)ionic cocrystal (lithium–salicylate–L-proline) Oral capsule. The Phase 1/2A multiple-ascending-dose study (NCT05363293) tested ascending doses in mild-to-moderate Alzheimer's patients and healthy non-elderly/elderly adults; the MGH 'Lithium in Brain' crossover studies dose three times daily for 14 days per period head-to-head against immediate-release lithium carbonate. | Oral | Three times daily | — |
Mechanism of action
The active moiety is lithium, whose canonical molecular actions include direct inhibition of glycogen synthase kinase-3 (GSK-3) and of inositol monophosphatase (IMPase). In Alzheimer's disease the rationale leans on lithium's GSK-3β inhibition — GSK-3β is a principal tau kinase, and low-dose lithium has been repeatedly explored as a disease-modifying candidate in AD — combined with AL001's delivery premise: an ionic cocrystal of lithium with salicylate and L-proline engineered to raise brain lithium relative to systemic exposure versus lithium carbonate, potentially enabling chronic dosing in an elderly population without the toxicity and therapeutic-drug-monitoring burden of marketed lithium salts.
| Target | Action | Affinity |
|---|---|---|
| GSK-3βprimaryGSK3B | Inhibitor | —ⓘ |
| IMPaseIMPA1 | Inhibitor | —ⓘ |
← Full AL001 compound page (identity, identifiers, all indications)
Sources
- Alzamend Neuro bipolar-study initiation PR (2026-03-16) — states the completed AD MAD study and announces future Alzheimer's, MDD and PTSD studies — Alzamend Neuro, Inc. via PRNewswire
- Alzamend Neuro Reports Encouraging Pharmacodynamic Data from AL001 'Lithium in Brain' Study at Massachusetts General Hospital (2026-04-07; qualitative MRS metabolite results, n=6 healthy subjects) — Alzamend Neuro, Inc. press release via BioSpace
- NCT05363293 (AL001-ALZ02) — Phase 1/2 multiple-ascending-dose study of AL001 in mild-to-moderate Alzheimer's patients and healthy adults; COMPLETED, primary completion 2023-04-14 — ClinicalTrials.gov (U.S. National Library of Medicine)
- NCT06921590 (AL001-ALZ03) — Phase 1 lithium brain/plasma PK crossover of AL001 vs lithium carbonate in healthy subjects; COMPLETED, primary completion 2025-12-15 — ClinicalTrials.gov (U.S. National Library of Medicine)
- NCT07540338 (AL001-BD01) — Phase 1/2 lithium brain/plasma PK crossover of AL001 vs lithium carbonate in bipolar I disorder; RECRUITING, started 2026-05-11, est. primary completion 2026-12 — ClinicalTrials.gov (U.S. National Library of Medicine)
- Validating GSK3 as an in vivo target of lithium action (open-access; lithium's direct inhibition of GSK-3 and IMPase) — PubMed Central