PROBLEM

Alzheimer’s disease is a progressive brain disorder that destroys memory and thinking, affecting 6–7 million people in the U.S. and over 50 million worldwide. There is currently no cure, and available treatments only temporarily manage symptoms or modestly slow disease progression. Healthy synapses are essential for forming and storing memories. Loss of these connections drives cognitive decline and dementia severity. Therapies that protect or strengthen these brain connections may help preserve memory, slow brain degeneration, and improve daily functioning. Increased extracellular glutamate concentration can lead to neuronal damage or death. EAAT2 is the major glutamate transporter and functions to remove glutamate from synapses. An increase in EAAT2 protein expression and function can provide a means to prevent insufficient glutamate re-uptake and consequently reduce neuronal damage.

SOLUTION

LH-001 is a clinical-stage, novel, small molecule activator of EAAT2 that strengthens and improves brain connections. In preclinical studies, LH-001 has been shown to be effective, stable, and safe. In disease models, it restores synaptic integrity, rebalances brain signaling, and improves memory and learning. Pre-clinical efficacy has been shown in Alzheimer's disease, Amyotrophic lateral sclerosis, Gulf War Illness, and epileptic mouse models.

DEVELOPMENT

IND-enabling studies have been completed and a Phase 1 first-in-human clinical trial in healthy volunteers is open, currently conducting a single ascending dose study. LH-001 is protected by a robust patent portfolio, with issuances in the United States, Europe, China, Japan, Hong Kong, Israel, and Australia.

Seeking opportunities for out-licensing and collaboration