FDA Clears Neuraly to Begin Phase 2 Trial of NLY01 in Alzheimer’s Patients

The U.S. Food and Drug Administration has given clearance for a Phase 2B clinical trial to evaluate the safety, tolerability, and efficacy of Nueraly‘s investigational therapy NLY01 in people with Alzheimer’s disease.

The randomized, double-blinded, placebo-controlled trial is expected to enroll more than 500 people with mild cognitive impairment due to Alzheimer’s disease at more than 100 sites in the U.S., Canada, and Europe. Results from the trial are expected by the end of 2023.

“Initiation of this Phase 2 study in Alzheimer’s disease represents an important milestone in the ongoing advancement of NLY01,” Seulki Lee, PhD, president and CEO of Neuraly, said in a press release.

NLY01, which was initially developed by researchers at Johns Hopkins University, works by activating a protein called glucagon-like peptide-1 receptor. This protein is highly expressed on microglia — a type of immune cell in the brain that plays a central role in controlling brain inflammation.

Inflammation in the brain, called neuroinflammation, is thought to be involved in the progression of numerous neurological diseases, including Alzheimer’s, Parkinson’s disease, and amyotrophic lateral sclerosis. Preclinical research has suggested that NLY01 can lessen this inflammation, which may ease symptoms in turn.

“Our internal studies have confirmed the same mechanism of action underlies the neuroinflammation observed in animal models of Alzheimer’s and Parkinson’s disease,” said Viktor Roschke, PhD, Neuraly’s chief scientific officer. “These findings reinforce our belief in the potential of NLY01 in treating both of these diseases.”

Neuraly previously funded a first-in-human Phase 1 clinical trial (NCT03672604) in which NLY01 was given via under-the-skin (subcutaneous) injections to healthy volunteers. The treatment was found to be well-tolerated in the study.

In addition to the newly-announced trial in Alzheimer’s, Neuraly is currently conducting a Phase 2 clinical trial of the investigational medication in people with early untreated Parkinson’s disease. The trial (NCT04154072) aims to enroll about 240 patients, age 30–80, in the U.S. and Canada. Like the Alzheimer’s study, this trial is designed to test the safety, tolerability, and efficacy of NLY01. Enrollment is currently ongoing at multiple sites in the U.S.; more information is available here.

“Alzheimer’s has proven to be a particularly challenging disease to address therapeutically,” Lee said. “We believe that targeting neuroinflammation via this novel mechanism of action represents a compelling pathway with the potential to be a disease-modifying agent for both Alzheimer’s and Parkinson’s disease.”