Synaptogenix has reported topline findings from the National Institute of Health (NIH)-sponsored Phase II clinical trial of its lead therapeutic candidate, Bryostatin-1, to treat advanced Alzheimer’s disease (AD).

The double-blind, randomised, placebo-controlled trial is designed to evaluate the long-term efficacy of bryostatin-1 versus placebo to treat advanced and moderately severe AD in the absence of memantine. 

The NIH units, the National Institute on Aging (NIA) and the National Cancer Institute (NCI), offered financial support for the trial.

Variation from baseline in the severe impairment battery (SIB) total score following the conclusion of the second course of treatment at week 28 was the primary endpoint of the trial.

According to the findings, the trial failed to meet the primary endpoint with statistical significance.

At week 28, an average rise in the SIB total score of 1.4 points and 0.6 points were seen in the Bryostatin-1 and placebo arms, respectively.

Preclinical research data showed that Bryostatin offered regenerative mechanisms of action in Fragile X syndrome as well for other neurodegenerative ailments such as multiple sclerosis, stroke, and traumatic brain injury.

The therapeutic candidate has also received Orphan Drug Designation from the US Food and Drug Administration to treat Fragile X syndrome. 

Synaptogenix CEO Alan Tuchman said: “We are disappointed in the topline results from this Phase II trial. 

“Having just received the primary endpoint data, we are conducting a full review of all of the trial data to determine potential next steps and will provide an update of our plans when appropriate. 

“We are committed to creating value for our shareholders and our substantial balance sheet offers us flexibility as we determine the next steps forward.”