Synaptogenix had an admirable goal, but, unfortunately, aspirations don’t directly translate to clinical success, particularly in neurodegenerative diseases.
That was the major takeaway from the company’s phase 2 trial failure reported Friday, which revealed that bryostatin-1 didn’t improve severe impairment scores in patients with advanced Alzheimer’s disease. The company said that the National Institutes of Health-sponsored study found that patients treated with the med experienced a 1.4-point increase on a symptom scale, compared to 0.6 for those on placebo. That was not a statistically significant difference, failing the trial’s primary endpoint.
The company was disappointed and will conduct a full review of the results before announcing the next steps for bryostatin-1, Synaptogenix CEO Alan Tuchman, M.D., said in the release.
Investors also expressed their concerns, with Synaptogenix’s shares plummeting nearly 73% to $1.28 a piece in the opening hour of trading from a Thursday closing price of $4.73. The biotech still had $38.5 million in cash reserves as of Dec. 15, a ray of financial light after reeling in a $15 million private placement in November.
But the results vanquish hope that the company would be able to show meaningful progress at slowing disease progression among patients with advanced Alzheimer’s. It was a valiant attempt, given that the efforts of larger pharmas to develop treatments for patients with early-stage disease have often proved equally unsuccessful.
The trial recruited 100 patients following promise from earlier pilot trials, with participants randomized 1:1 to receive either the treatment or placebo. When the recruitment was completed in April, Chief Scientific Officer Daniel Alkon, M.D., said in a release that an improvement on the symptom score of at least four points above baseline would likely register as significantly meaningful.
The failure follows a mixed bag of highly anticipated results from a number of top pharmas as phase 3 results of their Alzheimer’s hopefuls begin to readout. Eisai’s lecanemab continues to stand out, even in light of two patient deaths that are potentially tied to the drug. The therapy was found to slow clinical decline among early Alzheimer’s patients by 27%. The same promise couldn’t be said about Roche’s gantenerumab, however, which wasn’t able to keep pace. Last but not least is Eli Lilly’s donanemab, which is slated to read out a phase 3 trial by the middle of 2023.