Dive Brief:
- A small cohort of people with mild to moderate Alzheimer's disease who took a daily dose of Anavex 2-73 showed a stabilization of cognitive and functional measures at 41 weeks as compared to baseline, Anavex Life Sciences said Nov. 22 in an update on its Phase 2a study.
- Overall, 32 patients have participated in the study, which seeks to establish safety, tolerability and maximum tolerated dose for Anavex 2-73. The New York-based biotech company said planning is underway for a larger study.
- Anavex said patients tolerated oral daily dosing well and there were no significant adverse events from the investigational treatment, which targets cellular homeostasis.
Dive Insight:
For decades, clinical trials to develop pharmaceutical treatments for Alzheimer's – a degenerative brain disease affecting more than 5 million Americans – have been marked by a high level of failure. Scientists remain uncertain about its causes, and current therapies (comprised of several FDA-approved products) may temporarily improve symptoms but don't slow or halt the devastating disease's progression.
The day after Anavex announced its positive results from the small trial, Eli Lilly & Co. announced the conclusion of its highly-anticipated EXPEDITION 3 trial. The big pharma reported a third failure for the drug, raising the skepticism around the amyloid-beta hypothesis, which assumes the decline in cognition is based on an accrual of these plaques in the brain.
Against this backdrop, Anavex's shares rallied with the latest news. Some investors have expressed doubts about the drug's efficacy, but others said the company's prospects are improved and that further positive updates from the Anavex 2-73 clinical trial could mean "strong upside" for the biotech's stock.
"Although this is an open label study with 32 patients, I have never seen mild-to-moderate Alzheimer’s patients maintain near baseline cognitive and activities of daily living function and positive correlation with all other measures over a 41-week trial period in any prior study with an approved or experimental drug," said George Perry, dean and professor at the University of Texas at San Antonio and Editor-in-Chief of the Journal of Alzheimer’s Disease.
"It is quite plausible that complex [central nervous system] diseases like Alzheimer’s may require a comprehensive approach, including restoration of cellular homeostasis," Perry added in a statement accompanying Anavex's update.
Anavex President and CEO Christopher Missling cautioned that, while data from the Anavex 2-73 trial are encouraging, "these endpoints are exploratory" and must be confirmed by a larger study. Yet, information from the biotech's Phase 2a study "might allow us to also target shorter-term endpoints including behavioral and psychological symptoms," he said.