A year ago, we featured a Q & A with CEO Christopher U. Missling, Ph.D., from Anavex Life Sciences Corp. Anavex is a biopharmaceutical company based in New York City and serves as one of ACT-AD’s sponsors. We recently circled back with the team at Anavex to get an update. Please note that the views and opinions of Anavex are purely its own.
Q: In our last interview, we discussed your lead drug candidate, ANAVEX 2-73. Could you give us an update on this?
A: Last year, we started the Phase 2a trial in mild-to-moderate Alzheimer’s patients with the oral compound, ANAVEX 2-73. It acts by selectively activating the stress reducing protein, the sigma-1 receptor, and muscarinic receptors, which are believed to reduce cognitive impairment. In November 2015, we presented five-week data, and recently, at AAIC 2016, we presented preliminary 31-week safety and exploratory efficacy data. So we have been actively advancing the trial.
Q: What have you found in this trial?
A: Preliminary Phase 2a data demonstrated that the study met the primary objective of safety as ANAVEX 2-73 was well tolerated and results were consistent with prior Phase 1 clinical trial data. The secondary objectives were also met, with ANAVEX 2-73 showing what appears to be a converging and consistent response for all quantitative endpoints. This includes cognitive and functional measures: Mini Mental State Examination (MMSE), Alzheimer’s Disease Co-operative Study – Activities of Daily Living (ADCS-ADL), as well as Cogstate and electroencephalographic activity and event-related potentials (EEG/ERP), which consistently demonstrated improvement and stabilization from baseline in a patient population that would normally be expected to experience ongoing cognitive and functional decline.
Additionally, the Phase 2a data demonstrated positive unexpected therapeutic response events, such as improved mood, improved social engagement, and increased independent activities through 31 weeks. ANAVEX 2-73 also showed preliminary beneficial effects on reducing insomnia, agitation, and anxiety, which may suggest additional important roles of ANAVEX 2-73 in conditions correlated with multiple underlying psychiatric diseases and dementia.
The purpose of this adaptive and Population PK Phase 2a trial was specifically designed to get all relevant information needed to be able to move as efficiently as possible into larger studies. Given the positive data, we are encouraged to proceed with the plan to prepare a larger Phase 2/3 trial, for which planning is underway.
Q: What are your thoughts on the next steps for this drug?
A: The new findings this year were the observation of positive efficacy with ANAVEX 2-73 in neurodevelopmental rare disease models such as Rett syndrome, Infantile Spasms and Fragile X, Autism-related disorders. The distinct mechanistic nature of ANAVEX 2-73 to address both neurodegenerative and neurodevelopmental diseases, which have cognitive impairment in common, suggests the compound could be a candidate treatment for a precision medicine approach across a spectrum of different neurological and psychiatric diseases alike.
Two orphan designations by the FDA for ANAVEX 2-73 support the exploration of ANAVEX 2-73 as a potential therapeutic in these disorders coupled with positive human safety data.
Q: What motivates Anavex to support the work of ACT-AD?
A: We understand the pursuit to provide relief to all those affected by Alzheimer’s disease and dementia requires a team effort to solve the current unmet need of a lack of a viable therapeutics. We find the Accelerate Cure/Treatments for Alzheimer’s Disease (ACT-AD) Coalition to be one of the leaders in the Alzheimer’s research space, and we look forward to continuing to work with ACT-AD to advance awareness about the importance of this research.