The phase IIb/III study is scheduled to initiate enrollment of approximately 450 patients, randomized 1:1:1 to 2 different ANAVEX 2-73 doses or placebo.
“The phase IIa is still in the extension period, so there was a 57-week, 1-year study establishing safety, it’s a safe drug so far."
Harald Hampel, MD, PhD, MA, MSc, professor at Sorbonne Universities, Department of Neurology, presented results of the entire genome DNA and RNA analysis of all study participants in the phase IIa study of ANAVEX 2-73 for the treatment of early Alzheimer disease at the 2018 Alzheimer’s Association International Conference in Chicago, Illinois.
Hampel sat down with NeurologyLive to discuss these results and dive a bit deeper into the phase IIa study, which is still in the extension phase. The 57-week, 1-year study established a favorable safety, bioavailability, dose-response curve and tolerability/risk profile at doses between 10 mg and 50 mg of oral daily ANAVEX 2-73. A dose-dependent improvement in exploratory endpoints of cognition (MMSE) and function (ADCS-ADL) were also demonstrated. The study met both primary and secondary endpoints.
The positive data from the phase IIa study led to an extension observation study of 2 additional years, as well as another 2 years beyond the end phase to examine the toxicity over a 5-year period. Additionally, a phase IIb/III trial has been approved to launch where Anavex will aim to enroll approximately 450 patients randomized 1:1:1 to 2 different ANAVEX 2-73 doses or placebo assessing patients with early-stage Alzheimer disease.
The design of the study includes genomic precision medicine biomarkers, identified in the phase IIa study. Primary and secondary endpoints will assess safety, as well as cognitive and functional efficacy which will be measured through ADAS-Cog, ADCS-ADL and CDR-SB. The international study will begin in Australia and also include a few North American sites.