Anavex Life Sciences Corp (NASDAQ: AVXL) has announced encouraging results from its third phase double-blind, randomized, placebo-controlled AVATAR study of ANAVEX 2-73 (blarcamesine) in adult female Rett syndrome patients.
ANAVEX 2-73 met study endpoints
ANAVEX 2-73 demonstrated considerable improvement relative placebo for the primary efficacy objective and secondary efficacy endpoints. Also, the convenient once per day oral liquid doses of ANAVEX 2-73 of up to 30mg were well tolerated with amazing drug compliance. It is vital to note that Rett syndrome is a chronic central nervous system condition resulting from a spontaneous alteration of one gene, MECP2.
ANAVEX®2-73 activates the sigma-1 receptor (SIGMAR1).SIGMAR1 activation appears to restore homeostatic body function and is important for restoring brain cell balance and promoting neuroplasticity, according to research findings. Recent independent discoveries add to our understanding of SIGMAR1 activation’s favorable implications as a compensating mechanism for chronic CNS disorders.
ANAVEX 2-73 induced a substantially important and clinically significant improvement of 72.2 in the primary objective, RSBQ AUC, relative to 38.5% in placebo. Equally, the secondary endpoints show clinically meaningful and statistically significant improvement.
Principal study investigator Professor Terence O’Brien stated, “The outcome of this trial has confirmed the promising results of the early lower-dose study in adults with Rett syndrome. ANAVEX®2-73 was not only safe but it also demonstrated clinically meaningful improvements in multiple common areas of impairment, which are known to impair the quality of life of girls and women affected by the disorder.”
ANAVEX 2-73 can benefit drug-responsive patients
The promising findings point to a high chance of significant benefits in younger, more drug-responsive Rett syndrome patients, like those enrolled in the current pediatric EXCELLENCE research. In addition, the Phase 3 AVATAR study’s consistent effectiveness across both primary and secondary endpoints demonstrates ANAVEX 2-73’s promise for treating Rett syndrome, as shown by a previous Phase 2 study.
The company’s Chief Scientific Officer, Walter Kaufmann, said, “This strong body of data opens the possibility of successful treatment for both adults and children with Rett syndrome and early interventions for modifying the course of the disease.”
