Anavex Inc. (AVXL) stock gained by 4.29% at the last close while the AVXL stock price surged by 12.38% in the pre-market trading session. Anavex Life Sciences Corp. is a publicly listed pharmaceutical company focused on developing distinguishable therapies for the cure of neurodegenerative and neurodevelopmental diseases such as Alzheimer’s disease, Parkinson’s disease, Rett syndrome, and other CNS diseases, as well as pain and cancer.
What is happening?
The U.S. Phase 2 randomized, double-blind, placebo-controlled trial of ANAVEX 2-73 (blarcamesine) in adult woman patients with Rett syndrome found that a predictive biomarker of response established with SIGMAR1 mRNA expression correlates importantly with responses in initial diagnostic effectiveness endpoints.
The sigma-1 receptor is activated by ANAVEX 2-73. SIGMAR1 activation appears to restore full housekeeping function inside the body and is critical for restoring brain cell homeostasis and fostering neuroplasticity, according to research. Recent independent results add to our knowledge of SIGMAR1 activation as a compensatory strategy for chronic pain.
An unprompted mutation of one gene, MECP2, causes Rett syndrome, a persistent CNS illness. For the first time, a biomarker is linked to clinical effectiveness in Rett syndrome, according to this study. Increases in the mRNA expression of SIGMAR1, the gene coding for the receptor targeted by ANAVEX 2-73, were associated with clinical effectiveness as evaluated by both primary efficacy endpoints (ITT population), namely RSBQ (p = 0.035) and CGI-I (p = 0.029) after treatment with ANAVEX 2-73.
In addition, in the clinical study, prespecified patients with WT SIGMAR1 showed a clinically relevant and statistically significant 14.5-point (p = 0.009) increase in the RSBQ total score, the trial’s main effectiveness objective, compared to placebo. The degree of improvement with ANAVEX 2-73 is comparable to published results from clinical trials presently underway. Despite an advantage of greater dosage and lower age compared to the ANAVEX 2-73-RS-001 study, it reported an average difference of 4.4 points in RSBQ total score over placebo.
Walter E. Kaufmann, MD, Principal Investigator and Chief Medical Officer of Anavex stated,
The biomarker-driven clinical data is highly encouraging since it opens the door to effective therapy for adults as well as children with Rett syndrome, also early treatments to change the disease’s trajectory. In terms of both safety and clinical improvement, the results of this experiment are highly promising. ANAVEX 2-73 revealed clinically substantial improvements in outcome measures measuring numerous impairments, which are confirmed by correlations with objective biomarkers, despite the cohort’s older age (patients were on average 24 years old) and the comparatively modest dosage (5 mg daily).