AVXL share price rises more than 10% before market: why it happened
- The Anavex Life Sciences Corp (NASDAQ: AVXL) share price rose more than 10% before market. That’s why it happened.
The share price of Anavex Life Sciences Corp (NASDAQ: AVXL) – a clinical-stage biopharmaceutical company that develops differentiated therapies for the treatment of neurodegenerative and neurodevelopmental disorders, including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) disorders – increased by more than 10% pre-marketing. Investors appear to respond positively to the fact that the response predictive biomarker established with SIGMAR1 mRNA expression significantly correlates with responses to key clinical efficacy endpoints in the US phase trial 2 randomized, double-blind, placebo-controlled ANAVEX 2-73 (blarcamesine) in adult patients with Rett syndrome.
ANAVEX 2-73 is known to activate the sigma-1 receptor (SIGMAR1). And data suggests that activating SIGMAR1 results in the restoration of full housekeeping function in the body and is essential for restoring neural cell homeostasis and promoting neuroplasticity. In addition, recent independent findings strengthen the understanding of the beneficial effect of activating SIGMAR1 as a compensatory mechanism for chronic CNS diseases.
Rett syndrome is a chronic disease of the CNS caused by a spontaneous mutation of a gene called MECP2. And this study demonstrates for the first time that a biomarker correlates with clinical efficacy in Rett syndrome. ANAVEX 2-73 treatment resulted in an increase in mRNA expression of SIGMAR1, the gene encoding the receptor targeted by ANAVEX 2-73 – which was correlated with clinical efficacy as measured by the two endpoints. ‘main assessment (ITT population), namely RSBQ (p = 0.035) and CGI-I (p = 0.029).
Patients pre-specified with WT SIGMAR1 in the clinical trial demonstrated a clinically significant and statistically significant 14.5 point (p = 0.009) improvement over placebo in the RSBQ total score, which is the endpoint of key effectiveness of the trial. And this magnitude of improvement with ANAVEX 2-73 compares favorably with published data currently in clinical development, which reported an average difference of 4.4 points in RSBQ total score compared to placebo despite the benefit of a higher dose and younger age compared to ANAVEX 2-73 -RS-001 trial.3
The RSBQ demonstrated balanced improvements across all instrument subscales over the 7 week trial period, such as general mood, breathing, hand behavior, repetitive facial movements, body sway, nocturnal behavior, fear / anxiety, walking / standing.
The Anxiety, Depression, and Mood Scale (ADAMS) – which is a measure of anxiety and mood symptoms in people with developmental disabilities – has been clinically validated for use in Rett syndrome and fragile X syndrome6, shown to be clinically significant and statistically significant 12.9 -point of improvement (p = 0.005) for adult patients treated with ANAVEX 2-73 with Rett syndrome compared to placebo in pre- specified with WT SIGMAR1.
ADAMS also demonstrated balanced improvements in all different subscales over the 7 week trial period including manic / hyperactive behavior, depressed mood, social avoidance, general anxiety , obsessive-compulsive behavior.
With this more compelling biomarker correlating efficacy data from the US phase 2 study (ANAVEX 2-73-RS-001) in adult patients with Rett syndrome, Anavex plans to meet with the FDA to discuss the way approval. Currently, there is no FDA approved drug for Rett syndrome.
ANAVEX 2-73 has Fast Track Designation, Rare Pediatric Disease Designation, and Orphan Drug Designation from the FDA for the treatment of Rett Syndrome and may be considered for expedited approval. This study was supported by the Rettsyndrome.org Foundation.
ANAVEX 2-73 is currently being evaluated for Rett syndrome in 2 other late stage placebo-controlled clinical studies: And the AVATAR trial in adult Rett syndrome (ANAVEX 2-73-RS-002) 8 and the trial EXCELLENCE in Pediatric Rett Syndrome (ANAVEX 2-73-RS-003).
The Anavex Life Sciences product portfolio platform includes the small molecule drug candidate ANAVEX 2-73 for the treatment of Alzheimer’s disease, Parkinson’s disease and Rett syndrome, and ANAVEX 3- 71 for frontotemporal dementia.
“The clinical evidence based on biomarkers is very interesting and opens the possibility of successful treatment for adults and children with Rett syndrome and early interventions to modify the course of the disease. The result of this trial is very promising in terms of safety and clinical improvement. Despite the challenges of the older cohort age (patients were on average 24 years old) and the relatively low dose (5 mg per day), ANAVEX 2-73 demonstrated clinically significant improvements in outcome measures assessing multiple impairments, which are supported by correlations with objective biomarkers.
– Walter E. Kaufmann, MD, Principal Investigator and Medical Director of Anavex
“This is strong, consistent data demonstrating rapid and clinically significant improvements correlated with biomarkers in key measures of Rett syndrome symptoms in the ANAVEX 2-73 treatment group compared to placebo. Our team is dedicated to addressing this urgent unmet need of Rett Syndrome patients, and we believe our Rett Syndrome program ANAVEX 2-73 sets us on the right track to potentially offer a unique and new treatment option. mechanically differentiated also for other diseases associated with autism spectrum disorder.
– Christopher U. Missling, PhD, President and CEO of Anavex
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