Here's the link to today's press release, for the record, so we can find it again easily.
http://newsfile.refinitiv.com/getnewsfile/v1/...theme=true
NEW YORK, June 21, 2021 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) disorders, today reported predictive biomarker of response established with SIGMAR1 mRNA expression correlates significantly with responses in primary clinical efficacy endpoints from the U.S. Phase 2 randomized, double-blind, placebo-controlled trial of ANAVEX®2-73 (blarcamesine) in adult female patients with Rett syndrome.
ANAVEX®2-73 activates the sigma-1 receptor (SIGMAR1). Data suggests that activation of SIGMAR1 results in the restoration of complete housekeeping function within the body and is pivotal to restoring neural cell homeostasis and promoting neuroplasticity.1 Recent independent findings strengthen the understanding of the beneficial effect of SIGMAR1 activation as compensatory mechanism to chronic CNS diseases.2
Rett syndrome is a chronic CNS disease caused by a spontaneous mutation of one gene, MECP2. This study demonstrates for the first-time that a biomarker correlates with clinical efficacy in Rett syndrome. ANAVEX®2-73 treatment resulted in increases in the mRNA expression of SIGMAR1, the gene coding for the receptor targeted by ANAVEX®2-73, which correlated with clinical efficacy as measured by both primary efficacy endpoints (ITT population), namely RSBQ (p = 0.035) and CGI-I (p = 0.029).
In addition, prespecified patients with WT SIGMAR1 in the clinical trial demonstrated a clinically meaningful and statistically significant 14.5-point (p = 0.009) improvement over placebo in the RSBQ total score, the trial’s key efficacy endpoint. This magnitude of the improvement with ANAVEX®2-73 compares favorably to published data currently in clinical development, which reported an average difference of 4.4 points in RSBQ total score versus placebo, despite an advantage of higher dose and lower age compared to ANAVEX®2-73-RS-001 trial.3
The RSBQ demonstrated balanced improvements across all the instrument’s subscales during the trial period of 7 weeks, including general mood, breathing, hand behavior, repetitive face movements, body rocking, night-time behavior, fear/anxiety, walking/standing.
The Anxiety, Depression, and Mood Scale (ADAMS), which is a measure of anxiety and mood symptoms in individuals with intellectual disability,4 has been clinically validated for use in Rett syndrome5 and in Fragile X syndrome,6 demonstrated clinically meaningful and statistically significant 12.9-point (p = 0.005) improvement for ANAVEX®2-73 treated adult patients with Rett syndrome vs placebo in prespecified patients with WT SIGMAR1.
The ADAMS also demonstrated balanced improvements across all different subscales during the trial period of 7 weeks, including manic/hyperactive behavior, depressed mood, social avoidance, general anxiety, obsessive compulsive behavior.
“The biomarker-driven clinical evidence is very exciting and opens the possibility of successful treatment for both adults and children with Rett syndrome and early interventions for modifying the course of the disease,” commented Walter E. Kaufmann, MD, Principal Investigator and Chief Medical Officer of Anavex. “The outcome of this trial is very promising in terms of both safety and clinical improvement. Despite the challenges of the older age of the cohort (patients were on average 24 years of age) and the relatively low dose (5 mg daily), ANAVEX®2-73 demonstrated clinically meaningful improvements in outcome measures evaluating multiple impairments, which are supported by correlations with objective biomarkers.”
http://newsfile.refinitiv.com/getnewsfile/v1/...theme=true
NEW YORK, June 21, 2021 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) disorders, today reported predictive biomarker of response established with SIGMAR1 mRNA expression correlates significantly with responses in primary clinical efficacy endpoints from the U.S. Phase 2 randomized, double-blind, placebo-controlled trial of ANAVEX®2-73 (blarcamesine) in adult female patients with Rett syndrome.
ANAVEX®2-73 activates the sigma-1 receptor (SIGMAR1). Data suggests that activation of SIGMAR1 results in the restoration of complete housekeeping function within the body and is pivotal to restoring neural cell homeostasis and promoting neuroplasticity.1 Recent independent findings strengthen the understanding of the beneficial effect of SIGMAR1 activation as compensatory mechanism to chronic CNS diseases.2
Rett syndrome is a chronic CNS disease caused by a spontaneous mutation of one gene, MECP2. This study demonstrates for the first-time that a biomarker correlates with clinical efficacy in Rett syndrome. ANAVEX®2-73 treatment resulted in increases in the mRNA expression of SIGMAR1, the gene coding for the receptor targeted by ANAVEX®2-73, which correlated with clinical efficacy as measured by both primary efficacy endpoints (ITT population), namely RSBQ (p = 0.035) and CGI-I (p = 0.029).
In addition, prespecified patients with WT SIGMAR1 in the clinical trial demonstrated a clinically meaningful and statistically significant 14.5-point (p = 0.009) improvement over placebo in the RSBQ total score, the trial’s key efficacy endpoint. This magnitude of the improvement with ANAVEX®2-73 compares favorably to published data currently in clinical development, which reported an average difference of 4.4 points in RSBQ total score versus placebo, despite an advantage of higher dose and lower age compared to ANAVEX®2-73-RS-001 trial.3
The RSBQ demonstrated balanced improvements across all the instrument’s subscales during the trial period of 7 weeks, including general mood, breathing, hand behavior, repetitive face movements, body rocking, night-time behavior, fear/anxiety, walking/standing.
The Anxiety, Depression, and Mood Scale (ADAMS), which is a measure of anxiety and mood symptoms in individuals with intellectual disability,4 has been clinically validated for use in Rett syndrome5 and in Fragile X syndrome,6 demonstrated clinically meaningful and statistically significant 12.9-point (p = 0.005) improvement for ANAVEX®2-73 treated adult patients with Rett syndrome vs placebo in prespecified patients with WT SIGMAR1.
The ADAMS also demonstrated balanced improvements across all different subscales during the trial period of 7 weeks, including manic/hyperactive behavior, depressed mood, social avoidance, general anxiety, obsessive compulsive behavior.
“The biomarker-driven clinical evidence is very exciting and opens the possibility of successful treatment for both adults and children with Rett syndrome and early interventions for modifying the course of the disease,” commented Walter E. Kaufmann, MD, Principal Investigator and Chief Medical Officer of Anavex. “The outcome of this trial is very promising in terms of both safety and clinical improvement. Despite the challenges of the older age of the cohort (patients were on average 24 years of age) and the relatively low dose (5 mg daily), ANAVEX®2-73 demonstrated clinically meaningful improvements in outcome measures evaluating multiple impairments, which are supported by correlations with objective biomarkers.”
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