Clinical-stage biopharmaceutical company Anavex Life Sciences Corp. (AVXL:NASDAQ), which is focused on the development of therapeutics for treating neurodegenerative and neurodevelopmental and other central nervous system (CNS) disorders including Rett syndrome, Alzheimer's and Parkinson's disease, today announced "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."
The company explained that ANAVEX®2-73 works by activating the sigma-1 receptor (SIGMAR1), a key factor in restoring neural cell homeostasis and promoting neuroplasticity, which from recent independent findings is thought to provide beneficial effects as a compensatory mechanism to chronic CNS diseases.
Anavex Life Sciences indicated that "Rett syndrome is a chronic CNS disease caused by a spontaneous mutation of one gene, MECP2, and that this study demonstrates for the first-time that a biomarker correlates with clinical efficacy in Rett syndrome." The company added that "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."
Anavex Life Sciences' Principal Investigator and Chief Medical Officer Walter E. Kaufmann, M.D., remarked, "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…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."
The firm advised that based upon the positive results demonstrating convincing biomarker correlating efficacy data in its Phase 2 adult Rett syndrome study of ANAVEX®2-73 (RS-001), it now plans to initiate meetings with the U.S. Food and Drug Administration (FDA) to discuss the pathway forward for approval. The company stated that ANAVEX®2-73 has previously received Fast Track, Rare Pediatric Disease and Orphan Drug designations from the FDA for Rett syndrome treatment and it further believes that it may be considered for accelerated approval as thus far there are no FDA-approved medicines that have been approved for treating Rett syndrome.
The company advised that ANAVEX®2-73 is presently being studied in two additional Rett syndrome clinical trials. These include the AVATAR trial in adults with Rett syndrome and the EXCELLENCE study targeting pediatric Rett syndrome.
The company's President and CEO Christopher U. Missling Ph.D., commented, "These are strong and consistent data demonstrating biomarker-correlated rapid and clinically meaningful improvements in key measures of Rett syndrome symptoms in the ANAVEX®2-73 treatment group compared to placebo…we believe our ANAVEX®2-73 Rett syndrome program sets us on a course to potentially offer a new, unique and mechanistically differentiated treatment option also for other diseases associated with autism spectrum disorder."
The firm described Rett syndrome as "a devastating, non-inherited genetic post-natal progressive neurodevelopmental disorder that occurs almost exclusively in girls and leads to severe impairments, affecting nearly every aspect of the child's life: their ability to speak, walk, eat and easily breathe. The hallmark of Rett syndrome is near constant repetitive hand movements while awake." The company stated that there are about 11,000 patients diagnosed with Rett syndrome in the U.S.
The company noted that the double-blind Phase 2 oral liquid ANAVEX®2-73-RS-001 Clinical Trial evaluated 31 adult patients with Rett syndrome over a period of 7 weeks. The primary objectives in the study were to measure the safety, tolerability and pharmacokinetics of oral liquid once-daily dosing of 5 mg ANAVEX®2-73.
Anavex Life Sciences is a biopharmaceutical company based in New York City that is engaged in finding and developing therapeutics to address neurodegenerative and neurodevelopmental disorders such as Alzheimer's and Parkinson's diseases and Rett syndrome. The firm is also working on developing treatments for pain, other CNS diseases and certain cancers. The company statted that "its lead drug candidate, ANAVEX®2-73 (blarcamesine), successfully completed a Phase 2a clinical trial for Alzheimer's disease and recently a Phase 2 proof-of-concept study in Parkinson's disease dementia and a Phase 2 study in adult patients with Rett syndrome."
Anavex started the day with a market cap of around $1.6 billion with approximately 70.0 million shares outstanding and a short interest of about 9.3%. AVXL shares opened almost 9% higher today at $24.06 (+$1.92, +8.67%) over Friday's $22.14 closing price. The stock has traded today between $24.06 and $27.85 per share and closed at $25.17 (+$3.03, +13.69%).[NLINSERT]
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