Quote: "Imagine if the Rett results are not so good as everyone is expecting, this will drop to under $5."
Sure. Anything is possible. If it was a slam dunk then everyone would be invested in AVXL...and our SP would be North of $1,000/share.
But let's get real. I'm invested in AVXL because of past results from the P2 AD, P2 Rett trials (see below), and especially the P2 PDD trial with over 130 participants...all of which enrolled in the OLE.
So, if results don't come in as great as I expect they will, then I believe it would contradict all of the previous trials that I mentioned above.
Personally, I think the odds are definitely in my favor.
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 top-line results from a U.S. Phase 2 randomized, double-blind, placebo-controlled trial of ANAVEX®2-73 (blarcamesine) in adult female patients with Rett syndrome.
The primary endpoint of the trial was safety. The convenient oral liquid once-daily dosing of 5 mg ANAVEX®2-73 was well-tolerated and demonstrated dose-proportional PK (pharmacokinetics). Adverse events related to study drug were similar between ANAVEX®2-73 (13.3%) and placebo (10%), with no reported serious adverse events (SAEs). The safety profile of ANAVEX®2-73 in this trial is consistent with prior clinical trial data.
All secondary efficacy endpoints of the trial showed statistically significant and clinically meaningful sustained improvements for ANAVEX®2-73 compared to placebo, consisting of the Rett Syndrome Behaviour Questionnaire (RSBQ) (p = 0.048) and the Clinical Global Impression Improvement Scale (CGI-I) score (p = 0.014) in the intent-to-treat (ITT) population (n = 25). Statistically significant differences in patient symptoms between the active and placebo groups occurred as early as 4 weeks following the initiation of ANAVEX®2-73 administration. Improvements in RSBQ Total scores were correlated with parallel decreases (improvements) in glutamate plasma levels.
ANAVEX®2-73 activates the sigma-1 receptor (SIGMAR1). Data suggests that activation of the sigma-1 receptor (SIGMAR1) is pivotal to restoring neural cell homeostasis and promoting neuroplasticity.[1] Consistent with previous ANAVEX®2-73 clinical trials, patients carrying the common form of the SIGMAR1 gene treated with ANAVEX®2-73 experienced stronger improvements in the prespecified efficacy endpoints.
All twenty-five patients in this randomized study elected to enter a 12-week ANAVEX®2-73 extension study. Anavex will be advancing its Expanded Access Policy in order to provide long-term therapy to current participants with Rett syndrome under an expanded access program for ANAVEX®2-73.
“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 over 18 years of age) and the relatively low dose (5 mg daily), ANAVEX®2-73 demonstrated clinically meaningful improvements in outcome measures evaluating multiple impairments,” commented Walter E Kaufmann, MD, Principal Investigator. Subsequent to his appointment as Principal Investigator of this Phase 2 ANAVEX®2-73 trial in adult Rett syndrome patients, Dr. Kaufmann joined Anavex as Chief Medical Officer. He also said, “Moreover, the convergent clinical evidence was supported by parallel changes in a key biomarker of disease. 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.”
Based on the results in this first of its kind U.S. Phase 2 (ANAVEX®2-73-RS-001)[2] study in adult patients with Rett syndrome, Anavex is planning to meet with FDA to discuss the approval pathway. There are no FDA-approved drugs 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 accelerated approval.
ANAVEX®2-73 is currently being evaluated for Rett syndrome in two other ongoing placebo-controlled clinical studies: The Phase 2 AVATAR trial in adult Rett syndrome (ANAVEX®2-73-RS-002)[3] and the EXCELLENCE Phase 2/3 pediatric Rett syndrome study (ANAVEX®2-73-RS-003)[4].
“These are strong and consistent data demonstrating tolerability and rapid and clinically meaningful improvements in key measures of Rett syndrome symptoms in the ANAVEX®2-73 treatment group compared to placebo,” said Christopher U Missling, PhD, President & Chief Executive Officer of Anavex. I would like to thank the patients and caregivers who participated in this trial, the Anavex team, trial clinics, and doctors who have worked tirelessly on this program. Special thanks go to the Rettsyndrome.org Foundation, which provided financial support for this trial; we look forward to continuing the journey together.”
Anavex Life Sciences Announces Preliminary Clinical Efficacy Data of its U.S. Phase 2 Clinical Trial of ANAVEX®2-73 in Patients with Rett Syndrome
• Both global efficacy endpoints, RSBQ and CGI-I, showed significant improvement with respect to baseline after 7 weeks of treatment with ANAVEX®2-73 (blarcamesine) • ANAVEX®2-73 (blarcamesine) treatment effect was significantly correlated with changes in two different biomarkers linked to the neurobiology of Rett syndrome, Glutamate and GABA • Detailed Data to be Presented at 6th Annual European Rett Syndrome Conference in Tampere, Finland, September 27-28, 2019
NEW YORK – September 16, 2019 – 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 announced preliminary clinical data of the U.S. Phase 2 Rett syndrome clinical trial.
Preliminary Clinical Data is derived from the ANAVEX®2-73-RS-001 study on the first 6-patient cohort ranging in age from 18 to 36 years, who completed the pharmacokinetic (PK) part of the study and who received a low dose of approx. 5 mg daily oral liquid dose of ANAVEX®2-73 (blarcamesine) for 7 weeks. Patients are continuing participation in the ANAVEX®2-73-RS-001 open label extension study. Both efficacy endpoints, the Rett Syndrome Behaviour Questionnaire (RSBQ) and the Clinical Global Impression – Improvement (CGI-I) showed significant improvement with respect to baseline after 7 weeks of treatment. The RSBQ Total average scores improved from 50 to 34 points (2-tailed Wilcoxon signed rank test, p = 0.027) and the CGI-I scores were positively correlated with RSBQ Total scores at 7 weeks (2-tailed Spearman’s rho = 0.956, p = 0.003).
Supporting the clinical assessments, plasma levels of the biomarker Glutamate also decreased significantly (Week 0 vs. Week 7; 2-tailed Wilcoxon signed rank test, p = 0.046) and levels of Glutamate at Week 7 were directly correlated with CGI-I scores at Week 7 (2-tailed Spearman’s rho = 0.837, p = 0.038) with greater decreases in Glutamate associated with greater improvement in these efficacy scores. Glutamate is the main excitatory neurotransmitter in the brain and is known to be higher in patients with Rett syndrome compared to healthy subjects in the brain, as measured by magnetic resonance imaging spectroscopy (MRS), as well as in cerebrospinal fluid (CSF) and blood plasma.
Additionally, the magnitude of GABA change was inversely correlated with the magnitude of decrease in RSBQ Total scores (2-tailed Spearman’s rho = -0.812, p = 0.050) and GABA changes demonstrated an inverse correlation of the magnitude of Glutamate changes (2-tailed Spearman’s rho = -0.829, p = 0.042). GABA is the main inhibitory neurotransmitter in the brain, known to be deficient in animal models of Rett syndrome. Excitatory-inhibitory imbalances postulated in many neurologic disorders, including Rett syndrome, have been linked to imbalances between Glutamate and GABA1,2.
1 Kaufmann et al. Expert Opin Orphan Drugs 4:1043-1055, 2016 2 Banerjee et al. Brain 142:239-248, 2019
An independent DSMB review determined that ANAVEX®2-73 (blarcamesine) was well tolerated, with no SAEs reported and with all patients completing the study. Therefore, the DSMB issued a positive recommendation for the continuation of the Phase 2 Rett syndrome study without any modifications.
“This is a remarkable first strong signal for patients with Rett syndrome especially given that the strong effects were seen in adult patients, and we look forward to discussing these results with the FDA and the European regulatory agency as we continue our Rett Syndrome Program including pediatric patients,” said Walter E Kaufmann, MD, Principal Investigator of the study and Chief Medical Officer of Anavex. “Importantly, we've now observed that the ANAVEX®2-73 (blarcamesine) effect is correlated with changes of Glutamate and GABA levels, objective measures and biomarkers in several neurodevelopmental disorders.”
Detailed results will be presented at the 6th European Rett Syndrome Conference in Tampere, Finland, September 27-28, 2019 and submitted for publication in a peer-reviewed journal.
Neurobehavioral effects of ANAVEX®2-73 (blarcamesine) previously observed in preclinical studies were also detected in patients with Rett syndrome, pointing to the ability of translation of preclinical to clinical data. ANAVEX®2-73 (blarcamesine) has received orphan drug designation from the FDA and EMA for the treatment of Rett syndrome.
Christopher U Missling, PhD, President and Chief Executive Officer of Anavex stated, “We are encouraged by the insights gleaned from these first clinical data for ANAVEX®2-73 (blarcamesine) in patients with Rett syndrome and we look forward to both confirm this clinical data and continue the Rett syndrome program with determination. In addition to Rett syndrome3, Anavex has ongoing clinical development programs for ANAVEX®2-73 (blarcamesine) for the treatment of Alzheimer’s disease4 and Parkinson’s disease dementia5.”
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