Anavex Life Sciences Presents New Three-Year, Longitudinal Clinical Efficacy Data for ANAVEX®2-73 in Alzheimer’s Disease at the 2018 Clinical Trials on Alzheimer’s Disease (CTAD) Meeting
Late-Breaking Data Demonstrate Significant Association Between Higher ANAVEX®2-73 Concentration and Maintained Activities of Daily Living and Reduced Cognitive Decline in Alzheimer’s Disease Patients
Data Confirm SIGMAR1 and COMT as Genetic Biomarkers of Response to ANAVEX®2-73
NEW YORK – October 26, 2018 – 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 diseases including Alzheimer’s disease, Rett syndrome and other central nervous system (CNS) diseases, today presented new clinical data for ANAVEX®2-73 in a late-breaking oral presentation at the 2018 Clinical Trials on Alzheimer’s Disease (CTAD) Meeting.
At 148 weeks into the five-year extended Phase 2a clinical study of ANAVEX®2-73, data confirmed a significant association between ANAVEX®2-73 concentration and both exploratory functional and cognitive endpoints as measured by the Alzheimer’s Disease Cooperative Study-Activities of Daily Living (ADCS-ADL) evaluation and the Mini Mental State Examination (MMSE), respectively. The cohort of patients treated with higher ANAVEX®2-73 concentration maintained ADCS-ADL performance compared to the lower concentration cohort (p<0.0001). The patient cohort with the higher ANAVEX®2-73 concentration performed better at MMSE compared to the lower concentration cohort (p<0.0008).
A significant impact on the drug response levels of both the SIGMAR1 (p<0.0080) and COMT (p<0.0014) genomic biomarkers, identified and specified at week 57, was also confirmed over the 148-week period.
ANAVEX®2-73 demonstrated continued favorable safety and tolerability through 148 weeks.
“These results further confirm the impact of actionable genetic variants that were previously identified through a full, unbiased genomic analysis of ANAVEX®2-73 in Alzheimer’s disease, raising optimism for the future of biomarker-guided precision medicine to effectively combat this devastating disease,” said Professor Harald Hampel, M.D., Ph.D., M.A., M.Sc., AXA Research Fund & Sorbonne University Excellence Chair, Departments of Neuroscience and Neurology, Sorbonne University, Paris, France. Professor Hampel is the president of the Alzheimer Precision Medicine Initiative (APMI).
“We are excited to see our initial biomarker hypothesis confirmed at 148 weeks. The biomarkers were selected through a systematic, data-driven analysis of all available genomic and clinical data, which identified a genomic alteration of SIGMAR1, the target of ANAVEX®2-73. The consistency of the DNA and RNA data, as well as multiple endpoints and time-points further strengthen this biomarker hypothesis,” said Mohammad Afshar, M.D., Ph.D., CEO of Ariana Pharma. “This innovative data analytics approach, using our KEM® Artificial Intelligence platform has the potential to expand the access to precision medicine and precision pharmacology for a wide range of neurodegenerative diseases, thus, identifying the right patients that can benefit from the right therapy, at the right moment.”
“We are encouraged by these data which now show the clinical impact of ANAVEX®2-73 on patients who have been receiving therapy for almost three years,” said Christopher U Missling, Ph.D., President and Chief Executive Officer of Anavex. “We continue to build on the body of clinical evidence to support the development of ANAVEX®2-73 as we further our recently initiated Phase 2b/3 study, and we will continue our targeted precision medicine approach to advance ANAVEX®2-73 as a potential therapeutic option for patients with Alzheimer’s disease who will most benefit from treatment.”
Title: Longitudinal 148-Week Extension Study for ANAVEX®2-73 Phase 2a Alzheimer’s Disease Demonstrates Maintained Activities of Daily Living Score (ADCS-ADL) and Reduced Cognitive Decline (MMSE) for Patient Cohort on Higher Drug Concentration and Confirms Role of Patient Selection Biomarkers
The slides are accessible through the investor relations section of the Company’s website at www.anavex.com.
About ANAVEX®2-73-002 Phase 2a Clinical Study (ClinicalTrials.gov NCT02244541)
The multicenter Phase 2a clinical trial of ANAVEX®2-73 consisted of two parts and a total of 32 mild-to-moderate Alzheimer’s patients. PART A was a randomized, open-label, two-period, cross-over between oral (30mg/50mg) and IV (3mg/5mg) administration, adaptive trial lasting up to five weeks for each patient. PART B was an open-label extension for an additional 52 weeks. Initially planned for 26 weeks, PART B was extended to 52 weeks as a result of requests from patients and caregivers.
The primary endpoints of the Phase 2a trial were safety, tolerability and maximum tolerated dose (MTD) of ANAVEX®2-73, which had shown potential in preclinical studies to prevent, halt and/or reverse the course of the disease. Secondary endpoints included dose response, bioavailability, and exploratory cognitive and functional measures using the Mini Mental State Examination (MMSE) and evaluation of Alzheimer’s Disease Co-operative Study – Activities of Daily Living Inventory (ADCS-ADL), as well as Cogstate test battery and biomarker EEG/ERP.
About ANAVEX®2-73-003 Phase 2a Clinical Study (ClinicalTrials.gov NCT02756858)
The multi-center Phase 2a clinical trial of ANAVEX®2-73 consists of an open-label extension for an additional 208 weeks, allowing for the collection of potential safety data for ANAVEX®2-73 cumulatively over five years. The 208-week (four-year) extension of the multi-center Phase 2a clinical trial of ANAVEX®2-73 will follow mild-to-moderate Alzheimer’s patients who have already completed 52 weeks in PART B of the study. Every three months, patients will be scheduled for physician visits to assess primary and secondary endpoints.
The primary endpoints of the new Phase 2a trial are safety and tolerability of ANAVEX®2-73. Secondary endpoints are exploratory cognitive and functional measures using the Mini Mental State Examination (MMSE) and evaluation of Alzheimer’s Disease Co-operative Study – Activities of Daily Living Inventory (ADCS-ADL), respectively.
About the Alzheimer’s Disease Precision Medicine Initiative (APMI)
The Alzheimer’s Disease Precision Medicine Initiative (APMI), established in 2016 by founding president Professor Harald Hampel of Sorbonne University, Paris, France, is an international interdisciplinary consortium of leading neuroscientists aimed to facilitate breakthrough reforms towards early detection and successful drug development of neurological diseases, such as Alzheimer’s disease, away from a traditional “one-size fits all” approach to drug development of biologically heterogeneous populations towards individualized biomarker-guided targeted therapy for the right patient at the right time. Further information is available at www.apmiscience.com.
About Ariana Pharma
Ariana Pharma is a leading digital health Company focused on developing advanced therapeutic decision support systems. Ariana’s innovative clinical data analysis and diagnostic testing solutions help the healthcare sector better adapt patient treatments to individual biological characteristics. Ariana KEM® Artificial Intelligence technology enables personalization of therapies, improves the efficacy and safety of patient treatment, reduces risks and drug development costs, and accelerates time to market. With a growing number of successful therapeutic development applications, KEM® is the only FDA-reviewed technology that systematically explores combinations of biomarkers, producing more effective biomarker signatures for precision medicine. Ariana has developed Onco KEM®, the most advanced, clinically tested, oncology treatment selection system. Founded in 2003 as a spin-off of the Institut Pasteur, Paris, France, the company opened a subsidiary in the United States in 2012. Further information is available at www.arianapharma.com.
About Anavex Life Sciences Corp.
Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company dedicated to the development of differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental diseases including Alzheimer’s disease, Rett syndrome and other central nervous system (CNS) diseases, pain and various types of cancer. Anavex’s lead drug candidate, ANAVEX®2-73, recently completed a successful Phase 2a clinical trial for Alzheimer’s disease. ANAVEX®2-73 is an orally available drug candidate that restores cellular homeostasis by targeting sigma-1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer’s disease. ANAVEX®2-73 also exhibited anticonvulsant, anti-amnesic, neuroprotective and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson’s Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX®2-73 for the treatment of Parkinson’s disease. ANAVEX®3-71, which targets sigma-1 and M1 muscarinic receptors, is a promising preclinical drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimer’s disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid and tau pathologies. In preclinical trials, ANAVEX®3-71 has shown beneficial effects on neuroinflammation and mitochondrial dysfunction. Further information is available at www.anavex.com. You can also connect with the company on Twitter, Facebook and LinkedIn.
Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks set forth in the Company’s most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Anavex Life Sciences Corp. undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.
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 Harald Hampel1, Mohammad Afshar2, Frédéric Parmentier2, Coralie Williams2, Adrien Etcheto2, Federico Goodsaid3, Christopher U Missling4; (1) Department of Neurology, Sorbonne University, Paris, France; (2) Ariana Pharma, Paris, France; (3) Regulatory Pathfinders LLC, San Francisco, CA; (4) Anavex Life Sciences Corp., New York, NY