ANAVEX 2-73 has U.S. FDA Orphan Drug Designation for Rett Syndrome
NEW YORK and Cincinnati, Ohio – January 19, 2017 – Anavex Life Sciences Corp. (Nasdaq: AVXL) and the International Rett Syndrome Foundation, doing business as Rettsyndrome.org today announced that Rettsyndrome.org has committed a financial grant of a minimum of $0.6 million to cover the majority of a planned U.S. multicenter Phase 2 clinical trial of ANAVEX 2-73 for the treatment of Rett syndrome.
Scheduled to begin in 2017, the trial will be a randomized, double blind, placebo-controlled study of ANAVEX 2-73 in patients with Rett syndrome lasting up to 12 weeks. Primary and secondary endpoints include safety as well as Rett syndrome conditions such as cognitive impairment, motor impairment, behavioral symptoms and seizure activity. Existing independent pre-clinical research demonstrated that ANAVEX 2-73 improves all of these measures. Hence, ANAVEX 2-73 might have a chance to positively affect a wide range of the symptoms associated with Rett syndrome.
Steven Kaminsky, PhD, Chief Science Officer of Rettsyndrome.org, stated, “This is a very exciting time for us. Pre-clinical data from ANAVEX 2-73 is highly encouraging and demonstrates the potential of this investigational drug to significantly improve Rett patients’ lives. Success in the Phase 2 clinical trial could result in a truly safe and effective drug for this patient population, for which no effective treatment currently exists. We are very motivated to support this trial financially and by involving our knowledgeable clinical and educated community network.”
Rettsyndrome.org sponsored Anavex’s pre-clinical studies of Rett syndrome, which successfully demonstrated benefits from treatment with ANAVEX 2-73. As reported at the 2016 Epilepsy Pipeline Conference and the 2016 Rett Syndrome Research Symposium, ANAVEX 2-73 demonstrated dose-related and significant improvements in an array of behavioral and gait paradigms in a mouse model with a MECP2-null mutation that causes neurological symptoms that mimic Rett syndrome.
“We are delighted to continue our partnership with Rettsyndrome.org and its network of experts to accelerate the clinical development of ANAVEX 2-73 for the treatment of Rett syndrome,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “We look forward to initiating the planned Phase 2 clinical trial as soon as reasonably possible in 2017.”
Anavex received Orphan Drug Designation from the U.S. Food and Drug Administration’s (FDA) for ANAVEX 2-73 in Rett syndrome on May 18, 2016. The designation provides sponsors with development and commercial incentives, including seven years of market exclusivity in the U.S., prioritized consultation by the FDA on clinical studies and certain exemptions from or reductions in regulatory fees.
About Rett Syndrome
Rett syndrome is a rare non-inherited genetic postnatal 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 even breathe easily. The hallmark of Rett syndrome is near constant repetitive hand movements while awake. It is characterized by normal early growth and development (6 to 18 months) followed by a slowing of development, loss of purposeful use of the hands, distinctive hand movements, slowed brain and head growth, problems with walking, seizures and intellectual disability. There is currently no cure for Rett syndrome and treatment of the disorder is symptomatic. Management of symptoms is done through a multidisciplinary approach utilizing medication for motor difficulties, breathing irregularities and control of seizures through anticonvulsant drugs. Rett syndrome is caused by mutations in the MECP2 gene and strikes all racial and ethnic groups and occurs worldwide in approximately 1 in every 10,000 to 15,000 live female births.
Rettsyndrome.org is the most comprehensive nonprofit organization dedicated to accelerating research of treatments and a cure for Rett syndrome and related disorders while providing information and family empowerment. As the world’s leading private funder of Rett syndrome research, Rettsyndrome.org has funded over $40M in high-quality, peer-reviewed research grants and programs to date. The organization hosts the largest global gathering of Rett researchers and clinicians to establish research direction for the future. Rettsyndrome.org, a 501(c)3 organization, has earned Charity Navigator’s most prestigious 4 star rating year after year. To learn more about our work and Rett syndrome, visit www.rettsyndrome.org or call (800) 818-7388 (RETT).
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, other central nervous system (CNS) diseases, pain and various types of cancer. Anavex’s lead drug candidate, ANAVEX 2-73, recently completed successfully a 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 and successfully completed Phase 1. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer’s disease. It has also exhibited anticonvulsant, anti-amnesic, neuroprotective and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy and others. The Michael J. Fox Foundation for Parkinson’s Research has awarded Anavex a research grant to develop ANAVEX 2-73 for the treatment of Parkinson’s disease to fully fund a preclinical study, which could justify moving ANAVEX 2-73 into a Parkinson’s disease clinical trial. ANAVEX 3-71, also targeting sigma-1 and M1 muscarinic receptors, is a promising preclinical drug candidate demonstrating disease modifications against the major Alzheimer’s hallmarks in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid and tau pathologies, and also with beneficial effects on neuroinflammation and mitochondrial dysfunctions. Further information is available at www.anavex.com and 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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Dennis Dobson, Jr.
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Steven Kaminsky, PhD, Chief Science Officer