NEWARK, N.J., March 17, 2015 /PRNewswire/ -- Neurotrope, Inc. (OTCQB: NTRP) today announced secondary and exploratory endpoint results from its randomized, double-blind, placebo-controlled, single dose Phase 2a clinical trial evaluating bryostatin-1 for the treatment of Alzheimer's disease (AD). Bryostatin is a potent modulator of an enzyme called protein kinase C epsilon (PKCe). The Company is approaching the treatment of Alzheimer's disease through the activation of PKCe. In animal models of Alzheimer's disease, activation of PKCe has been shown to improve learning and memory, induce synaptogenesis or growth of new synapses and prevent neurodegeneration.
Final analysis of this Phase 2a safety study, in nine Alzheimer's patients with mild dementia as measured by MMSE-2 scores, confirms the previously announced result. The study has met its primary endpoint demonstrating preliminary safety and tolerability of bryostatin. No safety signals have been identified.
As a secondary objective, the Phase 2a safety study examined the correlation of the changes in PKCe with plasma levels of bryostatin after a single dose. Preliminary assessment of PKCe levels in peripheral monocytes demonstrated a significant increase in total PKC protein levels at the end of the bryostatin infusion consistent with target engagement.
Commenting on the study results, Charles S. Ramat, President and Chief Executive Officer of Neurotrope, Inc., said, "We are pleased to confirm the preliminary findings of the Phase 2astudy we disclosed last month, the Phase 2a met its primary endpoint, showing good safety and tolerability. Now we can add that we achieved expected outcomes on the exploratory endpoint of PKCe activation. While we continue to recognize that this is a small trial population we are still greatly encouraged and intend to move this treatment forward to our next planned clinical trial."
An additional secondary objective of the study was the evaluation of efficacy following a single dose of bryostatin. As expected with a single dose of bryostatin, there was no measurable improvement in cognition in this mildly impaired patient population. It is important to note that in previous animal studies improvement of learning and memory was first observed following multiple doses of bryostatin.
Warren W. Wasiewski, MD, Executive Vice President and Chief Medical Officer of Neurotrope, noted, "Given these additional encouraging results, we are actively planning our Phase 2b, multi-site, double-blind, placebo controlled trial of approximately 150 patients in moderately severe to severe AD patients."
About Neurotrope's Approach to Alzheimer's
In contrast to the industry's historic focuses on removal of amyloid plaque and Tau pathology in the brain – which have resulted in numerous, failed clinical trials – Neurotrope is following a new and novel approach. Specifically, research conducted in animal models by the Company's Chief Scientific Officer, Dr. Dan Alkon, of the Blanchette Rockefeller Neurosciences Institute, has shown that PKCe, when stimulated, initiates a cascade of enzymatic events, ultimately improves synaptic function, induces formation of new synapses and inhibits cell death. The stimulation of PKCe is far upstream from the formation of plaques and tangles, which may be considered pathologic markers of AD, rather than causes. As a potent modulator of PKCe at low dose levels, bryostatin has been shown to stimulate this key enzyme, thus showing promise in restoring memory and learning function.
Neurotrope Bioscience Inc., the operating subsidiary of Neurotrope, Inc., was formed in October 2012 principally to license, develop and commercialize various novel therapeutic and diagnostic technologies from the Blanchette Rockefeller Neuroscience Institute (BRNI) which are focused on the development of conventional small molecules that are extraordinarily potent in the activation of the enzyme PKCe. PKCe has been shown to play a central role in the regrowth or repair of nervous tissues, cells or cell products. Neurotrope's pipeline, under its license from BRNI, includes the drug candidate, bryostatin, for the treatment of Alzheimer's disease, and a minimally invasive, diagnostic biomarker analysis system which would assess the presence of Alzheimer's in patients. In addition, Neurotrope has a world-wide, exclusive license agreement with the Icahn School of Medicine at Mount Sinai located in New York City to utilize its proprietary information and data package for the use of bryostatin-1 in the treatment of Niemann-Pick Type C Disease, a rare disease, mostly of children who are afflicted with Alzheimer-like symptoms. Also, the Company, under its BRNI license, has the rights to develop the licensed technology for other cognitive dysfunctions, including orphan diseases, such as Fragile X Syndrome.
About The Blanchette Rockefeller Neurosciences Institute
Located in Morgantown, WV, BRNI, at West Virginia University, is a unique, independent, non-profit institute dedicated to the study of memory and finding solutions to memory disorders. BRNI was founded in 1999 in memory of Blanchette Ferry Hooker Rockefeller, an Alzheimer's patient and mother of U. S. Senator John D. Rockefeller IV. BRNI is operated in alliance with West Virginia University as well as in collaboration with other academic institutions.
Any statements contained in this press release that do not describe historical facts may constitute forward-looking statements. These forward-looking statements include statements regarding the plans and scope for the Company's next clinical trial with bryostatin in the treatment of Alzheimer's disease and potential for the diagnostic system to assess Alzheimer's disease in patients. Such forward- looking statements are subject to a number of risks and uncertainties and other influences, many of which the Company has no control over. Actual results and the timing of certain events and circumstances may differ materially from those described by the forward-looking statements as a result of these risks and uncertainties. Factors that may influence or cause actual results to differ materially from expected or desired results may include, without limitation, the Company's inability to obtain adequate financing, the significant length of time associated with drug development and related insufficient cash flows and resulting illiquidity, the Company's patent portfolio, the Company's inability to expand the Company's business, significant government regulation of pharmaceuticals and the healthcare industry, lack of product diversification, availability of the Company's raw materials, existing or increased competition, stock volatility and illiquidity, and the Company's failure to implement the Company's business plans or strategies. These and other factors are identified and described in more detail in the Company's filings with the SEC, including the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2013 and Quarterly Report on Form 10-Q for the fiscal nine months ended September 30, 2014. The Company does not undertake to update these forward-looking statements.
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