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Neurotrope Enters into Collaboration with the Nemours / Alfred I. duPont Hospital for Children to Initiate a Clinical Trial in Fragile X Syndrome

NEW YORK, Sept. 5, 2018 /PRNewswire/ -- Neurotrope Inc. (NTRP) a clinical-stage biopharmaceutical company developing novel therapies for neurodegenerative and neurodevelopmental disorders, including Alzheimer's disease (AD), is announcing a collaboration with The Nemours / Alfred I. duPont Hospital for Children ("Nemours") to initiate a clinical trial in children with Fragile X Syndrome ("Fragile X").  The planned protocol for the pilot open-label trial will dose Fragile X patients from 8 to less than 18 years of age, using a dosing regimen similar to that being tested in AD patients. In addition to the primary objective of safety and tolerability, measurements will be made of working memory, language and other functional aspects such as anxiety, repetitive behavior, executive functioning, and social behavior.   "We are delighted to be collaborating with clinical scientists at Nemours on this important study for Fragile X.  As one of the premier children's hospitals in the United States, this hospital has both the patient population and the clinical team required to execute this study," stated Dr. Charles Ryan, Chief Executive Officer of Neurotrope.

Fragile X is characterized by synaptic immaturity, cognitive impairment, and behavioral changes that currently have no effective therapeutics. The disorder is caused by transcriptional shutdown in neurons of the FMR1 gene product, the Fragile X mental retardation protein (FMRP).  Fragile X, a childhood neurological disorder that afflicts approximately 135,000 individuals in the U.S., is characterized by widespread loss of synaptic connections and occurs early in the neurological development process.

"Alzheimer's disease and Fragile X have a common devastating consequence in the brain: the loss of functional and anatomically normal synaptic networks – the loss of the brain's "wiring."  Bryostatin's target, PKC epsilon, was shown in extensive pre-clinical testing to restore these lost synaptic structures.  Neurotrope is collaborating with Nemours on an early clinical trial with dose frequency that is similar to the completed Phase 2 trial with AD patients that suggested promising improvement in cognitive functions," stated Dr. Daniel Alkon, President and Chief Scientific Officer of Neurotrope.

With a transgenic, pre-clinical model of Fragile X, Bryostatin-1 caused benefits similar to those produced in AD transgenic mice - restoration of synaptic structures. Bryostatin-1 is a relatively selective PKC epsilon activator which appears to induce synaptogenesis and synaptic maturation by activating synaptic growth factors present in the brain. Such cognitive benefits and synaptic restoration have not been demonstrated by any drugs tested in pre-clinical Fragile X models.  Bryostatin-1 has been granted Orphan Drug status for the treatment of Fragile X by the Food and Drug Administration ("FDA"). "The mechanism of Bryostatin-1 is novel compared to all prior drug treatments studied in Fragile X. It will be important to explore this mechanism in an early phase Fragile X human study. Early signs of positive target engagement and potential change on quantitative outcome measures will work to justify a larger-scale future study," stated Professor Craig Erickson, MD, a leading authority on Fragile X.

Pre-clinical studies have shown that chronic treatment with Bryostatin-1 rescues young Fragile X mice from the disorder phenotypes, including normalization of most Fragile X abnormalities in: 1) hippocampal brain-derived neurotrophic factor expression; 2) postsynaptic density-95 levels; 3) transformation of immature dendritic spines to mature synapses; 4) densities of the presynaptic and postsynaptic membranes, and; 5) spatial learning and memory. Our pre-clinical results demonstrate that synaptic and cognitive function of young Fragile X mice can potentially be normalized through pharmacological treatment without down-regulation of mGluR5 and that Bryostatin-1-like agents may represent a novel class of drugs to treat the effects of Fragile X at a young age and in adults. (J Pharmacol Exp Ther. 2016 May;357(2):300-10).

About Neurotrope

Neurotrope is at the forefront of developing a new approach to combating AD and other neurodegenerative diseases. The Company's world-class science offers the potential to realize a paradigm shift to overcome one of today's most challenging clinical problems — finding a way to slow or even prevent the progression of AD.

In addition to the Company's Phase 2 trial of Bryostatin-1 in advanced AD, Neurotrope has also conducted preclinical studies of Bryostatin-1 as a potential treatment for Stroke, Traumatic Brain Injury, and Fragile X Syndrome, Niemann-Pick Type C disease and Rett Syndrome—rare genetic diseases for which only symptomatic treatments are currently available. The FDA has granted Orphan Drug Designation to Neurotrope for Bryostatin-1 as a treatment for Fragile X. Bryostatin-1 has already undergone testing in more than 1,500 people in cancer studies, thus creating a large safety data base that will further inform clinical trial designs.

Please visit www.neurotrope.com for further information.

About Nemours

About Nemours Children's Health System

Nemours is an internationally recognized children's health system that owns and operates the Nemours / Alfred I. duPont Hospital for Children in Wilmington, Del., and Nemours Children's Hospital in Orlando, Fla., along with outpatient facilities in six states, delivering pediatric primary, specialty and urgent care. Nemours also powers the world's most-visited website for information on the health of children and teens, KidsHealth.org and offers on-demand, online video patient visits through Nemours CareConnect.

Established as The Nemours Foundation through the legacy and philanthropy of Alfred I. duPont, Nemours provides pediatric clinical care, research, education, advocacy, and prevention programs to families in the communities it serves. 

Please visit www.nemours.org for additional information.

Forward-Looking Statements

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 Phase 2 study and further studies, and continued development of use of Bryostatin-1 for Alzheimer's dementia and other cognitive diseases. Such forward-looking statements are subject to risks and uncertainties and other influences, many of which the Company has no control over. There can be no assurance that the clinical program for Bryostatin-1 will be successful in demonstrating safety and/or efficacy that we will not encounter problems or delays in clinical development, or that Bryostatin-1 will ever receive regulatory approval or be successfully commercialized. 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. Additional 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 year ended December 31, 2017, and on Form 10-Q for the quarter ended June 30, 2018. The Company does not undertake to update these forward-looking statements.

Contact information:
Investors and Media
Jeffrey Benison, Director of Corporate Communications
Neurotrope, Inc.
516.286.6099 (C) or 212.334.8709 (O)


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