Synaptogenix Signs Memorandum Of Understanding with Nemours A.I. DuPont Hospital to Initiate Trial Using Bryostatin, Under Orphan Drug Status, to Treat Fragile X Syndrome

- Bryostatin-1 to benefit from previously granted Orphan Drug Status for the treatment of Fragile X syndrome ("Fragile X") by the Food and Drug Administration ("FDA")

- In a recent Nature publication, co-authored by the Fragile X Research Foundation and Synaptogenix scientists, Bryostatin was shown to successfully treated both Fragile X Cognitive and Autistic Spectrum Disorder Deficits in animal models

- Nemours Foundation, founded by A.I. Dupont with an estimated value at over $5.0 Billion, supports one of the nation's largest multistate pediatric health systems

News provided by

Synaptogenix, Inc.

Aug 05, 2021, 10:00 ET

NEW YORK, Aug. 5, 2021 /PRNewswire/ -- Synaptogenix, Inc. (Nasdaq: SNPX), an emerging biopharmaceutical company developing regenerative therapeutics for neurodegenerative disorders, today announced that it signed a memorandum of understanding ("MOU") with the Nemours A.I. DuPont Hospital for Children, an internationally recognized leader in the comprehensive care of childhood diseases, with the intention of initiating a trial to treat Fragile X with Bryostatin.

Alan Tuchman M.D., Chief Executive Officer, stated, "We are very pleased to take this important step forward with the Nemours A.I. du Pont Hospital for Children, a leading institution for the treatment of childhood diseases, to partner with as we explore a second indication for Bryostatin across a new population: children and young adults. This study builds on positive preclinical studies of Fragile X by Synaptogenix scientists, most recently in collaboration with FRAXA Research Foundation, as published in a Science Reports (a Nature publication) article that extends the potential efficacy of Bryostatin from Fragile X intellectual disability to behavioral phenotypes of Autism Spectrum Disorder."

Dr. Michael Tranfaglia, Medical Director and Chief Scientific Officer of FRAXA Research Foundation stated, "The therapeutic strategy employed by Synaptogenix is exciting and entirely novel. The mechanism of action of Bryostatin in the treatment of Fragile X is unique, and preclinical results have been very promising. We believe that this compound has the potential to make a real difference in the lives of patients with Fragile X, and we look forward to supporting this important new clinical trial."

Dr. Mary M. Lee, Enterprise Chief Scientific Officer and Executive Vice-President for Nemours Children's Health System, Physician-in-Chief Delaware Valley, added, "We are excited to participate in a potentially breakthrough therapeutic drug strategy to address this huge unmet medical need."

Synaptogenix and the Nemours A.I. Dupont Hospital will collaborate on trial(s) of Bryostatin to treat children with Fragile X which is the most common genetic cause of intellectual disability that often includes Autistic Spectrum disorder behavioral abnormalities. Bryostatin, a lead compound of Synaptogenix, has previously been granted Orphan Drug Status by the FDA based on extensive preclinical publications authored by Synaptogenix scientists and further validated in collaboration with FRAXA Research Foundation. These Bryostatin studies in animal models demonstrated the only known normalization of genetically determined synaptic abnormalities at the ultrastructural level. Bryostatin restored synaptic structures and rectified cognitive deficits at both juvenile and young adult stages of Fragile X animal models programmed with the human gene for the disease.

Nemours' team of clinical experts will guide the direct interactions with the children, oversee administration of drug protocols, conduct a broad range of psychometric evaluations, and implement a novel Bryostatin efficacy biomarker. Synaptogenix staff will help design the overall protocol, provide expertise on drug development, and advise on dose optimization. Synaptogenix will also provide the study drug, ensure safe storage and distribution, as well as share knowledge of dosing in relation to the drug's regenerative mechanism of action, demonstrated in extensive preclinical studies.

"Bryostatin has shown promising preclinical efficacy in animal models to reverse synaptic loss and restore cognitive functions in a broad range of pre-clinical models of neurodegeneration, including Alzheimer's disease and those associated with both Fragile X and Autistic Spectrum Disorder. Based on those studies, the FDA has approved Bryostatin as an Orphan drug to treat Fragile X. We are encouraged by our collaboration with such an outstanding children's hospital as Nemours to initiate clinical testing on a new neurorestorative clinical indication for Bryostatin," stated Dr. Daniel Alkon, President and Chief Scientific Officer.

About Synaptogenix, Inc.

Synaptogenix is a clinical-stage biopharmaceutical company that has historically worked to develop novel therapies for neurodegenerative diseases. Synaptogenix has conducted clinical and preclinical studies of its lead therapeutic candidate, Bryostatin-1, in Alzheimer's disease. Preclinical studies have also demonstrated Bryostatin's regenerative mechanisms of action for the rare disease, Fragile X syndrome, and for other neurodegenerative disorders such as multiple sclerosis, stroke, and traumatic brain injury. The U.S. Food and Drug Administration has granted Orphan Drug Designation to Synaptogenix for Bryostatin-1 as a treatment for Fragile X syndrome. 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.

Additional information about Synaptogenix, Inc. may be found on its website: www.synaptogen.com.

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 anticipated initiation of a clinical trial to treat Fragile X with Bryostatin and continued development of use of Bryostatin-1 for Fragile X, Autistic Spectrum Disorder Deficits 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 its 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 its business plans or strategies. These and other factors are identified and described in more detail in the Company's filings with the Securities and Exchange Commission. The Company does not undertake to update these forward-looking statements.

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Robert Weinstein
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Synaptogenix, Inc.
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SOURCE Synaptogenix, Inc.