Bryostatin-1 from Synaptogenix Shows Statistically Significant Results as an ALS Treatment in Pre-Clinical Independent Study

News provided by

Sep 07, 2023, 09:00 ET

Positive changes in ALS gene expression and increased cellular viability met with statistical significance of p<.001

NEW YORK, Sept. 7, 2023 /PRNewswire/ -- Synaptogenix, Inc. (Nasdaq: SNPX) ("Synaptogenix" or the "Company"), an emerging biopharmaceutical company developing therapeutics for neurodegenerative disorders, today announced findings from an independent study demonstrating the potential for Bryostatin-1 as a treatment strategy for amyotrophic lateral sclerosis ("ALS"), also known as Lou Gehrig's Disease.

An open access article about the study was published in a special issue of the International Journal of Molecular Sciences titled "Neurodegenerative Disease: From Molecular Basis to Therapy." Under the leadership of Professor Sebastiano Cavallaro, an internationally recognized expert in the genetics of ALS, the study was conducted by scientists at the Institute for Biomedical Research and Innovation and the National Research Council of Italy, in collaboration with the University of Catania. Dr. Cavallaro has served for over 20 years as Research Director for the Institute for Biomedical Research and Innovation.

The new article demonstrates that the gene encoding for the enzyme protein kinase C epsilon ("PKCε"), the primary initial target of Bryostatin-1, showed an expression level that was significantly reduced (p<.001, 2-tailed) in the motor cortex samples taken from a genetically identified population of ALS patients when compared to control patients. Furthermore, in pre-clinical murine motor neurons genetically engineered to model the genetic signature of these ALS patients, there was a significant (p<.001, 2-tailed) reduction in the level of activated PKCε. Bryostatin-1 treatment of these ALS motor neurons significantly reversed the PKCε deficits.

Dr. Sebastiano Cavallaro stated, "Taken together, our findings suggest that PKCε alteration could play a role in ALS pathophysiology, and that PKCε agonism by Bryostatin-1 may represent a potential neuroprotective strategy against motor neuronal degeneration — at least in a specific subgroup of sporadic ALS patients."

Dr. Alan Tuchman, Chief Executive Officer of Synaptogenix, commented, "We are excited about the statistical significance Bryostatin-1 achieved in improving ALS gene expression and increasing cellular viability. The therapeutic potential for this drug platform supports our work for indications with significant unmet needs including Alzheimer's disease, multiple sclerosis (MS), and the rare disease Fragile X Syndrome. Third party validation of Bryostatin-1 in ALS is of great value to our Company as we continue to review options for our lead compounds. In keeping with our international outlook for potential acquisitions of asset rights or R&D funding for other assets, we are exploring opportunities with world-leading institutions on cutting edge treatment innovations for central nervous system diseases."

Dr. Daniel Alkon, President and Chief Scientific Officer of Synaptogenix, added, "The difference between the biochemical PKC measurement in genetically engineered murine motor neurons that were treated with Bryostatin-1 versus those untreated was statistically significant (p<.001). This study provides evidence that two key disease factors for ALS are deficits in the gene for the PKCɛ enzyme and deficits in the amount of the activated PKCε in neurons from the motor cortex of ALS patients. It is encouraging that Bryostatin-1 reversed these deficits in motor neuron models of the ALS cells."

ALS is a fatal adult-onset neurodegenerative disorder characterized by the progressive degeneration of upper and lower motor neurons in the cortex, brainstem, and spinal cord. Motor neuron deterioration results in muscle weakness and, ultimately, death due to respiratory failure, typically within three to five years after diagnosis. The global ALS market value is projected to reach $1 billion by 2032.¹

About Synaptogenix
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 multiple sclerosis, amyotrophic lateral sclerosis (ALS), Fragile X syndrome, and for other neurodegenerative disorders such as 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 database 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. 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 the Company 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.

Contact
800-811-5591
[email protected]

¹ Allied Medical Research, Global Opportunity Analysis and Industry Forecast, 2023-2032. April 2023.

SOURCE Synaptogenix, Inc.