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
ir@synaptogen.com
1 Allied Medical Research, Global Opportunity
Analysis and Industry Forecast, 2023-2032. April 2023.
View original
content:https://www.prnewswire.com/news-releases/bryostatin-1-from-synaptogenix-shows-statistically-significant-results-as-an-als-treatment-in-pre-clinical-independent-study-301920491.html
SOURCE Synaptogenix, Inc.