NEW YORK, Sept. 5, 2018 /PRNewswire/ -- Neurotrope Inc.
(NASDAQ: 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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SOURCE Neurotrope, Inc.