NEW YORK, May 1, 2017 /PRNewswire/ -- Neurotrope,
Inc. (NASDAQ: NTRP) today announced positive top-line results
from its Phase 2 study (-202 Study) of Bryostatin-1 in patients
with moderate to severe Alzheimer's disease (AD), a population not
commonly targeted in AD clinical trials. Bryostatin-1,
a Protein Kinase C epsilon activator that works through
synaptic growth factors, as well as anti-amyloid and anti-tangle
signaling pathways in the brain, has been shown, in
non-clinical efficacy studies, to induce the growth of mature
synapses in the brain and prevent neuronal death. Thus,
Bryostatin-1 has a fundamentally different biological mechanism of
action with the potential for longer lasting effects than the other
currently marketed drugs for AD (e.g., donepezil (Aricept®) and
memantine (Namenda®)).
This Phase 2 study was the first repeat dose study of
Bryostatin-1 in patients with late stage AD (defined as a Mini
Mental State Exam 2 (MMSE-2) of 4-15), in which two dose levels of
Bryostatin-1 were compared with placebo to assess safety and
preliminary efficacy (p < 0.1, one-tailed) after 12 weeks of
treatment. The pre-specified primary endpoint, the Severe
Impairment Battery (SIB) (used to evaluate cognition in severe
dementia), compared each dose of Bryostatin-1 with placebo at Week
13 in two sets of patients: 1) the modified intent-to-treat (mITT)
population (consisting of all patients who received study drug and
had at least one efficacy/safety evaluation), and 2) the Completer
population (consisting of those patients within the mITT population
who completed the 13-week assessment).
Top-line results indicate that the 20 µg dose, administered
every two weeks, met the pre-specified primary endpoint in the
Completer population, but not in the mITT population. Among
the patients who completed the protocol (n = 113), the patients on
the 20 µg dose at 13 weeks showed a mean increase on the SIB of 1.5
vs. a decrease in the placebo group of -1.1 (improvement of 2.6) (p
< 0.07) (n = 80), whereas, in the mITT population, the 20 mcg
group had a mean increase on the SIB of 1.2 vs. a decrease in the
placebo group of -0.8 (improvement of 2.0) (p < 0.134) (n =
90).
A total of 147 patients were enrolled into the study; 135
patients in the mITT population and 113 in the Completer
population. The Alzheimer Disease Cooperative Study Activities of
Daily Living Inventory Severe Impairment version (ADCS-ADL-SIV) was
a secondary endpoint. The p values for the comparisons between 20
µg and placebo for the ADCS-ADL endpoint were 0.082 and 0.104,
respectively, among the patients who completed the protocol in the
mITT population. Analysis of secondary and numerous additional
exploratory endpoints are ongoing.
Together these results indicate, in this relatively small trial,
that Bryostatin-1, at the 20 µg dose, improved outcomes in
important dimensions that are impaired in patients with moderate to
severe Alzheimer's disease i.e., cognition and the ability to care
for oneself. Since most of the patients in this study were already
taking donepezil and/or memantine, the efficacy of Bryostatin-1 was
in addition to standard of care.
The safety profile of Bryostatin-1 20 µg was similar to that of
the placebo group except for a somewhat higher incidence of
diarrhea. Fewer adverse events were reported in patients in the 20
µg group, compared to the 40 µg group. The mean age of patients in
the study was 72 years and similar across all three treatment
groups.
"The results of this relatively small randomized, double-blind,
placebo controlled study of Bryostatin-1 shows that Bryostatin-1
has the potential to positively impact the lives of these severely
debilitated patients with moderate to severe AD, a population that
is in dire need of new therapies, especially drugs with a new
mechanism of action," said Dr. Susanne
Wilke, Neurotrope's Chief Executive Officer. "We are excited
to take the next steps in advancing the development of Bryostatin-1
to treat this serious disease that every year becomes a larger and
larger public health burden in the U.S. and around the world.
Additional development, with a path to Phase 3, is clearly
warranted."
"These results, which show improvement in patients with moderate
to severe Alzheimer's disease, the population that is generally
recognized as the most difficult to treat, provide exciting
evidence of a new therapeutic approach potentially could rejuvenate
synaptic networks in the brain. Improvements across the range of
important manifestations of the underlying neurodegenerative
disease, as shown in this Phase 2 study, could potentially
represent a shift in the paradigm to treat Alzheimer's disease,"
said Dr. Daniel Alkon, President and
Chief Scientific Officer of Neurotrope. "I would also like to thank
the National Cancer Institute for their generous donation of the
Bryostatin-1 we have used in our clinical trials."
Further analysis of the data from the Phase 2 -202 Study is
ongoing, and Neurotrope plans to present more data and analyses
from this study at future medical conferences. Once all of
the data and analyses have been reviewed, the Company plans to meet
with the Food and Drug Administration (FDA) to address the clinical
and regulatory path forward for Bryostatin-1.
Dial-in Information
A conference call will be held
today at 8:30 am Eastern Time. The
dial-in information to access the live call is listed below:
Participant Toll Free
Dial-In Number:
|
(877)
245-7303
|
Participant
International Dial-In Number:
|
(478)
219-0731
|
Conference ID:
|
16070779
|
The dial-in information to access the call replay, available for
30 days after the live call, is listed below:
Replay Toll Free
Dial-In
Number:
|
(800)
585-8367
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Replay International
Dial-In Number:
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(404)
537-3406
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Conference ID:
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16070779
|
About Neurotrope
Neurotrope is at the forefront of
developing a novel therapy to treat and potentially reverse
moderate to severe Alzheimer's dementia and other neurodegenerative
diseases. The Company's world-class science is a paradigm shifting
approach that treats some of the underlying causes of Alzheimer's
disease.
The scientific basis of our treatment is activation of Protein
Kinase C isozymes ε and α by Bryostatin-1, a natural product, which
in mouse Alzheimer's disease models was demonstrated to result in
repair of damaged synapses as well as synaptogenesis, the induction
of new neuronal networks, reduction of toxic beta-amyloid
generation, prevention of neuronal death, and enhancement of memory
and learning, thus having the potential to improve cognition and
behavior in Alzheimer's dementia.
Neurotrope has conducted a Phase 2 trial of Bryostatin-1 in the
treatment of moderate to severe Alzheimer's dementia, as well as
preclinical studies of Bryostatin-1 as a treatment for Fragile X
Syndrome, Niemann-Pick Type C disease and Rett Syndrome, three 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
Syndrome. Bryostatin-1 has undergone testing in over 1,500
people establishing a large safety database.
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. These statements are subject to the risk that further
analyses of the Phase 2 data may lead to different interpretations
of the data than the analyses conducted to date and/or may identify
important implications of the Phase 2 data that are not reflected
in these statements. Clinical trial data are subject to
differing interpretations, and regulatory agencies, medical and
scientific experts and others may not share the Company's views of
the Phase 2 data. 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, 2016.
The Company does not undertake to update these forward-looking
statements.
Please visit www.neurotropebioscience.com for further
information.
For additional information, please contact:
Neurotrope Bioscience, Inc.
Jeffrey Benison, Director of Corporate Communications
973.242.0005 Ext. 101
jbenison@neurotropebioscience.com
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SOURCE Neurotrope, Inc.