Neurotrope Nears Breakthrough in New Alzheimer’s Treatment

Top Line Data Expected in April 2017 in Placebo Controlled Study of Bryostatin-1 in 148 Moderate to Severe Alzheimer’s Patients

NEW YORK, NY / ACCESSWIRE / April 4, 2017 / Too much modern medicine relies on treating symptoms, not underlying cause. This is especially clear with Alzheimer’s Disease ((AD)), where 5.4 million Americans and 44 million people worldwide suffer in a market of $236 billion here and $605 billion globally. Old drugs do not work. Needed now is something new, especially in light of demographics. Neurotrope, Inc. (NASDAQ: NTRP), a stand-out in progressive AD solutions led by visionary Daniel Alkon, MD, President of Neurotrope, endeavors to reverse AD by restoring synaptic response. Neurotrope, under Dr. Alkon’s leadership, recently concluded its first step in a Phase II trial using proprietary drug bryostatin on moderate to severe AD patients. Results are imminent.

Neurotrope’s approach is exceptional. Bryostatin is clinically known to stimulate neurons, key brain components that command higher and lower functions from complex problem solving to memory to simple movement, in addition their electrical lieutenants synapses which send signals throughout the brain to help mammals learn to survive and adapt. Neurotrope’s latest study, like its former, smaller ones, should show the importance of bryostatin in synaptic well-being.

Four drugs for AD are FDA-approved. New studies involve monoclonal antibodies (mAbs) that aim, like marketed drugs, to tackle amyloid proteins leading to plaque (and fibrotic brain tangles, a structural hallmark of AD), again sidestepping treatment of the underlying cause of AD dementia – destruction of synaptic activity that would otherwise keep patients sharp. Another commonality among past and trialed AD drugs is treating mild disease. Neurotrope treats patients in advanced AD, a bold testimony to its belief that bryostatin can reverse synaptic destruction at any stage.

AD and the Outdated Amyloid Hypothesis

After 20 years of research and failed late-stage trials by big name Pharma, scientists began to understand the lack of evidence between ridding plaques and tangles and better cognitive function: drugs controlling plaque build-up do not restore memory or prevent the decline into severe dementia and eventual death.

Work published in 2008 took an early stab at refuting amyloid hypothesis, effectively showing that of AD sufferers treated for plaque removal with success, 87.5% still died from severe AD dementia. This pointed to synaptic health as a key factor in AD mortality.

Recognizing a connection with drops in plaque load and no halt to cognitive decline, other AD researchers adopted a new theory – one that Neurotrope ascribes to – that neuronal structures in the brain and resulting synaptic loss are the cause of dementia, not plaques or tangles. Recent studies advance this renegade supposition. Autopsy of AD patients reveal significant plaques and tangles show up in 40% of elderlies without cognitive problems.

Other work advances the idea that amyloid plaques emit toxicity, so decreasing plaque should yield a good therapeutic result. Failed clinical trials dash this theory.

Brief History of Bryostatin

Bryostatin comes from the sea animal Bugula neritina, a rich source of raw material to make the compound (although less labor-intensive synthesized versions with equivalent potency are available and nicely locked up within Neurotrope’s substantial IP estate). Originally investigated as an anti-cancer drug, lack of efficacy canceled studies. Government agencies retained interest in bryostatin as a memory booster via a biochemical cascade that pumps up brain enzymes and proteins to stimulate synaptic connections. Targeting AD, with its known role in memory loss, was a natural progression and Neurotrope became the cause’s advocate.

Neurotrope found bryostatin delivers a doubled-barreled approach to AD: damage from plaques and tangles decline while synapses repair themselves and even grow, unheard of in the medical community. To the point: synaptic loss goes hand-in-hand with a precipitous plunge in cognition necessary for daily living.

Dr. Alkon, who studied the intricacies of memory loss early in his career, is passionate about reversing brain deterioration to herald a new era of brain medicine. Upon completion of Phase II enrollment of nearly 150 AD patients last November, he stressed the importance of Neurotrope’s aim to treat severe disease as competitor’s stock clinical trials with only those mildly impaired. His conviction of success is based on Neurotrope’s “experience with severe patients through our compassionate use studies” that indicated “reversal of some of the manifestations of the disease.” Bryostatin would be the first new drug for AD in 15 years.

Bryostatin works fast: less than two weeks to see initial cognitive improvement as synaptic connections heal and grow. Dr. Suzanne Wilke, CEO of Neurotrope, lends insight, telling me in a recent interview “the fast and magnitude of effect of bryostatin on synaptic function may have to do with the sheer increase in the number of synapses per neuron being regenerated or maturing.” This makes sense when considering there are 10 billion neurons in the human brain, comprising 100 trillion synapses.

Clinical Trials Backgrounder

More than 1,500 patients under government-sponsored experimentation have shown bryostatin safe and well tolerated. Mice induced with stroke, then fed bryostatin rebounded well from disability and did not require new training to find their way through a maze that had been obliterated from memory. FDA allowed compassionate use of bryostatin, or treatment of AD patients without the structure of a formal clinical trial. Originally meant to be support of safety data, Neurotrope found a marked increase of cognitive ability, still prevailing after one year since bryostatin treatment.

Now on to a bigger Phase IIb: 148+ AD patients with moderate to severe disease have been tested in over two dozen US sites. Dosing and patient monitoring is complete. Primary and secondary endpoints involve standard paper-based tests to assess cognitive impairment (or improvement) and how patients cope with daily living, and other neuropsychiatric metrics. Binary, top line data is expected next month.

Neurotrope has no revenue, but through a recent funding has approximately $25 million in cash. With a low monthly burn of $300,000, runway is respectable but larger trials may make future funding necessary.

Risks to an investment in Neurotrope are high. Curing mice and a handful of humans may not translate to efficacy in larger trials, although safety data seems established. Alzheimer’s is a physiologically complex condition, and attempting to treat later-stage patients may be tricky. Money may be needed to finance ongoing trials. A future raise may cause dilution. Competition looms large, despite recent failures. If Merck & Co. (MRK), who surrendered defeat in Phase III for AD but insists on re-evaluation of the same drug to be used for earlier stages of AD, and succeeds, the competitive landscape could change drastically.

Although injectable now, there is a chance for bryostatin to be made into oral form. Imagine you as a caretaker for a progressively degenerating AD family member and able to give a pill to enhance cognitive function quickly, with long term results. Your job and everyone’s quality of life improves.

Neurotrope’s near-term market moving catalyst is top-line efficacy data which is due towards the end of April. Positive Phase IIb results would entirely transform the way Alzheimer’s is treated and impact the lives of a generation of patients and their families.

About Small Cap Forecasting, Inc.

Sharon di Stefano has spent 20 years as an analyst, beginning her career at Smith Barney, Harris Upham & Co. specializing in medical devices, pharmaceuticals, healthcare information technology, and bio-pharmacology. Ms. di Stefano had also served as Senior Venture Officer for the Edison Innovation Fund, implemented through the New Jersey Economic Development Authority that provided funding for early-stage life sciences companies. Industry experience includes laboratory research for Johns Hopkins Hospital and the Department of Defense. Ms. di Stefano received a Master’s of Science degree, in Business, from Johns Hopkins University in 1986, and a Bachelor of Arts from the University of Delaware in 1984 with a minor in biology.

Media contact:

Jackie Rodriguez

646-430-5783

SOURCE: Small Cap Forecasting, Inc.

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