Results Pave the Way for Future, Larger Trials
NEW YORK, NY / ACCESSWIRE / May 2, 2017 / A new world of clinical trial data testing is upon us. Older methods to determine a drug's worth, are being replaced by more robust, accurate methods that, through advanced computing, better predict future results. A pioneer using 'modified intent-to-treat' (mITT) analysis, Neurotrope, Inc. (NTRP) released its first top-line results for lead compound Bryostatin in very sick Alzheimer's Disease ((AD)) patients, with superb results.
Its Phase IIb of 147 patients underwent different dosages of Bryostatin along different timelines to see which worked best. Although the study was geared mostly for safety and tolerability, a surprise ensued - efficacy. Even more impressive was the generation of statistically significant results that exceeded FDA standards, which hold that a 'p-value', defined as the probability that good results are not due to chance, should be less than 0.1 in Phase II trials. In other words, 90% of patients are responding to a treatment. Neurotrope, on two standardized tests blessed by leading AD organizations, delivered figures even lower, meaning greater probability of results not being accidental.
Investors should recognize that Neurotrope's study was not necessarily about p-values but more about trends in cognitive improvement, clearly shown, and can lay groundwork for a larger study. Commenting on Bryostatin results in my conversation with Dr. Daniel Alkon, President and Chief Scientific Officer, he said "Ours is the first drug to show reversal of cognitive impairment in severe AD patients." Further, Dr. Alkon told me post-treatment follow-up reveals a continued benefit of Bryostatin treatment over time, in the absence of further dosing. This, I believe, can transform AD medicine, a sorely-needed remedy in light of so many failed trials, many of them at the hands of Big Pharma.
Neurotrope, with scientific vigor, showed positive, significant results from its 'Completer' group of patients, a standard in mITT analysis, comprising all those who crossed the finish line to 13 weeks of treatment. Theirs was a strong data test, not a prerequisite by FDA at Phase II, but forward-thinking in terms of validity. Dr. Susanne Wilke, CEO, told me the company purposely chose mITT/Completer metrics to show trends in increased cognition, stating they "wanted to be more rigorous". I applaud that stance, given much data today, particularly in AD, can be fraught with problems in data collection. Clinical trial AD patients are not known for their compliance (including drop-outs), but mITT/Completer methods smooth away data-gathering issues.
Bryostatin is unique because not only does it reduce the formation of toxic brain pollutants implicated in AD but also revitalizes neuronal function - it acts like a car battery jump-start to grey matter, restoring life and energy to brain components that have died or gone dormant from AD. Early proof from the Phase II study should be viewed with excitement from the AD community.
Pointing to Neurotrope's results, Maria Carrillo, Chief Science Officer of the Alzheimer's Association said even though this Phase II study was small and of short duration, "there was a trend towards improvement in memory, thinking and behavior" in a patient population hardest to treat. This was precisely Neurotrope's intent, resulting in a brave decision to test patients with advanced disease to restore synaptic health. Dr. Dean Hartley, another research heavyweight at the Association, admits there is nothing else approved that "slows down or prevents the disease." A true medical need exists and if more detailed data unveiled later this summer is good, FDA may grant its coveted Breakthrough Designation.
There is always the possibility that big trials don't produce equivalent results as smaller ones. Also, investors must better understand new trial methods - newer, in the case of Neurotrope's Phase II, mean superior. Many pharma companies are adopting this type of testing. Case in point is Biogen Inc. (BIIB), whose recent results in AD trials using mITT/Completer analysis were published in one of science's most respected journals - Nature.
Detailed results from a secondary endpoint, how AD patients perform effectively in daily living, are forthcoming. Bryostatin's future studies will be more highly-powered by virtue of a greater number of patients and more well-defined dosing ranges. In this study, improved trends in thinking and functioning were of ultimate importance and have, because of Neurotrope, become a reality.
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.
SOURCE: Small Cap Forecasting, Inc.