A month ago, we discussed Neurotrope (NASDAQ: NTRP) and its anticipated results from its study of Bryostatin-1 as a therapy for patients with moderate to severe Alzheimer’s disease. The company announced results this morning that showed no statistically significant improvement in patients vs placebo.
There hasn’t been a new medication approved for Alzheimer’s disease in 14 years, and anticipation is always high – emotionally and financially – whenever a company teases reasons for optimism, as Neurotrope had done. The crucible, of course, is always in the clinic, and the results from this Phase 2 study represented yet another disappointment for those who were hoping for a breakthrough.
The company tried to put a positive spin on the results with a press release this morning headlining “Positive Top-Line Results,” but the market swiftly parsed beyond the headline to see that the results were not statistically significant. As of this writing at mid-day, the stock is trading down over 57% to $8.01.
The primary endpoint of the 148-patient study was the Severe Impairment Battery (SIB), a series of 40 questions that are commonly used to evaluate cognition in severe dementia. The company released results on two subgroups of patients:
- A modified intent-to-treat (mITT) population, consisting of 90 patients who received Bryostatin-1 and had at least one efficacy and safety evaluation.
- A “Completer” population , consisting of 80 patients within the mITT population who completed the 13-week assessment
The press release stated that the 90 patients in the mITT population receiving a 20 microgram (µg) dose of Bryostatin-1 showed a mean increase on the SIB of 1.2 points vs. a decrease in the placebo group of -0.8 point – a difference of 2.0 points with a P value < 0.134. Further, the 80 patients who were on the 20 µg dose at 13 weeks showed a mean increase on the SIB of 1.5 points vs. a decrease in the placebo group of -1.1 points, for a difference of 2.6 points with a P value < 0.07.
We won’t belabor you with statistics, but the P value here is a measure of the probability that there is no difference between the groups you’re testing – in this case, between the group on Bryostatin-1 and the group on placebo. Results like these are considered to have statistical significance if the P value is less than 0.05, or 5%. In other words, a low P value indicates that your testing sample is meaningful. Despite the positive claims from the company, it’s clear to us and to the market that the results showing only a 2.6 point improvement after 13 weeks with a P value < 0.07 is simply not a statistically significant clinical result.
We spoke with Jeff Benison, Neurotrope’s head of IR, a couple of weeks ago, and he shared the company’s optimism about what the study results would show. We were hopeful too, as was anyone who follows the market or is affected in some way by Alzheimer’s disease. We look forward to speaking with him again, and perhaps also with Dr. Daniel Alkon, Neurotrope’s President and Chief Scientific Officer. We would lead with two questions:
1. The study tested two doses of Bryostatin-1: 20 µg and 40 µg. The press release only discusses results from the testing of the lower dose. What happened with patients on the higher dose? Those results are conspicuous by their absence.
2. The press release states that additional development of Bryostatin-1, “with a path to Phase 3, is clearly warranted.” We would challenge this assessment based on the results, and would like to hear greater exposition on why the company feels that further study is indicated.
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(GIF source: Elvis Weathercock)
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