"The Ariana and PK/PD analysis is sussing that all out (what dosages are tolerated and most effective for different types of people). Ph 2a wasn't even optimized or powered, yet Anavex saw unexpected, stat sig efficacy results; that's unprecedented at 57 weeks (and soon 15 mos)."
From what I have read on this board I believe most people here would like to see Neurotrope and Anavex both succeed. I would include myself in that group. Unfortunately, for me anyway, I have done enough DD that certain things make me extremely nervous when I see it occurring with respect to claims of statistical efficacy. I'll put this here as a cautionary tail for Neurotrope investors as well, so they will be aware in the event that Neurotrope uses similar techniques in analyzing and presenting their data. You simply cannot, or at least should not, post charts that indicate "all patients" without defining "all patients" when there are dropouts. Why one might ask?
There were significantly more dropouts in the 23 mg SR group (30%) than in 10 mg IR group (18%). While the primary Intent-to-Treat Population-Last observation Carried forward (ITT- LOCF) analysis and the secondary Observed Cases (OC) analysis of the change from baseline in SIB at Week 24 were nominally significant in favor of the high dose, significance was lost for an analysis assigning the lowest rank for dropouts and some other similar sensitivity analyses. In most cases where significance was lost the high dose was still numerically better than the low dose, but these analyses still raise the importance of the question of whether the trial had assay sensitivity. The mean SIB was between 10 and 20 points higher at baseline in this trial than in the previous Donepezil trials in which the SIB was used as an endpoint; this may further complicate cross trial comparisons and the lack of a placebo control issue.
See how the method of data imputation for dropouts can change the statistical significance of the results? I even emailed IR to ask for clarification. No reply
Next, investors should really be cautious when they see words like "unprecedented" being used, it means "never done or known before".
This is where doing DD tends to make one even more nervous. It looks like, to me anyway, that AVXL's results are indeed not "unprecedented."
"Myriad Presents Additional Flurizan™ Phase 2 Study Data Demonstrating Benefit in Alzheimer's Patients 42% of Patients on Flurizan™ Had Improved or Not Declined After 24 Months
SALT LAKE CITY, UT, March 05, 2007—Myriad Genetics, Inc. (NASDAQ: MYGN) (www.myriad.com) announced today that it presented additional results of its completed Phase 2 follow-on study of Flurizan™ in patients with mild Alzheimer's disease at the annual meeting of the American Association for Geriatric Psychiatry (AAGP), held March 1-4, 2007 in New Orleans. The data indicate that Flurizan may be capable, not only of slowing the decline of Alzheimer's disease, but of halting the disease in its tracks. In this study, many patients with Alzheimer's disease got no worse over two full years, and in some cases, patients treated with Flurizan appear to have improved. At 24 months, study participants in the Phase 2 trial with mild Alzheimer's disease taking 800 mg twice daily Flurizan experienced a 67% improvement in their level of cognitive decline compared with placebo, as measured by the Mini Mental State Exam (MMSE) score. This difference was highly significant statistically (p=0.001). Additionally, based upon the MMSE score, three times the percentage of patients on Flurizan demonstrated improvement in cognition or zero decline, compared to patients on placebo: Forty-two percent of patients on 800 mg twice daily Flurizan experienced improvement or zero decline, compared with 14% of patients taking placebo. MMSE is the primary test used by most clinicians to help diagnose, assess and monitor progression of patients with Alzheimer's disease. It was also the principal criterion for selecting patients to enroll in the Phase 2 study, and is a secondary endpoint in the two ongoing Phase 3 trials of Flurizan.
Overall, 42% of patients on Flurizan showed improvement or no decline in one or more of the three primary endpoints of cognition, global function and activities of daily living, compared to 10% of patients on placebo. On the test that measures cognition, ADAS-cog, 25% of study participants taking 800 mg BID of Flurizan showed cognitive improvement or experienced zero decline in cognition after 24 months, compared with none on placebo. With the CDR-sb test, a measure of overall function in Alzheimer's patients, 29% of study participants on Flurizan experienced an improved or zero decline score, compared with none of those on placebo.
Robert C. Green, M.D., MPH, Co-Director, Alzheimer's Disease Clinical & Research Program, Professor of Neurology, Genetics and Epidemiology at the Boston University School of Medicine, and a lead investigator on the Phase 3 trial of Flurizan, commented, "This analysis of patient response to Flurizan in the Phase 2 trial suggests that the drug may, in many patients, actually halt disease progression over a 24-month time frame. Since Flurizan appears to slow the biological progression of the disease, this is an exciting and novel finding, and if replicated in the ongoing Phase 3 trials will be extraordinarily important."
To recap the efficacy results of the Phase 2 study at month 24 presented earlier, mild patients taking 800 mg of Flurizan twice daily had an effect size of 72%, with a statistically significant value of p=0.0005, as measured by their global function on the CDR-sb test. In activities of daily living, the patients showed a statistically significant 67% effect size (p=0.015). Cognition improvement showed an effect size of 52% at 24 months on the ADAS-cog scale. These data suggest that there is a substantial benefit from Flurizan. The additional data presented at the AAGP meeting and announced today add detail to these effect sizes by demonstrating that, not only did the population as a whole respond to the treatment, but a meaningful portion of the patients who responded to the treatment did so by experiencing either zero decline after two years or a reversal of their decline to actual improvement, something that is very rare in Alzheimer's disease. Comparisons to placebo at 24 months refer to the placebo group as originally randomized.
The vast majority of patients in this Phase 2 study, approximately 94% at the time of enrollment, were receiving stable doses of acetylcholinesterase inhibitors, which are FDA-approved drugs for symptomatic treatment of Alzheimer's disease. Thus, the benefits of Flurizan observed in these patients were over and above the current standard of care.
"The 24 month Phase 2 responder analysis provides further evidence of efficacy against mild Alzheimer's disease that is consistent with our understanding of Flurizan's mechanism of action as a SALA," said Adrian Hobden, Ph.D., President of Myriad Pharmaceuticals, Inc. "The results support our belief that Flurizan is modifying the course of the underlying disease process."
It would be great if we don't "shoot the messenger" here. I did not make any of this up nor did I actually "want" to find it, nonetheless there it is. Nothing above means A2-73 or Bryostatin will succeed or fail but it's important to keep in mind as an investor imho.
Be very cautious when companies start "data mining" for subgroup "super responders" and proceeding to Phase 3 trials based on that type of data analysis. Here's why.
"Flurizan, a drug that reduced the production of apparently harmful amyloid in laboratory and animal experiments, has failed to show a significant benefit in a large-scale, “Phase III” trial in about 1,700 people with Alzheimer’s disease. Its maker, Myriad Pharmaceuticals of Salt Lake City, announced the results today and indicated that it would discontinue development of the drug."
News 
Market Data 
Discover 
