Synaptogenix Inc (SNPX)

[runncoach]

17 week data.
Important to those of us following the science. London abstract is now published on line. https://ep70.eventpilot.us/web/planner.php?id=AAIC17

Bryostatin-1 Improves Cognition and Daily Living Tasks in Moderate to Severe Alzheimer’s Disease: Preliminary Report of a Phase 2 Study

Abstract ID: a19955

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Abstract:

Martin R. Farlow, MD1,2;Jeffrey M Burns, MD, MS3,4;Kenneth J Gorelick, MD5;David R Crockford, BA5;Elaine Grenier, BS5;Susanne Wilke, PhD5;Ellen C Cooper, MD6 and Daniel L. Alkon, MD5, (1)Indiana Alzheimer Disease Center, Indianapolis, IN, USA, (2)Indiana University School of Medicine, Indianapolis, IN, USA, (3)University of Kansas Alzheimer's Disease Center, Kansas City, KS, USA, (4)University of Kansas Alzheimer's Disease Center, Fairway, KS, USA, (5)Neurotrope Bioscience Inc, New York, NY, USA, (6)ClinReg Solutions LLC, Rockville, MD, USA

Background: In preclinical studies, the PKCe activator bryostatin-1 induces growth of mature synapses, prevents neuronal death, and influences amyloid plaques and tau tangles. We report the first safety and efficacy study of repeated doses of bryostatin in Alzheimer’s disease (AD). Methods: Adults with moderate–severe AD (MMSE-2: 4-15 inclusive) received biweekly infusions of placebo or bryostatin-1 (20 or 40µg) for 12 weeks. Endpoints included the Severe Impairment Battery (SIB) and an Activities of Daily Living Inventory-Severe Impairment Version (ADCS-ADL-SIV) at baseline, weeks 5,9,13, and 30d after last infusion. Safety evaluations included routine blood tests and treatment emergent adverse events (TEAEs). A mixed-model for repeated measures was used to assess change from baseline to week 13 (?13) between each bryostatin group and placebo (1-tailed a=0.10 and power=80%) in predefined populations of the Full Analysis Set (=1 post-baseline efficacy assessment) and Completer AnalysisSet (completed week 13 visit). Randomization was stratified by MMSE-2 4-9 vs. 10-15. Bryostatin-1 was provided by the National Cancer Institute. Results: 147 adults were randomized in 28 centers with n=135 in the Full Analysis Set and n=113 in the Completer Analysis Set. Full Analysis Set mean(sd) age=71.6(7.9), MMSE-2=10.2(3.4), and 51.9% females with no imbalances among arms. 83.0% used acetylcholinesterase inhibitors, 67.4% memantine, and 58.5% both. The ?13 (80%CI) in SIB was 1.9(-0.3,4.2) for 20µg and 0.8(-1.4,4.0) for 40µg in the Full Analysis Set, and this finding was strengthened when analyses were confined to the Completer Analysis Set (2.6 [0.4,4.9] for 20µg and 1.5[-0.7,3.8] for 40µg). Subjects who completed the 30d followup had sustained benefit. In the FAS, ?13 for the ADCS-ADL-SIV was 1.4(0.0,2.8) for 20µg and 0.8 (-0.6,2.3) for 40µg group. TEAE rates were 58% in placebo, 65% in 20µg and 83% in the 40µg groups. TEAEs >placebo in both arms included diarrhea, while the 40µg arm also included fatigue, weight loss and myalgia. Conclusions: 20µg of bryostatin-1 every other week for 12 weeks appeared safe and benefited cognition and activities of daily living in moderate to severe AD. These results suggest acceptable safety and clinical benefits for the 20 µg group and support further development of bryostatin-1.

Sentence that is striking is this one: "Subjects who completed the 30d followup had sustained benefit." Seems to validate everything Wilkes and Alkon alluded to in May 1 conference call. Seems to validate synapses regrowth as NO Drug was given during that time period. Seems to validate detailed treatment info given for compassionate use patients which showed upswing in 12-13 week data trend which reached a high point in 17ish week timeframe before leveling off and remaining at that level for months. If improvement was sustained a month later than week 13, almost a certainly becomes stat significant at that point. Heck, it was easily stat sig in completer population at week 5 already. Basically everything pointed out as a potential positive May 1 has born to be the truth with this presentation IMO.

So question is if you had a bunch of patients like CU patient #3 from the 2a Alz Journal write up, would you consider that a success because it seems that its the trend line several dozen other patients were on in this trial. If you think not, then might be time to move on. Company will need funding at some point in the future or will need to partner. Probably plenty of money for FragileX and a few dozen patients on open label extension. From there company needs to tell us the plan. JMHO