Schmiggins
22 hours ago
Good discussion. Seeing as they are favoring the "Milds" at 71% (even though the mean is right in the middle), they've designed it to make a strong case for a "Milds" approval. The "Moderates" - though there are less of them - will include some pretty advanced cases, and they are probably looking to see some good signs in that group but they are not relying on them to help much overall. I think they'll succeed for what they want and maybe there's a chance they luck out with the "Moderates". But if not, not a deal breaker.
The trial wrapping up 6 months later will possibly show that over more time the "Moderates" start to do better as the corrected proteins come in at higher numbers in their brains compared to the damaged ones. Also there will be data on the differences between the doses to look at.
I think the second trial may help the first trial if it needs it. Between the two trials, there will likely be enough positives to see to warrant approval, it seems reasonable, to me, to expect that approval.
The new seriousness seen in management - (I think Nicaise (Alexion) was brought in at the end of last year to become the CEO....He's been on the Sarepta board with Barry for years) - and the rising stock price and a whole bunch of new "lookers and likers" is going to keep the stock in focus up until the climactic moment at year's end. If ambiguity still ensues and a buying opportunity arises, take it .... The second trial will fix everything.
I bought in on Tuesday at 19. Had bought all the negativity for a year previous and didn't dive in deep until this week. I've been with Anavex for a year and Sarepta for 19 years. I sure wish Anavex had a Robertson, a Barry, a Nicaise, and this Matt N guy routing for it and helping it. Anavex does have the bad press that Cassave has had, so maybe that's one thing going for it! ....It's got the same screwy enemies!
Doc328
2 weeks ago
Agree that the chance of success is lower than 92% (imo quite a bit lower). AD is difficult and small cap bio's in this space are very speculative.
The corporate presentation showed 71% of the patients in the two trials are Mild (though table should say MCI or mild). With an average CDR-global of 0.75-0.79, many of the mild are actually MCI (CDR-G 0.5).
We don't know what the SAP actually has as the specifics of primary endpoint analysis but as long as pre-specified before the study is complete, it would be legitimate to have a primary endpoint analysis where the highest hierarchy is analysis of the 71% MCI/mild patients and a supplemental analysis (or secondary) to be the entire cohort. However, given the company's reputation being below that of most politicians, if this is true, they should state it before the trial is complete. Cassava should also release the dated SAP when the P3 results are released (at least IMO), especially if the results are good, to avoid claims of bias.
investingdog
2 weeks ago
from Matt N. on X
"I believe short sellers and big pharma are using the government to stop a promising and safe Alzheimer's drug from getting to patients. Dr. Wang, as a professor and researcher at a small university, is an easy target for the attack and is all alone to mount a fair defense. "
and more..
"Dr. Wang has dedicated his life to curing Alzheimer's disease, but now his safe, twice a day pill, in phase 3 trials at the FDA is coming under attack by short sellers, big pharma competitors, and the DOJ. The DOJ has indicted Dr. Wang 3 months before the first phase three trial finishes in October of this year.
Families on the drug and clinical trial sites that have run the trials have said that the drug does turn around Alzheimer's disease. "
investingdog
2 weeks ago
The Cognition Maintenance Study (CMS) was a Phase II randomized withdrawal trial evaluating simufilam, an oral therapy developed by Cassava Sciences, for the treatment of Alzheimer's disease. Here are the key findings from the study:
Study Design:
The CMS enrolled 157 patients with mild-to-moderate Alzheimer's disease.
All patients first received open-label simufilam 100mg twice daily for 12 months.
Patients were then randomized 1:1 to either continue simufilam or switch to placebo for 6 months.
Primary Endpoint:
The pre-specified cognitive endpoint was mean change in ADAS-Cog11 scores over 6 months, comparing simufilam to placebo.
Key Results:
Simufilam slowed cognitive decline by 38% compared to placebo over the 6-month randomized period.
The placebo arm declined 1.5 points on ADAS-Cog, while the simufilam arm declined 0.9 points.
Patients with mild Alzheimer's disease (MMSE 21-26) who continued on simufilam showed no material decline in ADAS-Cog scores over 18 months, indicating stable cognition.
Safety:
Simufilam 100 mg twice daily was reported to be safe and well-tolerated.
There were no drug-related serious adverse events.
No treatment-emergent adverse events occurred in 5% or more of study participants.
The placebo group did better than expected due to the lingering drug effects, especially for the first 3 months.