Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
The Shake It Up group sponsored the earlier trial and wouldn’t be helping sponsor this plus the Australian government if the earlier trial data wasn’t SOLID.
TGD has commented that he expects the Rett to price in the $400-600K per patient.
The total US Rett population is about 10,000.
Do the math.
That was an easy ignore call.
Only a projected 350% increase over todays price.
Maybe I should buy some crypto instead.
In the past we often saw a sell off after these events. Unfulfilled expectations.
Today is different.
If the company ultimately isn’t able to show they met the end points there will be a shareholder suit.
Missling is smart enough to not set the company up for that.
Now can we get the PDD OLE data we were promised by year end 2022.
I believe Missling understands the best way to silence a short attack it to shove a peer reviewed journal article down their collective throats.
Answering my own question.
I see in clinicaltrials.gov the Pediatric Rett trial is described as a dose escalation trial.
No maximum aimed at however due to a child’s size probably less than the 50 max in the adult AD trial.
Should give even better results than the 5mg used in the Adult Rett.
Yes 5 mg for adult Rett seemed very low compared to the 50mg most adults in the Phase 2 AD trial could tolerate.
I wonder why such a low number?
Are they titrating to a higher dose in the pediatric?
I can understand the children are fragile and the risk could be too great.
A death would do a lot of harm to 2-73 credibility as being safe.
However if tolerated the clinical response to a dose above 5mg could exceed the impressive response shown in the adult Rett trial.
Yes 5 mg for adult Rett seemed very low compared to the 50mg most adults in the Phase 2 AD trial could tolerate.
I wonder why such a low number?
Are they titrating to a higher dose in the pediatric?
I can understand the children are fragile and the risk could be too great.
A death would do a lot of harm to 2-73 credibility as being safe.
However if tolerated the clinical response to a dose above 5mg could exceed the impressive response shown in the adult Rett trial.
Such a short trial period for a relatively slowly progressive disease makes our 48 weeks look more likely to be statistically significant.
Hiring of three FDA veterans to me sends a strong signal that Missling is not holding back expecting to hand over the filing job to partners.
Waiting to partner after filing strong NDA’s will maximize shareholder value.
TGD said I believe in December that they were close to final enrollment in Pediatric Rett.
No PR saying it is fully enrolled.
If yes time to run trial and crunch data I think no TLD until mid year.
Lack of OLE TLD for PDD has no explanation from TGD even though he had repeatedly told us to expect by year end 22.
Lack of any explanation obviously undermines confidence in the company's ability to meet stated deadlines.
AD, Rett, PDD.
Three NDA’s
Three FDA veterans.
?
Missling’s masterful raising of needed cash is one reason I support his leadership of the company.
Anavex could market Blarcamasine as a medication to substantially reduce falls in the Alzheimers population, even if nothing else.
?????
Straight line decline markers if with a good p value should be enough.
Surrogate markers are only used when straight line decline markers are too weak to justify approval on their own.
Unfortunately it will be difficult to show a meaningful
slowing of Amyloid build up because it normally would only increase a slight amount over the time of the trial.
Biogen is more clearly showing its reduction.
Activation of increased Sigmar1 expression to me is a better bio marker but so far the FDA has not been asked to see it as a surrogate marker for AD.
Give us some simple graphs
comparing placebo, 30mg and 50 mg cohorts.
Clear easily understood by even non experts in the field like the Alzheimers advocacy groups.
Get some retail press!!
I messaged Joseph.walker@wsj.com
to take a look at the Anavex AD program.
Let’s all do it.
Missling has said the application will be for all ages not separate pediatric and adult.
I assume he still believes it will still be eligible for the childhood rare disease voucher.
From March 22.
Old news, but still great.
Since they are just finishing trial enrollment as we speak the time to run the trial plus time to crunch the numbers I think second quarter at the earliest for us to see the data.
Third to fourth quarter best hope for FDA approval if data is as good as Adult Rett phase 3 trial data.
Since they are just finishing trial enrollment as we speak the time to run the trial plus time to crunch the numbers I think second quarter at the earliest for us to see the data.
Third to fourth quarter best hope for FDA approval if data is as good as Adult Rett phase 3 trial data.
I’m really mystified as to why the trial for 2-73 in Fragile-X hasn’t been started by now.
It’s a screaming target that even Randi Hagerman is very excited about yet nothing for so long.
Is TGD saving the company cash for a follow up Phase 3 in AD?
Damaging the reputation of the Alzheimers Association will do no good for when good therapies like Blarcamasine are developed their recommendation will be meaningless.
Make it $32 billion and we’ll talk, but just for the AD IP.
“management responses”
Is this Anavex management complaining of misrepresentation of the recent data for the purpose of driving down share price?
Roche presented their failed trial data at CTAD the day before Anavex presented.
Roche must have been in the room to see it.
Of course it will cross their mind whether to approach Anavex to have a conversation, which in fact may have already been started earlier.
I like 40 minute time slot for the oral presentation.
Unlike the 15 minute oral at CTAD plenty of time to drill down on the details of the 2b/3 trial including how biomarkers relate to response, and breakout of 30 and 50 mg cohort responses.
I agree.
Combining the 30 and 50 mg data gives the result of a base hit.
From what we see in the Phase 2a the 50mg data could be the home run that gets the crowd in the stands on their feet cheering.
I agree the peer reviewed article should push the nay sayers back under their rocks, however since the raw data was obtained only days before the CTAD conference it may take months for the results to be calculated including all the genomic data, put into an article then time for the peer review then time to publish.
I’m hoping less than six months but wouldn’t be surprised if longer.
After again listening to todays presentation I remain highly confident of 2-73 success in AD.
https://video.wixstatic.com/video/79bcf7_6566f1d485d2424eadf745c64a05ff7a/480p/mp4/file.mp4
Expert in the field with no connection to the company saying “improvement” after looking at the data presented.
How much clearer does that have to be said?
Before the open.
I like that.
50 mg data please!
I suspect the company would have preferred more time to analyze and put data into a clear presentation including graphs.
However they felt CTAD was the best venue to present the top line and did the best they could in an extremely tight time line.
My guess the CRO did the data lock later than anticipated.
I agree.
The 30 50 breakout is key to showing eye popping results no AF smoke can hide.
50 was always going to be the dose the company was going to be applying to the FDA to be the new Standard of Care in AD.
I find it highly significant that the data was a combination of the 30 and 50 mg treatment groups.
We know from the Phase 2 trial the 50 mg group did significantly better than the 30 mg group.
If the segregated data in this trial follows that of the Phase 2 the 50 mg treatment arm data will be very very strong and convincing and will become the intended standard of care.
Ease of administration, lower cost and minimal side effects.
Pick you winner.