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Msg  3834 of 3936  at  12/3/2023 8:09:54 AM  by

chvoja


Rett syndrome


Hello,
I'm new to AVLX and I'm trying to understand the data already gathered on blarcamesine.
 
I try to analyze the Rett syndrome indication, there is one drug DAYBUE on market,
BTW generating 60M in Q3, pricing is around $385,075 and $924,180 per year.
 
It was approved based on 13% clinically significant improvement in Clinical Global Impression-I (CGI-I) scores (scores 1 and 2) and 37.7% any kind improvement (scores 1-3), see Fig. 2 in the link below.
 
https://reverserett.org/news/articles/daybue-trofinetide-key-facts-for-parents/
 
 
From the results released on 2022 blarcamesine in 36 patients treated for only 7 weeks:
 
Additionally, a significantly greater proportion of patients on study drug (72.2%) achieved clinically meaningful response on Clinical Global Impression-I (CGI-I) scores throughout the trial relative to those on placebo (38.5%; P = .037; Cohen d = 1.91).
 
The very high placebo effect is probably due to the short duration, in DAYBUE trial there was similar effect (Fig 1 in the link above). This might be the reason why the protocol was extended to treatment for 12 weeks.
 
Now my question is if you know if "clinically meaningful response" in CGI-I is the same as "clinically significant improvement", i.e., did AVLX include scores 1 and 2 or 1, 2, and 3?
 
In any case the benefit for patients should be at least double to DAYBUE.
With known mechanistic path for improvement in the case of blarcamesine (unlike for DAYBUE) I think FDA should love it!
Anyone can see a flow in my logic?
 
Good luck,
Chvoja
 
 


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