Anavex Life Sciences Corp (AVXL)

[Steady_T]

Corrected version of this post.

I did some research to try to understand what that improvement on the RSBQ represents. I was trying to put that number in context.

Here's what I found and it blew me away.

RSBQ is scored in 8 sub areas. Each of those sub areas is added to generate a total score with a maximum of 90 points.

So with that in mind, the response of the 6 subjects to 2-73 was an average reduction of 16 points from 50 to 34 on a 90 point scale. 90 points represents the worst symptoms of the disease. lower number represent improvement by reduced symptoms.

THAT IS HUGE. Unheard of in the Rett community.

Remember this is on a 5 mg oral dose. Maybe there could be more improvement with a larger dose. Maybe not. If there is room for increased dose related improvement, this is even bigger than it first appears.

As word of this small trial spreads as Falconer suggested it will, recruitment goals for the larger trial should be filled quickly.

As I suggested in an earlier post, the very young girls that are still developing might develop in a more normal manner and avoid some of the crippling effects of Rett all together. That is pure speculation on my part but given that Rett syndrome interferes with normal brain development it seems possible.

https://www.anavex.com/anavex-life-sciences-announces-preliminary-clinical-efficacy-data-of-its-u-s-phase-2-clinical-trial-of-anavex2-73-in-patients-with-rett-syndrome/

Quote:
Both efficacy endpoints, the Rett Syndrome Behaviour Questionnaire (RSBQ) and the Clinical Global Impression – Improvement (CGI-I) showed significant improvement with respect to baseline after 7 weeks of treatment. The RSBQ Total average scores improved from 50 to 34 points (2-tailed Wilcoxon signed rank test, p = 0.027) and the CGI-I scores were positively correlated with RSBQ Total scores at 7 weeks (2-tailed Spearman’s rho = 0.956, p = 0.003).


https://n.neurology.org/content/92/16/e1912

Quote:
The Rett Syndrome Behaviour Questionnaire (RSBQ) is a validated caregiver-completed rating scale assessing a wide range of neurobehavioral symptoms known to be impaired in RTT.27,–,31 The RSBQ has been correlated with functioning and quality of life and characterized across a range of ages and genetic variations in RTT.13,14,30,32 The scale includes 45 items rated on a Likert scale from 0 to 2, 39 of them grouped into 8 subscales (General Mood, Breathing Problems, Hand Behaviors, Repetitive Face Movements, Body Rocking and Expressionless Face, Night-time Behaviors, Fear/Anxiety, and Walking/Standing) whose ratings reflect severity and frequency of symptoms. A total, representing the sum of the 45 items (maximum score 90), and 8 subscale scores are obtained.