Comparing Trofinetide and Anavex2-73. This Installment We Try to Estimate RSBQ Score of AVATAR RS-002 From Hearsay and Formulas for Cohen’s d for RSBQ-ACU. All for Rett Syndrome Winning Drug. $AVXL $ACAD

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The Way of the Engineer….

In engineering if you can not find an analytical solution you asymptotically move toward solution through iterative calculations making your error smaller with every cycle and making assumptions. I once talked with Phd (mathematics) guy and he derisively asserted that if in engineering you are within 20% of solution you are OK.

I don’t think I nailed it but this is very interesting so let’s dive in. I might be within the twenty percent.


Link to Presentation AVATAR RESULTS

The Hearsay Goes…


  1. Dr Missling said that the RSBQ scores ranged from 10 to 29 of improvement among the ~72% how were classified as Responders in Dosed Arm
  2. Dr Missling said that CGI-I score 3 meant that patient reached 12.5 point improvement on RSBQ score.

My Assumptions

  • I can create spreadsheet where I list hypotethical RSBQ scores based on 2 statement from Dr Missling suggesting that those who passed CGI-I gatekeepers were at least 12.5 point down (improvement), otherwise thay were assumed to be zero.
  • Assuming that time factor is 5 weeks for the 7 weeks duration and that I will take 70% of so calculated Standard Deviations into the formula for RSBQ-ACU Cohen’s d.
  • I can plug in RSBQ scores into the Dosed cohort sample and watch my calculations for RSBQ-ACU Cohen’s d update till they reach near or converge on 1.91 as was calculated by the company.
  • I assume that Placebo Arm is composed of 4 patients who are -12.5 RSBQ and the rest zero.
  • I assume that Dosed Arm is composed of 14 patients with RSBQ scores of below -12.5 from baseline, and 6 who are zero.
  • I assume the RSBQ scores are in the range of -12.5 to -30.

Let’s See The Calculations…

  • Yellow filled cell contains value of 1.88 which for my purposes is close enough to 1.91 calculated by the company.
  • Row 8 contains possible values of patients improvements of about -30 RSBQ points from baseline. Marked green.
  • The MEAN of RSBQ scores is -19 (tales are in positive values) RSBQ
  • The results are in line with what Dr Missling have been saying in answers to analysts’s questions without giving them a real number.
  • I don’t think that this analysis is 100% correct but it points to the possibility of very high improvements scores attainable with ANAVEX2-73 (Blarcamesine)
  • The comparison between Trofinetide and Anavex2-73 is respectively -5.1 to -19. We have almost 4 times larger response with Anavex2-73 than with Trofinetide. It doesn’t matter whether you convert Trofinetide RSBQ scores into RSBQ-AUC or try to arrive at an estimate of RSBQ scores for Anavex2-73, whichever way Trofinetide is grossly inferior to Anavex2-73.

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