Anavex Life Sciences Corp (AVXL)

[F1ash]

Taken together, the changes in the measurement scales that correlate with the biomarker changes make strong argument for 2-73 even though it is a small N unblinded drug trial.

Kudos on an excellent post.

The correlation between the questionnaire and the biomarkers is exactly what one would hope to see. Certainly better than not seeing the correlation. Could both be from the placebo effect? Yes probably, but it’s a bit stronger evidence since both results concur.

There is something else positive that hasn’t gotten much mention.

The results came from a dose that is likely to avoid issues with toxicity.

“Preliminary Clinical Data is derived from the ANAVEX®2-73-RS-001 study on the first 6-patient cohort ranging in age from 18 to 36 years, who completed the pharmacokinetic (PK) part of the study and who received a low dose of approx. 5 mg daily oral liquid dose of ANAVEX®2-73 (blarcamesine) for 7 weeks. “

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/

Note that that dose is significantly different than a potential example that was given in the patent application.

“EXAMPLE 4
Eating and Choking
A 20 year-old female presents with Rett syndrome. She experiences feeding difficulties exhibiting as choking and poor food intake prior to commencing therapy. She begins treatment with oral doses of A2-73 at 40 mg every other day. After 60 days of treatment, the instances of choking are reduced and she exhibits a 10% weight gain. “

“Therapeutic doses of A2-73 are particularly noted in a range of from about 0.5 mg/day to about 100 mg/day with particular reference to doses of from about 1 to about 60 mg/day and, optionally, wherein said dosage is administered daily for at least about 10 days. Adult doses of about 20 mg to about 50 mg/day are noted, with particular reference to about 30 mg to about 40 mg/day. Infant and child doses are to the lower end of the range with particular reference to from about 0.5 mg/day to about 20 mg/day. Chronic administration for chronic conditions is further contemplated.”

https://patentscope.wipo.int/search/en/detail.jsf?docId=US236451262&tab=PCTDESCRIPTION

There is probably a reason that the Rett doses are less than initially planned and it’s good that the lower doses may be effective unlike the results in the Alzheimer’s trial seem to show.