Replies to post #197478 on Anavex Life Sciences Corp (AVXL)

[Doc328]

If Avexis succeeds they may have something close to a cure for Rett and not just a treatment. They just got approval for Zolgensma, a one time 2.3 million dollar gene therapy that had remarkable results for infants with SMA type 1. Expensive but for a one-time treatment that cures a lifelong neurologic disease, worth every penny. The drug is a complex gene therapy using a modified adenovirus to deliver.

Similarly, AVXS-201 would probably only be used in studies at the first stage of Rett (age 1-2) when symptoms first emerge and diagnosis is made. I don't think the drug would have benefit by stage 3-4 when neurologic symptoms are mostly stable, so there may still ultimately be a place for band-aid drugs like A273

They intend to get an IND very soon and start phase 1. Given the nature and speed of the disease they will not need all the phases. From a PR:

Preclinical mouse studies using AVXS-201 led to the longest living Rett mouse to date with sustained symptom reduction.

Primate studies using AVXS-201 show no signs of adverse effects or over-expression of MECP2.

Production of clinical trial grade AVXS-201 using AveXis’ own scalable manufacturing platform is underway.