The Trouble With AVATAR $AVXL Blarcamesine Rett Syndrome

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Evercore ISI December 2, 2021

• First order of the day, Plateau in Blarcamesine therapetic effect, 1:57′ into interview
• Blarcamsine therapetic effect proportionaly to SIGMAR1 mRNA level in blood
• Rising the dosing levels first meets with plateau then with protective mechanism (antagonist)
• We can dose high without adverse effects even in spite plateau
• The Glutomate/Gaba balance in Rett patients with 50% increase in Glutomate levels up vs. steady GABA.
• This finding is arrived to by comparision between siblings and patients.

I suggest reading two of my posts on Rett syndrome results.

Link 1 https://piotrpeterblog.com/2020/12/07/rett-syndrome-data-already-out-anybody-pays-attention/

Link 2 https://piotrpeterblog.com/2020/12/11/rett-syndrome-continued-blarcamesine-avxl/

Let’s see slide from the results.

Observe that by dossing 5mg/kg we have dropped levels of Glutomate by over 40%, leaving almost no room for further improvement. If level at which regaining of healthy levels in this biomarker is set at 50% drop in Glutomate levels per Dr. Missling remark. The conclusion which we may reach is that in Rett syndrome even with the dose of 5mg/kg we hit the plateau or close so in therapetic effect, as the biomarker Glutomate/GABA levels indicate. The only degree of freedom we have to increase the therapetic effect is to dose younger patients as their higher neuroplasticity than adult patients shall allow for greater therapetic effect to develop in their lives.

The question which is right now unanswered is whether Blarcamesine can take the full advantage of younger patients neuroplasticity or/and rather prevent the neurodevelopmental damage done by the mutation. If indeed we can treat 5 years olds with it why not those freshly diagnosed? In adults we restored the Glutomate/GABA balance but the damage has been done irrevocably. Would we dose the 18 months old and prevent the devastation of the disease?

It seems for now that is almost all we can do for the adult sufferes of the devastating disease. Do not expect AVATAR to drastically change this fact.