Anavex Life Sciences Corp (AVXL)

[Investor2014]

A collection of info from previous Rett trial announcements...and more
This recent publication gives an idea why a drug targeting both S1R and M4 Muscarinic Receptor might be useful in Rett. We seem to be in for interesting times ahead.

Total RNA-sequencing of Rett Syndrome Autopsy Samples Identifies the M4 Muscarinic Receptor as a Novel Therapeutic Target

A survey of our RNA-seq results also identified a significant decrease in expression of the muscarinic acetylcholine receptor 4 (CHRM4) gene, which encodes a receptor (M4) that is the subject of multiple large drug discovery efforts for schizophrenia and Alzheimer’s disease. We confirmed that CHRM4 expression was decreased in RTT patients, and, excitingly, we demonstrated that M4 potentiation normalizes social and cognitive phenotypes in Mecp2+/- mice.

Furthermore, Anavex has recently reported that the sigma 1 / M1 agonist ANAVEX 2-73, which is currently undergoing phase 2 clinical trials for Alzheimer’s disease and received orphan drug status for RTT, also has a benefit in RTT model mice. These results may point to the global repression of muscarinic acetylcholine signaling in RTT, and, more broadly, a responsiveness of RTT model mice to positive modulation of this form of neurotransmission.

ANAVEX 2-73

Biological Activity
s1 agonist (IC50 = 860 nM); also displays affinity for muscarinic M1-M4 receptors (Ki values < 500 nM), but not for s2 receptors. Exhibits neuroprotective effects, prevents tau hyperphosphorylation, and attenuates scopolamine- and (+)-MK 801-induced learning deficits in a mouse model of amyloid toxicity.

This gives an idea of what might constitute efficacy endpoints and biomarkers.
Do you or someone you know have a child with Rett Syndrome?

Also supported by:


Anavex awarded grant to fund clinical trial of Rett syndrome drug

Anavex Life Sciences Corp. will receive funding for a phase 2 trial of its Anavex 2-73 drug from the International Rett Syndrome Foundation to study the rare neurological disorder, the company announced early Thursday.

The 12-week trial — which will cost about $1 million total and enroll between 50 and 80 patients

The full PR: Anavex Life Sciences Receives Grant From Rettsyndrome.org to Commence U.S. Phase 2 Trial in 2017

Now I think we can finally say Go Anavex! 2019 will be an interesting year for patients and long investors.