Response from RTT (Right to Try)...
Sent a msg to the RTT org a couple days ago and received the below reply. Makes me think they haven't heard of Anavex prior. Now they know!
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Hi,
Thank you for contacting us. I work for the Goldwater Institute, the organization that developed the idea for Right to Try.
I appreciate you sharing this research with us and I’ll certainly keep an eye on these treatment options as they move through the trial process. Would you like me to add you to our distribution list for any updates regarding the efforts with Right to Try?
Regards,
Austin Anton
_________________________________________
Austin Anton
National Policy Manager
Goldwater Institute | www.GoldwaterInstitute.org | 602.462.5000
“The Goldwater Institute is simply in the liberty business, and there’s no institution in the country that performs that business better.” – George Will
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From: basparks79
Sent: Sunday, February 18, 2018 10:34 AM
To: Right To Try <righttotry@goldwaterinstitute.org>
Subject: Anavex Life Sciences (AVXL)
Just recently discovered your wonderful organization via a well-written article by Patrick Cox.
A company that I (and many others) have been following is Anavex. Its initial drug, A2-73, should be a prime candidate for RTT. It has been proven safe and has demonstrated cellular homeostasis restoration, as an effective Sigma-1 and Muscarinic Receptor agonist. Most (if not all) CNS diseases begin with cellular stress and neuroinflammation, which ultimately lead to mitochondrial dysfunction.
In its Alz Ph2a trial, A2-73 has produced patients demonstrating stabilization and even improvement from baseline after 109 weeks of treatment. Those patients were in a subgroup having higher serum levels of the drug, which mostly associate with the higher dosage (50mg). The company contracted Ariana to perform detailed PK/PD analysis to better understand the biometrics of the “super responder” subgroup, and that information is now available. Genomic sequencing (Illumina) is also playing a role from a precision-medicine perspective. Additionally, unexpected therapeutic benefits were observed during the trial. Most all patients were reported to experience less agitation, less depression and less insomnia during the treatment period. All patients and caregivers have requested to remain on the drug.
Three trials will begin this year: Rett Syndrome (ODD) Phase-2, Parkinson’s Phase-2 and Alzheimer’s Phase-2/3. The Rett trial, funded by the Rett Syndrome Org, will begin as soon as the FDA completes its review of the modified IND. The Parkinson’s trial will likely begin in Q2, in Sweden, in association with Lund University. And the larger Alzheimer’s trial (200-300 patients) is expected to begin sometime midyear in Australia and presumably in the US via the recently established ACTC (Alzheimer’s Clinical Trial Consortium).
The company believes that, in low doses (i.e. 14mg), A2-73 can be a prophylactic (preventative) for a host of neurodegenerative diseases: Alz, PD, MS, ALS, etc.
With the proven safety and initial efficacy results to date, A2-73 is no doubt a prime candidate for RTT. Many thoroughly believe, that sometime this year, A2-73 will receive Accelerated Approval designation by the FDA.
The time has come for the FDA to evolve itself from the traditional bureaucracy to a more progressive review agency for these technological advancements. Only then might we be able to save our healthcare system and the lives of millions of afflicted individuals.
Sincerely,
