In honesty, I do not know enough organic chemistry to detail the minute (but important) structural differences between the Anavex 2-73 and Anavex 3-71 (previously named AF710B) molecules.
But those arcane structural chemical differences are irrelevant to any Anavex investor. Important only to the organic chemists that will be synthesizing the molecules and the neurochemists who will be conducting detailed cellular therapies with them. That’s not us.
(Note below the two words “potentially” were underlined by Anavex. This is in delicate conformance to SEC rules, where a company cannot make incompletely-substantiated or promotional statements about their products. For some (the SEC, many financial “advisors”) the profound efficacies of either Anavex 2-73 and Anavex 3-71 are as of yet not sufficiently substantiated; hence, the feeble adverb “potentially.”)
Regardless of SEC rules, both of these molecules have profound scientific substantiation; first in animal (rats and mice) models of the central nervous system diseases Anavex is targeting, and now with the early Anavex 2-73 human clinical trials in Australia, profound human efficacies are known.
Those who for either investment tepidness or biochemical ignorance wish to wait for more substantial clinical results are welcome to do so. As a biologist I’ve scrutinized dozens of pages of technical papers on the Anavex pipeline drugs, and I have no doubt that a) FDA approval of Anavex 2-73 will be forthcoming for both Rett Syndrome and Alzheimer’s (with others to follow), and b) Anavex 3-71 presents efficacies for Parkinson’s Disease. Clinical trials will reveal those in humans, when conducted.
For us retail AVXL investors, here’s what we really need to know (copied from the Pipelines page of the Anavex website).
Anavex’s summary of Anavex 2-73:
ANAVEX 2-73
ANAVEX™ 2-73 is an orally available drug candidate developed to potentially modify Alzheimer’s disease rather than temporarily address its symptoms. It has a clean Phase 1 data profile and shows reversal of memory loss (anti-amnesic properties) and neuroprotection in several models of Alzheimer’s disease.
Successful Phase 1 Clinical Trial
A Phase 1 single ascending dose human clinical trial of ANAVEX 2-73 was successfully completed in healthy human volunteers. It was a randomized, placebo-controlled study. Healthy male volunteers aged 18 to 55 received single, ascending oral doses over the course of the trial. The trial objectives were to define the maximum tolerated dose, assess pharmacokinetics (PK), clinical and lab safety.
Results: – Dosing from 1-60 mg. – Maximum tolerated dose 55-60 mg; above the equivalent dose shown to have positive effects in mouse models of Alzheimer’s disease. – Well tolerated below the 55-60 mg dose with only mild adverse events in some volunteers. – Observed adverse events at doses above the maximum tolerated single dose included headache and dizziness, which were moderate in severity and reversible. These side effects are often seen with drugs that target central nervous system (CNS) conditions, including Alzheimer’s disease. – No significant changes in blood safety measurements. – No changes in ECG. – Favorable PK profile. – Rapid absorption into blood. – Dose proportional kinetics.
*********** Anavex’s Summary of ANAVEX 3-71
ANAVEX 3-71, previously named AF710B is a preclinical drug candidate with a novel mechanism of action via sigma-1 receptor activation and M1 muscarinic allosteric modulation, which has shown to enhance neuroprotection and cognition in Alzheimer’s disease. ANAVEX 3-71 is a CNS-penetrable mono-therapy that bridges treatment of both cognitive impairments with disease modifications. It is highly effective in very small doses against the major Alzheimer’s hallmarks in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid and tau pathologies, and also has beneficial effects on inflammation and mitochondrial dysfunctions. ANAVEX 3-71 indicates extensive therapeutic advantages in Alzheimer’s and potentially other protein-aggregation-related diseases given its ability to enhance neuroprotection and cognition via sigma-1 receptor activation and M1 muscarinic allosteric modulation.