Anavex Life Sciences Corp (AVXL)

[falconer66a]

Final Rett Readout To Be Positive

Still on track for Q4 readout on the US phase 2 Rett trial, nice to see. ... How do you do it?

Good question. All of what we see from blarcamesine tested in girls with Rett syndrome (but only 6 of those, so far) is entirely positive. Significant suppression of glutamate (very positive, to suppress this hyperexcitability-inducing molecule) and significant elevation of gamma-aminobutyric acid (GABA). GABA suppresses nerve impulse excitability. Both of these quantified results are highly desirable for treating Rett (and other CNS diseases).

But an often overlooked matter was the safety of blarcamesine in these humans. Simply, no debilitating or disqualifying adverse events, side effects. Profoundly safe. Uncommon in drugs acting so powerfully in nerves and neurons.

Why won't these safety and efficacy results appear in the much larger, soon to be completed new Rett trial? The only thing different (as far as I can tell) is that these girls will have been treated for much longer periods of time; closely monitored for both safety and efficacy again.

The safety factor should continue. No indication in either humans or murines (lab rodents) that chronic (long-term) dosing with blarcamesine induces eventual side effects. No indication whatsoever of adverse accumulation of escalating side effects. Just the opposite; it appears that the body can learn to adapt to higher doses, obviating or reducing the only minor side effects of headaches or dizziness. (Most of those were at elevated doses.)

So, safety should continue.

But what about the really important outcome, does the drug continue to work, be effective? Many (I was one) were astonished that in the first trial such good positive results could appear in such a relatively short period of time. The new trial will have much longer treatment periods. If anything, that longer period should allow even better therapeutic outcomes. GABA levels may be even higher; glutamate levels even lower. Overall, even better, more normalized health for the Rett girls.