Too good to be true or just objective facts and corroborating data that leads to the inescapable conclusion: blarcamesine works (IMO). Especially in regards to Rett Syndrome as evidenced by the data on glutamate and gaba. I can tell you from firsthand knowledge that there is so much excitement within the Retts community about blarcamesine's potential.
As a reminder (copied and pasted from earlier AVXL PR):
plasma levels of the biomarker Glutamate also decreased significantly (Week 0 vs. Week 7; 2-tailed Wilcoxon signed rank test, p = 0.046) and levels of Glutamate at Week 7 were directly correlated with CGI-I scores at Week 7 (2-tailed Spearman’s rho = 0.837, p = 0.038) with greater decreases in Glutamate associated with greater improvement in these efficacy scores.
Glutamate is the main excitatory neurotransmitter in the brain and is known to be higher in patients with Rett syndrome compared to healthy subjects in the brain, as measured by magnetic resonance imaging spectroscopy (MRS), as well as in cerebrospinal fluid (CSF) and blood plasma.
Additionally, the magnitude of GABA change was inversely correlated with the magnitude of decrease in RSBQ Total scores (2-tailed Spearman’s rho = -0.812, p = 0.050) and GABA changes demonstrated an inverse correlation of the magnitude of Glutamate changes (2-tailed Spearman’s rho = -0.829, p = 0.042).
GABA is the main inhibitory neurotransmitter in the brain, known to be deficient in animal models of Rett syndrome. Excitatory-inhibitory imbalances postulated in many neurologic disorders, including Rett syndrome, have been linked to imbalances between Glutamate and GABA[1],[2].
An independent DSMB review determined that ANAVEX®2-73 (blarcamesine) was well tolerated, with no SAEs reported and with all patients completing the study. Therefore, the DSMB issued a positive recommendation for the continuation of the Phase 2 Rett syndrome study without any modifications.