Anavex Life Sciences Provides an Update on Rett Syndrome Program - Seite 2

When looking at other placebo-controlled Rett syndrome trials, ANAVEX2-73 compares favorably in terms of absolute RSBQ improvements, with the caveat that cross trials comparisons have their limitations.

The key secondary endpoint, the Anxiety, Depression, and Mood Scale (ADAMS), trended favorably. In the same analysis, scores for all RSBQ and ADAMS subscales improved over the course of the study. Collectively, the RSBQ and ADAMS demonstrated improvements in multiple areas, impacting positively in particular repetitive movements, nighttime disruptive behaviors and social avoidance.

In the EXCELLENCE study, a large placebo effect was observed which may have masked the compound’s therapeutic effect. Anavex believes to have identified the probable causes.

Walter E Kaufmann, MD, Chief Scientific Officer of Anavex commented, “We believe that a high placebo response may have masked the therapeutic effect of this innovative orally available molecule. High placebo responses are well documented especially in pediatric clinical studies. Although data analysis is ongoing, the early conclusion is that the placebo rate could have been higher in the study due to a slight imbalance in disease severity at baseline, across the treatment arms, and the 2 to 1 drug to placebo randomization ratio. We intend to further assess the collective results and discuss with the regulatory authorities next steps.”

A preliminary review of the safety results indicates there were no new safety signals in the EXCELLENCE study, reinforcing the favorable and manageable safety profile observed with ANAVEX2-73 to date. The most common treatment-related adverse events in the drug-treated group were somnolence and lethargy and were predominantly mild to moderate in severity. There were no clinically meaningful changes observed in SAEs associated with known risks of ANAVEX2-73.

Over 91% of patients completing the trial continued into a 48-week open-label extension study (OLE), which is ongoing. Upon patient’s caregivers and investigators request, Anavex has established Compassionate Use Programs in Canada, Australia, and the UK for pediatric patients upon completion of the OLE study, similarly to its existing program for adult patients with Rett syndrome. To date, of the pediatric patients who completed the OLE, 93% have joined the Compassionate Use Program. This rate is comparable to the Compassionate Use level seen for adult patients which is over 96%. As of today, some patients with Rett syndrome have been on ANAVEX2-73-treatment for over 4 years, combined OLE and Compassionate Use Program.