Anavex Life Sciences Corp (NASDAQ:AVXL) has announced that it has exceeded its ANAVEX®2-73 (blarcamesine) second Phase study in Rett syndrome enrolment target by 50%. The company is anticipating reporting topline data from the study in the second half of 2020.
ANAVEX®2-73 promotes neuroplasticity and restoration neural cell homeostasis
ANAVEX®2-73 is a small molecule sigma-1 receptor activator available orally according to data. It is vital in neural cell homeostatic restorations as well as the promotion of neuroplasticity. The double-blind, multi-center clinical study conducted in eight sites in the US evaluates the efficacy, safety, and tolerability of daily oral ANAVEX®2-73 doses relative to placebo. After finalizing the study, the subjects will be eligible to join a voluntary open-label ANAVEX®2-73 extension study.
The company has previously reported preliminary data from the PART A intensive PK subgroup in the first six subjects in the second phase US Rett syndrome trial. In the results, ANAVEX®2-73 showed considerable improvements in their two efficacy endpoints the Clinical Global Impression Improvement (CGI-I) and the Rett Syndrome Behaviour Questionnaire Total score (RSBQ). Similarly, ANAVEX®2-73 substantially enhanced RSBQ Hand Behaviours as well as the RSBQ Breathing Abnormalities. Also, there was a significant improvement in efficacy indications on caregivers and clinician-based processes of severity related to glutamate levels, which are the main biomarker in the disease’s pathogenesis.
ANAVEX®2-73 study among other Rett Syndrome Programs
The study is among the three currently ongoing clinical trials in the company’s Rett Syndrome Program. The programs include U.S. RTT (ANAVEX®2-73-RS-001)2, EXCELLENCE (ANAVEX®2-73-RS-003)4, and AVATAR (ANAVEX®2-73-RS-002)3. The FDA had previously granted the company’s ANAVEX®2-73 Fast Track Designation, Orphan Drug designation, and Rare Paediatric Disease Designation for Rett Syndrome treatment.
Anavex Chief Executive Officer and President Christopher Missling stated that since currently there is no approved therapy for the treatment of Rett Syndrome, there is, therefore, a huge unmet medical need for this indication. The CEO added that the company was delighted to complete enrolment of the prudently conducted ANAVEX®2-73 study in Rett Syndrome.