Anavex completes dosing in trial of Anavex 2-73 for Rett

EXCELLENCE study testing safety of oral therapy in girls ages 5-17

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Anavex Life Sciences has completed dosing all 92 girls with Rett syndrome taking part in a clinical trial testing the safety of its oral treatment candidate Anavex 2-73 (blarcamesine).

Top-line data from this Phase 2/3 study of Anavex 2-73 — which aims to ease the symptoms of the rare neurodevelopmental disorder — are expected in the second half of the year.

“We would like to thank the investigators and clinical site coordinators as well as all the participating families for their dedication to this clinical study,” Christopher U. Missling, PhD, president and CEO of Anavex, said in a company press release.

Noting the disorder affects “nearly every aspect of the child’s life,” Missling said the company is set to “continue our fast-paced development program of Anavex 2-73 (blarcamesine) in Rett syndrome.”

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Rett syndrome causes changes to how the brain develops in early childhood, resulting in severe mental and physical problems. It mainly affects girls, and only very rarely occurs in boys. Symptoms differ from one child to another, and usually get worse over the years.

A small molecule, Anavex 2-73 is designed to work by turning on, or activating, a receptor protein in the brain called sigma-1 receptor, or SIGMAR1. By activating SIGMAR1, it’s expected that Anavex 2-73 will help restore balance in how brain cells work. This should contribute to neuroplasticity, the brain’s ability to rewire and adapt.

The ongoing study, dubbed EXCELLENCE (NCT04304482), is testing how safe and well tolerated Anavex 2-73 is versus a placebo in girls with Rett syndrome who have lost spoken language or motor skills. The trial also is evaluating the therapy’s efficacy, or how well it works among the 92 total participants, who range in age from 5 to 17.

The girls were randomly assigned to receive either an oral solution of Anavex 2-73 or the placebo, and neither they nor the researchers knew which treatment each was given. After 12 weeks, or about three months, when the treatment period ends, the girls will be offered the possibility to enter a 48-week open-label extension study that will assess the therapy’s effects for about one year.

Besides side effects, the researchers will be looking at changes in the Rett Syndrome Behavior Questionnaire (RBSQ) score, which assesses behavioral challenges, and the Clinical Global Impression Improvement Scale (CGI-I) score, which measures disease severity, as primary outcome measures.

Before launching EXCELLENCE, Avanex already had completed two other studies of the therapy — in adult women with Rett. These were a Phase 2 study (NCT03758924) known as ANAVEX2-73-RS-001, and a Phase 3 trial called AVATAR (NCT03941444).

Both studies showed that Anavex 2-73 was safe and well tolerated in women with Rett, and worked better than a placebo in easing their behavioral challenges. The results also showed treatment reduced disease severity and improved quality of life.

Earlier this month, Anavex was granted a new U.S. patent to add to its previous ones for the therapy. Issued by the U.S. Patent and Trademark Office, patent number 11,661,405 covers the use of crystal forms of Anavex 2-73, their composition, and methods of preparation.

This new patent relating to Anavex 2-73 is exceptionally important for Anavex, because all clinical trials with Anavex 2-73 (blarcamesine) … are using the patent protected crystal forms as the active pharmaceutical ingredient.

The patent, which expands the list of covered crystal forms and their use in a range of diseases, is expected to remain in force at least until October 2036.

“This new patent relating to Anavex 2-73 is exceptionally important for Anavex, because all clinical trials with Anavex 2-73 (blarcamesine) … are using the patent protected crystal forms as the active pharmaceutical ingredient,” Missling said in another press release, adding, “We are extremely pleased with the continued development of the patent portfolio for ANAVEX 2-73 (blarcamesine) which demonstrates our continued commitment to protecting all aspects of the Anavex product portfolio.”