Rett Syndrome Trial of Oral Anavex 2-73 to Continue with at-Home Visits

Anavex Life Sciences announced plans to continue to screen and enroll patients into its active trials for Rett syndrome and Alzheimer’s disease, in accordance to institutional rules and government guidance related to the COVID-19 pandemic.

A Phase 2 study of people with dementia linked to Parkinson’s disease is fully enrolled, and the company expects to announce topline results by June.

Contingency plans are in place for “remote or virtual assessments” of participants in the Alzheimer’s and Parkinson’s primary trials, it added, while the Rett trials allow at-home visits.

“We understand that some activities will be temporarily slower because of COVID-19, however, our risk-mitigation actions were implemented for every project,” Christopher U. Missling, PhD, president and CEO of Anavex, said in a press release.

“Anavex’s primary focus is on the safety of all involved and the continued conduct of our clinical programs as we navigate the pandemic together,” Missling added. “Our sites understand why their patients participate in research and are committed to the importance of clinical trials for those patient communities currently without effective treatments.”

All trials are testing Anavex 2-73, which was originally developed as a potential disease-modifying therapy for Alzheimer’s disease.

Anavex 2-73 is given orally to activate a cellular receptor called Sigma-1 (SIGMAR1), known to have neuroprotective effects. Specifically, activation of SIGMAR1 can help reduce neuroinflammation, as well as the accumulation of beta-amyloid and tau proteins and oxidative stress, all known to contribute to progression in neurodegenerative disorders.

Two placebo-controlled trials are underway in adults with Rett syndrome, ANAVEX2-73-RS-001 (NCT03758924) in the U.S., and AVATAR (NCT03941444) taking place at the Royal Melbourne Hospitals in Victoria, Australia. Both are recruiting eligible patients, ages 18 to 45.

Patients who complete these seven-week studies, which have always allowed at-home visits, will have the option of continuing — or starting — treatment in a 12-week open-label extension.

The Phase 2 trial ANAVEX2-73-PDD-001 (2017-004335-36) is testing Anavex 2-73, at two different doses against placebo, as a treatment for Parkinson’s dementia. This 14-week treatment trial, completely enrolled, is taking place at several clinical sites in Spain.

The Phase 2b/3 ANAVEX2-73-AD-004 (NCT03790709) study, taking place in Australia, is testing two doses of oral Anavex 2-73 (a middle and high dose) against a placebo for about one year in up to 450 people (ages 60 to 85) with mild cognitive impairment or early stage mild dementia due to Alzheimer’s disease.

People completing this trial are invited to continue treatment in its long-term extension study, (NCT04314934), that runs through December 2023 in Australia.

For these disease studies, the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and the Australian Therapeutic Goods Administration (TGA) recommended contingency plans to ensure that all active patients can be assessed remotely or virtually.

Anavex reaffirmed in its release its commitment to ensuring the safety of all trial participants and to its studies continuing to be conducted in line with scientific and ethical guidelines. It is working closely with stakeholders to mitigate the effects of the COVID-19 pandemic, with a goal of continuing its clinical program.