Patients in Australian Trials of Anavex 2-73 May Continue Using Therapy, Agency Says

People in Australia with Alzheimer’s disease who have finished five years of treatment with Anavex 2-73 (blarcamesine) in Phase 2 clinical trials may continue to use Anavex Life Sciences‘ investigational oral therapy at their doctor’s request.

Under a special access scheme, the Therapeutic Goods Administration (TGA) — a part of the Australian Government Department of Health — allowed for compassionate use based on safety findings and early evidence of benefit in these clinical studies.

A larger Phase 3 trial in early Alzheimer’s patients is now underway and enrolling in Australia and parts of Europe. Canada is expected to open sites as well.

Normally, therapies have to undergo thorough evaluations for efficacy, quality, and safety before they are made available. However, physicians may request to use medications that aren’t approved but showing benefit to patients.

“We are honored to support medical professionals and their patients seeking treatment for Alzheimer’s disease,” Christopher Missling, PhD, the president and CEO of Anavex, said in a press release.

Anavex 2-73 is an investigational oral therapy that is believed to reduce inflammation, lessen cellular stress, and prevent the accumulation of toxic proteins in the brain. It works by binding to sigma-1, a receptor protein present in neurons.

The Phase 2a clinical trial ANAVEX 2-73-002 (NCT02244541), which ran for 57 weeks in Australia, aimed to find the optimum dose for Anavex 2-73 in about 32 people with Alzheimer’s. Those who completed this trial could enroll in its 208-week open-label extension study, ANAVEX 2-73-003 (NCT02756858), which is set to conclude in November.

Results have been positive, suggesting that the investigational therapy is generally safe and well-tolerated. Although the trial was not designed with the primary intent of assessing efficacy, preliminary data indicated that the medication may slow cognitive decline. These findings were the basis for the TGA opening its access scheme to Anavex 2-73.

“The Phase 2a clinical ANAVEX 2-73-002/-003 studies are exploring the long-term effect of daily treatment with Anavex 2-73 (blarcamesine) over 5 years, however it is pleasing that physicians have requested extended treatment of their patients with Anavex 2-73 (blarcamesine) beyond these 5 years,” Missling said.

Anavex 2-73 is also being tested in a larger and placebo-controlled Phase 2b/3 clinical trial, called ANAVEX 2-73-AD-004 (NCT03790709). This trial’s primary goal is to determine treatment effectiveness in aiding cognition and daily life activities in up to 450 adults, ages 60 to 85, with mild cognitive impairment or mild dementia due to Alzheimer’s.

The ANAVEX 2-73-AD-004 trial is currently recruiting in Australia and the Netherlands, and Anavex recently received approvals to begin enrolling patients in Canada and the U.K. Additional trial and contact information can be found here.