Following a proof-of-concept and extended trial, Anavex Life Sciences’ investigational drug Anavex 2-73 shows promise as a therapy for cognitive declines associated with Parkinson’s disease dementia. Anavex is a biopharmaceutical company developing small molecule treatments for debilitating central nervous system disorders like Alzheimer’s disease, Rett syndrome, and Parkinson’s.
Parkinson’s disease is a complex and progressive CNS disorder that greatly impacts the motor system, causing tremors, movement difficulties, and memory loss. In the United States, nearly 1 million people are living with Parkinson’s, with up to 70% of those with the disease developing dementia as symptoms progress.
Anavex 2-73 (blarcamesine) is currently making its way through clinical trials as a viable treatment for Alzheimer’s-related cognitive decline and Parkinson’s-related dementia.
Anavex 2-73 Is Designed To Activate a Key Protein in the Brain
Anavex 2-73 is an orally available small molecule activator of the sigma-1 protein that is key to maintaining neuroplasticity — a process essential to creation of neurons and formation of memories and learned experiences. Because CNS disorders often reduce the levels of sigma-1 found in the brain, activating the protein can preserve healthy brain functioning and reduce levels of oxidative stress and cognitive decline for those with Alzheimer’s and Parkinson’s.
In the proof-of-concept Phase 2 Anavex 2-73 trial, the drug was double-blind tested against a placebo in 132 adults diagnosed with Parkinson’s disease dementia. This trial lasted 14 weeks. Participants who took a daily dose of Anavex 2-73 showed improvements in memory and cognitive skills such as choice reaction time and vigilance.
Anavex 2-73 also improved patients’ REM sleep and had a safety profile greater than other common dementia treatments. Taken together, these findings warrant an extended study that concluded in 2023.
“Our strategy has been consistently to advance Anavex 2-73 (blarcamesine) with focus on precision medicine and to validate this approach in clinical studies in patients with significant cognitive impairments,” Christopher Missling, Ph.D., Anavex president and CEO, said in a press release. “We are pleased with these study results that will be further supplemented by actigraphy movement data and whole genome exome DNA and RNA data. Finally, we would like to thank all the patients and participating families, as well the investigators and clinical site coordinators, for their dedication to this study.”[4]
The Anavex 2-73 Extension Trial Further Validated the Drug’s Efficacy
Following Anavex’s proof-of-concept trial, participants were given the option to enroll in an open-label, 48-week extended trial to further validate the drug’s safety, tolerability, and efficacy. This study measured a variety of endpoints, including improvements to the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) parts I, II, and III, and Clinical Global Impression – Improvement (CGI-I). Both test the severity of Parkinson’s symptoms.
Given an extended delay to the trial’s start due to the COVID-19 pandemic, the study provided significant data on the effects of an Anavex 2-73 “drug holiday” — a term used to describe a lengthy gap in treatment. During an average 41-week gap in Anavex 2-73 treatment, all tested endpoints worsened compared to their initial trial results.
Total MDS-UPDRS scores, which trended the worst during this drug holiday, showed improvements throughout the 48-week extended trial. By week 48, the median score for the 20 patients who completed the extension was similar to that of the previous trial before the drug holiday. Through the compassionate use program[5], those who took part in the extended study will have the option to continue using the medication.
“It is encouraging that the patients’ clinical symptoms consistently improved longitudinally over time during the extension phase under active Anavex 2-73 treatment,” said Anavex President & CEO Christopher Missling, PhD. “This data suggests Anavex 2-73’s potential capability to slow and potentially reverse the life-altering symptoms of Parkinson’s disease, an urgent unmet global need.”
Anavex 2-73 Will Now Go Through a Phase 3 Trial
With the promising results of the first two trials, Anavex’s Phase 3 Parkinson’s trial will commence subsequently. The trial will allow researchers to further evaluate MDS-UPDRS scores and Clinical Global Impression improvement for patients on Anavex 2-73 over a six-month period.
The company has taken a deliberate break between its Phase 2 and subsequent Phase 3 trial. With a disease as complex as Parkinson’s, that time allows for meticulous analysis of all results and the potential introduction of new, useful biomarkers.
“The Anavex 2-73 (blarcamesine) study results represent a meaningful step forward toward urgently needed treatment for this serious complication of Parkinson’s disease,” said Dag Aarsland, professor and the head of the Department of Old Age Psychiatry at King’s College London. He added that this is especially important “given that cognitive impairment of patients with Parkinson’s disease dementia is very distressing to patients and their families and is associated with greater risk of institutionalization and accelerated progression to severe dementia and death.”
More about Anavex: https://www.nasdaq.com/market-activity/stocks/avxl
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