Alzheon has released one-year data from a phase 2 study of its Alzheimer’s disease candidate ALZ-801, linking the oral beta amyloid blocker to a reduction in a marker of neuronal stress and brain injury.
ALZ-801, also known as valiltramiprosate, is an inactive prodrug form of a drug candidate that failed a phase 3 clinical trial in Alzheimer’s 15 years ago. Alzheon struck a licensing deal for the molecule in 2013 on the strength of signs of efficacy in patients with two copies of the APOE4 gene, a mutation associated with a higher risk of Alzheimer’s and greater burden of beta amyloid.
After twice failing to pull off an IPO intended to support a pivotal program, Alzheon began a phase 2 trial of its drug candidate in September 2020. By then, the biotech had secured a $47 million grant from the National Institutes of Health to bankroll a pivotal phase 3 study.
Alzheon presented the latest data from the phase 2 study on Tuesday, revealing a 41% reduction from baseline in the neuronal stress and brain injury marker plasma p-tau181. Levels of the phosphorylated tau rise as Alzheimer’s progresses and patients deteriorate. Alzheon hailed the extent of the reduction in the biomarker as “industry leading.”
“The several-fold greater reduction on the p-tau181 biomarker in plasma compared to plaque-clearing anti-amyloid antibodies provides further support for the robust clinical benefits observed in Alzheon’s prior Alzheimer’s studies,” Alzheon CEO Martin Tolar, M.D., Ph.D., said in a statement.
The biotech also reported “robust preservation of brain volume” and “positive memory effects” without providing data in the statement to quantify the effects. According to Alzheon, participants’ performance on memory tests improved from baseline after three and six months of treatment. Performance was still above baseline after one year. The 84-subject phase 2 study lacks a control arm.
Alzheon could show whether ALZ-801 is better than placebo at improving cognition when it reports data from its phase 3 study, which has a primary completion date of June 2024 on ClinicalTrials.gov. The trial is enrolling 300 patients with early Alzheimer’s and two copies of the APOE4 gene.