2020-08-22| R&D

Alzheon’s Bags $47 Million from NIA for Oral Alzheimer’s Drug

by Ruchi Jhonsa
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The Framingham, MA-based small biotech has received funding to pursue ALZ-801 as a treatment AD patients who are positive for APOE4 protein.

By Ruchi Jhonsa, Ph.D.

Neurodegenerative diseases pose the greatest therapeutic challenge of our time. An estimated 60 million people are affected by the disease worldwide and are expected to double every 20 years as the baby-boomers age. Yet, other than drugs that slow down the progression of the disease for a brief time, there are no drugs that can completely stop or reverse this debilitating disease.

Many biotech companies are trying their luck to come up with a treatment that can cure the degenerative brain disorders. However, greater than 99% of the drugs that have entered the clinical trials have failed. Nevertheless, not all hope is lost. As the disease biology is becoming clearer, more companies are targeting the affected proteins to treat AD.

The dominant “amyloid hypothesis” in AD research holds amyloid deposition as the principal etiologic factor. Presently, two drug candidates are working on treating amyloid plaques, Biogen’s aducanumab, and Lilly’s solanezumab. Both the drugs promote clearance of amyloid deposits in the brain. While these drug candidates are showing promise in the clinical trials, they also pose the risk of vasogenic edema, which can limit their use or effectiveness.

Competing with the big players is Alzheon, a small biotech company in Framingham, Massachusetts that is working with ALZ-801, a prodrug of an old compound tramiprosate. While tramiprosate failed to meet the primary outcome in the Phase3 AD trial, Alzheon believes that the drug showed signs of efficacy in AD patients who were positive for APOE4 protein. Showing faith in the molecule, the US National Institute of Aging, a part of NIH, awarded a $47 million grant to the company over five years to conduct Phase 3 trial of their lead oral AD candidate ALZ-801 in APOE4 positive Alzheimer’s patients.

“We are grateful to the NIA for their rigorous scientific review and funding support to advance ALZ-801 for Alzheimer’s patients in need of an effective treatment,” said Martin Tolar, MD, Ph.D., Founder, President and Chief Executive Officer of Alzheon. “Proposals to NIA are highly competitive, and this award recognizes the contribution of Alzheon scientists to the targeting of toxic beta-amyloid oligomers, which are now established as the key drivers of cognitive decline in Alzheimer’s patients. We are proud that Alzheon has stayed the course and, after seven years of pathbreaking efforts, emerged as a frontrunner in the development of the first oral treatment for Alzheimer’s that can slow or stop the progression of the disease.”



In its singular form, amyloid protein is essential for maintaining brain health. However, when it starts aggregating and forming chains with other monomers, it forms bad soluble oligomers, which are toxic to brain cells. This is one of the triggers for Alzheimer’s disease. While Biogen’s and Lilly’s drugs work by clearing these aggregates, Alzheon’s ALZ-801 works by inhibiting the aggregation of monomers into oligomers.

ALZ-801 has been previously studied in large, multinational Phase3 study in 2000 patients with mild to moderate AD. Although the study did not show any significant outcome in general AD patients, it showed promising efficacy in a subgroup of patients that were positive for APOE4/4 protein. Moreover, the drug could preserve hippocampal volume on brain magnetic resonance imaging scans compared with placebo and showed favorable safety and tolerability.

At present, the drug has some advantages over Lilly and Biogen’s potential AD med. First, it is an oral drug. So, unlike the other two that need to be injected and require physician’s help, Alzheon’s drug can be taken at home. But, more importantly, the drug showed no cases of vasogenic edema, a side effect of the beta-amyloid antibodies developed by Biogen and Lilly.


NIA Sponsored Phase 3 Study

With the NIA grant, Alzheon will conduct phase 3 study in 300 patients with early AD and the APOE4/4 genotype. All the patients will be randomized to receive 265mg tablets of ALZ-801 or a matching placebo twice daily for 18 months. The study will investigate the drug’s effect on Alzheimer’s Disease Assessment Scale-cognitive subscale as well as on other functional, behavioral, and global clinical endpoints. The clinical trial will also determine changes in biomarkers including plasma and cerebrospinal fluid levels of amyloid and phosphorylated tau protein and other neurodegeneration and neuroinflammatory markers along with changes in hippocampal volume and cortical thickness

“We have designed this study to have a higher probability of success than previous trials in Alzheimer’s patients. We are applying for precision medicine by targeting patients who can be readily diagnosed, who develop the disease earlier when they are less likely to have comorbidities, and who are likely to benefit from the treatment,” said Susan Abushakra, MD, Alzheon’s Chief Medical Officer and principal investigator for the NIA grant. “We are integrating advanced fluid biomarkers into Phase 3 program to increase our understanding of the effects of ALZ-801 treatment on the course of the disease. While initially, we will focus on Early Alzheimer’s patients with the APOE4/4 genotype, future expansions will include investigations of ALZ-801 in prevention trials in healthy individuals with high risk for Alzheimer’s, and in APOE4 heterozygotes, who carry one copy of the APOE4 gene.”

Related Article: UCB Joins Forces with Roche to Develop Antibody Treatment for Alzheimer’s






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