A Look at This Tiny Massachusetts Biotech That Could Take Down Alzheimer's

October 11, 2016 (Last Updated: 10:10am PT)
By Alex Keown, BioSpace.com Breaking News Staff

Framingham, Mass. – Finding a cure or long-term treatment for Alzheimer’s disease is something that many companies, such as Biogen , Genentech , Eli Lilly and others have taken aim at, but have yet to deliver. Joining those pharma giants is tiny Alzheon, Inc., which believes its experimental drug may be able to modify and underlying cause of the dreaded form of dementia.

In July, Alzheon moved its oral tramiprosate-based drug candidate, ALZ-801, into a Phase III trial. Tramiprosate was originally developed by Bellus Health as an amyloid-plaque targeting drug to treat Alzheimer’s, but like so many other drugs before it, this one failed. However, Alzheon saw potential in the drug, particularly if it was aimed at one specific target population—patients who are homozygous for the e4 allele of the apolipoprotein E gene.

There is a widespread belief that the buildup of amyloid plaque in the brain is one of the primary causes of the disease. Alzheon said it uses a precision medicine approach to improve the critical pharmaceutical properties of the active molecule tramiprosate—tolerability, pharmacokinetic and pharmacodynamic profile, metabolic stability—to make its drug candidate better suited as a treatment for patients with Alzheimer’s.

In July, Alzheon announced it was moving ALZ-801 into Phase III trials in 2017 after two successful Phase Ib studies. Martin Tolar, chief executive officer of Alzheon, said in July that the company has “all of the necessary clinical, toxicology and tablet data to start the pivotal Phase III program with ALZ-801.” The company said the APOE4 subgroup analyses of the tramiprosate Phase II studies showed “clinically meaningful benefits” on cognitive and functional endpoints in Mild and Moderate AD patients who are APOE4/4 homozygotes.

In its Phase Ib trials, ALZ-801 showed an equivalent or improved PK profile compared to tramiprosate, including plasma exposures and dose proportionality, thereby allowing bioequivalence and bridging to the prior clinical data with tramiprosate.

It’s been a tough road for Alzheimer’s treatment hopes. Last month, Danish company H. Lundbeck A/S felt the sting of failure. The company said its experimental Alzheimer’s treatment, idalopirdine failed in a late stage trial. The company said both doses being studied in the Phase III trial showed an “a weak efficacy profile” and failed to meet primary and secondary endpoints.

Although amyloid plaque is the target for many companies when it comes to Alzheimer’s, New York-based Anavex is focused on oxidation and nitration in the brain as the principle cause. The company’s lead product is Anavex 2-73, an oral treatment being developed to “modify” Alzheimer’s disease, rather than treat it. The drug candidate “inhibits oxidation, nitration and cell death by inhibiting the cascade which follows NMDA receptor over-activation.”

Alzheimer's disease, a type of dementia, affects 15 million people worldwide, a number that is expected to grow to 75 million by 2030 due, in part, to the lack of effective treatments. In total, there are about 50 million people suffering from some form of dementia worldwide. There are currently no drugs that target the cause of Alzheimer’s the most common form of dementia. There are several drugs on the market that help manage Alzheimer’s, but none treat the primary cause, including Eisai Co. ’s Aricept.