FDA Fast-Tracks Possible Alzheimer's Modifier

OCTOBER 25, 2017
Kevin Kunzmann
alzheimer's disease, ALZ-801, FDAThe US Food and Drug Administration (FDA) has given Fast Track Designation to an Alzheimer’s disease (AD) clinical drug that targets an underlying — and often untreated — pathology of the disease.

Biopharmaceutical company Alzheon Inc.’s lead clinical investigational drug ALZ-801, a novel, oral anti-amyloid optimized prodrug of tramiprosate, was green-lit for FDA fast track after a series of promising clinical developments. The investigative drug was discovered to have a novel mechanism of blocking the formation of toxic amyloid oligomers — a noted agent of AD development and progression.

Thus far, clinical data of ALZ-801 has shown long-term clinical efficacy in AD patients homozygous for the E-4 allele of apolipoprotein E (APOE4/4) genotype. These genetically-defined, high-risk patients will be targeted in the fast-tracked phase 3 study.

APOE, a gene located in the brain that helps transfer cholesterol to neurons and support their function, separated into 3 alleles — E-2, E-3, and E-4. The E-4 allele has been indicated in previous research to correlate with higher and earlier risk of onset AD. About 65% of US patients with AD carry at least one E-4 allele, and about 560,000 of those patients are APOE4/4 homozygotes. These patients' rates of cognitive decline and dementia stages of AD appear faster than the rest of the patient population.

Martin Tolar, MD, PhD, founder, president and chief executive officer of Alzheon said the company has built a strong body of clinical research and analysis that supports the treatment of ALZ-801 in AD subpopulation group.

Such clinical backing enables the company to “pioneer a Precision Medicine approach in Alzheimer’s, and to move toward an upcoming pivotal study and potential approval as quickly as possible,” Tolar said.

Following the phase 3 trial, Alzheon intends to expand ALZ-801 trials into additional AD patient populations, as part of its “Precision Medicine” approach. With the approach, the company evaluates the clinical drug based on genetic markers and disease stages.

This is adverse to the state of developed AD drugs, which Gary Small, MD, director of Geriatric Psychiatry at UCLA’s Brain Research Institute, said have been historically developed for symptomatic response. Speaking at the Pri-Med West 2017 conference earlier this year, Small advocated for the pursuit of disease-modifying drugs in mainstream AD research.

As there are currently no FDA approved drugs that target the underlying pathology, as well as slow AD’s progressive cognitive and functional decline, ALZ-801 stands to become a possible breakthrough. Its Fast Track Designation only supplants the potential.

“We look forward to working closely with the FDA, as we seek to make progress in developing a disease modifying treatment for Alzheimer’s disease to address the needs of Alzheimer’s patients and their families who struggle with this devastating disease,” Tolar said.

A press release regarding the designation was made available.

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