AUG 23, 2016

Investigational Alzheimer Drug Wins Fast-Track Status

WRITTEN BY: Xuan Pham

For the 44 million people worldwide who are affected by Alzheimer’s disease, and the countless more who are at risk, a treatment drug is one step closer to being approved. The Food and Drug Administration recently granted a Fast-Track Designation to a compound made by partners AstraZeneca and Eli Lilly. This means the first drug to stop the progression of Alzheimer is very close within reach.

The compound, named AZD3293, is an oral beta secretase cleaving enzyme (BACE) inhibitor. It works by preventing one of the main cuts in the amyloid precursor protein (APP). When APP is cut, it is prone to forming the β–amyloid clusters, which are the distinguishing neurological hallmark of Alzheimer’s disease. By preventing APP from getting cut, BACE inhibitors would prevent the buildup of β–amyloid clusters in the brain, thereby slowing or preventing Alzheimer’s brain.

The small molecule AZD3293 passed Phase I (safety in healthy volunteers) clinical trial in 2014, and since has been in a Phase II/III trial (safety and efficacy on patients). With the fast-track status, the compound is now entering the Phase III portion to test safety, efficacy, and effectiveness in a larger number of patients. According to the FDA, “Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need.”

“The Fast Track Designation in the US for this promising potential therapy reinforces the ambition of the AstraZeneca-Lilly BACE Alliance to advance science for patients and their families managing this devastating illness. BACE inhibitors have the potential to transform the treatment of Alzheimer’s disease, one of the biggest challenges facing medical science today,” said Craig Shering, AZD3293 Project Lead in Global Medicines Development at AstraZeneca.

Of note, many other pharmaceutical companies are in contention to create effective BACE inhibitors. However, one of the main challenges was how to design compounds that could pass the blood-brain barrier. Indeed, this challenge has been recently met not only by AstraZeneca and Eli Lilly, but also by Biogen, and Merck independently. But, by nabbing the fast-track designation, AstraZeneca and Eli Lilly are the front-runners in the BACE inhibitor race to cure Alzheimer’s.

Final data from the trials won’t be available until August of 2019. But the excitement for the prospects of an Alzheimer’s drug is already very high. “We are pleased the FDA places a high priority on the development of drugs that target Alzheimer’s disease, one of the most critical health issues of our time. Most importantly, this is a positive step forward for the millions of patients, families, caregivers, advocates and healthcare providers who fight every day for progress,” said Phyllis Ferrell, Vice President and Global Development Leader for Alzheimer’s disease at Lilly.

Of note, Alzheimer’s disease is expected to exceed 74 million by 2030 and 131 million by 2050. The estimated cost of this disease is projected to reach $2 trillion by 2030. Given this, a fast-track designation still doesn’t seem quite fast enough.

Additional sources: Fierce Biotech, Astrazeneca release