Pharmaceutical powerhouses AstraZeneca and Eli Lilly have announced that they will be pushing their experimental Alzheimer’s candidate into a Phase III clinical trial. Unlike many other investigational treatments for the neurodegenerative disease, their drug showed no harmful side effects in initial safety testing in human patients.
The new drug – dubbed AZD3293 – is known as a BACE inhibitor. This class of pharmaceuticals is designed to block the enzyme beta-secretase 1, which could contribute to the build-up of amyloid plaques commonly seen in the brain tissue of people with Alzheimer’s disease. Researchers are still unsure as to whether this amyloid protein is responsible for the symptoms of Alzheimer’s, or whether the plaques are simply a symptom of the disease.
AstraZeneca and Eli Lilly plan to start enrolling patients with early stages of the disease for the upcoming Phase III clinical trial. Their goal is to recruit 2,200 patients representing 14 different countries, before starting the trial.
According to the two companies, they will also conduct a separate trial to test the effectiveness of the drug in patients with mild Alzheimer’s. This trial will begin enrolling participants in the third quarter of this year.
“Alzheimer’s disease remains one of the biggest challenges facing medical science today,” said Menelas Pangalos, executive vice president of AstraZeneca’s IMED Biotech unit. “BACE inhibitors have the potential to target one of the key drivers of disease progression and we are delighted that our combined efforts have resulted in the development of AZD3293 moving into the next phase of study.”
BACE inhibitors are some of the most promising drugs in development, but they could still fail once they enter late-stage clinical trials. In 2013, Eli Lilly abandoned a BACE inhibitor – known as LY2886721 – when it was established that the drug could impact normal liver function.
According to research published by the trade group Pharmaceutical Research & Manufacturers of America (PhRMA), there were 101 Alzheimer’s drug failures between 2000 and 2012. This high failure rate has increased the number of risk-sharing partnership deals in the Alzheimer’s space.
Under the agreement between the two companies, Eli Lilly has taken responsibility for designing and executing all clinical trials – a role previously filled by AstraZeneca alone. The two have agreed to split the cost development, along with any future revenues of the drug, should it gain FDA approval.
According to the Alzheimer’s Association, approximately 5.3 million Americans have the neurological disease. In 2013, the Alzheimer’s drug market – consisting of drugs that can only manage symptoms – was a $4.9 billion industry; this number is expected to grow to $13.3 billion by 2023, according to statistics compiled by GlobalData.