Eli Lilly and AstraZeneca announce continuation of pivotal clinical trial for people with early Alzheimer's disease

Phase II/III trial of AZD3293, an oral potent small molecule BACE inhibitor, will continue to Phase III after positive interim safety data.

The AMARANTH independent data monitoring committee recommended the study continue without modification after a scheduled interim safety analysis was conducted. The analysis was not designed to review efficacy.

"This is an important and meaningful step forward on the path to better understand the Alzheimer's puzzle," said Phyllis Ferrell, vice president and global development leader for Alzheimer's disease at Lilly. "We'd like to thank the AMARANTH participants and the trial investigators for taking part in this important study, and thank our colleagues at AstraZeneca for their partnership."

AZD3293 has been shown in Phase I studies to reduce levels of amyloid beta in the cerebro-spinal fluid of people with Alzheimer's and healthy volunteers. The progression of Alzheimer's disease is characterized by the accumulation of amyloid plaque in the brain. BACE is an enzyme associated with the development of amyloid beta. Inhibiting BACE is expected to prevent the formation of amyloid plaque and eventually slow the progression of the disease.

"Alzheimer's disease remains one of the biggest challenges facing medical science today. 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," said Menelas Pangalos, executive vice president, IMED Biotech Unit, AstraZeneca. "Disease-modifying approaches, such as this, have the potential to transform the treatment of Alzheimer's disease and help patients in this area of large unmet medical need."

Under the terms of the agreement, AstraZeneca will receive a milestone payment from Lilly now that AZD3293 has moved into Phase III testing. The payment will result in a second-quarter charge of $100 million (pre-tax) to Lilly's GAAP and non-GAAP research and development expense.

Lilly and AstraZeneca have also announced the planned initiation of a new Phase III trial for AZD3293. The trial, named DAYBREAK, will study the safety and efficacy of AZD3293 in people with mild Alzheimer's dementia. DAYBREAK will begin enrolling participants in the third quarter of 2016.

AstraZeneca and Lilly announced an alliance in 2014 for the development and commercialisation of AZD3293/LY3314814. Under the agreement, Lilly leads clinical development, working with researchers from AstraZeneca's Neuroscience Research and Development Team, while AstraZeneca will be responsible for manufacturing. The companies will take joint responsibility for commercialisation of the molecule and will share all future costs equally for development and commercialisation, as well as net global revenues post-launch.