Lilly’s Drug Receives Breakthrough Therapy for Alzheimer’s Disease

Eli Lilly and Company recently announced that FDA granted breakthrough therapy designation for its investigational antibody therapy, donanemab, intended for Alzheimer’s disease.

This closely follows the contentious FDA accelerated approval of Biogen’s Aduhelm, another drug intended for Alzheimer’s disease.

Donanemab targets a modified form of beta amyloid called N3pG. FDA based its breakthrough designation for the therapy on clinical evidence for Eli Lilly and Company’s Phase 2 trial, TRAILBLAZER-ALZ, which studied the efficacy and safety of donanemab in patients with early, symptomatic Alzheimer’s disease. 

Researchers enrolled 272 patients based on cognitive assessments in conjunction with amyloid plaque imaging and tau staging by PET imaging. The trial’s primary endpoint was change from baseline until 76 weeks in the Integrated Alzheimer’s Disease Rating Scale (iADRS).

Trial results showed that donanemab slowed cognitive and functional decline in patients with early symptomatic Alzheimer’s disease and slowed tau accumulation across key brain regions in patients. 

Specifically, at 76 weeks compared to baseline, treatment with donanemab slowed decline by 32 percent compared to placebo. Forty percent of participants treated with donanemab achieved amyloid negativity as early as six months after starting treatment, while 68 percent of patients achieved this target by 18 months.

Eli Lilly and Company stated that it intends to submit a biologics license application for donanemab under the accelerated approval pathway later this year based on data from TRAILBLAZER-ALZ. 

Currently, the therapy’s safety, tolerability, and efficacy are being evaluated in the ongoing, placebo-controlled, double-blind Phase 3 study, TRAILBLAZER-ALZ 2. 

Over six million Americans of all ages have Alzheimer’s disease. Dementia due to Alzheimer’s disease is the most common form of dementia, accounting for 60 to 80 percent of all cases.
Most recently, FDA officially approved Biogen’s aducanumab, marking the first Alzheimer’s drug approval in nearly 20 years and the first one to address cognitive decline in those with illness.