Donanemab is an investigational antibody therapy that has shown positive results in treating Alzheimer's disease in clinical trials
Eli Lilly and Company's Corporate Center in Indianapolis, Indiana. (Credit: Momoneymoproblemz/Wikipedia.)
Eli Lilly and Company (Lilly) has received the US Food and Drug Administration (FDA) Breakthrough Therapy designation for its donanemab to treat Alzheimer’s disease.
Donanemab is an investigational antibody therapy that targets a modified form of beta amyloid called N3pG and has shown positive results in two previous clinical trials.
FDA’s Breakthrough Therapy designation facilitates the development and review of drugs for serious conditions, which show promising results in early-stage clinical trials.
Lilly intends to submit a biologics license application (BLA) for donanemab under the accelerated approval pathway later this year, based on TRAILBLAZER-ALZ study data.
The company is currently evaluating the safety, tolerability and efficacy of the drug in the ongoing Phase 3 TRAILBLAZER-ALZ 2 study.
The FDA granted Breakthrough Therapy designation for donanemab based on clinical evidence from Phase 2 TRAILBLAZER-ALZ, a randomised, placebo-controlled trial.
The Phase 2 study evaluated the safety, tolerability and efficacy of donanemab in 272 patients with early symptoms of Alzheimer’s disease.
Participants in the trial were selected based on cognitive assessments in combination with amyloid plaque imaging and tau staging by PET imaging.
The change from baseline until 76 weeks in the Integrated Alzheimer’s Disease Rating Scale (iADRS) is the primary endpoint of the study.
iADRS is a composite tool combining the Alzheimer’s Disease Assessment Scale-Cognitive subscale (ADAS-Cog13) and the Alzheimer’s Disease Cooperative Study – instrumental Activities of Daily Living (ADCS-iADL) for function.
The changes in ADAS-Cog13, ADCS-iADL, MMSE, and Clinical Dementia Rating Scale Sum of Boxes (CDR-SB) scores include key secondary endpoints.
Other secondary biomarker endpoints included changes in brain amyloid deposition and brain tau deposition and volumetric MRI.