Objective: To describe the design and status of the BIIB054 SPARK trial of the safety and potential efficacy of a monoclonal antibody (mAb) targeting alpha-synuclein (α-syn).

Background: Aggregated α-syn is a major constituent of Lewy bodies and is thought to play a central role in the pathology and progression of Parkinson’s disease (PD). BIIB054 is a mAb that binds with sub-nanomolar affinity to the N-terminal region of aggregated α-syn with much lower affinity for the monomer. Favorable toxicology has been observed in chronic exposure studies in rats and monkeys. A first-in-human, single ascending dose study of BIIB054 in healthy controls and PD patients demonstrated a favorable safety and pharmacokinetic profile.

Methods: The SPARK trial is an adaptively-designed randomized, double-blind, placebo-controlled parallel-group study testing the safety and biological effects of three dosages of BIIB054 compared to placebo studied in two staggered cohorts (24 in cohort A, 287 in cohort B). Participants with early, untreated PD recruited at approximately 90 international sites will receive randomized treatment (monthly IV infusions) for two years. The primary objective is to evaluate dose-related safety of BIIB054. Secondary objectives are to assess the pharmacodynamic effects of BIIB054 through dopaminergic imaging and clinical symptom burden. The SPARK study is sponsored by Biogen and is being conducted in North America in cooperation with the Parkinson Study Group.

Results: The first subject in the BIIB054 study was enrolled in January 2018. Enrollment is expected to continue through 2018. The design and status of the study will be presented.

Conclusions: The SPARK study will assess the dose-related safety of BIIB054. It is hoped that results of the study may support further evaluation of BIIB054 as a potential treatment for PD.

« Back to 2018 International Congress

MDS Abstracts - https://www.mdsabstracts.org/abstract/design-and-status-of-the-biib054-spark-trial/