The study is double-blind, randomized, placebo-controlled, sequential allocation, and multicenter at 16 sites in the USA, Canada, Israel, Norway, Spain, and the UK. The age range for recruitment is between 35 and 80. Patients must be less than seven years since diagnosis, have an H&Y score of 3, and be free of major motor fluctuations or dyskinesia. Standard exclusionary criteria apply. The single ascending dose (SAD) is evaluating six dose levels of BIIB094, and the multiple ascending dose (MAD) will test three dose levels. The study is planned to complete in September 2023.
There are one primary and six secondary outcome measures. The primary one is the number of participants with adverse events (AEs) and serious adverse events (SAEs). The SAD will be conducted from screening (day -42) up to day 85. The MAD will run from screening (day -77) up to day 253. Secondary outcomes are: 1. Serum concentrations of BIIB094; 2. Area under the concentration-time curve from time zero extrapolated to infinity (AUCinf); 3. Area under the concentration-time curve from time zero to last quantifiable concentration (AUClast); 4. Maximum concentration (Cmax); 5. Time to reach maximum concentration (Tmax); 6. Terminal elimination half-life (t1/2). The timeframe for each of the secondary outcomes for the SAD is pre-dose through day 57; and for the MAD, pre-dose through day 169.
BIIB094 is the first ASO drug to reach the clinical phase of development for PD. The ultimate goal is to attempt to modify the course of PD, slowing or even stopping the progression. Establishing safety and tolerability parameters with a relatively new technology are thus crucial.