New Therapy for Parkinson’s ‘Off’ Periods Shows Promising Results in Preclinical Studies

CVT-301, an inhalable drug under development by Acorda Therapeutics to treat Parkinson’s symptoms during “off” periods when currently available drugs don’t adequately work, showed promising results in preclinical Phase 1 and 2 studies.

Since the late 1960s, levodopa has been the most common and efficient oral drug used for managing Parkinson’s motor symptoms. But as the disease progresses, levodopa tends to lag in effectiveness. Approximately 50 percent of patients on levodopa experience off periods. The new levadopa-based inhalable aims to counter “off” periods with therapeutic “on” times.

“‘Off’ periods can be hugely disruptive to the lives of people with Parkinson’s and their families, and are considered one of the greatest unmet medical needs in the treatment of Parkinson’s,” Dr. Ron Cohen, MD, president and CEO of Acorda, said in a press release.

Published in the Science Translational Medicine journal, the study, “Preclinical and clinical assessment of inhaled levodopa for OFF episodes in Parkinson’s disease,” demonstrated CVT-301’s ability to restore levodopa blood levels and the drug’s clinical effects in 24 patients experiencing “off” episodes.

The study reported that a single inhaled dose of CVT-301 increases levodopa faster than oral medicines because it enters the body through the lungs, bypassing the digestive system, reaching the brain quicker. Study patients showed overall motor function improvement between five and 15 minutes after administration.

CVT-301 will be additionally tested for safety and effectiveness in Phase 3 clinical trials.

“Following two successful Phase 2 clinical trials, our Phase 3 program is assessing the extent to which CVT-301, used when people with Parkinson’s begin to experience ‘off’ periods, can restore motor function. If approved, CVT-301 may provide a valuable new treatment option for these individuals,” Cohen said.