Clinical data from a phase 3 trial of CVT-301 (Acorda Therapeutics), an investigational, inhalable formulation of levodopa (L-dopa), have shown a statistically significant improvement in motor function in patients with Parkinson’s disease (PD) experiencing OFF periods. CVT-301 is being studied as a treatment for OFF periods in PD patients receiving an oral carbidopa/levodopa regimen. OFF periods refer to the re-emergence of PD symptoms.
The randomized, double-blind, placebo-controlled trial evaluated the efficacy and safety of CVT-301 compared with that of placebo in PD patients who experience motor fluctuations (OFF periods). The trial had three arms: CVT-301 84 mg and CVT-301 60 mg (equivalent to 50-mg and 35-mg fine-particle doses, respectively), and placebo. All of the subjects were receiving a stable regimen of oral carbidopa/levodopa and were maintained on their other existing PD therapies. A total of 339 subjects received at least one dose of CVT-301 or placebo. The subjects self-administered treatment up to five times daily for 12 weeks.
The study’s primary endpoint was the change at week 12 in the Unified Parkinson’s Disease Rating Scale–Part III (UPDRS III) score––a measure of Parkinson’s motor involvement––compared with placebo at 30 minutes after treatment for the 84-mg dose of CVT-301. The UPDRS III change for the 84-mg dose was –9.83 compared with –5.91 for placebo (P = 0.009).
CVT-301 uses Acorda Therapeutics’ investigational ARCUS platform for inhaled medications. CVT-301 delivers a dry-powder formulation of L-dopa to the lungs. Oral medications can be associated with a slow and variable onset of action, as the medication is absorbed through the gastrointestinal tract before reaching the brain. Inhaled treatments enter the body through the lungs and reach the brain shortly thereafter, bypassing the digestive system.
Approximately one million people in the United States have PD; OFF periods are experienced by approximately 350,000 of those individuals.
PD is a progressive neurodegenerative disorder resulting from the gradual loss of certain neurons responsible for producing dopamine. The disease causes a range of symptoms, including impaired movement, muscle stiffness, and tremors. As PD progresses, individuals experience OFF periods, which are characterized by the re-emergence of symptoms. This re-emergence can occur even when an individual’s treatment regimen has been optimized.
Source: Acorda Therapeutics; February 9, 2017.