Successful Phase IIb trial for CVT 301 for Off periods in Parkinson's disease- Acorda

Data from the Phase IIb clinical trial of CVT 301, published in the peer-reviewed journal Movement Disorders, showed that people with Parkinson’s disease (PD) experiencing OFF periods and who were treated with CVT 301, from Acorda, had significantly greater improvements in motor function than those treated with placebo. A treatment effect was evident at 10 minutes after dosing and was sustained for at least one hour, the longest time point at which patients were assessed.

The Phase IIb trial was a randomized, double blind, placebo-controlled, multicenter study in 86 people with PD for the treatment of OFF periods. Participants were randomized to self-administer CVT 301 or placebo to the lung via an inhaler, as an adjunct to their established oral PD medications. Participants received 35mg of CVT 301 or placebo in study weeks 1 and 2, and 50mg of drug or placebo in weeks 3 and 4. The primary endpoint was defined as the mean change from baseline OFF state in Unified Parkinson’s Disease Rating Scale motor function (UPDRS Part III) score, (measured at 10-60 minutes post dose) after 4 weeks of treatment. The UPDRS is an established assessment method to monitor PD motor impairment that has been used extensively in clinical research.

In this study, participants receiving CVT 301 showed a statistically significant and clinically important reduction in average UPDRS III score compared to placebo (p<0.01) and across all measured time points beginning at 10 and up to 60 minutes post-administration (p<0.05). There were no concerning safety signals observed in either CVT 301 dose group, with no increase relative to placebo in troublesome or non-troublesome dyskinesias during ON periods. There were no serious adverse events reported in the drug group, and the incidence of drug-related adverse events was similar between treatment groups (23% drug group; 21% placebo group). The most common adverse events were dizziness (7% drug group; 5% placebo), cough (7% drug group; 2% placebo) and nausea (7% drug group; 0% placebo); there were no adverse events related to cardiovascular or lung function. PD patients were able to self-administer treatment while in an OFF state.

Comment: OFF periods are characterized by a re-emergence of PD symptoms, including motor symptoms such as the impaired ability to move, muscle stiffness and tremor. This re-emergence can occur even when treatment regimens of oral levodopa (L-dopa) and other standard of care medications have been optimized.