Acorda Therapeutics said today it plans to file for regulatory approvals in the U.S. and Europe this year for its Parkinson’s disease candidate CVT-301 in the second quarter after the inhalable levodopa (L-dopa) met its primary endpoint in a Phase III trial.

CVT-301 showed statistically significant improvement in motor function in people with Parkinson’s who experience re-emergence of disease symptoms or OFF periods in the SPAN-PD trial. The primary endpoint for SPAN-PD was the change at week 12 in Unified Parkinson’s Disease Rating Scale–Part 3 (UPDRS III) score relative to placebo at 30 minutes post-treatment for the 84-mg dose. That dose showed a UPDRS III change of –9.83 compared to –5.91 for placebo.

Key secondary endpoints, also measured at week 12, included: proportion of participants achieving an ON state within 60 minutes of treatment and maintained at 60 minutes; change in UPDRS III score at 10 and 20 minutes following treatment; Patient Global Impression of Change (PGI-C) improvement; and total daily OFF time as recorded in participant diary.

SPAN-PD was designed to assess efficacy of two doses of CVT-301—84 mg and 60 mg (equivalent to 50 mg and 35 mg fine-particle doses, respectively)—comparing them to placebo. A total of 339 participants were randomized and received at least one dose of CVT-301 or placebo. Participants self-administered treatment up to five times daily for 12 weeks.

Six participants in the 60-mg arm reported serious adverse events, compared with three in the placebo arm and two in the 84-mg arm. One participant in the 60-mg group committed suicide, but the study’s investigator said the death was not related to the study drug.

Detailed trial results will be presented at an unspecified future medical meeting, Acorda said.

Acorda said the safety profile of CVT-301 in the study was consistent with that observed in the Phase IIb trial. Spirometry and diffusing capacity of the lung for carbon monoxide (DLCO) tests showed no notable pulmonary safety signals. By the end of the first quarter, the company expects to report up to 12-month data from two safety studies assessing the long-term safety profile of CVT-301.

“We are greatly encouraged by the efficacy and safety results of this trial, which validate the positive Phase IIb results,” Acorda CMO Burkhard Blank, M.D., said in a statement.

The company said it will file an NDA with the FDA by the end of the second quarter, and a marketing authorization application with the European Medicines Agency by the end of 2017, pending additional data analyses.

CVT-301 is designed to deliver a precise dose of a dry powder formulation of L-dopa to the lung, using Acorda’s investigational ARCUS® platform for inhaled therapeutics.

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