Acorda Therapeutics (NSDQ:ACOR) said today that the phase III clinical trial evaluating its inhaled formulation of Parkinson’s drug levodopa met its primary endpoint. The drug was studied as a therapy for people with Parkinson’s disease experiencing “off” periods, or a re-emergence of symptoms.
The randomized phase III trial enrolled 339 patients to evaluate the efficacy and safety of CVT-301 compared with a placebo in patients with Parkinson’s who experience motor fluctuations. Study participants self-administered the therapy up to 5 times per day for 12 weeks.
The study’s primary endpoint was a change in the Unified Parkinson’s Disease Rating Scale score at week 12 relative to a placebo at 30 minutes post-treatment. Secondary endpoints included the proportion of participants who achieved an “on” state within 60 minutes of treatment and maintained at 60 minutes, as well as total daily “off” time as recorded in participants’ logs.
The trial participants were randomized into 3 arms – a group that received an 84 mg dose, a 60 mg dose and a placebo. The UPDRS change for the 84 mg dose was -9.83 compared to -5.91 for their counterparts in the control group.
The inhaled drug’s safety profile was consistent with results observed in the phase IIb trial. The company said it is conducting 2 studies to assess the long-term safety profile of CVT-301, which it will present at the end of the 1st quarter this year.
“We are greatly encouraged by the efficacy and safety results of this trial, which validate the positive Phase IIb results,” chief medical officer Dr. Burkhard Blank said in prepared remarks. “We would like to express our gratitude to the study volunteers and clinical investigators who participated in this trial to advance our understanding of this potentially important therapy for people with Parkinson’s.”
Acorda said it plans to file a New Drug Application in the U.S. by the end of the 2nd quarter this year, contingent on the company’s long-term safety studies. The company also said it anticipates it will file a Marketing Authorization Application in Europe by the end of this year.
“The re-emergence of Parkinson’s disease symptoms has a major negative impact on the lives of people with this disease, as well as on their families and care partners,” lead investigator Dr. Peter Lewitt added. “Managing symptoms of ‘Off’ periods continues to be a significant unmet need for people taking oral carbidopa/levodopa regimens. Delivering levodopa by the pulmonary route offers an important treatment option for people with Parkinson’s disease.”
ACOR shares were trading at $23.08 apiece in mid-morning activity, up +13%.