Civitas Therapeutics has released positive results from a Phase IIb clinical trial of CVT-301, an inhaled formulation of levodopa (L-dopa), being developed for the treatment of intermittent debilitating motor fluctuations (OFF episodes) associated with Parkinson's disease (PD).
The randomised, double blind, placebo-controlled, multicentre Phase IIb trial (CVT-301-003) is carried out in the US and Europe.
According to the company, CVT-301 will provide rapid and reliable relief from OFF episodes that affect many patients with PD.
OFF episodes result from the unpredictable nature of oral baseline therapies and they affect more than half of all patients on an oral L-dopa treatment regimen.
Civitas president and CEO Mark Iwicki said: "CVT-301 provided onset of action by ten minutes with durable effects lasting for at least 60 minutes.
"CVT-301 is being developed to address a significant unmet need facing Parkinson's disease patients today, and these results, particularly the efficacy and ease-of-use seen in the outpatient setting, give us confidence that CVT-301 can have a transformative impact on the daily lives of patients."
Part of the funding for the Phase IIb trial of CVT-301 is provided by a grant from The Michael J Fox Foundation for Parkinson's Research. The foundation's Parkinson's research CEO Todd Sherer said OFF episodes are debilitating events for patients with PD.
"A rapid and reliable therapy that can address these episodes would be a major advancement in treatment," Sherer said.
"These results suggest that CVT-301 could have a transformative impact on patients' lives.
"As supporters of this programme from its early days, we are pleased with its performance thus far and look forward to continued success in Phase III trials."
The Phase IIb trial evaluated the efficacy, safety and tolerability of two doses of CVT-301, while motor responses were assessed during regularly-scheduled clinical visits using the Unified Parkinson's Disease Rating Scale Part 3 (UPDRS III).
In addition, efficacy was also assessed during out-patient use with commonly used diary-based outcomes measures.
Eighty six patients treated in the trial underwent an initial screen and run-in period of two to four weeks.
The trial's primary endpoint was successfully met with CVT-301 achieving a clinically important and statistically significant reduction in average UPDRS III motor score versus placebo at time points ranging from ten to 60 minutes post-administration.
In addition, clinically important and statistically significant improvements in UPDRS III were seen at every time point including ten minutes, the earliest time point tested, for both tested doses.
Civitas is focused on developing and commercialising transformative therapeutics using its proprietary ARCUS technology, which delivers a reliable and consistent drug dose with a compact, breath-actuated inhaler.
Image: PDB rendering of Parkin (ligase). Photo: courtesy of Emw.