Biothech company Bellerophon Therapeutics met the enrollment goal for its Phase 2 clinical trial evaluating INOpulse in patients with pulmonary arterial hypertension. Eighty patients, with age limits of 16-80 years, were enrolled at 52 sites across the United States and Canada.
INOpulse is pulsed, inhaled nitric oxide. Nitric oxide is naturally secreted by endothelial cells lining blood vessels to cause relaxation of smooth muscle cells and dilate blood vessels. This lowers blood pressure and decreases strain on the heart to pump blood to the lungs and throughout the body.
“The US Food and Drug Administration has granted orphan drug designation for nitric oxide for the treatment of PAH, which will give us seven years of exclusivity in the US if INOpulse is the first inhaled nitric oxide therapy approved in this indication,” said Daniel Tasse, interim chief executive officer of Bellerophon, in a press release. “There is presently no cure for PAH and, despite several approved therapies, the mortality rate remains high. The completion of enrollment in this important Phase 2 trial is therefore a key milestone for Bellerophon, and we look forward to the continued development of this potential new first-in-class therapy.”
The study is randomized, placebo-controlled, and designed to evaluate the safety and efficacy of inhaled nitric oxide. Three treatments groups will be studied: 0.025 mg/kg ideal body weight (IBW)/hr or 0.075 mg/kg IBW/hr inhaled nitric oxide or 0.075 mg/kg IBW/hr placebo. The study is split into two parts: weeks 0-16 will be used to evaluate the safety, tolerability, and efficacy of the different inhaled nitric oxide doses, and the remaining weeks will evaluate the long-term effects of inhaled nitric oxide.
Change in vascular resistance from baseline at 16 weeks is the primary outcome measure, and changes in mean pulmonary arterial pressure and cardiac index are included in the secondary outcome measures. The completion date is December 2014.
INOpulse is convenient for patients, as it is a special, portable delivery device. It enables ambulatory patients to receive daily treatments without the burden of visiting a hospital. Although only 80 patients are enrolled in the trial, fewer than 20,000 pulmonary arterial hypertension patients are diagnosed in the United States, giving the condition orphan disease status from the FDA.