Bellerophon reached its enrollment goal for the second group of patients in its ongoing Phase 2/3 trial evaluating different doses of INOpulse therapy for pulmonary hypertension associated with interstitial lung disease (PH-ILD), a illness for which no treatments exist.
PH-ILD patients in group (or cohort) 2 will test a higher dose — 45 mcg/kg ideal body weight (IBW) per hour — of inhaled nitric oxide (iNO, which is a potent vasodilator that relaxes blood vessels) and for a longer 16-week period than the first cohort of patients who experienced meaningful improvements with a lower dose (30 mcg/kg ideal body weight per hour).
The optimal dose identified from these studies will be used in a subsequent Phase 3 part, which is necessary for Bellerophon to apply for INOpulse’s approval. This last phase is expected to start in the first quarter of 2020.
“We have been extremely pleased with the rate of enrollment in cohort 2, and are thankful for the continued support and enthusiasm from our sites. We look forward to study completion by year-end 2019,” Fabian Tenenbaum, Bellerophon’s CEO, said in a news release.
In January, results from the first group of patients (cohort 1, with 41 patients) in the clinical trial (NCT03267108) demonstrated that an eight-week treatment with the lowest dose of iNO (30 mcg/kg IBW/hr) is safe, and produces clinically meaningful improvements in moderate to vigorous physical activity, or MVPA, as well as other physical activity parameters.
This dose of iNO, delivered through INOpulse, made it easier to walk, climb stairs, or do yardwork in 23% of the patients, compared with no improvements observed in these parameters in the placebo group.
Bellerophon has now reached its target enrollment of 44 subjects for cohort 2. In this group, patients will be randomized to a higher dose of iNO45 (45 mcg/kg IBW/hr) or a placebo, over 16 weeks. Subjects can then proceed to a long-term open-label study.
Cohort 3 includes the larger and pivotal Phase 3 part, which is hoped to provide key data to allow Bellerophon to request INOpulse’s approval. It is expected to enrol about 300 subjects who will be randomly and blindly assigned to either iNO or a placebo for 16 weeks. MVPA will be the primary efficacy measure, or endpoint, as recently agreed upon between Bellerophon and the U.S. Food and Drug Administration.
“PH-ILD is a debilitating and life threatening disease where patients suffer severe functional impairment that limit their capacity to perform even the most basic daily tasks,” Tenenbaum said. “The capability of INOpulse to improve MVPA would directly benefit a patient’s ability to function, and provides the potential for the first approved therapy in this unmet medical need.”