Bellerophon halts Phase 3 trial for futility after interim data check
- Bellerophon Therapeutics halted its Phase 3 trial for futility after 16 weeks of testing a pulsatile nitric oxide delivery system, INOpulse, for pulmonary arterial hypertension, the company said Tuesday.
- The study's Data Monitoring Committee recommended the trial be halted after completion of a pre-specified interim analysis on the first 75 enrolled subjects — half the intended size of the full study.
- The company has two other INOpulse programs: one for pulmonary hypertension associated with interstitial lung disease (PH-ILD) and the other for pulmonary hypertension with chronic obstructive pulmonary disease (PH-COPD). The company's Phase 2b trial of INOpulse for PH-ILD is ongoing, and results are expected at the end of this year.
The premature halt of the pulmonary arterial hypertension trial is a setback for Bellerophon, which had already reported mounting financial losses in June.
For the Phase 3 trial, Bellerophon had agreed with the Food and Drug Administration to an interim analysis of the data after half the subjects completed 16 weeks of treatment. The results on the primary endpoint of measuring improvement in 6-minute walking distance were not sufficient to continue the study, the company said.
Yet the data also suggested some improvement in pulmonary vascular resistance among treated subjects, and INOpulse therapy was well tolerated.
"While we are disappointed in the overall efficacy results of the study, we are encouraged by the positive data in hemodynamics and pleased with the safety and tolerability profile of INOpulse," Bellerophon CEO Fabian Tenenbaum said in a statement.
Tenenbaum noted that INOpulse achieved favorable results in Phase 2 studies for PH-ILD and PH-COPD. Since these conditions cannot be treated effectively with systemic vasodilators, INOpulse could fill a need if further trial results show efficacy.
In addition to its ongoing Phase 2b trial in PH-ILD, the company has finalized the design of a Phase 2b study in PH-COPD.
Yet due to its financial losses and the negative results of the Phase 3 trial, Bellerophon's future may need some help.
It reported a net loss of $11.5 million in the second quarter of 2018. Bellerophon says it has sufficient financial resources to continue its planned research and other activities until the first half of 2019.
- Bellerophon Therapeutics Statement