Bellerophon Therapeutics Inc (NASDAQ:BLPH) a clinical-stage biotherapeutics firm reported that it obtained confirmation from the U.S. FDA of the approval of all modifications proposed to Phase III plan for INOpulse in PAH.
Under the modified Phase III program, the underway 1-year INOvation-1 trial, and another confirmatory randomized withdrawal trial with almost 40 subjects who will be moving from the INOvation-1 trial, can serve as the two well-controlled and adequate studies to support a NDA submission for INOpulse in PAH patients on long term oxygen treatment. The randomized withdrawal study and INOvation-1 plan are planned to be performed on near parallel timelines, which could minimize the time to market for company’s drug in PAH by almost two years.
Both trials comprise an interim analysis almost half-way through each trial, projected to occur by the close of 2017 for the INOvation-1 trial and in 2H2018 for the withdrawal trial. The interim assessment will allow both trials to be stopped early if efficacy is shown.
The original Phase III program called for 2 trials, the INOvation-1 trial, followed by the INOvation-2 trial. Based on these metrics, the firm expected that INOpulse could get regulatory nod in 2022. With the accepted modifications to the Phase III program, the INOvation-2 trial will be substituted with the randomized withdrawal trial, a much smaller trial in almost 40 patients over a 4-month enrichment period and 2-month randomized withdrawal. Following this revised clinical study protocol, INOpulse could get regulatory nod as early as 2020.
Fabian Tenenbaum, the CEO and President of Bellerophon, reported that they are gratified that the FDA has approved to the proposed modifications to their PAH Phase III program. It has the prospect to make INOpulse accessible to PAH patients almost 2 years earlier than planned under the original Phase III program.
PAH is a rare disease and numerous patients continue to show poor results with currently available treatments. They consider INOpulse has the potential, if permitted, to be a well-tolerated and effective treatment option for these patients.