Both studies include an interim analysis approximately half-way through each study, estimated to occur by the end of 2017 for the INOvation-1 study and in the second half of 2018 for the withdrawal study. The interim analysis will allow both studies to be stopped early if efficacy is demonstrated.
The original Phase 3 program called for two studies, the INOvation-1 study (n=188; placebo arm and iNO 75 arm), followed by the INOvation-2 study (n=282; placebo arm, iNO 50 arm and iNO 75 arm). Based on these parameters, the Company anticipated that INOpulse could receive regulatory approval in 2022. With the approved modifications to the Phase 3 program, the INOvation-2 study will be replaced with the randomized withdrawal study, a much smaller study in approximately 40 subjects over a four-month enrichment period and two-month randomized withdrawal. Under this revised clinical trial protocol, INOpulse could receive regulatory approval as early as 2020.
“We are gratified that the
In the modified Phase 3 program, following completion of INOvation-1, subjects will receive at least four months of active, open-label iNO treatment with a dose of 75 mcg/kg IBW/hr (iNO75). Those subjects who demonstrate ≥ 30 meter improvement in six minute walk distance, the primary endpoint of INOvation-1, and can tolerate the iNO, will constitute an "enriched" population of iNO responders who will be randomized to either placebo or iNO75 in the withdrawal study. Changes in clinical status (defined as clinical worsening) within an individual patient during the randomized withdrawal period will provide the necessary evidence of whether the improvements observed during the enrichment phase were related or non-related to iNO therapy. The proposed study design, therefore, provides the formal confirmatory evidence of whether iNO at a dose of 75 mcg/kg IBW/hr delivered via the INOpulse delivery device for up to 24 hours per day is efficacious in subjects with PAH concomitantly using approved PAH medication and LTOT.
About the INOpulse Clinical Program
The lead indication for INOpulse is Pulmonary Arterial Hypertension (PAH). In
Any statements in this press release about Bellerophon’s future expectations, plans and prospects, including statements about the clinical development of its product candidates, regulatory actions with respect to the Company’s clinical trials and expectations regarding the sufficiency of the Company’s cash balance to fund clinical trials, operating expenses and capital expenditures, and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of future clinical trials, availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary or interim results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, expectations for regulatory approvals, the FDA’s substantial discretion in the approval process, availability of funding sufficient for our foreseeable and unforeseeable operating expenses and capital expenditure requirements and other factors discussed in the “Risk Factors” section of the Company’s most recent filings with the
Bellerophon Therapeutics: Fabian Tenenbaum, Chief Executive Officer (908) 574-4819 LifeSci Advisors Bob Yedid, Managing Director (646) 597-6989 firstname.lastname@example.org