Unlike prostacyclin vasodilators which provide a much more systemic (i.e. indiscriminate) effect throughout the pulmonary arteries, INOpulse delivers iNO mostly to the well-ventilated areas of the lungs. This targeted delivery provides for distinct advantages, particularly as it relates to the optimization of treatment (and, specifically, to the mitigation of related side effects) of patients with certain lung diseases in which both respiratory function is impaired and blood flow is constricted.

Treatment with systemic pulmonary vasodilators can cause ‘ventilation-perfusion mismatch’ in patients with these diseases, a potentially serious complication resulting in abnormal oxygen desaturation of the blood (i.e. hypoxemia). INOpulse avoids this ventilation-perfusion (V/Q) mismatch through targeted delivery made possible by its novel pulsed action (synchronized with a patient’s breathing rate) and the rapid inactivation of inhaled nitric oxide upon contact with hemoglobin. INOpulse’s ability to safely, effectively and significantly improve vasodilation/ventilation balance, increase blood vessel volume, decrease pulmonary arterial pressure (PAP) and increase exercise capacity in patients with highly debilitating and progressive pulmonary diseases has been demonstrated in randomized controlled clinical trials.

BLPH is pursuing three specific PH-related (potential orphan) chronic indications; PH associated with interstitial lung disease (PH-ILD) which includes PH associated with idiopathic pulmonary fibrosis (PH-IPF), PH associated with chronic obstructive pulmonary disease (PH-COPD) and PH associated with Sarcoidosis (PH-Sarc). As each of these are associated with impairment to both lung function and pulmonary vasculature and characterized by high unmet therapeutic needs and severe limitation to quality of life, they offer highly attractive commercial opportunities for BLPH and INOpulse.

While results of a Phase 3 study evaluating INOpulse in the treatment of pulmonary arterial hypertension, or PAH, (a specific subgroup of PH, the underlying causes of which have key differences than those of the PH-associated diseases that BLPH is now pursuing) was discontinued (in August 2018) due to a determination that the primary endpoint would not be met, it did provide valuable clinical (such as clinically and statistically significant improvement in NT-ProBNP, a marker of right ventricle dysfunction) and safety data, helping to reinforce the opportunity for INOpulse in diseases more likely to benefit from targeted iNO administration (such as PH-ILD, PH-COPD and PH-Sarc).

Earlier this year BLPH announced positive topline data from a Phase 2 portion (i.e. ‘Cohort 1’) of an ongoing Phase 2/3 study of INOpulse in PH-ILD. This includes a statistically significant improvement in multiple clinically meaningful activity parameters, which is particularly encouraging as the proposed Phase 3 (pivotal) study is expected to use the same measures as its primary endpoint. On August 1st BLPH announced that Cohort 2 (higher dose group) of the Phase 2 portion completed enrollment, results of which are anticipated later this year and which will inform on the optimal dose to be used in the Phase 3 study. If all goes well the Phase 3 study could begin early next year.

Meanwhile, BLPH also expects to build on statistically significant Phase 2a results in PH-COPD, which were announced in September 2017. Design of a Phase 2b (n=90) study was recently reviewed and finalized by FDA. The goal of this study, which will assess multiple endpoints including activity monitoring and oxygen saturation, is to inform design, including specifics around patient population and clinical endpoints, of a subsequent Phase 3 (pivotal?) study. BLPH anticipates commencement in 2020.

And finally, the PH-Sarc program currently consists of an ongoing Phase 2a dose-escalation study which will use right heart catheterization to assess the safety and hemodynamic effect of INOpulse. The study commenced in Q1 and could have topline results later this year.

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