PB1046 for Pulmonary Arterial Hypertension
PAH is a rare, rapidly progressive and fatal disease characterized by pulmonary arterial remodeling, severe pulmonary hypertension and right heart failure. Despite currently approved treatments, prognosis in PAH remains poor, with approximately 15 percent mortality each year. Consequently, there is a high unmet need for PAH therapies that reverse the disease process and improve long-term health outcomes.
Preclinical studies in animal models and clinical studies in patients with PAH have demonstrated that the absence of VIP may be a critical factor in disease development. VIP aids in the regulation of blood pressure, inhibits pulmonary vascular smooth muscle proliferation and remodeling, and has anti-inflammatory and anti-fibrotic actions. As a novel analogue of VIP, PB1046 has the potential to provide significant therapeutic benefit to PAH patients who have suffered disease progression on currently approved agents.
PhaseBio is conducting an exploratory open-label, single cohort trial examining the safety, tolerability and hemodynamic response of individually dose-titrated PB1046 in adult PAH subjects with a permanently implanted hemodynamic monitor (CardioMEMS™ HF System). Other endpoints include evaluation of pharmacokinetics, exercise capacity by the 6-minute walk distance test and exploratory biomarkers.
We expect to initiate a larger Phase 2 clinical trial in mid-2018, which will evaluate improvements in patients on currently approved therapies.
PB1046 has received orphan drug designation from the U.S. Food and Drug Administration (FDA) in PAH.