PB1046 for Pulmonary Arterial Hypertension

PAH is a rare, rapidly progressive and fatal disease that is 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 novel PAH targets and therapies that reverse the disease process and improve long-term health outcomes.

Clinical studies in patients with PAH and studies in animal models 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.

We expect to initiate a phase 2 clinical study of PB1046 for the treatment of PAH in early 2017, 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.