PhaseBio Announces Dosing of First Patient in an Exploratory Clinical Study of PB1046 for the Treatment of Pulmonary Arterial Hypertension

Malvern, PA, November 1, 2017 — PhaseBio Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing therapies for the treatment of orphan diseases, today announced that the first patient has been dosed in an exploratory study evaluating PB1046, a once-weekly vasoactive intestinal peptide (VIP) receptor agonist, for the treatment of pulmonary arterial hypertension (PAH).

The open-label, single cohort study is 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 are delighted to announce the dosing of the first patient in our PAH program. This marks a significant step toward our goal of building a portfolio of clinical-stage therapies that treat orphan cardiovascular disorders,” said John Lee, M.D., Ph.D., Chief Medical Officer of PhaseBio. “Previous studies have demonstrated that reduced VIP levels may be a critical factor in PAH disease development and progression. Consequently, VIP-based therapies such as PB1046 have the potential to provide benefit to patients who currently lack adequate treatment options. This exploratory study will provide safety and hemodynamic data to support a larger, placebo-controlled Phase 2 study, which is planned for early 2018.”

“There is a clear need for new therapeutic options that reverse the progression of PAH and improve long-term patient outcomes,” said Raymond L. Benza, M.D., Director, Heart Failure, Transplant, Mechanical Circulatory Devices and Pulmonary Hypertension at the Allegheny Health Network and lead investigator of the study. “VIP-based approaches have been shown to be highly effective in preclinical models of PAH and I look forward to working with PhaseBio to evaluate PB1046 in PAH patients.”

Additional information on the study can be found on using the identifier NCT03315507.

About PB1046

PB1046 is a first-in-class, once-weekly vasoactive intestinal peptide (VIP) receptor agonist that targets VPAC receptors in the cardiovascular, pulmonary and immune systems. VIP is known to have vasodilatory, inotropic, lusitropic and antifibrotic effects and several cardiopulmonary disorders are associated with alterations in levels of VIP or its receptors, VPAC1 and VPAC2. Consequently, VIP-based therapies are expected to provide benefit in PAH, cardiomyopathy and other cardiovascular diseases.

PB1046, which comprises an analogue of VIP fused to PhaseBio’s elastin-like polypeptide (ELP) biopolymer, is designed to overcome the poor in vivo stability and bioavailability of the native VIP peptide, and to preferentially bind to the VPAC2 receptor to minimize potential gastrointestinal side effects. PB1046 was well-tolerated and demonstrated a prolonged, dose-dependent effect on blood pressure in a Phase 1 study in patients with essential hypertension, and demonstrated clear efficacy in animal models of heart failure, PAH and DMD cardiomyopathy.

PhaseBio is currently evaluating PB1046 in two clinical studies: a Phase 2a multiple ascending dose study in subjects with heart failure, and an exploratory study in PAH. The U.S. Food and Drug Administration has granted PB1046 orphan drug designation for the treatment of PAH (WHO Group 1 PH) and cardiomyopathy associated with dystrophinopathies.

About PhaseBio

PhaseBio Pharmaceuticals, Inc., is a clinical-stage biopharmaceutical company developing therapies for the treatment of orphan diseases. PhaseBio is leveraging its proprietary ELP biopolymer technology platform to develop therapies with less-frequent dosing and the potential for better patient compliance. PhaseBio is privately owned, with headquarters and research laboratories in Malvern, PA. For more information, please visit


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