Malvern, PA, February 27, 2018 — PhaseBio Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing therapies for the treatment of orphan diseases, today announced that the Company has been awarded a $2.8 million Fast Track Small Business Innovation Research (SBIR) grant from the National Institutes of Health (NIH) to support the clinical development of PB1046, a first-in-class, sustained-release vasoactive intestinal peptide (VIP) analogue, in patients with pulmonary arterial hypertension (PAH).
The Phase I portion of the grant will provide funding for an ongoing exploratory clinical trial of PB1046, which is currently enrolling adult PAH patients with a permanently implanted hemodynamic monitor (CardioMEMS™ HF System) (NCT03315507). The Phase II portion of the grant will support a larger, Phase 2 clinical trial in PAH planned to initiate by mid-2018.
“Recent advancements in the treatment of PAH have improved patient symptoms and exercise capacity, but are not curative and long-term disease prognosis remains poor, leaving the door open for innovative new therapeutic options like PB1046,” said John Lee, M.D., Ph.D., Chief Medical Officer of PhaseBio and Principal Investigator for the grant.
“PB1046’s novel mechanism of action targets VPAC receptors, which, along with VIP, are believed to be a critical factor in the development and progression of PAH. Consequently, VIP-based therapies, like PB1046, may provide compelling new treatment options that could reverse disease progression and improve long-term patient outcomes,” said Jonathan P. Mow, Chief Executive Officer of PhaseBio. “This grant award validates our therapeutic approach and PhaseBio’s position as an emerging player in the development of innovative therapies for rare diseases.”
PB1046 is a 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 pulmonary arterial hypertension (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 clinical trial in patients with essential hypertension, and demonstrated clear efficacy in animal models of heart failure, PAH and Duchenne muscular dystrophy-related 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.
PhaseBio Pharmaceuticals, Inc., is a clinical-stage biopharmaceutical company developing therapies for the treatment of orphan diseases. PhaseBio is leveraging its proprietary elastin-like polypeptide (ELP) biopolymer technology platform to develop therapies with the potential for less-frequent dosing and better patient compliance. PhaseBio’s lead development candidate, PB1046, is a first-in-class weekly vasoactive intestinal peptide (VIP) receptor agonist for the treatment of pulmonary arterial hypertension. The company is also developing PB2452, a reversal agent for the antiplatelet therapy ticagrelor. PhaseBio is privately owned, with headquarters and research laboratories in Malvern, PA. For more information, please visit www.phasebio.com.
Laura Bagby, 6 Degrees