Rare Disease Report

NIH Awards $2.8 Million Grant to PhaseBio for Potential PAH Therapy

FEBRUARY 28, 2018
Kaitlynn Ely
A $2.8 million Fast Track Small Business Innovation Research (SBIR) grant has been awarded to PhaseBio Pharmaceuticals from the National Institutes of Health (NIH) to support the clinical development of PB1046 in treating patients with pulmonary arterial hypertension (PAH).

PB1046 is a first-in-class, once-weekly, sustained-release vasoactive intestinal peptide (VIP) analogue that targets VPAC receptors in the cardiovascular, pulmonary, and immune systems. Due to its vasodilatory, inotropic, lusitropic and antifibrotic effects, VIP-based therapies benefit patients with PAH, cardiomyopathy and other cardiovascular diseases.

The potential PAH therapy has already been granted orphan drug designation for the treatment of PAH and cardiomyopathy associated with dystrophinopathies by the U.S. Food and Drug Administration (FDA).

“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 in a press release.

PAH is a progressive condition affecting the heart and lungs. Patients will have abnormally high blood pressure in the pulmonary artery, causing shortness of breath and fainting spells. The condition will worsen as patients age, resulting in dizziness, swelling of the ankles or legs, chest pain and a racing pulse.

In some cases, the condition is the result of mutations in the BMPR2 gene. Others times, however, it can be caused by connective tissue diseases, like HV infections, chronic hemolytic anemia, and congenital heart disease. Once diagnosed with PAH, patients are estimated to survive 2.8 years depending on their age and the severity of the disease.

While current treatments manage symptoms, they have no effect on improving survival.

The Phase 1 part 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). In the Phase 1 clinical trial, PB1046 was well tolerated and demonstrated a prolonged, dose-dependent effect on blood pressure in patients with essential hypertension, and demonstrated clear efficacy in preclinical studies in animal models of heart failure, PAH and Duchenne muscular dystrophy-related cardiomyopathy.

The Phase 2 clinical trial in PAH will be initiated by mid-2018.

“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,” stated 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.”

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