PhaseBio Doses First Patients in Two-Part Phase 2a Trial for Cardiopulmonary Disorder Drug

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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DMD clinical trial results

The first patients have been dosed in PhaseBio Pharmaceuticals‘ part one of a Phase 2a clinical trial for PB1046, a once-weekly vasoactive intestinal peptide (VIP) receptor activator currently in development to treat cardiopulmonary disorders.

Part one is designed to assess the effect of PB1046 in adult patients with stable heart failure with reduced ejection fraction (measure of how well the heart pumps blood.) Later this year, part two of the trial will evaluate the therapy in patients with heart impairment secondary to Duchenne or Becker muscular dystrophy.

“There is a clear need for heart failure treatments that both alleviate symptoms and slow or reverse disease progression,” Dr. John Lee, Ph.D., chief medical officer of PhaseBio said in a recent press release. “We look forward to building on our compelling preclinical data in models of heart failure, and the positive data from our Phase 1 studies, which demonstrated the safety and tolerability of PB1046 at effective doses in patients with hypertension.”

Vasoactive intestinal peptide (VIP) is a naturally occurring 28-amino acid peptide, with vasodilatory biological properties that are mediated by two G-protein-coupled receptors: VPAC1 and VPAC2. Nerve fibers enclosing VIP are broadly distributed in the central and peripheral nervous systems, in myocardial tissue, and in systemic and coronary arteries.

Several cardiovascular diseases are associated with changes in VIP concentration or with alteration of affinity, density or physiological responsiveness of VIP receptors. VIP based treatments may help patients’ with heart failure, hypertension, pulmonary arterial hypertension and other cardiovascular conditions.


The randomized, double-blind, placebo-controlled, Phase 2a clinical trial will assess the drug’s safety, tolerability and pharmacokinetics (how the drug works in the body) and explore the pharmacodynamic response (how the body respond to the drug) following 4 weeks of treatment. The once weekly skin injections of PB1046 will be given to adult patients with stable heart failure with reduced ejection fraction (part one) and then in patients with cardiac dysfunction secondary to Duchenne or Becker Muscular Dystrophy (part two).

The trial will be a sequential multiple-dose escalation study that will enroll 28 patients in four cohorts composed of 3 active and 1 placebo in Cohort 1; and 6 active and 2 placebo in subsequent cohorts. Randomized patients will be treated with a fixed weekly dose of PB1046 or a placebo for 4 weeks. Dose levels that show as safe and well-tolerated in part one will be selected for further clinical assessment in part two.

“This is an important milestone in the progression of our pipeline and the expansion of our programs in cardiopulmonary disorders and orphan diseases,” said Jonathan P. Mow, chief executive officer of PhaseBio. “We look forward to initiating part two of this study later this year in DMD patients with cardiomyopathy, which is a leading cause of death in people suffering from this devastating disease.”