In a landmark deal that could someday provide help for the 5.1 million Americans suffering from heart failure, pharmaceutical giant Bristol-Myers Squibb recently purchased Cardioxyl Pharmaceuticals—a company co-founded by John Toscano, vice dean for natural sciences and a professor in the Krieger School’s Department of Chemistry, and two researchers at the School of Medicine, David Kass and Nazareno Paolocci—for $300 million. The total value could top $2 billion depending on future development and sales achievements of Cardioxyl’s CXL-1427, an intravenous treatment for late-stage heart failure known as acute decompensated heart failure.
Early clinical data has shown that CXL-1427 improves how the heart muscle relaxes and contracts without the complications incurred by current treatments, which frequently produce an increase in heart rate, oxygen consumption, or both. Other drugs have been known to restrict blood flow to parts of the body or cause severe arrhythmia, which can lead to death.
CXL-1427 releases nitroxyl, a gaseous molecule that previously had demonstrated beneficial effects on heart muscle and vascular health. Toscano and his collaborators were able to overcome the issue of how to safely and effectively deliver the notoriously unstable molecule into the bloodstream—something that had eluded scientists for decades.
Toscano, who was working independently on nitroxyl research, met up serendipitously with Kass and Paolocci after a researcher at the National Institutes of Health connected them in 2004. A dozen years later, CXL-1427, which is currently in Phase 2a clinical trials to evaluate efficacy and safety in selected populations, could provide hope for a condition that contributes to one in nine deaths annually, according to the Centers for Disease Control.
“I started here as a professor in ’95 and I certainly didn’t come here to develop drugs for heart failure,” says Toscano, who continues to study the chemistry and biology of nitroxyl in his lab. “But it has been a pretty cool experience to say the least.”