What makes a PhII drug worth up to $665M? Researchers spell it out for Novartis


Kenneth I. Ataga MD, UNC Chapel Hill

Two weeks ago, Novartis stepped in with a deal to buy Selexys Pharmaceuticals and its Phase III-ready sickle cell disease drug crizanlizumab, an anti-P-selectin antibody designed to slash the number of vaso-occlusive pain crises among patients. New Phase II data was promised for ASH this weekend, so we already knew the data were positive.

Today we got some of the details, and they look good for Novartis’ new drug (originally optioned in 2012), at least for the high dose group.

These pain crises occur when sickle-shaped cells block the flow of blood in small blood vessels. And it’s the most obvious symptom of the disease.

Outlining the response in the high-dose arm, investigators reported in the New England Journal of Medicine the median rate of crises per year was “1.63 with high-dose crizanlizumab versus 2.98 with placebo (indicating a 45.3% lower rate with high-dose crizanlizumab, P=0.01). The median time to the first crisis was significantly longer with high-dose crizanlizumab than with placebo (4.07 vs. 1.38 months, P=0.001), as was the median time to the second crisis (10.32 vs. 5.09 months, P=0.02). The median rate of uncomplicated crises per year was 1.08 with high-dose crizanlizumab, as compared with 2.91 with placebo (indicating a 62.9% lower rate with high-dose crizanlizumab, P=0.02).”

The low dose did not deliver a statistically significant response.

Novartis will look to see if it can upgrade physicians’ response to sickle cell crises with this drug. And in the meantime, some of the researchers involved believe that the treatment could do better than simply relieve pain.

Kenneth I. Ataga, MD, professor of medicine and director of the comprehensive sickle cell program at University of North Carolina at Chapel Hill, told HemOnc Today. (“M)y assumption is … ultimately we may find a drug like this could actually improve survival.”

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