Novartis’ Phase II antibody trial reduces frequency of sickle cell pain crises

Results from the Phase II sustain study show that SEG101 (crizanlizumab, formerly SelG1), an anti-P-selectin antibody, reduced the median annual rate of sickle cell-related pain crises by 45.3% compared to placebo (1.63 vs 2.98, p=0.010) in patients with or without hydroxyurea therapy.

“Acute painful episodes, commonly referred to as vaso-occlusive crises, are a substantial cause of morbidity in sickle cell disease with limited treatment options,” said Kenneth Ataga, MD, University of North Carolina. “These findings show that crizanlizumab significantly reduces the frequency of painful crises and represents a potentially novel disease-modifying therapeutic option.”

Sustain study

In the sustain study, patients were assigned to high-dose (5.0 mg/kg), low-dose (2.5 mg/kg) and placebo arms. The study met its primary endpoint, reduction of the annual rate of SCPC in the high-dose arm by 45.3% vs. placebo (medians of 1.63 vs. 2.98, p=0.010).

In the low dose arm, the annual rate of SCPC was reduced by 32.6% vs. placebo (medians of 2.01 vs. 3.0, p = 0.180). For patients in the high dose arm, time to first SCPC vs. placebo was 2.9 times longer (medians of 4.07 vs. 1.38 months, p = 0.001) and time to second SCPC was 2.0 times longer than placebo (medians of 10.32 vs. 5.09 months, p = 0.022).

Despite its availability, hydroxyurea often is not utilised primarily due to concerns about patient compliance and potential adverse events.