Novartis has announced results from Phase II Sustain study for treatment of sickle cell-related pain crises (SCPC).
Results shown 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, explained Novartis.
Vaso-occlusive crises, also known as sickle cell-related pain crises, occur episodically when sickle-shaped red blood cells block blood flow through blood vessels. The therapeutic blockade of P-selectin can prevent painful vaso-occlusion in small blood vessels and maintain blood flow.
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 sickle cell-related pain crises 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 sickle cell-related pain crises 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 sickle cell-related pain crises vs. placebo was 2.9 times longer (medians of 4.07 vs. 1.38 months, p = 0.001) and time to second sickle cell-related pain crises was 2.0 times longer than placebo (medians of 10.32 vs. 5.09 months, p = 0.022).
“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 I. Ataga, M.D., Division of Hematology/Oncology, University of North Carolina, Chapel Hill. “These findings show that crizanlizumab significantly reduces the frequency of painful crises and represents a potentially novel disease-modifying therapeutic option.”
“Patients have long been in need of a new therapy for treatment of sickle cell-related pain crises, the most common and debilitating complication of sickle cell disease,” said Bruno Strigini, CEO of Novartis Oncology. “We are pleased that data from the SUSTAIN study show SEG101 may have the potential to become the first new option for patients dealing with sickle cell-related pain crises since hydroxyurea was approved for use in sickle cell anemia about 20 years ago.”