Novartis SEG101 (crizanlizumab, formerly SelG1) significantly reduces frequency of sickle cell pain crises in Phase II study - Seite 2

"Patients have long been in need of a new therapy for treatment of SCPC, 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 SCPC since hydroxyurea was approved for use in sickle cell anemia about 20 years ago[2]."

Despite its availability, hydroxyurea often is not utilized primarily due to concerns about patient compliance and potential adverse events[3],[4]. 

About the SUSTAIN trial
The SUSTAIN trial was a multicenter, multinational, randomized, placebo-controlled, double-blind, 12-month study to assess safety and efficacy of the anti-P-selectin antibody SEG101 with or without hydroxyurea therapy in sickle cell disease patients with sickle cell-related pain crises. Patients included in the study had a history of 2 to 10 pain crises in the previous 12 months. Patients receiving hydroxyurea or erythropoietin were included if prescribed for the preceding 6 months and dose was stable for at least 3 months. The trial randomized 198 patients age 16 to 65 to receive high dose SEG101, low dose SEG101 or placebo[1].

Adverse events that occurred in 5% or more of patients in an active dose group and were elevated over placebo by at least 2-fold were arthralgia, pruritus, vomiting, chest pain, diarrhea, road traffic accident, fatigue, myalgia, musculoskeletal chest pain, abdominal pain, influenza and oropharyngeal pain. There were no apparent increases in infections with SeG101 treatment. Five deaths occurred during the study, 2 at 5.0 mg/kg, 1 at 2.5 mg/kg and 2 in placebo; no deaths were deemed related to the study drug[1].

About SEG101 (crizanlizumab)
SEG101 (crizanlizumab, formerly SelG1) is a humanized anti-P-selectin monoclonal antibody that binds a molecule called P-selectin on the surface of endothelial cells and platelets in the blood vessels, causing a blockade of P-selectin[1],[5]. P-selectin drives the vaso-occlusive process[1],[6].  Vaso-occlusive crises, also known as SCPC, occur episodically when sickle-shaped red blood cells block blood flow through blood vessels[7]. The therapeutic blockade of P-selectin can prevent painful vaso-occlusion in small blood vessels and maintain blood flow[1],[7].