Data presented at ASH from pivotal phase II/III trial of emapalumab-lzsg in primary haemophagocytic lymphohistiocytosis.- SOBI + Novimmune

Swedish Orphan Biovitrum AB (publ) (Sobi�) and Novimmune SA presented data from the pivotal phase II/III clinical study of emapalumab-lzsg in primary haemophagocytic lymphohistiocytosis (HLH) as part of the late-breaking abstract session at the 60th Annual Meeting of the American Society of Hematology (ASH), taking place 1-4 December 2018, in San Diego.

�Primary HLH is an aggressive hyper-inflammatory syndrome that can quickly become fatal if not treated. This study demonstrated that emapalumab treatment induced rapid and sustained responses, helping these fragile and often very young patients control HLH activity and reach stem cell transplant, which is the only cure for this devastating disease, potentially without the side effects of prolonged chemotherapy. Further, the 90 per cent survival post-transplant in this study is very encouraging, particularly given the high morbidity and mortality associated with primary HLH,� said first author and presenter of the late-breaking abstract Professor Franco Locatelli, Head of the Department of Onco-Haematology, Bambino Ges� Children�s Hospital IRCCS, Rome, Italy.

The trial achieved its primary endpoint, with 64.7 per cent of all patients treated (22 of 34; p=0.0031) and 63 per cent of the patients who had failed prior conventional HLH therapy (17 of 27; p=0.0134) demonstrating an overall response at the end of treatment, defined as achievement of either a complete or partial response, or HLH improvement. Across both cohorts, the overall response rate (ORR) was significantly higher than the pre-specified null hypothesis of 40 per cent. The median time to response was eight days, and patients remained in response for a median of 75 per cent of the time.The majority of all patients treated � 64.7 per cent (22 of 34) of the full patient group and 70.4 per cent (19 of 27) of the patients who had failed prior conventional HLH therapy � proceeded to haematopoietic stem cell transplantation (HSCT), with 90.9 per cent (20 of 22) of the full group and 89.5 per cent (17 of 19) of the subgroup surviving post HSCT.

The most common adverse reactions reported during the study were infections (56 per cent), hypertension (41 per cent), infusion-related reactions (27 per cent) and fever (24 per cent).