Amsterdam, Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced its plan to continue the development of efgartigimod to Phase 3 in adults with primary Sjögren’s disease (SjD), following the analysis of topline data from the Phase 2 RHO study. Detailed results will be presented at a future medical meeting.
“We are excited to be advancing efgartigimod’s development in Sjogren’s disease based on the totality of the data generated from the RHO study,” said Luc Truyen, M.D., Ph.D., Chief Medical Officer of argenx. “Consistent with our indication selection strategy, we confirmed our IgG biology hypothesis with these data, and now have a demonstrated clinical effect across multiple efficacy scales to support proof-of-concept. Sjögren’s disease can be debilitating, predominantly affects women, and given its heterogeneous nature, is often misdiagnosed with its symptoms poorly understood. With no current approved therapies to treat the underlying disease, the unmet need is substantial, and we recognize the opportunity to advance a new potential alternative treatment to these patients.”
The decision to advance the clinical development of efgartigimod in SjD was supported by the safety, efficacy and biomarker results from the study. The observed safety and tolerability profile was consistent with other clinical trials. Efficacy assessments showed a treatment effect across multiple clinical endpoints, which were also consistent with biomarker data.
RHO Study Design
The Phase 2 RHO study was a randomized, double-blinded, placebo-controlled multicenter proof of concept study to evaluate the safety and efficacy of VYVGART in adults with SjD. In order to enter the study, patients needed to test positive for anti-Ro autoantibodies and maintain residual salivary flow. Thirty four patients were randomized 2:1 to receive either efgartigimod or placebo for up to 24 weeks. Multiple endpoints and biomarkers were evaluated in the signal-finding study, including the primary endpoint of CRESS (Composite of Relevant Endpoints for Sjögren’s Syndrome). Within CRESS there are five components spanning: systemic disease activity as measured by the ESSDAI (EULAR Sjogren’s Syndrome Activity Index), patient reported outcomes as measured by the ESSPRI (EULAR Sjögren’s Syndrome Patient Reported Index), tear and salivary gland function and serology. To be a CRESS responder, patients needed to demonstrate a clinically meaningful benefit in at least 3 of the 5 composite items. Additional datapoints were gathered including the clinESSDAI, STAR (Sjogren’s Tool for Assessing Response), biomarker data, and the change in lymphocytic infiltrate levels through parotid biopsies.
About Sjögren’s Disease
Sjögren’s Disease (SjD) is a chronic, slowly progressive inflammatory systemic autoimmune disease characterized by immune-mediated destruction of exocrine glands. SjD can be severely debilitating and have a negative impact on patient quality of life, with common symptoms reported as dry eyes and mouth, fatigue, joint point and impaired cognitive function. In addition, a substantial subset of patients suffer from extraglandular systemic disease. While the presence of anti-Ro and anti-LA IgG autoantibodies are considered a hallmark of disease, the underlying cause of SjD is believed to be multi-factorial, triggered by environmental factors, leading to auto-immunity and chronic inflammation. SjD predominantly impacts women with a 9:1 female:male incidence ratio. Given the heterogeneous nature of the disease, the treatment journey can be challenging with long delays and high rates of misdiagnosis. There are no FDA-approved treatments targeting the disease itself, leaving current treatments to focus primarily on individual symptom management.
About Efgartigimod
Efgartigimod is an antibody fragment designed to reduce pathogenic immunoglobulin G (IgG) antibodies by binding to the neonatal Fc receptor and blocking the IgG recycling process. Efgartigimod is being investigated in several autoimmune diseases known to be mediated by disease-causing IgG antibodies, including neuromuscular disorders, blood disorders, and skin blistering diseases, in both an intravenous and subcutaneous (SC) formulation. Efgartigimod is marketed as VYVGART® for the treatment of generalized myasthenia gravis in more than 30 regions globally and immune thrombocytopenia in Japan.
About argenx
argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first approved neonatal Fc receptor (FcRn) blocker, globally in the U.S., Japan, Israel, the EU, the UK, China and Canada. The Company is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises.
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