The acquisition adds to the company’s growing pipeline with the addition of clinical-stage SYNT001, a humanized monoclonal antibody that inhibits the interaction of neonatal Fc receptor (FcRn) with Immunoglobulin G (IgG) and IgG immune complexes with the potential to treat a number of rare IgG-mediated diseases.
Alexion CEO Ludwig Hantson said: “We’ve made significant progress rebuilding our pipeline this year, and the acquisition of Syntimmune is another critical step in continuing to expand and diversify our portfolio.
“We’re very excited to continue the development of SYNT001, which we believe holds great promise in transforming patient care across a number of rare IgG-mediated diseases. We are rapidly advancing the current development programs and look forward to beginning two pivotal programs next year, including one in warm autoimmune hemolytic anemia.”
SYNT001 is currently being evaluated in Phase 1b/2a studies in patients with warm autoimmune hemolytic anemia (WAIHA) and in patients with pemphigus vulgaris (PV) or pemphigus foliaceus (PF) and has demonstrated proof of mechanism showing rapid IgG reduction.
Alexion plans to initiate two pivotal trials in 2019 – one in WAIHA, following successful completion of the current Phase 1b/2a study, and one in an undisclosed indication.
About FcRn
Antibodies play an important role in a healthy body’s defense by fighting infections from bacteria and other invaders. In autoimmune diseases, however, the body mistakenly attacks itself through the production of pathogenic (disease-causing) antibodies of the Immunoglobulin G (IgG) subtype. Neonatal Fc receptor (FcRn) rescues IgGs from lysosomal degradation by binding them to endosomes and returning them to the bloodstream. This helps prolong the half-life of IgG. In healthy individuals, this function contributes to a normal immune response. In many autoimmune conditions, however, FcRn prevents lysosomal degradation of pathogenic IgGs associated with driving the disease. Therefore, blocking the FcRn-IgG interaction has the potential to drive degradation of IgG within cells and rapidly reduce circulating pathogenic IgG.
About WAIHA
Warm autoimmune hemolytic anemia (WAIHA) is a rare autoimmune disorder caused by pathogenic Immunoglobulin G (IgG) antibodies that react with and cause the premature destruction of red blood cells at normal body temperature. The disease is often characterized by profound, and potentially life-threatening anemia and other acute complications, including severe and life-threatening hemolysis, severe weakness, enlarged spleen and/or liver, rapid heart rate (tachycardia), chest pain, heart failure and fainting (syncope). There are approximately 65,000 patients across the United States, France, Germany, Italy, Spain and the United Kingdom. There are currently no approved treatments for WAIHA.
About SYNT001
SYNT001 is an investigational humanized IgG4 monoclonal antibody optimized to inhibit FcRn binding to IgG at both neutral and acidic pH. Studies have shown that SYNT001 rapidly facilitates clearance of IgG and IgG circulating immune complexes (CICs), with the potential to block innate immune responses induced by IgG and CIC, as well as inhibit T-cell and B-cell activation in response to CIC. Additionally, studies suggest that SYNT001 accomplishes its effects on IgG without destroying immune cells or impacting other types of immunoglobulin. SYNT001 has the potential to exert a rapid therapeutic effect in a wide range of IgG-mediated autoimmune diseases.
Source: Company Press Release