Publication date: 06/06/2018
argenx (Euronext & Nasdaq: ARGX), a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, today announced the receipt of guidance from the U.S. Food & Drug Administration (FDA) following an End-of-Phase 2 meeting. The company has identified the key elements of the trial design and CMC framework of the Phase 3 program to support a Biologics License Application (BLA) for efgartigimod in generalized myasthenia gravis (gMG).
argenx expects to initiate a global pivotal Phase 3 clinical trial of efgartigimod in gMG before the end of 2018. The placebo-controlled 26-week trial is expected to evaluate the efficacy of a 10 mg/kg dose of efgartigimod in approximately 150 gMG patients, including both AChR autoantibody positive and AChR autoantibody negative patients. In addition, patients can roll over into an open-label extension study for a period of one year.
"The outcome of the End-of-Phase 2 meeting is an important step in our strategic plan to advance efgartigimod in gMG patients. We plan to proceed with one study and one dose for our path to approval, and to include AChR autoantibody negative patients in our recruitment plan as this subset represents a particular high unmet need among the MG population," commented Nicolas Leupin, CMO of argenx. "We believe our Phase 3 clinical trial, in combination with the positive Phase 2 data, has the potential to support a BLA submission. We will continue to work very closely with the regulatory authorities as we advance efgartigimod towards approval to help patients suffering from this severe autoimmune disease."
Efgartigimod (ARGX-113) is an investigational therapy for IgG-mediated autoimmune diseases and was designed to exploit the natural interaction between IgG antibodies and the recycling receptor FcRn. Efgartigimod is the Fc-portion of an antibody that has been modified by the argenx proprietary ABDEG(TM) technology to increase its affinity for FcRn beyond that of normal IgG antibodies. As a result, efgartigimod blocks antibody recycling through FcRn binding and leads to fast depletion of the autoimmune disease-causing IgG autoantibodies. The development work on efgartigimod is done in close collaboration with Prof. E. Sally Ward (University of Texas Southwestern Medical and Texas A&M University Health Science Center, a part of Texas A&M University (TAMHSC)).
argenx is a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe auto-immune diseases and cancer. We are focused on developing product candidates with the potential to be either first-in-class against novel targets or best-in-class against known, but complex, targets in order to treat diseases with a significant unmet medical need. Our ability to execute on this focus is enabled by our suite of differentiated technologies. Our SIMPLE AntibodyTM Platform, based on the powerful llama immune system, allows us to exploit novel and complex targets, and our three antibody engineering technologies are designed to enable us to expand the therapeutic index of our product candidates.