argenx(US:ARGX)2021-05-14 18:28Business Overview - argenx aims to reach patients through immunology innovation, focusing on late-stage pipeline development and commercial franchises[3] - The company has a strong balance sheet with a pro-forma cash position of approximately $3.1 billion[3] - Efgartigimod has achieved proof-of-concept in 4 out of 4 indications, including 2 out of 2 in the neuromuscular franchise[13] Efgartigimod Development - A bridging study (n=50) is underway to support registration of subcutaneous efgartigimod, demonstrating non-inferiority of pharmacodynamic effect of 1000 mg SC efgartigimod to 10mg/kg IV efgartigimod[31] - In Myasthenia Gravis (MG), a person with MG on a good day operates at 70%, while on bad days, they can get 10% out of their battery[22] - Approximately half (1/2) of MG patients have been diagnosed with depression or anxiety in addition to gMG[22] Pipeline and Programs - Cusatuzumab is being developed for newly diagnosed elderly AML patients who are unfit for intensive chemotherapy[59] - In the Phase 2 CULMINATE trial of Cusatuzumab + Azacitidine 20 mg/kg, the 30-day mortality rate was 9.6% (5/52)[67] - ARGX-117, targeting the innate immune system, is in Phase 1 with healthy volunteer data expected mid-2021[55] Regulatory and Global Expansion - The BLA for IV efgartigimod for the treatment of gMG has been accepted for review by the FDA, with a PDUFA date of December 17, 2021[47]