Breda, the Netherlands / Ghent, Belgium – 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 its fourth quarter business update and full year results for 2018.
The full year results will be discussed during a conference call and webcast presentation today at 3 pm CET/9 am EST. To participate in the conference call, please select your phone number below and use the confirmation code 9795988. The webcast may be accessed on the homepage of the argenx website at www.argenx.com or by clicking here.
“By translating fundamental immunology breakthroughs into novel product candidates, we have built a robust pipeline of differentiated antibodies with the potential to truly impact patients' lives,” commented Tim Van Hauwermeiren, CEO of argenx. “We made great progress in 2018 with our wholly owned lead asset efgartigimod, advancing development across four severe autoimmune indications, in both intravenous and subcutaneous formulations. We signed a transformational global collaboration and license agreement with Janssen to develop cusatuzumab in AML while retaining key commercial rights to the product in the U.S. We also announced that AbbVie exercised its option to in-license ARGX-115 targeting GARP.
“Based on our deep and diversified product candidate portfolio, a solid cash position and an ambitious business plan we are facing an abundance of opportunity ahead in 2019. We will continue to build out our novel pipeline through our Innovative Access Program and our commercial capabilities as we drive enrollment of the Phase 3 ADAPT trial of efgartigimod in gMG and plan for the start of a second Phase 3 trial in ITP.”
FOURTH QUARTER 2018 AND RECENT HIGHLIGHTS
argenx continues to build a deep pipeline of differentiated antibody-based product candidates, including wholly-owned efgartigimod for the treatment of severe autoimmune diseases and cusatuzumab for the treatment of acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS) in collaboration with Janssen. Through its Innovative Access Program, argenx grows its proprietary and partnered pipeline by leveraging its antibody expertise to match fundamental biology breakthroughs at leading research institutions.
Efgartigimod (ARGX-113) Program
• Full Phase 2 immune thrombocytopenia (ITP) trial data presented during American Society of Hematology (ASH) Annual Meeting
o Correlation between IgG antibody reduction, platelet count increase and reduction of bleeding events following efgartigimod treatment.
o Increasing separation between treatment arms and placebo at increasing response thresholds observed, with response rates of 46% and 67% in primary trial and first dosing cycle of open-label extension trial, respectively.
o Primary endpoint analysis revealed efgartigimod to be well-tolerated in all patients consistent with tolerability analysis from Phase 1 healthy volunteer study.
• Orphan drug designation granted in February 2019 by U.S. Food and Drug Administration (FDA) for use for primary ITP.
o argenx additionally received orphan drug designation for efgartigimod in generalized myasthenia gravis (gMG) from FDA and the European Medicines Agency, which was announced, respectively, in September 2017 and March 2018.
• By mid-2019, argenx expects to close its ongoing open-label extension study as part of the completed Phase 2 proof-of-concept trial in ITP in anticipation of the start of a more chronic dosing regimen in its Phase 3 trial.
Cusatuzumab (ARGX-110) Program
• Updated Phase 1/2 AML trial data in combination with azacitidine presented during ASH Annual Meeting.
o Overall response rate (ORR) of 92% in 12 newly diagnosed AML patients unfit for intensive chemotherapy, including 10 patients (91%) with a complete remission with or without hematologic recovery (CR/CRi) and 1 patient (9%) with partial remission (PR).
o Five patients (42%) achieved minimal residual disease (MRD) negativity. Translational data demonstrated that cusatuzumab monotherapy and in combination with azacitidine
significantly reduced leukemic stem cells in the bone marrow of AML patients.
o Well-tolerated with no exacerbation of azacitidine toxicity.
• Orphan drug designation granted by FDA in January 2019 for use of cusatuzumab for AML. Collaborations
• Entered global collaboration and license agreement with Halozyme for ENHANZE® technology (February 2019).
• Gained access to ENHANZE® subcutaneous delivery technology for up to three
targets, including exclusive rights to develop therapeutic products targeting human neonatal Fc receptor FcRn.
• Upfront payment of $30 million paid to Halozyme with potential future payments up to $160 million per selected target subject to achievement of specified development, regulatory and sales-based milestones.
• Entered exclusive global collaboration and license agreement with Janssen for cusatuzumab
• Deal totaling up to $1.6 billion, plus royalties ranging from the low double digits to the high teens, to develop cusatuzumab in AML, MDS and other hematological malignancies.
• Received $300 million upfront cash payment, and Johnson & Johnson Innovation
made equity investment of €176.7 million ($200.0 million based on exchange rate on date of signing) in argenx, representing 4.68% of then-outstanding share capital.
• argenx retains right to co-promote cusatuzumab in the U.S. and share such royalties with Janssen on a 50-50 basis.
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