•Enrollment on track for Phase 3 ADAPT trial of efgartigimod (ARGX-113) in generalized myasthenia gravis (gMG) patients with topline results expected in 2H20
•First of two registration trials from Phase 3 program of efgartigimod in primary immune thrombocytopenia (ITP) patients expected to start in 2H19
•First patient dosed in Phase 1 healthy volunteer (HV) trial evaluating ENHANZE® subcutaneous (SC) formulation of efgartigimod Management to host conference call today at 3:00 pm CEST (9:00 am ET); details provided below
August 1, 2019
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 financial results for the first half of 2019 and provided its second quarter business update and the outlook for the remainder of the year.
"We continue to execute on accelerating and expanding our robust, late-stage innovative clinical development programs, as we invest in forward integration across our organization to maximize value. Efgartigimod is the most advanced and also the broadest FcRn antagonist program with four indications and both intravenous and subcutaneous formulations. Enrollment for the Phase 3 ADAPT trial in generalized myasthenia gravis is progressing as planned, and we are on track to initiate before the end of the year the first of two pivotal Phase 3 trials, ADVANCE, for our global primary immune thrombocytopenia program, as well as a Phase 2 trial in chronic inflammatory demyelinating polyneuropathy. Our Phase 2 trial of efgartigimod in pemphigus vulgaris remains a priority, and we expect to report topline data in the first half of 2020," commented Tim Van Hauwermeiren, CEO of argenx. "As we move towards becoming a fully-integrated biotechnology company, we continue to invest in commercial infrastructure with the build-out of our neuromuscular and hematology franchises and the expansion of our global supply chain through our longstanding collaboration with Lonza, which is expected to support the commercial launch of efgartigimod in generalized myasthenia gravis in 2021."
SECOND QUARTER 2019 AND RECENT HIGHLIGHTS
During its 2019 R&D Day in May, argenx announced its plan to become a fully integrated, global immunology company in accordance with its “argenx 2021” vision, which includes building two successful commercial franchises in neuromuscular and hematological disorders.
Efgartigimod (ARGX-113): Potential to be best-in-class with broad applicability
Efgartigimod is a human IgG1 Fc fragment engineered to increase affinity for FcRn versus endogenous IgG, whilst preserving characteristic pH-dependent binding, which may contribute to efgartigimod’s relatively long serum half-life and pharmacodynamic effect, and may promote tissue penetration. Treatment with efgartigimod results in a targeted reduction of IgG autoantibodies and is a rational approach to diseases where IgGs are directly pathogenic. argenx is evaluating efgartigimod as a potential treatment for four high-value indications, including:
•Generalized Myasthenia Gravis (gMG)
o Global, multi-center Phase 3 ADAPT clinical trial, including ADAPT+ one-year open-label extension study, currently ongoing
o With current enrollment on track, topline data are expected in second half of 2020
o Results from completed Phase 2 clinical trial were published in Neurology
•?Primary Immune Thrombocytopenia (ITP)
o Global Phase 3 program to include two registration trials that will be run concurrently
? First trial (ADVANCE) to evaluate 10 mg/kg intravenous (IV) efgartigimod on top of standard of care medication, with enrollment up to 158 patients; primary endpoint includes achieving sustained platelet count response of at least 50×109/L
? Second trial to evaluate 10 mg/kg IV induction period followed by subcutaneous (SC) injections, all on top of standard of care medication, to evaluate potential of SC product to maintain clinical benefit
? Phase 3 program was developed following consultation with key regulatory agencies
o ADVANCE Phase 3 clinical trial expected to start in second half of 2019
•Pemphigus Vulgaris (PV)
o Phase 2 proof-of-concept clinical trial ongoing and currently enrolling patients in third cohort with extended dosing of efgartigimod
o Data from Phase 2 clinical trial expected in first half of 2020
•Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
o Phase 2 clinical trial on track to start in second half of 2019
o Key opinion leader (KOL) event planned for fourth quarter 2019 to discuss Phase 2 trial design and unmet needs in CIDP
?argenx entered into a global collaboration with Halozyme in February 2019 to develop a SC formulation of efgartigimod using Halozyme’s proprietary ENHANZE® drug delivery technology, gaining exclusive rights to the technology for the FcRn target.
•First subject dosed in Phase 1 healthy volunteer (HV) trial evaluating safety, pharmacokinetics, pharmacodynamics and bioavailability of ENHANZE® SC formulation of efgartigimod
•?Initiation of study triggered $5 million milestone payment to Halozyme
•?Data from Phase 1 HV trial are expected by end of 2019 after which argenx will disclose a path forward in patients for ENHANZE® SC formulation of efgartigimod
?Cusatuzumab (ARGX-110): First-in-class opportunity in acute myeloid leukemia (AML)
Cusatuzumab is a first-in-class monoclonal antibody inhibiting CD70, a target that is uniquely present on both leukemic stem cells and AML blasts but not healthy cells. It is being developed under an exclusive global collaboration and license agreement with Janssen for the treatment of AML, high-risk myelodysplastic syndromes and other hematological malignancies.
•Phase 2 and registration-directed clinical trial in AML on track to start in second half of 2019
o Trial to enroll up to 150 patients with previously untreated AML and who are not eligible for intensive chemotherapy
o In this two-part trial, patients will first be randomized to receive one of two dose levels of cusatuzumab (10 mg/kg and 20 mg/kg) in combination with azacytidine (75 mg/m2) followed by an expansion cohort to evaluate efficacy
of the selected dose of cusatuzumab
?Early Development Programs
argenx announced during its R&D Day the expansion of its product pipeline with the addition of two new proprietary therapeutic candidates, ARGX-117 and ARGX-118. Both emerged from argenx’s Innovative Access Program, in which it collaborates closely with disease biology experts, bringing the argenx cutting-edge antibody discovery and engineering technologies to the heart of novel target research.
•ARGX-117 is a complement-targeting antibody against C2, a component of both the classical and lectin pathways in the complement cascade
o Potential therapeutic applications in multiple autoimmune diseases
o argenx exercised its second exclusive license to Halozyme’s ENHANZE® technology for use with this molecule
o Expected to file Clinical Trial Application (CTA) by end of 2019 with first-in-human trial expected to start in first quarter of 2020
•?ARGX-118 is a highly differentiated antibody against Galectin-10, the protein of Charcot-Leyden crystals (CLCs), which play a major role in severe asthma and the persistence of mucus plugs
o Immunology breakthrough in airway inflammation
o SIMPLE Antibody™ observed to have unique crystal-dissolving properties
o Currently in final stages of lead optimization work
o Data were published in Science by argenx collaborator Dr. Bart Lambrecht from VIB Inflammation Research Center supporting role of CLCs and potential of ARGX-118 in airway inflammation
Appointed Wim Parys, M.D. as Chief Medical Officer effective July 1, 2019. Most recently, Dr. Parys served as Head of R&D of the Global Public Health group of Janssen.
Based on the current objectives of the Company’s business plan, argenx expects that its existing cash, cash equivalents and investments will fund planned operating and capital expense requirements into 2021. With the launch of a second global Phase 3 trial for efgartigimod, the execution of the development plan for cusatuzumab, the build-out of the commercial organization, and the expansion of the Company’s ambition level within its growing business plan, argenx expects operating and capital expense requirements to continue to increase year-over-year.
U.S. SEC and Statutory Financial Reporting
argenx’s primary accounting framework is International Financial Reporting Standards (IFRS) as issued by the International Accounting Standards Board (IASB). Unaudited condensed half yearly interim financial statements prepared in accordance with International Accounting Standards IAS 34 Interim Financial Reporting are available on www.argenx.com.
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