On October 24, 2019 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, reported its financial results for the third quarter ended September 30, 2019 and provided a business update (Press release, argenx, OCT 24, 2019, View Source [SID1234542472]).
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"We have had another strong quarter of execution across our development pipeline and look forward to a busy remainder of the year that should set the stage for an exciting 2020. With efgartigimod, we are planning for three upcoming readouts, including Phase 3 ADAPT data in generalized myasthenia gravis, which remains on track with enrollment and for data readout in the second half of 2020; Phase 2 data in pemphigus vulgaris in the first half of 2020; and Phase 1 healthy volunteer data from the ENHANZE SC formulation of efgartigimod by the end of this year, which will guide our subcutaneuous development path forward. In tandem, we plan to initiate new trials of efgartigimod, cusatuzumab and ARGX-117 as we continue to build a multitude of opportunities across our pipeline and strengthen our focus within important therapeutic franchises," commented Tim Van Hauwermeiren, CEO of argenx.
THIRD QUARTER 2019 AND RECENT HIGHLIGHTS
argenx is executing on its "argenx 2021" vision to become a fully integrated, global immunology company, which includes its anticipated first commercial launch of efgartigimod in generalized myasthenia gravis (gMG) in 2021 and the building of two potential commercial franchises in neuromuscular and hematology with its three core assets: efgartigimod, cusatuzumab and ARGX-117.
Efgartigimod: First-in-class opportunity with potential across range of high-value autoimmune indications
Efgartigimod is a human IgG1 Fc fragment engineered for optimal blocking of FcRn. Treatment with efgartigimod is intended to result in a targeted reduction of IgG autoantibodies. argenx is evaluating efgartigimod across four indications where IgG autoantibodies are directly pathogenic, including:
· Generalized Myasthenia Gravis
· Global, multi-center Phase 3 ADAPT clinical trial, including ADAPT+ one-year open-label extension study, currently ongoing
· Based on current enrollment, argenx continues to expect topline data from ADAPT in second half of 2020
· Primary Immune Thrombocytopenia (ITP)
· Global Phase 3 program to include two registrational trials to be run concurrently
· First trial, ADVANCE, on track to start in second half of 2019 and will evaluate 10mg/kg intravenous (IV) efgartigimod on top of standard of care medication
· Second trial to evaluate 10mg/kg IV efgartigimod to induce IgG antibody reduction and clinical response followed by fixed dose 330mg subcutaneous (SC) injectable efgartigimod to maintain clinical benefit
· Pemphigus Vulgaris (PV)
· Interim data from ongoing Phase 2 proof-of-concept clinical trial expected in first half of 2020
· Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
· Phase 2 proof-of-concept clinical trial on track to start in second half of 2019
· Key opinion leader (KOL) event planned for December 5, 2019 to present Phase 2 trial design and market opportunity in CIDP
argenx is developing three formulations of efgartigimod to address the needs of patients, physicians and payors across indications and geographies, including IV efgartigimod and two SC formulations.
· A standalone SC formulation of efgartigimod as part of argenx’s collaboration with Halozyme enabling co-formulation of efgartigimod with Halozyme’s proprietary ENHANZE drug delivery technology
· Data from Phase 1 healthy volunteer (HV) trial expected by end of 2019
· After Phase 1 HV trial data are available, argenx to disclose path forward for testing in patients for ENHANZE SC formulation of efgartigimod, including potential bridging strategy in gMG
· argenx has exclusive access to ENHANZE technology for FcRn target
·A fixed 330mg SC injection to be dosed as maintenance treatment following IV induction; this formulation to be evaluated in a second Phase 3 ITP trial
Cusatuzumab: First-in-class opportunity with potential in hematological malignancies
Cusatuzumab is an anti-CD70 monoclonal antibody being developed under an exclusive global collaboration and license agreement with Janssen for the treatment of acute myeloid leukemia (AML), high-risk myelodysplastic syndromes and other hematological malignancies.
·Phase 2 and registration-directed clinical trial of cusatuzumab currently enrolling up to 150 patients with previously untreated AML who are not eligible for intensive chemotherapy
· In two-part trial, patients will first be randomized to receive one of two dose levels of cusatuzumab (10mg/kg and 20mg/kg) in combination with azacytidine (75mg/m2) followed by an expansion cohort to evaluate efficacy of the selected dose of cusatuzumab
ARGX-117: First-in-class anti-C2 antibody expected to enter clinic in first quarter 2020
ARGX-117 is a complement-targeting antibody against C2 with potential therapeutic applications in multiple autoimmune diseases. A Clinical Trial Application (CTA) is on track to be filed by end of 2019 with first-in-human trial expected to start in first quarter of 2020.
UPCOMING MILESTONES
·Before the end of 2019, argenx expects:
Data from Phase 1 HV trial of ENHANZE SC formulation of efgartigimod after which argenx will communicate on path forward for testing in patients with this co-formulation
· Start of first Phase 3 ADVANCE trial of efgartigimod in primary ITP
· Start of Phase 2 proof-of-concept trial of efgartigimod in CIDP
· In 2020, argenx expects:
· Data from Phase 2 proof-of-concept trial of efgartigimod in PV in first half
· First-in-human Phase 1 trial to start with ARGX-117 in first quarter
· Data from global Phase 3 ADAPT trial of efgartigimod in gMG in second half
·Announcement of fifth indication with efgartigimod and new pipeline asset ARGX-119
Update from ongoing cusatuzumab development
FINANCIAL OUTLOOK
Based on current development plan, argenx expects that its cash, cash equivalents and investments will fund planned operating and capital expense requirements up to the expected launch of efgartigimod in gMG in 2021. This development plan excludes any potential upcoming milestone payments under existing collaborations.
With the advancement of development of efgartigimod across indications, build-out of the argenx commercial organization, commitment to fund 40% of the development plan for cusatuzumab, and the expansion of its business plan, argenx expects operating and capital expense requirements to continue to increase year-over-year.