Annual Report 2022

Annual Report 2022

2022 In Brief

In 2022, we executed on our global launch of VYVGART® (efgartigimod alfa) (VYVGART) our first-in-class neonatal Fc receptor (FcRn) blocker, which is now approved in the U.S., Japan and Europe. There are now over 3,000 people globally living with gMG who are on VYVGART.

gMG is just the beginning and efgartigimod is now being evaluated in ten autoimmune indications with three more to start in 2023. We announced positive Phase 3 data from ADAPT-SC evaluating subcutaneous (SC) efgartigimod in gMG and also from ADVANCE evaluating VYGART (intravenous (IV)) in ITP. We are well on our way to achieve our ‘argenx 2025’ vision of efgartigimod either being commercially available or in clinical development in fifteen indications by 2025.

We are advancing a pipeline of differentiated immunology assets across four commercial franchises including neurology, hematology/rheumatology, dermatology and nephrology. Our wholly-owned pipeline currently consists of efgartigimod, ARGX-117 targeting component 2 (C2) and ARGX-119 targeting muscle-specific kinase (MuSK). Our core mission is innovation, and we continue to invest in our immunology innovation program (IIP) from which we drive pipeline expansion by collaborating with leading disease biologists who are researching first-in-class disease targets or pathways. We believe that our IIP has a track record of success and eight programs have demonstrated human proof-of-concept since our inception.

The infinity sign symbolizes process of our commitment that every year we try to devlop best solutions for our patients and moves us forward

Global Efgartigimod Launch

Generated global net product VYVGART revenue of $400.7 million in 2022.

VYVGART was approved for the treatment of gMG in the U.S., Japan and Europe:

  • On December 17, 2021, the U.S. Food and Drug Administration (FDA) approved VYVGART for the treatment of gMG in adult patients who are anti-acetylcholine receptor antibody positive (AChR.AB+). The U.S. commercial launch was initiated in January 2022.
  • On January 20, 2022, Japan’s Ministry of Health, Labour and Welfare (MHLW) approved VYVGART (efgartigimod alfa) for the treatment of adult patients with gMG who do not have sufficient response to steroids or non-steroidal immunosuppressive therapies (ISTs). The Japan commercial launch was initiated in May 2022.
  • On August 11, 2022, the EU Commission granted marketing authorization for VYVGART (efgartigimod alfa-fcab) as an add-on to standard therapy for the treatment of adult patients with gMG who are AChR-AB+. The approval is applicable in all 27 European Union (EU) Member States, Iceland, Norway and Liechtenstein. The commercial launch in Germany was initiated in September 2022.

In addition, in 2022, we made the following progress towards our global launch of VYVGART:

  • argenx Canada was established in February 2022 in preparation for a potential Health Canada approval, expected in the third quarter of 2023, and, if granted, commercial launch in Canada.
  • argenx UK Ltd. was established in August 2022 in preparation for launch in the United Kingdom (UK).
  • argenx Netherlands Services B.V. was established in September 2022 in preparation for launch in the Netherlands.
  • argenx Italy S.r.l. incorporated under the laws of Italy, having its registered office in Milan, Italy and its address at Largo Francesco Richini 6, 20122 Milan, Italy.
  • Zai Lab Ltd (Zai Lab) filed for approval of efgartigimod in the People’s Republic of China (PRC) in the second quarter of 2022. The approval decision is expected in 2023.
  • Medison Pharma Ltd (Medison) filed for approval of efgartigimod in Israel in the second quarter of 2022. The approval decision expected in 2023.
  • Additional distribution partnership agreements for other territories were announced in 2022 to further expand global patient reach. For example, we entered into VYVGART commercial and distribution agreements with Medison in Central and Eastern Europe and with Genpharm Services FZ-LLC (Genpharm) for the Gulf Cooperation Council, comprising Saudi Arabia, Kuwait, the United Arab Emirates, Qatar, Bahrain and Oman (collectively, the GCC).
  • Expanded large-scale manufacturing capabilities and capacity through collaboration with FUJIFILM Diosynth Biotechnologies Denmark ApS (Fujifilm) based in Hillerød, Denmark, to provide large-scale drug substance manufacturing of efgartigimod (in addition to Lonza Sales AG (Lonza)).
See Our Products and Product Candidates

Pipeline of Differentiated Antibody Candidates

Click to explore

Efgartigimod

Efgartigimod is our first-in-class neonatal FcRn blocker. We expect our leadership in FcRn blockade to expand to include thirteen autoimmune indications in the pipeline by the end of 2023, including in our commercial franchises listed below:

ARGX-117 (C2 inhibitor):

ARDA: Phase 2 proof-of-concept clinical trial of ARGX-117 in multifocal motor neuropathy (MMN) ongoing with interim results expected in mid-2023.

Phase 2 proof-of-concept clinical trial to start in delayed graft function (DGF) in the second half of 2023.

DM was announced as the third indication for ARGX-117.

ARGX-119 (muscle-specific tyrosine kinase (MuSK) agonist):

Phase 1 dose-escalation trials in healthy volunteers started in the first quarter of 2023 with subsequent Phase 1b clinical trial to assess early signal detection in patients.

Leo Pharma

LEO Pharma exercised it’s exclusive, worldwide option to ARGX-112 targeting IL22 receptor, which triggered a €5.0 million payment to us.

Creation of OncoVerity, Inc. (OncoVerity):

argenx, the University of Colorado Anschutz Medical Campus and the University of Colorado Health (UCHealth) created an asset-centric spin-off, OncoVerity, focused on optimizing and advancing the development of cusatuzumab, an anti-CD70 antibody, in acute myeloid leukemia (AML). OncoVerity is an entity of co-creation, combining the extensive translational biology insights from Dr. Clayton Smith, M.D. from the University of Colorado with the experience from argenx on the CD70/CD27 pathway. OncoVerity is the fourth spin-off company from our IIP.

Neurology franchise:
  • ADAPT-SC: Positive topline data of SC efgartigimod, announced on March 22, 2022, followed by acceptance of a biologics license application (BLA) by the FDA for SC efgartigimod for gMG in adult patients. The BLA was granted a Prescription Drug User Fee Act (PDUFA) target action date that was recently extended by three months to June 20, 2023.
  • ADHERE: Topline data from registrational ADHERE clinical trial of SC efgartigimod for chronic inflammatory demyelinating polyneuropathy (CIDP) expected in thesecond quarter of 2023.
  • ALKIVIA: Registrational Phase 2/3 ALKIVIA clinical trial ongoing of SC efgartigimod for three subtypes of idiopathic inflammatory myopathies (Myositis), including immune-mediated necrotizing myopathy (IMNM), anti-synthetase syndrome (ASyS) and dermatomyositis (DM), with an analysis planned for the Phase 2 portion of the clinical trial including 30 patients of each subtype.
  • Thyroid eye disease (TED): Registrational clinical trial to start in TED in the fourth quarter of 2023.
  • gMG data from our neuromuscular franchise presented during the American Association of Neuromuscular and Electrodiagnostic Medicine annual meeting and Myasthenia Gravis Foundation of America scientific session on September 21, 2022, including new data analyses from ADAPT+ and real-world case studies on the adult AChR antibody negative gMG patient population.
    • Hematology/rheumatology franchise:
  • ADVANCE: Positive topline data of VYVGART for ITP, announced on May 5, 2022.
  • ADVANCE-SC: Topline data from the second registrational ADVANCE-SC clinical trial of SC efgartigimod for primary ITP expected in the second half of 2023.
  • Sjögren’s syndrome (Primary SjS): Phase 2 proof-of-concept clinical trial ongoing through partnership with IQVIA Ltd (IQVIA) with topline results expected in 2024.
  • Post-COVID-19 Postural Orthostatic Tachycardia Syndrome (PC-POTS): Phase 2 proof-of-concept clinical trial ongoing through partnership with IQVIA with topline results expected in fourth quarter of 2023.
  • Anti-neutrophil cytoplasmic antibody-associated vasculitis (AV): Phase 2 proof-of-concept clinical trial to start in fourth quarter of 2023.
    • Dermatology franchise:
  • ADDRESS: Topline data from registrational ADDRESS trial of SC efgartigimod for pemphigus vulgaris (PV) and pemphigus foliaceus (PF) expected in the second half of 2023.
  • BALLAD: Registrational Phase 2/3 BALLAD trial of SC efgartigimod in bullous pemphigoid (BP) ongoing with interim results after the first 40 patients expected in 2024.
  • Novel translational data from the open-label Phase 2 clinical trial of efgartigimod for the treatment of PV and PF that further support the potential role of FcRn blockade and potential of efgartigimod in autoimmune skin blistering disorders were published in the journal Frontiers of Immunology and presented during the Society for Investigative Dermatology Annual Meeting in May 2022.
    • Nephrology franchise
  • Phase 2 proof-of-concept clinical trial ongoing through partnership with Zai Lab. Membranous Nephrology (MN).
  • Phase 2 proof-of-concept clinical trial ongoing through partnership with Zai Lab. Lupus Nephritis (LN).
  • Antibody-mediated rejection (AMR): Phase 2 proof-of-concept clinical trial to start in the fourth quarter of 2023.

    • Corporate Achievements

    Board of Directors

    Appointment of Camilla Sylvest and Ana Cespedes in 2022, and Steve Krognes in the first quarter as non-executive directors to our board of directors (Board of Directors).

    See Board of Directors

    843

    Employees

    Expansion to 843 employees (as of December 31, 2022) to support further growth of our business, including fully staffed commercial teams in the U.S., Europe and Japan.

    See Employees

    Hans de Haard

    Prof. Hans de Haard, our chief scientific officer, retired effective January 1, 2023 and transitioned to consult within our IIP and as a strategic advisor to the research and development committee of our Board of Directors. Peter Ulrichts, Ph.D., our former head of clinical science, assumed the chief scientific officer role.

    See Senior Management

    Keith Woods

    Keith Woods, our chief operating officer, was succeeded as chief operating officer by Karen Massey effective March 13, 2023. Mr. Woods will transition to serve as an advisor to our Board of Directors.

    See Senior Management

    Financial Highlights

    $ 400.7

    million

    Global net product VYVGART revenue

    $ 720.3

    million

    Operating loss

    $ 2.2

    billion

    Cash

    (cash, cash-equivalents and current financial assets) enabling execution of our ambitious strategy objectives.

    $ 804.1

    million

    Raised

    In gross proceeds in global offering of 2,683,334 ordinary shares (including ordinary shares represented by American Depositary Shares (ADSs)), which included the full exercise of the underwriters’ option to purchase 350,000 additional ADSs

    $ 709.6

    million

    Loss

    See our Financial Review