2023 In Brief
At the start of 2023, we shared our key drivers for the year to continue our trajectory of value creation. First, we aimed to reach more patients globally with VYVGART, our first-in-class neonatal Fc receptor (FcRn) blocker. VYVGART and VYVGART SC are now approved in more than 30 countries or regions and by the end of 2023, we had treated over 6,000 gMG patients globally with our innovations.
gMG is just the beginning of our mission to transform autoimmunity. Our second key driver was to pioneer the FcRn class of medicines, including broadening the scope of indications we are evaluating with efgartigimod. As of the end of 2023, efgartigimod is approved or under regulatory review in three indications, including gMG, CIDP and primary immune thrombocytopenia (ITP), and is being evaluated in more than 10 additional serious autoimmune indications. We are well on our way to achieve our ‘argenx 2025’ vision of efgartigimod being commercially available or in clinical development in 15 indications by 2025.
Third, we worked to advance our pipeline of differentiated immunology assets. Beyond efgartigimod, our wholly-owned clinical pipeline consists of empasiprubart (ARGX-117) targeting complement component 2 (C2) and ARGX-119 targeting muscle-specific kinase (MuSK). We believe both have potential as a novel treatment modality in multiple serious indications.
The fourth key driver was to build out our innovation ecosystem, serving our core mission to sustainably deliver immunology innovations to the patients who need them. We continue to invest in our IIP from which we drive pipeline expansion by collaborating with leading disease biologists who are researching first-in-class disease targets or pathways. Our IIP has a track record of success and nine programs have been tested in humans 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
VYVGART
Reach More Patients Globally with VYVGART
- VYVGART is now approved in the U.S., Japan, Europe, the UK, Israel, Mainland China and Canada for the treatment of gMG. VYVGART SC is now approved in the U.S., in Europe, the UK and Japan for the treatment of gMG. This makes VYVGART the only gMG treatment available as both an intravenous (IV) and a simple SC injection, providing choice to patients in how and where they are treated. In 2023, we generated product net sales of $1.2 billion
- Pricing and reimbursement discussions for VYVGART and VYVGART SC remain ongoing in multiple jurisdictions, including in several countries in the EU
- We filed for approval of VYVGART for ITP in Japan and a decision is expected in the first quarter of 2024
- A supplemental Biologics License Application (sBLA) for SC efgartigimod for CIDP has been accepted for priority review by the FDA, with a Prescription Drug User Fee Act (PDUFA) target date of June 21, 2024
- We have filed for approval of VYVGART SC in Mainland China and we expect a decision on approval by the end of 2024 through our partnership with Zai Lab
- We entered into a VYVGART commercial and distribution agreement with Handok Inc. (Handok) in South Korea (the Handok Agreement)
- We filed for approval of VYVGART for gMG in several jurisdictions and we are expecting multiple decisions by the end of 2024
Advance Extensive Pipeline
We continue to demonstrate breadth and depth within our immunology pipeline and have advanced multiple pipeline-in-a-product candidates. With efgartigimod, we are furthering our leadership in FcRn and we expect to be approved or in development in 15 autoimmune indications by 2025. Beyond efgartigimod, we are advancing earlier stage pipeline programs, including empasiprubart (C2 inhibitor) with Phase 2 POC clinical trials ongoing in MMN, delayed graft function (DGF) and dermatomyositis (DM). In addition, we expect to initiate Phase 1b/2a clinical trials of ARGX-119, a MuSK agonist, in congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS) in 2024.
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Build out Innovation Ecosystem
- In January 2024, we announced the nomination of four new pipeline candidates, including: ARGX-213 targeting FcRn and furthering argenx’s leadership in this new class of medicine; ARGX-121 and ARGX-220, which are first-in-class targets broadening argenx’s focus across the immune system; and ARGX-109, targeting IL-6, which plays an important role in inflammation. Preclinical work is ongoing in each candidate
- We entered into a collaboration with Genmab A/S (Genmab) to jointly discover, develop and commercialize novel therapeutic antibodies with applications in immunology, as well as in oncology therapeutic areas
Neurology indications:
- ADHERE: following the positive topline results from the ADHERE clinical trial in CIDP, an sBLA for SC efgartigimod for CIDP was submitted on December 21, 2023 and is under review by the FDA with a PDUFA date of June 21, 2024
- Clinical trial met primary endpoint (p=0.000039); SC efgartigimod demonstrated 61% reduction (HR: 0.39 95% CI: 0.25; 0.61) in risk of relapse versus placebo
- ALKIVIA: operationally seamless Phase 2/3 clinical trial is ongoing with SC efgartigimod for three subtypes of idiopathic inflammatory myopathies (Myositis), including immune-mediated necrotizing myopathy (IMNM), anti-synthetase syndrome (ASyS) and dermatomyositis (DM); analysis planned for first 30 patients of each subtype
- Registrational clinical trial in thyroid eye disease (TED) expected to start in first quarter of 2024
Hematology/rheumatology indications:
- ADVANCE-IV: positive clinical trial results formed basis of filing in Japan for ITP; topline results published in The Lancet in September 2023
- ADVANCE-SC: topline data from SC efgartigimod in ITP announced on November 28, 2023
- Primary endpoint was not met (p=0.5081); 13.7% (17/124) of treated patients demonstrated sustained platelet count response compared to 16.2% (11/68) of placebo patients. Secondary endpoints were also not met
- RHO: Phase 2 POC clinical trial in primary Sjögren’s disease (SjD) is ongoing through our partnership with IQVIA Ltd (IQVIA)
- ALPHA: Phase 2 POC clinical trial in postural orthostatic tachycardia syndrome post-COVID-19 (POTS post-COVID-19) ongoing through our partnership with IQVIA
Dermatology indications:
- ADDRESS: announced topline data of SC efgartigimod in pemphigus vulgaris (PV) and pemphigus foliaceus (PF) on December 20, 2023
- Primary endpoint was not met; proportion of PV patients achieving primary endpoint of complete remission on minimal dose of steroids (CRmin) was not significantly different between SC efgartigimod and placebo
- Treatment with SC efgartigimod led to CRmin in 35.5% of patients compared to 30.3% with placebo (p=0.5956). Secondary endpoints were also not met
- BALLAD: in light of ADDRESS results and the comparable biology between PV and bullous pemphigoid (BP), we decided to stop enrollment of BALLAD. We will integrate key learnings from ADDRESS and data from already-enrolled patients in BALLAD and we plan to communicate on a revised development plan before end of 2024
Nephrology indications:
- Membranous Nephrology (MN): Phase 2 POC clinical trial ongoing through our partnership with Zai Lab
- Lupus Nephritis (LN): Phase 2 POC clinical trial ongoing through our partnership with Zai Lab
- Antibody-mediated rejection (AMR): IND submission planned for the second quarter of 2024
Corporate Achievements
Steve Krognes
Mr. Steve Krognes joined our Board of Directors in February 2023 as a non-executive director and chairperson of the audit and compliance committee
See Board of DirectorsJ. Donald deBethizy
Mr. J. Donald deBethizy, who has served as a director since May 2015, was appointed to serve as vice-chairman of the Board of Directors as of February 2023
See Board of DirectorsKaren Massey
Karen Massey joined argenx as chief operating officer (COO) in March 2023 succeeding Keith Woods. Mr. Woods transitioned to serve as strategic advisor to the commercialization committee of the Board of Directors
See Board of Directors1,148
Employees
Expansion to 1,148 full-time employees (as of December 31, 2023) to support further growth of our business, including fully staffed commercial teams in the U.S., Europe, Japan and Canada
See Employees
Financial Highlights
billion
Product net sales
million
Operating loss
billion
Cash
(cash, cash-equivalents and current financial assets) enabling execution of our ambitious strategy objectives
billion
Raised
In gross proceeds in global offering of 2,581,633 ordinary shares (including ordinary shares represented by ADSs, which included the full exercise of the underwriters’ option to purchase 336,734 additional ADSs
million
Loss