UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 6-K
REPORT OF FOREIGN PRIVATE ISSUER
PURSUANT TO RULE 13a-16 OR 15d-16
UNDER THE SECURITIES EXCHANGE ACT OF 1934
For the Month of January 2025
Commission File Number: 001-38097
ARGENX SE
(Translation of registrant’s name into English)
Laarderhoogtweg 25
1101 EB Amsterdam, the Netherlands
(Address of principal executive offices)
Indicate by check mark whether the registrant files or will file annual reports under cover of Form 20-F or Form 40-F.
Form 20-F x Form 40-F ¨
Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(1): ¨
Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(7): ¨
EXPLANATORY NOTE
On January 13, 2025, argenx SE (the “Company”) issued a press release and an investor presentation, copies of which are attached hereto as Exhibits 99.1 and 99.2, respectively, and are incorporated by reference herein.
The information contained in this Current Report on Form 6-K, including Exhibits 99.1 and 99.2, shall be deemed to be incorporated by reference into the Company’s Registration Statements on Forms F-3 (File No. 333-258251) and S-8 (File Nos. 333-225375, 333-258253, and 333-274721), and to be part thereof from the date on which this Current Report on Form 6-K is filed, to the extent not superseded by documents or reports subsequently filed or furnished.
| Exhibit | Description | |
| 99.1 | Press Release dated January 13, 2025 | |
| 99.2 | Investor Presentation dated January 13, 2025 |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
| ARGENX SE | ||
| Date: January 13, 2025 | By: | /s/ Hemamalini (Malini) Moorthy |
Name: Hemamalini (Malini) Moorthy Title: General Counsel | ||
Exhibit 99.1
argenx Highlights 2025 Strategic Priorities
Reported $2.2 billion in preliminary* full-year 2024 global product net sales, inclusive of $737 million in fourth quarter sales
Pre-filled syringe FDA PDUFA on track for April 2025 to support reaching patients earlier in treatment paradigm
10 ongoing registrational studies in 2025 across efgartigimod and empasiprubart enable next wave of indications
Empasiprubart to be evaluated in two head-to-head registrational studies against IVIg to position C2 inhibitor for broad, early-line use in MMN and CIDP
Transition to sustainable profitability in 2025 enables continued investment in innovation
January 13, 2025, 7:00 AM CET
Amsterdam, the Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today reported preliminary financial results for the full-year 2024, including global product net sales, and announced its strategic priorities for 2025.
“2024 was a transformative year as we significantly expanded our global patient reach with VYVGART and advanced a world-class pipeline of precision therapies,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx. “The team’s strong execution has positioned argenx to be a profitable company in 2025, providing us flexibility to invest in the next wave of innovation across the company. Today, we are all in on our innovation mission, applying our successful innovation playbook to bring transformational outcomes to even more patients by unleashing the next wave of autoimmune indications and therapies, and securing our future by advancing multiple programs against first-in-class targets. We are positioned for significant expansion in 2025 with the FDA decision on approval of our pre-filled syringe, global CIDP rollout, and label-expansion studies underway for MG. To further support our growth, we are thrilled to have 10 ongoing registrational and 10 proof-of-concept studies in 2025, teeing us up for several data readouts across our pipeline in the next 12-24 months.
“Innovation is the cornerstone of everything we do, from the foundational science all the way to payor negotiations; it is our goal to deliver innovative and disruptive science for the benefit of patients who need better access to transformational safe, effective, and convenient precision therapies. Innovation has no meaning unless it reaches the marketplace, and we will continue to prioritize patient outcomes in all that we do.”
2025 Strategic Priorities
argenx established its ‘Vision 2030’ to outline the next phase of growth as part of its long-term commitment to transform the treatment of autoimmune diseases. Through this vision, argenx aims to treat 50,000 patients globally with its medicines, secure 10 labeled indications across all approved medicines, and advance five pipeline candidates into Phase 3 development by 2030.
To achieve the goals set out in its ‘Vision 2030’, argenx has set the following priorities for 2025:
| · | Expand the global VYVGART opportunity by reaching more patients broadly across MG, CIDP and ITP through additional regulatory approvals and continuous evidence generation |
| · | Launch VYVGART SC as a pre-filled syringe to innovate on the patient experience and move earlier in the MG and CIDP treatment paradigms |
| · | Execute 10 registrational and 10 proof-of-concept studies to fuel pipeline growth across efgartigimod, empasiprubart and ARGX-119 |
| · | Advance four new molecules into Phase 1 development, expanding the next wave of innovation |
| · | Generate sustainable value through continued investment in the Immunology Innovation Program, focused on first-in-class, antibody-based medicines with pipeline-in-a-product potential |
Expand the global VYVGART opportunity and launch VYVGART SC as a pre-filled syringe
VYVGART® (IV: efgartigimod alfa-fcab and SC: efgartigimod alfa and hyaluronidase-qvfc) is a first-in-class FcRn blocker approved in three indications, including generalized myasthenia gravis (gMG) globally, primary immune thrombocytopenia (ITP) in Japan, and chronic inflammatory demyelinating polyneuropathy (CIDP) in the U.S., Japan, and China.
| · | Regulatory decisions on approval of VYVGART for gMG expected in first half of 2025, including in Israel (SC), South Korea (IV), and Kuwait (IV) |
| · | Continued innovation around the patient experience for VYVGART SC, including four key regulatory decisions on approval expected in 2025 |
| o | FDA review ongoing of pre-filled syringe (PFS) for gMG and CIDP with Prescription Drug User Fee Act (PDUFA) target action date of April 10, 2025 |
| o | PFS decisions on approval for gMG and CIDP expected in Europe in first half of 2025 and Japan and Canada in second half of 2025 |
| o | Autoinjector development underway with launch planned for 2027 |
| · | Evidence generation through Phase 4 and label-enabling studies in MG, CIDP and ITP |
| o | Label-enabling studies ongoing to reach broader MG populations, including ADAPT-SERON (seronegative gMG), ADAPT-JR (pediatric) and ADAPT-OCULUS (ocular MG), with topline results expected in second half of 2025 (SERON) and first half of 2026 (OCULUS and JR) |
| o | Phase 4 switch study ongoing in CIDP to inform treatment decisions when switching a patient on IVIg to VYVGART SC |
| o | ADVANCE-NEXT confirmatory study ongoing of VYVGART IV in primary ITP to support FDA submission with topline results expected in second half of 2026 |
Execute 10 registrational and 10 proof-of-concept studies across efgartigimod, empasiprubart and ARGX-119
argenx continues to demonstrate breadth and depth within its immunology pipeline, advancing multiple first-in-class product candidates with potential across multiple high-need indications. argenx is solidifying its leadership in FcRn biology with efgartigimod, complement inhibition with empasiprubart and in the role of MuSK at the neuromuscular junction with ARGX-119. In 2025, argenx plans to execute 10 registrational and 10 proof-of-concept studies across efgartigimod, empasiprubart and ARGX-119 to advance its next wave of launches.
Efgartigimod Development
Efgartigimod is being evaluated in more than 15 severe autoimmune diseases (including MG, CIDP and ITP), exploring the significance of FcRn biology across neurology and rheumatology indications, as well as new therapeutic areas. To prioritize those indications that can drive transformative benefit, argenx has made the decision to discontinue development of efgartigimod in bullous pemphigoid (BP).
| · | Registrational ALKIVIA study ongoing evaluating three myositis subsets (immune-mediated necrotizing myopathy (IMNM), anti-synthetase syndrome (ASyS), and dermatomyositis (DM); topline results expected in second half of 2026 |
| · | Two registrational UplighTED studies ongoing in thyroid eye disease (TED); topline results expected in second half of 2026 |
| · | Registrational UNITY study ongoing in primary Sjögren’s disease; topline results expected in 2027 |
| · | Decision made to discontinue development in BP based on results from 98 patients in the Phase 2 BALLAD study |
| · | Proof of concept studies ongoing in lupus nephritis (LN), systemic sclerosis (SSc) and antibody mediated rejection (AMR); topline results expected in LN in fourth quarter of 2025, SSc in second half of 2026, and AMR in 2027 |
| · | Two new indications nominated, including autoimmune encephalitis (AIE) and one that will be disclosed later in 2025 |
| · | Externally sponsored research studies ongoing in early MG, MG crisis, Guillain-Barré syndrome (GBS), stiff person syndrome (SPS), and neuromyelitis optica spectrum disorder (NMO-SD) |
Empasiprubart Development
argenx is evaluating empasiprubart in registrational studies in multifocal motor neuropathy (MMN) and CIDP, and proof-of-concept studies in delayed graft function (DGF) and DM.
| · | Registrational EMPASSION study ongoing in MMN evaluating empasiprubart head-to-head versus IVIg; topline results expected in second half of 2026 |
| · | Registrational EMVIGORATE study in CIDP evaluating empasiprubart head-to-head versus IVIg to start in first half of 2025 |
| · | Proof of concept studies ongoing in DGF and DM; topline results expected in DGF in second half of 2025 and in DM in first half of 2026 |
ARGX-119 Development
argenx is evaluating ARGX-119 in congenital myasthenic syndromes (CMS), amyotrophic lateral sclerosis (ALS), and spinal muscular atrophy (SMA).
| · | Phase 1b proof-of-concept study ongoing in CMS; topline results expected in second half of 2025 |
| · | Phase 2a proof-of-concept study ongoing in ALS; topline results expected in first half of 2026 |
| · | SMA nominated as third indication with proof-of-concept study to start in 2025 |
Advance four new pipeline molecules and generate sustainable value through continued investment in Immunology Innovation Program
argenx continues to invest in its Immunology Innovation Program (IIP) to drive long-term sustainable pipeline growth. Through the IIP, four new pipeline candidates have been nominated, including: ARGX- 213, targeting FcRn and further solidifying argenx’s leadership in this new class of medicine; ARGX- 121, a first-in-class molecule targeting IgA; ARGX-109, targeting IL-6, which plays an important role in inflammation, and ARGX-220, a first-in-class sweeping antibody for which the target has not yet been disclosed.
| · | Investigational new drug (IND) applications to be filed in 2025 for ARGX-213, ARGX-121, ARGX-109 and ARGX-220; Phase 1 results expected for ARGX-109 in second half of 2025 and for ARGX-213 and ARGX-121 in first half of 2026 |
Preliminary* Fourth Quarter and Full-Year 2024 Financial Results
Today, argenx also announced preliminary* global product net sales for the fourth quarter and full-year 2024 of approximately $737 million and $2.2 billion, respectively.
As of December 31, 2024, argenx had approximately $3.4 billion in cash, cash equivalents and current financial assets*.
* - The preliminary selected financial information is unaudited, subject to adjustment, and provided as an approximation in advance of the company’s announcement of complete financial results in February 2025. Refer to the Preliminary Financial Results note in this document.
2025 Financial Guidance
Based on its current operating plans, argenx expects its combined R&D and SG&A expenses in 2025 to be approximately $2.5 billion.
43rd Annual J.P. Morgan Healthcare Conference Presentation and Webcast
Tim Van Hauwermeiren will highlight these updates in a corporate presentation at the 43rd Annual J.P. Morgan Healthcare Conference today, Monday, January 13, 2025, at 9:45 a.m. PT. The live webcast of the presentation may be accessed under Investors on the argenx website. A replay will be available for 30 days following the presentation.
About VYVGART and VYVGART SC
VYVGART® is a human IgG1 antibody fragment that binds to the neonatal Fc receptor (FcRn), resulting in the reduction of circulating IgG autoantibodies. It is the first approved FcRn blocker for the treatment of generalized myasthenia gravis (gMG), chronic inflammatory demyelinating polyneuropathy (CIDP), and primary immune thrombocytopenia (ITP). VYVGART SC is a subcutaneous combination of efgartigimod alfa and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE® drug delivery technology to facilitate subcutaneous injection delivery of biologics. It is marketed as VYVGART® Hytrulo in the U.S., VYVGART SC in Europe, VYVDURA® in Japan, and may be marketed under different proprietary names following approval in other regions.
About argenx
argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first approved neonatal Fc receptor (FcRn) blocker, globally in the U.S., Japan, Israel, the EU, the UK, China and Canada. The company is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit www.argenx.com and follow us on LinkedIn, X (formerly known as Twitter), and Instagram.
Media:
Ben Petok
bpetok@argenx.com
Investors:
Alexandra Roy (US)
aroy@argenx.com
Lynn Elton (EU)
lelton@argenx.com
Preliminary Financial Results
The financial information presented in this press release is preliminary, estimated, and unaudited. They are subject to the completion and finalization of argenx’s financial and accounting closing procedures. They reflect management’s estimates based solely upon information available to management as of the date of this press release. Further information learned during that completion and finalization may alter the final results. In addition, the preliminary estimates should not be viewed as a substitute for full quarterly and annual financial statements prepared in accordance with IFRS. There is a possibility that argenx’s financial results for the quarter ended December 31, 2024, and full year financial results for 2024 could vary materially from these preliminary estimates. In addition to the completion of the financial closing procedures, factors that could cause actual results to differ from those described above are set forth below. Accordingly, you should not place undue reliance upon this preliminary information.
Additional information regarding the company’s fourth quarter 2024 financial results and full year financial results for 2024 will be available in the company’s annual report and Form 20-F, which will be filed with the Netherlands Authority for the Financial Markets and U.S. Securities and Exchange Commission (the “SEC”), respectively.
Forward Looking Statements
The contents of this announcement include statements that are, or may be deemed to be, “forward-looking statements.” These forward-looking statements can be identified by the use of forward-looking terminology, including the terms “aim,” “can,” “continue,” “expect,” “goal,” “may,” “ongoing,” “plan,” “possible,” “target,” and “will,” and include statements argenx makes regarding its expected profitability in 2025; its 2025 strategic priorities, including its launch of pre-filled syringes, 10 Phase 3 studies and 10 Phase 2 studies across efgartigimod, empasiprubart and ARGX-119, the advancement of four molecules in Phase 1 studies and the continued investment in the Immunology Innovation Program; its significant expansion in 2025; the continued growth of VYVGART, including its expected autoinjector launch in 2027 and four global decisions in 2025; its expectations regarding the continued growth in CIDP, including its plan to launch multiple CIDP products in 2025 and the expected timing of the EMVIGORATE study; its expectations regarding the growth of the MMN market opportunity, including the expected timing of the EMPASSION study; the anticipated timing of data readouts and regulatory milestones and plans, including the timing of planned clinical trials and regulatory filings and approvals; its vision for 2030, including having 5 new molecules in Phase 3, 10 labeled indications and having 50,000 patients on treatment; the anticipated timing of pending regulatory decisions in Israel, South Korea, Kuwait, the U.S., Europe, Japan and Canada; the expected timing of Phase 4 and label-enabling studies in MG, CIDP and ITP; and its expected 2025 research and development and selling, general and administrative expenses. By their nature, forward-looking statements involve risks and uncertainties and readers are cautioned that any such forward-looking statements are not guarantees of future performance. argenx’s actual results may differ materially from those predicted by the forward-looking statements as a result of various important factors, including the results of argenx's clinical trials; expectations regarding the inherent uncertainties associated with the development of novel drug therapies; preclinical and clinical trial and product development activities and regulatory approval requirements in products and product candidates; the acceptance of argenx's products and product candidates by patients as safe, effective and cost-effective; the impact of governmental laws and regulations on our business; disruptions caused on our reliance of third party suppliers, service providers and manufacturing; inflation and deflation and the corresponding fluctuations in interest rates; and regional instability and conflicts. A further list and description of these risks, uncertainties and other risks can be found in argenx’s U.S. Securities and Exchange Commission (SEC) filings and reports, including in argenx’s most recent annual report on Form 20-F filed with the SEC as well as subsequent filings and reports filed by argenx with the SEC. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this document. argenx undertakes no obligation to publicly update or revise the information in this press release, including any forward-looking statements, except as may be required by law.
| The Next Wave of Innovation 43rd ANNUAL JP MORGAN HEALTHCARE CONFERENCE JANUARY 13, 2025 |
| Forward Looking Statements This presentation has been prepared by argenx se (“argenx” or the “company”) for informational purposes only and not for any other purpose. Nothing contained in this presentation is, or should be construed as, a recommendation, promise or representation by the presenter or the company or any director, employee, agent, or adviser of the company. This presentation does not purport to be all-inclusive or to contain all of the information you may desire. Certain information contained in this presentation relates to or is based on studies, publications, surveys and other data obtained from third-party sources and the company’s own internal estimates and research. While argenx believes these third-party studies, publications, surveys and other data to be reliable as of the date of this presentation, it has not independently verified, and makes no representation as to the adequacy, fairness, accuracy or completeness of, any information obtained from third-party sources. In addition, no independent source has evaluated the reasonableness or accuracy of argenx’s internal estimates or research, and no reliance should be made on any information or statements made in this presentation relating to or based on such internal estimates and research. Certain statements contained in this presentation, other than present and historical facts and conditions independently verifiable at the date hereof, may constitute forward-looking statements. These forward-looking statements can be identified by the use of forward-looking terminology, including the terms “aim,” “continue,” “future,” “ongoing,” “opportunity,” “plan,” and “potential,” and include statements argenx makes regarding its expected profitability in 2025; its 2025 strategic priorities, including its PFS launch, 10 Phase 3 studies and 10 Phase 2 studies, and four molecules in Phase 1 studies; the continued growth of VYVGART Hytrulo, including its expected autoinjector launch in 2027 and four global decisions in 2025; its expectations regarding the increase in the MG Total Addressable Market in the U.S. in 2030; its expectations regarding the continued growth in CIDP, including its plan to launch multiple CIDP products in 2025 and the expected timing of the EMVIGORATE study; its expectations regarding the growth of the MMN market opportunity; data readouts and regulatory milestones and plans, including the timing of planned clinical trials, expected data readouts, and regulatory approvals; the Wave 2 growth plan for 2026-2027; the Wave 3 growth plan for 2028-2030; its vision for 2030, including having 5 new molecules in Phase 3, 10 labeled indications and having 50,000 patients on treatment; and the anticipated timing of pending PFS regulatory decisions in the U.S., Europe, Canada and Japan. By their nature, forward-looking statements involve risks and uncertainties and readers are cautioned that any such forward-looking statements are not guarantees of future performance. argenx’s actual results may differ materially from those predicted by the forward-looking statements as a result of various important factors, including the results of argenx's clinical trials; expectations regarding the inherent uncertainties associated with the development of novel drug therapies; preclinical and clinical trial and product development activities and regulatory approval requirements in products and product candidates; the acceptance of argenx's products and product candidates by patients as safe, effective and cost-effective; the impact of governmental laws and regulations on our business; disruptions caused on our reliance of third parties suppliers, service provides and manufacturing; inflation and deflation and the corresponding fluctuations in interest rates; and regional instability and conflicts. A further list and description of these risks, uncertainties and other risks can be found in argenx’s U.S. Securities and Exchange Commission (the “SEC”) filings and reports, including in argenx’s most recent annual report on Form 20-F filed with the SEC as well as subsequent filings and reports filed by argenx with the SEC. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this document. argenx undertakes no obligation to publicly update or revise the information in this presentation, including any forward-looking statements, except as may be required by law. This presentation contains trademarks, trade names and service marks of other companies, which are the property of their respective owners. 2 |
| 3 We are ALL IN on Innovation |
| Innovation has no meaning unless it provides real benefit to patients 4 |
| VYVGART® Builds Foundation of Innovation Foundational Immune Target FcRn First-in-Class Potential Best-in-Class Fc Fragment Pipeline in a Product Opportunity VYVGART 15 In Development 3 Approved Indications 5 ABDEG Precision IgG Degradation Expansive Development Portfolio |
| VYVGART is Setting a New Standard for Patients MG MSE = MG-ADL SCORE of 0 or 1 MG-ADL SCORE ≤ 5 Response rate No/minimal symptoms Meaningful response ECI STAGE A 54% 8/10 CIDP 6 7/10 I feel fantastic! Amazing! My wife keeps telling me to shut up because I am talking too much since I am so excited! I cried a tear of happiness when I was eating because I was able to chew and swallow with no problem. I am so happy! – VYVGART Patient Fred, MG Patient Jamilah, CIDP Patient Substantial improvement in 32 functional ability % ≥2 POINT DECREASE IN INCAT FROM RUN-IN BASELINE |
| Financial Strength to Invest in Sustainable Innovation 21 75 131 173 218 269 329 374 398 478 573 737 0 50 100 150 200 250 300 350 400 450 500 550 600 650 700 750 Q1 2022 Q2 2022 Q3 2022 Q4 2022 Q1 2023 Q2 2023 Q3 2023 Q4 2023 Q1 2024 Q2 2024 Q3 2024 Q4 2024 $M Product Net Sales by Quarter $2.2B 2024 Product Net Sales Cash reflects cash, cash equivalents and current financial assets as of December 31, 2024 $3.4B Strong Cash Position Profitable in 2025 Disciplined Capital Allocation and Scaling 7 Q4 and FY 2024 selected financial information is preliminary, unaudited, and subject to adjustment |
| Fuel pipeline growth 2025 Strategic Priorities PFS Launch Expand next wave of innovation 4 New Molecules in Phase 1 8 Reach more patients with VYVGART 10 Phase 3s 10 Phase 2s |
| Reach More Patients with VYVGART 9 |
| PFS to Accelerate VYVGART Growth in MG and CIDP PFS PDUFA April 10, 2025 Aiming for Self-Administration 4 Global Decisions on Autoinjector Approval in 2025 2027 Planned Launch 10 Pre-Filled Syringe* Autoinjector* *FPO Application Pending Not FDA Approved *FPO Not FDA Approved |
| Growing VYVGART Leadership in MG U.S. Addressable MG Patients Total Addressable Market in 2030 Addressable Market at Launch 17K +11K +7K +25K 60K Seronegative Ocular Growth in Biologics Share of Market Path to 60K Addressable Patients Consistent QoQ growth VYVGART has set a high bar 11 Source: argenx market research #1 BRANDED BIOLOGIC for gMG |
| Ocular Domain Effect in ADAPT (1) Opportunity To Set New Standard in Ocular MG High Disease Burden Impaired ability to work, drive and participate in social activities High Treatment Burden Frequent chronic, high doses of oral corticosteroids(2) Pioneer and Transform + Strong rationale from ADAPT and case reports + Upside potential to delay generalization to gMG + OCULUS: First and only study in oMG (2) 89% ≥10mg/kg/day and 46% ≥20 mg/kg/day and 18% ≥;30mg/kg/day | source: PROMISE MG Addressing Unmet Need EFGARTIGIMOD PLACEBO 12 (1) Source: Vera Bril et al. Effect of Efgartigimod on Muscle Group Subdomains in Participants With Generalized Myasthenia Gravis: Post Hoc Analyses of the Phase 3 Pivotal ADAPT Study Weeks %change from baseline in MG-ADL ocular subdomain |
| Continued Momentum in CIDP Patients Physicians Payors ~1,000 Patients on Therapy 90% Lives Covered Majority policies favorable 25% New Prescribers Global Expansion Multiple planned launches in 2025 Majority IVIg-experienced Breadth and depth of prescribers 13 |
| Fuel Pipeline Growth 14 |
| Investigate/ESR Expand GBS NMOSD MG Crisis MG Steroid Taper CIDP gMG oMG snMG CMS SMA ALS MMN CIDP SJD TED DGF AIE Lead ON SSC DM DM ASyS IMNM AMR LN ITP ITP Other I&I Our Pipeline is Positioned to Fuel Continuous Growth Neuromuscular Rheumatology ARGX-119 Empasiprubart Efgartigimod 15 Prevalence Bubble Size and Color Indicates Asset <15k 15-100k 100-300k |
| Empasiprubart is Now a Phase 3 Asset DGF and DM Proof of Concept Studies MMN and CIDP Registrational Studies IgG IgM Mannose sugar C5 C4 C2 C3 CP LP 16 Foundational Immune Target Complement Factor C2 First-in-Class Potential Best-in-Class C2-Specific Antibody Pipeline in a Product Opportunity Empasiprubart Intersection of Classical and Lectin Pathways NHance |
| Phase 2 ARDA Study: Transformational Data in MMN Significant Unmet Need Empasiprubart Treated Patients Feel Better than their Best on IVIg Life-Limiting Symptoms Progressive, disabling, asymmetric limb weakness Severe disability in 20% of patients Frequently misdiagnosed as ALS IVIg as only approved therapy Cohort 1: 94.4% improved 0 20 40 60 80 100 Percentage of Participants (%) Empasiprubart Placebo Very much improved Minimally improved Much improved No change Minimally worse Much worse Very much worse Study Met Primary Endpoint 91% Empasiprubart reduced risk of IVIg retreatment by up to 17 Cohort 1 Presented at the 10 Presented at the 2024 Peripheral Nerve Society (PNS) th Congress of the European Academy of Neurology (EAN) |
| 18 Disrupting Blockbuster Markets EMPASSION Study Ongoing EMVIGORATE Study to Begin 1H 2025 CIDP Opportunity to shape CIDP with two argenx medicines MMN Two Head-to-Head Phase 3 Studies with IVIg >400 patients enrolled in iMMersioN natural history study Innovation Builds Markets Today Following similar analogues in MG and MS Future More innovation brings better outcomes for more patients MMN Market $750-800M Market1 18 1. Opta, Takeda, CSL, argenx analysis |
| ARGX-119 is Now in Proof-of-Concept Studies Foundational Immune Target MuSK First-in-Class Potential Best-in-Class MuSK Agonist Antibody Pipeline in a Product Opportunity ARGX-119 SMA Next Indication CMS and ALS Proof-of-concept Studies P P MuSK SIMPLE Antibody Crucial for Neuromuscular Junction Function 19 |
| Expand Next Wave of Innovation 20 |
| 4 Phase 1 Molecules in 2025 Continued Leadership with Broad Immune System Targets ARGX-213 FcRn ARGX-121 IgA ARGX-220 Target Undisclosed ARGX-109 IL-6 Fc-ABDEGTM Potential for monthly dosing Rapid, deep IgA reduction Best-in-class potency NHance® First-in-class sweeper Convenient dosing Prolonged IgG reduction Fc-ABDEGTM α-albumin VHH Enables flexible dosing Leverages FcRn biology 21 |
| Innovation Model Generating World-Class Pipeline Active IIP Programs 4 PHASE 1 10 Proof of Concept 10 PHASE 3 APPROVED Neuro Target Neuro Target ARGX-213 (FcRn) Lupus Nephritis Seronegative MG Neuro Target Neuro Target ARGX-109 (IL6) AMR Ocular MG Neuro Target Rheum Target ARGX-121 (IgA) Scleroderma ITP Rheum Target Rheum Target ARGX-220 AIE TED Rheum Target Rheum Target Undisclosed Sjogren’s Disease Heme Target Skin Target Delayed Graft Function IMNM Skin Target Renal Target Dermatomyositis ASyS Renal Target Other CMS Dermatomyositis Other Other ALS MMN SMA CIDP Efgartigimod Empasiprubart ARGX-119 Other PROGRAMS 22 |
| Significant Momentum Ahead Regional PFS Approvals 1H25 PFS FDA, EMA decisions on approval 2H25 PFS Canada, Japan decisions on approval Efgartigimod IVIg Switch CIDP Ph4 Efgartigimod Seronegative MG Ph3 Efgartigimod Lupus Nephritis Ph2 Empasiprubart DGF Ph2 ARGX-119 CMS Ph1b ARGX-109 Ph1 1H26 Efgartigimod Ocular MG Ph3 Empasiprubart DM Ph2 ARGX-119 ALS Ph2a ARGX-121 Ph1 ARGX-213 Ph1 2H26 Empasiprubart MMN Ph3 Efgartigimod TED Ph3 Efgartigimod Myositis Ph3 Efgartigimod ITP (US) Ph3 Efgartigimod SSc Ph2 4 6 6 4 DECISIONS ON APPROVAL Ph3 READ OUTS Ph2 READ OUTS NEW MOLECULES IN Ph1 23 |
| Wave 1: TODAY Wave 2 (2026-2027) Wave 3 (2028-2030) TED Myositis oMG snMG Sjogren’s gMG CIDP ITP DM MMN CMS CIDP 50K 24 Strong Growth Trajectory to 50K Patients VYVGART Empasiprubart ARGX-119 |
| 25 Vision 2030 COMMITMENT TO OUR INNOVATION MISSION 5 New Molecules in Phase 3 10 Labeled Indications 50k Patients on Treatment 25 |
| Appendix 26 |
| Top 5 Autoimmunology Launches Our Innovation is Now Enabling our Sustainable Future 0 100 200 300 400 500 600 700 800 900 1000 L+0 L+1 L+2 L+3 L+4 L+5 L+6 L+7 L+8 L+9 L+10 L+11 L+12 PRODUCT NET REVENUE (USD, M) QUARTERS POST LAUNCH 27 |
| 28 |
| This is just the Beginning PHASE 3 STUDIES IN PROGRESS 2025 PROOF OF CONCEPT STUDIES snMG 81K* TED 100K ITP-US 75K Sjogren’s 330K IMNM 6K oMG 81K* ASyS 12K DM 70K LN 40K AMR 8K SSc 87K AIE 45K argenx market research; US prevalence and TAM numbers (except Japan ITP), sn gMG and oMG are in-market expansion studies *81k represents total MG prevalence, with snMG and oMG each representing 15% MG Launched 2022 81K CIDP Approved June 21, 2024 43K ITP Approved in Japan | March 26, 2024 17K Unnamed Indication 29 |
| Next Wave of Growth for VYVGART • Anti-Ro/Anti-La AutoAbs • Passive transfer model evidence • IgG reduction associated with improvement Treatment effect across multiple clinical endpoints, consistent with biomarker data CRESS/ESSDAI Steroids/NSISTs Cholinergic agonists Artificial tears • Myositis AutoAbs • Passive transfer model evidence (IMNM) • AutoAb titer correlates with disease activity POC established – GO decision Strong signal across subtypes inclusive of TIS ALKIVIA Phase 3 readout 2H 26 • IGF-1R, TSHR AutoAbs • Pathogenic potential of IgG (in vitro and in vivo) • AutoAbs correlate with clinical activity and severity POC established FcRn antagonization validated in TED* uplighTED Phase 3 readout 2H 2026 Teprotumumab is only FDA approved treatment BIOLOGY Steroids IVIg 330K 100K Sjogren’s Disease Myositis (IMNM, ASyS, DM) Thyroid Eye Disease CLINICAL COMMERCIAL 6K IMNM 11K ASyS 70K DM 30 *Immunovant POC established – GO decision UNITY Phase 3 ongoing |
