Argenx SE 6-K, filed Jan 8, 2024
Argenx SE (ARGX)
About this 6-K
Argenx SE filed this 6-K on January 8, 2024.
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 2024
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 8, 2024, argenx SE (the “Company”) issued a press release and a presentation the Company intends to use at the 42nd Annual J.P. Morgan Healthcare Conference on January 8, 2024 at 9:00 a.m. PT, 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 Exhibit 99.1 and Exhibit 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333-274721), and to be part thereof from the date on which this Current Report on Form 6-K is filed, to the extent not superseded by documents or reports subsequently filed or furnished.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 8, 2024 | |
| 99.2 | Investor Presentation, January 8, 2024 |
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 8, 2024 | By: | /s/ Hemamalini (Malini) Moorthy |
| Name: Hemamalini (Malini) Moorthy | ||
| Title: General Counsel | ||
Exhibit 99.1
argenx Highlights 2024 Strategic Priorities
Reported $1.2 billion in preliminary* full-year 2023 global net product sales
Submitted sBLA to FDA for VYVGART® Hytrulo for CIDP with priority review voucher (PRV); if approved, launch expected mid-2024
Reported positive data from Phase 2 ARDA study establishing proof-of-concept for empasiprubart in MMN
Data from six Phase 2 proof-of-concept trials expected by end of 2024
Nominated four new pipeline candidates with IND filings expected by end of 2025
January 8, 2024, 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 2023, including global net product sales (inclusive of both VYVGART® and VYVGART Hytrulo), and announced its strategic priorities for 2024.
“In 2023, we reached more than 6,000 patients globally, making VYVGART available to gMG patients around the world,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx. “We continued to invest in and demonstrate the sustainability of our business by successfully launching our subcutaneous VYVGART product, and are poised for continued expansion in gMG and beyond in 2024. argenx is delivering on its promise to transform how the world understands autoimmunity. It is with this commitment in mind that we submitted our sBLA for VYVGART Hytrulo in CIDP and, if approved, expect to launch in the U.S. in mid-2024. We will continue to be aggressive in advancing our pipeline this year and expect to report efgartigimod data from six Phase 2 studies in 2024, and to further develop empasiprubart in MMN. Through sustained investment in our IIP, we expect to see repeat value creation, and plan to submit four new INDs by the end of 2025.”
“Two years ago, argenx’s key goal was to launch efgartigimod in the U.S. Today, we have built a formidable global commercial organization with product approvals in over 30 countries worldwide and a vibrant pipeline of promising new therapeutics to address immune-mediated diseases. We have forged important partnerships that support our mission to advance the human understanding of immunology to best benefit patients. argenx is well positioned for sustained growth throughout 2024 and well into the future.”
2024 Strategic Priorities
argenx will focus on three strategic priorities in 2024 to drive sustainable long-term growth, including:
| · | Reach more patients with VYVGART by building upon its strong commercial foundation to address ongoing unmet patient need, broaden the MG opportunity, and expand into CIDP |
| · | Advance its extensive pipeline through new data readouts, creating multiple opportunities to demonstrate transformative clinical benefit |
| · | Leverage its repeatable innovation engine, driving pipeline growth through its Immunology Innovation Program |
Reach More Patients with VYVGART
VYVGART (efgartigimod alfa fcab) is a first-in-class antibody fragment targeting the neonatal Fc receptor (FcRn) and is now approved in more than 30 countries globally. VYVGART subcutaneous (SC) (efgartigimod alfa and hyaluronidase-qvfc) is approved in the U.S. (as VYVGART Hytrulo) and Europe, making VYVGART the only gMG treatment available as both an IV and simple SC injection. argenx is planning to reach more patients commercially in 2024 through its multi-dimensional expansion efforts. argenx will work to reach patients earlier in the MG treatment paradigm and improve the lives of new MG patient populations through additional global regulatory approvals, and the expansion of uses to treat additional autoimmune indications.
| · | Regulatory approval decisions of VYVGART for gMG expected in Switzerland, Australia, Saudi Arabia and South Korea by end of 2024 |
| · | Through strategic collaboration with Zai Lab, VYVGART to be included on China’s 2023 National Reimbursement Drug List (NRDL), starting in January 2024 |
| · | Decision on approval of VYVGART SC for gMG in Japan expected in first quarter of 2024 and in China through Zai Lab by end of 2024 |
| · | Decision on approval of VYVGART for primary immune thrombocytopenia (ITP) in Japan expected in first quarter of 2024 |
| · | Supplemental Biologics License Application (sBLA) submitted to FDA for VYVGART Hytrulo for CIDP with priority review voucher (PRV); if approved, launch expected mid-2024 |
| · | Regulatory submissions of VYVGART SC for CIDP in Japan, Europe, China and Canada expected in 2024 |
| · | Registrational studies to expand VYVGART label into broader MG populations, including in seronegative patients, to start in 2024 |
| · | Update on pre-filled syringe development expected in first half of 2024; ongoing studies to support potential approval in gMG and CIDP in 2024 |
Advance Current Pipeline through Upcoming Data Readouts
argenx continues to demonstrate breadth and depth within its immunology pipeline and is advancing multiple pipeline-in-a-product candidates. With efgartigimod, argenx is solidifying its leadership in FcRn and is on track to be approved or in development in 15 autoimmune indications by 2025. Beyond efgartigimod, argenx is advancing its earlier stage pipeline programs, including empasiprubart (C2 inhibitor) with Phase 2 studies ongoing in multifocal motor neuropathy (MMN), delayed graft function and dermatomyositis (DM). In addition, ARGX-119, a muscle-specific kinase (MuSK) agonist, will initiate Phase 1b/2a studies in congenital myasthenic syndrome and amyotrophic lateral sclerosis in 2024.
Today, argenx reported positive clinical data from the first cohort of the Phase 2 ARDA study of empasiprubart, establishing proof-of-concept in MMN. After confirming IVIg dependence, 27 patients were withdrawn from IVIg treatment and randomized 2:1 to either empasiprubart or placebo for 16 weeks. Patients were monitored for clinical deterioration that required IVIg retreatment, which was the main efficacy endpoint of the study.
| · | Empasiprubart demonstrated a 91% reduction in the need for IVIg rescue compared to placebo [HR: 0.09 95% CI (0.02; 0.044)] |
| · | According to the Patient Global Impression of Change scale, 94% (17/18) of empasiprubart-treated patients rated their condition as improved since study start, including 55% (10/18) who were much or very much improved. Of placebo patients, 89% (8/9) worsened or had no change. |
| · | Empasiprubart demonstrated improvement on all six efficacy measurements compared to baseline |
| · | Safety and tolerability profile were consistent with Phase 1 results |
| · | Cohort 2 is ongoing to determine dose response ahead of a Phase 3 study start |
argenx is on track to report topline data from five additional proof-of-concept studies in 2024, including:
| · | Phase 2 RHO study evaluating efgartigimod in primary Sjogren’s syndrome expected in first half of 2024 |
| · | Phase 2 ALPHA study evaluating efgartigimod in post-COVID-19 postural orthostatic tachycardia syndrome (PC-POTS) expected in first half of 2024 |
| · | Seamless Phase 2/3 ALKIVIA study evaluating efgartigimod across three myositis subsets (immune-mediated necrotizing myopathy (IMNM), antisynthetase syndrome (ASyS), and DM) expected in the second half of 2024 |
Leverage Repeatable Innovation Playbook to Drive Long-Term Pipeline Growth
argenx continues to invest in its discovery engine, the 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 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 and argenx is on track to file four investigational new drug (IND) applications by the end of 2025.
Preliminary* Fourth Quarter and Full-Year 2023 Financial Results
Today, argenx also announced preliminary* global net VYVGART revenues for the fourth quarter and full-year 2023 of approximately $374 million and $1.2 billion, respectively.
As of December 31, 2023, argenx had approximately $3.2 billion in cash, cash equivalents and current financial assets*. Based on its current operating plans, argenx expects its combined R&D and SG&A expenses in 2024 to be less than $2 billion. The projected 2024 cash burn will be approximately $500 million. argenx expects its existing cash, cash equivalents and current financial assets, together with anticipated future product revenues, to fund the Company to profitability.
* - The preliminary selected financial results are unaudited, subject to adjustment, and provided as an approximation in advance of the Company’s announcement of complete financial results in February 2024.
42nd Annual J.P. Morgan Healthcare Conference Presentation and Webcast
Mr. Van Hauwermeiren will highlight these updates in a corporate presentation at the 42nd Annual J.P. Morgan Healthcare Conference today, Monday, January 8, 2024, at 9:00 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.
Phase 2 ARDA Study Design
The Phase 2 ARDA study is a randomized, double-blinded, placebo-controlled multicenter study to evaluate the safety and tolerability, efficacy, pharmacokinetics, pharmacodynamics, and immunogenicity of two dose regimens of empasiprubart in adults with multifocal motor neuropathy (MMN). The study consists of an IVIg dependency and monitoring period and two 16-week treatment cohorts of 24 MMN patients receiving empasiprubart or placebo in a 2x1 randomization. The dosing for Cohort 2 was established after a planned interim analysis of the first nine patients to complete the 16-week treatment period from Cohort 1. The primary endpoint is safety and tolerability. Additional endpoints include time to IVIg retreatment, biomarker analyses of C2 levels, and changes in measurements on key functional scores (modified medical research council (mMRC)-10 sum score, grip strength, MMN-RODS) as well as several patient-reported quality of life outcome measures (fatigue severity score (FSS), chronic acquired polyneuropathy patient-reported index (CAP-PRI), and values of the patient global impression change (PGIC) scale).
About Multifocal Motor Neuropathy
Multifocal motor neuropathy (MMN) is a rare, chronic autoimmune disease of the peripheral nervous system. The disease is characterized by slowly progressive, asymmetric muscle weakness mainly of the hands, forearms and lower legs. MMN is often associated with anti-GM1 IgM autoimmunity, leading to activation of the classical complement pathway, driving subsequent axon damage. High-dose IVIg is the only approved treatment for MMN and patients typically experience disease progression despite therapy, indicating an unmet need for efficacious and better tolerated therapeutic options.
About Empasiprubart
Empasiprubart (ARGX-117) is a first-in-class humanized sweeping antibody that binds specifically to C2 thereby blocking both the classical and lectin pathways of the complement cascade. By blocking upstream complement activity, empasiprubart has the potential to reduce tissue inflammation representing a broad pipeline opportunity across multiple severe autoimmune indications. In addition to multifocal motor neuropathy, argenx is evaluating empasiprubart in delayed graft function following kidney transplant and dermatomyositis.
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 globally for the treatment of adults with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive and in Japan for the treatment of adults with gMG who do not have sufficient response to steroids or non-steroidal immunosuppressive therapies (ISTs).
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. and VYVGART SC in Europe, 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, 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 results presented in this press release are 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, 2023, and full year financial results for 2023 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 2023 financial results and full year financial results for 2023 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 “plans,” “aims,” “believes,” “continues,” “hope,” “estimates,” “preliminary,” “anticipates,” “expects,” “intends,” “may,” “will,” “should,” or “commitment” and include statements argenx makes concerning its preliminary financial results for the full year 2023; its expansion efforts, including reaching more patients with VYVGART within the MG treatment paradigm, through geographic expansion and into new autoimmune indications, expanding into CIDP, and the anticipated development of empasiprubart and ARGX-119; the anticipated timing of its launch of SC efgartigimod for CIDP in the U.S.; the initiation, timing, progress and results of its anticipated clinical development, data readouts and regulatory milestones and plans; its strategic priorities, including the timing and outcome of regulatory filings and regulatory approvals; its expectations of future profitability; the potential of its innovative clinical programs; and the nomination of new development candidates. 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 but not limited to argenx’s ability to successfully execute its business and growth strategies, the inherent uncertainties associated with development of novel drug therapies, preclinical and clinical trial and product development activities and regulatory approval requirements, the acceptance of our products and product candidates by our patients as safe, effective and cost-effective, volatile market conditions, and the impact of governmental laws and regulations on our business. 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.
| argenx The Next Chapter JP MORGAN HEALTHCARE CONFERENCE JANUARY 8, 2024 1 |
| 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. The financial results presented in this presentation are 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 presentation. 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, 2023, and full year financial results for 2023 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 2023 financial results and full year financial results for 2023 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. These forward-looking statements can be identified by the use of forward-looking terminology, including the terms “plans,” “aims,” “believes,” “continues,” “hope,” “estimates,” “preliminary,” “anticipates,” “expects,” “intends,” “may,” “will,” “should,” or “commitment” and include statements argenx makes concerning its preliminary financial results for the full year 2023; its expansion efforts, including reaching more patients with VYVGART within the MG treatment paradigm, through geographic expansion and into new autoimmune indications, expanding into CIDP, and the anticipated development of empasiprubart and ARGX-119; the anticipated timing of its launch of SC efgartigimod for CIDP in the U.S.; the initiation, timing, progress and results of its anticipated clinical development, data readouts and regulatory milestones and plans; its strategic priorities, including the timing and outcome of regulatory filings and regulatory approvals; its expectations of future profitability; the potential for innovation of its clinical programs; its pipeline; and the nomination of new development candidates. 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 but not limited to argenx’s ability to successfully execute its business and growth strategies, the inherent uncertainties associated with development of novel drug therapies, preclinical and clinical trial and product development activities and regulatory approval requirements, the ability of our clinical trials to reach their endpoints, the ability to maintain, expand, and deliver on our pipeline; the acceptance of our products and product candidates by our patients as safe, effective and cost-effective, volatile market conditions, and the impact of governmental laws and regulations on our business. 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 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 |
| On a Journey to Transform Autoimmunity Pioneering novel target biology Leading antibody engineering capabilities Pipeline-in-a-product opportunities Creating optionality across and within molecules 3 |
| We are Hearing Transformational Stories with VYVGART 4 Mike, VYVGART Patient |
| PFS in development 15 indications in development by 2025 ITP MAA filed CIDP sBLA submitted ARGX-109 $1.2B in gMG revenue in 2023 (Anti-IL-6) ARGX-213 (Anti-FcRn) ARGX-220 ARGX-121 Empasiprubart POC established in MMN Trials in DGF and DM ARGX-119 Phase 1b/2a trials in CMS and ALS Our Innovation Horizons 5 |
| PFS in development 15 indications in development by 2025 ITP MAA filed CIDP sBLA submitted ARGX-109 $1.2B in gMG revenue in 2023 (Anti-IL-6) ARGX-213 (Anti-FcRn) ARGX-220 ARGX-121 Empasiprubart POC established in MMN Trials in DGF and DM ARGX-119 Phase 1b/2a trials in CMS and ALS VYVGART Opportunity Horizon 6 |
| Leadership in FcRn Pioneering FcRn Unique modulation of FcRn 15 indications by 2025* Fc fragment and proprietary ABDEG™ mutations Transformational data in gMG and CIDP Generating key learnings *Indications in development 7 |
| Estimated 4,000 patient years of safety follow-up between clinical trial and real-world experience 45% MSE QoL comparable to healthy population* 78% MG-ADL ≤4** Meaningful steroid tapering by at least 5mg/day within first 6 months Superior cost/benefit over IVIg*** Enables significantly faster access to treatment VYVGART is Setting New Expectations in gMG * Real world evidence **Source: ADAPT and ADAPT+ clinical trial data ***Leading Health Technology Assessment agency 8 |
| 25% YoY increase >2,300** Prescribers in the US PRESCRIBER EXPANSION Strong Commercial Execution 21% 2023 CAGR $1.2B* Global Product Revenue GROWTH 2023 Performance Favorable payor policies ~90% Access VYVGART after ≤2 Orals BROAD PATIENT ACCESS 55% patients from orals >6,000** Global VYVGART Patients EARLIER LINE PATIENTS * Preliminary Financials. Unaudited and subject to change ** As of Q3 2023 Financial Results 9 |
| Innovation Builds Autoimmune Market Opportunities 10 17,000 6,000* Growing MG Market gMG Patients on VYVGART Current VYVGART TAM Expanding VYVGART TAM Growing VYVGART Share US RoW argenx markets (exc. China) Global 31,000 Label-enabling trials in broader gMG populations Phase 3b studies and externally sponsored research Geographic expansion, including South Korea and Australia Growing VYVGART share US: VYVGART Hytrulo J-Code in effect; field force expansion PFS development Addition to China NRDL Expanding VYVGART TAM Growing MG market Targeted biologics are expanding gMG market by providing patients more treatment options *As of Q3 2023 Financial Results Based on argenx market research |
| VYVGART Has Potential to Transform CIDP 11 67% Response rate demonstrates IgG autoantibodies play significant role in underlying CIDP biology HR: 0.39 P = 0.000039 99% Rollover of eligible patients to open-label extension Favorable safety and tolerability profile consistent with previous clinical trials Stage B sBLA submitted with priority review voucher SET NEW STANDARD FOR HOW CIDP TRIALS ARE RUN SIGNIFICANT IMPACT ON CIDP PATIENTS 99% Study Compliance Stage A ESTABLISHED CIDP AS IgG MEDIATED 61% reduced risk of relapse 11 |
| CONFIDENTIAL FOR INTERNAL USE ONLY. © 2023 argenx. We Aim to Address the Unseen Suffering in CIDP ≤20% of patients achieve remission on current SOC (CDAS=2)* >50% of patients are dissatisfied with their symptom burden** >42K treated CIDP patients in US & ROW argenx markets (ex-China)*** * Gorson KC, et al. 2010 ** Mendoza M, et al. 2023 *** argenx market research 12 12 |
| Transforming the Patient Treatment Experience 13 Pre-filled Syringe Autoinjector Single 30-90s injection HCP administered Increasingly convenient delivery Preparing for self-administration Approved June 2023 Ongoing in clinical trials Industrialization phase Exclusive FcRn license to ENHANZE® High concentration formulation with low viscosity, no back pressure |
| This is Just the Beginning 14 Generalized Myasthenia Gravis Chronic Inflammatory Demyelinating Polyneuropathy Expected 2024 PoC study readouts ITP Launched 2022 Anticipated approval 2024 Japan submission in review TED 100K IMNM 6K Sjogren’s 330K PC-POTS 500K Beyond 2024 PoC studies BP 52K LN 40K MN 80K AAV 105K AMR 8K Potential 2024 Phase 3 Start Phase 2 Proof of Concept 65K 24K 17K 2024 Phase 3 Start ASyS 11K DM 70K #14 #15 *** argenx market research; US prevalence numbers (except Japan ITP) |
| Phase 2 Readouts Present Significant Commercial Opportunities 15 • Anti-Ro/Anti-La AutoAbs • Passive transfer model evidence • IgG reduction associated with improvement RCT - Phase 2 CRESS/ESSDAI • Steroids/NSISTs • Cholinergic agonists • Artificial tears • Anti-adrenergic receptor AutoAbs • IVIG/PLEX effective RCT - Phase 2 MaPS/COMPASS • No approved therapies • Myositis AutoAbs • Passive transfer model evidence (IMNM) • AutoAb titer correlates with disease activity RCT - P2/P3 TIS BIOLOGIC RATIONALE • Steroids • IVIg 330K 500K 6K IMNM 11K ASyS 70K DM Sjogren’s Syndrome PC-POTS Myositis (IMNM, ASyS, DM) CLINICAL FEASIBILITY U.S. COMMERICAL OPPORTUNITY |
| PFS in development 15 indications in development by 2025 ITP MAA filed CIDP sBLA submitted ARGX-109 $1.2B in gMG revenue in 2023 (Anti-IL-6) ARGX-213 (Anti-FcRn) ARGX-220 ARGX-121 Empasiprubart POC established in MMN Trials in DGF and DM ARGX-119 Phase 1b/2a trials in CMS and ALS Pipeline Horizon 16 |
| Rewriting Immunology Textbook with Empasiprubart Pioneering complement factor C2 Unique sweeping antibody Ongoing development in 3 indications ~80-day half-life supports favorable dosing Defining MMN as POC established in MMN auto-IgM mediated disease 17 |
| Empasiprubart has Potential to Transform MMN 18 HR (95% CI) = 0.09 (0.02; 0.44) • 94% of treated patients rated their condition improved since starting therapy, including 55% who were much/very much improved 8/9 placebo patients had no change or worsened (Patient Global Impression of Change scale) • Empasiprubart demonstrated improvement compared to baseline on 6/6 efficacy measurements • Safety profile consistent with Phase 1 data 91% reduction in need for IVIg rescue with empasiprubart Cohort 2 is ongoing; results to inform dose for Phase 3 study initiation 18 |
| MMN Patients are Waiting Clear opportunity for empasiprubart… …I’m not asking to be able to run and jump like I used to. I just want to be able to stand like I used to. “ ” IVIg only treatment option Patient journey characterized by deep frustration and anxiety …to transform MMN outcomes ADDRESSABLE MARKET ~10k patients US + argenx ROW markets (ex China)* *argenx market research; Arnold et al 2013; Park et al 2022 19 |
| ARGX-119: Enhancing Neuromuscular Junction Pioneering MuSK biology at NMJ Initial development in CMS and ALS Agonistic SIMPLE Antibody™ Safety and tolerability data from extensive Phase 1 study support advancement into PoC studies 20 |
| CMS and ALS Trials to Start in 2024 21 ARGX-119 rescues early neonatal lethality and disease relapse in adult DOK7 CMS mice Nature, Oury et al. 2021 ARGX-119 preserves NMJ numbers and restores muscle contraction in ALS patient derived NMJs on-a-chip Fidelity (%) Biomaterials, Badu-Mensah et al. 2022; Advanced Therapeutics, Guo et al. 2020 50 100 0 Frequency (Hz) Healthy ALS ALS + ARGX-119 argenx internal data mAb mAb |
| PFS in development 15 indications in development by 2025 ITP MAA filed CIDP sBLA submitted ARGX-109 $1.2B in gMG revenue in 2023 (Anti-IL-6) ARGX-213 (Anti-FcRn) ARGX-220 ARGX-121 Empasiprubart POC established in MMN Trials in DGF and DM ARGX-119 Phase 1b/2a trials in CMS and ALS Immunology Innovation Horizon 22 |
| ARGX-109 (Anti-IL-6) Pipeline Growth Driven By Immunology Innovation Program 23 ARGX-213 ARGX-220 DISEASE INSIGHT ANTIBODY ENGINEERING CLINICAL DEVELOPMENT Continued Leadership in FcRn Broad Immune System Targets ARGX-121 First in class, novel targets 4 IND FILINGS BY END OF 2025 Strong Track Record with Repeatable Innovation Playbook LEADING TRANSLATIONAL BIOLOGY LABS TECHNOLOGY KNOW-HOW |
| VYVGART gMG Decision on approval: Switzerland, Australia, Saudi Arabia, South Korea By Year End Seronegative trial initiation By Year End ITP Japan decision on approval 1Q 2024 VYVGART SC gMG Japan decision on approval By 1Q 2024 China decision on approval (Zai Lab) By Year End CIDP U.S. launch, if approved Mid-2024 Regulatory submissions Japan, Europe, China, Canada By Year End MG, CIDP Update on PFS development 1H 2024 Efgartigimod Primary Sjogren’s syndrome Proof of concept data 1H 2024 PC-POTS Proof of concept data 1H 2024 Myositis Proof of concept data 2H 2024 Empasiprubart MMN Full Phase 2 data 2024 ARGX-119 CMS, ALS Phase 1b/2a study initiations 2024 IIP Not Disclosed 4 INDs filed By End of 2025 24 Indication Milestone Timing Strong Cadence of Milestones in 2024 |
| Q4 2023 Product Net Sales of $374 million 21 75 131 173 218 269 329 374 2022 Q1 2022 Q2 2022 Q3 2022 Q4 2023 Q1 2023 Q2 2023 Q3 2023 Q4* ($B) 2023 Product Net Sales(1) 1.2 Cash, cash equivalents and current financial assets(1) 3.2 2024 Financial Guidance (1) - Preliminary Financials. Unaudited and subject to change. ($B) 2024 Cash burn(2) ~ 0.5 Combined R&D and SG&A expenses < 2.0 *Preliminary Financials. Unaudited and subject to change Preliminary 2023 Financial Results (2) - Cash burn is equal to the decrease in our cash, cash equivalents and current financial assets On Track To Be Sustainable 2023 CAGR: 21% 25 |
| 6 Leading to multiple 2024 Strategic Priorities Committed to Driving Continued Growth 26 Broaden leadership in MG market Launch CIDP Advance PFS Phase 2 data readouts Phase 3 initiations INDs by 2025 4 |
| THANK YOU 27 |
