Argenx SE 6-K, filed Jun 21, 2023
Argenx SE (ARGX)
About this 6-K
Argenx SE filed this 6-K on June 21, 2023.
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 June 2023
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 June 20, 2023, argenx SE (the “Company”) issued two press releases and on June 21, 2023, the Company issued an investor presentation, copies of which are attached hereto as Exhibits 99.1, 99.2 and 99.3, respectively, and are incorporated by reference herein.
The information contained in this Current Report on Form 6-K, including Exhibits 99.1, 99.2 and 99.3, are incorporated by reference into the Company’s Registration Statements on Forms F-3 (File No. 333-258251) and S-8 (File Nos. 333-225375 and 333-258253).
| Exhibit | Description | |
| 99.1 | Press Release dated June 20, 2023 | |
| 99.2 | Press Release dated June 20, 2023 | |
| 99.3 | Investor Presentation dated June 21, 2023 |
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: June 21, 2023 | By: | /s/ Hemamalini (Malini) Moorthy |
| Hemamalini (Malini) Moorthy | ||
Exhibit 99.1
argenx initiates second cohort of Phase 2 ARDA study of empasiprubart in multifocal motor neuropathy
| · | Independent Data Monitoring Committee recommended study continuation based on the favorable safety profile observed in the first dose cohort |
| · | Early efficacy signals support proof-of-concept of empasiprubart in multifocal motor neuropathy |
Regulated Information/Inside Information
Amsterdam, the Netherlands—June 20, 2023—argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced its plan to advance to a second dose cohort with the Phase 2 ARDA study of empasiprubart (ARGX-117) in multifocal motor neuropathy (MMN). The decision follows a planned interim analysis of the first dose cohort by an Independent Data Monitoring Committee (IDMC) meeting held on June 19, 2023.
“We are encouraged by the favorable safety profile and early efficacy signals from the ARDA study, as well as the IDMC recommendation to advance the study to the next cohort. Looking forward, we hope to build on the promising safety and efficacy we observed in the first cohort while populating our PK/PD model to understand the full potential of empasiprubart in MMN,” commented Luc Truyen, M.D., Ph.D., Chief Medical Officer, argenx. “People living with MMN continue to face significant burden associated with this chronic autoimmune disease, including progressive muscle weakness, which can lead to an inability to walk or move one’s arms or hands. Our mission is to transform treatment paradigms for autoimmune patients by changing expectations on what well-controlled means. Based on these data, we are hopeful that we can accomplish this in MMN and other autoimmune indications with our first-in-class C2 inhibitor.”
The IDMC reviewed interim safety data from all patients (n=22) currently enrolled in the first cohort of the ARDA study, including nine patients who completed the full 16-week treatment period. The IDMC confirmed a favorable safety and tolerability profile of empasiprubart consistent with results from the Phase 1 study and recommended advancing to the second cohort. An early efficacy assessment of all 22 patients supports proof-of-concept of empasiprubart in MMN. The data showed distinct separation between treated patients and placebo based on a suite of clinical outcome measures, including time to IVIg retreatment.
In total, the ARDA study is expected to enroll 48 patients across two cohorts. The study’s objective, in addition to assessing safety and efficacy of empasiprubart, is to populate a PK/PD model to inform the Phase 3 study dose selection. Based on the IDMC recommendation to continue enrollment, argenx will aim to build on the promising efficacy and safety observations from the first cohort by evaluating a next dose level of empasiprubart.
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 will be 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 clinical efficacy scores (modified medical research council (mMRC)-14 sum score, grip strength, MMN-RODS) as well as several patient-reported quality of life outcome measures.
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 planning to evaluate empasiprubart in delayed graft function following kidney transplant and dermatomyositis.
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 IV immunoglobulin (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 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-and- only approved neonatal Fc receptor (FcRn) blocker in the U.S., Japan, the EU and the UK. 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.
For further information, please contact:
Media:
Erin Murphy
EMurphy@argenx.com
Investors:
Alexandra Roy (US)
ARoy@argenx.com
Lynn Elton (EU)
LElton@argenx.com
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 “believes,” “hope,” “estimates,” “anticipates,” “expects,” “intends,” “may,” “will,” or “should” and include statements argenx makes concerning the safety profile and efficacy signals from the ARDA study; the prospects of empasiprubart as a treatment for MMN and other indications; and the expected enrollment, objectives and results of the ARDA study. 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. 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 publicly update or revise the information in this press release, including any forward-looking statements, except as may be required by law.
Exhibit 99.2
argenx Announces U.S. Food and Drug Administration Approval of VYVGART Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) Injection for Subcutaneous Use in Generalized Myasthenia Gravis
| • | VYVGART® Hytrulo is first FDA-approved subcutaneous (SC) injectable for generalized myasthenia gravis (gMG) |
| • | With this approval, argenx broadens innovative gMG product offering and demonstrates continued commitment to providing more choice and flexibility for patients |
| • | Efficacy of VYVGART Hytrulo was established by demonstrating a comparable pharmacodynamic (PD) effect to VYVGART® in Phase 3 ADAPT-SC bridging study |
| • | Management to host conference call tomorrow at 2:30pm CET (8:30 am ET) |
Regulated Information/Inside Information
Amsterdam, the Netherlands—June 20, 2023—argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) approved VYVGART® Hytrulo (efgartigimod alfa and hyaluronidase-qvfc). VYVGART Hytrulo is an injection for subcutaneous (SC) use for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. These patients represent approximately 85% of the total gMG population.
VYVGART Hytrulo is a subcutaneous product combination of efgartigimod alfa, a human IgG1 antibody fragment marketed for intravenous use as VYVGART, and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE® drug delivery technology to facilitate subcutaneous delivery of biologics. The product is to be administered subcutaneously by a healthcare professional as a single injection (1,008 mg fixed dose) over 30-90 seconds in cycles of once weekly injections for four weeks.
“Today’s approval of VYVGART Hytrulo is another significant milestone on our path to redefine what well-controlled means for gMG patients. The availability of a second argenx innovation in just 18 months also underscores our longstanding commitment to the gMG community by providing more choice and flexibility in how patients receive treatment,” said Luc Truyen M.D., Ph.D., Chief Medical Officer, argenx. “With our broad gMG offering of both a first-in-class infusion and SC injection, we continue to offer an individualized treatment approach and possibility of staying symptom free, while providing patients options of how and where they want to seek treatment. We want to thank the gMG patient community and their supporters, clinical investigators, our employees and all stakeholders who have collaborated with us to advance this subcutaneous option, including our partners at Halozyme.”
“The availability of another gMG treatment option from argenx, now in a subcutaneous delivery, is a meaningful advancement for the patient community. Patients now have the opportunity to receive treatment in an infusion center, at home or at a physician’s office - providing more flexibility and freedom of choice that can make daily living easier for gMG patients and their caregivers,” said Allison Foss, Executive Director of the Myasthenia Gravis Association.
Phase 3 ADAPT-SC Bridging Study
“The clinical trials of VYVGART continue to show significant benefit to patients with a favorable safety profile and clear improvements in gMG disease scores. Now with the approval of VYVGART Hytrulo, we have a broad gMG treatment offering with both IV and SC administration options and can select based on patient needs and preference without sacrificing clinical benefit or safety,” said James F. Howard Jr., M.D.,
Professor of Neurology (Neuromuscular Disease), Medicine and Allied Health, Department of Neurology, The University of North Carolina at Chapel Hill School of Medicine and Principal Investigator for the ADAPT-SC trial.
This FDA approval is based on positive results from the Phase 3 ADAPT-SC study, which established the efficacy of VYVGART Hytrulo by demonstrating a reduction in anti-AChR antibody levels comparable to intravenous VYVGART in adult gMG patients. ADAPT-SC was a bridging study to the Phase 3 ADAPT study, which formed the basis for approval of intravenous VYVGART in December 2021.
In the ADAPT-SC study, the primary endpoint of noninferiority was met (p< 0.0001) and VYVGART Hytrulo demonstrated mean total IgG reduction of 66.4% from baseline at day 29, compared to 62.2% with VYVGART. Additional key secondary endpoints were met, which were consistent with efficacy measures from the ADAPT study identifying the correlation between total IgG reduction and clinical benefit in gMG.
VYVGART Hytrulo has a demonstrated safety profile, consistent with the ADAPT clinical trial with the exception of injection site reactions (ISRs), which were higher with VYVGART Hytrulo. It was generally well-tolerated with ISRs being the most frequent adverse events. All ISRs, which are commonly observed with biologics administered subcutaneously, were mild to moderate, and resolved over time.
Access to VYVGART Hytrulo
VYVGART Hytrulo is expected to be available for patients in the U.S. in July 2023. argenx is committed to supporting access for patients to its medicines and has decided to price VYVGART Hytrulo at parity to VYVGART on a net annual revenue basis.
Throughout their treatment journey, patients can access VYVGART Hytrulo in the same personalized way they access VYVGART today with support from My VYVGART Path. Program resources include disease and product education, access support and benefits verification, and financial assistance programs for eligible patients. Patients and clinicians can access more information at VYVGARTHytrulo.com.
Marketing authorization applications for SC efgartigimod are under review by the European Medicines Agency with a decision expected by the end of 2023, and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) with a decision expected by the first quarter of 2024.
Conference Call Details
argenx will host a conference call tomorrow, June 21, 2023, at 2:30 pm CET (8:30am ET) to discuss the approval. A webcast of the live call and replay may be accessed on the Investors section of the argenx website.
Dial-in numbers:
Please dial in 15 minutes prior to the live call.
| Belgium | 32 800 50 201 |
| France | 33 800 943355 |
| Netherlands | 31 20 795 1090 |
| United Kingdom | 44 800 358 0970 |
| United States | 1 888 415 4250 |
| Japan | 81 3 4578 9752 |
| Switzerland | 41 43 210 11 32 |
See Important Safety Information below and full Prescribing Information for VYVGART Hytrulo for additional information.
Important Safety Information
What is VYVGART® HYTRULO (efgartigimod alfa and hyaluronidase-qvfc)?
VYVGART HYTRULO is a prescription medicine used to treat a condition called generalized myasthenia gravis, which causes muscles to tire and weaken easily throughout the body, in adults who are positive for antibodies directed toward a protein called acetylcholine receptor (anti-AChR antibody positive).
IMPORTANT SAFETY INFORMATION
What is the most important information I should know about VYVGART HYTRULO?
VYVGART HYTRULO may cause serious side effects, including:
| • | Infection. VYVGART HYTRULO may increase the risk of infection. The most common infections for efgartigimod alfa-fcab-treated patients were urinary tract and respiratory tract infections. More patients on efgartigimod alfa-fcab vs placebo had below normal levels for white blood cell counts, lymphocyte counts, and neutrophil counts. The majority of infections and observed lower white blood cell counts were mild to moderate in severity. Your healthcare provider should check you for infections before starting treatment, during treatment, and after treatment with VYVGART HYTRULO. Tell your healthcare provider if you have any history of infections. Tell your healthcare provider right away if you have signs or symptoms of an infection during treatment with VYVGART HYTRULO such as fever, chills, frequent and/or painful urination, cough, pain and blockage of nasal passages/sinus, wheezing, shortness of breath, fatigue, sore throat, excess phlegm, nasal discharge, back pain, and/or chest pain. If a serious infection occurs, your doctor will treat your infection and may even stop your VYVGART HYTRULO treatment until the infection has resolved. |
| • | Undesirable immune reactions (hypersensitivity reactions). VYVGART HYTRULO and efgartigimod alfa-fcab can cause the immune system to have undesirable reactions such as rashes, swelling under the skin, and shortness of breath. Hives were also observed in patients treated with VYVGART HYTRULO. In clinical studies, the reactions were mild or moderate and occurred within 1 hour to 3 weeks of administration, and the reactions did not lead to VYVGART HYTRULO discontinuation. Your healthcare provider should monitor you during and after treatment and discontinue VYVGART HYTRULO if needed. Tell your healthcare provider immediately about any undesirable reactions to VYVGART HYTRULO. |
Before taking VYVGART HYTRULO, tell your healthcare provider about all of your medical conditions, including if you:
| • | Have a history of infection or you think you have an infection. |
| • | Have received or are scheduled to receive a vaccine (immunization). Discuss with your healthcare provider whether you need to receive age-appropriate immunizations before initiation of a new treatment cycle with VYVGART HYTRULO. The use of vaccines during VYVGART HYTRULO treatment has not been studied, and the safety with live or live-attenuated vaccines is unknown. Administration of live or live-attenuated vaccines is not recommended during treatment with VYVGART HYTRULO. |
| • | Are pregnant or plan to become pregnant and are breastfeeding or plan to breastfeed. |
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
What are the common side effects of VYVGART HYTRULO?
The most common side effects of efgartigimod alfa-fcab-treated patients were respiratory tract infection, headache, and urinary tract infection. Additional common side effects of VYVGART HYTRULO are injection site reactions, including rash, redness of the skin, itching sensation, bruising, pain, and hives.
These are not all the possible side effects of VYVGART HYTRULO. Call your doctor for medical advice about side effects. You may report side effects to the US Food and Drug Administration at 1-800-FDA-1088.
Please see the full Prescribing Information for VYVGART HYTRULO and talk to your doctor.
About Phase 3 ADAPT-SC Trial
The Phase 3 ADAPT-SC trial was a multicenter, randomized, open-label, parallel-group study evaluating the noninferiority of the pharmacodynamic (PD) effect of VYVGART Hytrulo compared with VYVGART in adult patients with gMG. The pharmacodynamic effect was measured by percent change from baseline in autoantibody (AChR) levels at day 29. Safety, clinical efficacy, immunogenicity and pharmacokinetics (PK) were also assessed. A total of 110 adult patients with gMG in North America, Europe and Japan enrolled in the ADAPT-SC trial. Patients were randomized in a 1:1 ratio to receive VYVGART Hytrulo or VYVGART for one treatment cycle consisting of four doses at once-weekly intervals. The total study duration was approximately 12 weeks, including seven weeks of follow-up after the treatment cycle. At the completion of ADAPT-SC, patients had the opportunity to roll-over to ADAPT-SC+, an open-label extension study.
About VYVGART® Hytrulo
VYVGART Hytrulo is a subcutaneous combination of efgartigimod alfa, a human IgG1 antibody fragment marketed for intravenous use as VYVGART®, and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE® drug delivery technology to facilitate subcutaneous injection delivery of biologics. In binding to the neonatal Fc receptor (FcRn), VYVGART Hytrulo results in the reduction of circulating IgG. It is the first-and-only approved FcRn blocker administered by subcutaneous injection.
About Generalized Myasthenia Gravis
Generalized myasthenia gravis (gMG) is a rare and chronic autoimmune disease where IgG autoantibodies disrupt communication between nerves and muscles, causing debilitating and potentially life-threatening muscle weakness. Approximately 85% of people with MG progress to gMG within 24 months, where muscles throughout the body may be affected. Patients with confirmed AChR antibodies account for approximately 85% of the total gMG population.
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-and-only approved neonatal Fc receptor (FcRn) blocker in the U.S., Japan, Israel, the EU and the UK. 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.
For further information, please contact:
Media:
Erin Murphy
EMurphy@argenx.com
Investors:
Alexandra Roy (US)
ARoy@argenx.com
Lynn Elton (EU)
LElton@argenx.com
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 “believes,” “hope,” “estimates,” “anticipates,” “expects,” “intends,” “may,” “will,” or “should” and include statements argenx makes concerning the benefits and safety profile of VYVGART; the expected availability of VYVGART Hytrulo; and the timing and outcome of marketing authorizations for VYVGART Hytrulo by the European Medicines Agency and Japan’s Pharmaceuticals and Medical Devices Agency. 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. 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 publicly update or revise the information in this press release, including any forward-looking statements, except as may be required by law.
| VYVGART ® Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) FDA Approval Call June 21, 2023 |
| 2 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 our own internal estimates and research. While we believe these third-party studies, publications, surveys and other data to be reliable as of the date of this presentation, we have not independently verified, and make 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 our 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. Examples of such forward-looking statements include those argenx makes concerning the benefits and safety profile of VYVGART; the expected availability of VYVGART Hytrulo; the timing and outcome of marketing authorizations for VYVGART Hytrulo in China, Canada, Europe and Japan; the safety profile and efficacy signals from the ARDA study; and the prospects of empasiprubart as a treatment for MMN and other indications. 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. 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 presentation. 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. This presentation contains trademarks, trade names and service marks of other companies, which are the property of their respective owners. |
| VYVGART for Generalized Myasthenia Gravis VYVGART and VYVGART Hytrulo are indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive NOW TWO FDA-APPROVED PRODUCTS 3 |
| GENERALIZED MYASTHENIA GRAVIS PATIENT EXPERIENCE gMG is characterized by debilitating muscle weakness and fatigue. Despite taking an average of 2.3 current treatments, 61% of patients have poor well-being according to WHO-5 Index 1/2 of Patients have been diagnosed with depression or anxiety in addition to gMG 2.6 Years mean time from symptom expression to diagnosis 51% of Patients stopped working completely from disease impact ANDREA. Living with gMG VYVGART® Hytrulo has redefined my experience with gMG. I am grateful to have found a treatment built to my needs, empowering me to return to the activities I love. Information from argenx market research 35% of Patients need help from a caregiver with daily activities and 33% of caregivers have to cut back or stop working altogether 4 |
| Redefining What ‘Well-Controlled’ Means for the Patient 5 Achieve minimal symptom expression Reduce reliance on broad immunosuppressants Regain control of their lives, including professionally and socially Minimize treatment burden We want to transform gMG treatment for patients |
| EXPLORATORY MSE: MG-ADL score of 0 or 1 n=20/54 n=21/55 VYVGART Hytrulo VYVGART Innovative Phase 3 Bridging Study Consistent Responses Across IV and SC MG-ADL, Myasthenia Gravis Activities of Daily Living; QMG, Quantitative Myasthenia Gravis; MSE, minimal symptom expression Howard JF, et al. Lancet Neurol. 2021;20(7):526-536. Primary endpoint of noninferiority met [p<0.0001] IgG reduction (%) in all ADAPT-SC and ADAPT participants SECONDARY QMG responder: ≥3-point reduction for ≥4 consecutive weeks during the first cycle MINIMAL SYMPTOM EXPRESSION QMG RESPONDERS VYVGART n=36/55 51.9% n=28/54 ADAPT-SC 6 VYVGART n=38/55 69.1% SECONDARY MG-ADL responder: ≥2-point reduction ≥4 consecutive weeks during the first cycle n=38/55 MG-ADL RESPONDERS 69.1% 65.5% 37.0% 38.2% VYVGART Hytrulo VYVGART Hytrulo VYVGART Hytrulo |
| ADVERSE REACTIONS • The most common adverse reactions ( ≥10%) in patients treated with intravenous VYVGART were respiratory tract infections, headaches, and urinary tract infection. Additional common adverse reactions with VYVGART Hytrulo are injection site reactions. VYVGART® Hytrulo (efgartigimod alfa and hyaluronidase -qvfc) Injection for Subcutaneous Use INDICATION STATEMENT VYVGART Hytrulo is a combination of efgartigimod alfa, a neonatal Fc receptor blocker, and hyaluronidase, an endoglycosidase, indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti -acetylcholine receptor (AChR) antibody positive. DOSING AND ADMINISTRATION • Administer by a healthcare professional only • Recommended dose is 1,008 mg / 11,200 units (1,008 mg efgartigimod alfa and 11,200 units hyaluronidase) administered subcutaneously over approximately 30 to 90 seconds in cycles of once weekly injections for 4 weeks • Subsequent treatment cycles to be administered based on clinical evaluation • Safety of initiating subsequent cycles sooner than 50 days from the start of the previous treatment cycle has not been established WARNINGS AND PRECAUTIONS • Infections: delay administration to patients with active infection. Monitor for signs and symptoms of infection. If serious infection occurs, administer appropriate treatment and consider withholding VYVGART Hytrulo until infection has resolved • Hypersensitivity reactions: angioedema, dyspnea, and rash have occurred. If a hypersensitivity reaction occurs, discontinue the administration and institute appropriate supportive measures if needed VYVGART is a registered trademark of argenx. VYVGART Hytrulo is a trademark of argenx. © 2023 argenx. All rights reserved 7 |
| 8 Best-in-Class Patient Support Simplicity of Access Flexibility and Choice for Patients Chronic autoimmune diseases are complicated VYVGART makes treatment simple Broad HCP Use and Adoption |
| VYVGART Simplifies Treatment of gMG in the Community ~17,000 Lowering the bar for treatment initiation and expanding access for the current gMG patients we can address with VYVGART Demonstrated Efficacy: 78% responders across first two treatment cycles in ADAPT Simple Treatment Initiation: broad access, no vaccination requirements Choice in HOW to be treated: IV infusion or SC injection Predictable safety and tolerability: 18 months and over 3000* real world patients Options for site of care: infusion center, HCP office, home administration *As of end of 2022 9 |
| Simplicity of Access: Driving Choice By Value to the Patient Creating a new standard for gMG treatment Flexibility and choice for patients IV has broad and early access Annual net price to be similar across IV and SC treatment options ESTABLISHED VALUE TO PATIENTS PARITY PRICING PRODUCT CHOICE BY MARKET Patient Payor HCP Expected annual SC and IV net price for typical VYVGART patient is approximately $225,000 Price to vary based on individualized dosing and specific insurance coverage, and mandatory government rebates and discounts 10 |
| My VYVGART Path is Available to Provide Access Support and Education 11 PERSONALIZED SUPPORT Educate on VYVGART and VYVGART Hytrulo Treatment Resources and Information Navigate Insurance Process Financial Assistance Available |
| Reaching gMG Patients Across the Globe VYVGART Approved December 2021 VYVGART Hytrulo Approved June 2023 Japan Approved January 2022 Europe Approved September 2022 China (Zai Lab) Expected approval in 2023 Canada Expected approval in 2023 United Kingdom Approved March 2023 Israel (Medison) Approved April 2023 Subcutaneous efgartigimod approval decisions expected in Europe and Japan by 1Q24 US Global 12 |
| gMG is just the beginning 13 PC-POTS >500K ANCA 105K TED 100K Sjogren’s 330K Membranous Nephropathy 80K BP 52K gMG 65K ITP 57K Myositis 63K Pemphigus 19K CIDP 16K Lupus Nephritis 40K AMR 8K Patient numbers are U.S. prevalence from argenx market research clinical indications with successful proof-of-concept or Phase 3 data |
| A phase 2, randomized, double-blind, placebo-controlled, parallel-group, multicenter study to evaluate the safety and tolerability, efficacy, pharmacokinetics, pharmacodynamics, and immunogenicity of two dose regimens of empasiprubart (ARGX-117) in adults with multifocal motor neuropathy Empasiprubart for Multifocal Motor Neuropathy Advancing Phase 2 ARDA Study to Cohort 2 ARGX-117 Dose Regimen R a n d o miz atio n (2:1) Day 1 Screening and IVIg Monitoring Period 1st IVIg 2nd IVIg 3rd IVIg 16 weeks Placebo ARDA+ Long-term Extension Study Treatment-free Follow-up Phase 14 Primary Efficacy Measure • IVIg-retreatment Additional Efficacy Measures • Grip Strength – MRC sum score • MMN-RODS, CAP-PRI • Euro-Quality of Life 5 Dimensions 5 Levels • Timed Peg Board Test • Patient Global Impression of Change (PGIC) • Fatigue Severity Scale (FSS) |
| Our mission continues… Humility Innovation Excellence Co-Creation Empowerment 15 |
