6-K
Argenx SE (ARGX)
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 May 2022
Commission File Number: 001-38097
ARGENX SE
(Translation of registrant’s name into English)
Willemstraat54811 AH, Breda, 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): ¨
argenx SE
On May 5, 2022, argenx SE (the “Company”) issued press releases, copies of which are attached here as Exhibit 99.1 and 99.2 and are incorporated by reference herein.
Theinformation contained in this Current Report on Form 6-K, including Exhibit 99.1 and Exhibit 99.2, are incorporated byreference into the Company’s Registration Statements on Forms F-3 (File No. 333-258251)and S-8 (File Nos. 333-225375 and 333-258253).
EXHIBITS
| Exhibit | Description |
|---|---|
| 99.1 | Press Release dated May 5, 2022 |
| 99.2 | Press Release dated May 5, 2022 |
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: May 5, 2022 | By: | /s/ Hemamalini (Malini) Moorthy |
| Hemamalini (Malini) Moorthy | ||
| General Counsel |
Exhibit 99.1

argenx Reports First Quarter 2022 FinancialResults and Provides Business Update
- $21.2 million in VYVGART^®^ (efgartigimod alfa-fcab) net product sales during initial quarter of U.S. commercial launch
- Met primary endpoint in Phase 3 ADVANCE trial of VYVGART for treatment of primary immune thrombocytopenia (ITP)
- Japan commercial launch of VYVGART on track to start this month
- Management to host conference call today at 2:30 pm CET (8:30 am ET)
May 5, 2022
Breda, 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 financial results for the first quarter 2022 and provided a business update.
In a separate press release issued today, argenx also announced positive results from the Phase 3 ADVANCE trial evaluating VYVGART for the treatment of adult patients with ITP. The primary endpoint, demonstrating a significantly higher proportion of VYVGART-treated patients achieved a sustained platelet response than patients receiving placebo, and additional key secondary endpoints were met.
“Our VYVGART commercial launch is off to a strong start, underscoring the significant unmet need for a new treatment option in gMG. We are very encouraged by the early demand from patients and physicians and our team continues to meet the challenge with outstanding execution and deep engagement with our key stakeholders. We look forward to our imminent commercial launch in Japan and an upcoming regulatory decision in Europe, which support our goal to make VYVGART available worldwide. We are confident that the relationships we are building today with the gMG community will establish a strong foundation to continue to deliver on behalf of patients,” commented Tim Van Hauwermeiren, Chief Executive Officer of argenx.
“The positive readout from our first registrational ITP trial highlights the promise of efgartigimod as a pipeline-in-a-product with the potential to reach a variety of IgG-mediated autoimmune diseases, even beyond the ten indications we are currently evaluating. We are on track to achieve our argenx 2025 goal to build the next great immunology company while bringing breakthrough innovations to patients and creating long-term value for our stakeholders.”
FIRST QUARTER 2022 AND RECENT BUSINESS UPDATE
VYVGART Launch Progress
VYVGART is the first-approved neonatal Fc receptor (FcRn) blocker in the U.S. and Japan. VYVGART is approved in the U.S. for the treatment of adult generalized myasthenia gravis (gMG) patients who are anti-acetylcholine receptor (AChR) antibody positive and in Japan for adult gMG patients. The global launch strategy is on track to make VYVGART available in Europe, China and Canada, as well as select additional regions.

| - | Generated net product revenues of $21.2 million for first full quarter of VYVGART commercial launch in<br>U.S. |
|---|---|
| - | Japan commercial launch to start this month following addition of VYVGART to National Health Insurance<br>(NHI) drug price list on April 20, 2022 |
| - | Decision from European Medicines Agency on Marketing Authorization Application expected in second half<br>of 2022 |
| - | Zai Lab to file for approval in China in mid-2022 and Medison in Israel in second quarter of 2022 |
Efgartigimod Research and Development
argenx is positioned to expand its leadership position in FcRn blockade to include ten total autoimmune indications by the end of 2022. Six registrational trials are ongoing with four new proof-of-concept trials to start this year across multiple therapeutic franchises.
| - | Neuromuscular franchise |
|---|---|
| o | Biologics License Application (BLA) on track to be filed by end of year for subcutaneous (SC) efgartigimod<br>for gMG, following positive topline results from Phase 3 ADAPT-SC trial |
| --- | --- |
| o | Topline data from registrational ADHERE trial of SC efgartigimod for chronic inflammatory demyelinating<br>polyneuropathy (CIDP) expected in first quarter of 2023 |
| o | Registrational ALKIVIA trial on track to start this quarter for three subtypes of idiopathic inflammatory<br>myopathies (myositis), including immune-mediated necrotizing myopathy, anti-synthetase syndrome and dermatomyositis; interim analysis<br>planned of first 30 patients of each subtype |
| - | Hematology franchise |
| --- | --- |
| o | Positive topline data of VYVGART for primary ITP reported today |
| --- | --- |
| § | Primary endpoint was met; significantly higher proportion of patients receiving VYVGART achieved a sustained<br>platelet response than patients receiving placebo |
| --- | --- |
| § | Statistically significant separation from placebo in key platelet-derived secondary endpoints |
| § | Safety and tolerability profile confirmed in second indication |
| o | Topline data from second registrational ADVANCE-SC trial of SC efgartigimod for primary ITP expected in<br>first quarter of 2023 |
| --- | --- |
| - | Dermatology franchise |
| --- | --- |
| o | Enrollment expanded in registrational ADDRESS trial of SC efgartigimod for pemphigus vulgaris and foliaceus<br>in order to manage ongoing impact of war in Ukraine; topline data now expected in second half of 2023 |
| --- | --- |
| o | Registrational BALLAD trial ongoing of SC efgartigimod for bullous pemphigoid with interim analysis planned<br>of first 40 patients |

| - | Proof-of-concept trials to be launched in collaboration with Zai Lab and IQVIA |
|---|---|
| o | Zai Lab to launch Phase 2 trials in lupus nephritis and membranous nephropathy in 2022 with argenx to<br>lead global registrational programs for each potential indication |
| --- | --- |
| o | IQVIA to launch Phase 2 trials in primary Sjogren’s syndrome in second half of 2022 and COVID-19-mediated<br>postural orthostatic tachycardia syndrome (POTS) in mid-2022 |
Pipeline Progress
argenx is developing ARGX-117 and ARGX-119, which both have pipeline-in-a-product potential for multiple autoimmune indications.
| - | ARGX-117 (C2 inhibitor) |
|---|---|
| o | Proof-of-concept ARDA trial ongoing to evaluate safety, tolerability, and potential dosing regimen in<br>multifocal motor neuropathy (MMN) |
| --- | --- |
| o | Phase 2 proof-of-concept trial expected to start in 2022 for prevention of delayed graft function and/or<br>allograft failure after kidney transplantation |
| - | ARGX-119 (muscle-specific kinase (MuSK) agonist) |
| --- | --- |
| o | Phase 1 dose-escalation trial in healthy volunteers expected to start after Clinical Trial Application<br>filing in fourth quarter of 2022 |
| --- | --- |
| o | A subsequent Phase 1b trial will assess early signal detection in patients with congenital myasthenic<br>syndrome and MuSK-associated myasthenia gravis |
Upcoming Medical Meeting Presentations
| - | 14^th^ Myasthenia Gravis Foundation of America International Conference on Myasthenia and Related<br>Disorders (May 10-12, Miami, FL) |
|---|---|
| - | Society for Investigative Dermatology Annual Meeting (May 18-21, Portland, Oregon) |
| - | Annual Meeting of the Japanese Society of Neurology (May 18-22, Tokyo, Japan) |
| - | 8^th^ Congress of the European Academy of Neurology (June 25-28, Vienna, Austria) |
| - | 17^th^ International Congress on Neuromuscular Diseases (July 5-9, Brussels, Belgium) |

FIRST QUARTER 2022 FINANCIAL RESULTS (CONSOLIDATED)
| (in thousands of except for shares and EPS) | 2021 | Variance | ||||||
| Product net sales | 21,163 | $ | — | $ | 21,163 | |||
| Collaboration revenue | 2,249 | 158,155 | (155,906 | ) | ||||
| Other operating income | 8,068 | 20,412 | (12,344 | ) | ||||
| Total operating income | 31,480 | 178,567 | (147,087 | ) | ||||
| Cost of sales | (1,372 | ) | — | (1,372 | ) | |||
| Research and development expenses | (151,968 | ) | (122,328 | ) | (29,640 | ) | ||
| Selling, general and administrative expenses | (100,866 | ) | (56,253 | ) | (44,613 | ) | ||
| Total operating expenses | (254,206 | ) | (178,580 | ) | (75,626 | ) | ||
| Operating loss | (222,726 | ) | $ | (13 | ) | $ | (222,713 | ) |
| Financial income | 821 | 764 | 57 | |||||
| Financial expenses | (953 | ) | (1,184 | ) | 231 | |||
| Exchange gains/(losses) | (7,213 | ) | (28,817 | ) | 21,604 | |||
| Loss before taxes | (230,072 | ) | $ | (29,249 | ) | $ | (200,823 | ) |
| Income tax (expense) / benefit | 2,885 | $ | (11,184 | ) | $ | 14,069 | ||
| Loss for the period | (227,187 | ) | $ | (40,433 | ) | $ | (186,754 | ) |
| Weighted average number of shares outstanding | 52,084,335 | 49,946,515 | ||||||
| Basic and diluted loss per share (in ) | (4.36 | ) | (0.81 | ) | ||||
| Net increase/(decrease) in cash and cash equivalents and current financial assets compared to year-end 2021 and 2020 | 518,656 | $ | 910,903 | |||||
| Cash and cash equivalents and current financial assets at the end of the period | 2,855,384 | $ | 2,907,355 |
All values are in US Dollars.
DETAILS OF THE FINANCIAL RESULTS
Total operating income for the three months ended March 31, 2022 was $31.5 million, compared to $178.6 million for the three months ended March 31, 2021, and consists of:
| · | Product net sales from sales of VYVGART in the U.S. for the three months ended March 31, 2022<br>were $21.2 million, following the approval of VYVGART by the U.S. Food and Drug Administration (FDA) on December 17, 2021. No product<br>sales were recognized during the comparable prior period. |
|---|

| · | Collaboration revenue for the three months ended March 31, 2022 was $2.2 million, compared<br>to $158.2 million for three months ended March 31, 2021, resulting in a decrease of $155.9 million. The collaboration revenue for<br>the three months ended March 31, 2021 was primarily attributable to the closing of the strategic collaboration for efgartigimod with<br>Zai Lab, resulting in the recognition of $151.9 million in collaboration revenue. |
|---|---|
| · | Other operating income for the three months ended March 31, 2022 was $8.1 million, compared<br>to $20.4 million for three months ended March 31, 2021, resulting in a decrease of $12.3 million. During the three months ended March 31,<br>2021, the fair value of the argenx profit share in AgomAb Therapeutics NV increased by $11.2 million. There was no change in the fair<br>value during the three months ended March 31, 2022. |
Total operating expenses for the three months ended March 31, 2022 were $254.2 million, compared to $178.6 million for the three months ended March 31, 2021, and consists of:
| · | Cost of sales for the three months ended March 31, 2022 amounted to $1.4 million. The cost<br>of sales was recognized with respect to the sale of VYVGART in the U.S. during the first quarter of 2022. There was no cost of sales recognized<br>in the comparable prior period. |
|---|---|
| · | Research and development expenses increased by $29.6 million for the three months ended March 31,<br>2022 to $152.0 million, compared to $122.3 million for the three months ended March 31, 2021. The increase resulted primarily from<br>higher external research and development expenses, mainly related to the efgartigimod program in various indications and other clinical<br>and preclinical programs. |
| · | Selling, general and administrative expenses totaled $100.9 million for the three months ended<br>March 31, 2022, compared to $56.3 million for the three months ended March 31, 2021. The increase resulted primarily from higher<br>professional and marketing fees linked to the commercialization of VYVGART in the U.S. and Japan and higher personnel expenses increased<br>due to a planned increase in headcount. |
Exchange losses totaled $7.2 million for the three months ended March 31, 2022, compared to $28.9 million for the three months ended March 31, 2021 and are mainly attributable to unrealized exchange rate losses on cash, cash equivalents and current financial assets position in Euro.
Income tax totaled $2.9 million of tax income for the three months ended March 31, 2022, compared to $11.2 million of tax expense for the comparable prior period. Tax income for the three months ended March 31, 2022 consists of $5.0 million of income tax expense and $7.9 million of deferred tax income, compared to $6.2 million of income tax expense and $5 million of deferred tax expense for the comparable prior period.
Net loss for the three months ended March 31, 2022 was $227.2 million compared to $40.4 million for the comparable prior year period. On a per weighted average share basis, the net loss was $4.36 and $0.81 for the three months ended March 31, 2022 and 2021, respectively.

Cash, cash equivalents and current financialassets totaled $2,855.4 million as of March 31, 2022, compared to $2,336.7 million as of December 31, 2021. The increase in cash and cash equivalents and current financial assets resulted primarily from the closing of a global offering of shares, including a U.S. offering and a European private placement, which resulted in the receipt of $761.0 million in net proceeds in March 2022, partially offset by the net cash flows used in operating activities, primarily towards the commercial launch of VYVGART in the U.S. and Japan and continued investment in pipeline expansion.
FINANCIAL GUIDANCE
As of March 31, 2022, argenx had $2.9 billion in cash, cash equivalents and current financial assets. Based on current plans to fund anticipated operating expenses and capital expenditures, argenx expects to utilize approximately $1 billion of its available cash in 2022. The increased spend will support the global VYVGART launches, clinical development of efgartigimod in 10 indications and ARGX-117 in two indications, investment in the global supply chain, and continued focus on pipeline expansion through the Immunology Innovation Program.
EXPECTED 2022 FINANCIAL CALENDAR
| - | July 28, 2022: HY 2022 financial results and business update |
|---|---|
| - | October 27, 2022: Q3 2022 financial results and business update |
CONFERENCE CALL DETAILS
The first quarter 2022 business update will be discussed during a conference call and webcast presentation today at 2:30 pm CEST/8:30 am ET. A webcast of the live call may be accessed on the Investors section of the argenx website at argenx.com/investors. A replay of the webcast will be available on the argenx website.
Dial-in numbers:
Please dial in 15 minutes prior to the livecall.
Dial-in numbers:
Use the access code 073235 to join the call.Please dial in 15 minutes prior to the live call.
| Belgium | 32 800 548 13 |
|---|---|
| United Kingdom | 44 808 189 6484 |
| United States | 1 844 200 6205 |
| All other locations | 1 929 526 1599 |
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. and Japan. 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:
Kelsey Kirk
kkirk@argenx.com
Investors:
Beth DelGiacco
bdelgiacco@argenx.com
Michelle Greenblatt
mgreenblatt@argenx.com
Forward-looking Statements
The contents of this announcement include statementsthat are, or may be deemed to be, “forward-looking statements.” These forward-looking statements can be identified by theuse of forward-looking terminology, including the terms “believes,” “hope,” “estimates,” “anticipates,” “expects,” “intends,” “may,” “will,” or “should” and include statements argenxmakes concerning execution of its global launch strategy and expected therapy delivery to patients in Japan, Europe, China and Canada;the expected long-term safety, tolerability and efficacy of VYVGART® (efgartigimod alfa-fcab) in adult patients with Primary Immunethrombocytopenia; its expectation concerning its development pipeline and ability to deliver shareholder value as a result thereof; developmentof efgartigimod in up to ten indications and ARGX-117 in up to two indications by end of 2022; expected advancement of ARGX-119 into first-in-humanstudies; expected broad U.S. policy coverage of VYVGART by the end of second quarter 2022; the estimated number of covered patients inthe U.S.; anticipated pathway for approval in Japan and launch in the second quarter of 2022; plans for Medison to file for approval inIsrael in second quarter of 2022; partnership agreements expected to be announced in 2022; the timing and its expectations with respectto reporting data from registrational trials; expectations with respect to expansion of efgartigimod portfolio into ten indications byend of 2022; expected launch and timing of proof of concept trials, including by Zai Labs and IQVIA, and dose escalation trials in 2022;and its expectations with respect to its use of available cash and liquidity needs for 2022. 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, includingin argenx’s most recent annual report on Form 20-F filed with the SEC as well as subsequent filings and reports filed by argenxwith the SEC. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. Theseforward-looking statements speak only as of the date of publication of this document. argenx undertakes no obligation to publicly updateor revise the information in this press release, including any forward-looking statements, except as may be required by law.
Exhibit 99.2
argenxAnnounces Positive Phase 3 Data from ADVANCE Trial of VYVGART^®^ (efgartigimod alfa-fcab) in Adults with PrimaryImmune Thrombocytopenia
Study met primary endpoint, demonstrating ahigher proportion of sustained platelet response with VYVGART treatment compared to placebo (p=0.0316); responders observed across patienttypes regardless of prior therapy or disease severity
Statistically significant separation from placeboin key platelet-derived secondary endpoints
Safety and tolerability profile of VYVGART isconsistent with previous clinical trials; ADVANCE is first registrational trial with chronic dosing out to 24 weeks
Topline data expected in first quarter 2023from ADVANCE-SC, the second pivotal trial required for registration in primary immune thrombocytopenia (ITP)
argenx to host investor call today at 8:30amET / 2:30pm CET
Regulated Information/Inside Information
**Breda,the Netherlands—May 5, 2022—**argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced positive data from the Phase 3 ADVANCE trial of VYVGART^®^ (efgartigimod alfa-fcab)
in adults with primary ITP. ADVANCE met its primary endpoint demonstrating that a higher proportion of chronic ITP patients receiving VYVGART achieved a sustained platelet count response compared to placebo. ADVANCE is the first Phase 3 clinical trial of a neonatal Fc receptor (FcRn) blocker in ITP.
“Immune thrombocytopenia is a rare, debilitating autoimmune disease that can be very difficult to treat, especially in patients who have an insufficient response to previous ITP therapies. There is no clear standard of care and many patients continue to experience significant symptoms and decreased quality of life,” said Catherine Broome, M.D., Associate Professor of Medicine at Georgetown University Lombardi Comprehensive Cancer Center, and Principal Investigator in the ADVANCE study. “These data are very promising as they show that platelet counts can rapidly improve to clinically meaningful levels following VYVGART treatment in a proportion of a heavily pretreated patient population. We are excited that targeting pathogenic IgG autoantibodies could represent a new, potential approach to help alleviate the disease burden in this patient community.”
The ADVANCE trial enrolled 131 adult patients with chronic and persistent ITP. Patients were heavily pretreated and 67% of patients had received three or more prior ITP therapies, including 59% who had prior thrombopoietin receptor agonist (TPO-RAs) experience, 34% with prior rituximab experience and 37% with a history of splenectomy. Patients were insufficiently controlled at baseline with mean platelet counts of 17x10^9^/L across all patients. Of patients who completed the full ADVANCE study, 94% (63/67) of VYVGART-treated patients and 97% (38/39) of placebo patients continued to the ADVANCE+ open-label extension study.
Highlights of Phase 3 ADVANCE Data
Primary endpoint met
ADVANCE met its primary endpoint demonstrating a significantly higher proportion of patients with chronic ITP receiving VYVGART (17/78; 21.8%) compared to placebo (2/40; 5%) achieved a sustained platelet response (p=0.0316), defined as having platelet counts greater than or equal to 50x10^9^/L on at least four of the last six scheduled visits between weeks 19 and 24 of treatment.
Primary endpoint responders were observed across patient types regardless of age, disease severity, time since diagnosis, prior ITP treatment or background medication.
Key platelet-derived secondary endpoints demonstratedstatistical significance
Key platelet-derived secondary endpoints showed VYVGART-treated patients had a statistically significant benefit compared to placebo on (1) cumulative number of weeks where platelet counts were at least 50x10^9^/L in the chronic ITP population (p=0.0009) and (2) sustained platelet response in the overall population, including both chronic and persistent ITP patients (p=0.0108). Numerically fewer WHO-classified bleeding events occurred in treated patients throughout the trial but the difference from placebo was not statistically significant. A higher proportion of treated patients in the overall population achieved a durable, sustained platelet response compared to placebo, defined as a sustained platelet response on at least six of the last eight scheduled visits between weeks 17 and 24 of treatment (p=0.0265), but was not considered statistically significant based on hierarchical testing.
Additional secondary endpoints provided clinicallymeaningful data on platelet count responses throughout 24-week trial
Additional secondary endpoint data from the ADVANCE trial are consistent with primary and secondary platelet-derived endpoints and provide additional context on metrics that often drive treatment decisions.
| ● | International Working Group (IWG) responder status: 51.2% of VYVGART-treated patients were classified<br>as IWG responders and 27.9% as complete responders compared to 20% of placebo patients as IWG responders and 4.4% as complete responders.<br>IWG responders are defined as having a platelet count of at least 30x10^9^/L, a two-fold increase in platelet count from baseline,<br>and the absence of bleeding for two separate, consecutive weekly visits. Complete responders are patients with platelet counts of 100x10^9^/L<br>and the absence of bleeding for two separate, consecutive weekly visits. |
|---|---|
| ● | Mean platelet count change from baseline: VYVGART-treated patients demonstrated a rapid onset of<br>platelet count improvement with statistically significant separation from placebo observed at week one and maintained through 20 out of<br>24 weeks of the trial. |
| --- | --- |
| ● | Switch to biweekly dosing: Ten VYVGART-treated patients switched to a biweekly (every two weeks)<br>dosing schedule after achieving platelet counts of 100x10^9^/L for three out of four consecutive visits, compared to one placebo<br>patient. Nine of the ten treated patients achieved a sustained platelet response. |
| --- | --- |
Consistent safety and tolerability profile
ADVANCE is the second registrational trial of VYVGART and the first to evaluate chronic weekly dosing. VYVGART was well-tolerated in this 24-week study and the observed safety and tolerability profile was consistent with previous clinical trials.
“In listening to and learning from people in the ITP community, we understand the impact of living with this disease can extend beyond physical signs, taking a serious toll on a person’s quality of life. These compelling preliminary data emphasize the potential for VYVGART to drive responses in ITP regardless of prior lines of therapy, history of splenectomy or time from diagnosis. We look forward to learning more about the potential approach of targeting pathogenic IgGs in ITP through our ADVANCE-SC trial, which is on track to read out in the first quarter of next year,” said Luc Truyen, MD, Ph.D., Chief Medical Officer at argenx. “The totality of data generated thus far continue to support the key attributes of VYVGART, including its onset of action and safety profile. These data further reinforce our confidence in FcRn blockade as a precision tool with the potential to reach a broad spectrum of IgG-mediated severe autoimmune diseases.”
The Phase 3 ADVANCE trial is the first of two registrational trials being conducted as part of the ongoing ITP development program. ADVANCE-SC is evaluating subcutaneous efgartigimod for the treatment of primary ITP. Topline data from the ADVANCE-SC study are expected in the first quarter of 2023.
Phase 3 ADVANCE Trial Design
The Phase 3 ADVANCE trial was a randomized, double-blind, placebo-controlled, multicenter, global trial evaluating the efficacy and safety of VYVGART in adult patients with chronic or persistent primary ITP. A total of 131 adult patients with primary ITP in North America, Europe and Japan enrolled in the trial and received VYVGART or placebo for a total of 24 weeks as part of the primary trial. Enrolled patients had a confirmed ITP diagnosis and a mean entry platelet count of less than 30x10^9^/L. Patients were on a stable dose of at least one ITP treatment prior to randomization and had received at least one prior therapy. Concomitant medications permitted included corticosteroids, nonsteroidal immunosuppressive drugs, fostamatinib or TPO-RAs. If patients were on 'watch and wait' at baseline, they had to have received at least 2 prior treatments for ITP.
Patients were randomized in a 2:1 ratio to receive VYVGART or placebo for a total of 24 weeks as part of the primary trial. Randomized patients received weekly infusions from weeks 1-4 and were eligible to adjust frequency to bi-weekly depending on platelet count. Administration frequency was fixed from study visits 16-24. The primary endpoint was measured by the proportion of patients with chronic ITP with a sustained platelet count response defined as achieving platelet counts of greater than or equal to 50x10^9^/L for at least four of the last six scheduled visits between weeks 19 and 24. Patients who received rescue therapy at week 12 or later, or for whom dose and/or frequency of concurrent ITP therapies increased at week 12 or later, were considered non-responders. Key secondary endpoints included extent of disease control over 24-week treatment period, proportion of overall population with sustained platelet count response, incidence and severity of WHO-classified bleeding events and an extended primary endpoint analysis between weeks 17 and 24.
About Immune Thrombocytopenia (ITP)
Immune thrombocytopenia (ITP) is an autoimmune disorder where immunoglobulin G (IgG) autoantibodies destroy platelets and reduce platelet production, which can lead to an increased risk of excessive bleeding and bruising. In severe cases, frequent bleeding events can cause anemia or even brain hemorrhage in rare cases. ITP is also associated with debilitating fatigue and significant impacts on mental health, including anxiety, fear and depression. Many ITP patients are inadequately controlled on current therapies so there remains a significant unmet need for additional treatment options.
AboutVYVGART**^®^ (efgartigimod alfa-fcab)**
VYVGART is a human IgG1 antibody fragment that binds to the neonatal Fc receptor (FcRn), resulting in the reduction of circulating immunoglobulin G (IgG) autoantibodies. It is the first and only approved FcRn blocker. VYVGART is approved in the United States 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 is being studied in adults with primary immune thrombocytopenia (ITP) and other IgG autoantibody-mediated diseases.
Important Safety Information for VYVGART^®^(efgartigimodalfa-fcab) intravenous (IV) formulation (U.S. prescribing information) for the treatment of generalized myasthenia gravis (gMG) inadult patients who are anti-acetylcholine receptor (AChR) antibody positive.
What is VYVGART^®^ (efgartigimodalfa-fcab)?
VYVGART 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).
What is the most important information I should know about VYVGART?
VYVGART may cause serious side effects, including:
| ● | Infection. VYVGART may increase the risk of infection. In a clinical study, the most common<br>infections were urinary tract and respiratory tract infections. More patients on VYVGART vs placebo had below normal levels for white<br>blood cell counts, lymphocyte counts, and neutrophil counts. The majority of infections and blood side effects were mild to moderate in<br>severity. Your health care provider should check you for infections before starting treatment, during treatment, and after treatment with<br>VYVGART. Tell your health care provider if you have any history of infections. Tell your health care provider right away if you have signs<br>or symptoms of an infection during treatment with VYVGART such as fever, chills, frequent and/or painful urination, cough, pain and blockage<br>of nasal passages/sinus, wheezing, shortness of breath, fatigue, sore throat, excess phlegm, nasal discharge, back pain, and/or chest<br>pain. |
|---|---|
| ● | **Undesirable immune reactions (hypersensitivity reactions).**VYVGART can cause the immune system<br>to have undesirable reactions such as rashes, swelling under the skin, and shortness of breath. In clinical studies, the reactions<br>were mild or moderate and occurred within 1 hour to 3 weeks of administration, and the reactions did not lead to VYVGART discontinuation.<br>Your health care provider should monitor you during and after treatment and discontinue VYVGART if needed. Tell your health care provider<br>immediately about any undesirable reactions. |
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Before taking VYVGART, tell your health care provider about all of your medical conditions, including if you:
| ● | Have a history of infection or you think you have an infection. |
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| ● | Have received or are scheduled to receive a vaccine (immunization). Discuss with your health care provider<br>whether you need to receive age-appropriate immunizations before initiation of a new treatment cycle with VYVGART. The use of vaccines<br>during VYVGART treatment has not been studied, and the safety with live or live-attenuated vaccines is unknown. Administration of live<br>or live-attenuated vaccines is not recommended during treatment with VYVGART. |
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| ● | Are pregnant or plan to become pregnant and are breastfeeding or plan to breastfeed. |
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Tell your health care 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?
The most common side effects of VYVGART are respiratory tract infection, headache, and urinary tract infection.
These are not all the possible side effects of VYVGART. 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 and talk to your doctor.
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. and Japan. 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:
Kelsey Kirk
kkirk@argenx.com
Investors:
Beth DelGiacco
bdelgiacco@argenx.com
Michelle Greenblatt
mgreenblatt@argenx.com
Forward-looking Statements
*Thecontents of this announcement include statements that are, or may be deemed to be, “forward-looking statements.” These forward-lookingstatements 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 expected long-term safety, tolerability and efficacy of VYVGART®(efgartigimod alfa-fcab) in adult patients with Primary Immune thrombocytopenia. By their nature, forward-looking statements involve risksand uncertainties and readers are cautioned that any such forward-looking statements are not guarantees of future performance. argenx’sactual 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 ExchangeCommission (SEC) filings and reports, including in argenx’s most recent annual report on Form 20-F filed with the SEC as wellas subsequent filings and reports filed by argenx with the SEC. Given these uncertainties, the reader is advised not to place any unduereliance 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.