作者: Holak, Tad A. ; Sitar, Tomasz ; Kitel, Radoslaw ; Bielski, Przemyslaw ; Musielak, Bogdan ; Barczynski, Jan ; Tyrcha, Urszula ; Hec-Galazka, Aleksandra ; Plewka, Jacek ; Mikitiuk, Michal ; Gudz, Ganna P.

Therapeutic antibodies directed against either programmed cell death-1 protein (PD-1) or its ligand PD-L1 have demonstrated efficacy in the treatment of various cancers.In contrast with antibodies, small mols. have the potential for increased tissue penetration; better pharmacol.; and therefore, improved antitumor activity.A series of nonsym. C2 inhibitors were synthesized and evaluated for PD-1/PD-L1 interaction inhibition.These compounds induced PD-L1 dimerization and effectively blocked PD-L1/PD-1 interaction in a homogeneous time-resolved fluorescence (HTRF) assay with most inhibitors exhibiting IC50 values in the single-digit nM range and below.Their high inhibitory potency was also demonstrated in a cell-based coculture PD-1 signaling assay where 2 exhibited an EC50 inhibitory activity of 21.8 nM, which approached that of the PD-L1 antibody durvalumab (EC50 = 0.3-1.8 nM).Structural insight into how these inhibitors interact with PD-L1 was gained by using NMR and X-ray cocrystal structure studies.These data support further preclin. evaluation of these compounds as antibody alternatives.

2021-05-01·The Lancet Haematology1区 · 医学

Addition of iptacopan, an oral factor B inhibitor, to eculizumab in patients with paroxysmal nocturnal haemoglobinuria and active haemolysis: an open-label, single-arm, phase 2, proof-of-concept trial

1区 · 医学

Article

作者: Benajiba, Lina ; Soret, Juliette ; Junge, Guido ; End, Peter ; Risitano, Antonio M ; Peffault de Latour, Régis ; Nidamarthy, Prasanna K ; Milojevic, Julie ; Frieri, Camilla ; Röth, Alexander ; de Fontbrune, Flore Sicre ; Marotta, Serena ; Rozenberg, Izabela ; Marano, Luana ; Alashkar, Ferras

BACKGROUNDThe haematological benefit of standard-of-care anti-C5 treatment for haemolytic paroxysmal nocturnal haemoglobinuria is limited by residual intravascular haemolysis or emerging C3-mediated extravascular haemolysis. Therefore, the aim of this phase 2 study was to assess the safety, tolerability, pharmacokinetics and pharmacodynamics, and activity of the new complement factor B inhibitor, iptacopan, in patients with paroxysmal nocturnal haemoglobinuria who have active haemolysis despite anti-C5 therapy.METHODSIn this multicentre, open-label, single-arm, phase 2 trial, we enrolled adult patients (aged 18-80 years) with paroxysmal nocturnal haemoglobinuria who showed signs of active haemolysis despite receiving eculizumab treatment. Patients were enrolled at Federico II University Hospital (Naples, Italy), Hôpital Saint-Louis (Paris, France), and University Hospital Essen (Essen, Germany). For enrolment, patients were required to show lactate dehydrogenase more than 1·5-times the upper limit of normal and a paroxysmal nocturnal haemoglobinuria type 3 erythrocyte or granulocyte clone size of 10% or greater. Patients with bone marrow failure, on systemic steroid or immunosuppressive drugs, or with severe comorbidities were excluded from the study. Iptacopan was given orally as an add-on therapy at a dose of 200 mg twice daily. The primary endpoint was the effect of iptacopan on the reduction of chronic residual intravascular haemolysis measured as change in lactate dehydrogenase from baseline value to week 13. At 13 weeks, patients could opt into a long-term study extension (ongoing), allowing for modifications of standard treatment. This trial is registered at ClinicialTrials.gov, NCT03439839.FINDINGSBetween May 31, 2018, and April 9, 2019, ten patients had twice daily 200 mg iptacopan. Iptacopan resulted in marked reduction of lactate dehydrogenase from baseline versus at week 13 (mean 539 IU/L [SD 263] vs 235 IU/L [44], change from baseline -309·2 IU/L [SD 265·5], 90% CI -473·77 to -144·68, p=0·0081), associated with significant improvement of haemoglobin concentrations (mean 97·7 g/L [SD 10·5] vs 129·5 g/L [18·3] change from baseline 31·9 g/L [14·5], 90% CI 23·42-40·28, p<0·0001). All biomarkers of haemolysis improved on iptacopan treatment. Observed haematological benefits were maintained longer than the 13-week study period, throughout the study extension, including seven patients who stopped concomitant standard-of-care treatment and continued iptacopan as monotherapy. There were no deaths or treatment-related serious adverse events during the study period. Of three non-related serious adverse events, two occurred in the same patient (one during run-in and before exposure to iptacopan).INTERPRETATIONIptacopan at a chronic dose of 200 mg twice daily was well tolerated without any major drug-related safety findings and shows lactate dehydrogenase reduction and haemoglobin normalisation in most patients with paroxysmal nocturnal haemoglobinuria at week 13 and beyond, even in monotherapy. On the basis of these data, iptacopan will be tested as monotherapy in pivotal trials investigating its haematological benefit in a broader paroxysmal nocturnal haemoglobinuria population.FUNDINGNovartis Institutes for Biomedical Research.

2014-04-24·Blood1区 · 医学

Decrypting C2 inhibitors

1区 · 医学

Article

作者: Voorberg, Jan ; Meems, Henriët

A review.In this issue of Blood, Nguyen et al employ high-resolution mapping to precisely define epitopes on the C2 domain of blood coagulation factor VIII (FVIII).Their results nicely complement a recent report in Blood describing the structure of a ternary complex of 2 inhibitory antibodies with the C2 domain.Together, these studies reveal fascinating mol. details on the unexpectedly large number of exposed surfaces in the C2 domain that contribute to the binding of inhibitory antibodies.These findings are relevant in the context of neutralizing anti-FVIII antibodies that develop in patients with hemophilia A.Insight into the antigenic properties of the C2 domain is needed to design FVIII variants with decreased antigenicity.Pioneering studies by Dorothea Scandella on the epitope mapping of FVIII inhibitors already pointed toward the C2 domain as a major binding site for FVIII inhibitors.The recent studies by Nguyen and Walter, combined with earlier work by Meeks et al, have provided evidence for 3 major binding sites for inhibitory anti-FVIII antibodies within the C2 domain.The overall dimensions of the C2 domain are small when compared to antibodies.Nevertheless, at least 2 and probably 3 monoclonal antibodies can simultaneously bind to the C2 domain.The mapping studies reported by Nguyen also suggest the presence of 3 distinct clusters of surface-exposed side chains on the FVIII C2 domain (see figure).

项与 Oral C2 Inhibitor 相关的新闻（医药）

2024-10-31

·GlobeNewswire-Health Care

argenx Reports Third Quarter 2024 Financial Results and Provides Business Update

$573 million in third quarter global net product sales CIDP global expansion on track, with decisions on approval under review in Japan, Europe, China, and Canada Management to host conference call today at 1:30 PM CET (8:30 AM ET) Regulated information - Inside information October 31, 2024 7:00AM 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 announced its third quarter 2024 financial results and provided a business update. “We delivered significant patient impact with VYVGART over the quarter, expanding our gMG footprint and delivering innovation to CIDP patients three months into launch,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx. “We continued to advance our goal of reaching more gMG patients earlier in their treatment journey, supported by VYVGART’s strong safety and efficacy profile, and real-world data showing the ability to meaningfully reduce steroid use. Expanding upon our leadership in gMG, we are now paving the future in CIDP. The strength of our data, combined with execution across the team to reach key stakeholders, contributed to the initial success of our CIDP launch, with more than 300 patients on therapy at the end of the third quarter. There remains significant opportunity ahead as we work towards achieving our Vision 2030, with innovation implemented across our pipeline to deliver transformative outcomes to more patients.” Advancing ‘Vision 2030’ in Third Quarter 2024 Vision 2030 is the next phase in argenx’s long-term commitment to transform the treatment of autoimmune diseases by strengthening its leadership in FcRn biology, investing in its continuous pipeline of differentiated antibody candidates, and scaling in a disciplined way to ensure innovation remains core to the argenx mission. As a part of this vision, argenx plans to reach at least 50,000 patients globally, advance the pipeline to achieve 10 labeled indications, and bring five new molecules into Phase 3 by 2030. Reaching 50,000 Patients Globally by 2030 VYVGART® (efgartigimod alfa-fcab) is a first-in-class antibody fragment targeting FcRn and is now approved for both intravenous use and subcutaneous injection (SC) (efgartigimod alfa and hyaluronidase-qvfc) in three indications, including generalized myasthenia gravis (gMG) globally, primary immune thrombocytopenia (ITP) in Japan (IV only), and chronic inflammatory demyelinating polyneuropathy (CIDP) in the U.S. (SC only). Generated global net product revenues (inclusive of both VYVGART and VYVGART SC) of $573 million in the third quarter of 2024Multiple VYVGART regulatory submissions completed or underway for gMG, including: Swissmedic approved VYVGART for the treatment of gMG in SwitzerlandRegulatory decisions on approval expected in Australia and Saudi Arabia in 2024, and South Korea in 2025 Multiple VYVGART SC regulatory submissions under review or planned for CIDP, including: Regulatory submissions completed in Japan, Europe, and China with decisions on approval expected in 2025Regulatory submission to be completed in Canada by end of 2024 VYVGART now reimbursed in 11 countries in Europe, with new agreements in place in France, Luxembourg, and BelgiumFDA review of VYVGART SC pre-filled syringe (PFS) for gMG and CIDP ongoing with Prescription Drug User Fee Act (PDUFA) target action date of April 10, 2025 Advancing Pipeline to Achieve 10 Labeled Indications by 2030 argenx continues to demonstrate breadth and depth within its immunology pipeline, advancing multiple pipeline-in-a-product candidates. argenx is solidifying its leadership in FcRn biology, with efgartigimod currently in development in 15 indications. argenx is also advancing its first-in-class C2 inhibitor, empasiprubart, which is being evaluated in multifocal motor neuropathy (MMN), delayed graft function (DGF), dermatomyositis (DM), and CIDP. In addition, argenx is evaluating ARGX-119, a muscle-specific kinase (MuSK) agonist in both congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS). Registrational studies ongoing of efgartigimod in thyroid eye disease (TED)Registrational studies ongoing to support label-expansion into broader MG, including ADAPT SERON in seronegative gMG and ADAPT OCULUS in ocular MGRegistrational study in primary Sjögren’s disease (SjD) on track to start by end of 2024Confirmatory study of efgartigimod in primary ITP to start by end of 2024 to enable registration in U.S.Topline data from seamless Phase 2/3 ALKIVIA study evaluating efgartigimod across three myositis subsets (immune-mediated necrotizing myopathy (IMNM), anti-synthetase syndrome (ASyS), and DM) expected by end of 2024Update on BALLAD study development plan evaluating efgartigimod in bullous pemphigoid (BP) expected by end of 2024Decision made to discontinue development of efgartigimod in membranous nephropathy (MN); proof-of concept study ongoing with efgartigimod in lupus nephritis (LN)Proof-of-concept study ongoing with efgartigimod in antibody mediated rejection (AMR), with systemic sclerosis (SSc) to start by end of 2024Registrational study of empasiprubart in MMN to start by end of 2024Additional proof-of-concept studies of empasiprubart ongoing, including VARVARA study in DGF and EMPACIFIC study in DMRegistrational study of empasiprubart in CIDP to start in 2025Ongoing Phase 1b/2a studies of ARGX-119 to assess early signal in patients with CMS and ALS Investing in Immunology Innovation Program to Support Five New Molecules in Phase 3 by 2030 argenx continues to invest in its Immunology Innovation Program (IIP) to drive long-term sustainable pipeline growth. Through the IIP, four new pipeline candidates have been nominated, including: ARGX-213, targeting FcRn and further solidifying argenx’s leadership in this new class of medicine; ARGX-121, a first-in-class molecule targeting IgA; ARGX-109, targeting IL-6, which plays an important role in inflammation, and ARGX-220, a first-in-class sweeping antibody for which the target has not yet been disclosed. Phase 1 studies of ARGX-213 and ARGX-121 expected to start in second half of 2025Investigational new drug (IND) applications for ARGX-220 and ARGX-109 on track to be filed by end of 2025 THIRD QUARTER 2024 FINANCIAL RESULTS argenx SE UNAUDITED CONDENSED CONSOLIDATED INTERIM STATEMENTS OF PROFIT OR LOSS Three Months Ended Nine Months Ended September 30 September 30(in thousands of $ except for shares and EPS) 2024 2023 2024 2023Product net sales $ 572,997 $ 329,097 $ 1,448,915 $ 816,432 Collaboration revenue 239 692 2,905 3,047 Other operating income 15,642 10,050 38,999 31,275 Total operating income $ 588,878 $ 339,839 $ 1,490,819 $ 850,754 Cost of sales $ (59,072) $ (35,999) $ (154,633) $ (78,358)Research and development expenses (235,940) (191,755) (686,195) (553,119)Selling, general and administrative expenses (277,698) (191,930) (769,392) (503,079)Loss from investment in a joint venture (1,981) (743) (5,294) (2,623)Total operating expenses $ (574,691) $ (420,427) $ (1,615,514) $ (1,137,179) Operating profit/(loss) $ 14,187 $ (80,588) $ (124,695) $ (286,425) Financial income $ 40,586 $ 30,049 $ 118,414 $ 67,078 Financial expense (676) (231) (1,760) (626)Exchange gains/(losses) 33,927 (32,509) 6,712 (23,345) Profit/(loss) for the period before taxes $ 88,024 $ (83,279) $ (1,329) $ (243,318)Income tax (expense)/benefit $ 3,386 $ 10,637 $ 60,208 $ 47,437 Profit/(loss) for the period $ 91,410 $ (72,642) $ 58,879 $ (195,881)Profit/(loss) for the period attributable to: Owners of the parent $ 91,410 $ (72,642) $ 58,879 $ (195,881)Weighted average number of shares outstanding 60,087,498 58,128,233 59,633,179 56,512,254 Basic profit/(loss) per share (in $) 1.52 (1.25) 0.99 (3.47)Diluted profit/(loss) per share (in $) 1.39 (1.25) 0.91 (3.47)Net increase in cash, cash equivalents and current financial assets compared to year-end 2023 and 2022 $ 194,523 $ 993,035 Cash and cash equivalents and current financial assets at the end of the period $ 3,374,367 $ 3,185,583 DETAILS OF THE FINANCIAL RESULTS Total operating income for the third quarter and year-to-date in 2024 was $589 million and $1,491 million, respectively, compared to $340 million and $851 million for the same periods in 2023, and mainly consists of: Product net sales of VYVGART for the three months ended and nine months ended September 30, 2024, were $573 million and $1,449 million, compared to $329 million and $816 million for the same periods in 2023.Other operating income for the third quarter and year-to-date in 2024 was $16 million and $39 million, respectively, compared to $10 million, and $31 million for the same periods in 2023. The other operating income for the three and nine months ended September 30, 2024 primarily relates to research and development tax incentives and payroll tax rebates. Total operating expenses for the third quarter and year-to-date in 2024 were $575 million and $1,616 million, respectively, compared to $420 million and $1,137 million for the same periods in 2023, and mainly consists of: Cost of sales for the third quarter and year-to-date in 2024 was $59 million and $155 million, respectively, compared to $36 million and $78 million for the same periods in 2023. The cost of sales was recognized with respect to the sale of VYVGART and VYVGART Hytrulo.Research and development expenses for the third quarter and year-to-date in 2024 were $236 million and $686 million, respectively, compared to $192 million and $553 million for the same periods in 2023. The research and development expenses mainly relate to external research and development expenses and personnel expenses incurred in the clinical development of efgartigimod in various indications and the expansion of other clinical and preclinical pipeline candidates.Selling, general and administrative expenses for the third quarter and year-to-date in 2024 were $278 million and $769 million, respectively, compared to $192 million and $503 million for the same periods in 2023. The selling, general and administrative expenses mainly relate to professional and marketing fees linked to global commercialization of VYVGART and VYVGART Hytrulo, and personnel expenses. Financial income for the third quarter and year-to-date in 2024 was $41 million and $118 million, respectively, compared to $30 million and $67 million for the same periods in 2023. The increase in financial income is mainly due to an increase in interest income on current financial assets and cash and cash equivalents. Exchange gains for the third quarter and year-to-date in 2024 were $34 million and $7 million respectively, respectively, compared to $(33) million and $(23) million of exchange losses for the same periods in 2023. Exchange gains/losses are mainly attributable to unrealized exchange rate gains or losses on the cash, cash equivalents and current financial assets position in Euro. Income tax for the third quarter and year-to-date in 2024 was $3 million and $60 million of tax benefit, respectively, compared to $11 million and $47 million of tax benefit for the same periods in 2023. Tax benefit for the nine months ended September 30, 2024 consists of $29 million of income tax expense and $89 million of deferred tax income, compared to $24 million of income tax expense and $71 million of deferred tax income for the comparable prior period. Net income for the three and nine month periods ended September 30, 2024, was $91 million and $59 million, respectively, compared to a net loss of $(73) million and $(196) million over the prior year periods. On a per weighted average share basis, the earnings per share was $0.99 for the nine months ended September 30, 2024 and a net loss per share of $(3.47) for the nine months ended September 30, 2023. Cash, cash equivalents and current financial assets totaled $3.4 billion as of September 30, 2024, compared to $3.2 billion as of December 31, 2023. The increases in cash and cash equivalents and current financial assets over the period was from financing activities due to the exercise of stock options which is offset by net cash flows used in operating and investing activities. FINANCIAL GUIDANCE With the increase in cash, cash equivalents and current financial assets in the quarter and year-to-date, the previously issued cash guidance no longer applies. The financial guidance on the combined selling, general and administrative expenses and research and development expenses remains unchanged at approximately $2 billion. EXPECTED 2024 FINANCIAL CALENDAR February 27, 2025: Full-year 2024 financial results and 4Q 2024 business update CONFERENCE CALL DETAILS The third quarter 2024 financial results and business update will be discussed during a conference call and webcast presentation today at 1:30 pm CET/8:30 am ET. A webcast of the live call and replay may be accessed on the Investors section of the argenx website at argenx.com/investors. Dial-in numbers: Please dial in 15 minutes prior to the live call. Belgium 32 800 50 201France 33 800 943355Netherlands 31 20 795 1090United Kingdom 44 800 358 0970United States 1 888 415 4250Japan 81 3 4578 9081Switzerland 41 43 210 11 32 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 in the U.S., Japan, Israel, the EU, the UK, China and Canada. The Company is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit www.argenx.com, and follow us on LinkedIn, X/Twitter, Instagram, Facebook, and YouTube. For further information, please contact: Media: Ben Petokbpetok@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 “aim,” “achieve,” “advance,” “bring,” “complete,” “expect,” “initiate,” “plan,” “reach,” “start,” “support,” “to be,” or “will,” and include statements argenx makes regarding its goal to reach more gMG patients in an early-line setting; its growth opportunity; its Vision 2030 plan, including (1) transforming the treatment of autoimmune diseases by strengthening its leadership in FcRn biology, (2) investing in its continuous pipeline of differentiated antibody candidates, (3) scaling in a disciplined way to ensure innovation, and (4) reaching 50,000 patients globally with an argenx medicine, 10 labeled indications across all approved assets, and five new molecules in Phase 3 development; the advancement of anticipated clinical development, data readouts and regulatory milestones and plans, including (1) the anticipated timing of additional VYVGART regulatory decisions on approval for gMG in Australia, Saudi Arabia, and South Korea, (2) the anticipated timing of VYVGART SC regulatory submissions for CIDP, including decisions on approval in China, Japan, and Europe and an anticipated regulatory submission filing in Canada, (3) the anticipated timing of the initiation of a registrational study for efgartigimod in primary SjD, (4) the anticipated timing of a confirmatory study of efgartigimod in primary ITP, (5) the anticipated timing of topline data from Phase 2/3 ALKIVIA study evaluating efgartigimod across three myositis subsets, (6) the anticipated timing of an update on the BALLAD study development plan evaluating efgartigimod in BP, (7) the anticipated timing of proof-of-concept study data for efgartigimod in LN, (8) the anticipated timing of the initiation of a study on efgartimod in SSc, (9) the anticipated timing of the initiation of a registrational study of empasiprubart for MMN, (9) the anticipated timing of the initiation of a registrational study of empasiprubart in CIDP, (10) the anticipated timing of the initiation of Phase 1 studies of ARGX-213 and ARGX-121, (11) the anticipated timing of the filing of investigational new drug applications for ARGX-220 and ARGX-109; the advancement of empasiprubart; its 2024 selling, general and administrative expenses and research and development expenses; the anticipated timing of releases of future financial results and business updates; and its goal of translating immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. By their nature, forward-looking statements involve risks and uncertainties and readers are cautioned that any such forward-looking statements are not guarantees of future performance. argenx’s actual results may differ materially from those predicted by the forward-looking statements as a result of various important factors, including the results of argenx's clinical trials; expectations regarding the inherent uncertainties associated with the development of novel drug therapies; preclinical and clinical trial and product development activities and regulatory approval requirements in products and product candidates; the acceptance of argenx's products and product candidates by patients as safe, effective and cost-effective; the impact of governmental laws and regulations on our business; disruptions caused on our reliance of third parties suppliers, service provides and manufacturing; inflation and deflation and the corresponding fluctuations in interest rates; and regional instability and conflicts. A further list and description of these risks, uncertainties and other risks can be found in argenx’s U.S. Securities and Exchange Commission (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.

上市批准财报临床1期免疫疗法

2024-07-25

·GlobeNewswire-Health Care

argenx Reports Half Year 2024 Financial Results and Provides Second Quarter Business Update

$478 million in second quarter global net product sales First CIDP patients treated with VYVGART® Hytrulo following June 21st FDA approval On track to begin four additional registrational studies across efgartigimod and empasiprubart by end of 2024 Management to host conference call today at 2:30 PM CET (8:30 AM ET) Regulated Information – Inside Information July 25, 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 announced its half year 2024 results and provided a second quarter business update. “We were excited to unveil our ambition for the future of argenx – Vision 2030 – last week, outlining our plan to develop and deliver continued and sustainable innovation for patients,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx. “We are already delivering on this promise with impressive commercial execution throughout the first half of the year, expanding our patient reach in MG, and launching in CIDP with our broad FDA label. Our development pipeline is stronger than ever, driven by our unique innovation engine. And we are well-positioned to capture the sizeable growth opportunity before us as we seek to reach earlier-line MG patients over the next 12-18 months with potential label expansions and a pre-filled syringe.” Vision 2030 During its R&D Day on July 16, 2024, argenx unveiled its ‘Vision 2030’ as part of its long-term commitment to transform the treatment of autoimmune diseases by strengthening its leadership in neonatal Fc receptor (FcRn) biology, investing in its continuous pipeline of differentiated antibody candidates, and scaling in a disciplined way to ensure innovation remains core to the argenx mission. ‘Vision 2030’ includes the following goals: 50,000 patients globally on treatment with an argenx medicine10 labeled indications across all approved assets, including VYVGART and potentially empasiprubart and ARGX-119Five new molecules in Phase 3 development indicating ongoing investment in internal discovery engine, the Immunology Innovation Program Reaching 50,000 Patients Globally VYVGART (efgartigimod alfa-fcab) is a first-in-class antibody fragment targeting FcRn and is now approved for both intravenous use and subcutaneous injection (SC) (efgartigimod alfa and hyaluronidase-qvfc) in three indications, including generalized myasthenia gravis (gMG) globally, primary immune thrombocytopenia (ITP) in Japan, and chronic inflammatory demyelinating polyneuropathy (CIDP) in the U.S. Generated global net product sales (inclusive of both VYVGART and VYVGART SC) of $478 million in second quarter of 2024National Medical Products Administration (NMPA) approved VYVGART SC for treatment of gMG in China through Zai Lab on July 16, 2024Additional VYVGART regulatory decisions on approval expected for gMG in 2024, including in Switzerland, Australia, and Saudi ArabiaLaunched VYVGART Hytrulo in CIDP in U.S. with first patients injected in JulyMultiple VYVGART SC regulatory submissions under review or planned for CIDP, including: Regulatory submissions completed in China, Japan, and Europe with decisions on approval expected in 2025Regulatory submission filing in Canada by end of 2024 Announced plan to evaluate VYVGART in ocular MG with registrational study (ADAPT OCULUS) to start by end of year; OCULUS to support label-expansion strategy into broader MG populations along with ongoing ADAPT SERON study in seronegative MGRegulatory submission filed and under review for VYVGART SC pre-filled syringe (PFS) for gMG and CIDP Advancing Pipeline to Achieve 10 Labeled Indications by 2030 argenx continues to demonstrate breadth and depth within its immunology pipeline, advancing multiple pipeline-in-a-product candidates. argenx is solidifying its leadership in FcRn biology, with efgartigimod currently in development in 15 indications. argenx is also advancing its first-in-class C2 inhibitor, empasiprubart, which is being evaluated in multifocal motor neuropathy (MMN), delayed graft function (DGF), dermatomyositis (DM), and CIDP. In addition, argenx is evaluating ARGX-119, a muscle-specific kinase (MuSK) agonist in both congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS). Registrational studies ongoing of efgartigimod in thyroid eye disease (TED)Advancing development of efgartigimod in primary Sjogren’s disease (SjD) with Phase 3 study to begin by end of 2024Following alignment meeting with FDA, confirmatory study of VYVGART (IV) in primary ITP to start by end of 2024 to enable registration in U.S.Topline data from seamless Phase 2/3 ALKIVIA study evaluating efgartigimod across three myositis subsets (immune-mediated necrotizing myopathy (IMNM), anti-synthetase syndrome (ASyS), and DM expected in fourth quarter of 2024Update on BALLAD study development plan evaluating efgartigimod in bullous pemphigoid (BP) expected by end of 2024Proof-of-concept studies ongoing with efgartigimod in membranous nephropathy (MN) and lupus nephritis (LN) with studies expected to initiate this year in antibody mediated rejection (AMR) and systemic sclerosis (SSc)Phase 3 study of empasiprubart for MMN to initiate in fourth quarter of 2024CIDP nominated as fourth empasiprubart indication, recognizing opportunity to bring multiple innovative treatments to patientsPhase 1b/2a studies of ARGX-119 to assess early signal detection in patients with CMS and ALS to start by end of 2024 Investing in Immunology Innovation Program to Support Five New Molecules in Phase 3 by 2030 argenx continues to invest in its Immunology Innovation Program (IIP) to drive long-term sustainable pipeline growth. Through the IIP, four new pipeline candidates have been nominated, including: ARGX-213, targeting FcRn and further solidifying argenx’s leadership in this new class of medicine; ARGX-121, a first-in-class molecule targeting IgA; ARGX-109, targeting IL-6, which plays an important role in inflammation, and ARGX-220, a first-in-class sweeping antibody for which the target has not yet been disclosed. Phase 1 studies of ARGX-213 and ARGX-121 expected to start in second half of 2025Investigational new drug (IND) applications for ARGX-220 and ARGX-109 on track to be filed by end of 2025 SECOND QUARTER 2024 FINANCIAL RESULTS argenx SEUNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF PROFIT OR LOSS Three Months Ended Six Months Ended June 30, June 30,(in thousands of $ except for shares and EPS) 2024 2023 2024 2023Product net sales $477,635 $269,313 $875,918 $487,335Collaboration revenue - 1,237 2,718 2,355Other operating income 11,793 10,485 23,305 21,225 Total operating income $489,428 $281,035 $901,941 $510,915 Cost of sales $(52,383) $(24,024) $(95,561) $(42,359)Research and development expenses (225,286) (195,509) (450,255) (361,364)Selling, general and administrative expenses (255,699) (161,977) (491,694) (311,149)Loss from investment in joint venture (1,521) (1,619) (3,313) (1,880)Total operating expenses $(534,889) $(383,129) $(1,040,823) (716,752) Operating loss $(45,461) $(102,094) $(138,882) $(205,837) Financial income $38,933 $20,441 $77,828 $37,029Financial expense (572) (207) (1,084) (395)Exchange gains/(losses) (7,903) (2,001) (27,215) 9,164 Loss for the period before taxes $(15,003) $(83,861) $(89,353) $(160,039)Income tax benefit/(expense) $44,069 $(10,507) $56,822 $36,800Profit/(loss) for the period $29,066 $(94,368) $(32,531) $(123,239)Profit/(loss) for the period attributable to: Owners of the parent $29,066 $(94,368) $(32,531) $(123,239)Weighted average number of shares outstanding 59,490,437 55,828,239 59,400,217 55,690,873Basic profit/(loss) per share (in $) 0.49 (1.69) (0.55) (2.21)Diluted profit/(loss) per share (in $) 0.45 (1.69) (0.55) (2.21)Net increase/(decrease) in cash, cash equivalents and current financial assets compared to year-end 2022 and 2023 (77,497) $(195,580)Cash and cash equivalents and current financial assets at the end of the period 3,102,347 $1,996,968 DETAILS OF THE FINANCIAL RESULTS Total operating income for the three and six months ended June 30, 2024 was $489 million and $902 million compared to $281 million and $511 million for the same periods in 2023, and mainly consists of: Product net sales of VYVGART and VYVGART SC for the three and six months ended June 30, 2024 were $478 million and $876 million compared to $269 million and $487 million for the same periods in 2023.Other operating income for the three and six months ended June 30, 2024 was $12 million and $23 million compared to $10 million and $21 million for the same periods in 2023. The other operating income for the three and six months ended June 30, 2024 and 2023, primarily relates to research and development tax incentives. Total operating expenses for the three and six months ended June 30, 2024 were $535 million and $1,041 million compared to $383 million and $717 million for the same periods in 2023, and mainly consists of: Cost of sales for the three and six months ended June 30, 2024 was $52 million and $96 million compared to $24 million and $42 million for the same periods in 2023. The cost of sales was recognized with respect to the sale of VYVGART and VYVGART SC.Research and development expenses for the three and six months ended June 30, 2024 were $225 million and $450 million compared to $196 million and $361 million for the same periods in 2023. The research and development expenses mainly relate to external research and development expenses and personnel expenses incurred in the clinical development of efgartigimod in various indications and the expansion of other clinical and preclinical pipeline candidates.Selling, general and administrative expenses for the three and six months ended June 30, 2024 were $256 million and $492 million compared to $162 million and $311 million for the same periods in 2023. The selling, general and administrative expenses mainly relate to professional and marketing fees linked to the commercialization of VYVGART and VYVGART SC, and personnel expenses. Financial income for the three and six months ended June 30, 2024 was $39 million and $78 million compared to $20 million and $37 million for the same periods in 2023. The increase in financial income is mainly due to an increase in interest income coming from an increase of cash, cash equivalents and current financial assets as a result of the July 2023 financing round. Exchange losses for the three and six months ended June 30, 2024 were $8 million and $27 million compared to $2 million exchange losses and $9 million of exchange gains for the same periods in 2023. Exchange gains/losses are mainly attributable to unrealized exchange rate gains or losses on the cash, cash equivalents and current financial assets denominated in Euro. Income tax for the three and six months ended June 30, 2024 was $44 million and $57 million of income tax benefit, respectively, compared to $11 million of income tax expense and $37 million of income tax benefit for the same periods in 2023. Net Result for the three and six months ended June 30, 2024 was $29 million profit and $33 million loss compared to $94 million and $123 million loss for the same periods in 2023. On a per weighted average share basis, the basic profit was $0.49 and diluted profit was $0.45 for the three months ended June 30, 2024, compared to a basic and diluted loss of $1.69 for the same period in 2023. On a per weighted average share basis, the basic and diluted loss was $0.55 for the six months ended June 30, 2024, compared to a basic and diluted loss of $2.21 for the same period in 2023. Cash, cash equivalents and current financial assets totalled $3.1 billion as of June 30, 2024, compared to $3.2 billion as of December 31, 2023. The decrease in cash and cash equivalents and current financial assets result from a net cash flows used in operating activities. FINANCIAL GUIDANCE Based on its current operating plans, argenx expects its combined research and development and selling, general and administrative expenses in 2024 to be less than $2 billion. argenx updated its cash burn guidance and now expects to utilize less than $500 million of net cash1 in 2024 on anticipated operating expenses as well as working capital and capital expenditures. EXPECTED 2024 FINANCIAL CALENDAR October 31, 2024: 3Q 2024 financial results and business updateFebruary 27, 2025: Full-year 2024 financial results and 4Q 2024 business update CONFERENCE CALL DETAILS The half-year 2024 financial results and second quarter business update will be discussed during a conference call and webcast presentation today at 2:30 PM CET/8:30 AM ET. A webcast of the live call and replay may be accessed on the Investors section of the argenx website at argenx.com/investors. Dial-in numbers: Please dial in 15 minutes prior to the live call. Belgium 32 800 50 201France 33 800 943355Netherlands 31 20 795 1090United Kingdom 44 800 358 0970United States 1 888 415 4250Japan 81 3 4578 9081Switzerland 41 43 210 11 32 About argenx argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first approved neonatal Fc receptor (FcRn) blocker, globally in the U.S., Japan, Israel, the EU, the UK, China and Canada. The Company is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit www.argenx.com and follow us on LinkedIn, X/Twitter, Instagram, Facebook, and YouTube. For further information, please contact: Media:Ben Petokbpetok@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 “advance,” “aim,” “anticipates,” “continue,” “expect,” “expand,” “plan,” or “seek” and include statements argenx makes regarding its Vision 2030 plan to develop and deliver continued and sustainable innovation for patients; its long-term commitments; its Vision 2030 goals, including having 50,000 patients globally on treatment with an argenx medicine, 10 labeled indications across all approved assets, including VYVGART franchise and potentially empasiprubart and ARGX-119, and five new molecules in Phase 3 development indicating ongoing investment in the immunology innovation program; its growth opportunity; its plans to reach earlier-line MG patients over the next 12-18 months with potential label expansions and a pre-filled syringe; the advancement of, and anticipated clinical development, data readouts and regulatory milestones and plans, including the anticipated timing of four additional Phase 3 registrational studies across efgartigimod and empasiprubart, the expected timing of additional VYVGART regulatory decisions on approval for gMG, including in Switzerland, Australia, and Saudi Arabia, the expected timing of additional VYVGART SC regulatory submissions under review or planned for CIDP, including decisions on approval in China, Japan, and Europe and a regulatory submission filing in Canada, the anticipated timing of the initiation of a Phase 3 study for efgartigimod in primary Sjogren’s disease (SjD), the anticipated timing of a confirmatory study of VYVGART (IV) in primary ITP, the anticipated timing of topline data from Phase 2/3 ALKIVIA study evaluating efgartigimod across three myositis subsets, the anticipated timing of an update on BALLAD study development plan evaluating efgartigimod in BP, the anticipated timing of proof-of-concept studies for efgartigimod in AMR and SSc, the anticipated timing of the initiation of a Phase 3 study of empasiprubart for MMN, the anticipated timing of the initiation of Phase 1b/2a studies of ARGX-119 to assess early signal detection in patients with CMS and ALS, the anticipated timing of the initiation of Phase 1 studies of ARGX-213 and ARGX-121; its investigational new drug applications for ARGX-2220 and ARGX-109 expected to be filed by the end of 2025; its 2024 research and development and selling, general and administrative expenses and operating expenses; its 2024 cash burn; and its goal of translating immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. By their nature, forward-looking statements involve risks and uncertainties and readers are cautioned that any such forward-looking statements are not guarantees of future performance. argenx’s actual results may differ materially from those predicted by the forward-looking statements as a result of various important factors, including the results of argenx's clinical trials; expectations regarding the inherent uncertainties associated with the development of novel drug therapies; preclinical and clinical trial and product development activities and regulatory approval requirements in products and product candidates; the acceptance of argenx's products and product candidates by patients as safe, effective and cost-effective; the impact of governmental laws and regulations on our business; disruptions caused on our reliance of third parties suppliers, service provides and manufacturing; inflation and deflation and the corresponding fluctuations in interest rates; and regional instability and conflicts. A further list and description of these risks, uncertainties and other risks can be found in argenx’s U.S. Securities and Exchange Commission (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. 1 reflects cash, cash equivalents and current financial assets

上市批准财报临床3期免疫疗法临床1期

2024-05-09

·GlobeNewswire-Health Care

argenx Reports First Quarter 2024 Financial Results and Provides Business Update

$398 million in first quarter global net product sales FDA review ongoing for CIDP sBLA with PDUFA target action date of June 21, 2024 On track to submit filing for pre-filled syringe (PFS) in second quarter 2024 Management to host conference call today at 2:30 PM CET (8:30 AM ET) May 9, 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 announced its first quarter 2024 results and provided a business update. “The team at argenx has made significant progress executing across the ambitious plan we set out at the beginning of the year,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx. “We are driven by our commitment to provide patients with the broadest gMG product offering that consistently delivers on safety and efficacy. VYVGART SC played a key role in our growth over the quarter, expanding the breadth of our prescriber base and reaching new patients. The relationships we have built and key market learnings in gMG position us for success as we scale the organization and prepare for CIDP. After generating the data required for filing, we are also excited to advance the development of our pre-filled syringe, which should further enhance the patient experience.” “The clinical opportunity ahead is expansive – we are preparing for registrational trials across multiple programs including empasiprubart in MMN and efgartigimod in Sjogren's disease, in addition to those already underway in TED and seronegative gMG. We look forward to deepening our understanding of FcRn with additional Phase 2 data points expected this year, while rapidly working to deliver on our promise of innovation by bringing the next wave of molecules to the clinic.” FIRST QUARTER 2024 AND RECENT BUSINESS UPDATE Reaching More Patients with VYVGART VYVGART (efgartigimod alfa-fcab) is a first-in-class antibody fragment targeting the neonatal Fc receptor (FcRn), and is now the first FcRn antagonist approved in two indications. VYVGART is approved in more than 30 countries globally for the treatment of generalized myasthenia gravis (gMG) and is approved in Japan for the treatment of primary immune thrombocytopenia (ITP). VYVGART subcutaneous (SC) (efgartigimod alfa and hyaluronidase-qvfc) is approved in the U.S. (as VYVGART® Hytrulo), Japan (as VYVDURA®) and Europe, making VYVGART the only gMG treatment available as both an IV and simple SC injection. Generated global net product sales (inclusive of both VYVGART and VYVGART SC) of $398 million in the first quarter of 2024VYVGART approved in Japan for treatment of ITP on March 26, 2024, marking first global approval for ITPAdditional VYVGART and VYVGART SC regulatory decisions on approval expected for gMG in 2024, including VYVGART in Switzerland, Australia, Saudi Arabia and South Korea, and VYVGART SC in China through Zai LabMultiple VYVGART SC regulatory submissions under review or planned for chronic inflammatory demyelinating polyneuropathy (CIDP), including: FDA review of Supplemental Biologics License Application (sBLA) ongoing with Prescription Drug User Fee Act (PDUFA) target action date of June 21, 2024Regulatory submissions completed in China and JapanRegulatory submissions expected in Europe and Canada by end of 2024 Registrational study of VYVGART in seronegative gMG patients ongoing with aim to expand label into broader MG populationsFDA submission for VYVGART SC prefilled syringe for gMG and CIDP expected in second quarter of 2024, following positive data outcomes from bioequivalence and human factor studies Advancing Current Pipeline argenx continues to demonstrate breadth and depth within its immunology pipeline and is advancing multiple pipeline-in-a-product candidates. argenx is solidifying its leadership in FcRn biology and expects that efgartigimod will be approved or under evaluation in at least 15 indications by 2025. argenx is also advancing its earlier stage pipeline programs, including empasiprubart (C2 inhibitor) with Phase 2 studies ongoing in multifocal motor neuropathy (MMN), delayed graft function (DGF) and dermatomyositis (DM). In addition, argenx is evaluating ARGX-119, a muscle-specific kinase (MuSK) agonist in both congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS). Decision announced to advance development of efgartigimod in primary Sjogren’s disease (SjD) to Phase 3 following analysis of topline data from Phase 2 RHO studyTopline data from Phase 2 ALPHA study of efgartigimod in post-COVID-19 postural orthostatic tachycardia syndrome (PC-POTS) expected in second quarter of 2024Topline data from seamless Phase 2/3 ALKIVIA study evaluating efgartigimod across three myositis subsets (immune-mediated necrotizing myopathy (IMNM), anti-synthetase syndrome (ASyS), and DM expected in second half of 2024Update on BALLAD study development plan evaluating efgartigimod in bullous pemphigoid (BP) expected by end of 2024Registrational studies ongoing of efgartigimod in thyroid eye disease (TED)Decision made to discontinue planned development of efgartigimod in ANCA-associated vasculitis (AAV) following risk assessment of all ongoing studies based on learnings from ADDRESS (pemphigus) and ADVANCE SC (ITP) studiesProof-of-concept studies ongoing with efgartigimod in membranous nephropathy (MN) and lupus nephritis (LN) with studies expected to start this year in antibody mediated rejection (AMR) and newly nominated indication, systemic sclerosis (SSc)Full Phase 2 topline data (cohorts 1 and 2) from ARDA study of empasiprubart in MMN expected in 2024; cohort 2 ongoing to determine dose response ahead of Phase 3 study startPhase 1b/2a trials of ARGX-119 to assess early signal detection in patients with CMS and ALS expected to start in 2024 Leveraging 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. Investigational new drug (IND) applications for each program are expected to be filed by end of 2025. Appointment of Brian L. Kotzin, MD as Non-executive Director to Board of Directors Dr. Brian Kotzin has been appointed as non-executive director to the Board of Directors and Chair of the Research & Development Committee for a term of four years. He is currently a consultant for companies developing therapeutics for autoimmune and inflammatory diseases. His prior roles include Chief Medical Officer for Nektar Therapeutics and Vice President of Global Clinical Development, Head of the Inflammation Therapeutic Area and Vice President and Head of Medical Sciences at Amgen. FIRST QUARTER 2024 FINANCIAL RESULTS argenx SE UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF PROFIT OR LOSS Three months ended March 31,(in thousands of $ except for shares and EPS) 2024 2023 VarianceProduct net sales $398,283 $218,022 $180,261Collaboration revenue 2,718 1,118 (1,600)Other operating income 11,512 10,740 772Total operating income 412,513 229,880 182,633 Cost of sales (43,178) (18,335) (24,843)Research and development expenses (224,969) (165,855) (59,114)Selling, general and administrative expenses (235,995) (149,172) (86,823)Loss from investment in joint venture (1,792) (261) (1,531)Total operating expenses (505,934) (333,623) (172,311) Operating loss $(93,421) $(103,743) $10,322 Financial income 38,895 16,588 22,307Financial expense (512) (188) (324)Exchange gains/(losses) (19,312) 11,165 (30,477) Loss for the period before taxes $(74,350) $ (76,178) $1,828Income tax benefit/(expense) $12,753 $47,307 $(34,554)Loss for the period $(61,597) $ (28,871) $(32,726)Loss for the period attributable to: Owners of the parent $(61,597) $(28,871) $(32,726)Weighted average number of shares outstanding 59,309,996 55,555,186 3,754,810Basic and diluted (loss) per share (in $) (1.04) (0.52) (0.52)Net increase/(decrease) in cash, cash equivalents and current financial assets compared to year-end 2023 and 2022 (75,378) (185,035) 109,657Cash and cash equivalents and current financial assets at the end of the period 3,104,466 2,007,513 DETAILS OF THE FINANCIAL RESULTS Total operating income for the three months ended March 31, 2024, was $413 million compared to $230 million for the same period in 2023, and consists of: Product net sales of VYVGART and VYVGART SC for the three months ended March 31, 2024, were $398 million compared to $218 million for the same period in 2023.Collaboration revenue for the three months ended March 31, 2024, was $3 million compared to $1 million for the same period in 2023. Collaboration revenue for the three months ended March 31, 2024, includes $2 million in royalty revenue from VYVGART sales in China.Other operating income for the three months ended March 31, 2024, was $12 million compared to $11 million for the same period in 2023. The other operating income for the three months ended March 31, 2024 and 2023, primarily relates to research and development tax incentives. Total operating expenses for the three months ended March 31, 2024, were $506 million compared to $334 million for the same period in 2023, and mainly consists of: Cost of sales for the three months ended March 31, 2024, was $43 million compared to $18 million for the same period in 2023. The cost of sales was recognized with respect to the sale of VYVGART and VYVGART SC.Research and development expenses for the three months ended March 31, 2024, were $225 million compared to $166 million for the same period in 2023. The research and development expenses mainly relate to external research and development expenses and personnel expenses incurred in the clinical development of efgartigimod in various indications and the expansion of other clinical and preclinical pipeline candidates.Selling, general and administrative expenses for the three months ended March 31, 2024, were $236 million compared to $149 million for the same period in 2023. The selling, general and administrative expenses mainly relate to professional and marketing fees linked to the commercialization of VYVGART and VYVGART SC, and personnel expenses. Financial income for the three months ended March 31, 2024, was $39 million compared to $17 million for the same period in 2023. The increase in financial income is mainly due to an increase in interest income coming from an increase of cash, cash equivalents and current financial assets as a result of the July 2023 financing round. Exchange losses for the three months ended March 31, 2024, were $19 million compared to $11 million of exchange gains for the same period in 2023. Exchange gains/losses are mainly attributable to unrealized exchange rate gains or losses on the cash, cash equivalents and current financial assets denominated in Euro. Income tax for the three months ended March 31, 2024, was $13 million of income tax benefit compared to $47 million of income tax benefit for the same period in 2023. Income tax benefit for the three months ended March 31, 2024, consists of $6 million of current income tax expense and $19 million of deferred tax benefit, compared to $11 million of current income tax expense and $58 million of deferred tax benefit for the comparable prior period. Net loss for the three months ended March 31, 2024, was $62 million compared to $29 million for the same period in 2023. On a per weighted average share basis, the net loss was $1.04 and $0.52 for the three months ended March 31, 2024 and 2023, respectively. Cash, cash equivalents and current financial assets totalled $3.1 billion as of March 31, 2024, compared to $3.2 billion as of December 31, 2023. The decrease in cash and cash equivalents and current financial assets result from net cash flows used in operating activities. FINANCIAL GUIDANCE Based on its current operating plans, argenx expects its combined Research and development and Selling, general and administrative expenses in 2024 to be less than $2 billion. argenx expects to utilize up to $500 million of net cash in 2024 on these anticipated operating expenses as well as working capital and capital expenditures. EXPECTED 2024 FINANCIAL CALENDAR July 25, 2024: Q2 2024 financial results and business updateOctober 31, 2024: Q3 2024 financial results and business update CONFERENCE CALL DETAILS The first quarter 2024 financial results and business update will be discussed during a conference call and webcast presentation today at 2:30 PM CET/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 live call. Belgium 32 800 50 201France 33 800 943355Netherlands 31 20 795 1090United Kingdom 44 800 358 0970United States 1 800 715 9871Japan 81 3 4578 9081Switzerland 41 43 210 11 32 About argenx argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first approved neonatal Fc receptor (FcRn) blocker, globally in the U.S., Japan, Israel, the EU, the UK, China and Canada. The Company is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit www.argenx.com and follow us on LinkedIn, X/Twitter, Instagram, Facebook, and YouTube. For further information, please contact: Media:Ben Petokbpetok@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 “aim,” “anticipates,” “believes,” “continue,” “expects,” “will,” “plan,” “prepare,” or “should” and include statements argenx makes regarding its commitment to provide patients with the broadest generalized myasthenia gravis (gMG) product offerings; the ability to scale the organization and prepare for CIDP; the preparation for registrational trials across multiple programs including empasiprubart in MMN and efgartigimod in Sjogren's disease; the pending regulatory decisions for gMG in Switzerland, Australia, Saudi Arabia and South Korea, and VYVGART SC in China through Zai Lab; regulatory submissions in Europe and Canada; its plans to expand label for VYVGART in seronegative gMG patients into broader MG populations; the planned FDA submission for VYVGART SC prefilled syringe for gMG and CIDP in second quarter of 2024; its aim to solidify its Fc receptor (FcRn) leadership and expectation that efgartigimod will be approved or under evaluation in at least 15 autoimmune diseases by 2025; its advancement of earlier stage pipeline programs; its evaluation of ARGX-119; its expected updates on BALLAD study development plan by end of 2024; data readouts and regulatory milestones and plans, including the timing of planned clinical trials and expected data readouts; its investigational new drug applications for four new pipeline candidates through the Immunology Innovation Program expected to be filed by the end of 2025; and its 2024 research and development and selling, general and administrative expenses and operating expenses. By their nature, forward-looking statements involve risks and uncertainties and readers are cautioned that any such forward-looking statements are not guarantees of future performance. argenx’s actual results may differ materially from those predicted by the forward-looking statements as a result of various important factors, including the results of argenx's clinical trials; expectations regarding the inherent uncertainties associated with the development of novel drug therapies; preclinical and clinical trial and product development activities and regulatory approval requirements in products and product candidates; the acceptance of argenx's products and product candidates by patients as safe, effective and cost-effective; the impact of governmental laws and regulations on our business; disruptions caused on our reliance of third parties suppliers, service provides and manufacturing; inflation and deflation and the corresponding fluctuations in interest rates; the results of the PDUFA review; and regional instability and conflicts. A further list and description of these risks, uncertainties and other risks can be found in argenx’s U.S. Securities and Exchange Commission (SEC) filings and reports, including in argenx’s most recent annual report on Form 20-F filed with the SEC as well as subsequent filings and reports filed by argenx with the SEC. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this document. argenx undertakes no obligation to publicly update or revise the information in this press release, including any forward-looking statements, except as may be required by law.

上市批准临床2期高管变更财报临床3期

100 项与 Oral C2 Inhibitor 相关的药物交易

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