Antibody-mediated rejection

$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.

The anti-CD38 mAb market is projected to experience substantial growth in the coming years owing to strong uptake of DARZALEX (along with DARZALEX FASPRO), the expected entry of subcutaneous formulation of SARCLISA in Multiple Myeloma. This growth is also driven by a rising number of cancer and autoimmune disorder diagnoses, as well as an increasing number of Anti-CD38 mAbs undergoing clinical trials and seeking approval. LAS VEGAS, Oct. 23, 2024 /PRNewswire/ -- DelveInsight's Anti-CD38 mAb Market Insights report includes a comprehensive understanding of current treatment practices, emerging anti-CD38 mAb, market share of individual therapies, and current and forecasted anti-CD38 mAb market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Key Takeaways from the Anti-CD38 mAb Market Report As per DelveInsight's analysis, the total market size of anti-CD38 mAb in the 7MM is expected to surge significantly by 2034. Multiple Myeloma is the only indication for which anti-CD38 therapies, Johnson&Johnson's (J&J) DARZALEX and Sanofi's SARCLISA have been approved until now. Around 50% of DARZALEX usage comes from second-line treatment in Multiple Myeloma. Additionally, in the frontline multiple myeloma clinical trials, DARZALEX has emerged triumphant, surpassing all expectations and solidifying its position as the gold standard of therapy. Its unparalleled effectiveness and exceptional safety profile have exceeded rivals' predictions and soon positioned DARZALEX to dominate the multiple myeloma market. The filing submission of SARCLISA subcutaneous in 3L R/R MM (IRAKLIA) is anticipated in 2025 and beyond. Somehow, SARCLISA is way behind DARZALEX in terms of market expansion, and DARZALEX has also got an early mover advantage of over 4 years. Multiple Myeloma is the leading indication, other emerging indications with commercial opportunity include Systemic Lupus Erythematosus (SLE), Chronic Kidney Disease (CKD), and IgA Nephropathy Leading emerging anti-CD38 mAb companies such as Takeda Pharmaceuticals, HI-Bio, Genmab, CASI Pharmaceuticals, and others are developing novel anti-CD38 mAb that can be available in the market in the coming years. Some of the key anti-CD38 mAb in the pipeline include Mezagitamab (TAK-079), Felzartamab, GEN3014, CID-103 , and others. In June 2024, data from the IMROZ Phase III study by Sanofi demonstrated that SARCLISA is the first anti-CD38 to significantly improve progression-free survival in combination with bortezomib, lenalidomide, and dexamethasone (VRd) for newly diagnosed transplant-ineligible multiple myeloma. In May 2024, Biogen and Human Immunology Biosciences (HI-Bio), announced the companies have entered into a definitive agreement under which Biogen has agreed to acquire HI-Bio for USD 1.15 billion upfront and up to USD 650 million in potential milestone payments. Discover which therapies are expected to grab the anti-CD38 mAb market share @ Anti-CD38 mAb Market Report Anti-CD38 mAb Market Dynamics The market for anti-CD38 mAbs has undergone significant growth and transformation in recent years, driven primarily by the increasing prevalence of multiple myeloma and the d emand for more effective treatment options. The market dynamics are further influenced by ongoing research into the broader application of anti-CD38 mAbs beyond multiple myeloma. There is growing interest in exploring their efficacy in other CD38-expressing malignancies, such as chronic lymphocytic leukemia and certain solid tumors. Additionally, research into combination therapies, where anti-CD38 mAbs are paired with immune checkpoint inhibitors, CAR-T cells, or other novel agents, is expanding the horizon for these treatments. Such advancements are likely to further propel the market, attracting substantial investment and fueling innovation. However, challenges such as biosimilar competition, pricing pressures, and the need for differentiation in a crowded market pose potential hurdles. The entrance of biosimilars could reduce costs but also threaten market share for existing products. Companies must navigate these challenges through strategic pricing, market access strategies, and continuous innovation to maintain their competitive edge. The anti-CD38 mAb market is poised for continued expansion, with a promising pipeline of new therapies and combinations on the horizon, making it a dynamic and evolving sector within oncology. Anti-CD38 mAb Treatment Market Monoclonal antibodies targeting CD38 are crucial in the treatment of newly diagnosed and refractory multiple myeloma. Currently, DARZALEX and SARCLISA are the two approved anti-CD38 therapies. SARCLISA is a prescription medication used to treat multiple myeloma in adults. It is administered alongside pomalidomide and dexamethasone for patients who have undergone at least two previous treatments, including lenalidomide and a proteasome inhibitor. Additionally, SARCLISA is used with carfilzomib and dexamethasone for patients who have received one to three prior therapies that were either ineffective or have stopped working. In March 2020, the US FDA approved SARCLISA in combination with pomalidomide and dexamethasone for multiple myeloma patients who had at least two prior therapies, including lenalidomide and a proteasome inhibitor. Later, in May 2020, the European Commission approved SARCLISA. By March 2021, the US FDA also approved SARCLISA with carfilzomib and dexamethasone for those with relapsed or refractory multiple myeloma who had received one to three previous treatments. DARZALEX is an innovative monoclonal antibody that the US FDA approved for treating multiple myeloma in patients with at least one prior treatment. It works by inducing tumor cell death through several mechanisms, including antibody-dependent cell-mediated cytotoxicity, complement-dependent cytotoxicity, and antibody-dependent cellular phagocytosis. In November 2015, the FDA granted DARZALEX accelerated approval as a monotherapy for multiple myeloma patients who had undergone at least three prior treatments. By November 2016, it was also approved for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for patients who had received at least one prior therapy. From 2015 to 2021, DARZALEX gained further approvals for use in various drug combinations. Their effectiveness in managing multiple myeloma and plasma cell disorders has been confirmed by numerous Phase III clinical trials. Moreover, CD38-targeted therapies are being explored for other conditions such as primary immune thrombocytopenia, IgA nephropathy, antibody-mediated transplant rejection, lupus nephritis, and various hematologic cancers. Learn more about the FDA-approved anti-CD38 mAb @ Anti-CD38 mAb Drugs Key Emerging Anti-CD38 mAb and Companies Several key players, including Takeda Pharmaceuticals, HI-Bio, CASI Pharmaceuticals, and others, are involved in developing drugs for Anti-CD38 mAb for various indications such as multiple myeloma, myasthenia gravis, and others. Mezagitamab is a fully human IgG1 monoclonal antibody that targets CD38 and is being developed by Takeda for the treatment of multiple myeloma, myasthenia gravis, and primary immune thrombocytopenia. On March 14, 2024, the FDA granted Fast Track designation and Orphan Drug status to mezagitamab for idiopathic thrombocytopenic purpura (ITP). Takeda also revealed plans to begin a global Phase III trial for mezagitamab in ITP later in 2024. The drug is currently in Phase II of clinical development. HI-Bio's leading candidate, felzartamab, is a fully human anti-CD38 monoclonal antibody. Clinical studies have demonstrated its ability to selectively target and deplete CD38+ cells, including plasma cells and NK cells, potentially enabling broader therapeutic applications and enhanced clinical outcomes across various immune-mediated diseases. Felzartamab has been granted Breakthrough Therapy and Orphan Drug Designations by the FDA for primary membranous nephropathy and also received Orphan Drug Designation for treating antibody-mediated kidney transplant recipients. Phase II trials have been completed for primary membranous nephropathy and antibody-mediated transplant rejection, with ongoing studies in IgA nephropathy. HI-Bio intends to progress each of these indications to Phase III. The anticipated launch of these emerging therapies are poised to transform the anti-CD38 mAb market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the anti-CD38 mAb market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. To know more about anti-CD38 mAb clinical trials, visit @ Anti-CD38 mAb Treatment Drugs Anti-CD38 mAb Overview Anti-CD38 monoclonal antibodies (mAbs) are a class of therapeutic agents that target CD38, a surface protein widely expressed on plasma cells, including malignant ones in multiple myeloma. By binding to CD38, these mAbs can induce direct cytotoxicity, promote antibody-dependent cellular cytotoxicity (ADCC), and facilitate complement-dependent cytotoxicity (CDC) to destroy the targeted cells. Beyond their role in multiple myeloma, anti-CD38 mAbs are being explored for their potential in treating other hematologic malignancies and autoimmune diseases. CD38 is not only a marker for plasma cells but also involved in processes like calcium signaling and NAD+ metabolism, which are crucial for cell survival and proliferation. By targeting CD38, these antibodies can modulate the immune microenvironment, making them a promising tool for both oncology and immunology. The ongoing research is focused on optimizing their efficacy, managing resistance, and exploring their use in combination with other immunotherapies to enhance patient outcomes. Anti-CD38 mAb Epidemiology Segmentation Epidemiology assessed for multiple myeloma showed that the United States, in 2023, accounted for 36,800 incident cases of multiple myeloma. The anti-CD38 mAb market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Incident Cases of Selected Indication for Anti-CD38 mAb Total Eligible Patient Pool for Anti-CD38 mAb in Selected Indication Total Treatable Cases of Selected Indications for Anti-CD38 mAb Scope of the Anti-CD38 mAb Market Report Anti-CD38 mAb Therapeutic Assessment: Anti-CD38 mAb current marketed and emerging therapies Anti-CD38 mAb Market Dynamics: Conjoint Analysis of Emerging Anti-CD38 mAb Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Anti-CD38 mAb Market Access and Reimbursement Discover more about anti-CD38 mAb in development @ Anti-CD38 mAb Clinical Trials Table of Contents Related Reports Multiple Myeloma Market Multiple Myeloma Market Insight, Epidemiology and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key multiple myeloma companies, including Johnson & Johnson (Janssen), Pfizer, AbbVie and Roche (Genentech), Regeneron Pharmaceuticals, Bristol-Myers Squibb, Celgene, Roche (Genentech), Arcellx, Novartis, Regeneron Pharmaceuticals, BeiGene, CARsgen Therapeutics, Cartesian Therapeutics, C4 Therapeutics, Heidelberg Pharma, Bristol-Myers Squibb, RAPA Therapeutics, AbbVie (TeneoOne), Takeda , among others. Multiple Myeloma Pipeline Multiple Myeloma Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key multiple myeloma companies, including Janssen, Pharmacyclics, Novartis, Incyte Corporation, Gracell Biotechnology, SpringWorks Therapeutics, Sana Biotechnology, Cytovia Therapeutics, Pfizer, MorphoSys, Biokine Therapeutics, Arcellx, Rapa Therapeutics, iTeos Therapeutics, Sorrento Therapeutics, CARsgen, Ascentage Pharma, Cartesian Therapeutics, Bristol-Myers Squibb, Juno Therapeutics, Nanjing IASO Biotherapeutics, Arch Oncology, Oncoceutics, TeneoBio, Nerviano Medical Sciences, among others. Relapse/Refractory Multiple Myeloma Market Relapse/Refractory Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key R/R multiple myeloma companies, including NexImmune, Amgen, AbbVie, Allogene Therapeutics, Precision Biosciences, Takeda Oncology, ORIC Pharmaceuticals, Ionis Pharmaceuticals, Cellectis S.A., YZY Biopharma, Janssen Research & Development, Bristol Myers Squibb, Lava Therapeutics, Prelude Therapeutics, among others. Relapse/Refractory Multiple Myeloma Pipeline Relapse/Refractory Multiple Myeloma Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key R/R multiple myeloma companies, including Bristol-Myers Squibb, I-MAB Biopharma, Pfizer, Arcellx, Gilead Sciences, Novartis, Array Biopharma, Hrain Biotechnology Co., Ltd., Cartesian Therapeutics, Xencor, Takeda, Sorrento Therapeutics, Heidelberg Pharma AG, Ichnos Sciences, Allogene Therapeutics, Harpoon Therapeutics, Cellectis, Poseida Therapeutics, Regeneron Pharmaceuticals, ONK Therapeutics, TeneoOne, iTeos Therapeutics, Oricell Therapeutics, Anaveon AG, Luminary Therapeutics, Seagen Inc., Trillium Therapeutics Inc., Virtuoso BINco, Inc., Seagen Inc., Trillium Therapeutics Inc., among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Designation is based on data from the clinical development program which demonstrated clinical proof of conceptFelzartamab, an investigational anti-CD38 monoclonal antibody, is a potential first-in-class therapeutic candidate for a range of rare immune-mediated indications with planning underway for Phase 3 developmentFDA Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious or life-threatening conditions and which have potential to show substantial improvement over existing therapies CAMBRIDGE, Mass., Oct. 09, 2024 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) – Biogen announced today that felzartamab, an investigational anti-CD38 monoclonal antibody, has received Breakthrough Therapy Designation (BTD) from the U.S. Food and Drug Administration (FDA) for the treatment of late antibody-mediated rejection (AMR) without T-cell mediated rejection in kidney transplant patients. The FDA grants BTD to drug candidates for serious or life-threatening conditions and that have preliminary clinical evidence demonstrating potential to provide substantial improvement over existing therapies. The designation provides additional opportunities to engage the FDA and to support the drug development program through Fast Track designation features. Data from the clinical development program that supported the designation were published in the New England Journal of Medicine and presented as a late-breaking presentation at the 61st European Renal Association (ERA) Congress in Stockholm, May 2024. "Antibody-mediated rejection is a major reason why kidney transplants fail, and currently patients suffering from AMR have tremendous unmet medical need,” said Travis Murdoch, Head of HI-Bio at Biogen. “We are focused on tackling this important challenge, and the breakthrough therapy designation will enable us to work efficiently with the FDA to accelerate development of felzartamab in AMR.” Felzartamab previously received BTD and Orphan Drug Designation (ODD) from the FDA for development in the treatment of primary membranous nephropathy (PMN) and ODD in the treatment of AMR in kidney transplant recipients. Phase 2 studies have been completed in AMR, PMN and IgA nephropathy (IgAN). Biogen plans to initiate Phase 3 trials for felzartamab across AMR, IgAN, and PMN in 2025. Biogen acquired Human Immunology Biosciences (HI-Bio) in July 2024. About FelzartamabFelzartamab is an investigational therapeutic human monoclonal antibody directed against CD38, a protein expressed on mature plasma cells. Felzartamab has been shown in clinical studies to selectively deplete CD38+ plasma cells, which may allow applications that ultimately improve clinical outcomes in a broad range of diseases driven by pathogenic antibodies. Felzartamab was originally developed by MorphoSys AG for multiple myeloma. HI-Bio exclusively licensed the rights to develop and commercialize felzartamab across all indications in all countries and territories excluding China (including Macau and Hong Kong and Taiwan). Felzartamab is an investigational therapeutic candidate that has not yet been approved by any regulatory authority and its safety and effectiveness have not been established. About Antibody-Mediated Rejection (AMR) in Kidney Transplant RecipientsAntibody-mediated rejection (AMR) is a major cause of kidney transplant failure, with chronic AMR affecting ~12% of patients that receive kidney transplants annually in the U.S.1 AMR has emerged as the leading cause of late graft loss in kidney transplant recipients. Effective treatment options for chronic AMR are currently limited.2 About BiogenFounded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients’ lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth. We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media - Facebook, LinkedIn, X, YouTube. Biogen Safe Harbor This news release contains forward-looking statements, including related to the potential clinical effects of felzartamab; the potential benefits, safety and efficacy of felzartamab; the clinical development program for felzartamab; the identification and treatment of AMR; our research and development program for the treatment of AMR; the potential of our commercial business and pipeline programs, including SPINRAZA; and risks and uncertainties associated with drug development and commercialization. These forward-looking statements may be accompanied by words such as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “intend,” “may,” “plan,” “potential,” “possible,” “will,” “would” and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on our forward-looking statements. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation, uncertainty of success in the development and potential commercialization of SPINRAZA; the risk that we may not fully enroll our clinical trials or enrollment will take longer than expected; unexpected concerns may arise from additional data, analysis or results obtained during our clinical trials; regulatory authorities may require additional information or further studies, or may fail or refuse to approve or may delay approval of our drug candidates, including SPINRAZA; the occurrence of adverse safety events; the risks of unexpected hurdles, costs or delays; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; product liability claims; results of operations and financial condition. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement, as well as the risk factors identified in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements speak only as of the date of this news release. We do not undertake any obligation to publicly update any forward-looking statements. References: Schinstock et al. (2018) Kidney Transplant with Low Levels of DSA or Low Positive B-Flow Crossmatch: An Underappreciated Option for Highly-Sensitized Transplant Candidates (Page 8). Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5481511/pdf/nihms837168.pdf#page=8 ; Ciancio et al. 2018 Antibody-Mediated Rejection Implies a Poor Prognosis in Kidney Transplantation: Results From a Single Center. Available at: https://onlinelibrary.wiley.com/doi/10.1111/ctr.13392Rodriguez-Ramirez et al. 2022 Antibody-mediated rejection: prevention, monitoring and treatment dilemmas (Page 1). Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9475491/ MEDIA CONTACT:Jack Cox+ 1 781 464 3260public.affairs@biogen.comINVESTOR CONTACT:Stephen Amato+1 781 464 2442IR@biogen.com