Commercialization and Competition - The company currently generates commercial revenue from VYVGART and VYVGART SC for gMG, CIDP, and ITP (Japan only) and faces intense competition from other biopharmaceutical companies[44]. - The company anticipates significant competition for its drug discovery and development efforts, particularly in the autoimmune field, with multiple mAbs and other biologics already marketed or in development[42]. - The company is aware of competitors developing biosimilar products for VYVGART and VYVGART HYTRULO, which could lead to substantial competition and decreased sales[56]. - The commercialization of VYVGART in new indications will require further expansion of the sales and marketing organization and collaboration with third parties[51]. - The company faces significant competition in establishing successful relationships with third-party service providers and collaborative partners[107]. Regulatory and Legislative Environment - The base regulatory exclusivity period for VYVGART and VYVGART HYTRULO is expected to extend until December 2033 in the U.S., while regulatory protection in the EU is expected to expire in August 2032 in the EEA and March 2033 in the UK[55]. - Legislative changes in the U.S., UK, and EU could adversely affect the company's future results of operations, particularly regarding price controls and patient access[60]. - The Inflation Reduction Act (IRA) allows the HHS to negotiate prices for a specified number of high-expenditure drugs, with the first 10 drugs selected for negotiation announced in August 2023, effective in 2026[61]. - The IRA imposes penalties on drug manufacturers for price increases exceeding inflation rates and caps out-of-pocket spending for Medicare Part D enrollees at $2,000 starting in 2025[62]. - The European Commission's proposal to revise pharmaceutical legislation could reduce data and market exclusivity for products in the EEA if adopted[66]. - The UK is not required to align with future EU pharmaceutical legislation, but other regulatory changes may still impact the company's operations[67]. Financial Performance and Capital Needs - The company expects to increase expenses for the foreseeable future and may not be able to raise additional capital or sustain net profitability[38]. - Future profitability is uncertain, as the company must generate significant product net sales to sustain profitability[159]. - The company may need to raise additional capital through equity or debt financing, which could be impacted by market conditions[160]. - The company's investments are subject to various risks, including credit, liquidity, and foreign currency risks, which may adversely affect financial condition[162]. - Revenue from international markets is influenced by foreign exchange rates, particularly the euro and Japanese Yen, impacting net income and expenses[164]. Clinical Development and Regulatory Approval - Regulatory approval for products and candidates is uncertain and can take many years, with clinical trials being expensive and outcomes unpredictable[85]. - Delays in clinical trials can increase costs and jeopardize the ability to commence product sales and generate revenues[90]. - Certain clinical trials have not met primary endpoints, leading to a decision to prioritize the clinical development of efgartigimod in severe autoimmune indications[84]. - The company anticipates submitting applications for approval of VYVGART in new indications, but cannot guarantee acceptance or timely approval[134]. - Delays or failures in obtaining regulatory approvals for VYVGART could materially adversely affect the company's business[135]. Supply Chain and Manufacturing Risks - The company relies on third-party suppliers for raw materials, which poses risks of supply disruptions that could materially impact revenue potential[111]. - The company contracts with Lonza and Fujifilm for manufacturing, and any issues with these CMOs could lead to significant manufacturing delays or product defects[117]. - Regulatory restrictions on biological source materials may limit or delay production, impacting clinical development and commercial activities[112]. - The company operates with a master cell bank for each product, and losing multiple cell banks could significantly impact manufacturing and financial results[229]. Intellectual Property and Legal Risks - The company faces challenges in maintaining, enforcing, and protecting its intellectual property rights, which are critical for its competitive advantage[179]. - There is uncertainty regarding the issuance and scope of patents for the company's product candidates, which could affect market potential[180]. - The company may need to divert substantial resources to enforce its intellectual property rights, and adverse rulings could allow competitors to commercialize similar products[186]. - The upcoming EU Product Liability Directive (Directive (EU) 2024/2853) will expand the definition of "damage" and create rebuttable presumptions to help claimants prove their case, potentially increasing liability claims[168]. - The company may face significant costs and distractions from complex litigation, which could adversely affect its business and operating expenses[170]. Compliance and Operational Risks - Compliance with healthcare laws is critical, as violations could lead to significant penalties and reputational harm[128]. - The company is subject to evolving privacy laws and regulations, which may require significant compliance efforts and associated costs[140]. - The company faces risks related to environmental compliance, which could result in substantial fines and reputational damage[155]. - The company is dependent on accurate financial information from collaboration partners, and any inaccuracies could adversely impact financial reporting[119]. Market and Economic Factors - Global geo- and socio-political threats and macro-economic uncertainties could materially and adversely affect the company's business and financial performance[38]. - Changes in U.S.-Mainland China relations, including tariffs and export controls, may negatively impact collaborations with Chinese biotechnology companies[223]. - The company may face challenges in obtaining favorable pricing and reimbursement terms in foreign markets, which could hinder product commercialization[77]. - The company’s ability to attract and retain key personnel may be challenged by competitive remuneration practices and an inflationary environment[217]. Research and Development - The long-term growth strategy includes developing and marketing additional products and candidates such as efgartigimod, empasibrubart, and ARGX-119, which requires substantial resources[83]. - The company benefits from research and development tax incentives, including tax credits and payroll withholding tax exemptions[230]. - Public health issues may disrupt clinical trials and operations, affecting the ability to recruit patients and obtain regulatory approvals[229].

Product Approvals and Commercialization - VYVGART® has been approved in multiple regions including the U.S., EU, Japan, and China for the treatment of generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP) with ongoing commercialization efforts[9][12]. - VYVGART SC has been launched in the U.S. and China as VYVGART HYTRULO™ and in Japan as VYVDURA®, with pricing and reimbursement discussions ongoing in several EU countries[11][12]. - The company aims to expand its product approvals and commercialization efforts in additional jurisdictions for both VYVGART and VYVGART SC[12]. - VYVGART is now approved in multiple regions, including the U.S., Japan, and the EU, for various indications[62]. - VYVGART is approved in over 30 countries for gMG treatment, with VYVGART SC also approved in the U.S., EU, UK, Japan, Israel, China, and Australia for CIDP[121]. - VYVGART received approval in Mainland China in June 2023 for the treatment of adult gMG patients, with additional approvals for gMG and CIDP expected in 2024[152]. - Regulatory approvals for VYVGART have been received in multiple regions, including the U.S., Japan, and the EU, with further reviews ongoing[192][200]. Financial Performance and Projections - The financial statements are prepared in accordance with IFRS and presented in U.S. dollars, ensuring compliance with international accounting standards[15][16]. - The company anticipates benefiting from the Belgian innovation income deduction, which may positively impact future financial performance[21]. - Global product net sales reached $2.2 billion in 2024, reflecting strong demand for innovative therapies[45]. - Product net sales reached $2.2 billion, indicating strong market performance[87]. - Research and development expenses totaled $1.0 billion, reflecting continued investment in innovation[87]. - The company plans to transition to sustainable operating profitability by 2025, enabling further growth[87]. - The 2025 outlook suggests a focus on long-term growth strategies, including the development of new products and indications[28]. Clinical Trials and Pipeline Development - The company is actively pursuing collaborations and partnerships to enhance its product development and market reach[21][25]. - The company plans to advance five new molecules into Phase 3 clinical trials by 2030[47]. - Four new investigational new drugs (INDs) are expected to enter Phase 1 clinical trials in 2025[60]. - The company is conducting registrational clinical trials for efgartigimod in multiple indications, with topline results expected in 2026[70]. - The company has initiated Phase 3 clinical trials for empasiprubart in multifocal motor neuropathy (MMN) and CIDP, expected to start in 2025[58]. - The Phase 3 clinical trial for efgartigimod PFS in Primary Sjogren's Disease (RHO) was initiated at the end of 2024, with topline results expected in 2027[74]. - The company has a strong focus on expanding its pipeline with multiple candidates in various stages of clinical trials[124]. - The company aims to have five new molecules in Phase 3 development and ten labeled indications by 2030[137]. - The pipeline includes empasiprubart in Phase 3 for multiple autoimmune diseases, with topline results expected in 2H 2025[123]. - The company is evaluating efgartigimod in over 10 additional serious autoimmune indications[123]. - The ongoing clinical trial for the ADAPT-SC study in gMG has shown promising results, with the proportion of responders based on the MG-ADL score being a primary endpoint[173]. - The ongoing clinical trial for the treatment of Sjögren's disease (SjD) has a target of 30 responders by the end of the treatment period[175]. - The ongoing partnership with Zai Lab aims to evaluate the change in urine protein creatinine ratio in lupus nephritis, with results expected in the second half of 2025[175]. Strategic Initiatives and Collaborations - The company is committed to maintaining strong governance practices as outlined in the Dutch Corporate Governance Code[33]. - The Immunology Innovation Program (IIP) is central to the company's strategy, enabling the development of novel pipeline candidates through collaborations[124]. - The company has entered into a non-exclusive research agreement with Chugai Pharmaceutical for technologies aimed at expanding the therapeutic index of product candidates[130]. - The company has entered into multiple distribution agreements, including with Medison for commercialization in Israel and 14 other countries[153]. - The partnership with Zai Lab for Greater China includes sales-based milestone payments and royalties based on annual product net sales of efgartigimod[152]. Market Challenges and Risk Factors - The risk factors section highlights potential challenges related to commercialization and regulatory approvals that could impact future results[22][31]. - The competitive landscape includes multiple monoclonal antibodies and biologics, with ongoing challenges from other biopharmaceutical companies in the autoimmune field[136]. Patient Impact and Awareness - The company is committed to improving MG awareness and ensuring healthcare providers have access to critical information about MG patients[102]. - The company aims to transform the lives of at least 50,000 patients by 2030 through innovative immunology-based medicines[130]. - A Pre-Approval Access program for gMG patients was opened in February 2021, providing access to over 403 patients across 14 countries in 2024[159]. - Approximately 1,000 patients were on therapy for CIDP within the first two quarters of its launch[46]. - The company aims to reach at least 50,000 patients globally and secure 10 labeled indications by 2030[47].

Core Insights - Amgen reported positive results from the phase III MINT study for Uplizna, indicating its potential for label expansion in generalized myasthenia gravis (gMG) [1][4] Group 1: Study Results - Uplizna demonstrated durable efficacy in AChR-positive gMG patients over 52 weeks, with a 2.8-point improvement in MG-ADL scores compared to placebo [2] - 72% of AChR+ patients treated with Uplizna showed an improvement of three or more points in MG-ADL scores, versus 45% in the placebo group [2] - In the QMG score, 69% of AChR+ patients on Uplizna improved by three or more points, compared to nearly 42% in the placebo group [3] Group 2: Regulatory and Market Position - Amgen plans to submit a regulatory filing for Uplizna with the FDA by the first half of 2025, currently approved for neuromyelitis optica spectrum disorder [4] - A regulatory filing for Uplizna in immunoglobulin G4-related disease is under review, with a decision expected by April 3, 2025 [4] - Uplizna, part of Amgen's rare disease franchise, was acquired through the $28 billion purchase of Horizon Therapeutics in 2023 [9] Group 3: Competitive Landscape - The 52-week results from the MINT study provide Uplizna an advantage over competitors like argenx's Vyvgart and UCB's Rystiggo, which require weekly dosing, while Uplizna requires dosing every six months [8] - Uplizna has received orphan drug designation from the FDA for the gMG indication [8] Group 4: Stock Performance - Year to date, Amgen's shares have increased by over 20%, outperforming the industry growth of 6% [5]

Financial Data and Key Metrics Changes - Total operating income for Q3 2024 was $589 million, comprising $573 million in product net sales and $16 million in other operating income, reflecting a 20% quarter-over-quarter growth and a 74% increase year-over-year [20][21] - Operating expenses for Q3 totaled $575 million, an increase of $40 million compared to Q2 2024, driven by higher SG&A and R&D expenses [22][23] - The company reported an operating profit of $14 million for Q3, while the year-to-date operating loss stood at $125 million [23] Business Line Data and Key Metrics Changes - Product revenue breakdown: $492 million from the U.S., $24 million from Japan, $46 million from the rest of the world, and $11 million from product supply to Zai Lab in China [21] - The CIDP launch has successfully reached over 300 patients, with revenue approaching that of the first quarter of the MG launch [10][32] Market Data and Key Metrics Changes - The company has secured favorable payer policies covering approximately 54% of U.S. commercial lives for CIDP, with most policies requiring prior IVIg utilization [28] - VYVGART is now available to over 80% of the gMG population in the EU, with ongoing regulatory reviews in China, Japan, and Europe for CIDP [37] Company Strategy and Development Direction - The company is focused on expanding its commercial presence and advancing its pipeline, with a Vision 2030 strategy aimed at addressing unmet needs in neuromuscular medicine [7][15] - The decision to discontinue the development of efgartigimod in MN reflects a strategic shift to prioritize programs with higher potential for patient impact [17][18] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the CIDP launch trajectory, noting that patient starts have been relatively linear and in line with expectations [43][44] - The company is optimistic about the potential to reach 50,000 patients by 2030, leveraging growth in both MG and CIDP, as well as future pipeline expansions [68] Other Important Information - The company has over $3.4 billion in cash and equivalents, with cash guidance for 2024 no longer applicable [23][24] - The upcoming PDUFA date for the prefilled syringe (PFS) is in April 2025, which is expected to enhance patient convenience and drive growth [102] Q&A Session Summary Question: Trajectory of the CIDP launch - Management indicated that CIDP patient starts have been relatively linear, with most starts occurring in September as payer policies came online [43][44] Question: Update on payer formulary and treatment timelines - Management confirmed that the time from prescription to treatment is in line with expectations, with smooth transitions for patients getting on therapy [49][50] Question: Breakdown of sales from CIDP and MG - Management confirmed that most sales were still driven by MG, with CIDP contributing approximately $20 million, primarily in the last month of the quarter [52][54] Question: Patients not switching from IVIg - Management noted that the majority of CIDP patients are switching from IVIg, with the remaining patients primarily coming from steroids or other immunosuppressants [59] Question: Dosing frequency for CIDP patients - Management stated it is too early to determine dosing frequency trends, as most patients are just starting therapy [80][81] Question: Expected price for Hytrulo in CIDP - Management reiterated that the expected price for Hytrulo is $450,000, but real-world utilization will determine the final cost [84] Question: Pipeline news flow expectations - Management indicated that a detailed clinical calendar for 2025 will be announced at the start of the new year, with significant activity expected across multiple trials [92]

| --- | --- | --- | --- | --- | --- | --- | |-------------|-------|-------|-------------------------|-----------------------------------------------------|-----------|-------| | | | | | | | | | | | | | | | | | 3 Q 2 0 2 | 4 | | E A R N I N G S C A L L | Reaching Patients Through \| O c t o b e r 3 1 | , 2 0 2 4 | | | | | | | Immunology Innovation | | | | | | | | | | | Forward Looking Statements This presentation has been prepared by argenx se ("argenx" or the "company") for informational purposes only and n ...

After grappling with recession fears, the Federal Reserve’s jumbo interest rate cuts helped the broader S&P 500 to defy the odds of a notorious September and notch record highs. The S&P 500 posted its best three-quarters of the 21st century, and if history is any guide, the index is well-poised to continue its winning streak over the next three months. The 30-stock Dow and the tech-laden Nasdaq also closed in the green in the first nine months of 2024. Thus, it’s judicious for astute investors to place thei ...

argenx SE (NASDAQ:ARGX) Q2 2024 Earnings Conference Call July 25, 2024 8:30 AM ET Company Participants Beth DelGiacco - Vice President, Global Head of Corporate Communications & Investor Relations Tim Van Hauwermeiren - Chief Executive Officer Karl Gubitz - Chief Financial Officer Karen Massey - Chief Operating Officer Conference Call Participants James Gordon - JPMorgan Tazeen Ahmad - Bank of America Allison Bratzel - Piper Sandler Rajan Sharma - Goldman Sachs Derek Archila - Wells Fargo Akash Tewari - Jef ...

| --- | --- | --- | --- | --- | --- | --- | |-------|---------|-------|----------------------------------------------------|----------------------------|-------|-------| | | | | | | | | | | | | | | | | | Q 2 | 202 4 | | Reaching Patients through E A R N I N G S C A L L | \| J u l y 2 5 , 2 0 2 4 | | | | | | | Immunology Innovation | | | | | | | | | | | | Forward Looking Statements 2 This presentation has been prepared by argenx se ("argenx" or the "company") for informational purposes only and not for any o ...

argenx SE (NASDAQ:ARGX) Q1 2024 Earnings Conference Call May 9, 2024 8:30 AM ET Company Participants Beth DelGiacco - Vice President, Global Head of Corporate Communications and Investor Relations Tim Van Hauwermeiren - Chief Executive Officer Karl Gubitz - Chief Financial Officer Karen Massey - Chief Operating Officer Conference Call Participants Tazeen Ahmad - Bank of America Securities Rajan Sharma - Goldman Sachs Derek Archila - Wells Fargo James Gordon - JPMorgan Akash Tewari - Jefferies Yaron Werber - ...

Financial Performance - The company reported net product sales of $1.2 billion from global product net sales in fiscal year 2023[35]. - Cumulative operating losses since inception total $2,405 million, with expectations of continued losses for the foreseeable future[34]. - As of December 31, 2023, the company had cash and cash equivalents of $2,049 million and current financial assets of $1,131 million[36]. - Cash and cash equivalents and current financial assets increased to $3.2 billion as of December 31, 2023, up from $2.2 billion a year earlier[38]. - The company raised $1.3 billion in gross proceeds from a global offering in July 2023, selling 1,917,715 ADSs at $490.00 each and 663,918 ordinary shares at €436.37 each[37]. Competition and Market Dynamics - The company faces significant competition in the autoimmune field, with multiple monoclonal antibodies and other biologics in development by various companies[49]. - The commercialization of VYVGART in new indications will require further expansion of the sales and marketing organization[40]. - Distribution agreements have been established with partners in various regions, which may affect product revenues and profitability[42]. - The overall use of biosimilars is increasing, which may lead to greater competition and impact sales of innovative products[56]. Regulatory Environment - The regulatory exclusivity periods for VYVGART and VYVGART HYTRULO are expected to extend until December 2033 in the U.S. and until August 2032 in the EU[55]. - The Inflation Reduction Act (IRA) allows the U.S. Department of Health and Human Services to negotiate prices for a specified number of drugs, with the first negotiations affecting prices starting in 2026[59]. - The IRA imposes penalties on drug manufacturers that increase prices at a rate greater than inflation, which could significantly impact revenue[60]. - The European Commission's proposal to revise pharmaceutical legislation may shorten data and market exclusivity periods, affecting future product demand[58]. - Compliance with government pricing laws is mandatory, and failure to comply could result in significant civil monetary penalties[64]. - The American Rescue Plan of 2021 eliminated the cap on Medicaid rebates, potentially increasing the financial burden on pharmaceutical manufacturers[66]. - The Infrastructure Investment and Jobs Act requires manufacturers to provide refunds for unused portions of certain drugs, impacting financial results[67]. - Legislative changes at both federal and state levels are expected to continue affecting pharmaceutical pricing and demand for products[62]. Clinical Development and Trials - The company is developing products for different indications, with potential negative results in clinical trials impacting overall perception and commercialization efforts[45]. - The ADVANCE-SC clinical trial for VYVGART HYTRULO in adults with ITP did not meet its primary endpoint, indicating challenges in clinical development[113]. - The Phase 3 ADDRESS clinical trial for SC efgartigimod in adults with pemphigus vulgaris showed no significant difference in primary endpoint achievement compared to placebo, leading to a decision not to pursue further development in this area[113]. - Patient enrollment for clinical trials is critical and may be hindered by the small target population for rare diseases, affecting the ability to realize full commercial potential[127]. - The company faces potential delays in clinical trials due to regulatory requirements, which could jeopardize development and approval processes[87]. - Significant delays in clinical trials may increase costs and harm commercial prospects, allowing competitors to market products earlier[89]. Compliance and Legal Risks - Compliance with healthcare laws is critical, as violations could result in significant penalties and operational disruptions[81]. - The company is expanding its internal compliance program to meet diverse healthcare regulations, which involves substantial costs[84]. - Compliance costs with regulatory authorities could be substantial, and failure to comply may result in sanctions or delays in product development[93]. - The company is subject to evolving privacy laws, including the EU GDPR, which imposes fines of up to €20 million or 4% of total worldwide annual turnover for serious offenses[95]. - Non-compliance with environmental laws could result in substantial fines and reputational damage[109]. Intellectual Property and Market Position - The success of the company depends on obtaining and maintaining intellectual property rights, and failure to do so could adversely affect its competitive advantage[161]. - The scope of patent protection is uncertain, and competitors may market similar products if patents are not adequately enforced[162]. - The company may face challenges in preparing and filing necessary patent applications in a timely manner, which could affect its ability to commercialize products[163]. - The company may not be successful in acquiring or in-licensing necessary third-party intellectual property rights, which could hinder product development[175]. - The company relies on trade secret protection for proprietary information, but the difficulty in protecting these secrets increases the risk of competitors duplicating its technological achievements[184]. Operational Challenges - The company faces operational challenges as it increases the number of clinical trials, which may lead to higher costs and delays[118]. - The company relies on third parties for research and clinical trials, and any issues in these relationships could adversely affect business operations[132]. - The company faces risks related to the reliance on single-source suppliers for raw materials, which could lead to delays in research or clinical trials if supply is interrupted[140]. - Manufacturing processes are dependent on contract manufacturing organizations (CMOs), and any failure in their ability to meet specifications could adversely affect clinical trials and regulatory approvals[144]. - The company collaborates with Lonza and FUJIFILM for drug substance manufacturing, which involves complex processes that could lead to product defects if not managed properly[145]. Strategic Growth and Management - The company may engage in strategic transactions such as acquisitions, collaborations, or investments to enhance its business but may face challenges in identifying suitable targets or satisfactory conditions for these transactions[156]. - Integration of newly acquired companies or technologies could be expensive and time-consuming, potentially straining managerial and operational resources[157]. - The company must continue to implement and improve its managerial and operational systems to manage anticipated growth effectively[203]. - The company is increasingly dependent on its information technology systems, which are critical for maintaining sensitive information and could be vulnerable to cyber-attacks[158]. - Cybersecurity incidents pose evolving risks, and despite efforts to secure information systems, there is no assurance that breaches will not occur, potentially disrupting operations and increasing costs[159]. Tax and Financial Regulations - The company has benefited from research and development tax incentives, including tax credits and payroll withholding tax exemptions, which may be challenged by tax authorities, potentially impacting future cash flows[204]. - The effective tax rates could be adversely affected by changes in tax laws and regulations, particularly regarding the Belgian innovation income deduction, which could significantly impact the company's tax assets[208]. - The Organisation of Economic Co-operation and Development (OECD) has introduced the Pillar Two Directive, which may subject the company to new tax regulations as early as 2025, although no impact is expected in 2024[210]. - The company is subject to transfer pricing regulations that may change and could materially impact financial results as international operations expand[206]. - The company has considerable tax assets in Belgium, which may be forfeited or restricted due to various transactions or changes in statutory law[211]. Shareholder and Governance Issues - Holders of ADSs do not have the same voting rights as ordinary shareholders, which may limit their ability to influence corporate governance[216]. - The company is exempt from certain U.S. securities laws as a foreign private issuer, allowing it to file less information with the SEC compared to U.S. domestic companies[227]. - The company may face challenges in maintaining effective internal controls over financial reporting, which could lead to financial statement restatements and negatively impact the trading price of its ADSs[214]. - The company is not obligated to comply with all best practice provisions of the Dutch Corporate Governance Code (DCGC), which may affect shareholders' rights[225].