Latest news with #VYVGART
Yahoo
22-05-2025
- Business
- Yahoo
Johnson & Johnson's IMAAVY Gets FDA Approval, Taking on Top Players in Myasthenia Gravis Market
The FDA has approved Johnson & Johnson's anti-FcRn antibody, nipocalimab, for the treatment of generalized myasthenia gravis. The drug will be sold under the brand name IMAAVY. This approval positions J&J to challenge leading competitors in the myasthenia gravis market, such as AstraZeneca, which markets the monoclonal antibodies SOLIRIS and ULTOMIRIS. J&J will also be competing with Argenx's VYVGART and UCB's RYSTIGGO and ZILBRYSQ. LAS VEGAS, May 22, 2025 /PRNewswire/ -- Myasthenia gravis is an autoimmune disorder in which antibodies disrupt the communication between nerves and muscles, resulting in skeletal muscle weakness. This weakness primarily affects voluntary muscles that control eye movement, facial expression, swallowing, and limb function. The condition is predominantly driven by autoantibodies targeting either the acetylcholine receptor (AChR-Ab) or a receptor-associated protein known as muscle-specific tyrosine kinase (MuSK-Ab). According to DelveInsight's analysis, there were approximately 300,000 diagnosed prevalent cases of myasthenia gravis across the 7MM in 2024. This number is expected to grow steadily over the forecast period (2025–2034), with a projected CAGR. The current myasthenia gravis therapeutics market mainly consists of symptomatic treatments such as acetylcholinesterase inhibitors like Mestinon. However, achieving full clinical remission with symptomatic therapy alone is challenging. As a result, immunosuppressive drugs are frequently introduced. First-line immunotherapy typically involves high-dose oral corticosteroids such as prednisone or prednisolone, which can induce a rapid response within 2 to 4 weeks. To reduce reliance on steroids and improve long-term disease control, nonsteroidal immunosuppressants (NSISTs) are often used. These include antimetabolites (e.g., azathioprine, mycophenolate mofetil, methotrexate, and cyclophosphamide) and calcineurin inhibitors (e.g., tacrolimus and cyclosporine). While NSISTs may be used as standalone treatments, they usually take 6 to 9 months to produce noticeable effects. Immunomodulatory therapies, including intravenous immunoglobulin (IVIg) and plasmapheresis (PLEX), are typically reserved for acute exacerbations or myasthenic crises. They are also employed as chronic treatments in patients who cannot tolerate or do not respond to standard immunosuppressants. Although traditional immunosuppressive strategies can help manage symptoms, achieving long-term, stable remission remains difficult. Additionally, about 15% of patients are considered treatment-refractory, meaning they either fail to respond to or cannot tolerate multiple immunosuppressive options. These patients often face the dual challenge of persistent symptoms and the adverse effects of long-term immunosuppression. Learn more about the myasthenia gravis disease market @ Myasthenia Gravis Treatment Medications The myasthenia gravis treatment market is well-established, with multiple approved drugs for myasthenia gravis, including SOLIRIS, ULTOMIRIS, RYSTIGGO, ZILBRYSQ, and VYVGART, among others. Alexion Pharmaceuticals' SOLIRIS is a monoclonal antibody that targets and inhibits complement protein C5, thereby blocking the formation of the terminal complement complex. This action helps reduce complement-driven hemolysis in PNH and thrombotic microangiopathy in aHUS. Although its exact mechanism in gMG and NMOSD is not fully known, it is believed to decrease complement complex accumulation. Between 2017 and 2023, SOLIRIS gained regulatory approvals in Japan, the US, and the EU for treating gMG, including pediatric indications in Japan. ULTOMIRIS (ravulizumab), also from Alexion Pharmaceuticals, is the first long-acting C5 inhibitor, offering rapid, complete, and sustained suppression of complement activity. It binds to C5, preventing its cleavage and subsequent formation of the membrane attack complex (C5b-9). ULTOMIRIS is effective in managing terminal complement-mediated hemolysis in PNH and thrombotic microangiopathy in aHUS. Although the precise mechanism in gMG and NMOSD remains unclear, it is thought to reduce complement deposition at the neuromuscular junction and block aquaporin-4 antibody-mediated complement activation. It received approvals from the US FDA, EMA, and Japan's PMDA in 2022. Get an in-depth assessment on generalized myasthenia gravis medication for adults @ ULTOMIRIS vs SOLIRIS for Myasthenia Gravis UCB Biopharma's RYSTIGGO is a subcutaneously administered, humanized monoclonal antibody that binds with high affinity to the human neonatal Fc receptor (FcRn). It received regulatory approval from the US FDA and Japan's PMDA in 2023, followed by the EC in January 2024. ZILBRYSQ, also developed by UCB Biopharma, is the first once-daily subcutaneous peptide-based inhibitor specifically targeting complement protein C5. It works by blocking C5 cleavage and the formation of the terminal complement complex C5b-9. Although its precise action in gMG remains uncertain, it is thought to limit C5b-9 accumulation at the neuromuscular junction. ZILBRYSQ gained approval from the US FDA, EMA, and PMDA in 2023. VYVGART, from Argenx, is a human IgG1 antibody fragment that targets FcRn, leading to a decrease in circulating IgG autoantibodies. It is the first FcRn blocker to receive regulatory approval. Argenx's VYVGART HYTRULO (also marketed as VYVDURA) combines efgartigimod alfa, an FcRn inhibitor, with hyaluronidase, an endoglycosidase, for treating adults with generalized myasthenia gravis. This formulation was approved by the US FDA and EMA in 2023 and by the PMDA in January 2024. Uncover the mechanism of FcRn inhibitor myasthenia gravis medication @ Myasthenia Gravis MoA On April 30, 2025, the FDA approved Johnson & Johnson's nipocalimab, branded as IMAAVY, as a new therapy for gMG. This approval puts IMAAVY among the list of approved drugs for myasthenia gravis treatment. The approval, which J&J says encompasses the widest eligible patient population with gMG, includes individuals aged 12 and above who test positive for AChR or anti-muscle-specific kinase (MuSK) antibodies. IMAAVY functions by inhibiting the FcRn protein, which plays a role in maintaining circulating IgG antibodies, key drivers in many autoimmune disorders. By blocking FcRn, IMAAVY significantly reduces harmful IgG autoantibodies while preserving the body's overall immune response, according to clinical data. J&J sees broader potential for IMAAVY beyond gMG, believing its mechanism of action could apply across multiple autoimmune conditions due to IgG's involvement in various diseases. The FDA's decision followed results from the ongoing Phase III Vivacity-MG3 trial, which evaluates IMAAVY in combination with standard care versus placebo and standard care in 199 adult gMG patients, including 153 antibody-positive individuals. Additionally, J&J is conducting a Phase II/III pediatric trial called Vibrance, where IMAAVY, combined with standard care, achieved its primary goal by reducing total serum IgG levels by 69% over 24 weeks. J&J acquired IMAAVY through its USD 6.5 billion acquisition of Momenta Pharmaceuticals in 2020, with the FcRn inhibitor myasthenia gravis serving as the centerpiece of that deal. The company is aiming for blockbuster status, forecasting peak annual sales of over USD 5 billion for the myasthenia gravis medication. Dive deep into the myasthenia gravis treatment cost @ Generalized Myasthenia Gravis Drug with Copay Myasthenia gravis pipeline possesses some drugs in mid- and late-stage development to be approved in the near future. The expected launch of myasthenia gravis treatment medications, such as Batoclimab (Immunovant Sciences GmbH), Descartes-08 (Cartesian Therapeutics), and others, are expected to further create a positive impact on the myasthenia gravis disease market. Discover which therapies are expected to grab major myasthenia gravis therapeutics market share @ Market Analysis on Myasthenia Gravis Batoclimab is a fully humanized monoclonal antibody designed to inhibit neonatal Fc receptors by blocking the interaction between FcRn and IgG, thereby promoting the breakdown of autoantibodies. It has received Orphan Drug Designation (ODD) from both the US FDA and the EMA. In March 2025, Immunovant announced topline results from its Phase III trial evaluating batoclimab in patients with myasthenia gravis. Descartes-08 represents the first RNA-engineered CAR T-cell (rCAR-T) therapy developed for autoimmune conditions. This therapy uses cytotoxic T-cells programmed to target and eliminate pathogenic plasma cells that produce autoantibodies. Created from a patient's own blood, Descartes-08 has also received ODD from the US FDA for treating myasthenia gravis. In December 2024, Cartesian Therapeutics shared encouraging updated findings from its Phase IIb study of Descartes-08 in myasthenia gravis. Furthermore, in January 2025, the FDA agreed to the company's Phase III AURORA trial design under a Special Protocol Assessment, with trial initiation planned for the first half of 2025. Curious about what the new myasthenia gravis treatments are in development? Visit Myasthenia Gravis Clinical Trials The anticipated launch of these emerging myasthenia gravis treatments are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the myasthenia gravis market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the myasthenia gravis market size in the 7MM is expected to grow from USD 5.2 billion in 2024 at a significant CAGR by 2034. According to DelveInsight's analysis, the myasthenia gravis market growth is anticipated with the introduction of emerging therapies, leading to an expansion in the myasthenia gravis market size throughout the forecast period (2025–2034). This anticipated myasthenia gravis market growth is driven by advancements in treatment options, greater healthcare access, and a rising myasthenia gravis prevalence, which together foster higher demand for innovative and effective therapies. DelveInsight's latest published market report titled Myasthenia Gravis Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which myasthenia gravis treatment market leader is going to capture the largest market share. The report provides comprehensive insights into the country-specific myasthenia gravis treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The myasthenia gravis market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into: Total Diagnosed Prevalent Cases of Myasthenia Gravis Gender-specific Diagnosed Prevalent Cases of Myasthenia Gravis Age-specific Diagnosed Prevalent Cases of Myasthenia Gravis Diagnosed Prevalent Cases of Myasthenia Gravis by MGFA Classification Diagnosed Prevalent Cases of Generalized Myasthenia Gravis by Antibody Serology The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM myasthenia gravis market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this myasthenia gravis market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the myasthenia gravis market. Also, stay abreast of the mitigating factors to improve your market position in the myasthenia gravis therapeutic space. Related Reports Myasthenia Gravis Epidemiology Forecast Myasthenia Gravis Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted myasthenia gravis epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Myasthenia Gravis Pipeline Myasthenia Gravis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key myasthenia gravis companies, including Harbour BioMed (Guangzhou) Co. Ltd., Kyverna Therapeutics, Cabaletta Bio, Takeda, Hoffmann-La Roche, Immunovant Sciences GmbH, Regeneron Pharmaceuticals, Novartis Pharmaceuticals, Janssen Research & Development, LLC, Momenta Pharmaceuticals, Inc., Amgen, Dianthus Therapeutics, Cartesian Therapeutics, COUR Pharmaceutical Development Company, Inc., Alexion Pharmaceuticals, Inc., among others. Generalized Myasthenia Gravis Market Generalized Myasthenia Gravis Market Insights, 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 gMG companies including Alexion Pharmaceuticals, Argenx, UCB Biopharma, Argenx-Halozyme Therapeutics, Horizon Therapeutics, Hoffmann-La Roche, Janssen Research & Development, LLC, Immunovant Sciences GmbH, Bristol Myers Squibb, Biogen Inc., Pfizer Inc., Novartis AG, Sanofi S.A., Roche Holding AG, Johnson & Johnson, Takeda Pharmaceutical Company Limited, AbbVie Inc., Eli Lilly and Company, Merck & Co., Inc., GlaxoSmithKline plc, AstraZeneca plc, Amgen Inc., Boehringer Ingelheim International GmbH, Genentech, Inc., Gilead Sciences, Inc., Celgene Corporation, Vertex Pharmaceuticals Incorporated, among others. Generalized Myasthenia Gravis Pipeline Generalized Myasthenia Gravis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key gMG companies, including Biocon, Cartesian Therapeutics, UCB, Momenta Pharmaceuticals, HanAll Biopharma, Roche, Alexion, Novartis, Takeda, BioMarin, 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 UsShruti Thakur info@ +14699457679 Logo: View original content: SOURCE DelveInsight Business Research, LLP Sign in to access your portfolio
Yahoo
08-05-2025
- Business
- Yahoo
Zai Lab Announces First Quarter 2025 Financial Results and Recent Corporate Updates
– Total revenues grew 22% y-o-y to $106.5 million for the first quarter of 2025; reaffirming full-year 2025 revenue guidance of $560 million to $590 million – Operating loss improved significantly, declining 20% year-over-year to $56.3 million for the first quarter of 2025, and 25% to $37.1 million on an adjusted basis1; on track to achieve profitability1 in the fourth quarter of 2025 – ZL-1310 (DLL3 ADC) is advancing rapidly, with upcoming ASCO 2025 data presentation in ES-SCLC; initiation of registrational study in ES-SCLC expected in the second half of 2025 – AACR 2025 presentations of ZL-6201 (LRRC15 ADC) and ZL-1222 (PD-1/IL-12) underscore the promising potential of Zai Lab's internally developed next-generation oncology therapies – Strong balance sheet with a cash position2 of $857.3 million as of March 31, 2025, compared to $879.7 million as of December 31, 2024 Conference call and webcast today, May 8, 2025, at 8:00 a.m. ET (8:00 p.m. HKT) SHANGHAI & CAMBRIDGE, Mass., May 08, 2025--(BUSINESS WIRE)--Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) today announced financial results for the first quarter of 2025, along with recent product highlights and corporate updates. "In the first quarter, we continued to advance both our global pipeline and commercial business," said Dr. Samantha Du, Founder, Chairperson, and CEO of Zai Lab. "We are rapidly expanding our global portfolio, having recently presented promising data for two next-generation oncology therapies at AACR, and we look forward to presenting updated results for ZL-1310 (DLL3 ADC) at the 2025 ASCO Annual Meeting. We remain on track to initiate a pivotal study for ZL-1310 in SCLC this year, with the goal of securing FDA approval in 2027. In parallel, we are also exploring opportunities in first-line SCLC and other neuroendocrine tumors to unlock the full potential of this important global asset. On the commercial front, we continued to expand patient access for key products and are leveraging our existing infrastructure to support upcoming launches and our next wave of blockbuster opportunities. With a strong foundation in place, we are well positioned to drive growth and profitability, and to further our mission of becoming a leading global biopharmaceutical company." "2025 marks a transformational year for Zai Lab, and we are executing against the key objectives we set at the start of the year," said Josh Smiley, President and COO of Zai Lab. "Following a seasonal slowdown of VYVGART early in the year, we saw patient volumes rebound in March and April, and we anticipate a return to strong sequential growth throughout the rest of the year. Looking ahead, our late-stage pipeline, including bemarituzumab and KarXT, is expected to fuel the next wave of topline growth alongside VYVGART. At the same time, we continue to strengthen our financial position, achieving a 20% year-over-year reduction in operating loss and a 25% year-over-year reduction in adjusted operating loss1, keeping us on track to achieve profitability1 in the fourth quarter of 2025. With a strong cash position,2 a growing commercial business and an expanding global portfolio, Zai Lab is poised to capitalize on multiple upcoming catalysts and drive long-term shareholder value." 1 Refers to adjusted income (loss) from operations (non-GAAP), calculated as GAAP income (loss) from operations adjusted to exclude certain non-cash expenses, including depreciation, amortization, and share-based compensation. For additional information on this adjusted profitability measure, refer to the "Non-GAAP Measures" section. 2 Cash position includes cash and cash equivalents, current restricted cash, and short-term investments. First Quarter 2025 Financial Results Product revenue, net was $105.7 million in the first quarter of 2025, compared to $87.1 million for the same period in 2024, representing 21% y-o-y growth, 23% y-o-y growth at constant exchange rate (CER). This increase was primarily driven by increased sales for VYVGART, ZEJULA, and NUZYRA. VYVGART and VYVGART Hytrulo were $18.1 million in the first quarter of 2025, compared to $13.2 million for the same period in 2024. Although quarter over quarter growth was impacted by seasonality, sales grew year over year driven by increasing market coverage and penetration since VYVGART's launch in September 2023 and listing on China's National Reimbursement Drug List (NRDL) for the treatment of generalized Myasthenia Gravis (gMG) effective January 1, 2024. ZEJULA was $49.5 million in the first quarter of 2025, compared to $45.5 million for the same period in 2024. ZEJULA sales remained strong as it continued to be the leading PARP inhibitor in hospital sales for ovarian cancer in mainland China. NUZYRA was $15.1 million in the first quarter of 2025, compared to $9.9 million for the same period in 2024. This growth was supported by increasing market coverage and penetration. Research and Development (R&D) expenses were $60.7 million in the first quarter of 2025, compared to $54.6 million for the same period in 2024. This increase was primarily due to upfront fees totaling $20.0 million for our license and collaboration agreements. Other R&D expenses decreased as a result of resource prioritization and efficiency efforts. Selling, General and Administrative expenses were $63.4 million in the first quarter of 2025, compared to $69.2 million for the same period in 2024. This decrease was primarily driven by decreased personnel costs as a result of resource prioritization and efficiency efforts. Loss from operations was $56.3 million in the first quarter of 2025, $37.1 million when adjusted to exclude non-cash expenses including depreciation, amortization, and share-based compensation. A reconciliation of loss from operations (GAAP) to adjusted loss from operations (non-GAAP) is included at the end of this release. Net loss was $48.4 million in the first quarter of 2025, or a loss per ordinary share attributable to common stockholders of $0.04 (or loss per American Deposit Share (ADS) of $0.45), compared to a net loss of $53.5 million for the same period in 2024, or a loss per ordinary share of $0.05 (or loss per ADS of $0.55). These decreases in net loss were primarily due to product revenue growing faster than net operating expenses. Cash and cash equivalents, short-term investments, and current restricted cash totaled $857.3 million as of March 31, 2025, compared to $879.7 million as of December 31, 2024. Recent Pipeline Highlights Below are key product updates since our last earnings release: Oncology Pipeline ZL-1310 (DLL3 ADC): Second-Line+ Extensive-Stage SCLC (ES-SCLC): In April 2025, Zai Lab announced that updated data from an ongoing, global Phase 1a/1b clinical trial of ZL-1310 for the treatment of ES-SCLC, will be presented during a poster session at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting. The updated results will include additional patients and follow-up from the ongoing trial. Other neuroendocrine tumors: In April 2025, Zai Lab initiated a global Phase 1/2 study in patients with selected solid tumors, exploring its therapeutic potential beyond ES-SCLC. Tisotumab Vedotin (TIVDAK, Tissue Factor ADC): In March 2025, China's National Medical Products Administration (NMPA) accepted the Biologics License Application (BLA) for TIVDAK for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after systemic therapy. Zai Lab will leverage its commercial footprint of ZEJULA in women's cancer to accelerate patient access to this therapy in China if approved. Tumor Treating Fields (TTFields): In April 2025, Zai Lab partner Novocure announced that the additional results from the Phase 3 PANOVA-3 trial for pancreatic cancer will be presented as a late-breaking oral presentation at the 2025 ASCO Annual Meeting. Zai Lab participated in the study in Greater China (mainland China, Hong Kong, Macau and Taiwan, collectively) and plans to file for regulatory approval in China in 2025. Repotrectinib (ROS1/TRK): In April 2025, China's NMPA accepted the supplemental New Drug Application for repotrectinib for patients with NTRK-positive solid tumors. Repotrectinib has the potential to become a next-generation TKI that can be used across a broad range of NTRK fusion-positive solid tumors for both TKI-naïve and TKI-pretreated patients. ZL-6201 (LRRC15 ADC): In April 2025, Zai Lab presented new data at the American Association for Cancer Research (AACR) Annual Meeting 2025. The findings demonstrate that ZL-6201 efficiently internalizes within and kills tumor cells, while also exhibiting a strong bystander killing effect in the tumor microenvironment where the target is often expressed. Based on these findings, Zai Lab plans to initiate Investigational New Drug (IND)-enabling studies of ZL-6201 as a potential treatment for patients with sarcoma and other LRRC15-positive solid tumors, such as breast cancer and other malignancies, in 2025. ZL-1222 (PD-1/IL-12): In April 2025, Zai Lab presented data at the AACR Annual Meeting 2025, marking the first public disclosure of this global asset. Findings from preclinical studies demonstrate its potent anti-tumor activity in both anti-PD-1 sensitive and resistant tumor models, with improved systemic safety. These results indicate its potential to benefit patients who are unresponsive or resistant to the current immuno-oncology therapies. Immunology Pipeline Efgartigimod (FcRn): In April 2025, Zai Lab partner argenx announced the U.S. Food and Drug Administration (FDA) approved VYVGART Hytrulo prefilled syringe (PFS) for self-injection in gMG and Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). It is the third administration option providing additional flexibility and convenience for patients. Zai Lab plans to submit the Chemical Manufacturing and Control (CMC) variation for PFS for these indications in China in 2025. ZL-1102 (IL-17 Humabody®): Zai Lab has decided to discontinue the global Phase 2 clinical trial of ZL-1102 following a comprehensive review of the data from interim analysis from the first 40 enrolled participants and the subsequent recommendation of the independent Data and Safety Monitoring Board. Povetacicept (APRIL/BAFF): Zai Lab partner Vertex has completed enrollment of the interim analysis cohort in the global Phase 3 RAINIER study of povetacicept in IgA nephropathy. Zai Lab participated in the study in Greater China. Anticipated Major Milestones in 2025 Upcoming Potential NMPA Submissions Bemarituzumab (FGFR2b): BLA submission in first-line gastric cancer. Tumor Treating Fields (TTFields): Marketing Authorization Application submissions in first-line pancreatic cancer. Efgartigimod (FcRn): submission for the CMC variation for PFS in gMG and CIDP. Expected Clinical Developments and Data Readouts Global Pipeline ZL-1310 (DLL3 ADC) Second-Line+ ES-SCLC: Zai Lab to present updated data at the 2025 ASCO Annual Meeting on June 2, 2025. Zai Lab plans to initiate a pivotal study in the second half of 2025. First-Line ES-SCLC: Zai Lab to provide a data readout for dose escalation of ZL-1310 doublet in combination with atezolizumab. ZL-1503 (IL-13/IL-31R) Zai Lab to provide a preclinical data update and advance into a global Phase 1 study in moderate-to-severe atopic dermatitis. ZL-6201 (LRRC15 ADC) Zai Lab to advance into global Phase 1 development for patients with sarcoma and potentially other LRRC15-positive solid tumors, such as breast cancer and other malignancies. Regional Pipeline Bemarituzumab (FGFR2b) Zai Lab partner Amgen to provide data readout from the Phase 3 FORTITUDE-101 study of bemarituzumab combined with chemotherapy versus chemotherapy alone in first-line gastric cancer in the second quarter of 2025. Zai Lab participated in the study in Greater China. Zai Lab partner Amgen to provide data readout from the Phase 3 FORTITUDE-102 study of bemarituzumab plus chemotherapy and nivolumab versus chemotherapy and nivolumab in first-line gastric cancer in the second half of 2025. Zai Lab participated in the study in Greater China. Xanomeline-Trospium (or KarXT) (M1/M4-agonist) Zai Lab partner Bristol Myers Squibb (BMS) to provide data readout from the Phase 3 ADEPT-2 study of KarXT in Alzheimer's Disease Psychosis in the second half of 2025. Zai Lab participated in the study in Greater China. Efgartigimod (FcRn) Seronegative gMG: Zai Lab partner argenx to provide topline results from the Phase 3 ADAPT-SERON study in seronegative gMG. Zai Lab participated in the study in Greater China. Lupus Nephritis (LN): Zai Lab to provide topline results from the Phase 2 study in LN. Povetacicept (APRIL/BAFF) Primary Membranous Nephropathy (pMN): Zai Lab plans to partner with Vertex to execute the global pivotal (Phase 2/3) study of povetacicept in pMN in Greater China. Conference Call and Webcast Information Zai Lab will host a live conference call and webcast today, May 8, 2025, at 8:00 a.m. ET (8:00 p.m. HKT). Listeners may access the live webcast by visiting the Company's website at Participants must register in advance of the conference call. Details of registration links are as follows: For webcast: For dial-in: All participants must use the link provided above to complete the online registration process in advance of the conference call. Dial-in details will be in the confirmation email which the participant will receive upon registering. A replay will be available shortly after the call and can be accessed by visiting the Company's website. About Zai Lab Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) is an innovative, research-based, commercial-stage biopharmaceutical company based in China and the United States. We are focused on discovering, developing, and commercializing innovative products that address medical conditions with significant unmet needs in the areas of oncology, immunology, neuroscience, and infectious disease. Our goal is to leverage our competencies and resources to positively impact human health worldwide. For additional information about Zai Lab, please visit or follow us at Non-GAAP Measures In addition to results presented in accordance with GAAP, we disclose growth rates that have been adjusted to exclude the impact of changes due to the translation of foreign currencies into U.S. dollars. We have also presented a measure of adjusted loss from operations that adjusts GAAP loss from operations to exclude the impact of certain non-cash expenses including depreciation, amortization, and share-based compensation, which we refer to as "profitability." These adjusted growth rates and adjusted loss from operations are non-GAAP measures. We believe that these non-GAAP measures are important for an understanding of the performance of our business operations and financial results and provide investors with an additional perspective on operational trends and greater transparency into our historical and projected operating performance. Although we believe the non-GAAP financial measures enhance investors' understanding of our business and performance, these non-GAAP financial measures should not be considered an exclusive alternative to accompanying GAAP financial measures. Zai Lab Forward-Looking Statements This press release contains certain forward-looking statements, including statements relating to our strategy and plans; potential of and expectations for our business, commercial products, and pipeline programs; our goals, objectives, and priorities and our expectations under our growth strategy (including our expectations regarding our commercial products and launches, clinical stage products, revenue growth, profitability, and cash flow); clinical development programs and related clinical trials; clinical trial data, data readouts, and presentations; risks and uncertainties associated with drug development and commercialization; regulatory discussions, submissions, filings, and approvals and the timing thereof; the potential benefits, safety, and efficacy of our products and product candidates and those of our collaboration partners; the anticipated benefits and potential of investments, collaborations, and business development activities; our profitability and timeline to profitability; our future financial and operating results; and financial guidance, including with respect to our planned sources and uses of cash and our expected path to profitability. All statements, other than statements of historical fact, included in this press release are forward-looking statements, and can be identified by words such as "aim," "anticipate," "believe," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "plan," "poised," "positioned," "possible," "potential," "will," "would," and other similar expressions. Such statements constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are not guarantees or assurances of future performance. Forward-looking statements are based on our expectations and assumptions as of the date of this press release and are subject to inherent uncertainties, risks, and changes in circumstances that may differ materially from those contemplated by the forward-looking statements. We may not actually achieve the plans, carry out the intentions, or meet the expectations or projections disclosed in our forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including but not limited to (1) our ability to successfully commercialize and generate revenue from our approved products; (2) our ability to obtain funding for our operations and business initiatives; (3) the results of our clinical and pre-clinical development of our product candidates; (4) the content and timing of decisions made by the relevant regulatory authorities regarding regulatory approvals of our product candidates; (5) risks related to doing business in China; and (6) other factors identified in our most recent annual and quarterly reports and in other reports we have filed with the U.S. Securities and Exchange Commission (SEC). We anticipate that subsequent events and developments will cause our expectations and assumptions to change, and we undertake no obligation to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise, except as may be required by law. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release. Our SEC filings can be found on our website at and on the SEC's website at Zai Lab LimitedUnaudited Condensed Consolidated Balance Sheets(in thousands of U.S. dollars ($), except for number of shares and per share data) March 31, 2025 December 31, 2024 Assets Current assets Cash and cash equivalents 757,263 449,667 Restricted cash, current 100,000 100,000 Short-term investments — 330,000 Accounts receivable (net of allowance for credit losses of $22 and $25 as of March 31, 2025 and December 31, 2024, respectively) 76,555 85,178 Notes receivable 11,118 4,233 Inventories, net 53,054 39,875 Prepayments and other current assets 43,878 41,527 Total current assets 1,041,868 1,050,480 Restricted cash, non-current 1,113 1,114 Property and equipment, net 49,654 47,961 Operating lease right-of-use assets 19,081 21,496 Land use rights, net 2,882 2,907 Intangible assets, net 56,198 56,027 Other non-current assets 2,534 5,768 Total assets 1,173,330 1,185,753 Liabilities and shareholders' equity Current liabilities Accounts payable 103,017 100,906 Current operating lease liabilities 6,574 8,048 Short-term debt 173,405 131,711 Other current liabilities 36,811 58,720 Total current liabilities 319,807 299,385 Deferred income 30,126 31,433 Non-current operating lease liabilities 12,319 13,712 Other non-current liabilities 325 325 Total liabilities 362,577 344,855 Commitments and contingencies Shareholders' equity Ordinary shares (par value of $0.000006 per share; 5,000,000,000 shares authorized; 1,089,076,400 and 1,082,614,740 shares issued as of March 31, 2025 and December 31, 2024, respectively; 1,084,164,200 and 1,077,702,540 shares outstanding as of March 31, 2025 and December 31, 2024, respectively) 7 7 Additional paid-in capital 3,283,800 3,264,295 Accumulated deficit (2,501,521 ) (2,453,083 ) Accumulated other comprehensive income 49,303 50,515 Treasury Stock (at cost, 4,912,200 shares as of both March 31, 2025 and December 31, 2024) (20,836 ) (20,836 ) Total shareholders' equity 810,753 840,898 Total liabilities and shareholders' equity 1,173,330 1,185,753 Zai Lab LimitedUnaudited Condensed Consolidated Statements of Operations(in thousands of $, except for number of shares and per share data) Three Months Ended March 31, 2025 2024 Revenues Product revenue, net 105,650 87,149 Collaboration revenue 837 — Total revenues 106,487 87,149 Expenses Cost of product revenue (38,452 ) (33,619 ) Cost of collaboration revenue (195 ) — Research and development (60,729 ) (54,645 ) Selling, general, and administrative (63,422 ) (69,194 ) Loss from operations (56,311 ) (70,309 ) Interest income 8,606 9,658 Interest expenses (1,187 ) (113 ) Foreign currency gains (losses) 651 (2,068 ) Other (expense) income, net (197 ) 9,361 Loss before income tax (48,438 ) (53,471 ) Income tax expense — — Net loss (48,438 ) (53,471 ) Loss per share - basic and diluted (0.04 ) (0.05 ) Weighted-average shares used in calculating net loss per ordinary share - basic and diluted 1,080,825,300 973,145,760 Zai Lab LimitedUnaudited Condensed Consolidated Statements of Comprehensive Loss(in thousands of $) Three Months Ended March 31, 2025 2024 Net loss (48,438 ) (53,471 ) Other comprehensive income, net of tax of nil: Foreign currency translation adjustments (1,212 ) 1,542 Comprehensive loss (49,650 ) (51,929 ) Zai Lab LimitedNon-GAAP Measures(unaudited)($ in thousands)Growth on a Constant Exchange Rate (CER) Basis Three Months EndedMarch 31, Year over Year %Growth 2025 2024 As reported At CER* Product revenue, net 105,650 87,149 21 % 23 % Loss from operations (56,311 ) (70,309 ) (20 )% (20 )% * The growth rates at CER were calculated assuming the same foreign currency exchange rates were in effect for the current and prior year period. Reconciliation of Loss from Operations (GAAP) to Adjusted Loss from Operations (Non-GAAP) Three Months Ended March 31, 2025 2024 GAAP loss from operations (56,311 ) (70,309 ) Plus: Depreciation and amortization expenses 3,458 3,012 Plus: Share-based compensation 15,800 17,980 Adjusted loss from operations (37,053 ) (49,317 ) View source version on Contacts Investor Relations: Christine Chiou / Lina Zhang+1 (917) 886-6929 / +86 136 8257 / Media: Shaun Maccoun / Xiaoyu Chen+1 (415) 317-7255 / +86 185 0015 / Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
08-05-2025
- Business
- Business Wire
Zai Lab Announces First Quarter 2025 Financial Results and Recent Corporate Updates
- – Total revenues grew 22% y-o-y to $106.5 million for the first quarter of 2025; reaffirming full-year 2025 revenue guidance of $560 million to $590 million – Operating loss improved significantly, declining 20% year-over-year to $56.3 million for the first quarter of 2025, and 25% to $37.1 million on an adjusted basis 1; on track to achieve profitability 1 in the fourth quarter of 2025 – ZL-1310 (DLL3 ADC) is advancing rapidly, with upcoming ASCO 2025 data presentation in ES-SCLC; initiation of registrational study in ES-SCLC expected in the second half of 2025 – AACR 2025 presentations of ZL-6201 (LRRC15 ADC) and ZL-1222 (PD-1/IL-12) underscore the promising potential of Zai Lab's internally developed next-generation oncology therapies – Strong balance sheet with a cash position 2 of $857.3 million as of March 31, 2025, compared to $879.7 million as of December 31, 2024 Conference call and webcast today, May 8, 2025, at 8:00 a.m. ET (8:00 p.m. HKT) SHANGHAI & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) today announced financial results for the first quarter of 2025, along with recent product highlights and corporate updates. "With a strong foundation in place, we are well positioned to drive growth and profitability, and to further our mission of becoming a leading global biopharmaceutical company." Share 'In the first quarter, we continued to advance both our global pipeline and commercial business,' said Dr. Samantha Du, Founder, Chairperson, and CEO of Zai Lab. 'We are rapidly expanding our global portfolio, having recently presented promising data for two next-generation oncology therapies at AACR, and we look forward to presenting updated results for ZL-1310 (DLL3 ADC) at the 2025 ASCO Annual Meeting. We remain on track to initiate a pivotal study for ZL-1310 in SCLC this year, with the goal of securing FDA approval in 2027. In parallel, we are also exploring opportunities in first-line SCLC and other neuroendocrine tumors to unlock the full potential of this important global asset. On the commercial front, we continued to expand patient access for key products and are leveraging our existing infrastructure to support upcoming launches and our next wave of blockbuster opportunities. With a strong foundation in place, we are well positioned to drive growth and profitability, and to further our mission of becoming a leading global biopharmaceutical company.' '2025 marks a transformational year for Zai Lab, and we are executing against the key objectives we set at the start of the year,' said Josh Smiley, President and COO of Zai Lab. 'Following a seasonal slowdown of VYVGART early in the year, we saw patient volumes rebound in March and April, and we anticipate a return to strong sequential growth throughout the rest of the year. Looking ahead, our late-stage pipeline, including bemarituzumab and KarXT, is expected to fuel the next wave of topline growth alongside VYVGART. At the same time, we continue to strengthen our financial position, achieving a 20% year-over-year reduction in operating loss and a 25% year-over-year reduction in adjusted operating loss 1, keeping us on track to achieve profitability 1 in the fourth quarter of 2025. With a strong cash position, 2 a growing commercial business and an expanding global portfolio, Zai Lab is poised to capitalize on multiple upcoming catalysts and drive long-term shareholder value.' First Quarter 2025 Financial Results Product revenue, net was $105.7 million in the first quarter of 2025, compared to $87.1 million for the same period in 2024, representing 21% y-o-y growth, 23% y-o-y growth at constant exchange rate (CER). This increase was primarily driven by increased sales for VYVGART, ZEJULA, and NUZYRA. VYVGART and VYVGART Hytrulo were $18.1 million in the first quarter of 2025, compared to $13.2 million for the same period in 2024. Although quarter over quarter growth was impacted by seasonality, sales grew year over year driven by increasing market coverage and penetration since VYVGART's launch in September 2023 and listing on China's National Reimbursement Drug List (NRDL) for the treatment of generalized Myasthenia Gravis (gMG) effective January 1, 2024. ZEJULA was $49.5 million in the first quarter of 2025, compared to $45.5 million for the same period in 2024. ZEJULA sales remained strong as it continued to be the leading PARP inhibitor in hospital sales for ovarian cancer in mainland China. NUZYRA was $15.1 million in the first quarter of 2025, compared to $9.9 million for the same period in 2024. This growth was supported by increasing market coverage and penetration. Research and Development (R&D) expenses were $60.7 million in the first quarter of 2025, compared to $54.6 million for the same period in 2024. This increase was primarily due to upfront fees totaling $20.0 million for our license and collaboration agreements. Other R&D expenses decreased as a result of resource prioritization and efficiency efforts. Selling, General and Administrative expenses were $63.4 million in the first quarter of 2025, compared to $69.2 million for the same period in 2024. This decrease was primarily driven by decreased personnel costs as a result of resource prioritization and efficiency efforts. Loss from operations was $56.3 million in the first quarter of 2025, $37.1 million when adjusted to exclude non-cash expenses including depreciation, amortization, and share-based compensation. A reconciliation of loss from operations (GAAP) to adjusted loss from operations (non-GAAP) is included at the end of this release. Net loss was $48.4 million in the first quarter of 2025, or a loss per ordinary share attributable to common stockholders of $0.04 (or loss per American Deposit Share (ADS) of $0.45), compared to a net loss of $53.5 million for the same period in 2024, or a loss per ordinary share of $0.05 (or loss per ADS of $0.55). These decreases in net loss were primarily due to product revenue growing faster than net operating expenses. Cash and cash equivalents, short-term investments, and current restricted cash totaled $857.3 million as of March 31, 2025, compared to $879.7 million as of December 31, 2024. Recent Pipeline Highlights Below are key product updates since our last earnings release: Oncology Pipeline ZL-1310 (DLL3 ADC): Second-Line+ Extensive-Stage SCLC (ES-SCLC): In April 2025, Zai Lab announced that updated data from an ongoing, global Phase 1a/1b clinical trial of ZL-1310 for the treatment of ES-SCLC, will be presented during a poster session at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting. The updated results will include additional patients and follow-up from the ongoing trial. Other neuroendocrine tumors: In April 2025, Zai Lab initiated a global Phase 1/2 study in patients with selected solid tumors, exploring its therapeutic potential beyond ES-SCLC. Tisotumab Vedotin (TIVDAK, Tissue Factor ADC): In March 2025, China's National Medical Products Administration (NMPA) accepted the Biologics License Application (BLA) for TIVDAK for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after systemic therapy. Zai Lab will leverage its commercial footprint of ZEJULA in women's cancer to accelerate patient access to this therapy in China if approved. Tumor Treating Fields (TTFields): In April 2025, Zai Lab partner Novocure announced that the additional results from the Phase 3 PANOVA-3 trial for pancreatic cancer will be presented as a late-breaking oral presentation at the 2025 ASCO Annual Meeting. Zai Lab participated in the study in Greater China (mainland China, Hong Kong, Macau and Taiwan, collectively) and plans to file for regulatory approval in China in 2025. Repotrectinib (ROS1/TRK): In April 2025, China's NMPA accepted the supplemental New Drug Application for repotrectinib for patients with NTRK -positive solid tumors. Repotrectinib has the potential to become a next-generation TKI that can be used across a broad range of NTRK fusion-positive solid tumors for both TKI-naïve and TKI-pretreated patients. ZL-6201 (LRRC15 ADC): In April 2025, Zai Lab presented new data at the American Association for Cancer Research (AACR) Annual Meeting 2025. The findings demonstrate that ZL-6201 efficiently internalizes within and kills tumor cells, while also exhibiting a strong bystander killing effect in the tumor microenvironment where the target is often expressed. Based on these findings, Zai Lab plans to initiate Investigational New Drug (IND)-enabling studies of ZL-6201 as a potential treatment for patients with sarcoma and other LRRC15-positive solid tumors, such as breast cancer and other malignancies, in 2025. ZL-1222 (PD-1/IL-12): In April 2025, Zai Lab presented data at the AACR Annual Meeting 2025, marking the first public disclosure of this global asset. Findings from preclinical studies demonstrate its potent anti-tumor activity in both anti-PD-1 sensitive and resistant tumor models, with improved systemic safety. These results indicate its potential to benefit patients who are unresponsive or resistant to the current immuno-oncology therapies. Immunology Pipeline Efgartigimod (FcRn): In April 2025, Zai Lab partner argenx announced the U.S. Food and Drug Administration (FDA) approved VYVGART Hytrulo prefilled syringe (PFS) for self-injection in gMG and Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). It is the third administration option providing additional flexibility and convenience for patients. Zai Lab plans to submit the Chemical Manufacturing and Control (CMC) variation for PFS for these indications in China in 2025. ZL-1102 (IL-17 Humabody ®): Zai Lab has decided to discontinue the global Phase 2 clinical trial of ZL-1102 following a comprehensive review of the data from interim analysis from the first 40 enrolled participants and the subsequent recommendation of the independent Data and Safety Monitoring Board. Povetacicept (APRIL/BAFF): Zai Lab partner Vertex has completed enrollment of the interim analysis cohort in the global Phase 3 RAINIER study of povetacicept in IgA nephropathy. Zai Lab participated in the study in Greater China. Anticipated Major Milestones in 2025 Upcoming Potential NMPA Submissions Bemarituzumab (FGFR2b): BLA submission in first-line gastric cancer. Tumor Treating Fields (TTFields): Marketing Authorization Application submissions in first-line pancreatic cancer. Efgartigimod (FcRn): submission for the CMC variation for PFS in gMG and CIDP. Expected Clinical Developments and Data Readouts Global Pipeline ZL-1310 (DLL3 ADC) Second-Line+ ES-SCLC: Zai Lab to present updated data at the 2025 ASCO Annual Meeting on June 2, 2025. Zai Lab plans to initiate a pivotal study in the second half of 2025. First-Line ES-SCLC: Zai Lab to provide a data readout for dose escalation of ZL-1310 doublet in combination with atezolizumab. ZL-1503 (IL-13/IL-31R) Zai Lab to provide a preclinical data update and advance into a global Phase 1 study in moderate-to-severe atopic dermatitis. ZL-6201 (LRRC15 ADC) Zai Lab to advance into global Phase 1 development for patients with sarcoma and potentially other LRRC15-positive solid tumors, such as breast cancer and other malignancies. Regional Pipeline Bemarituzumab (FGFR2b) Zai Lab partner Amgen to provide data readout from the Phase 3 FORTITUDE-101 study of bemarituzumab combined with chemotherapy versus chemotherapy alone in first-line gastric cancer in the second quarter of 2025. Zai Lab participated in the study in Greater China. Zai Lab partner Amgen to provide data readout from the Phase 3 FORTITUDE-102 study of bemarituzumab plus chemotherapy and nivolumab versus chemotherapy and nivolumab in first-line gastric cancer in the second half of 2025. Zai Lab participated in the study in Greater China. Xanomeline-Trospium (or KarXT) (M1/M4-agonist) Zai Lab partner Bristol Myers Squibb (BMS) to provide data readout from the Phase 3 ADEPT-2 study of KarXT in Alzheimer's Disease Psychosis in the second half of 2025. Zai Lab participated in the study in Greater China. Efgartigimod (FcRn) Seronegative gMG: Zai Lab partner argenx to provide topline results from the Phase 3 ADAPT-SERON study in seronegative gMG. Zai Lab participated in the study in Greater China. Lupus Nephritis (LN): Zai Lab to provide topline results from the Phase 2 study in LN. Povetacicept (APRIL/BAFF) Primary Membranous Nephropathy (pMN): Zai Lab plans to partner with Vertex to execute the global pivotal (Phase 2/3) study of povetacicept in pMN in Greater China. Conference Call and Webcast Information Zai Lab will host a live conference call and webcast today, May 8, 2025, at 8:00 a.m. ET (8:00 p.m. HKT). Listeners may access the live webcast by visiting the Company's website at Participants must register in advance of the conference call. Details of registration links are as follows: All participants must use the link provided above to complete the online registration process in advance of the conference call. Dial-in details will be in the confirmation email which the participant will receive upon registering. A replay will be available shortly after the call and can be accessed by visiting the Company's website. About Zai Lab Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) is an innovative, research-based, commercial-stage biopharmaceutical company based in China and the United States. We are focused on discovering, developing, and commercializing innovative products that address medical conditions with significant unmet needs in the areas of oncology, immunology, neuroscience, and infectious disease. Our goal is to leverage our competencies and resources to positively impact human health worldwide. For additional information about Zai Lab, please visit or follow us at Non-GAAP Measures In addition to results presented in accordance with GAAP, we disclose growth rates that have been adjusted to exclude the impact of changes due to the translation of foreign currencies into U.S. dollars. We have also presented a measure of adjusted loss from operations that adjusts GAAP loss from operations to exclude the impact of certain non-cash expenses including depreciation, amortization, and share-based compensation, which we refer to as 'profitability.' These adjusted growth rates and adjusted loss from operations are non-GAAP measures. We believe that these non-GAAP measures are important for an understanding of the performance of our business operations and financial results and provide investors with an additional perspective on operational trends and greater transparency into our historical and projected operating performance. Although we believe the non-GAAP financial measures enhance investors' understanding of our business and performance, these non-GAAP financial measures should not be considered an exclusive alternative to accompanying GAAP financial measures. Zai Lab Forward-Looking Statements This press release contains certain forward-looking statements, including statements relating to our strategy and plans; potential of and expectations for our business, commercial products, and pipeline programs; our goals, objectives, and priorities and our expectations under our growth strategy (including our expectations regarding our commercial products and launches, clinical stage products, revenue growth, profitability, and cash flow); clinical development programs and related clinical trials; clinical trial data, data readouts, and presentations; risks and uncertainties associated with drug development and commercialization; regulatory discussions, submissions, filings, and approvals and the timing thereof; the potential benefits, safety, and efficacy of our products and product candidates and those of our collaboration partners; the anticipated benefits and potential of investments, collaborations, and business development activities; our profitability and timeline to profitability; our future financial and operating results; and financial guidance, including with respect to our planned sources and uses of cash and our expected path to profitability. All statements, other than statements of historical fact, included in this press release are forward-looking statements, and can be identified by words such as 'aim,' 'anticipate,' 'believe,' 'could,' 'estimate,' 'expect,' 'forecast,' 'goal,' 'intend,' 'may,' 'plan,' 'poised,' 'positioned,' 'possible,' 'potential,' 'will,' 'would,' and other similar expressions. Such statements constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are not guarantees or assurances of future performance. Forward-looking statements are based on our expectations and assumptions as of the date of this press release and are subject to inherent uncertainties, risks, and changes in circumstances that may differ materially from those contemplated by the forward-looking statements. We may not actually achieve the plans, carry out the intentions, or meet the expectations or projections disclosed in our forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including but not limited to (1) our ability to successfully commercialize and generate revenue from our approved products; (2) our ability to obtain funding for our operations and business initiatives; (3) the results of our clinical and pre-clinical development of our product candidates; (4) the content and timing of decisions made by the relevant regulatory authorities regarding regulatory approvals of our product candidates; (5) risks related to doing business in China; and (6) other factors identified in our most recent annual and quarterly reports and in other reports we have filed with the U.S. Securities and Exchange Commission (SEC). We anticipate that subsequent events and developments will cause our expectations and assumptions to change, and we undertake no obligation to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise, except as may be required by law. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release. Our SEC filings can be found on our website at and on the SEC's website at March 31, 2025 2024 Assets Current assets Cash and cash equivalents 757,263 449,667 Restricted cash, current 100,000 100,000 Short-term investments — 330,000 Accounts receivable (net of allowance for credit losses of $22 and $25 as of March 31, 2025 and December 31, 2024, respectively) 76,555 85,178 Notes receivable 11,118 4,233 Inventories, net 53,054 39,875 Prepayments and other current assets 43,878 41,527 Total current assets 1,041,868 1,050,480 Restricted cash, non-current 1,113 1,114 Property and equipment, net 49,654 47,961 Operating lease right-of-use assets 19,081 21,496 Land use rights, net 2,882 2,907 Intangible assets, net 56,198 56,027 Other non-current assets 2,534 5,768 Total assets 1,173,330 1,185,753 Liabilities and shareholders' equity Current liabilities Accounts payable 103,017 100,906 Current operating lease liabilities 6,574 8,048 Short-term debt 173,405 131,711 Other current liabilities 36,811 58,720 Total current liabilities 319,807 299,385 Deferred income 30,126 31,433 Non-current operating lease liabilities 12,319 13,712 Other non-current liabilities 325 325 Total liabilities 362,577 344,855 Commitments and contingencies Shareholders' equity Ordinary shares (par value of $0.000006 per share; 5,000,000,000 shares authorized; 1,089,076,400 and 1,082,614,740 shares issued as of March 31, 2025 and December 31, 2024, respectively; 1,084,164,200 and 1,077,702,540 shares outstanding as of March 31, 2025 and December 31, 2024, respectively) 7 7 Additional paid-in capital 3,283,800 3,264,295 Accumulated deficit (2,501,521 ) (2,453,083 ) Accumulated other comprehensive income 49,303 50,515 Treasury Stock (at cost, 4,912,200 shares as of both March 31, 2025 and December 31, 2024) (20,836 ) (20,836 ) Total shareholders' equity 810,753 840,898 Total liabilities and shareholders' equity 1,173,330 1,185,753 Expand Zai Lab Limited Unaudited Condensed Consolidated Statements of Operations (in thousands of $, except for number of shares and per share data) Three Months Ended March 31, 2025 2024 Revenues Product revenue, net 105,650 87,149 Collaboration revenue 837 — Total revenues 106,487 87,149 Expenses Cost of product revenue (38,452 ) (33,619 ) Cost of collaboration revenue (195 ) — Research and development (60,729 ) (54,645 ) Selling, general, and administrative (63,422 ) (69,194 ) Loss from operations (56,311 ) (70,309 ) Interest income 8,606 9,658 Interest expenses (1,187 ) (113 ) Foreign currency gains (losses) 651 (2,068 ) Other (expense) income, net (197 ) 9,361 Loss before income tax (48,438 ) (53,471 ) Income tax expense — — Net loss (48,438 ) (53,471 ) Loss per share - basic and diluted (0.04 ) (0.05 ) Weighted-average shares used in calculating net loss per ordinary share - basic and diluted 1,080,825,300 973,145,760 Expand Zai Lab Limited Unaudited Condensed Consolidated Statements of Comprehensive Loss (in thousands of $) Three Months Ended March 31, 2025 2024 Net loss (48,438 ) (53,471 ) Other comprehensive income, net of tax of nil: Foreign currency translation adjustments (1,212 ) 1,542 Comprehensive loss (49,650 ) (51,929 ) Expand Zai Lab Limited Non-GAAP Measures (unaudited) ($ in thousands) Growth on a Constant Exchange Rate (CER) Basis Contacts Investor Relations: Christine Chiou / Lina Zhang +1 (917) 886-6929 / +86 136 8257 6943 / Media: Shaun Maccoun / Xiaoyu Chen +1 (415) 317-7255 / +86 185 0015 5011 / Get RSS Feed Zai Lab to Present Data from Phase 1 Trial of DLL3-Targeted Antibody-Drug Conjugate (ADC) ZL-1310 at 2025 ASCO Annual Meeting SHANGHAI & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) today announced that updated data from an ongoing, global Phase 1a/1b clinical trial (NCT06179069) evaluating ZL-1310, the Company's potential first-in-class and best-in-class, Delta-like ligand (DLL3) antibody-drug conjugate (ADC) for the treatment of extensive-stage small cell lung cancer (ES-SCLC), will be presented during a poster session at the 2025 American Society of Clinical Oncology (ASCO) Annual M... Zai Lab Limited NASDAQ:ZLAB Release Versions English Contacts Investor Relations: Christine Chiou / Lina Zhang +1 (917) 886-6929 / +86 136 8257 6943 / Media: Shaun Maccoun / Xiaoyu Chen +1 (415) 317-7255 / +86 185 0015 5011 /
Yahoo
08-05-2025
- Business
- Yahoo
argenx Reports First Quarter 2025 Financial Results and Provides Business Update
VYVGART® (IV: efgartigimod alfa-fcab and SC: efgartigimod alfa and hyaluronidase-qvfc) is a first-and-only targeted IgG Fc-antibody fragment approved 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., Japan and China. The VYVGART-SC pre-filled syringe (PFS) is now approved for use in the U.S. and EU. argenx is well-positioned to sustain commercial growth through 2025, driven by global expansion, earlier treatment adoption, and the launch of the PFS to support growth momentum in both gMG and CIDP. In addition to bringing VYVGART to more patients early in the treatment paradigm, argenx is working to reach broader MG populations with ongoing studies in seronegative, ocular, and pediatric MG. argenx has established its strategic priorities to advance 'Vision 2030', aiming to treat 50,000 patients globally with its medicines, secure 10 labeled indications across all approved medicines, and advance five pipeline candidates into Phase 3 development by 2030. 'We continue to execute on our bold innovation agenda, guided by our 'Vision 2030' to reach 50,000 patients across 10 labeled indications,' said Tim Van Hauwermeiren, Chief Executive Officer of argenx. 'We remain committed to delivering meaningful outcomes with VYVGART by setting a new benchmark for sustained efficacy and safety, and generating data that matter most to improving the lives of patients. This strategy has driven strong launch fundamentals to date, and we see consistent patient and prescriber expansion in both gMG and CIDP. Looking forward, we have several reasons to be confident in our growth trajectory. We are thrilled to bring even more optionality to gMG and CIDP patients with the recent approval of our pre-filled syringe for self-injection in the United States, receiving an optimal label that supports our ability to reach patients earlier in the treatment paradigm. In line with our 'Vision 2030', we are advancing 10 Phase 2 and 10 Phase 3 studies across efgartigimod, empasiprubart and ARGX-119, creating significant opportunity to expand into new therapeutic areas and reach broader patient populations. By year end, we expect key insights from proof-of-concept and registrational studies across many of these programs, while continuing to progress four IND candidates that reflect the depth and diversity of our pipeline.' 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 2025 financial results and provided a business update. First patients treated with VYVGART Hytrulo pre-filled syringe for self-injection in US and Germany Story Continues Generated global product net sales (inclusive of both VYVGART and VYVGART SC) of $790 million in the first quarter of 2025 Strong underlying fundamentals across key patient and prescriber metrics with 99% product net sales growth year-over-year from first quarter 2024, and 7% product net sales growth from fourth quarter 2024 Multiple regulatory decisions on approval for PFS completed or underway: First patients treated with VYVGART-SC PFS for self-injection in the U.S. and Germany following regulatory approval Received positive recommendation from Committee for Medicinal Products for Human Use (CHMP) of European Medicines Agency (EMA) for VYVGART-SC (PFS and vial) for CIDP PFS decision on approval for gMG and CIDP expected in Japan and Canada by end of year Evidence generation through Phase 4 and label-enabling studies in MG, CIDP and ITP: Topline results expected in second half of 2025 for seronegative gMG (ADAPT-SERON) and first half of 2026 for ocular and pediatric MG (ADAPT-OCULUS, JR) Topline results from Phase 4 switch study to inform treatment decisions when switching patients on IVIg to VYVGART SC in CIDP expected in second half of 2025 and to be presented at an upcoming medical meeting ADVANCE-NEXT topline results expected in second half of 2026 to support FDA submission of VYVGART IV for primary ITP Execute 10 registrational and 10 proof-of-concept studies across efgartigimod, empasiprubart and ARGX-119 to advance the next wave of launches argenx continues to demonstrate breadth and depth within its immunology pipeline, advancing multiple first-in-class product candidates with potential across high-need indications. argenx is solidifying its leadership in FcRn biology with efgartigimod, complement inhibition with empasiprubart and in the role of MuSK at the neuromuscular junction with ARGX-119. Efgartigimod Development Efgartigimod is being evaluated in 15 severe autoimmune diseases (including MG, CIDP, and ITP), exploring the significance of FcRn biology across neurology and rheumatology indications, as well as new therapeutic areas. Registrational studies are currently ongoing in three subsets of myositis, thyroid eye disease (TED), and Sjögren's disease. Topline results from ALKIVIA study evaluating three myositis subsets (immune-mediated necrotizing myopathy (IMNM), anti-synthetase syndrome (ASyS), and dermatomyositis (DM)) expected in second half of 2026 Topline results from two registrational UplighTED studies (TED) expected in second half of 2026 Topline results from registrational UNITY study (Sjögren's disease) expected in 2027 Proof-of-concept studies ongoing in lupus nephritis (LN), systemic sclerosis (SSc) and antibody mediated rejection (AMR); topline results expected for LN in fourth quarter of 2025, SSc in second half of 2026, and AMR in 2027 Empasiprubart Development Empasiprubart is currently being evaluated in four indications, including two registrational studies in multifocal motor neuropathy (MMN) and CIDP, and proof-of-concept studies in delayed graft function (DGF) and DM. Topline results from registrational EMPASSION study (MMN) evaluating empasiprubart head-to-head versus IVIg expected in second half of 2026 Registrational EMVIGORATE study in CIDP evaluating empasiprubart head-to-head versus IVIg expected to start in first half of 2025 Topline results expected for DGF in second half of 2025 and for DM in first half of 2026 ARGX-119 Development ARGX-119 is being evaluated in congenital myasthenic syndromes (CMS), amyotrophic lateral sclerosis (ALS), and spinal muscular atrophy (SMA). Phase 1b proof-of-concept study ongoing in CMS; topline results expected in second half of 2025 Phase 2a proof-of-concept study ongoing in ALS; topline results expected in first half of 2026 SMA proof-of-concept study on track to start in 2025 Advance four new pipeline molecules and generate sustainable value through continued investment in Immunology Innovation Program 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 biology; ARGX-121, a first-in-class molecule targeting IgA; ARGX-109, targeting IL-6, which plays an important role in inflammation, and a fourth pipeline candidate, a first-in-class sweeping antibody for which the target has not yet been disclosed. Phase 1 results from ongoing ARGX-109 study expected in second half of 2025, and from ongoing ARGX-213 study and ARGX-121 study expected in first half of 2026. FIRST QUARTER 2025 FINANCIAL RESULTS argenx SE UNAUDITED CONDENSED CONSOLIDATED INTERIM STATEMENTS OF PROFIT OR LOSS Three Months Ended March 31, (in thousands of $ except for shares and EPS) 2025 2024 Product net sales $ 790,050 $ 398,283 Collaboration revenue 633 2,718 Other operating income 16,687 11,512 Total operating income 807,370 412,513 Cost of sales $ (80,805) $ (43,178) Research and development expenses (309,070) (224,969) Selling, general and administrative expenses (276,248) (235,995) Loss from investment in a joint venture (2,307) (1,792) Total operating expenses (668,430) (505,934) Operating profit/(loss) $ 138,940 $ (93,421) Financial income $ 37,118 $ 38,895 Financial expense (1,135) (512) Exchange gains/(losses) 27,438 (19,312) Profit/(loss) for the period before taxes $ 202,361 $ (74,350) Income tax (expense)/benefit $ (32,892) $ 12,753 Profit/(loss) for the period $ 169,469 $ (61,597) Profit/(loss) for the period attributable to: Owners of the parent $ 169,469 $ (61,597) Weighted average number of shares outstanding 60,983,325 59,309,996 Basic profit/(loss) per share (in $) $ 2.78 $ (1.04) Weighted average number of shares outstanding for diluted profit/(loss) per share 65,664,300 59,309,996 Diluted profit/(loss) per share (in $) $ 2.58 $ (1.04) DETAILS OF THE FINANCIAL RESULTS Total operating income for the three months ended March 31, 2025, was $807 million compared to $413 million for the same period in 2024, and consists of: Product net sales of VYVGART and VYVGART SC for the three months ended March 31, 2025, were $790 million compared to $398 million for the same period in 2024. Other operating income for the three months ended March 31, 2025, was $17 million compared to $12 million for the same period in 2024. The other operating income primarily relates to research and development tax incentives and payroll tax rebates. Total operating expenses for the three months ended March 31, 2025, were $668 million compared to $506 million for the same period in 2024, and mainly consists of: Cost of sales for the three months ended March 31, 2025, was $81 million compared to $43 million for the same period in 2024. 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, 2025, were $309 million compared to $225 million for the same period in 2024. The expenses mainly relate to: the clinical development and expansion of efgartigimod in 15 severe autoimmune diseases; the ramp-up of studies for our development of empasiprubart into MMN, DGF, DM and CIDP; the investments for ARGX-119 in proof-of-concept studies ongoing in ALS and CMS; and other discovery and preclinical pipeline candidates. Selling, general and administrative expenses for the three months ended March 31, 2025, were $276 million compared to $236 million for the same period in 2024. The selling, general and administrative expenses mainly relate to professional and marketing fees linked to global commercialization of the VYVGART franchise, and personnel expenses. Financial income for the three months ended March 31, 2025, was $37 million compared to $39 million for the same period in 2024. Exchange gains for the three months ended March 31, 2025, were $27 million compared to exchange losses of $19 million for the same period in 2024. Exchange gains and 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, 2025, consisted of $33 million of income tax expense compared to income tax benefit of $13 million for the same period in 2024. Income tax expense for the three months ended March 31, 2025, consists of $29 million of current income tax expense and $4 million of deferred tax expense, compared to $6 million of current income tax expense and $19 million of deferred tax benefit for the comparable prior period. Profit for the period of three months ended March 31, 2025, was $169 million compared to a loss for the period of $62 million in 2024. The profit per share was $2.78 compared to a loss per share of $1.04 for the three months ended March 31, 2025 and 2024, respectively. FINANCIAL GUIDANCE The financial guidance on the combined selling, general and administrative expenses and research and development expenses remains unchanged at approximately $2.5 billion. EXPECTED 2025 FINANCIAL CALENDAR May 27, 2025: Annual General Meeting of Shareholders in Amsterdam, the Netherlands July 31, 2025: Half Year and Second Quarter 2025 Financial Results and Business Update October 30, 2025: Q3 2025 Financial Results and Business Update CONFERENCE CALL DETAILS The first quarter 2025 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 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 50 201 France 33 800 943355 Netherlands 31 20 795 1090 United Kingdom 44 800 358 0970 United States 1 888 415 4250 Japan 81 3 4578 9081 Switzerland 41 43 210 11 32 This press release contains inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation (Regulation 596/2014). 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 and is evaluating its broad potential in multiple serious autoimmune diseases while advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit and follow us on LinkedIn, Instagram, Facebook, and YouTube. For further information, please contact: Media: Ben Petok bpetok@ Investors: Alexandra Roy aroy@ 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,' 'anticipate,' 'are,' 'believe,' 'can,' 'continue,' 'expect,' 'may,' 'strive,' and 'will' and include statements argenx makes concerning its innovation agenda and growth strategy, including its Vision 2030 to reach 50,000 patients globally across 10 labeled indications and to advance 10 Phase 2 and 10 Phase 3 studies across efgartigimod, empasiprubart and ARGX-119 to create significant opportunity to expand into new therapeutic areas and reach broader patient populations; its commitment to delivering meaningful outcomes with VYVGART by setting a new benchmark for sustained efficacy and safety, and generating data that matter most to improving the lives of patients; the patient and prescriber expansion in both gMG and CIDP; our confidence in our growth trajectory; its goal to bring even more optionality to gMG and CIDP patients; its ability to reach patients earlier in the treatment paradigm; its expectation regarding the insights from proof-of-concept and registrational studies across various programs; its belief that argenx is well-positioned to sustain commercial growth through 2025, driven by global expansion, earlier treatment adoption, and the launch of the PFS to support growth momentum in both gMG and CIDP; its goal to reach broader MG populations with ongoing studies in seronegative, ocular and pediatric MG; the advancement of anticipated clinical development, data readouts and regulatory milestones and plans, including: (1) PFS decision on approval for gMG and CIDP expected in Japan and Canada by end of 2025; (2) topline results for seronegative gMG (ADAPT-SERON) expected in second half of 2025 and for ocular and pediatric MG (ADAPT-OCULUS, JR) expected in first half of 2026; (3) topline results from Phase 4 switch study to inform treatment decisions when switching patient on IVIg to VYVGART SC in CIDP expected in the second half of 2025; (4) topline results for ADVANCE-NEXT to support FDA submission of VYVGART IV for primary ITP expected in second half of 2026; (5) its plan to execute 10 registrational and 10 proof-of-concept studies across efgartigimod, empasiprubart and ARGX-119 to advance the next wave of launches by exploring the significance of FcRn biology across neurology and rheumatology indications, as well as new therapeutic areas and ongoing registrational studies in three subsets of myositis, thyroid eye disease (TED), and Sjögren's disease, with topline results from (a) ALKIVIA expected in second half of 2026, (b) two registrational UplightTED studies expected in second half of 2026 and (c) registrational UNITY study expected in 2027, (6) proof-of-studies ongoing in LN, SSc and AMR, with topline results expected in fourth quarter of 2025, second half of 2026 and 2027, respectively; (7) its plans to develop empasiprubart, including (a) registrational EMPASSION study in MMN, with topline results expected in second half of 2026, (b) registrational EMVIGORATE study in CIDP, expected to start in first half of 2025 and (c) topline results for DGM and DM expected in second half of 2025 and first half of 2026, respectively; (8) its plans to develop ARGX-119, including: (a) Phase 1b proof-of-concept study in CMS, with topline results expected in second half of 2025; (b) Phase 2a proof-of-concept study in ALS, with topline results expected in first half of 2026; and (c) SMA proof-of-concept study, on track to start in 2025; and (9) its plans to advance four new pipeline molecules and generate sustainable value through continue investment in its IIP, through (a) ongoing studies for ARGX-213 and ARGX-121, with results expected in first half of 2026, (b) ARGX-109, with Phase 1 results expected in second half of 2025, and (c) a fourth pipeline candidate, a first-in-class sweeping antibody for which the target has not yet been disclosed; its 2025 anticipated research and development, selling, general and administrative expenses; 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 but not limited to, 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; the acceptance of its products and product candidates by its patients as safe, effective and cost-effective; the impact of governmental laws and regulations, including tariffs, export controls, sanctions and other regulations on its business; its reliance on third-party suppliers, service providers and manufacturers; 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.
Yahoo
28-04-2025
- Business
- Yahoo
argenx Announces Positive CHMP Opinion for VYVGART (efgartigimod alfa) Subcutaneous Injection for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
VYVGART® is first-and-only targeted IgG Fc-antibody fragment for CIDP First novel mechanism of action for CIDP treatment in more than 30 years CHMP positive opinion based on ADHERE data, the largest ever CIDP clinical trial European Commission (EC) decision on marketing authorization application (MAA) expected within approximately two months April 28, 2025, 07:00 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 the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended European Commission (EC) approval of VYVGART® 1000mg (efgartigimod alfa) for subcutaneous (SC) injection as a monotherapy for the treatment of adult patients with progressive or relapsing active chronic inflammatory demyelinating polyneuropathy (CIDP) after prior treatment with corticosteroids or immunoglobulins. 'Our mission is to develop innovative, targeted treatments for patients with rare and severe autoimmune diseases, who continue to face significant unmet needs. The positive CHMP opinion for VYVGART in CIDP brings us one step closer to providing patients across Europe with a transformational new treatment option that provides meaningful functional improvement,' said Luc Truyen M.D., Ph.D., Chief Medical Officer, argenx. 'VYVGART is the first and only targeted IgG Fc-antibody fragment for CIDP and if approved, would mark the first treatment in Europe with a novel, precision mechanism of action for CIDP patients in 30 years.' VYVGART for subcutaneous injection is available as a vial or prefilled syringe and can be administered by a patient, caregiver, or healthcare professional. Treatment is initiated with a weekly dose regimen and may be adjusted to every other week based on clinical evaluation. The CHMP recommendation is based on positive results from the ADHERE clinical trial, the largest study of CIDP patients to date. In the ADHERE study, 66.5% (214/322) of patients treated with VYVGART, regardless of prior treatment, demonstrated evidence of clinical improvement, including improvements in mobility, function and strength. ADHERE met its primary endpoint (p<0.0001) demonstrating a 61% reduction (HR: 0.39 95% CI: 0.25; 0.61) in the risk of relapse versus placebo. The study also demonstrated functional improvements across the Inflammatory Neuropathy Cause and Treatment (INCAT) disability scores (>1-point), grip strength (>17 kPa) and I-RODS scale (>8 points) at week 36 compared to baseline at entry to standard of care withdrawal phase. Ninety-nine percent of trial participants elected to participate in the ADHERE open-label extension. The safety results were generally consistent with the known safety profile of VYVGART in previous clinical studies. "For the patient population represented by the European Patient Organisation for Dysimmune and Inflammatory Neuropathies (EPODIN) and for those affected by CIDP, this is excellent news," said Jean-Philippe Plançon, President of EPODIN. "There are still considerable unmet medical needs in the management of CIDP, and the CHMP's recommendation brings renewed hope for improved treatment options and quality of life." The positive CHMP opinion is a scientific recommendation for marketing authorization, serving as a basis for the EC's final decision on argenx's CIDP application for subcutaneous VYVGART. The EC is expected to make a decision following CHMP recommendation and the decision will apply to all 27 European Union Member States, and also to Iceland, Norway and Liechtenstein. Currently, VYVGART is indicated as an add-on to standard therapy for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. About ADHERE The ADHERE trial was a multicenter, randomized, double-blind, placebo-controlled trial evaluating SC efgartigimod alfa for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP). ADHERE enrolled 322 adult patients with CIDP, 130 of whom were based in Europe, who were off treatment (not on active treatment within the past six months or newly diagnosed) or being treated with immunoglobulin therapy or corticosteroids. The trial consisted of an open-label Stage A followed by a randomized, placebo-controlled Stage B. In order to be eligible for the trial, the diagnosis of CIDP was confirmed by an independent panel of experts. Patients entered a run-in stage, where any ongoing CIDP treatment was stopped and in order to be eligible for Stage A had to demonstrate active disease, with clinically meaningful worsening on at least one CIDP clinical assessment tool, including INCAT, I-RODS, or mean grip strength. Treatment naïve patients were able to skip the run-in period with proof of recent worsening. To advance to Stage B, patients needed to demonstrate evidence of clinical improvement (ECI) with SC efgartigimod alfa. ECI was achieved through improvement of the INCAT score, or improvement on I- RODS or mean grip strength if those scales had demonstrated worsening during the run-in period. In Stage B, patients were randomized to either SC efgartigimod alfa or placebo for up to 48 weeks. The primary endpoint was measured once 88 total relapses or events were achieved in Stage B and was based on the hazard ratio for the time to first adjusted INCAT deterioration (i.e. relapse). After Stage B, all patients had the option to roll-over to an open-label extension study to receive SC efgartigimod alfa. About Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare and serious autoimmune disease of the peripheral nervous system. There is increasing evidence that IgG antibodies play a key role in the damage to the peripheral nerves. People with CIDP experience fatigue, muscle weakness and a loss of feeling in their arms and legs that can worsen over time or may come and go. These symptoms can significantly impair a person's ability to function in their daily lives. Without treatment, one-third of people living with CIDP will need a wheelchair. There are an estimated 31,413 people living with CIDP in the European Union. About EfgartigimodEfgartigimod is an antibody fragment designed to reduce pathogenic immunoglobulin G (IgG) antibodies by binding to the neonatal Fc receptor and blocking the IgG recycling process. Efgartigimod is being investigated in several autoimmune diseases known to be mediated by disease-causing IgG antibodies, including neuromuscular disorders, blood disorders, and skin blistering diseases, in both an IV and SC formulation. SC efgartigimod is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme's ENHANZE® drug delivery technology. In August 2022, efgartigimod received approval from the EC for IV administration as an add on to standard therapy for the treatment of adult patients with gMG who are AChR antibody positive. 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 and is evaluating its broad potential in multiple serious autoimmune diseases while advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit and follow us on LinkedIn, Instagram, Facebook, and YouTube. Contacts Media:Kate Dion kdion@ Investors: Alexandra Royaroy@ 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,' 'are,' 'believe,' 'can,' 'continue,' 'expect,' 'may,' and 'will' and include statements argenx makes concerning the expected timing and decision of the EC regarding VYVGART for SC injection for CIDP treatment and the application of such decision; the potential for improved treatment options and quality of life; 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 but not limited to, 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; the acceptance of its products and product candidates by its patients as safe, effective and cost-effective; the impact of governmental laws and regulations, including tariffs, export controls, sanctions and other regulations on its business; its reliance on third-party suppliers, service providers and manufacturers; 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 in to access your portfolio
