Latest news with #Sobi
Yahoo
8 hours ago
- Business
- Yahoo
TRYNGOLZA® (olezarsen) recommended for approval in the EU by CHMP for familial chylomicronemia syndrome (FCS)
– Recommendation based on Phase 3 Balance results, which showed a significant reduction of triglycerides and substantial reduction of acute pancreatitis events with TRYNGOLZA – – European Commission decision expected by Q4 2025 – CARLSBAD, Calif., July 25, 2025--(BUSINESS WIRE)--Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Sobi® today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion of TRYNGOLZA® (olezarsen) as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS). The positive opinion is now referred to the European Commission (EC) for an approval decision, which is expected by Q4 2025. "Building on the strong early launch of TRYNGOLZA in the U.S., the positive CHMP opinion advances our commitment to expand access to TRYNGOLZA globally," said Brett P. Monia, Ph.D., chief executive officer, Ionis. "TRYNGOLZA has demonstrated significant reductions in triglycerides and substantial reductions in acute pancreatitis events with favorable safety and tolerability. With this robust clinical profile, combined with Sobi's deep commercial market expertise in FCS, TRYNGOLZA has the potential to make a meaningful difference for people living with FCS in the EU." The CHMP opinion is based on positive data from the Phase 3 Balance study, in which TRYNGOLZA demonstrated a statistically significant reduction in triglyceride levels at six months that was sustained through 12 months. Additionally, TRYNGOLZA demonstrated a substantial and clinically meaningful reduction in acute pancreatitis events over 12 months. TRYNGOLZA showed a favorable safety and tolerability profile. Study results were published in The New England Journal of Medicine (NEJM). FCS is a rare and genetic form of severe hypertriglyceridemia (sHTG) that prevents the body from breaking down fats and severely impairs the ability to remove triglycerides from the bloodstream. People with FCS often have triglyceride levels of more than 880 mg/dL (10 mmol/L), compared to normal levels of <150 mg/dL (1.7 mmol/L), and are at high risk of developing acute pancreatitis, which can be life-threatening. In the EU, FCS is estimated to impact up to 13 people per million. Sobi has exclusive rights to commercialize TRYNGOLZA in countries outside the U.S., Canada and China. As Ionis' European commercial partner for Waylivra (volanesorsen), the only medicine currently approved for FCS in the EU, Sobi will leverage existing market expertise and distribution channels to enable an effective TRYNGOLZA launch in FCS, if approved. "The approval recommendation brings us one step closer toward delivering TRYNGOLZA to people living with FCS in the EU and is a testament to our long-standing support for the FCS community," said Lydia Abad-Franch, M.D., MBA, head of research, development and medical affairs and chief medical officer, Sobi. "Patients with FCS suffer from complications such as acute pancreatitis. These are very severe events, often requiring intensive care and sometimes causing multiorgan failure as well as increasing morbidity and mortality. We believe TRYNGOLZA has the potential to be an important treatment for people living with this rare and serious disease, and we look forward to the final decision from the EC later this year." TRYNGOLZA was approved in the United States in December 2024 and granted orphan designation in the EU. Olezarsen is also being evaluated for sHTG, a serious condition defined by dangerously high triglycerides (≥500 mg/dL), and data from the Phase 3 CORE and CORE2 studies are expected in Q3 2025. About the Balance Study Balance is a global, multicenter, randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of olezarsen in patients with FCS at six and 12 months. The primary endpoint was the percent change from baseline in fasting triglyceride levels at six months compared to placebo. Secondary endpoints included percent changes in triglyceride levels at 12 months, percent changes in other lipid parameters and adjudicated acute pancreatitis event rates over the treatment period. Following treatment and the end-of-trial assessments, patients were eligible to enter an open-label extension study to continue receiving olezarsen once every four weeks. About Familial Chylomicronemia Syndrome (FCS) FCS is a rare, genetic disease characterized by extremely elevated triglyceride levels. It is caused by impaired function of the enzyme lipoprotein lipase (LPL). Because of limited LPL production or function, people with FCS cannot effectively break down chylomicrons, lipoprotein particles that are 90% triglycerides. People living with FCS are at high risk of acute pancreatitis in addition to other chronic health issues such as fatigue and severe, recurrent abdominal pain. People living with FCS are sometimes unable to work, adding to the burden of disease. About TRYNGOLZA® (olezarsen) TRYNGOLZA® is an RNA-targeted medicine designed to lower the body's production of apoC-III, a protein produced in the liver that is a key regulator of triglyceride metabolism. TRYNGOLZA® (olezarsen) is approved in the United States as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). For more information about TRYNGOLZA, visit TRYNGOLZA is not yet approved for any indication in Europe. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS TRYNGOLZA is contraindicated in patients with a history of serious hypersensitivity to TRYNGOLZA or any of the excipients in TRYNGOLZA. Hypersensitivity reactions requiring medical treatment have occurred. WARNINGS AND PRECAUTIONS Hypersensitivity Reactions Hypersensitivity reactions (including symptoms of bronchospasm, diffuse erythema, facial swelling, urticaria, chills and myalgias) have been reported in patients treated with TRYNGOLZA. Advise patients on the signs and symptoms of hypersensitivity reactions and instruct patients to promptly seek medical attention and discontinue use of TRYNGOLZA if hypersensitivity reactions occur. ADVERSE REACTIONS The most common adverse reactions (incidence >5% of TRYNGOLZA-treated patients and >3% higher frequency than placebo) were injection site reactions, decreased platelet count and arthralgia. Please see full Prescribing Information for TRYNGOLZA. About Ionis Pharmaceuticals, Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has marketed medicines and a leading pipeline in neurology, cardiology and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit and follow us on X (Twitter), LinkedIn and Instagram. Ionis Forward-looking Statements This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of our commercial medicines, olezarsen, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2024, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company. In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals® and TRYNGOLZA® are trademarks of Ionis Pharmaceuticals, Inc. View source version on Contacts Ionis Investor Contact: D. Wade Walke, 760-603-2331Ionis Media Contact: Hayley Soffermedia@ 760-603-4679 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
8 hours ago
- Business
- Business Wire
TRYNGOLZA® (olezarsen) recommended for approval in the EU by CHMP for familial chylomicronemia syndrome (FCS)
CARLSBAD, Calif.--(BUSINESS WIRE)-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Sobi® today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion of TRYNGOLZA® (olezarsen) as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS). The positive opinion is now referred to the European Commission (EC) for an approval decision, which is expected by Q4 2025. 'Building on the strong early launch of TRYNGOLZA in the U.S., the positive CHMP opinion advances our commitment to expand access to TRYNGOLZA globally,' said Brett P. Monia, Ph.D., chief executive officer, Ionis. 'TRYNGOLZA has demonstrated significant reductions in triglycerides and substantial reductions in acute pancreatitis events with favorable safety and tolerability. With this robust clinical profile, combined with Sobi's deep commercial market expertise in FCS, TRYNGOLZA has the potential to make a meaningful difference for people living with FCS in the EU.' The CHMP opinion is based on positive data from the Phase 3 Balance study, in which TRYNGOLZA demonstrated a statistically significant reduction in triglyceride levels at six months that was sustained through 12 months. Additionally, TRYNGOLZA demonstrated a substantial and clinically meaningful reduction in acute pancreatitis events over 12 months. TRYNGOLZA showed a favorable safety and tolerability profile. Study results were published in The New England Journal of Medicine (NEJM). FCS is a rare and genetic form of severe hypertriglyceridemia (sHTG) that prevents the body from breaking down fats and severely impairs the ability to remove triglycerides from the bloodstream. People with FCS often have triglyceride levels of more than 880 mg/dL (10 mmol/L), compared to normal levels of <150 mg/dL (1.7 mmol/L), and are at high risk of developing acute pancreatitis, which can be life-threatening. In the EU, FCS is estimated to impact up to 13 people per million. Sobi has exclusive rights to commercialize TRYNGOLZA in countries outside the U.S., Canada and China. As Ionis' European commercial partner for Waylivra (volanesorsen), the only medicine currently approved for FCS in the EU, Sobi will leverage existing market expertise and distribution channels to enable an effective TRYNGOLZA launch in FCS, if approved. 'The approval recommendation brings us one step closer toward delivering TRYNGOLZA to people living with FCS in the EU and is a testament to our long-standing support for the FCS community,' said Lydia Abad-Franch, M.D., MBA, head of research, development and medical affairs and chief medical officer, Sobi. 'Patients with FCS suffer from complications such as acute pancreatitis. These are very severe events, often requiring intensive care and sometimes causing multiorgan failure as well as increasing morbidity and mortality. We believe TRYNGOLZA has the potential to be an important treatment for people living with this rare and serious disease, and we look forward to the final decision from the EC later this year.' TRYNGOLZA was approved in the United States in December 2024 and granted orphan designation in the EU. Olezarsen is also being evaluated for sHTG, a serious condition defined by dangerously high triglycerides (≥500 mg/dL), and data from the Phase 3 CORE and CORE2 studies are expected in Q3 2025. About the Balance Study Balance is a global, multicenter, randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of olezarsen in patients with FCS at six and 12 months. The primary endpoint was the percent change from baseline in fasting triglyceride levels at six months compared to placebo. Secondary endpoints included percent changes in triglyceride levels at 12 months, percent changes in other lipid parameters and adjudicated acute pancreatitis event rates over the treatment period. Following treatment and the end-of-trial assessments, patients were eligible to enter an open-label extension study to continue receiving olezarsen once every four weeks. About Familial Chylomicronemia Syndrome (FCS) FCS is a rare, genetic disease characterized by extremely elevated triglyceride levels. It is caused by impaired function of the enzyme lipoprotein lipase (LPL). Because of limited LPL production or function, people with FCS cannot effectively break down chylomicrons, lipoprotein particles that are 90% triglycerides. People living with FCS are at high risk of acute pancreatitis in addition to other chronic health issues such as fatigue and severe, recurrent abdominal pain. People living with FCS are sometimes unable to work, adding to the burden of disease. About TRYNGOLZA® (olezarsen) TRYNGOLZA® is an RNA-targeted medicine designed to lower the body's production of apoC-III, a protein produced in the liver that is a key regulator of triglyceride metabolism. TRYNGOLZA ® (olezarsen) is approved in the United States as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). For more information about TRYNGOLZA, visit TRYNGOLZA is not yet approved for any indication in Europe. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS TRYNGOLZA is contraindicated in patients with a history of serious hypersensitivity to TRYNGOLZA or any of the excipients in TRYNGOLZA. Hypersensitivity reactions requiring medical treatment have occurred. WARNINGS AND PRECAUTIONS Hypersensitivity Reactions Hypersensitivity reactions (including symptoms of bronchospasm, diffuse erythema, facial swelling, urticaria, chills and myalgias) have been reported in patients treated with TRYNGOLZA. Advise patients on the signs and symptoms of hypersensitivity reactions and instruct patients to promptly seek medical attention and discontinue use of TRYNGOLZA if hypersensitivity reactions occur. ADVERSE REACTIONS The most common adverse reactions (incidence >5% of TRYNGOLZA-treated patients and >3% higher frequency than placebo) were injection site reactions, decreased platelet count and arthralgia. Please see full Prescribing Information for TRYNGOLZA. About Ionis Pharmaceuticals, Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has marketed medicines and a leading pipeline in neurology, cardiology and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit and follow us on X (Twitter), LinkedIn and Instagram. Ionis Forward-looking Statements This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of our commercial medicines, olezarsen, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2024, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company. In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals ® and TRYNGOLZA ® are trademarks of Ionis Pharmaceuticals, Inc.
Yahoo
10-07-2025
- Business
- Yahoo
Apellis Sells Aspaveli Ex-US Royalties to Sobi for Up to $300M
Apellis Pharmaceuticals Inc. (NASDAQ:APLS) is one of the most promising stocks according to Wall Street analysts. On July 1, Apellis Pharmaceuticals announced a capped royalty purchase agreement with Sobi. Under this agreement, Apellis will receive up to $300 million in exchange for 90% of its future ex-US royalties for Aspaveli (systemic pegcetacoplan). This non-dilutive financing includes an upfront payment of $275 million in cash and up to $25 million in milestone payments contingent upon European Medicines Agency/EMA approval of Aspaveli for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). Apellis will retain exclusive commercialization rights for systemic pegcetacoplan in the US, where it is marketed as EMPAVELI. A biomedical scientist in a lab coat conducting research on biopharmaceutical compounds. The transaction reinforces the shared confidence between Apellis and Sobi in the growth potential of Aspaveli/EMPAVELI in rare kidney diseases. Aspaveli/EMPAVELI is currently approved in the European Union, other global countries, and the US for the treatment of paroxysmal nocturnal hemoglobinuria/PNH, which is a rare blood disorder. Apellis Pharmaceuticals Inc. (NASDAQ:APLS) is a commercial-stage biopharmaceutical company that discovers, develops, and commercializes novel therapeutic compounds for diseases with unmet needs. While we acknowledge the potential of APLS as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the . READ NEXT: and . Disclosure: None. This article is originally published at Insider Monkey.
Yahoo
06-07-2025
- Business
- Yahoo
Morgan Stanley Raises PT on Apellis Pharmaceuticals (APLS) to $26, Keeps a Hold Rating
Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) is one of the 13 Best Pharma Stocks to Buy According to Wall Street Analysts. On July 1, analyst Judah Frommer from Morgan Stanley raised the firm's price target on Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) to $26.00 from $25.00 while maintaining a Hold rating on the stock. Frommer attributed the rating to the company's market position and recent financial strategies. A biomedical scientist in a lab coat conducting research on biopharmaceutical compounds. He cited the company's significant royalty purchase agreement with Sobi, securing up to $300 million and including a $275 million upfront payment, stating that it would slash Sobi's royalty obligations on Aspaveli sales by 90%. This would result in immediate financial benefits for Apellis and may even ease investor concerns regarding the company's profitability. Despite this positive development, Frommer maintained a cautious outlook for Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) due to its future prospects. He stated that while the anticipated regulatory decisions from the FDA and CHMP and the launches of Empaveli/Aspaveli in new indications are significant events, they bring in as much uncertainty as confidence in the company's market potential. Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) is a commercial-stage biopharmaceutical company that discovers, develops, and commercializes novel therapeutic compounds for treating diseases with unmet needs. Its product portfolio primarily includes EMPAVELI and SYFOVRE. SYFOVRE treats geographic atrophy secondary to age-related macular degeneration (GA), while EMPAVELI treats paroxysmal nocturnal hemoglobinuria (PNH). While we acknowledge the potential of APLS as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: The Best and Worst Dow Stocks for the Next 12 Months and 10 Unstoppable Stocks That Could Double Your Money. Disclosure: None.
Yahoo
01-07-2025
- Business
- Yahoo
Apellis to Receive up to $300 Million from Royalty Purchase Agreement with Sobi for Ex-U.S. Royalties of Aspaveli® (systemic pegcetacoplan)
Apellis to receive $275 million upfront and up to $25 million in milestone payments upon EMA approval in C3G and IC-MPGN Non-dilutive financing further strengthens Apellis' balance sheet Apellis retains full U.S. commercialization rights for systemic pegcetacoplan Transaction underscores shared confidence in the meaningful growth potential in rare kidney diseases WALTHAM, Mass., July 01, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced a capped royalty purchase agreement with Sobi® under which Apellis will receive up to $300 million in exchange for 90% of Apellis' future ex-U.S. royalties for Aspaveli® (systemic pegcetacoplan). Under the companies' 2020 collaboration agreement, Apellis is eligible for tiered royalties on ex-U.S. sales of Aspaveli ranging from high teens to high twenties. Apellis retains exclusive commercialization rights for systemic pegcetacoplan in the United States, where the product is marketed as EMPAVELI®. 'This transaction reflects our shared conviction in the potential of Aspaveli/EMPAVELI to transform the treatment landscape for patients with rare diseases, including C3G and IC-MPGN,' said Timothy Sullivan, chief financial officer, Apellis. 'Through our collaboration, Sobi has developed a deep understanding of Aspaveli/EMPAVELI's potential to significantly improve patient outcomes and deliver long-term value as a rare disease franchise. Importantly, the non-dilutive funding from this transaction further strengthens our balance sheet and provides significant operational flexibility as we approach sustainable profitability, expand the reach of our approved products, and advance our innovative pipeline.' Aspaveli/EMPAVELI is approved in the European Union, other countries globally, and the U.S. for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. It is currently under review in the European Union and the U.S. for the treatment of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), rare kidney diseases. An opinion by the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) is expected before year-end. In the U.S., the Prescription Drug User Fee Act (PDUFA) action date is July 28, 2025. 'We are pleased to reaffirm our ongoing partnership with Apellis and share their strong belief in Aspaveli/EMPAVELI's potential to deliver significant long-term growth,' said Guido Oelkers, chief executive officer, Sobi. 'We are confident in our ability to reach patients with C3G and IC-MPGN globally following regulatory approvals, by leveraging our broad commercial footprint, deep rare disease expertise, and proven success in PNH.' Transaction details Under the terms of the agreement, Sobi will acquire 90% of Apellis' ex-U.S. royalties for Aspaveli for $275 million in cash. Apellis will also be eligible for up to $25 million in milestone payments upon EMA approval of Aspaveli for C3G and IC-MPGN. The agreement is subject to defined caps tied to Aspaveli's performance. Sobi retains 90% of ex-U.S. royalties until these caps are achieved, after which 100% of all ex-U.S. royalties revert to Apellis. About the Apellis and Sobi CollaborationApellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan, and its opt-in rights for future development programs are unchanged, exercisable at any time prior to commercialization. Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. About C3 Glomerulopathy (C3G) and Primary Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)C3G and primary IC-MPGN are rare and debilitating kidney diseases that can lead to kidney failure. Excessive C3 deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. Approximately 50% of people living with C3G and primary IC-MPGN suffer from kidney failure within five to 10 years of diagnosis, requiring a burdensome kidney transplant or lifelong dialysis.1 Additionally, approximately 90% of patients who previously received a kidney transplant will experience disease recurrence.2 The diseases are estimated to affect 5,000 people in the United States and up to 8,000 in Europe.3 About ApellisApellis Pharmaceuticals, Inc. is a global biopharmaceutical company that combines courageous science and compassion to develop life-changing therapies for some of the most challenging diseases patients face. We ushered in the first new class of complement medicine in 15 years and now have two approved medicines targeting C3. These include the first-ever therapy for geographic atrophy, a leading cause of blindness around the world. We believe we have only begun to unlock the potential of targeting C3 across many serious diseases. For more information, please visit or follow us on X and LinkedIn. Apellis Forward-Looking Statement Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute 'forward-looking statements' within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements regarding regulatory approval for the treatment of patients with C3G and IC-MPGN in the United States and other jurisdictions and commercial growth or prospects for systemic pegcetacoplan for those indications in the United States and other jurisdictions. The words 'anticipate,' 'believe,' 'continue,' 'could,' 'estimate,' 'expect,' 'intend,' 'may,' 'plan,' 'potential,' 'predict,' 'project,' 'should,' 'target,' 'will,' 'would' and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including whether systemic pegcetacoplan will receive approval for those indications from the FDA or equivalent foreign regulatory agencies when expected or at all; whether the commercialization of systemic pegcetacoplan for C3G or IC-MPGN in the United States or other jurisdictions will provide significant additional revenues or allow the Company to achieve profitability; and any other factors discussed in the 'Risk Factors' section of Apellis' Annual Report on Form 10-K with the Securities and Exchange Commission on February 28, 2025 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. Contacts: MediaTracy Vineismedia@ InvestorsNeil Carnahan, CFA References1. C3 glomerulopathy. National Institute of Health, Genetics Home Reference. Accessed November 21, 2019. 2. Tarragón, B, et al. C3 Glomerulopathy Recurs Early after Kidney Transplantation in Serial Biopsies Performed within the First 2 Years after Transplantation. Clinical Journal of the American Society of Nephrology. August 2024; 19(8)1005-1015. doi: 10.2215/CJN.0000000000000474.3. Data on file using literature 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
