Meningitis, Meningococcal

Statistically significant 68% (p<0.0001) proteinuria reduction compared to placebo, with reduction observed as early as Week 4 All secondary endpoints favoured treatment with pegcetacoplan, including: - Stabilised eGFR, a key measure of kidney function - 71% of patients achieved zero C3c staining intensity, indicating clearance of C3c deposits Results consistent across subgroups including C3G and primary IC-MPGN, adolescent and adult patients, and native and post-transplant kidneys STOCKHOLM, Oct. 27, 2024 /PRNewswire/ -- Sobi® (STO: SOBI) and Apellis Pharmaceuticals, Inc. today announced that detailed data from the phase 3 VALIANT study were presented as an oral presentation during the High-Impact Clinical Trials session at the 2024 American Society of Nephrology (ASN) Kidney Week. The results highlighted the potential of systemic pegcetacoplan treatment in patients with C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), which are rare, debilitating kidney diseases. "Pegcetacoplan is the only treatment to achieve substantial and clinically meaningful effects across all key markers of disease: proteinuria, eGFR stabilisation, and C3c staining," said Carla Nester, M.D. MSA, FASN, lead principal investigator for the VALIANT study, professor of internal medicine and paediatrics, and director of paediatric nephrology, University of Iowa Stead Family Children's Hospital. "C3G and primary IC-MPGN often affect patients as early as adolescence, often leading to either a kidney transplant or lifelong dialysis, so there is an urgent need for an approved treatment that can prolong kidney function." Statistically significant 68% proteinuria reduction across a broad study population, with reduction observed as early as Week 4 Pegcetacoplan-treated patients showed a statistically significant and clinically meaningful 68.1% (p<0.0001) proteinuria reduction (log-transformed ratio of urine protein-to-creatinine ratio) compared to placebo, both in addition to standard of care therapy, at Week 26. The proteinuria reduction was observed as early as Week 4 and continued through the six-month treatment period. Proteinuria reduction was consistent across broad patient subgroups including adolescent and adult patients, C3G and primary IC-MPGN patients, and patients with native and post-transplant kidneys. Pegcetacoplan stabilised eGFR and demonstrated substantial reduction in C3c staining Patients treated with pegcetacoplan achieved stabilisation of estimated glomerular filtration rate (eGFR), a key measure of kidney function, with a difference of +6.3 mL/min/1.73 m2 (95% CI 0.5, 12.1; nominal p value =0.03) over six months compared to placebo. Additionally, a substantial proportion of patients treated with pegcetacoplan also demonstrated a reduction in C3c staining intensity. Excessive C3c deposits are a key marker of disease activity which can lead to kidney inflammation, damage, and failure. 74.3% of patients in the pegcetacoplan group and 11.8% on placebo achieved a reduction in C3c staining intensity by two or more orders of magnitude from baseline, resulting in 27-fold higher odds ratio of achieving this reduction with pegcetacoplan (nominal p value <0.0001). 71.4% of pegcetacoplan-treated patients achieved zero C3c staining intensity, indicating clearance of C3c deposits. "C3G and primary IC-MPGN can have a severe impact on patients' lives, frequently resulting in kidney failure. The findings presented at ASN further highlight the potential of pegcetacoplan to preserve kidney function and address these unmet medical needs. We extend our heartfelt gratitude to the participants, their families, and the dedicated healthcare professionals who made this study possible," stated Lydia Abad-Franch, MD, Head of R&D, Medical Affairs, and Chief Medical Officer at Sobi. All secondary endpoints favoured treatment with pegcetacoplan In addition to the positive results on proteinuria, eGFR and C3c staining, pegcetacoplan demonstrated statistical significance on the key secondary endpoints of composite renal endpoint, which combines proteinuria reduction and eGFR stabilisation, and proteinuria reduction of at least 50% compared to baseline, as well as a numerical improvement in the C3G histologic index activity score. During the randomised, controlled 26-week treatment period, pegcetacoplan demonstrated favourable safety and tolerability, as well as a high compliance rate, consistent with its established profile. Rates of treatment-emergent adverse events (AEs) (84.1% in pegcetacoplan vs. 93.4% in placebo), serious AEs (9.5% vs. 9.8%), severe AEs (4.8 % vs. 6.6%), and AEs leading to study discontinuation (1.6% vs. 1.6%) were similar between the pegcetacoplan and placebo groups. There were no cases of meningococcal meningitis or serious infections attributed to encapsulated bacteria. Patients who complete the VALIANT study are eligible to enrol into the VALE long-term extension study. Sobi plans to submit a marketing application with the European Medicines Agency in 2025. Apellis plans to submit a supplemental new drug application to the U.S. Food and Drug Administration in early 2025. About the VALIANT study The VALIANT phase 3 study (NCT05067127) is a randomised, placebo-controlled, double-blinded, multi-centre study designed to evaluate pegcetacoplan efficacy and safety in 124 patients who are 12 years of age and older with C3G or primary IC-MPGN. It is the largest single trial conducted in these populations and the only study to include adolescent and adult patients, with native and post-transplant kidneys. Study participants were randomised to receive pegcetacoplan or placebo twice weekly for 26 weeks. Following this 26-week randomised controlled period, patients are able to proceed to a 26-week open-label phase in which all patients receive pegcetacoplan. The primary endpoint of the study was the log transformed ratio of urine protein-to-creatinine ratio (uPCR) at Week 26 compared to baseline. 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 C3c deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. There are no treatments that target the underlying cause of these diseases. Approximately 50% of people living with C3G and IC-MPGN suffer from kidney failure within five to 10 years of diagnosis, requiring a burdensome kidney transplant or lifelong dialysis.1 Additionally, 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 pegcetacoplan in rare diseases Pegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is under investigation for rare diseases across haematology and nephrology. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) as EMPAVELI®/Aspaveli® in the United States, European Union, and other countries globally. About the Sobi and Apellis collaboration Sobi and Apellis have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialisation rights for systemic pegcetacoplan, and Apellis has exclusive U.S. commercialisation rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. About Sobi® Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia, and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Statistically significant 68% (p<0.0001) proteinuria reduction compared to placebo, with reduction observed as early as Week 4 All secondary endpoints favored treatment with pegcetacoplan, including: Stabilized eGFR, a key measure of kidney function 71% of patients achieved zero C3c staining intensity, demonstrating complete clearance of C3c deposits Results consistent across subgroups including C3G and IC-MPGN, adolescent and adult patients, and native and post-transplant kidneysDemonstrated favorable safety profile WALTHAM, Mass., Oct. 26, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Sobi® today announced that positive results from the Phase 3 VALIANT study were presented as an oral presentation during the High-Impact Clinical Trials session at the American Society of Nephrology (ASN) Kidney Week. The results highlighted the strength of systemic pegcetacoplan treatment in patients with C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), which are rare, debilitating kidney diseases. “These unprecedented results represent a potential breakthrough for patients living with C3G and IC-MPGN. Pegcetacoplan is the only treatment to achieve substantial and clinically meaningful effects across all key markers of disease: proteinuria, eGFR stabilization, and C3c staining,” said Carla Nester, M.D., MSA, FASN, lead principal investigator for the VALIANT study, professor of internal medicine and pediatrics, and director of pediatric nephrology, University of Iowa Stead Family Children's Hospital. “C3G and IC-MPGN affect patients as early as adolescence, often leading to either a kidney transplant or lifelong dialysis, so there is an urgent need for an approved treatment that can prolong kidney function.” Statistically significant 68% proteinuria reduction across a broad study population, with reduction observed as early as Week 4Pegcetacoplan-treated patients showed a statistically significant and clinically meaningful 68.1% (p<0.0001) proteinuria reduction (log-transformed ratio of urine protein-to-creatinine ratio) compared to placebo, both in addition to standard of care therapy, at Week 26. The proteinuria reduction was observed as early as Week 4 and continued through the six-month treatment period. Proteinuria reduction was consistent across broad patient subgroups including adolescent and adult patients, C3G and IC-MPGN patients, and patients with native and post-transplant kidneys. Pegcetacoplan stabilized eGFR and demonstrated substantial reduction in C3c staining Patients treated with pegcetacoplan achieved stabilization of estimated glomerular filtration rate (eGFR), a key measure of kidney function, with a difference of +6.3mL/min/1.73m2 (nominal p value=0.03) over six months compared to placebo. Additionally, a substantial proportion of patients treated with pegcetacoplan demonstrated a reduction in C3c staining intensity. Excessive C3c deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. 74.3% of patients in the pegcetacoplan group and 11.8% on placebo achieved a reduction in C3c staining intensity by two or more orders of magnitude from baseline, resulting in 27-fold higher odds of achieving this reduction with pegcetacoplan (nominal p value <0.0001).71.4% of pegcetacoplan-treated patients achieved zero C3c staining intensity, demonstrating complete clearance of C3c deposits. “In the largest pivotal study in C3G and IC-MPGN, pegcetacoplan rapidly, significantly, and consistently improved key outcomes for patients with C3G and IC-MPGN,” said Peter Hillmen, M.B., Ch.B., Ph.D., chief medical advisor, rare, Apellis. “We are thrilled by these results, which underscore the potential for pegcetacoplan to significantly improve patients’ lives by directly targeting C3, the underlying cause of C3G and IC-MPGN.” All secondary endpoints favored treatment with pegcetacoplanIn addition to the positive results on proteinuria, eGFR, and C3c staining, pegcetacoplan demonstrated statistical significance on the key secondary endpoints of composite renal endpoint, which combines proteinuria reduction and eGFR stabilization, and proteinuria reduction of at least 50% compared to baseline, as well as a numerical improvement in the C3G histologic index activity score. During the randomized, controlled 26-week treatment period, pegcetacoplan demonstrated favorable safety and tolerability, as well as a high compliance rate, consistent with its established profile. Rates of treatment-emergent adverse events (AEs) (84.1% in pegcetacoplan vs. 93.4% in placebo), serious AEs (9.5% vs. 9.8%), severe AEs (4.8 % vs. 6.6%), and AEs leading to study discontinuation (1.6% vs. 1.6%) were similar between the pegcetacoplan and placebo groups. There were no cases of meningococcal meningitis or serious infections attributed to encapsulated bacteria. All patients who have already completed the VALIANT study have now enrolled into the VALE long-term extension study. Apellis plans to submit a supplemental new drug application to the U.S. Food and Drug Administration in early 2025. Sobi plans to submit a marketing application with the European Medicines Agency in 2025. About the VALIANT StudyThe VALIANT Phase 3 study (NCT05067127) is a randomized, placebo-controlled, double-blinded, multi-center study designed to evaluate pegcetacoplan efficacy and safety in 124 patients who are 12 years of age and older with C3G or primary IC-MPGN. It is the largest single trial conducted in these populations and the only study to include adolescent and adult patients, with native and post-transplant kidneys. Study participants were randomized to receive pegcetacoplan or placebo twice weekly for 26 weeks. Following this 26-week randomized controlled period, patients are able to proceed to a 26-week open-label phase in which all patients receive pegcetacoplan. The primary endpoint of the study was the log transformed ratio of urine protein-to-creatinine ratio (uPCR) at Week 26 compared to baseline. 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 C3c deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. There are no treatments that target the underlying cause of these diseases. Approximately 50% of people living with C3G and IC-MPGN suffer from kidney failure within five to 10 years of diagnosis, requiring a burdensome kidney transplant or lifelong dialysis.1 Additionally, 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 Pegcetacoplan in Rare DiseasesPegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is under investigation for rare diseases across hematology and nephrology. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) as EMPAVELI®/Aspaveli® in the United States, European Union, and other countries globally. 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 Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. 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 serious retinal, rare, and neurological diseases. For more information, please visit http://apellis.com or follow us on X (Twitter) 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 plans to submit applications for regulatory approval for the treatment of patients with C3G and IC-MPGN. 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; 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 27, 2024 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.Media:Tracy Vineismedia@apellis.com 617-420-4839 Investors: Meredith Kaya meredith.kaya@apellis.com617.599.8178 References 1. C3 glomerulopathy. National Institute of Health, Genetics Home Reference. https://ghr.nlm.nih.gov/condition/c3-glomerulopathy#resources. 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 consensus.

Mothers affected by meningitis urge national action to improve meningococcal meningitis vaccination rates in the United States FARMINGTON HILLS, Mich., Oct. 4, 2024 /PRNewswire/ -- In recognition of World Meningitis Day on October 5, the American Society for Meningitis Prevention (ASMP) is lighting up 55 landmarks and homes in 37 states across the country to raise awareness about meningococcal meningitis, an unpredictable and life-threatening bacterial infection. This national illumination campaign aims to encourage parents and caregivers to ensure their children are fully vaccinated against the deadly disease. Meningococcal meningitis, one of the most common types of bacterial meningitis, affects all ages, but is more common among young adults and adolescents. In 2023, the United States saw the highest number of meningococcal meningitis cases since 2014, prompting a nationwide alert from the Centers for Disease Control and Prevention (CDC).i To help protect against meningococcal meningitis, patients must be vaccinated against all five types of meningococcal bacteria most likely to cause meningococcal meningitis: A–B–C–W–Y.ii ASMP co-founder Patti Wukovits, BSN, RN, AMB-BC, emphasizes the critical role of vaccination in protecting against the disease. "As a nurse and a mother who lost her 17-year-old daughter to meningococcal meningitis, it's important to know that there is not only one meningitis vaccine. I encourage all parents to be proactive and ask their healthcare provider if their child is fully vaccinated against meningococcal meningitis," says Wukovits. Meningococcal meningitis is spread through close personal contact, such as coughing, kissing, or sharing utensils. The disease moves quickly and can kill in a matter of hours or cause permanent complications. "Meningitis is unpredictable —that's why it's so important to get protected. I lost my 19-year-old daughter 36 hours after her symptoms started," said Alicia Stillman, MBA, MPH, co-founder of ASMP. "Collaboration is key to preventing this devastating disease. It's critical that we work together—families, healthcare providers, policy makers and communities—to protect our loved ones. My daughter's life, and Patti's daughter's life, were tragically cut short by this disease, and we're determined to prevent others from experiencing the same heartbreak." As landmarks and homes across the country light up to promote meningitis awareness, the American Society for Meningitis Prevention urges everyone to ask their healthcare provider if they have received all the meningitis vaccines they need to be as protected as possible. ASMP's lighting initiative is part of a larger global effort to defeat meningitis by the Confederation of Meningitis Organisations (CoMO) and the Meningitis Research Foundation (MRF). For more information on how to protect against meningococcal meningitis, visit . For a list of all lighting locations in the United States, visit . About the American Society for Meningitis Prevention (ASMP) The American Society for Meningitis Prevention (ASMP) is a national 501(c)(3) organization dedicated to empowering all people to prevent meningococcal meningitis and achieve a world without this terrible disease. ASMP was born out of the vision and resilience of two mothers, Patti Wukovits and Alicia Stillman, who each lost their young, healthy daughters to meningococcal meningitis. In 2012, high school senior Kimberly, 17, Patti's daughter, died one week before her graduation. In 2013, college sophomore Emily, 19, Alicia's daughter, died just 36 hours after her first symptoms. In their memories, Patti and Alicia founded the Kimberly Coffey Foundation and the Emily Stillman Foundation. In addition to their Foundations, both mothers have joined forces to establish ASMP and ensure no other families needlessly suffer the same fate. Learn more at meningitisprevention.org. CONTACT: Mariana Rodrigues Communications Director [email protected] i Cases of Meningococcal Disease Are Increasing in the United States. Centers for Disease Control and Prevention. . ii Prevention. Centers for Disease Control and Prevention. SOURCE American Society for Meningitis Prevention WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED