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Globe and Mail
2 days ago
- Business
- Globe and Mail
IC-MPGN Therapeutics Market Expected to Grow With Complement Inhibitor Therapies: First FDA Approvals, Clinical Pipeline
"Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN) Market Insights, Epidemiology, and Market Forecast – 2034" The IC-MPGN market, valued at USD 12M in the US in 2023, is set to grow significantly by 2034, with ~7K cases across the 7MM. Novartis Pharmaceuticals and Apellis Pharmaceuticals lead innovation with iptacopan (LNP023) and pegcetacoplan (APL-2/EMPAVELI). In Aug 2025, the FDA approved Apellis' EMPAVELI as the first IC-MPGN therapy, marking a major milestone in treating this ultra-rare kidney disease. DelveInsight's ' Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN) Market Insights, Epidemiology, and Market Forecast – 2034 ' report delivers a comprehensive analysis of the IC-MPGN market, covering historical and forecasted patient pools, current treatment practices, emerging drugs, and market size trends across the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan. Key Takeaways Market size projection: As per DelveInsight's analysis, the total market size of IC-MPGN in the 7MM is expected to surge significantly by 2034, with the United States market alone growing significantly from approximately USD 12 million in 2023. Patient population data: The report provides the total IC-MPGN potential pool of more than 7K diagnosed prevalent cases across the 7MM in 2023, with the United States representing about 55% of these cases at approximately 3,770 diagnosed patients. Key companies: Leading IC-MPGN companies, such as Novartis Pharmaceuticals, Apellis Pharmaceuticals, and others, are driving innovation in this ultra-rare kidney disease therapeutic area. Pipeline assets: Some of the key IC-MPGN pipeline therapies include Iptacopan (LNP023) from Novartis, Pegcetacoplan (APL-2) from Apellis Pharmaceuticals, and others targeting complement pathway inhibition. In August 2025, the FDA approved pegcetacoplan (EMPAVELI) by Apellis Pharmaceuticals as the first treatment specifically for patients 12 years and older with primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). The approval was based on the Phase 3 VALIANT trial (NCT05067127), which demonstrated significant clinical benefits. Furthermore, Iptacopan (LNP023) is currently being evaluated in a multicenter, randomized, double-blind, placebo-controlled Phase 3 study specifically for idiopathic IC-MPGN. Discover recent advancements in the IC-MPGN treatment landscape @ IC-MPGN Recent Developments. IC-MPGN Market Dynamics The IC-MPGN market represents an ultra-rare therapeutic area with significant unmet medical needs and substantial growth potential. Currently valued at approximately USD 12 million in the United States in 2023, the market is characterized by the lack of approved medications specifically indicated for IC-MPGN, creating a substantial demand for treatments that address the root cause of this progressive kidney disease. IC-MPGN market drivers include increasing awareness of complement-mediated diseases, advances in understanding the pathophysiology of IC-MPGN, and the development of targeted complement inhibitors. The FDA approval of pegcetacoplan (EMPAVELI) by Apellis Pharmaceuticals, the first drug specifically approved for IC-MPGN heightens the optimisim in this landscape, further propelling the market. The disease is characterized by immune complex deposition in the glomeruli, triggering inflammation and damage that can lead to kidney failure within 5-10 years of diagnosis. Current treatment strategies involve supportive measures with or without traditional immunosuppression, showing limited effectiveness in slowing disease progression. IC-MPGN epidemiology includes moe than 7K diagnosed prevalent cases across the 7MM, with adults representing about 90% of all cases, typically resulting from chronic infections, autoimmune disorders, or underlying conditions such as hepatitis C, systemic lupus erythematosus (SLE), or monoclonal gammopathy. Clinical development activity is intensifying, with key assets advancing through Phase III trials. The IC-MPGN emerging therapies centers on targeted complement inhibition, addressing the underlying pathophysiology of IC-MPGN. The IC-MPGN competitive landscape features established pharmaceutical companies leveraging their expertise in nephrology and complement biology. Currently the market currently relies heavily on off-label prescription medications, creating opportunities for companies developing targeted therapies. However, challenges exist in developing effective complement inhibitors due to the complexity of the complement system and its adaptive capabilities. Furthermore, the IC-MPGN market outlook remains positive, driven by the anticipated launch of targeted therapies, increasing diagnosis rates, and growing recognition of complement-mediated kidney diseases. The market is expected to experience significant growth through 2034, supported by the entry of novel therapeutics that address the underlying disease mechanisms rather than merely providing symptomatic relief. Download the IC-MPGN Market report to understand which factors are driving the therapeutic market @ IC-MPGN Market Trends. IC-MPGN Epidemiology The IC-MPGN epidemiology across the 7MM reveals a rare but significant patient population requiring specialized therapeutic intervention. In 2023, more 7K diagnosed prevalent cases were identified across the seven major markets, representing a substantial patient population affected by this ultra-rare kidney disease. The United States dominates the global patient population, accounting for approximately 55% of all cases with around 3,770 diagnosed patients, followed by Japan as the second-largest market. The IC-MPGN epidemiology geographic distribution shows notable variations across the 7MM regions. Within the EU4 countries and the United Kingdom, Germany represents the largest patient population, followed by Spain, while Italy demonstrates the lowest number of cases. This distribution pattern reflects both demographic factors and potentially differential diagnostic capabilities across these regions. Japan shows a distinct age distribution pattern, with adults being significantly more prevalent compared to the pediatric population, consistent with the global trend of adult predominance in IC-MPGN cases. The IC-MPGN epidemiology age-specific segmentation reveals that approximately 90% of all IC-MPGN cases occur in adults, primarily due to underlying conditions that develop over time, including chronic infections, autoimmune disorders, hepatitis C, systemic lupus erythematosus, and monoclonal gammopathy. These conditions lead to persistent immune complex formation, triggering complement system activation and resulting in progressive kidney damage. The pediatric population represents a smaller but clinically significant segment requiring specialized care approaches. Additionally, DelveInsight's gender-specific analysis indicates a relatively balanced distribution between male and female patients, though specific regional variations exist across the 7MM. The total treated cases represent a subset of the diagnosed prevalent population, reflecting current treatment limitations and the challenge of managing this progressive disease with existing therapeutic options. The forecast period from 2024 to 2034 anticipates gradual growth in diagnosed cases, driven by improved awareness, enhanced diagnostic capabilities, and better recognition of IC-MPGN as a distinct clinical entity separate from other forms of membranoproliferative glomerulonephritis. Discover evolving trends in the IC-MPGN patient pool forecasts @ IC-MPGN Epidemiology Analysis. Key IC-MPGN Companies and Treatment Market The clinical and regulatory IC-MPGN landscape is evolving rapidly, with current treatment options limited to supportive care and off-label immunosuppressive therapies. Traditional approaches include renin-angiotensin-aldosterone system inhibitors, immunosuppressants such as corticosteroids, calcineurin inhibitors, and mycophenolate mofetil-based treatments, though these show limited effectiveness in slowing disease progression. The limited IC-MPGN approved therapies has created a significant treatment gap that emerging complement inhibitors aim to address. The IC-MPGN clinical pipeline activity is concentrated around complement pathway modulation, with two leading companies advancing promising therapeutics through late-stage development. Key players include Novartis Pharmaceuticals (stock symbol: NVS) with iptacopan (LNP023) and Apellis Pharmaceuticals (stock symbol: APLS) with pegcetacoplan (APL-2), both targeting different aspects of the complement cascade to address the underlying pathophysiology of IC-MPGN. Market positioning of these pipeline assets represents a paradigm shift from symptomatic management to targeted disease modification. Iptacopan functions as an investigational, first-in-class, orally administered factor B inhibitor of the alternative complement pathway, discovered at the Novartis Institutes for BioMedical Research. Currently in Phase III clinical development, the drug has the potential to become the first targeted therapy to delay progression to dialysis in complement-related kidney diseases. Pegcetacoplan operates as a targeted C3 inhibitor designed to regulate excessive complement activation, utilizing a unique 15-amino acid cyclic peptide conjugated to polyethylene glycol that binds to C3 and C3b, directly preventing activation of C3, C5, and the alternative pathway. Commercial arrangements include strategic collaborations, with Apellis partnering with Sobi for the VALIANT study results announcement. Both companies have established comprehensive development programs spanning multiple complement-related diseases, positioning their assets for potential label expansions beyond IC-MPGN. The regulatory pathway for these novel therapies benefits from the rare disease designation and the significant unmet medical need, potentially facilitating accelerated development timelines and priority review processes. Discover which companies are innovating in the IC-MPGN treatment landscape @ IC-MPGN Competitive Landscape. Conclusion DelveInsight's comprehensive analysis of the IC-MPGN market reveals a therapeutic area poised for transformation through targeted complement inhibition. With approximately 7,100 patients across the 7MM currently lacking effective treatment options, the anticipated market growth driven by iptacopan and pegcetacoplan represents a significant advancement for this ultra-rare kidney disease. The convergence of improved understanding of complement-mediated pathophysiology, positive late-stage clinical results, and the substantial unmet medical need positions the IC-MPGN market for meaningful expansion through 2034. As these innovative therapies progress toward regulatory approval and commercial launch, they offer the potential to fundamentally change the treatment paradigm and improve outcomes for patients suffering from this progressive and debilitating condition. Table of Contents 1. Key Insights 2. Executive Summary of IC-MPGN 3. Competitive Intelligence Analysis for IC-MPGN 4. IC-MPGN Market Overview at a Glance 5. IC-MPGN: Disease Background and Overview 6. IC-MPGN Patient Journey 7. IC-MPGN Epidemiology and Patient Population 8. Treatment Algorithm, Current Treatment, and Medical Practices 9. IC-MPGN Unmet Needs 10. Key Endpoints of IC-MPGN Treatment 11. IC-MPGN Marketed Products 12. IC-MPGN Emerging Therapies 13. IC-MPGN: Seven Major Market Analysis 14. Attribute analysis 15. 7MM: Market Outlook 16. Access and Reimbursement Overview of IC-MPGN 17. KOL Views 18. IC-MPGN Market Drivers 19. IC-MPGN Market Barriers 20. Appendix 21. DelveInsight Capabilities 22. Disclaimer 23. About DelveInsight About DelveInsight DelveInsight is a leading market research and consulting firm specializing in disease-specific insights and therapeutic market analysis. Their reports integrate real-world data, clinical trial findings, and expert interviews to deliver comprehensive industry intelligence. Media Contact Company Name: DelveInsight Business Research LLP Contact Person: Arpit Anand Email: Send Email Phone: +14699457679 Address: 304 S. Jones Blvd #2432 City: Las Vegas State: Nevada Country: United States Website:
Yahoo
29-07-2025
- Health
- Yahoo
FDA Approves Apellis' EMPAVELI® (pegcetacoplan) as the First C3G and Primary IC-MPGN Treatment for Patients 12 and Older
Proven efficacy across all three key markers of disease—68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits Broad label includes adults and adolescents with C3G or primary IC-MPGN, and post-transplant C3G disease recurrence Well-established safety profile, consistent across >2,200 patient years in approved indications C3G and primary IC-MPGN are rare kidney diseases with high risk of kidney failure Conference call tomorrow at 8:00 a.m. ETWALTHAM, Mass., July 28, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the U.S. Food and Drug Administration (FDA) has approved EMPAVELI® (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older, to reduce proteinuria. C3G and primary IC-MPGN are rare kidney diseases, affecting 5,000 people in the United States.1 'I'm excited to now have a highly effective therapy for a broad range of patients living with C3G and primary IC-MPGN,' 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. 'With standard of care, patients living with these rare and severe diseases frequently progress to kidney failure, necessitating lifelong dialysis and/or a kidney transplant. Given the urgent need, particularly in children, the approval of EMPAVELI marks a pivotal moment in the treatment of rare kidney diseases.' In the Phase 3 VALIANT study, EMPAVELI demonstrated an unprecedented 68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits as measured by C3 staining, compared to placebo. The positive results were consistent across adolescent and adult patients with C3G and primary IC-MPGN, and in C3G patients with post-transplant disease recurrence. 'EMPAVELI has the potential to be truly transformational for patients with C3G and primary IC-MPGN, who until now have had very few treatment options. In the largest pivotal study of these diseases, EMPAVELI demonstrated its potential to preserve kidney function by controlling all three key markers of disease,' said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer, Apellis. 'As Apellis' third approval in four years, this milestone underscores the unique ability of targeting C3 to improve patients' lives. We are deeply grateful to everyone who made this approval possible and look forward to building on this momentum as we advance pivotal studies of EMPAVELI in other rare kidney diseases.' 'The approval of EMPAVELI is a historic milestone for people living with C3G and primary IC-MPGN, many of whom are adolescents or young adults,' said Josh Tarnoff, chief executive officer, NephCure. 'We recognize Apellis' commitment to these patients and their families, and to the research and innovation that will bring this life-changing treatment into the hands of patients that need it most.' The approval of EMPAVELI is based on positive six-month results from the VALIANT study, demonstrating benefits across all three key markers of disease: Proteinuria reduction: The study met its primary endpoint, demonstrating a statistically significant 68% (p<0.0001) proteinuria reduction in EMPAVELI-treated patients compared to placebo. Stabilization of kidney function: EMPAVELI-treated patients achieved stabilization of kidney function compared to placebo (nominal p=0.03) as measured by estimated glomerular filtration rate (eGFR). Reduction of C3 staining: A majority of EMPAVELI-treated patients achieved a reduction in C3 staining intensity (nominal p<0.0001) compared to placebo. 71% of EMPAVELI-treated patients achieved zero C3 staining intensity, demonstrating complete clearance of C3 deposits. The safety profile of EMPAVELI is well-established, with >2,200 patient years across all approved indications. The most common adverse reactions in the VALIANT study (≥10%) were infusion site reactions, pyrexia, nasopharyngitis, influenza, cough, and nausea. Apellis is committed to helping patients with treatment access and support. ApellisAssist® is a program designed to help EMPAVELI patients along their treatment journey by providing a comprehensive system of support including help navigating insurance coverage, financial assistance for eligible patients, disease education, and ongoing product support. Patients and healthcare providers can call 1-888-273-5547 for more information. Conference Call and WebcastApellis will host a conference call and webcast to discuss the FDA's approval of EMPAVELI tomorrow, July 29, 2025 at 8:00 a.m. ET. To access the live call by phone, please pre-register for the call here. A live audio webcast of the event and accompanying slides may also be accessed through the 'Events and Presentations' page of the 'Investors and Media' section of the company's website. A replay of the webcast will be available for 30 days following the event. 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 therapy.2-4 Additionally, approximately 90% of patients who previously received a kidney transplant will experience disease recurrence.5 The diseases are estimated to affect 5,000 people in the United States and up to 8,000 in Europe.1 About the VALIANT StudyThe VALIANT Phase 3 study (NCT05067127) was a randomized, placebo-controlled, double-blinded, multi-center study that evaluated EMPAVELI® (pegcetacoplan) efficacy and safety in 124 patients who were 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 pediatric and adult patients, with native and post-transplant kidneys. Study participants were randomized to receive EMPAVELI or placebo twice weekly for 26 weeks. Following this 26-week randomized controlled period, patients were able to proceed to a 26-week open-label phase in which all patients received EMPAVELI. 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 EMPAVELI®/Aspaveli® (pegcetacoplan)EMPAVELI®/Aspaveli® (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. It is approved for the treatment of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in the United States and paroxysmal nocturnal hemoglobinuria (PNH) in the United States, European Union, and other countries globally. The therapy is also under investigation for other rare diseases. U.S. Important Safety Information for EMPAVELI BOXED WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA EMPAVELI, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as , , and type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update vaccination for encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, unless the risks of delaying therapy with EMPAVELI outweigh the risks of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria in patients receiving a complement inhibitor. Patients receiving EMPAVELI are at increased risk for invasive disease caused by encapsulated bacteria, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious infections and evaluate immediately if infection is suspected. Because of the risk of serious infections caused by encapsulated bacteria, EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the EMPAVELI REMS. CONTRAINDICATIONS Hypersensitivity to pegcetacoplan or to any of the excipients For initiation in patients with unresolved serious infection caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B WARNINGS AND PRECAUTIONS Serious Infections Caused by Encapsulated Bacteria EMPAVELI, a complement inhibitor, increases a patient's susceptibility to serious, life-threatening, or fatal infections caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis (caused by any serogroup, including non-groupable strains), and Haemophilus influenzae type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. The initiation of EMPAVELI treatment is contraindicated in patients with unresolved serious infection caused by encapsulated bacteria. Complete or update vaccination against encapsulated bacteria at least 2 weeks prior to administration of the first dose of EMPAVELI, according to the most current ACIP recommendations for patients receiving a complement inhibitor. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with EMPAVELI. Note that ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information. If urgent EMPAVELI therapy is indicated in a patient who is not up to date with vaccines against encapsulated bacteria according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer these vaccines as soon as possible. The benefits and risks of treatment with EMPAVELI, as well as the benefits and risks of antibacterial drug prophylaxis in unvaccinated or vaccinated patients, must be considered against the known risks for serious infections caused by encapsulated bacteria. Vaccination does not eliminate the risk of serious encapsulated bacterial infections, despite development of antibodies following vaccination. Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected. Inform patients of these signs and symptoms and instruct patients to seek immediate medical care if these signs and symptoms occur. Promptly treat known infections. Serious infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider interruption of EMPAVELI in patients who are undergoing treatment for serious infections. EMPAVELI is available only through a restricted program under a REMS. EMPAVELI REMS EMPAVELI is available only through a restricted program under a REMS called EMPAVELI REMS, because of the risk of serious infections caused by encapsulated bacteria. Notable requirements of the EMPAVELI REMS include the following: Under the EMPAVELI REMS, prescribers must enroll in the program. Prescribers must counsel patients about the risks, signs, and symptoms of serious infections caused by encapsulated bacteria, provide patients with the REMS educational materials, ensure patients are vaccinated against encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, prescribe antibacterial drug prophylaxis if patients' vaccine status is not up to date and treatment must be started urgently, and provide instructions to always carry the Patient Safety Card both during treatment, as well as for 2 months following last dose of EMPAVELI. Pharmacies that dispense EMPAVELI must be certified in the EMPAVELI REMS and must verify prescribers are certified. Further information is available at or 1-888-343-7073. Infusion-Related Reactions Systemic hypersensitivity reactions (eg, facial swelling, rash, urticaria, pyrexia) have occurred in patients treated with EMPAVELI, which may resolve after treatment with antihistamines. Cases of anaphylaxis leading to treatment discontinuation have been reported. If a severe hypersensitivity reaction (including anaphylaxis) occurs, discontinue EMPAVELI infusion immediately, institute appropriate treatment, per standard of care, and monitor until signs and symptoms are resolved. Interference with Laboratory Tests There may be interference between silica reagents in coagulation panels and EMPAVELI that results in artificially prolonged activated partial thromboplastin time (aPTT); therefore, avoid the use of silica reagents in coagulation panels. ADVERSE REACTIONS Most common adverse reactions in adult and pediatric patients 12 years of age and older with C3G or primary IC-MPGN (incidence ≥10%) were infusion-site reactions, pyrexia, nasopharyngitis, influenza, cough, and nausea. USE IN SPECIFIC POPULATIONS Females of Reproductive Potential EMPAVELI may cause embryo-fetal harm when administered to pregnant women. Pregnancy testing is recommended for females of reproductive potential prior to treatment with EMPAVELI. Advise female patients of reproductive potential to use effective contraception during treatment with EMPAVELI and for 40 days after the last dose. Please see full , including Boxed WARNING regarding serious infections caused by encapsulated bacteria, and . About Apellis Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company leading the way in complement science 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 C3-targeting medicines approved to treat four serious diseases. Breakthroughs for patients include the first-ever therapy for geographic atrophy, a leading cause of blindness, and the first treatment for patients 12 and older with C3G or primary IC-MPGN, two severe, rare kidney diseases. 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 LinkedIn and X. 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 the potential market opportunity of EMPAVELI for 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 the clinical trial results of EMPAVELI for C3G and IC-MPGN indicate an effect that is greater than the actual positive effect; 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. Media:Tracy Vineismedia@ Investors: Neil Carnahan, References1. Data on file using literature consensus. 2. Smith RJH, et al. Nat Rev Nephrol. 2019;15(3):129-143.3. Servais A, et al. Kidney Int. 2012;82(4):454-464.4. Zand L, et al. J Am Soc Nephrol. 2014;25(5):1110-1117.5. 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. A photo accompanying this announcement is available at
Globe and Mail
28-07-2025
- Health
- Globe and Mail
FDA Approves Apellis' EMPAVELI® (pegcetacoplan) as the First C3G and Primary IC-MPGN Treatment for Patients 12 and Older
Proven efficacy across all three key markers of disease—68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits Broad label includes adults and adolescents with C3G or primary IC-MPGN, and post-transplant C3G disease recurrence Well-established safety profile, consistent across >2,200 patient years in approved indications WALTHAM, Mass., July 28, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the U.S. Food and Drug Administration (FDA) has approved EMPAVELI ® (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older, to reduce proteinuria. C3G and primary IC-MPGN are rare kidney diseases, affecting 5,000 people in the United States. 1 'I'm excited to now have a highly effective therapy for a broad range of patients living with C3G and primary IC-MPGN,' 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. 'With standard of care, patients living with these rare and severe diseases frequently progress to kidney failure, necessitating lifelong dialysis and/or a kidney transplant. Given the urgent need, particularly in children, the approval of EMPAVELI marks a pivotal moment in the treatment of rare kidney diseases.' In the Phase 3 VALIANT study, EMPAVELI demonstrated an unprecedented 68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits as measured by C3 staining, compared to placebo. The positive results were consistent across adolescent and adult patients with C3G and primary IC-MPGN, and in C3G patients with post-transplant disease recurrence. 'EMPAVELI has the potential to be truly transformational for patients with C3G and primary IC-MPGN, who until now have had very few treatment options. In the largest pivotal study of these diseases, EMPAVELI demonstrated its potential to preserve kidney function by controlling all three key markers of disease,' said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer, Apellis. 'As Apellis' third approval in four years, this milestone underscores the unique ability of targeting C3 to improve patients' lives. We are deeply grateful to everyone who made this approval possible and look forward to building on this momentum as we advance pivotal studies of EMPAVELI in other rare kidney diseases.' 'The approval of EMPAVELI is a historic milestone for people living with C3G and primary IC-MPGN, many of whom are adolescents or young adults,' said Josh Tarnoff, chief executive officer, NephCure. 'We recognize Apellis' commitment to these patients and their families, and to the research and innovation that will bring this life-changing treatment into the hands of patients that need it most.' The approval of EMPAVELI is based on positive six-month results from the VALIANT study, demonstrating benefits across all three key markers of disease: Proteinuria reduction: The study met its primary endpoint, demonstrating a statistically significant 68% (p<0.0001) proteinuria reduction in EMPAVELI-treated patients compared to placebo. Stabilization of kidney function: EMPAVELI-treated patients achieved stabilization of kidney function compared to placebo (nominal p=0.03) as measured by estimated glomerular filtration rate (eGFR). Reduction of C3 staining: A majority of EMPAVELI-treated patients achieved a reduction in C3 staining intensity (nominal p<0.0001) compared to placebo. 71% of EMPAVELI-treated patients achieved zero C3 staining intensity, demonstrating complete clearance of C3 deposits. The safety profile of EMPAVELI is well-established, with >2,200 patient years across all approved indications. The most common adverse reactions in the VALIANT study (≥10%) were infusion site reactions, pyrexia, nasopharyngitis, influenza, cough, and nausea. Apellis is committed to helping patients with treatment access and support. ApellisAssist ® is a program designed to help EMPAVELI patients along their treatment journey by providing a comprehensive system of support including help navigating insurance coverage, financial assistance for eligible patients, disease education, and ongoing product support. Patients and healthcare providers can call 1-888-273-5547 for more information. Conference Call and Webcast Apellis will host a conference call and webcast to discuss the FDA's approval of EMPAVELI tomorrow, July 29, 2025 at 8:00 a.m. ET. To access the live call by phone, please pre-register for the call here. A live audio webcast of the event and accompanying slides may also be accessed through the 'Events and Presentations' page of the 'Investors and Media' section of the company's website. A replay of the webcast will be available for 30 days following the event. 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 therapy. 2 -4 Additionally, approximately 90% of patients who previously received a kidney transplant will experience disease recurrence. 5 The diseases are estimated to affect 5,000 people in the United States and up to 8,000 in Europe. 1 About the VALIANT Study The VALIANT Phase 3 study (NCT05067127) was a randomized, placebo-controlled, double-blinded, multi-center study that evaluated EMPAVELI® (pegcetacoplan) efficacy and safety in 124 patients who were 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 pediatric and adult patients, with native and post-transplant kidneys. Study participants were randomized to receive EMPAVELI or placebo twice weekly for 26 weeks. Following this 26-week randomized controlled period, patients were able to proceed to a 26-week open-label phase in which all patients received EMPAVELI. 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 EMPAVELI ® /Aspaveli ® (pegcetacoplan) EMPAVELI ® /Aspaveli ® (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. It is approved for the treatment of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in the United States and paroxysmal nocturnal hemoglobinuria (PNH) in the United States, European Union, and other countries globally. The therapy is also under investigation for other rare diseases. EMPAVELI, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update vaccination for encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, unless the risks of delaying therapy with EMPAVELI outweigh the risks of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria in patients receiving a complement inhibitor. Patients receiving EMPAVELI are at increased risk for invasive disease caused by encapsulated bacteria, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious infections and evaluate immediately if infection is suspected. Because of the risk of serious infections caused by encapsulated bacteria, EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the EMPAVELI REMS. CONTRAINDICATIONS Hypersensitivity to pegcetacoplan or to any of the excipients For initiation in patients with unresolved serious infection caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B WARNINGS AND PRECAUTIONS Serious Infections Caused by Encapsulated Bacteria EMPAVELI, a complement inhibitor, increases a patient's susceptibility to serious, life-threatening, or fatal infections caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis (caused by any serogroup, including non-groupable strains), and Haemophilus influenzae type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. The initiation of EMPAVELI treatment is contraindicated in patients with unresolved serious infection caused by encapsulated bacteria. Complete or update vaccination against encapsulated bacteria at least 2 weeks prior to administration of the first dose of EMPAVELI, according to the most current ACIP recommendations for patients receiving a complement inhibitor. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with EMPAVELI. Note that ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information. If urgent EMPAVELI therapy is indicated in a patient who is not up to date with vaccines against encapsulated bacteria according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer these vaccines as soon as possible. The benefits and risks of treatment with EMPAVELI, as well as the benefits and risks of antibacterial drug prophylaxis in unvaccinated or vaccinated patients, must be considered against the known risks for serious infections caused by encapsulated bacteria. Vaccination does not eliminate the risk of serious encapsulated bacterial infections, despite development of antibodies following vaccination. Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected. Inform patients of these signs and symptoms and instruct patients to seek immediate medical care if these signs and symptoms occur. Promptly treat known infections. Serious infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider interruption of EMPAVELI in patients who are undergoing treatment for serious infections. EMPAVELI is available only through a restricted program under a REMS. EMPAVELI REMS EMPAVELI is available only through a restricted program under a REMS called EMPAVELI REMS, because of the risk of serious infections caused by encapsulated bacteria. Notable requirements of the EMPAVELI REMS include the following: Under the EMPAVELI REMS, prescribers must enroll in the program. Prescribers must counsel patients about the risks, signs, and symptoms of serious infections caused by encapsulated bacteria, provide patients with the REMS educational materials, ensure patients are vaccinated against encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, prescribe antibacterial drug prophylaxis if patients' vaccine status is not up to date and treatment must be started urgently, and provide instructions to always carry the Patient Safety Card both during treatment, as well as for 2 months following last dose of EMPAVELI. Pharmacies that dispense EMPAVELI must be certified in the EMPAVELI REMS and must verify prescribers are certified. Further information is available at or 1-888-343-7073. Infusion-Related Reactions Systemic hypersensitivity reactions (eg, facial swelling, rash, urticaria, pyrexia) have occurred in patients treated with EMPAVELI, which may resolve after treatment with antihistamines. Cases of anaphylaxis leading to treatment discontinuation have been reported. If a severe hypersensitivity reaction (including anaphylaxis) occurs, discontinue EMPAVELI infusion immediately, institute appropriate treatment, per standard of care, and monitor until signs and symptoms are resolved. Interference with Laboratory Tests There may be interference between silica reagents in coagulation panels and EMPAVELI that results in artificially prolonged activated partial thromboplastin time (aPTT); therefore, avoid the use of silica reagents in coagulation panels. ADVERSE REACTIONS Most common adverse reactions in adult and pediatric patients 12 years of age and older with C3G or primary IC-MPGN (incidence ≥10%) were infusion-site reactions, pyrexia, nasopharyngitis, influenza, cough, and nausea. USE IN SPECIFIC POPULATIONS Females of Reproductive Potential EMPAVELI may cause embryo-fetal harm when administered to pregnant women. Pregnancy testing is recommended for females of reproductive potential prior to treatment with EMPAVELI. Advise female patients of reproductive potential to use effective contraception during treatment with EMPAVELI and for 40 days after the last dose. Please see full Prescribing Information, including Boxed WARNING regarding serious infections caused by encapsulated bacteria, and Medication Guide. About Apellis Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company leading the way in complement science 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 C3-targeting medicines approved to treat four serious diseases. Breakthroughs for patients include the first-ever therapy for geographic atrophy, a leading cause of blindness, and the first treatment for patients 12 and older with C3G or primary IC-MPGN, two severe, rare kidney diseases. 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 LinkedIn and X. 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 the potential market opportunity of EMPAVELI for 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 the clinical trial results of EMPAVELI for C3G and IC-MPGN indicate an effect that is greater than the actual positive effect; 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. Media: Tracy Vineis media@ 617.420.4839 Investors: Neil Carnahan, CFA 617.977.5703 References 1. Data on file using literature consensus. 2. Smith RJH, et al. Nat Rev Nephrol. 2019;15(3):129-143. 3. Servais A, et al. Kidney Int. 2012;82(4):454-464. 4. Zand L, et al. J Am Soc Nephrol. 2014;25(5):1110-1117. 5. 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. A photo accompanying this announcement is available at
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
Medscape
08-06-2025
- Health
- Medscape
Pegcetacoplan Maintains Kidney Benefits at 52 Weeks
Pegcetacoplan, a targeted C3/C3b inhibitor, shows sustained, robust effects on key markers of kidney disease in patients with C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), according to 52-week results from the VALIANT trial. The 26-week results, which were presented last October at Kidney Week 2024, showed a highly significant difference in change in proteinuria from baseline, as measured by the urine protein-to-creatinine ratio (uPCR); the reduction was 67.2% with pegcetacoplan vs 2.9% with placebo ( P < .0001). The current findings showed that not only were these benefits sustained in patients who remained on pegcetacoplan, patients who switched from placebo to pegcetacoplan also experienced reductions of similar magnitude, said Fadi Fakhouri, MD, of Lausanne University Hospital and the University of Lausanne, Switzerland. Fakhouri presented the 1-year results at the 62nd European Renal Association (ERA) Congress 2025. VALIANT Open-Label Period The phase 3 VALIANT trial involved 124 patients with native or post-transplant recurrent C3 glomerulopathy or primary IC-MPGN who were randomly assigned to treatment two times a week with either subcutaneous pegcetacoplan 1080 mg, or weight-based doses for adolescents weighing ≤50 kg (n = 63); or to placebo (n = 61). In the subsequent 26-week open-label period, the placebo group crossed over and all patients received pegcetacoplan. Overall, 59 (93.7%) people in the pegcetacoplan group and 55 (90.2%) in the placebo group completed the study treatment and 52-week assessment. At week 52, patients in the original pegcetacoplan group maintained the significant proteinuria reduction that was observed at week 26, with a mean urine protein-to-creatinine ratio (UPCR) change at week 26 of –67.2% and at week 52 of –68.3%. Patients in the placebo group, who experienced a +3.2% change in uPCR at week 26, experienced a reduction of –51.3% at week 52 after being switched to pegcetacoplan. In addition, eGFR levels in the pegcetacoplan group dropped slightly from -1.2 mL/min/1.73 m2 at week 26 to -3.7 mL/min/1.73 m2 at week 52; in the placebo-to-pegcetacoplan group, the corresponding changes were -7.9 mL/min/1.73 m2 at week 26 and -4.7 mL/min/1.73 m2 at week 52. In terms of safety, pegcetacoplan remained well-tolerated in the second 26 weeks, and treatment-emergent adverse events (TEAEs) were similar between the two arms during the open-label period (pegcetacoplan-to-pegcetacoplan, 77%; placebo-to-pegcetacoplan, 73.7%). Overall, infusion-related TEAEs decreased from the randomized to open-label period in the pegcetacoplan-to-pegcetacoplan group (33.3% vs 9.8%, respectively), suggesting an improvement in tolerability over time. Adherence was high, at 97.6% of patients having an adherence of ≥90% to ≤100%, overall. Infections included two cases of pneumococcal pneumonia (including one that was serious) occurring during the open-label period, and there was one case each of streptococcal pharyngitis and urinary tract infection caused by Escherichia . No patient deaths or allograft losses were reported. 'The open-label period results confirm pegcetacoplan's significant effect on kidney function,' Fakhouri said. 'Patients in the placebo arm experienced a decline in eGFR during the randomized controlled period, but a stabilization after switching to pegcetacoplan, comparable to randomized controlled period results in the pegcetacoplan group,' he added. Patients With Baseline Nephrotic Range Proteinuria The results of subanalyses looking at several other trial outcomes were also presented at the meeting. These included an analysis of patients with nephrotic range proteinuria at baseline, who are at a notably high risk of rapid disease progression to kidney failure. At baseline, 24 patients in the pegcetacoplan arm (38.1%) and 16 in the placebo arm (26.2%) had nephrotic range proteinuria, defined as a uPCR ratio ≥3 g/g. At the end of the initial 26-week period, among patients with nephrotic range proteinuria, those treated with pegcetacoplan had a relative reduction of proteinuria of 72.1% compared with placebo ( P < .0001). Patients Treated With Immunosuppressive Therapies A second subanalysis evaluated outcomes among 48 (76%) participants in the pegcetacoplan group and 42 (69%) in the placebo group who were receiving concomitant immunosuppressive therapy at baseline. Patients were permitted to continue the immunosuppressants provided that the doses had remained stable during the preceding 12-week period and throughout the trial. The outcomes between the two groups were similar: Immunosuppressant-treated patients who received pegcetacoplan had a relative reduction of 70% in proteinuria vs placebo ( P < .0001), and among patients who did not receive immunosuppressants, the relative reduction was 64.5% compared to placebo ( P = .0005). At week 26, 62.5% (30/48) of immunosuppressant‐treated patients receiving pegcetacoplan achieved a ≥50% reduction in proteinuria from baseline vs 4.8% (2/42) on placebo ( P < .0001). Among those not on immunosuppressants, 53.3% (8/15) of the pegcetacoplan group met the same endpoint compared with 5.3% (1/19) in the placebo arm ( P = .0087). 'Reassuringly, proteinuria was reduced in both groups, and the immunosuppressed group had a highly statistically significant reduction,' said David Kavanagh, MD, of the National Renal Complement Therapeutics Centre, Newcastle University, Newcastle upon Tyne, United Kingdom, who presented this subanalysis. Other outcomes, including stabilization of eGFR, were also similar to the overall reductions, including those patients not on immunosuppressants. Importantly, rates of TEAEs were also comparable between the immunosuppressed and non-immunosuppressed patients. 'If you can reduce your proteinuria by at least 50%, you're going to have an outcome that can reduce the risk of kidney failure,' Kavanagh concluded. The studies were sponsored by Apellis. Fakhouri reported relationships with Apellis, Sobi, Novartis, Roche, Alexion Pharmaceuticals, and AstraZeneca. Kavanagh reported relationships with Idorsia, Novartis, Chemocentryx, Alexion Pharmaceuticals, Samsung, Sobi, Gyroscope Therapeutics, Purespring, and Apellis.
