Pegcetacoplan

SYFOVRE is the only approved GA treatment to demonstrate a visual function benefit in a prespecified endpoint Data presented at the Clinical Trials at the Summit Meeting June 10, 2024 - Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that SYFOVRE® (pegcetacoplan injection) preserved visual function at 36 months in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). These positive data from the GALE long-term extension study were presented at the Clinical Trials at the Summit (CTS) Meeting on June 8 in Park City, Utah. “SYFOVRE is the only approved GA treatment to show a benefit on visual function in a prespecified endpoint,” said Dilsher Dhoot, M.D., presenting author, vitreoretinal surgeon, California Retina Consultants, Santa Barbara, CA. “The vision loss caused by GA is devastating for patients, taking away their ability to drive and read. These groundbreaking data clearly demonstrate SYFOVRE’s potential to make a meaningful difference for patients.” In a prespecified microperimetry endpoint, patients developed fewer new scotomatous points with 36 months of both continuous monthly (p=0.0156) and every-other-month (p=0.1233) treatment compared to patients from the sham crossover group (all p-values nominal). Scotomatous points measure areas of the retina that have lost all light sensitivity and therefore are no longer functioning. “These results further reinforce the importance of slowing GA lesion growth to preserve visual function, adding to the largest body of evidence for a GA treatment,” said Caroline Baumal, M.D., chief medical officer, Apellis. “As leaders in GA, we are committed to advancing our understanding of the benefits of SYFOVRE on this progressive and long-term disease.” View full table by clicking on this link or the image below: GALE (n=792) is a Phase 3, multicenter, open-label, extension study to evaluate the long-term efficacy and safety of SYFOVRE® (pegcetacoplan injection) in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The objectives of the study are to evaluate the long-term incidence and severity of ocular and systemic treatment emergent adverse events as well as change in the total area of GA lesions as measured by fundus autofluorescence. More than 80-percent of participants who completed the OAKS and DERBY studies entered the GALE study. GALE also includes 10 patients who were previously enrolled in the Phase 1b study of pegcetacoplan for GA. Patients in the sham crossover group completed sham treatment from Months 0-24 in the Phase 3 OAKS study and received SYFOVRE from Months 24-36. Microperimetry was a key secondary endpoint measured only in the OAKS study, and therefore, patients who crossed over from the OAKS study were included in this analysis. OAKS (n=637) and DERBY (n=621) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies comparing the efficacy and safety of SYFOVRE® (pegcetacoplan injection) with sham injections across a broad and heterogenous population of patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The studies evaluated the efficacy of monthly and every-other-month SYFOVRE in patients with GA assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence. In Phase 3 studies at 24 months, both every-other-month and monthly SYFOVRE reduced GA lesion growth with increasing effects over time and showed a well-demonstrated safety profile. SYFOVRE® (pegcetacoplan injection) is the first-ever approved therapy for geographic atrophy (GA). By targeting C3, SYFOVRE is designed to provide comprehensive control of the complement cascade, part of the body’s immune system. SYFOVRE is approved in the United States for the treatment of GA secondary to age-related macular degeneration. Geographic atrophy (GA) is an advanced form of age-related macular degeneration and a leading cause of blindness worldwide, impacting more than one million Americans and five million people worldwide.1,2 It is a progressive and irreversible disease caused by the growth of lesions, which destroy the retinal cells responsible for vision. The vision loss caused by GA severely impairs independence and quality of life by making it difficult to participate in daily activities. On average, it takes only 2.5 years for GA lesions to start impacting the fovea, which is responsible for central vision.3 Apellis 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 Twitter and LinkedIn. 1Rudnicka AR, Jarrar Z, Wormald R, et al. Age and gender variations in age-related macular degeneration prevalence in populations of European ancestry: a meta analysis. Ophthalmology 2012;119:571–580. 2Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health 2014;2:e106–116. 3Lindblad AS, et al, and AREDS Research Group. Arch Ophthalmol. 2009;127(9):1168-1174. 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Rapid reduction in disease activity seen at 12 weeks was sustained at one year 55% of patients showed zero C3c staining intensity, indicating that C3c deposits were cleared Highlighted as late-breaking oral presentation at the European Renal Association (ERA) Congress Sweden, May 24, 2024 -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Sobi® (STO:SOBI) today announced positive one-year results from the Phase 2 NOBLE study investigating systemic pegcetacoplan, a targeted C3 therapy, for the treatment of post-transplant recurrence of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). These data were presented during a late-breaking oral presentation at the European Renal Association (ERA) Congress taking place May 23-26 in Stockholm, Sweden. “It is exciting to see that treatment with pegcetacoplan rapidly reduced disease activity in only 12 weeks, and the effects were sustained over the long term,” said Fadi Fakhouri, M.D., Ph.D., presenting author and professor of nephrology at CHUV, Lausanne, Switzerland. “Post-transplant C3G and IC-MPGN patients are likely to experience disease recurrence, putting them at risk of needing another kidney transplant or dialysis. There is a huge need for a treatment that targets the cause of these diseases, and I am very encouraged by these unprecedented results.” Seven (64%) patients showed a reduction in C3c staining by two or more orders of magnitude of intensity from baseline. Six (55%) patients, including the three IC-MPGN patients, showed zero C3c staining intensity, indicating that C3c deposits were cleared. Consistent with C3c staining reduction, seven patients (64%) showed zero inflammation as measured by the activity score of the C3G histologic index. Excessive C3c deposits are a marker of disease activity, which can lead to kidney inflammation, damage, and failure. Clearance of both C3c deposits and inflammation allow the kidney to recover and prolong the function of the kidney. Additionally, treatment with pegcetacoplan resulted in sustained improvements in key measures of disease including proteinuria and continued stabilization of kidney function. “The NOBLE data further indicate that pegcetacoplan is treating the underlying cause of these diseases by directly targeting C3,” said Caroline Baumal, M.D., chief medical officer, Apellis. “Our ongoing Phase 3 VALIANT study evaluates the potential of pegcetacoplan in all patients with these rare kidney diseases, and we look forward to sharing the topline results later this year.” Pegcetacoplan was generally well-tolerated. The majority of adverse events were mild to moderate and consistent with previously reported results. "Patients with post-transplant C3G and IC-MPGN often face the daunting reality of disease relapse, so the need for a therapeutic approach addressing the root cause of these conditions cannot be overstated,” said Lydia Abad-Franch, M.D., head of R&D and medical affairs, chief medical officer, Sobi. “These findings underscore our conviction in pegcetacoplan’s potential to address these rare, severe, and life-threatening conditions in both native and post-transplant kidneys." The Phase 2 NOBLE study (NCT04572854) is a multicenter, open-label, randomized, controlled study designed to evaluate the efficacy and safety of pegcetacoplan in 13 adults who have post-transplant recurrence of C3G or IC-MPGN. Study participants were randomized in a 3:1 ratio to receive pegcetacoplan (IC-MPGN: n=2; C3G: n=8) or maintain standard of care (IC-MPGN: n=1; C3G: n=2) for 12 weeks, and all patients received pegcetacoplan from week 13 to week 52. The primary endpoint of the study was the proportion of patients with reduction in C3c staining on renal biopsy after 12 weeks of treatment with pegcetacoplan. Secondary endpoints include an evaluation of safety, the proportion of patients with reduction in C3c staining on renal biopsy after 52 weeks of treatment, and the proportion of patients achieving at least a 50% reduction in proteinuria. The VALIANT Phase 3 study (NCT05067127) is a randomized, placebo-controlled, double-blinded, multi-center study designed to evaluate pegcetacoplan efficacy and safety in approximately 90 patients who are 12 years of age and older with C3G or primary IC-MPGN. It is the only study to include both patients with native kidney disease and patients who have recurrent disease after receiving a kidney transplant. Study participants will be randomized to receive 1080 mg of pegcetacoplan or placebo twice weekly for 26 weeks. Following this 26-week randomized controlled period, patients will proceed to a 26-week open-label phase in which all patients receive pegcetacoplan. The primary endpoint of the study is the log transformed ratio of urine protein-to-creatinine ratio (uPCR) at week 26 compared to baseline. C3G and primary IC-MPGN are rare and debilitating kidney diseases that can lead to kidney failure. Excessive C3c deposits are a 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, two-thirds 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 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 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. Apellis 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. Apellis 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. 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. Learn more about Sobi at sobi.com and LinkedIn. 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. Zand L, et al Clinical findings, pathology, and outcomes of C3GN after kidney transplantation. J Am Soc Nephrol. 2014 May;25(5):1110-7. doi: 10.1681/ASN.2013070715. Epub 2013 Dec 19. 3. Data on file using literature consensus. 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