Pegcetacoplan

PDUFA target action date is July 28, 2025 WALTHAM, Mass., April 01, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review designation of the supplemental New Drug Application (sNDA) for EMPAVELI® (pegcetacoplan) for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), which are severe and rare kidney diseases. The Prescription Drug User Fee Act (PDUFA) target action date is July 28, 2025. “EMPAVELI demonstrated clinically meaningful benefits across all three key markers of disease – unprecedented proteinuria reductions, stabilization of kidney function, and substantial clearance of C3c staining,” said Bradley P. Dixon, M.D., FASN, professor of pediatrics and medicine, University of Colorado School of Medicine, and co-investigator of the VALIANT study. “Efficacy is critically important when treating C3G and IC-MPGN given the high risk of progression to kidney failure. As a disease-modifying therapy, EMPAVELI has the potential to make a life-changing difference for patients, if approved.” “This milestone represents a significant step toward our goal of bringing EMPAVELI to people living with C3G and primary IC-MPGN, regardless of their disease type, age, or transplant status,” said Cedric Francois, M.D., Ph.D., chief executive officer and co-founder at Apellis. “We look forward to working with the FDA to make this treatment available to patients in need as quickly as possible.” sNDA submission supported by positive Phase 3 VALIANT results at Week 26 The positive results were consistent across patients with C3G and IC-MPGN, adolescents and adults, and native and post-transplant kidney 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 (nominal p=0.03) as measured by eGFR. Reduction of C3c staining: A substantial proportion of EMPAVELI-treated patients achieved a reduction in C3c staining intensity (nominal p<0.0001). 71% of EMPAVELI-treated patients showed complete clearance of C3c staining compared to placebo. EMPAVELI showed favorable safety and tolerability, consistent with its established profile. Priority Review designation is granted to marketing applications for medicines that treat a serious condition and if approved, would provide a significant improvement in the safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition. 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. 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, 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 the VALIANT Study The 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 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 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 Collaboration 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. About Apellis 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 many serious 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 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: Lissa Pavluk media@apellis.com 617-977-6764 Investors: Meredith Kaya meredith.kaya@apellis.com 617-599-8178 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.

Seven years into its existence, Character Biosciences is ready to enter the clinic with a pair of experimental medicines targeting a bustling area of ophthalmology. The New Jersey biotech aims to treat age-related macular degeneration, a key driver of blindness in older adults, based on insights from genetic and imaging data that it collected from more than 6,500 people in an observational study. To get its first candidates into Phase 1 this year, Character announced Tuesday that it raised a $93 million Series B. The biotech previously raised $28 million in funding, including an $18 million Series A in 2022. The financing should take the 30-employee startup through Phase 2 testing for both intravitreal drug candidates, CEO and co-founder Cheng Zhang told Endpoints News . Investors are aMoon, Luma Group, Bausch + Lomb, Jefferson Life Sciences (an affiliate of Jefferson River Capital), Innovation Endeavors, Catalio Capital Management, S32 and KdT Ventures. Character is the second private biotech to announce a funding round on Tuesday after Hillstar Bio revealed its $67 million debut . It’s rare right now to see more than one venture funding round announcement per day, according to a Stifel report on Sunday. The average number of biotech venture rounds per month has slid 10% from two years ago and cratered 40% from the vibrant market of four years ago, according to the Stifel bankers. Character chose its drug targets based on the patient data from the observational study, and the startup believes that wealth of data can also give it a better shot at clinical success because it can hone in on the patients most likely to respond to treatment. Plus, the company worked with more than 150 ophthalmology centers for the observational study, and it plans to use some of those sites for upcoming clinical trials. “It’s been a long journey. It’s taken seven years, in fact, but we didn’t want to make any shortcuts,” Zhang said. “We wanted to build a rigorous company that is rooted in the fundamental science of patient data.” The company is exploring CTX114 as a treatment to slow the progression of geographic atrophy in advanced dry AMD, an increasingly popular category for drugmakers. Astellas and Apellis Pharmaceuticals both received FDA approval for geographic atrophy therapies in 2023 but have since faced regulatory setbacks. Last year, Astellas withdrew its application in Europe, and Apellis’ treatment was rebuffed by the bloc’s regulators, who raised questions about clinical benefit and safety. Like Astellas’ Izervay and Apellis’ Syfovre, Character is also pursuing the complement pathway with CTX114 but is tackling a “different genetically supported” part, according to Zhang. CTX114 is an engineered version of FHL-1, or factor H-like protein 1, a negative regulator of complement activation. Izervay is a complement 5 inhibitor and Syfovre is a C3 inhibitor. The company hopes CTX114 can also offer a less-frequent dosing schedule than Izervay and Syfovre, which are dosed monthy and every 25 to 60 days, respectively. As part of its due diligence on Character, aMoon investors talked with practicing ophthalmologists about the GA landscape, including Izervay and Syfovre. “They said, ‘Look, the risk-benefit for both of these drugs is just not amazing,'” aMoon partner Reut Shema said in an interview. “They said, ‘But we have nothing else. So if you give me something that’s similar efficacy, but just a little bit safer, it’s a multibillion-dollar drug.'” Meanwhile, with CTX203, Character hopes to thwart a patient’s progression to advanced AMD. CTX203 is an apolipoprotein A1 mimetic peptide that aims to stabilize ABCA1 expression. Character is also working with Bausch + Lomb to create new medicines for AMD. The duo began collaborating early last year and announced that work in January. Financial terms have’t been disclosed. “We would love, at a certain time, to move from incidence-driven drug development into a population-specific drug development,” Bausch + Lomb chief medical officer Yehia Hashad said in an interview. Character wants its platform to decipher which patients will be responders from the outset — before even going into clinical studies. “The more we are able to predict patients and treat them prior to getting to the late-stage part of the disease, the better,” Hashad said. The Bausch + Lomb tie-up does not involve Character’s CTX114 or CTX203. “Those could be highly competitive assets,” aMoon’s Shema said, “and because there’s so much interest from pharma companies, we wanted to make sure that this was open territory for different pharmas to bid for those because we think both of them have potential to be multibillion-dollar drugs.” Shema pointed out that Character also has datasets in glaucoma, which could form the basis for additional partnerships.