Pegcetacoplan

· Approximately 8,000 people in Europe are living with C3G or primary IC-MPGN · Aspaveli (pegcetacoplan) is the first treatment of primary IC-MPGN and the first treatment for C3G and primary IC-MPGN for patients 12 years and older Sobi® (STO: SOBI), today announced that the European Commission (EC) has approved Aspaveli® (pegcetacoplan) for the treatment of adult and adolescent patients aged 12 to 17 years with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) in combination with a renin-angiotensin system (RAS) inhibitor, unless RAS inhibitor treatment is not tolerated or contraindicated The approval follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in December 2025. Aspaveli is a targeted C3/C3b inhibitor designed to regulate excessive complement activation, a key driver of C3G and primary IC-MPGN. These conditions are associated with progressive kidney damage and limited treatment options. “The European Commission approval for Aspaveli represents an important milestone for people living with C3G or primary IC-MPGN in Europe, two severe and rare kidney diseases with limited treatment options and a high risk of progression to kidney failure,” said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. “This approval is underpinned by robust clinical evidence from the VALIANT trial, which demonstrated that pegcetacoplan is able to preserve kidney function by reducing proteinuria, stabilising eGFR and clearing C3 deposits. Aspaveli is now available for patients 12 years and older with these serious conditions. It is the first approved therapy for adolescent patients (12 to 17 years) and the first treatment indicated for primary IC-MPGN.” C3G and primary IC-MPGN are rare kidney diseases affecting approximately 8,000 patients in Europe. More than half of people living with C3G or primary IC-MPGN suffer from kidney failure within five to 10 years of diagnosis, requiring a burdensome kidney transplant or dialysis therapy.1-3 This approval is based on positive results from the Phase 3 VALIANT study, in which Aspaveli demonstrated benefits across the three key markers of disease, including significant reduction in proteinuria, stabilisation of kidney function, and substantial clearance of C3 deposits. These positive results were recently published in The New England Journal of Medicine.6 Sobi and its partner Apellis Pharmaceuticals, Inc. have global co-development rights for systemic pegcetacoplan. 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 or primary IC-MPGN suffer from kidney failure within five to 10 years of diagnosis, requiring a burdensome kidney transplant or dialysis therapy.1-3 Additionally, approximately 90% of patients who previously received a kidney transplant will experience disease recurrence.4 The diseases are estimated to affect 5,000 people in the United States and up to 8,000 in Europe.5 About the VALIANT StudyThe VALIANT Phase 3 study (NCT05067127) was a randomised, placebo-controlled, double-blinded, multi-center study that evaluated 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 paediatric 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 were able to proceed to a 26-week open-label phase in which all patients received 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 Aspaveli®/Empaveli® (pegcetacoplan) Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 and C3b 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 the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older approved in the United States, European Union, and other countries globally. Aspaveli/Empaveli is also approved for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) in the United States, European Union, and other countries globally. About the Sobi® and Apellis Collaboration Apellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialisation rights for systemic pegcetacoplan. Apellis has exclusive U.S. commercialisation rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. Sobi® Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 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. References 1. Smith RJH, et al. Nat Rev Nephrol. 2019;15(3):129-143. 2. Servais A, et al. Kidney Int. 2012;82(4):454-464. 3. Zand L, et al. J Am Soc Nephrol. 2014;25(5):1110-1117. 4. Tarragón, B, et al. Clin J Am Soc Nephrol 2024; 19(8)1005-1015. 5. Data on file using literature consensus. 6. Fakhouri F, et al. N Engl J Med 2025;393:2210-2220 We are a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Our therapies are concentrated within the areas of Haematology, Immunology and Specialty Care. We contribute to societies by improving access to treatment of rare diseases. Find out more about our business and financial performance. Here we present our most recent press releases, stories, news articles, and images. 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“We have made the decision that we don’t want to do rescue BD, which is to just fill that gap,” Astellas Pharma CEO Naoki Okamura said at Fierce JPM Week 2026.\n Xtandi, Astellas Pharma’s roughly $6 billion prostate cancer juggernaut by annual sales, is set to lose U.S. patent protection in 2027. As the company continues looking for external innovation to secure sustainable growth, CEO Naoki Okamura does not want the company to make acquisitions for the sole purpose of preventing a revenue decline.“We have made the decision that we don’t want to do rescue BD, which is to just fill that gap,” Okamura said during a fireside chat at Fierce JPM Week 2026. “We will invest in [the] much longer term.”Astellas has a “healthy appetite for business development,” Okamura said. But rather than as a way to scale up, dealmaking for Okamura is about “new technologies, new assets that strengthen our pipeline, or even better, a technology platform.”“We have no intention to have a large-scale M&A [deal] to elevate the size of the company,” he said.Last year, Astellas struck a potential $1.34 billion deal to license a CLDN18.2-targeted antibody-drug conjugate from Chinese biotech Evopoint Biosciences. That ADC program adds to Astellas’ first-in-class CLDN18.2 therapy Vyloy and ASP2138, a CLDN18.2 bispecific. With these three modalities, Astellas is “trying to dominate the CLDN18.2 space,” Okamura said. The bispecific agent could potentially treat patients with lower expression levels of CLDN18.2 compared with Vyloy, he explained. And, while Vyloy is currently approved alongside chemotherapy, an ADC construct holds potential to free patients from systemic chemotherapy.Astellas has not been a drugmaker defined by therapeutic areas or modalities. Having a select group of focus areas has its pros and cons, Okamura said. The upsides are apparent, including growing expertise and long-term relationships with relevant stakeholders.“The flip side is that if you stick to [a] certain therapeutic area too much, you will reduce the possibility of building up the long-term product portfolio,” Okamura said.Astellas instead starts with “biology with strong disease linkage,” Okamura explained. “We identify the best modality to address this biology. Then, we try to figure out what is the best patient population to benefit from this combination of biology-modality.”Nevertheless, Astellas intends to prioritize areas in which it already has expertise—both for internal research and business development. For now, these areas are targeted protein degradation, immuno-oncology, blindness/regeneration and genetic regulation.To navigate the post-Xtandi era, Astellas has identified five strategic brands with blockbuster sales potential. Besides Vyloy, these include Pfizer-partnered bladder cancer ADC Padcev, acute myeloid leukemia treatment Xospata, geographic atrophy med Izervay and menopause therapy Veozah.Among them, Padcev has seen explosive growth thanks to its establishment as the new standard of care in first-line bladder cancer. The Nectin-4 agent is expected to garner even more interest with its recent progress in muscle-invasive bladder cancer. Astellas is projecting Padcev peak annual sales between $2.8 billion and $3.5 billion, although the U.S. portion of that number is not booked by the Japanese pharma. In contrast to Padcev’s all-but-certain growth trajectory, the futures of Izervay and Veozah look less clear. For Izervay, Astellas’ peak sales projection ranges between $1.4 billion and $2.8 billion, and the company expects Veozah could reach around $1 billion to $1.7 billion at peak, according to Okamura’s presentation Monday at the J.P. Morgan Healthcare Conference.However, Astellas recently downgraded its Izervay sales projection from $750 million to $550 million for this fiscal year, which will end in March. Part of the problem relates to patient access, which Okamura said is difficult for Astellas to change. And it’s not just Izervay that’s suffering. Apellis Pharmaceuticals’ rival complement inhibitor Syfovre has also struggled to sustain its momentum, as the company Monday reported a preliminary 4% year-over-year sales decline for 2025.In turn, Astellas is trying to focus on the part of the drug\'s commercialization that it can control—physician education.Izervay’s initial uptake has been slower than expected because Astellas needs to educate retinal specialists about the drug’s treatment effect. Unlike other eye disease such as wet age-related macular degeneration, GA patients cannot feel how Izervay is working, Okamura told Fierce. Retinal specialists need to be assured that continued treatment with Izervay offers a better chance at disease improvement, he said. With that education effort in place, Astellas is now targeting community ophthalmologists. The company is taking a stratified approach to community outreach and is in the process of identifying key prescribers. Okamura noted that Astellas has experience targeting a relatively large, nonspecialist audience.As for menopause drug Veozah, the biggest uncertainty may lie in how competition with Bayer’s Lynkuet plays out.Okamura doesn’t view having Bayer, a women’s health veteran, as a competitor as necessarily bad given that both companies can help drive awareness. If the entire market grows bigger, Veozah may be better off even if it’s getting a smaller piece of the entire pie, the Astellas chief executive suggested.Bayer\'s Lynkuet is a dual-action drug targeting both NK3 and NK1 receptors, while Veozah, which holds a first-mover advantage, only targets NK3. Okamura argued that differentiation between the two approaches might come to the fore over time as real-world data emerge. Beyond specific products, one big uncertainty hovering over Astellas is changing U.S. drug pricing policy. While many Big Pharma companies have signed most-favored-nation deals with the Trump administration, it remains unclear whether midsized drugmakers like Astellas will be required to do the same.Astellas has been watching recent developments around MFN and is preparing proactively to respond if the scope of the policy reaches the company, Okamura said.For his prediction for 2026, Okamura said he remains optimistic about the biopharma industry despite the surprising lack of deal announcements at JPM this year.“There are many things moving under this,” Okamura said. “So, I still believe that 2026 is going to be another productive and prosperous year.”