Core Insights - Apellis Pharmaceuticals announced new data from the Phase 3 VALIANT study, highlighting the sustained efficacy of EMPAVELI (pegcetacoplan) in treating C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) [1][4] Efficacy and Safety - EMPAVELI demonstrated durable reductions in proteinuria and stable kidney function over one year, with significant proteinuria reduction maintained at 68% compared to placebo [2][10] - The treatment showed favorable safety and tolerability, with no new safety signals reported [5] - EMPAVELI was superior to iptacopan in reducing proteinuria levels and achieving composite renal endpoints, with a greater proportion of patients achieving significant UPCR reductions [6][7] Study Design and Results - The VALIANT study was a randomized, placebo-controlled trial involving 124 patients aged 12 and older, making it the largest trial for these conditions [13] - The primary endpoint was the log transformed ratio of urine protein-to-creatinine ratio (UPCR) at Week 26 compared to baseline [13] - Indirect treatment comparisons indicated that EMPAVELI was more effective than iptacopan in achieving proteinuria reduction and stabilization of estimated glomerular filtration rate (eGFR) [14][9] Patient Impact - Approximately one-third of patients treated with EMPAVELI achieved complete proteinuria remission, which was sustained through one year [10][9] - The treatment's efficacy was consistent across different patient groups, regardless of immunosuppressant use or baseline proteinuria levels [10][3] Market Position - EMPAVELI is the only approved treatment for C3G and primary IC-MPGN in patients aged 12 years and older, positioning it as a transformative option for patients at high risk of kidney failure [3][15] - The diseases affect an estimated 5,000 people in the U.S. and up to 8,000 in Europe, indicating a significant market opportunity for EMPAVELI [18]

Core Insights - Apellis Pharmaceuticals reported strong second quarter 2025 financial results, highlighting the FDA approval of EMPAVELI for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) as a significant milestone in their product portfolio [2][5][6] Financial Performance - Total revenue for Q2 2025 was $178.5 million, a decrease from $199.7 million in Q2 2024, driven by lower EMPAVELI sales [10] - U.S. net product sales included $151 million from SYFOVRE and $20.8 million from EMPAVELI, with licensing and other revenue contributing $7.1 million [10] - Cost of sales decreased to $13.6 million from $23.1 million year-over-year, attributed to lower product volumes supplied to Sobi and reduced expenses related to obsolete inventory [11] - R&D expenses were $67 million, down from $78 million in the same period last year, reflecting lower external costs and personnel expenses [14] - SG&A expenses increased to $131.1 million from $128.1 million, primarily due to higher office and travel expenses [15] - The net loss for Q2 2025 was $42.2 million, compared to a net loss of $37.7 million in Q2 2024 [16] Product Developments - EMPAVELI is the first FDA-approved treatment for C3G and primary IC-MPGN, showing a 68% reduction in proteinuria and stabilization of kidney function in clinical trials [6][20] - SYFOVRE continues to lead the market in geographic atrophy (GA) with a 55% share of new patient starts and total market share exceeding 60% [5][10] - The company is on track to initiate pivotal studies for delayed graft function (DGF) and focal segmental glomerulosclerosis (FSGS) in the second half of 2025 [6] Strategic Partnerships - Apellis entered a capped royalty purchase agreement with Sobi, allowing the company to receive up to $300 million for 90% of future ex-U.S. royalties for Aspaveli [5][13] - Sobi anticipates an opinion from the European Medicines Agency regarding Aspaveli's indication extension application by the end of 2025 [6] Cash Position - As of June 30, 2025, Apellis had $370 million in cash and cash equivalents, down from $411.3 million at the end of 2024, with expectations that current cash and future revenues will support operations until profitability [17]

Core Insights - The U.S. FDA has approved EMPAVELI (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients aged 12 and older, aimed at reducing proteinuria [2][3][4] Company Overview - Apellis Pharmaceuticals has achieved its third approval in four years with EMPAVELI, highlighting its focus on targeting C3 to improve patient outcomes [4][27] - The company is committed to supporting patients through programs like ApellisAssist, which offers assistance with insurance coverage, financial aid, and disease education [8][27] Clinical Study Results - The Phase 3 VALIANT study demonstrated a 68% reduction in proteinuria, stabilization of kidney function, and significant clearance of C3 deposits compared to placebo [3][12] - The study included 124 patients aged 12 and older, making it the largest trial for these rare kidney diseases [11] Disease Context - C3G and primary IC-MPGN are rare kidney diseases affecting approximately 5,000 individuals in the U.S., with a high risk of progression to kidney failure [10][12] - About 50% of patients with these conditions may experience kidney failure within five to ten years of diagnosis [10] Safety Profile - EMPAVELI has a well-established safety profile, with over 2,200 patient years of data across approved indications [5][7] - Common adverse reactions (≥10%) include infusion site reactions, pyrexia, and nasopharyngitis [7][25] Future Directions - Apellis plans to advance pivotal studies of EMPAVELI in other rare kidney diseases, building on the momentum from this approval [6][27]

Core Insights - Apellis Pharmaceuticals and Sobi presented new data from the Phase 3 VALIANT study for EMPAVELI (pegcetacoplan) targeting C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) at the European Renal Association Congress [1][5] Study Results - EMPAVELI showed a statistically significant 68% reduction in proteinuria compared to placebo at Week 26, with effects sustained for one year [2][4] - Patients switching from placebo to EMPAVELI also experienced similar benefits in proteinuria reduction and kidney function stabilization [3] Efficacy and Safety - The study confirmed EMPAVELI's efficacy and safety profile across a diverse patient population, including adults and adolescents with both native and post-transplant kidney disease [4][7] - No new safety signals were reported, indicating favorable safety and tolerability consistent with its established profile [4][7] Clinical Significance - The results highlight the urgent need for effective treatments for patients with C3G and primary IC-MPGN, as approximately 50% of these patients may face kidney failure within five to ten years of diagnosis [6] - The VALIANT study is the largest trial conducted in these populations, involving 124 patients aged 12 and older [8] Future Prospects - Marketing applications for EMPAVELI are currently under review with the FDA and EMA, with an FDA decision expected this summer [4][7] - The collaboration between Apellis and Sobi aims to expedite the availability of EMPAVELI for patients suffering from these rare kidney diseases [10]