据了解，作为一家处于商业化阶段的生物制药企业，Apellis致力于发现、开发和商业化治疗未满足医疗 需求的创新疗法。其核心产品线主要包括EMPAVELI和SYFOVRE两大产品：SYFOVRE用于治疗继发于 年龄相关性黄斑变性(GA)的地图样萎缩;而EMPAVELI则用于治疗阵发性睡眠性血红蛋白尿症(PNH)。 分析师特别指出，Apellis与Sobi公司达成的重大特许权购买协议具有战略意义。该协议不仅确保Apellis 可获得最高3亿美元收益(其中包含2.75亿美元首付款)，更将Sobi在Aspaveli销售中的特许权义务削减了 90%。这一举措不仅为Apellis带来即时财务收益，甚至有望缓解投资者对其盈利能力的担忧。 尽管存在这些积极因素，Frommer仍对Apellis的未来发展保持审慎态度。他认为，虽然FDA和CHMP即 将做出的监管决定，以及Empaveli/Aspaveli新适应症的上市都是重大利好，但这些因素在带来市场潜力 的同时，也伴随着同等程度的不确定性。 智通财经APP获悉，摩根士丹利分析师Judah Frommer发表研报，将Apellis Pharmaceuticals(APLS.U ...

Core Insights - Apellis Pharmaceuticals (APLS) shares increased by 3.2% following a capped royalty agreement with Sobi, allowing Apellis to receive up to $300 million for 90% of its future ex-U.S. royalties from Aspaveli [1][5] - The deal significantly enhances Apellis' financial position, providing operational flexibility with cash and marketable securities totaling $358.4 million as of the end of Q1 2025 [2] - The agreement includes a $275 million upfront payment and potential milestone payments of up to $25 million upon EU approval for Aspaveli in two severe kidney diseases [3][5] Financial Impact - The capped royalty deal will allow Sobi to receive 90% of ex-U.S. royalties until performance-based caps are met, after which all royalties will revert to Apellis [4] - Year-to-date, APLS shares have decreased by 44%, contrasting with a 3.4% decline in the industry [4] Regulatory Developments - Apellis and Sobi are seeking label expansion for Empaveli, with FDA and EU reviews currently underway for C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis [7] - A decision from the FDA is anticipated on July 28, 2025, while the EU decision is expected by year-end [7] Clinical Study Results - The phase III VALIANT study demonstrated a statistically significant 68% reduction in proteinuria for patients treated with Empaveli compared to placebo, indicating its effectiveness in C3G and IC-MPGN [9] - The study also showed that patients experienced stabilized kidney function, with 71% achieving complete clearance of C3c staining intensity [10][12]

Core Viewpoint - Apellis Pharmaceuticals has entered into a capped royalty purchase agreement with Sobi, allowing Apellis to receive up to $300 million in exchange for 90% of its future ex-U.S. royalties for Aspaveli, which is a treatment for rare diseases [1][2][3] Transaction Details - Apellis will receive $275 million upfront and may earn up to $25 million in milestone payments upon EMA approval for Aspaveli in treating C3G and IC-MPGN [3][7] - The agreement includes defined caps tied to Aspaveli's performance, after which 100% of ex-U.S. royalties will revert to Apellis [4] Product Information - Aspaveli (systemic pegcetacoplan) is approved in the EU and the U.S. for treating paroxysmal nocturnal hemoglobinuria (PNH) and is under review for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) [2][5] - Approximately 50% of patients with C3G and IC-MPGN may experience kidney failure within five to ten years of diagnosis, highlighting the urgency for effective treatments [6] Strategic Implications - The transaction is seen as a non-dilutive funding method that strengthens Apellis' balance sheet and provides operational flexibility as the company approaches sustainable profitability [2][7] - Sobi's expertise in rare diseases and its commercial footprint are expected to enhance the reach of Aspaveli globally following regulatory approvals [3]

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]

Group 1 - The market is highlighting the free cash flow (FCF) potential of Syfovre and Empaveli, with Syfovre projected to exceed $1 billion in annual sales despite initial rollout challenges due to infection reports [1] - The firm faced difficulties in launching Syfovre, but the outlook for its sales remains strong, indicating a recovery in market confidence [1] - A holistic investment approach is emphasized, considering both top-down and bottom-up valuation techniques, with a focus on capital structure and debt covenants impacting equity valuation [1]

WALTHAM, Mass., May 07, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), today announced its first quarter 2025 financial results and business highlights. "In the first quarter, we were pleased to see continued growth in SYFOVRE injection demand—a key indicator of long-term market strength—and an increase in share of new patient starts, already reaching 55% in April. However, revenue was impacted by funding shortages at third-party co-pay assistance programs and a larger- than-expected ...

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 Fe ...