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]

Financial Data and Key Metrics Changes - The company ended the quarter with just under $360 million in cash on the balance sheet, with outstanding debt obligations of $375 million in senior notes and approximately $187 million in convertible debt [42] - The normalized quarterly revenue is estimated to be in the range of $145 million to $150 million, which will serve as the new baseline for future growth [46][47] Business Line Data and Key Metrics Changes - The company has two commercial products: Empavedi and Cyfovri, with Cyfovri showing a gradual increase in market share, now at approximately 55% for new treatments compared to 45% for competitors [25] - Empavedi is positioned strongly in the market with a robust efficacy profile across various patient demographics, including adolescents and adults [28][29] Market Data and Key Metrics Changes - The company estimates approximately 5,000 patients in the U.S. with conditions treated by Empavedi, split roughly 50-50 between the two conditions [33] - The market for Cyfovri is large, with only about 10% of patients currently treated, indicating significant room for growth [16] Company Strategy and Development Direction - The company aims to enhance physician and patient education regarding the efficacy and safety of its products, particularly focusing on the long-term data available for Cyfovri [19][20] - There is a strategic focus on leveraging new imaging technologies to improve patient-physician communication and treatment adherence [20][21] Management's Comments on Operating Environment and Future Outlook - Management acknowledges a cautious view from the market but believes that the growth trajectory for Empavedi and Cyfovri will become clearer in the near future [4] - The company is optimistic about achieving profitability with modest growth and has built out necessary commercial capabilities in nephrology and ophthalmology [44] Other Important Information - The company is preparing for the launch of Empavedi, with commercial teams already profiling accounts and understanding patient locations [36] - The company has a developmental program targeting C3, which aims to enhance the efficacy of Cyfovri by significantly reducing systemic C3 levels [39][40] Q&A Session Summary Question: What is the company's outlook for Cyfovri given the first quarter's performance? - Management believes that the revenue has stabilized and that the impact of the Good Day's situation has been absorbed, setting a new baseline for growth [45][46] Question: How does the company differentiate its products from competitors? - The company emphasizes the superior efficacy of its products, particularly Cyfovri, which has shown a gradual increase in market share against competitors [25][24] Question: What is the competitive landscape for Empavedi? - The company highlights its strong efficacy profile across various patient demographics, which sets it apart from competitors [28][29]

Core Viewpoint - Apellis Pharmaceuticals has seen a 7.2% increase in share price over the past month, outperforming the S&P 500, but estimates have trended downward, indicating potential challenges ahead [1][2]. Financial Performance - The consensus estimate for Apellis Pharmaceuticals has shifted downward by 48.19% in the past month, reflecting a negative outlook [2]. - The company currently holds a Zacks Rank of 4 (Sell), suggesting expectations of below-average returns in the coming months [4]. VGM Scores - Apellis Pharmaceuticals has a subpar Growth Score of D and a Momentum Score of F, placing it in the bottom 40% for value strategy, resulting in an overall VGM Score of F [3]. Industry Comparison - In contrast, Vertex Pharmaceuticals, a peer in the same industry, reported revenues of $2.77 billion for the last quarter, with a year-over-year increase of 3% [5]. - Vertex is expected to post earnings of $4.24 per share for the current quarter, reflecting a significant year-over-year change of +133.1%, and holds a Zacks Rank of 3 (Hold) [6].

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]

Company Announcement - Apellis Pharmaceuticals, Inc. will present at the Goldman Sachs 46th Annual Global Healthcare Conference on June 11, 2025, at 10:40 a.m. ET [1] - A live conference webcast will be available on the company's website, with a replay accessible for approximately 90 days post-event [1] Company Overview - Apellis Pharmaceuticals is a global biopharmaceutical company focused on developing therapies for challenging diseases [2] - The company has introduced the first new class of complement medicine in 15 years, with two approved medicines targeting C3 [2] - Apellis has launched the first-ever therapy for geographic atrophy, a major cause of blindness globally, and aims to explore the potential of targeting C3 in various serious diseases [2]

Summary of Appellis Conference Call Company Overview - **Company**: Appellis Pharmaceuticals - **Core Product**: CyFovri, the first approved treatment for geographic atrophy in ophthalmology [2][5] Industry Context - **Industry**: Ophthalmology, specifically focusing on retinal diseases - **Market Penetration**: Approximately 10% of patients with geographic atrophy have been treated with CyFovri and its competitor [22] Key Points and Arguments Product and Pipeline - **CyFovri**: Demonstrated ability to slow down retinal tissue loss by up to 42% over two years [3] - **Market Usage**: Over 100,000 first eye treatments in the U.S. and more than 600,000 injections performed [4] - **Future Development**: Investigational siRNA product to be combined with CyFovri to potentially enhance treatment efficacy and extend dosing intervals [6][7] Competitive Landscape - **Competitors**: The primary competitor has been focusing on safety messaging, but CyFovri's efficacy has started to resonate more with physicians [16][19] - **Market Share**: CyFovri holds approximately 60% market share compared to 40% for the competitor, with a recent increase in first injections to 55% for CyFovri [20] - **Dosing Advantage**: CyFovri is approved for every two months dosing, while the competitor requires monthly dosing, which may lead to better patient adherence [18] Market Dynamics - **Patient Adoption**: The overall market is gradually adopting new treatments, similar to the historical adoption of anti-VEGF therapies [21] - **Patient Referrals**: Outreach programs to general ophthalmologists and optometrists are in place to increase patient referrals to retinal specialists [25][26] Financial and Operational Insights - **Tariff Management**: Most of the drug is manufactured in the U.S., mitigating some tariff risks, although some components are sourced from Switzerland and Japan [9][10] - **Co-Pay Assistance**: Approximately 20% of patients struggle with co-pay costs, but new funding avenues through Medicare Advantage Plans are being explored [31][32] - **Financial Position**: The company reported $315 million in cash at the end of the quarter, sufficient to fund operations towards profitability [48] Future Outlook - **Clinical Trials**: Upcoming Phase 2 clinical trial for the siRNA product is expected to provide significant data on treatment efficacy [6][47] - **Long-Term Confidence**: The company remains confident in its competitive position and the uniqueness of its product offerings in the complement pathway [46] Additional Important Insights - **Patient Education**: Programs are in place to educate patients and physicians about the benefits of CyFovri, including long-term data on treatment efficacy [34][35] - **Imaging Tools**: Development of imaging analyses to help physicians assess treatment effectiveness over time [36][38] This summary encapsulates the key points discussed during the Appellis conference call, highlighting the company's strategic positioning, product efficacy, market dynamics, and future growth potential.

EMPAVELI Launch and PNH Market - EMPAVELI (pegcetacoplan)获得美国FDA批准，成为首个获批的靶向C3疗法，用于治疗成人阵发性睡眠性血红蛋白尿症(PNH)[4, 5] - 美国现有约1500名接受补体治疗的PNH患者，每年新增约150名符合条件的PNH患者[10] - EMPAVELI上市初期反馈积极，医生已注册REMS项目，处方到首剂给药的平均时间为12天[9, 14] - 约75%从C5抑制剂转换到EMPAVELI的患者来自Ultomiris[14] PRINCE Trial Results - PRINCE试验在第26周达到主要终点，EMPAVELI组86%的患者实现血红蛋白稳定，而标准治疗组(SOC)为0%(p<0.0001)[20] - EMPAVELI组乳酸脱氢酶(LDH)降低90%，SOC组为14%(p<0.0001)[20] - EMPAVELI组9%的患者出现严重不良事件(SAE)，SOC组为17%[21] Pipeline Expansion - Apellis正在推进多种疾病的治疗方案，包括IC-MPGN/C3G、ALS、CAD和HSCT-TMA，预计在2021年下半年启动多项3期临床试验[23] - Apellis计划在2022年提交siRNA + EMPAVELI的IND申请，用于现有和新的适应症[33] Geographic Atrophy (GA) Program - DERBY和OAKS是两项针对GA患者的3期临床试验，共有1258名患者参与，预计9月份公布顶线结果[25] - Apellis计划在2022年启动一项针对中间期AMD的关键研究，前提是DERBY和OAKS的月度治疗组达到主要疗效终点[31] Financial Results - 2021年第二季度，净产品收入和总收入均为60万美元[32] - 研发费用为9590万美元，研究合作成本为5000万美元，一般及行政管理费用为4900万美元[32] - 净亏损为2.192亿美元[32] - 截至2021年6月30日，Apellis的现金为5.99亿美元，预计可为公司目前的运营计划提供资金至2022年下半年[32]

DERBY and OAKS Phase 3 Top-Line Results Conference Call September 9, 2021 Forward-Looking Statements 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 relating to Apellis' interpretation of results from the OAKS and DER ...

Leaders in Complement January 2022 Forward-looking statements Statements in this presentation 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. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "wo ...