Core Insights - Apellis Pharmaceuticals' stock has decreased by 31% over the past year despite receiving FDA approval for EMPAVELI, indicating a disconnect between regulatory success and commercial performance [2][7] Product Approval and Market Potential - EMPAVELI is the first and only approved treatment for C3 glomerulopathy and IC-MPGN, targeting approximately 5,000 patients in the U.S., with exclusive approval for about two-thirds of this population [3] - The positive opinion from the European CHMP in December 2025 enhances the global expansion potential and pricing power, typically associated with gross margins exceeding 90% [3] Patient Start Forms and Growth Expectations - Management has guided for 225 cumulative patient start forms by the end of 2025, having achieved 152 by the end of September, suggesting a slowdown to 24 forms per month in Q4 [4] - CEO Cedric Francois indicated that the company is moving past the initial wave of early adopters and anticipates steady growth moving into the next year [4] Revenue Challenges and Market Dynamics - SYFOVRE, which constitutes the majority of revenue, reported flat sales of $151 million in Q3 2025, with a mere 4% growth in injections, primarily driven by free goods costing the company $15 million [5] - The share of new patients for SYFOVRE has decreased to 52%, with management noting that retina specialists are adopting a cautious approach despite SYFOVRE's 60% market share [5] External Market Issues - The cessation of new co-pay assistance applications by Good Days Foundation has led to practices halting new patient enrollments, a challenge not unique to Apellis but reflective of broader issues in the geographic atrophy market [6]

Core Insights - Apellis is focused on the complement pathways, particularly complement factor C3, which allows exploration of various therapeutic options across multiple areas [1] - The company is currently self-funded and has identified growth drivers for both the near and long term [2] Company Overview - Apellis has established a foundation around its products SYFOVRE and EMPAVELI, which are expected to support its future pipeline [2] - SYFOVRE is generating a steady and durable revenue stream that aligns with the company's long-term growth ambitions [2]

2 Transforming innovation into durable value creation Apellis: The Leading Complement Company 44th Annual J.P. Morgan Healthcare Conference Cedric Francois, M.D., Ph.D. Co-Founder, Chief Executive Officer and President January 12, 2026 ARCHER Living with C3G 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 ...

Financial Data and Key Metrics Changes - The company is currently cash neutral and on a path to profitability, with over $450 million in cash available [6][64] - The company reported a dramatic reduction in proteinuria of approximately 68% in patients with C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis [10][11] - The company has been skating close to cash operational expenses, indicating strong financial health [64] Business Line Data and Key Metrics Changes - Apellis has two approved drugs: EMPAVELI for paroxysmal nocturnal hemoglobinuria (PNH) and recently approved indications for C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis [5][6] - SYFOVRE, the first treatment for geographic atrophy, was approved in 2023 and has stabilized its market share at just north of 60% [6][45] Market Data and Key Metrics Changes - The prevalence of C3G and IC-MPGN in the U.S. is approximately 5,000 patients, with about 50% progressing to end-stage renal disease over ten years [9] - The company has achieved around 5% market penetration with 225 start forms for the new indications within five months [14][32] Company Strategy and Development Direction - The company is focused on educating nephrologists about its new drug indications and differentiating its products from competitors [16][19] - Future trials are planned for FSGS and delayed graft function, with expectations of positive outcomes based on the underlying disease mechanisms [26][28] Management's Comments on Operating Environment and Future Outlook - Management noted that the competitive landscape for PNH has not led to significant patient loss, with 15% of patients returning to EMPAVELI after trying oral alternatives [24] - The company anticipates a strong 2026, with ongoing efforts to improve physician education and patient access to treatments [64][77] Other Important Information - The company is developing a siRNA combination therapy that aims to enhance the efficacy of SYFOVRE and potentially allow for less frequent dosing [67][69] - There is a focus on leveraging machine learning and AI to improve patient imaging and tracking disease progression [58][61] Q&A Session Summary Question: What is the opportunity with C3G and IC-MPGN? - The company highlighted the significant unmet need in these kidney diseases and the potential for its drug to provide a new treatment option [9][10] Question: How will the company increase its market share in the 5,000 prevalent pool? - Management emphasized the importance of education and building relationships with nephrologists to drive adoption [16][19] Question: What is the current status of SYFOVRE in the market? - The company reported stabilization in market share and ongoing efforts to communicate the drug's benefits to the retina community [40][45] Question: What are the timelines for upcoming studies? - The company indicated that trials for FSGS and delayed graft function have just opened, with updates expected next year [28][29] Question: How is the company addressing the impact of patient copay assistance foundations? - Management acknowledged the significant impact on patient access and is focusing on physician education to mitigate these challenges [75][76]

Core Insights - Apellis is in a strong position with a foundational technology targeting the complement cascade, specifically C3, which is believed to optimally control complement dysregulation [2] - The company has achieved three drug approvals in approximately 3 to 3.5 years, indicating a robust pipeline and growth potential [2] Drug Approvals - The first approval was for EMPAVELI, targeting paroxysmal nocturnal hemoglobinuria [3] - The second approval was for SYFOVRE, which addresses geographic atrophy, received about 2.5 years ago [3] - Recently, EMPAVELI received a second approval for C3G and IC-MPGN, bringing the total indications to four [3]

Core Insights - Apellis Pharmaceuticals, Inc. reported a revenue of $458.58 million for the quarter ended September 2025, marking a year-over-year increase of 133% [1] - The earnings per share (EPS) for the same period was $1.67, a significant improvement from -$0.46 a year ago [1] - The reported revenue exceeded the Zacks Consensus Estimate of $366.21 million by 25.22%, while the EPS surprise was 62.14% above the consensus estimate of $1.03 [1] Revenue Breakdown - Product revenue, net was $177.76 million, slightly below the nine-analyst average estimate of $178.43 million, reflecting a year-over-year change of 0.7% [4] - Licensing and other revenue reached $280.82 million, significantly surpassing the estimated $126.67 million, representing a year-over-year increase of 1286.2% [4] - Product Revenue from EMPAVELI was $26.83 million, exceeding the average estimate of $22.06 million, with a year-over-year change of 9% [4] - Product Revenue from SYFOVRE was $150.93 million, slightly below the average estimate of $156.55 million, showing a year-over-year decrease of 0.7% [4] Stock Performance - Shares of Apellis Pharmaceuticals have returned +30.1% over the past month, outperforming the Zacks S&P 500 composite's +3.6% change [3] - The stock currently holds a Zacks Rank 3 (Hold), indicating potential performance in line with the broader market in the near term [3]

Financial Data and Key Metrics Changes - Total revenue for Q3 2025 was $459 million, including a $275 million upfront payment from Sobi related to the EMPAVELI royalty purchase agreement [21] - SYFOVRE net product revenue for the quarter was $151 million, with approximately 101,000 doses delivered, including 86,000 commercial doses and 15,000 free goods doses [21] - Operating expenses were $235 million in Q3 2025, down from $244 million in the same quarter last year [24] Business Line Data and Key Metrics Changes - EMPAVELI generated total net product revenue of $27 million during Q3 2025, with a focus on C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis [24] - SYFOVRE maintained a leading position, accounting for an estimated 52% of new patient starts during Q3 2025, with a total injection growth of 4% [15][16] Market Data and Key Metrics Changes - The addressable market for EMPAVELI expanded by approximately 5,000 patients following FDA approval for C3 glomerulopathy and primary ICMPGN [6] - Only about 10% of patients diagnosed with geographic atrophy (GA) are currently treated with complement inhibitors, indicating significant room for growth [16] Company Strategy and Development Direction - The company aims to leverage its expertise in complement-mediated diseases to expand market opportunities for EMPAVELI and SYFOVRE while advancing its pipeline [8][9] - Initiatives to drive growth include targeted market education and the development of artificial intelligence tools to enhance physician understanding of GA [18] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term growth potential of EMPAVELI and SYFOVRE, highlighting positive early feedback from the nephrology community regarding EMPAVELI [26] - The company anticipates steady, measured injection growth for SYFOVRE, with expectations for renewed growth driven by new tools and education initiatives [8][16] Other Important Information - The company ended the quarter with $475 million in cash and cash equivalents, supported by the Sobi transaction [24] - The company plans to initiate pivotal trials for FSGS and DGF by the end of the year, expanding EMPAVELI's development into other rare kidney diseases [20] Q&A Session Summary Question: Regarding the launch of EMPAVELI and C3G - Management expects steady, consistent growth going into next year after the initial wave of early adopters [31] Question: On SYFOVRE sampling and market dynamics - Management is closely monitoring sampling and free goods programs to ensure they are utilized effectively [37] Question: Specifics on C3G patient demographics - Approximately 50 of the 152 start forms are from the Expanded Access Program, with positive feedback on efficacy leading to switches from competitive products [41] Question: Timeline for patient access to EMPAVELI - The typical timeframe for patients to start treatment is four to six weeks, with efforts underway to accelerate this process [52] Question: On the impact of co-pay assistance on patient treatment - The lack of co-pay support has created logistical and financial headwinds, affecting treatment discussions with patients [95] Question: Insights on physician behavior regarding biopsies for C3G - The availability of effective treatments like EMPAVELI motivates clinicians to diagnose and treat patients more aggressively [123]

Third Quarter 2025 Financial Results Conference Call October 30, 2025 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," "projec ...

Core Insights - Apellis is currently leading the field in complement therapies with two approved products, SYFOVRE and EMPAVELI, and has a promising pipeline ahead [2][3] - The company has been successfully launching EMPAVELI for PNH for a couple of years and recently received approval for C3G and IC-MPGN, presenting significant opportunities to impact patient lives [3] Company Overview - Apellis has established itself as a leader in complement therapies, focusing on complement factor C3, which plays a crucial role in controlling downstream effects of the complement cascade [2] - The company is positioned for growth with its innovative products and expanding indications, particularly in the latter part of the year [2][3]

Financial Data and Key Metrics Changes - The company has a strong balance sheet with a recent financing deal adding $275 million to its resources [11] - The company reported three approvals for its products in recent years, indicating significant progress in its commercial strategy [8][10] Business Line Data and Key Metrics Changes - SYFOVRE is the market leader in the geographic atrophy market, being the first product approved for this condition [9] - EMPAVELI has expanded its market by approximately 5,000 patients with recent approvals for C3G and IC-MPGN, in addition to its initial approval for PNH [10] Market Data and Key Metrics Changes - The company anticipates a steady growth trajectory in the ultra-rare disease market, particularly for EMPAVELI, with about 5,000 patients in the U.S. potentially qualifying for treatment [22][31] - The market for geographic atrophy is substantial, with estimates of over a million patients, indicating significant growth potential for SYFOVRE [45] Company Strategy and Development Direction - The company is focused on innovation and expanding its product pipeline, including the development of APL-3007, which aims to enhance the effectiveness of SYFOVRE [10][66] - The management emphasizes the importance of educating healthcare providers and patients about the benefits of their products to drive adoption [38][48] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the early launch of EMPAVELI in C3G and IC-MPGN, noting strong interest from key opinion leaders and positive early data [13][14] - The company believes it has a blockbuster product with the potential for significant market penetration across its indications, including PNH, C3G, and IC-MPGN [31][32] Other Important Information - The company has a differentiated approach in targeting the complement system, which is believed to be more effective than other treatments currently in development [64] - Management highlighted the challenges of demonstrating significant improvements in best-corrected visual acuity (BCVA) in geographic atrophy studies, emphasizing the importance of lesion slowing as a key measure [57][58] Q&A Session Summary Question: What is the current state of the business? - The company has made significant progress with its two commercial products and a strong balance sheet, feeling positive about its current position [11] Question: How is the launch of EMPAVELI progressing? - The launch is going well, with a broad label from the FDA and strong interest from healthcare providers [13][14] Question: What is the market opportunity for EMPAVELI? - The company believes it has a blockbuster product with a potential market of 5,000 patients in the U.S. and possibly more as awareness increases [31][32] Question: How does the company view competition in the market? - The company feels confident in its differentiated product offerings and the efficacy of its treatments compared to competitors [27][64] Question: What are the growth expectations for SYFOVRE? - Management anticipates low to mid-single-digit growth for SYFOVRE, with ongoing efforts to educate healthcare providers and patients [38][39] Question: How does the company plan to manage its patent lifecycle? - The company is focused on lifecycle management and has a solid patent strategy in place, with extensions potentially extending to 2034 or 2035 [34]