Summary of Apellis Pharmaceuticals (APLS) 2025 Conference Call Company Overview - **Company**: Apellis Pharmaceuticals (APLS) - **Date**: September 04, 2025 - **Focus**: Launch of new drugs, particularly in the complement inhibition space with products Ciforvi and Empavelli Key Points Current State and Outlook - Apellis is positioned as a leader in the complement inhibition field with two products on the market: Ciforvi and Empavelli [3][5] - The company is experiencing stable growth with guidance of low to mid single-digit growth for Ciforvi over the next several quarters [3][4] Product Launch and Market Dynamics - The launch of Empavelli for C3G and ICMPGN is progressing as expected, with a focus on addressing unmet needs in these disease areas [6][9] - Approximately 5,000 patients have been identified with C3G and ICMPGN, highlighting the significant unmet need [7][8] - The company is optimistic about the gradual uptake of Empavelli, emphasizing the importance of patient education and the functional benefits of the drug [9][10] Clinical Trials and Pipeline - Ongoing Phase 2 trial combining subcutaneous injection with Ciforvi aims to extend the dosing interval from every two months to every three months [4] - Two Phase 3 clinical trials for FSGS and delayed graft function are set to begin enrollment in the latter part of the year [4][52] - The company is also exploring preclinical programs that will be discussed in the following year [4][63] Financial Position - Apellis has a stable financial situation, ending the quarter with $370 million in the bank, which is expected to support the path to profitability [5][57] - A recent royalty deal with Sobe has strengthened the balance sheet and provided a growth platform [5][58] Competitive Landscape - Ciforvi is currently the market leader in new injections and total market share, with a recent shift in new treatment share favoring Ciforvi at approximately 55% [30][31] - The company is focused on generating additional data to maintain its competitive edge and grow the overall market [33][34] Regulatory and Market Access - Apellis is confident in navigating the regulatory landscape for its products, particularly in the context of delayed graft function [56] - The company is working on reimbursement strategies and co-pay assistance to ensure patient access to treatments [17][48] Future Developments - The company is exploring innovative approaches, such as functional OCT measurements, to enhance understanding of treatment impacts and patient experiences [38][41] - Apellis is also engaged in gene editing programs that aim to reduce immunoglobulin levels without affecting albumin, indicating a focus on novel therapeutic avenues [63][64] Additional Insights - The company is optimistic about the potential for Empavelli in transplant patients, which could change treatment paradigms in this area [22][23] - There is a strong emphasis on educating healthcare providers about the serious implications of diseases treated by Apellis, which could enhance market penetration [20][34] This summary encapsulates the key discussions and insights from the Apellis Pharmaceuticals conference call, highlighting the company's strategic direction, product pipeline, and market positioning.

Financial Data and Key Metrics Changes - In Q1 2025, Cifovri net product revenue was $130 million, which was down due to inventory dynamics and funding shortages at third-party co-pay assistance programs [12][14][23] - Cifovri injection demand grew 4% quarter over quarter, indicating a positive trend in market demand despite revenue shortfalls [8][12] - The company ended Q1 2025 with $358 million in cash and cash equivalents, indicating a strong liquidity position to fund operations towards profitability [17] Business Line Data and Key Metrics Changes - Cifovri maintained over 60% market share in geographic atrophy, with new patient starts exceeding 50% in Q1 and reaching 55% by late April [9][17] - Empavedi generated $20 million in net product revenue in Q1, with high compliance rates of 97% and a consistent safety profile [23] - The supplemental NDA for Empavedi was accepted by the FDA with a PDUFA date of July 28, indicating a significant upcoming launch opportunity [10][11] Market Data and Key Metrics Changes - The geographic atrophy market is expected to see continued but modest growth, with Cifovri positioned as a market leader [9] - The U.S. commercial opportunity for C3G and iCMPGN is estimated at 5,000 patients, with expectations for Empavedi to be used across all patient groups [22] Company Strategy and Development Direction - The company aims to capitalize on strong physician demand for Cifovri and is preparing for the launch of Empavedi in C3G and iCMPGN [24] - Strategic initiatives include enhancing disease state awareness and ensuring seamless access to treatment upon approval [20][22] - The company is focused on maintaining financial discipline while driving revenue growth and delivering meaningful treatments to patients [24] Management's Comments on Operating Environment and Future Outlook - Management acknowledged challenges in the market, including funding shortages impacting demand but expressed confidence in the long-term growth potential of Cifovri [6][8] - The company is optimistic about the upcoming launch of Empavedi and its potential to transform treatment in C3G and iCMPGN [24] - Management emphasized the importance of transitioning patients from samples to commercial doses to mitigate the impact of co-pay assistance program funding issues [113] Other Important Information - The company welcomed Craig Wheeler to its Board of Directors, highlighting his extensive experience in the biopharmaceutical industry [25] - The management is closely monitoring tariff situations and is prepared to take steps to mitigate any adverse impacts [17] Q&A Session Summary Question: Clarification on Q1 volume dynamics - Management clarified that Q1 saw a 4% growth in injections, with a drawdown of inventory impacting the number of vials delivered [30][34] Question: Expectations for copay assistance underfunding - Management does not expect the copay assistance underfunding issue to be resolved soon, but they are working to transition patients from samples to commercial doses [36][38] Question: Sales guidance considerations - Management indicated that they are not likely to provide sales guidance this year due to recent dynamics affecting the market [50][52] Question: Payer landscape post-label update - Management confirmed that Cifovri maintains preferred positioning with several large Medicare Advantage plans, and no significant changes were noted post-competitor label update [60] Question: Launch plans for C3G and ICMPGN - Management is actively profiling accounts and building relationships with key physician accounts in preparation for the launch of Empavedi [117]

Financial Data and Key Metrics Changes - Apellis Pharmaceuticals reported a net product revenue of $130 million for Q1 2025, which was down due to inventory dynamics and funding shortages at third-party co-pay assistance programs [10][12][19] - The company ended Q1 2025 with $358 million in cash and cash equivalents, indicating confidence in funding operations to profitability [12] Business Line Data and Key Metrics Changes - Cifovri injection demand grew by 4% quarter over quarter, but revenue fell short of expectations due to inventory drawdown and funding issues [5][10] - Empaveli generated $20 million in net product revenue for Q1 2025, with high compliance rates of 97% [19] Market Data and Key Metrics Changes - Cifovri maintains over 60% market share in geographic atrophy, with new patient starts exceeding 50% in Q1 and reaching 55% by late April [6][14] - The U.S. commercial opportunity for Empaveli in C3G and iCMPGN is estimated at 5,000 patients, with expectations for broad usage across patient groups [18] Company Strategy and Development Direction - The company is focused on expanding its market leadership in geographic atrophy and preparing for the launch of Empaveli in C3G and iCMPGN [4][7] - Apellis aims to drive revenue growth while maintaining financial discipline and delivering meaningful treatments to patients [20] Management's Comments on Operating Environment and Future Outlook - Management acknowledged challenges in Q1 but expressed confidence in the growth potential of Cifovri and the upcoming launch of Empaveli [4][20] - The company is preparing for pivotal studies in focal segmental glomerulosclerosis and delayed graft function in the second half of 2025 [8] Other Important Information - Apellis announced the addition of Craig Wheeler to its Board of Directors, bringing extensive biopharmaceutical leadership experience [21] - The company is actively working on educational programs to help practices manage patient reimbursement amid co-pay assistance funding shortages [15][16] Q&A Session Summary Question: Clarification on Q1 volume dynamics - Management clarified that Q1 saw a 4% growth in injections, with the reduction in vials delivered due to inventory drawdown and increased sample usage [27][30] Question: New patient share calculation - The company uses a site of care data set covering about 50% of the market for accurate new patient share calculations, differing from competitors' claims data [41] Question: Impact of co-pay assistance program - Management does not expect the co-pay assistance underfunding issue to resolve soon, and they are working to transition patients from samples to commercial doses [32][34] Question: Launch plans for C3G and ICMPGN - The company is actively profiling accounts and engaging with KOLs to ensure a strong launch for Empaveli upon approval [108][109] Question: Tariff exposure and manufacturing - The company is analyzing potential tariff exposure but has not disclosed specific details about manufacturing locations or IP domicile [74] Question: Net price dynamics in Q1 - Management noted that revenue recognition occurs when doses are shipped to distributors, which may not directly correlate with vials shipped to physicians [80][82]

Financial Data and Key Metrics Changes - Total revenue for Q4 2024 was approximately $13 million, with full-year revenue reaching $781 million, marking a nearly 100% increase compared to 2023 [48][50]. - The company reported a net loss of $36.4 million for Q4 2024 [49]. - Cash and cash equivalents at the end of 2024 stood at $411 million, indicating a strong financial position [51]. Business Line Data and Key Metrics Changes - Cipher generated over 120% year-over-year US net revenue growth, with nearly $900 million in sales for XIFAV in less than two years [5][7]. - XIFAV maintained a market share of over 60%, with approximately 94,000 doses delivered in Q4 2024 [21]. - Empavelli generated approximately $23 million in U.S. net product revenue in Q4 2024, with compliance rates at 97% [35]. Market Data and Key Metrics Changes - The overall GA market growth in Q1 2025 was tempered due to typical seasonal dynamics, including Medicare reverifications and winter storms affecting distribution [22]. - The company estimates that less than 10% of patients with geographic atrophy are currently receiving treatment, indicating significant growth potential [8][91]. Company Strategy and Development Direction - The company focuses on three key pillars: transforming the treatment of geographic atrophy, maximizing the impact of Empavelli in rare diseases, and advancing its innovative pipeline [5][6]. - Plans include launching Empavelli in C3G and ICMPGN in the second half of 2025, with ongoing development of next-generation treatments for GA [14][42]. Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's ability to create significant value for patients and shareholders, emphasizing a strong market position and growth potential in 2025 [53]. - The competitive landscape is viewed positively, with management noting that recent regulatory developments have reinforced their market leadership [19][92]. Other Important Information - The company plans to initiate a Phase 2 trial for a next-generation treatment for GA in Q2 2025 [40]. - Management highlighted the importance of patient access to retinal specialists and the need for higher utilization from physicians already treating patients with XIFAV [25]. Q&A Session Summary Question: Insights on Empavelli and FDA submission - Management expressed excitement about the submission for Empavelli and highlighted the importance of consistent results across various disease phenotypes [60][62]. Question: Expectations for Q1 results - Management indicated that Q1 results are expected to be lower than Q4 due to temporary factors but anticipates continued growth in 2025 [70][72]. Question: Competitive dynamics and label changes - Management discussed the impact of label changes for competitors and emphasized the unique advantages of XIFAV, including preferred status with payers [74][78]. Question: Market share and payer positioning - Management noted that they have secured preferred positioning with several major payers, which is expected to enhance market share [115][116]. Question: Patient adherence and sample usage - Management reported that adherence levels for XIFAV are positive and that increased sample usage is a sign of demand, which they believe is temporary [160][162].