Financial Data and Key Metrics Changes - Total revenue for Q2 2025 was $178 million, with Cyfovri net product revenue of $151 million, reflecting a 6% quarter-over-quarter growth in total injections [11][9] - Operating expenses decreased to $212 million in Q2 2025 from $229 million in 2024, with expectations for 2025 operating expenses to align with 2024 levels [15] - The company ended the quarter with $370 million in cash and cash equivalents, which, combined with a $275 million cash payment from a royalty purchase agreement, is expected to fund the business to sustainable profitability [15][13] Business Line Data and Key Metrics Changes - Cyfovri revenues were $151 million, with a 6% growth in total injections, maintaining a 55% share of new patient starts in the geographic atrophy (GA) market [9][11] - Empavli revenue was $21 million, up 5% quarter-over-quarter, with patient compliance rates at 97% [12] - The launch of Empavli for C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis is underway, with expectations for meaningful revenue generation from these opportunities in 2025 [12][20] Market Data and Key Metrics Changes - The market opportunity in GA is significant, with Cyfovri's market leadership and strong patent life indicating a low likelihood of new entrants in the coming years [9] - The company estimates approximately 5,000 patients in the U.S. for C3G and primary ICMPGN, with expectations for Empavli to become the preferred treatment option [20] - The company is focused on expanding awareness and access for Empavli, particularly among nephrologists and patient communities [21][22] Company Strategy and Development Direction - The company aims to maximize the potential of Empavli by raising awareness, establishing it as the treatment of choice, and securing broad access for patients [21][22] - The strategy includes expanding Empavli's development into other rare kidney diseases, such as delayed graft function and focal segmental glomerulosclerosis [16][17] - The company is also focused on maintaining Cyfovri's market leadership through education and engagement with the clinical community [24][26] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term potential of Cyfovri and the growth opportunities for Empavli, particularly in underserved populations [10][12] - The company is optimistic about the impact of recent FDA approvals and the ongoing demand for its products, despite challenges related to funding shortages in patient assistance programs [11][20] - Management highlighted the importance of continued education and awareness efforts to drive patient treatment and compliance [22][104] Other Important Information - The company announced key leadership appointments, including a new Chief Research and Development Officer and Chief People Officer, to support its growth strategy [28][29] - The company is actively working to address the funding gap affecting patient access to treatments, with ongoing efforts to transition patients from assistance programs to commercial products [94][95] Q&A Session Summary Question: Can you help clarify the GA market dynamics given competitor growth claims? - Management emphasized their clear leadership across all metrics, including revenue and market share, and noted the quality of their data compared to competitors [33][35][36] Question: What are the trends in sampling and patient starts? - Management indicated that growth in injections is expected to continue, with a slight bump in sampling due to ongoing demand [39][41] Question: What are the plans for FSGS trial endpoints? - Management confirmed they are aligned with recent work on endpoints and are excited about the upcoming trial [43][45] Question: How is the company addressing the funding gap for patient assistance? - Management acknowledged the ongoing challenges but expressed commitment to supporting patients through various programs [84][85] Question: What metrics will be reported for Empavli's launch? - Management stated that initial reporting will focus on total start forms, with more detailed breakdowns to follow as data becomes available [116][120]

Financial Data and Key Metrics Changes - Total revenue for Q2 2025 was $178 million, with Cyfovri net product revenue of $151 million, reflecting a 6% quarter-over-quarter growth in total injections [10][11] - Operating expenses decreased to $212 million from $229 million in Q2 2024, with expectations for 2025 operating expenses to align with 2024 levels [14] - The company ended the quarter with $370 million in cash and cash equivalents, which, along with a $275 million cash payment from a royalty purchase agreement, is expected to fund the business to sustainable profitability [14][12] Business Line Data and Key Metrics Changes - Cyfovri revenues were $151 million, with a 6% growth in total injections, maintaining a 55% share of new patient starts and over 60% total market share in geographic atrophy (GA) [7][10] - Empavli revenue was $21 million, up 5% quarter-over-quarter, with patient compliance rates at 97% [11] - The company anticipates meaningful revenue generation from C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (ICMPGN) opportunities in 2025 [11] Market Data and Key Metrics Changes - The market opportunity in GA is significant, with Cyfovri's leadership and strong patent life indicating low likelihood of new entrants in the next few years [7] - The company estimates approximately 5,000 C3G and primary ICMPGN patients in the U.S., with expectations for Empavli to become the preferred treatment option [20] Company Strategy and Development Direction - The company is focused on expanding into rare nephrology with the launch of Empavli for C3G and ICMPGN, and plans to develop Empavli for delayed graft function (DGF) and focal segmental glomerulosclerosis (FSGS) [15][16] - The strategy includes raising awareness of Empavli as a disease-modifying therapy and establishing it as the treatment of choice among nephrologists [21][22] - The company aims to leverage its strong clinical data to support the approval and adoption of Empavli [21] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term potential of Cyfovri, citing its market leadership and the significant unmet needs in the GA market [7][8] - The company is optimistic about the growth trajectory of Empavli, especially following its recent FDA approval for C3G and ICMPGN [20] - Management acknowledged the challenges posed by high levels of free goods usage due to funding shortages but remains focused on transitioning patients to commercial products [10][14] Other Important Information - The company announced key leadership appointments, including Dr. Leslie Meltzer as Chief Research and Development Officer and Kelly Boucher as Chief People Officer [28][29] - A capped royalty purchase agreement with Sobe was established, providing a $275 million upfront cash payment and potential future milestones [12][13] Q&A Session Summary Question: Concerns about GA market growth compared to competitors - Management highlighted their clear leadership in metrics such as revenue and market share, emphasizing the quality of their data and the larger database used for competitive analysis [34][35] Question: Inquiry about sampling trends - Management noted that growth in injections is expected to continue, with a slight bump in samples due to ongoing demand [39][42] Question: FSGS Phase III plans and endpoints - Management confirmed plans to enroll in pivotal trials for FSGS by the end of the year, aligning with recent work on endpoints [44][45] Question: Impact of funding dynamics on patient treatment - Management acknowledged that some patients are unable to start treatment due to funding gaps, but emphasized ongoing efforts to assist patients through various programs [103][104] Question: Expectations for future injection growth - Management indicated that they expect stable, steady, gradual growth in injections, with a long way to go in terms of market penetration [51][67] Question: Clarification on patient start forms and REMS enrollments - Management confirmed that initial reporting will be on total start forms, with more detailed breakdowns to follow as data becomes available [116][120]

Financial Performance - Apellis reported approximately $151 million in GA (Geographic Atrophy) revenue for 2Q 2025[10] - EMPAVELI U S net product sales were $208 million in 2Q 2025[18] - SYFOVRE U S net product sales were $1506 million in 2Q 2025[18] - The company received $275 million upfront from a royalty purchase agreement for 90% of future ex-U S royalties for Aspaveli[17] - Total revenue for 2Q 2025 was $1785 million, compared to $1997 million in 2Q 2024[18] EMPAVELI and Pipeline - EMPAVELI received U S FDA approval for C3G and primary IC-MPGN[10] - Apellis plans to initiate pivotal studies in DGF (Delayed Graft Function) and FSGS (Focal Segmental Glomerulosclerosis) by year end[10, 21] - APL-3007 reduced circulating C3 concentration by up to 90% in a Phase 1 healthy volunteer study[25] SYFOVRE - SYFOVRE's overall GA market share exceeds 60%[10] - Apellis initiated a Phase 2 study of SYFOVRE + APL-3007[10, 23] Market and Strategy - Approximately 5,000 C3G and primary IC-MPGN patients are estimated in the U S [28, 29] - The company aims to raise awareness about EMPAVELI as a disease-modifying therapy and establish it as the treatment of choice for relevant conditions[33, 34]

Received U.S. FDA approval for EMPAVELI® (pegcetacoplan) for treatment of patients 12 years and older with C3G and primary IC-MPGNAnnounced capped royalty purchase agreement with Sobi under which Apellis receives up to $300 million for 90% of ex-U.S. royalties of Aspaveli® (pegcetacoplan)Generated $178 million in 2Q 2025 revenues, including $171 million in U.S. net product salesSYFOVRE® (pegcetacoplan injection) injection demand grew 6% quarter-over-quarter, with U.S. net product revenue of $151 millionCash ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2025 OR Commission File Number: 001-38276 APELLIS PHARMACEUTICALS, INC. (Exact Name of Registrant as Specified in its Charter) Delaware 27-1537290 (State or other jurisdiction of incorporation or organization) 100 Fifth Avenue, Waltham, MA 02451 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR ...

Summary of Apellis Pharmaceuticals Conference Call on Empivalli FDA Approval Company and Industry Overview - **Company**: Apellis Pharmaceuticals - **Product**: Empivalli (systemic pegcetacoplan) - **Indications**: Approved for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (ICMPGN) in patients aged 12 years and older Key Points and Arguments 1. **FDA Approval Milestone**: Empivalli is the first C3-targeting therapy approved for C3G and primary ICMPGN, marking a significant milestone for Apellis and the treatment landscape for these diseases [4][7][24] 2. **Broad Label**: The approval includes pediatric patients and those with post-transplant C3G disease recurrence, addressing previously underserved populations [5][15] 3. **Trifecta of Outcomes**: Empivalli demonstrated significant efficacy by achieving proteinuria reduction (68% reduction compared to placebo), stabilization of estimated glomerular filtration rate (eGFR), and substantial clearance of C3 deposits [12][14] 4. **Clinical Trial Data**: The FDA approval was based on the phase three Valiant study, which enrolled 124 patients, making it the largest trial for these conditions [11] 5. **Safety Profile**: Empivalli's safety profile is well established, consistent with existing treatments for paroxysmal nocturnal hemoglobinuria (PNH) [5][7] 6. **Commercial Launch Plans**: Apellis has prepared a field team to educate physicians and secure access to Empivalli for patients, with expectations for meaningful revenue generation starting in 2025 [6][19][20] 7. **Market Size and Patient Population**: The U.S. market is estimated to have approximately 5,000 patients with C3G and primary ICMPGN, with a conservative methodology based on diagnostic codes [17][60] 8. **Pricing Strategy**: The annual wholesale acquisition cost (WAC) is approximately $505,000 per patient, with expectations for gross-to-net in the mid-teens for 2025 [19] 9. **Patient Compliance**: High compliance rates are anticipated, with over 90% adherence observed in clinical trials for both Empivalli and PNH [99] Additional Important Content 1. **Payer Engagement**: Apellis has engaged extensively with payers to ensure broad coverage and access for patients, with a focus on navigating prior authorization processes typical for rare disease treatments [53][55] 2. **Physician Education**: The company is actively working to raise awareness among nephrologists about the importance of treating key disease markers and establishing Empivalli as the treatment of choice [21][22] 3. **Future Clinical Programs**: Apellis is advancing registrational programs for additional complement-driven kidney diseases, including focal segmental glomerulosclerosis (FSGS) and delayed graft function (DGF) [7][8] 4. **Market Dynamics**: The launch is expected to be gradual due to the rarity of the disease, with initial uptake driven by pent-up demand and subsequent growth as awareness increases [18][134] This summary encapsulates the critical aspects of the conference call regarding the FDA approval of Empivalli, highlighting the company's strategic direction, market potential, and the transformative impact of the treatment on patient care.

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]

Company Overview - Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company focused on developing therapies for challenging diseases, having introduced the first new class of complement medicine in 15 years [3] - The company has two approved medicines targeting C3, including the first-ever therapy for geographic atrophy, a leading cause of blindness globally [3] Upcoming Events - The company will host a conference call and webcast to discuss its second quarter 2025 financial results on July 31, 2025, at 8:30 a.m. ET [1] - A live audio webcast and accompanying slides will be available on the company's website, with a replay accessible for 90 days post-event [2]

Group 1 - Morgan Stanley analyst Judah Frommer raised the target price for Apellis Pharmaceuticals (APLS.US) from $25 to $26 while maintaining a "Hold" rating, based on the company's market position and recent financial strategy [1] - The significant licensing agreement between Apellis and Sobi is strategically important, ensuring Apellis can receive up to $300 million in revenue, including a $275 million upfront payment, while reducing Sobi's royalty obligations on Aspaveli sales by 90% [1] - This agreement provides immediate financial benefits to Apellis and may alleviate investor concerns regarding its profitability [1] Group 2 - Apellis is a commercial-stage biopharmaceutical company focused on discovering, developing, and commercializing innovative therapies for unmet medical needs [2] - The company's core product lines include EMPAVELI, used for treating paroxysmal nocturnal hemoglobinuria (PNH), and SYFOVRE, which treats geographic atrophy (GA) secondary to age-related macular degeneration [2]

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]