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]

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.

Summary of Apellis Pharmaceuticals Conference Call Company Overview - **Company**: Apellis Pharmaceuticals (APLS) - **Event**: Bank of America Healthcare Conference - **Date**: May 13, 2025 - **Speakers**: Cedric Francois (CEO), Tim Sullivan (CFO) Key Points Industry Context - Discussion on the impact of recent executive orders related to drug pricing, particularly the "most favored nation" clause, which may affect Medicare pricing for Apellis' products [4][5][6] - Monitoring of tariff situations and their potential impact on gross margins [9] Product Insights - **Cyfoviry**: - Patients are responsible for approximately 20% copay, around $400 per injection [11][12] - A significant drop in funding from charitable foundations has affected patient access, leading to increased sample usage by physicians [14][15][19] - Despite challenges, there was a 4% quarter-over-quarter growth in patient treatments from Q4 to Q1 [19] - The company is only treating about 10% of the geographic atrophy patient population, indicating substantial unmet demand [20] Competitive Landscape - Apellis has gained market share against competitors, with a shift from 40% to 55% in new patients treated for Cyfoviry compared to 45% for the competitor [32][33] - Differentiation in efficacy and dosing frequency (every other month) is cited as a key driver for market share gains [34] Financial Health - Apellis ended the quarter with $358 million in cash, sufficient to reach profitability without needing to raise additional capital [45] Upcoming Launches - **C3G Program**: - Launch expected in August, targeting a patient population of approximately 5,000 [46][47] - Promising data showing 70% of patients had no trace of disease after six months of treatment [49] - Administered subcutaneously twice a week, similar to PNH [50] Future Directions - Plans to leverage learnings from previous product launches to ensure a strong market entry for C3G [56] - Ongoing development of a siRNA program in geographic atrophy, aiming to synchronize treatments for improved patient outcomes [58][59] - Two phase three trials in nephrology are set to begin in the second half of the year [60] Marketing and Awareness - A direct-to-consumer (DTC) campaign featuring Henry Winkler has shown early success in driving patient awareness and engagement [66][71] Guidance and Market Outlook - The company is cautious about providing sales guidance until after the C3G launch, given the recent challenges faced with Cyfoviry [62] Miscellaneous - Apellis has received recognition for its advertising campaigns, indicating effective marketing strategies [67][71] This summary encapsulates the critical insights and developments discussed during the conference call, highlighting Apellis Pharmaceuticals' strategic positioning, product performance, and future growth potential in the biotech industry.