Apellis(APLS) - 2025 FY - Earnings Call Transcript

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][61] - The company reported a dramatic reduction in proteinuria of approximately 68% in patients with C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis [10] Business Line Data and Key Metrics Changes - Apellis has two approved drugs: Empaveli for paroxysmal nocturnal hemoglobinuria (PNH) and recently for C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis, and Syfovre for geographic atrophy [5][6] - The company has achieved approximately 5% market penetration in the C3G and IC-MPGN indications, with 225 start forms reported by the end of the year [13][31] 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% expected to progress to end-stage renal disease over ten years [8] - The company has seen a significant adoption of Empaveli in the pediatric population, with interest growing among adult nephrologists [18] Company Strategy and Development Direction - The company is focused on educating nephrologists about its new drug indications and the unique benefits of its therapies compared to oral alternatives [15][17] - Future trials are planned for FSGS and delayed graft function, with expectations of positive outcomes based on the underlying disease mechanisms [25][26] Management's Comments on Operating Environment and Future Outlook - Management noted that 2025 was a challenging year for retina practices due to funding issues affecting patient access to treatments, but they expect a gradual recovery [39][40] - The company is optimistic about the long-term data from the Gale study and the potential for Syfovre to improve patient outcomes in geographic atrophy [41][42] 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 [64][66] - Management emphasized the importance of new imaging technologies to better track disease progression and treatment efficacy in retinal diseases [51][54] Q&A Session Summary Question: What is the potential for growth in PNH? - Management indicated that despite the introduction of oral alternatives, many patients have returned to Empaveli, demonstrating its strong efficacy and safety profile [22][23] Question: How does the company plan to increase market share in C3G and IC-MPGN? - The strategy focuses on education and building relationships with nephrologists, particularly in pediatric and transplant settings [15][18] Question: What is the current market share for Syfovre? - The company has stabilized its market share just north of 60%, with ongoing efforts to maintain and grow this position [43] Question: What are the timelines for upcoming trials? - Trials for FSGS and delayed graft function have just opened, with updates expected next year [27][28] Question: How is the company addressing the impact of patient copay assistance foundations? - Management acknowledged the significant impact of funding issues on patient access and is focusing on physician education to mitigate these challenges [72][74]