Apellis(US:APLS)2025-07-29 13:00Summary 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.