Apellis Pharmaceuticals (APLS) TD Cowen 46th Annual Health Care Conference summary

Company overview and strategic pillars

• Focused on three pillars: SYFOVRE for geographic atrophy, EMPAVELI for rare kidney diseases, and a gene editing program targeting FcRn, with clinical entry planned in H2 2025.

• SYFOVRE business is resilient, with plans for continued best-in-class therapy and a new prefilled syringe launch to broaden market reach.

• EMPAVELI is experiencing a strong launch in C3G and IC-MPGN, with over 5% market penetration in the first full quarter and expectations to reach up to 50% at peak.

• Pipeline includes phase III trials in FSGS and DGF, and a phase II combination program (3007 + SYFOVRE) targeting improved outcomes in geographic atrophy.

• On track toward profitability, supported by a solid organizational foundation and strong cash position.

Financial performance and outlook

• 2025 revenue was down year-over-year due to unique factors, including loss of patient co-pay assistance and inventory build, but underlying demand for SYFOVRE remained strong with 17% growth in injections.

• EMPAVELI revenue was steady in PNH and showed strong growth in new indications, with expectations for continued momentum into 2026.

• Free goods for SYFOVRE rose to 12–14% in 2025 from 5% in 2024 due to co-pay disruptions, but this is expected to decline as the market stabilizes.

• Cash balance exceeds $500 million, with additional milestone payments expected and a $94 million convertible note to address by September.

• Adjusted EBITDA was near break-even in some 2025 quarters, and profitability is anticipated with revenue growth and controlled expenses.

Product performance and competitive landscape

• SYFOVRE holds a stable 60% market share, with better patient compliance and longer injection intervals compared to competitors.

• Five-year data shows SYFOVRE significantly slows disease progression in geographic atrophy, with strong real-world evidence supporting efficacy.

• Safety profile for SYFOVRE is comparable to anti-VEGF injections, with only two vasculitis cases out of 370,000 injections in 2025, both resolved.

• EMPAVELI is the only approved product for pediatric and post-transplant populations in C3G/IC-MPGN, with a strong safety record and no observed encapsulated meningococcal infections.

• EMPAVELI's injectable format is less of a concern in severe diseases, where efficacy and safety are prioritized over convenience.