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 this year.

• SYFOVRE business is resilient, with ongoing efforts to expand market penetration and introduce innovations like a prefilled syringe and functional OCT technology.

• EMPAVELI is experiencing strong early launch momentum in C3G and IC-MPGN, with expectations to reach up to 50% market penetration at peak.

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

• Company is on a path to profitability, supported by a solid organizational foundation and strong cash position.

Financial performance and outlook

• Revenue declined year-over-year due to unique factors, including loss of co-pay assistance for SYFOVRE and inventory build impacts.

• EMPAVELI revenue remained flat in PNH but showed strong growth in new indications, with continued momentum expected.

• SYFOVRE faced headwinds from co-pay assistance disruptions, but underlying demand grew, evidenced by a 17% increase in injections.

• Free goods as a percentage of SYFOVRE sales rose to 12-14% in 2025 but are expected to decline as the market stabilizes.

• Cash balance exceeds $500 million, with additional milestone payments expected and manageable debt obligations.

Product performance and market dynamics

• SYFOVRE maintains a stable 60% market share, with strong patient retention and compliance compared to competitors.

• Five-year data shows significant efficacy in slowing vision loss in geographic atrophy, with real-world evidence supporting differentiation.

• Safety profile for SYFOVRE is comparable to anti-VEGF injections, with very low incidence of vasculitis.

• EMPAVELI is the only approved therapy for pediatric and post-transplant populations in its class, with a strong safety record and no observed cases of encapsulated meningococcal infection.

• Oral competitor Fabhalta offers convenience, but EMPAVELI's efficacy and safety are prioritized in severe disease settings.