Apellis Pharmaceuticals (APLS) Goldman Sachs 45th Annual Global Healthcare Conference summary

Syfovre relaunch and market dynamics

• Syfovre relaunch is considered highly successful, driven by significant patient need and differentiated efficacy, with over 250,000 injections by end of Q1.

• Safety profile is now well understood, with rare adverse events occurring at a rate of one in 4,000, primarily on the first injection.

• Efficacy data continues to mature, with the Gale extension study showing statistically significant visual benefit at 36 months.

• Market penetration is about 12% of diagnosed patients seen by retina specialists, with the total addressable market revised to 1.5 million patients.

• Growth is broad-based across practice types, though academic uptake lags; competition with Izervay is expected to result in a stable duopoly for at least five years.

Regulatory and financial outlook

• EU approval decision for Syfovre is expected by July, with a final decision likely by year-end after an appeal process.

• Profitability is primarily driven by US growth; European approval would require additional but manageable investment.

• Operating expenses are expected to be lower than last year due to a reduction in force and stable infrastructure.

• The company is now adequately capitalized, having refinanced obligations and secured $700 million in non-dilutive flexibility.

Pipeline and future opportunities

• Phase III Valiant data for C3G and IC-MPGN is expected in late summer, targeting a population with high unmet need and no current treatments.

• Empaveli has shown strong safety and efficacy in PNH, with future focus shifting to kidney indications and xenotransplantation.

• siRNA asset APL-3007 is on track for phase I data this year, with further pipeline updates expected at an investor day.

• Partnership with Beam aims to lead in gene editing for complement pathways, targeting liver, eye, and brain indications.