Apellis Pharmaceuticals (APLS) Baird's 2024 Global Healthcare Conference summary

Product portfolio and clinical progress

• Two approved products: Empaveli (PNH, 2021) and Syfovre (geographic atrophy, 2023), both considered on a path to blockbuster status.

• Empaveli demonstrated significant improvement in anemia for PNH and recently showed best-in-class phase III data in C3G and IC-MPGN, with potential to prevent transplantation or hemodialysis.

• Syfovre is the first approved therapy for geographic atrophy, with strong launch performance and meaningful reductions in lesion growth and functional vision preservation.

• Additional pipeline assets, including siRNA (APL-3007) and a Beam Therapeutics partnership, are expected to yield data and updates in late 2024 and 2025.

Market dynamics and launch updates

• Syfovre's launch benefited from retina community readiness and established reimbursement models, but only 7-8% of eligible GA patients are currently treated, indicating significant growth potential.

• Physician adoption is mixed, with about one-third embracing Syfovre and ongoing education efforts targeting both retina specialists and general ophthalmologists.

• Competition from Izervay introduced headwinds (J-code, contracting, safety messaging), but momentum is shifting back to Syfovre due to differentiated efficacy.

• Patient compliance with Syfovre is strong and comparable to anti-VEGF therapies.

Regulatory and geographic expansion

• European approval for Syfovre faces challenges after negative rapporteur opinions, but a final decision is expected in September; launch infrastructure is ready if approved.

• U.S. filing for Empaveli in C3G and IC-MPGN is planned for early 2025, with potential approval in the second half of the year.

• VALIANT phase III data for Empaveli will be presented at ASN, with strong efficacy across diverse patient groups.