EX-99.1 2 d436376dex991.htm EX-99.1 EX-99.1

Slide 1

2022 was a transformational year Submitted NDA to U.S. FDA Submitted MAA to EMA U.S. commercial preparations complete; deployed ~100 field-based employees mid-2022 Appointed leading retina specialist, Dr. Caroline Baumal as Chief Medical Officer (effective Q1 2023) ~$65 million1 in U.S. net product sales Submitted sNDA for PRINCE & 48-week PEGASUS data Submitted sNDA for EMPAVELI injector Completed >800 patient years of systemic dosing2 Progressed late-stage clinical studies with partner Sobi Advanced early-stage pipeline across therapeutic areas Completed enrollment in Phase 2 ALS study Geographic Atrophy (GA): Ready to launch first-ever treatment Paroxysmal Nocturnal Hemoglobinuria (PNH): Continued growth in demand Pipeline: Leaders in complement 1 Estimated 2022 EMPAVELI U.S. net product sales are unaudited, preliminary and based on management’s estimate as of the date of this presentation and are subject to completion of the Company’s financial closing procedures. 2. Includes 694 patients as of November 2022 from all subcutaneous pegcetacoplan clinical trials and post-marketing exposure of EMPAVELI. Exhibit 99.1