- Received approval for EMPAVELI™ (pegcetacoplan) – the first targeted C3 therapy – from U.S. Food and Drug Administration (FDA) for treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) and launched in U.S.
- Reported positive top-line results from Phase 3 PRINCE study of EMPAVELI in treatment-naïve patients with PNH
- Established exclusive research collaboration with Beam Therapeutics to apply base editing to discover novel therapies for complement-driven diseases
- Expect top-line results from Phase 3 geographic atrophy (GA) studies in September 2021
- Conference call scheduled today at 4:30 p.m. ET
WALTHAM, Mass., Aug. 09, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced its second quarter 2021 financial results and business highlights.
“The second quarter was an exceptional period for Apellis, underscored by the approval of EMPAVELI –– the first FDA-approved targeted C3 therapy –– for adults with PNH and a strong start to our U.S. commercial launch. We and our partner Sobi were also thrilled to report positive data from our Phase 3 PRINCE study in treatment-naïve PNH patients, emphasizing the potential for EMPAVELI to redefine treatment for all adults with PNH,” said Cedric Francois M.D., Ph.D., co-founder and chief executive officer of Apellis. “We are looking forward to the Phase 3 DERBY and OAKS readouts in September. GA is a leading cause of blindness worldwide, and we are excited about the potential to bring the first treatment to the millions of patients affected by this disease.”
“In addition to the broad platform potential of our targeted C3 therapy, our world-class research team is advancing multiple new molecular entities across several modalities, and we entered an exclusive collaboration with Beam Therapeutics to discover transformative therapies using its base editing technology. With our expanded pipeline across rare disease, ophthalmology, and neurology, we believe we are well positioned for long-term, global leadership in complement,” Dr. Francois continued.
Second Quarter 2021 Business Highlights and Upcoming Milestones:
Rare Disease
- In May 2021, Apellis announced that the FDA approved EMPAVELI for the treatment of adults with PNH including patients who are treatment naïve, as well as patients switching from the C5 inhibitors Soliris® (eculizumab) and Ultomiris® (ravulizumab).
- EMPAVELI is available commercially in the United States. Apellis recorded $0.6 million in net product revenue for the second quarter of 2021, which reflects recorded sales of EMPAVELI upon approval by the FDA in May 2021 through June 30, 2021. Apellis did not have any net product revenue in the quarter ended June 30, 2020.
- A decision on the Marketing Authorization Application is expected by the European Commission in the second half of 2021.
- In May 2021, Apellis and Sobi announced positive top-line results from the Phase 3 PRINCE study evaluating the efficacy and safety of EMPAVELI in adults with PNH who are treatment naïve. EMPAVELI demonstrated statistical superiority on the co-primary endpoints of hemoglobin stabilization (p