- Generated $5.3 million in EMPAVELI™ (pegcetacoplan) net product revenues
- Presented Phase 3 DERBY and OAKS results in geographic atrophy (GA); on track for NDA submission in 1H 2022
- Received positive CHMP opinion for pegcetacoplan for treatment of PNH; expect decision regarding approval by European Commission by end of 2021
- Conference call scheduled today at 4:30 p.m. ET
WALTHAM, Mass., Nov. 08, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in complement, today announced its third quarter 2021 financial results and business highlights.
“The third quarter was another exceptional period for Apellis, highlighted by successful commercial execution with EMPAVELI in PNH, the Phase 3 DERBY and OAKS results in geographic atrophy, and continued momentum advancing our broader pipeline,” said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis. “Our commercial launch of EMPAVELI is off to a strong start in the U.S. and we expect a decision regarding approval by the European Commission by the end of this year as we seek to elevate the standard of care in PNH worldwide.”
Dr. Francois continued, “With the DERBY and OAKS data, intravitreal pegcetacoplan has the potential to become the first treatment for patients living with geographic atrophy, a leading cause of blindness worldwide. We believe we have a robust data package that supports approval and remain on track to submit our New Drug Application to the FDA in the first half of 2022. Behind our lead programs, we are continuing to progress our broad pipeline addressing complement-driven diseases in rare disease, neurology, and ophthalmology.”
Third Quarter 2021 Business Highlights and Upcoming Milestones:
Paroxysmal Nocturnal Hemoglobinuria (PNH) Commercial Progress
- Apellis recorded $5.3 million in EMPAVELI™ (pegcetacoplan) net product revenue for the third quarter of 2021.
- In October 2021, Apellis and Sobi announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) had adopted a positive opinion recommending the marketing authorization of systemic pegcetacoplan for the treatment of adults with PNH who are anemic after treatment with a C5 inhibitor for at least three months.
- A decision regarding approval is expected by the European Commission by the end of 2021.
- Apellis is eligible for a $50.0 million milestone payment from Sobi upon first regulatory and reimbursement approval. If systemic pegcetacoplan is approved by the EMA in 2021, Apellis expects to achieve this milestone in the first half of 2022.
Rare Disease R&D Highlights
- Immune complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G): Apellis expects to initiate a Phase 3 study in the fourth quarter of 2021.
- Cold agglutinin disease (CAD): Sobi plans to initiate a Phase 3 study in the fourth quarter of 2021.
- Hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA): Sobi plans to initiate a potentially registrational program in the fourth quarter of 2021.
- Amyotrophic lateral sclerosis (ALS): Apellis now expects to complete enrollment for its ongoing and potentially registrational Phase 2 MERIDIAN study in the first half of 2022 (previously end of 2021). Enrollment is slower than expected due to the COVID-19 pandemic and competing ongoing trials recruiting in ALS.
- EMPAVELI + small interfering RNA (siRNA): Apellis plans to study the combination of EMPAVELI and an siRNA, which may offer the potential to reduce the treatment frequency of EMPAVELI. Apellis expects to submit an Investigational New Drug (IND) application in the first half of 2023.
Ophthalmology R&D Highlights
- Geographic atrophy:
- In September 2021, Apellis reported top-line results from the Phase 3 DERBY and OAKS studies evaluating intravitreal pegcetacoplan, an investigational targeted C3 therapy, in 1,258 adults with geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
- OAKS met the primary endpoint for both monthly and every-other-month treatment with pegcetacoplan, demonstrating a significant reduction in GA lesion growth of 22% (p=0.0003) and 16% (p=0.0052), respectively, compared to pooled sham at 12 months.
- DERBY narrowly missed the primary endpoint of GA lesion growth, showing a reduction of 12% (p=0.0528) and 11% (p=0.0750) with monthly and every-other-month treatment, respectively, compared to pooled sham at 12 months.
- In a prespecified analysis of the combined studies, pegcetacoplan decreased GA lesion growth in patients with extrafoveal lesions at baseline by 26% (p
- In September 2021, Apellis reported top-line results from the Phase 3 DERBY and OAKS studies evaluating intravitreal pegcetacoplan, an investigational targeted C3 therapy, in 1,258 adults with geographic atrophy (GA) secondary to age-related macular degeneration (AMD).