Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) announced today new data demonstrating that Empaveli™/Aspaveli® (pegcetacoplan) provides
Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) announced today new data demonstrating that Empaveli™/Aspaveli® (pegcetacoplan) provides consistent, sustained normalization of clinical measures across patients with paroxysmal nocturnal hemoglobinuria (PNH) who are treatment-naïve and patients with baseline hemoglobin levels greater than or equal to 10.0 g/dL. The data were presented at the American Society of Hematology Annual Meeting (ASH) taking place December 11 – 14, 2021.
Sustained Normalization and Superior Improvements of Clinical Measures in Treatment-Naïve Patients
New data from the Phase 3 PRINCE study in treatment-naïve patients with PNH showed that treatment with EMPAVELI resulted in sustained and superior improvements in the co-primary endpoints of hemoglobin stabilization through Week 26 and reduction in lactate dehydrogenase (LDH) compared to standard of care, which did not include complement inhibitors, at Week 26.
Improvements were seen as early as two weeks after starting treatment with EMPAVELI and patients showed sustained normalization across key markers of disease through Week 26:
- 46% of EMPAVELI patients achieved hemoglobin normalization in the absence of transfusions vs. 0% for standard of care (p<0.0010), reaching a mean hemoglobin level of 12.8 g/dL from a mean baseline of 9.4 g/dL
- Mean LDH levels rapidly fell from 9.5x the upper limit of normal (ULN) to below 1.5x the ULN by Week 2, normalized by Week 4, and were maintained through Week 26 with EMPAVELI
- 91% of EMPAVELI patients achieved transfusion avoidance vs. 6% for standard of care (p<0.0001), demonstrating superiority
The safety profile of EMPAVELI was consistent with previous studies. At Week 26, 9% of patients in the EMPAVELI group experienced a serious adverse event (SAE) compared to 17% on standard of care. No cases of thrombosis or meningococcal infection were reported in either group. The most common adverse events reported during the study in the EMPAVELI and standard of care groups, respectively, were injection site reaction (30% vs. 0%), hypokalemia (13% vs.11%), dizziness (11% vs. 0%), and fever (9% vs. 0%).
"The data presented at ASH add to a robust body of evidence that underscores the consistent efficacy and safety of EMPAVELI across a broad range of adults with PNH," said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. "EMPAVELI has the potential to elevate the standard of care for adults with PNH regardless of prior treatment or baseline hemoglobin levels."
Clinically Meaningful Improvements in Patients with Near-Normal Baseline Hemoglobin Levels
A new post hoc analysis across studies from the EMPAVELI PNH clinical development program showed that EMPAVELI-treated patients with baseline hemoglobin levels greater than or equal to 10.0 g/dL demonstrated clinically meaningful improvements across key markers of disease. The analysis included data from patients who were treatment-naïve and patients that remained anemic despite stable treatment with eculizumab, a C5 inhibitor.
Detailed data showed:
- EMPAVELI increased mean hemoglobin levels to 13.9 g/dL, 12.1 g/dL, and 12.7 g/dL from a mean baseline of 11.3 g/dL, 10.2 g/dL, and 10.4 g/dL in the PRINCE, PEGASUS, and PADDOCK studies, respectively
- EMPAVELI demonstrated mean improvements from baseline in the Functional Assessment of Chronic Illness Therapy (FACIT)-fatigue score of 9.9 points, 13.3 points, and 6.6 points in the PRINCE, PEGASUS, and PADDOCK studies. A three-point improvement is generally considered to be clinically meaningful
"The presented data reinforce the efficacy and safety profile of pegcetacoplan in PNH," said Ravi Rao, head of research and development and chief medical officer at Sobi. "We are committed to improve care, and to make a difference in the lives of people with this rare blood disease."