Apellis Updates Data from Study of Hemoglobinuria at ASH

• Treatment with APL-2 in eculizumab-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH) resulted in broad control of hemolysis and normalization of mean hemoglobin to 12.2 g/dL by day 85, an increase of 4.2 g/dL from baseline
• Previously transfusion-dependent patients did not require any transfusions during maintenance treatment with APL-2
• Rapid and durable normalization Lactate Dehydrogenase, Reticulocyte Count and Total Bilirubin was achieved

Apellis Pharmaceuticals Inc. (Nasdaq:APLS), a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds to treat disease through the inhibition of the complement system, today announced interim data from its Phase 1b study of APL-2 in treatment-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH). Data from the PADDOCK trial were presented in a poster session today at the 60^th Annual Meeting of the American Society of Hematology (ASH).

Interim results from the ongoing PADDOCK study evaluating 270mg subcutaneous APL-2 administered daily are presented. Data are presented for 19 patients at baseline, 15 patients on therapy at day 85 and 10 patients at day 169.

“There remains a significant unmet need in PNH driven by extravascular hemolysis, which is not addressed by C5 inhibitors such as eculizumab,” said Dr. Cedric Francois, MD, PhD, Apellis co-founder and CEO.  “A recent large study showed that over 70% of PNH patients continue to be anemic and nearly 40% had at least one transfusion in the prior year while on treatment with eculizumab, the only approved therapy for PNH. ¹ The study also showed that nearly all patients on eculizumab have reticulocytosis with an average of 1.9x the upper limit of normal (ULN).  In our PADDOCK study, patients achieved transfusion independence with an average hemoglobin increase of 4.2 g/dL by day 85 to 12.2 g/dL and average reticulocytes decrease of 50% from 2.0x ULN to 1.0x ULN. LDH was reduced from 9.7x ULN at baseline to 0.9x at day 85 with 80% of patients achieving normal LDH. We could not be happier with these results as they show APL-2 represents a promising potential improvement in treatment options for PNH patients.”

These data will be presented by Dr. Raymond SM Wong of the Department of Medicine & Therapeutics, Prince of Wales Hospital at The Chinese University of Hong Kong. Professor Wong is a principal investigator for the PADDOCK study.

“APL-2 shows meaningful improvement in hematological parameters in ways not seen with standard of care C5 inhibition,” said Dr. Raymond Wong. “The hemoglobin increases and transfusion avoidance are highly meaningful, as is the broader hematological correction, including reticulocytes and bilirubin. The safety is also promising and the subcutaneous route of administration is friendlier to patients.”

https://www.nasdaq.com/press-release/apellis-pharmaceuticals-presents-updated-data-from-a-clinical-study-of-apl2-in-patients-with-20181202-00024