Top-line Data Anticipated in December 2019
CRESTWOOD, Ky. and WALTHAM Mass., June 27, 2019 (GLOBE NEWSWIRE) -- 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 the completion of enrollment for its PEGASUS Phase 3 trial assessing the safety and efficacy of APL-2 (pegcetacoplan) in patients with paroxysmal nocturnal hemoglobinuria (PNH) compared to eculizumab (Soliris™). The study enrolled 80 patients, approximately half of whom were transfusion dependent. Apellis expects to release top-line data in December 2019.
"We are committed to developing new treatment options to improve the care of patients with complement-mediated diseases such as PNH, and completing enrollment of the PEGASUS study moves us closer to this goal," said Cedric Francois, MD, PhD, co-founder and CEO of Apellis. "The PEGASUS trial is designed to show whether APL-2 can improve upon the current standard of care and address the prevalence of anemia and blood transfusions in treated PNH patients. We believe that the unique mode of action of C3 inhibition has the potential to provide patients with broad hematologic correction relative to the standard of care and a meaningful improvement in their quality of life."
PEGASUS (Study APL2-302) is a randomized, active-controlled study comparing APL-2 monotherapy to eculizumab monotherapy in patients currently on treatment with eculizumab who have a hemoglobin level <10.5 g/dL, regardless of eculizumab dose or transfusion history. In the trial, patients are co-treated for 4 weeks with APL-2 (via subcutaneous infusion administered twice weekly) and eculizumab (maintaining the pre-study dose level and regimen), and then randomized to either APL-2 monotherapy or eculizumab monotherapy for a 16-week randomized controlled period. The primary endpoint is change from baseline to week 16 in hemoglobin level. Key secondary endpoints include transfusion avoidance during the 16-week randomized controlled period, as well as week 16 changes from baseline in reticulocyte count, lactate dehydrogenase (LDH) levels and FACIT-Fatigue scale score. The 16-week randomized controlled period is followed by a 32‑week open-label treatment period in which all subjects will receive treatment with APL-2 monotherapy.
APL-2 is being developed for patients with PNH, autoimmune hemolytic anemia (AIHA), complement-associated kidney diseases, and geographic atrophy (GA). Cumulative systemic exposure is over 50 patient years of treatment on APL-2. There have been no serious adverse drug reactions in the systemic development programs for hematology indications to date.
About Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, potentially life-threatening disease characterized by complement-mediated hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes and/or some degree of bone marrow dysfunction. A significant number of patients treated with the current standard of care still suffer from debilitating anemia and transfusion dependence.
About APL-2 (pegcetacoplan)
APL-2, an investigational drug, is designed to inhibit the complement cascade centrally at C3 and may have the potential to treat a wide range of complement-mediated diseases more effectively than is possible with partial inhibitors of complement. APL-2 is a synthetic cyclic peptide conjugated to a polyethylene glycol (PEG) polymer that binds specifically to C3 and C3b, effectively blocking all three pathways of complement activation (classical, lectin, and alternative). Apellis is currently evaluating APL-2 in clinical studies in patients with GA, in patients with PNH who are being treated with eculizumab or who are naïve to complement inhibitor treatment, in patients with AIHA and in patients with C3G and other glomerular diseases. For additional information regarding our clinical trials, visit www.apellis.com/clinical-trials.html.
About APL-2 in Hematologic Diseases
Apellis is currently evaluating APL-2 in PEGASUS, a Phase 3 trial for patients with PNH currently on treatment with eculizumab, as well as in two Phase 1b trials (PHAROAH and PADDOCK) for systemic administration to patients with PNH. Previously reported interim data from these Phase 1b trials showed improvements in lactate dehydrogenase and hemoglobin levels in patients who are suboptimal responders to eculizumab and untreated patients, respectively. Apellis is also testing APL-2 in a Phase 2 open-label trial assessing the safety, tolerability, efficacy, and PK of multiple subcutaneous (SC) doses of APL-2 administered daily in patients with warm autoimmune hemolytic anemia (wAIHA) or cold agglutinin disease (CAD). In this trial to date, APL-2 has shown the potential to improve hemoglobin, reticulocytes, bilirubin and lactate dehydrogenase levels.
Apellis Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds for the treatment of a broad range of life-threatening or debilitating autoimmune diseases based upon complement immunotherapy through the inhibition of the complement system at the level of C3. Apellis is the first company to advance chronic therapy with a C3 inhibitor into clinical trials. For additional information about Apellis and APL-2, please visit http://www.apellis.com.
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the implications of preliminary clinical data. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: whether the Company's clinical trials will be fully enrolled and completed when anticipated; whether preliminary or interim results from a clinical trial will be predictive of the final results of the trial; whether results obtained in preclinical studies and clinical trials will be indicative of results that will be generated in future clinical trials; whether APL-2 will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of such clinical trials will warrant regulatory submissions and whether APL-2 will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, wAIHA or any other indication; whether, if Apellis' products receive approval, they will be successfully distributed and marketed; and other factors discussed in the "Risk Factors" section of Apellis' Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on May 7, 2019 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
Source: Apellis Pharmaceuticals, Inc.