Results from positive Phase 3 PRINCE study in treatment-naive patients with PNH accepted for oral presentation
In new analysis, EMPAVELI demonstrated clinically meaningful improvements in key markers of disease in PNH patients with baseline hemoglobin levels greater than or equal to 10.0 g/dL
WALTHAM, Mass., Nov. 04, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. /zigman2/quotes/202103251/composite APLS -4.21% , a global biopharmaceutical company and leader in complement, today announced that five abstracts were accepted for presentation at the American Society of Hematology (ASH) Annual Meeting to be held December 11-14, 2021. The new data further support the positive efficacy and safety profile of EMPAVELI, the first and only targeted C3 therapy, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
Highlights include the first oral presentation of the positive Phase 3 PRINCE study of EMPAVELI in adults with PNH who are treatment-naive, meaning they had not received a complement inhibitor within three months before entering the study. As previously reported, EMPAVELI demonstrated superiority in both hemoglobin stabilization and reduction in lactate dehydrogenase (LDH) compared to standard of care, which did not include complement inhibitors, at Week 26.
Additionally, in a new post hoc analysis of the PRINCE, PEGASUS, and PADDOCK studies, EMPAVELI demonstrated clinically meaningful improvements in key markers of disease in PNH patients with pre-study hemoglobin levels greater than or equal to 10.0 g/dL. This highlights the potential of EMPAVELI to elevate the standard of care for all patients with PNH regardless of previous hemoglobin levels. The analysis included treatment-naive patients and patients who were taking eculizumab, a C5 inhibitor.
"The data that we are presenting at ASH underscore our leadership in PNH as well as the potential of EMPAVELI to redefine treatment for all adults with this debilitating disease," said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. "EMPAVELI demonstrated superiority to eculizumab in improving hemoglobin levels in PNH in our PEGASUS study and further evidence shows that EMPAVELI provides meaningful improvements in the treatment-naive patient population."
Accepted abstracts at ASH 2021 include:
Oral presentation: Efficacy and Safety of Pegcetacoplan Treatment in Complement-Inhibitor Naive Patients with Paroxysmal Nocturnal Hemoglobinuria: Results from the Phase 3 Prince Study - #606 - December 13, 11:45 AM ET, Georgia World Congress Center, Georgia Ballroom 1-3, in collaboration with Sobi
Poster presentation: Post Hoc Analysis of the Effect of Pegcetacoplan Treatment of Patients with Paroxysmal Nocturnal Hemoglobinuria and Baseline Hemoglobin Levels Greater Than 10 Grams per Deciliter - #2194 - December 12, 6:00 PM - 8:00 PM ET, in collaboration with Sobi
Poster presentation: Evaluation of the Long-Term Safety and Efficacy of Pegcetacoplan Treatment for Paroxysmal Nocturnal Hemoglobinuria Patients: An Extension Study - #2175 - December 12, 6:00 PM - 8:00 PM ET, in collaboration with Sobi
Poster presentation: Categorized Hematologic Response to Pegcetacoplan and Correlations with Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria: Post Hoc Analysis of Data from Phase 1b, Phase 2a, and Phase 3 Trials - #1104 - December 11, 5:30 PM - 7:30 PM ET
Poster presentation: Changes in Hemoglobin Measures Observed in PNH Patients Treated with Both C5 Inhibitors Ravulizumab and Eculizumab: Real-World Evidence from a US-Based EMR Network - #1112 - December 11, 5:30 PM - 7:30 PM ET
About the PRINCE Study
The PRINCE study (NCT04085601) is a randomized, multi-center, open-label, controlled Phase 3 study in 53 treatment-naive adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of EMPAVELI(TM) (pegcetacoplan) in patients who have not received treatment with any complement inhibitor within three months prior to screening. During the 26-week randomized, controlled period, patients received either 1080 mg of EMPAVELI twice weekly or standard of care therapy, which did not include complement inhibitors. Patients in the standard of care group had the option to receive EMPAVELI as escape therapy if their hemoglobin decreased by 2 g/dL or more from their baseline value.
About the PEGASUS Study
The PEGASUS study (NCT03500549) is a multi-center, randomized, head-to-head Phase 3 study in 80 adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of EMPAVELI compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a hemoglobin level of <10.5 g/dL at the screening visit. During the four-week run-in, patients were dosed with 1080 mg of EMPAVELI twice weekly (n=41) in addition to their current dose of eculizumab. During the 16-week randomized, controlled period, patients were randomized to receive either 1080 mg of EMPAVELI twice weekly or their current dose of eculizumab (n=39). All participants completing the randomized controlled period (n=77) opted to enter the open-label EMPAVELI treatment period.
About the PADDOCK Study
The PADDOCK study (NCT02588833) is a multicenter, open-label, multiple ascending dose, Phase 1b study in 23 adults with paroxysmal nocturnal hemoglobinuria (PNH) who have never received eculizumab. The primary objective of this study, designed with two cohorts, was to establish the safety and efficacy of 270 mg of EMPAVELI administered daily by subcutaneous injection in adults with PNH. Patients in Cohort One received a suboptimal dose of 180 mg of EMPAVELI once daily for 28 days and subjects in Cohort Two received 270 mg of EMPAVELI once daily for up to one year.
About EMPAVELI(TM) (pegcetacoplan)
EMPAVELI(TM) (pegcetacoplan) is the first and only approved therapy targeting C3, the central protein in the complement cascade. EMPAVELI acts proximally in the complement cascade controlling both C3b-mediated extravascular hemolysis and terminal complement-mediated intravascular hemolysis. EMPAVELI is approved in the United States for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH).
U.S. Important Safety Information for EMPAVELI
BOXED WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA
Meningococcal infections may occur in patients treated with EMPAVELI and may become rapidly life-threatening or fatal if not recognized and treated early. Use of EMPAVELI may predispose individuals to serious infections, especially those caused by encapsulated bacteria, such asStreptococcus pneumoniae,Neisseria meningitidistypes A, C, W, Y, and B, andHaemophilus influenzaetype B.
Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria.
Vaccinate patients at least 2 weeks prior to administering the first dose of EMPAVELI unless the risks of delaying therapy with EMPAVELI outweigh the risk of developing a serious infection.
Vaccination reduces, but does not eliminate, the risk of serious infections. Monitor patients for early signs of serious infections and evaluate immediately if infection is suspected.
EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the EMPAVELI REMS, prescribers must enroll in the program.
Hypersensitivity to pegcetacoplan or to any of the excipients
Not currently vaccinated against certain encapsulated bacteria, unless the risks of delaying EMPAVELI treatment outweigh the risks of developing a bacterial infection with an encapsulated organism
Unresolved serious infection caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae
WARNINGS AND PRECAUTIONS
Serious Infections Caused by Encapsulated Bacteria
The use of EMPAVELI may predispose individuals to serious, life-threatening, or fatal infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis types A, C, W, Y, and B, and Haemophilus influenzae type B (Hib). To reduce the risk of infection, all patients must be vaccinated against these bacteria according to the most current ACIP recommendations for patients with altered immunocompetence associated with complement deficiencies. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with EMPAVELI.
For patients without known history of vaccination, administer required vaccines at least 2 weeks prior to receiving the first dose of EMPAVELI. If immediate therapy with EMPAVELI is indicated, administer required vaccine as soon as possible and provide patients with 2 weeks of antibacterial drug prophylaxis.
Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected. Promptly treat known infections. Serious infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider discontinuation of EMPAVELI in patients who are undergoing treatment for serious infections.
Because of the risk of serious infections, EMPAVELI is available only through a restricted program under a REMS. Under the EMPAVELI REMS, prescribers must enroll in the program and must counsel patients about the risk of serious infection, provide the patients with the REMS educational materials, and ensure patients are vaccinated against encapsulated bacteria. Enrollment and additional information are available by telephone: 1-888-343-7073 or at www.empavelirems.com .
Systemic hypersensitivity reactions (e.g., facial swelling, rash, urticaria) have occurred in patients treated with EMPAVELI. One patient (less than 1% in clinical studies) experienced a serious allergic reaction which resolved after treatment with antihistamines. If a severe hypersensitivity reaction (including anaphylaxis) occurs, discontinue EMPAVELI infusion immediately, institute appropriate treatment, per standard of care, and monitor until signs and symptoms are resolved.
Monitoring PNH Manifestations after Discontinuation of EMPAVELI
After discontinuing treatment with EMPAVELI, closely monitor for signs and symptoms of hemolysis, identified by elevated LDH levels along with sudden decrease in PNH clone size or hemoglobin, or reappearance of symptoms such as fatigue, hemoglobinuria, abdominal pain, dyspnea, major adverse vascular events (including thrombosis), dysphagia, or erectile dysfunction. Monitor any patient who discontinues EMPAVELI for at least 8 weeks to detect hemolysis and other reactions. If hemolysis, including elevated LDH, occurs after discontinuation of EMPAVELI, consider restarting treatment with EMPAVELI.
Interference with Laboratory Tests
There may be interference between silica reagents in coagulation panels and EMPAVELI that results in artificially prolonged activated partial thromboplastin time (aPTT); therefore, avoid the use of silica reagents in coagulation panels.
The most common adverse reactions (incidence greater-than or equal to10% of patients) with EMPAVELI vs. eculizumab were injection-site reactions (39% v. 5%), infections (29% v. 26%), diarrhea (22% v. 3%), abdominal pain (20% v. 10%), respiratory tract infection (15% v. 13%), viral infection (12% v. 8%), and fatigue (12% v. 23%).
USE IN SPECIFIC POPULATIONS
Females of Reproductive Potential
EMPAVELI may cause embryo-fetal harm when administered to pregnant women. Pregnancy testing is recommended for females of reproductive potential prior to treatment with EMPAVELI. Advise female patients of reproductive potential to use effective contraception during treatment with EMPAVELI and for 40 days after the last dose.
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, hemoglobinuria and difficulty breathing (dyspnea).
About the Apellis and Sobi Collaboration
Apellis and Sobi collaborate to develop and commercialize systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan. Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and retains worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy (GA). The companies have global co-development rights for systemic pegcetacoplan.
Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com .
Apellis Forward-Looking Statement
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 in respect of the expected closing of the exchanges. 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 results of the company's clinical trials will warrant regulatory submissions and other factors discussed in the "Risk Factors" section of Apellis' Quarterly Report on Form 10-Q with the Securities and Exchange Commission on August 9, 2021 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.
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