LOUISVILLE, Ky., Nov. 21, 2016 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company focused on inhibition of the complement system, announced today it will present data from Phase 1 clinical trials of APL-2 in paroxysmal nocturnal hemoglobinuria (PNH) at two scientific meetings in December in San Diego. PNH is a rare, acquired, potentially life-threatening disease characterized by complement-mediated hemolytic anemia. Unlike other complement inhibitors, APL-2 targets C3, blocking all three activation pathways of the complement system, which may provide a more effective treatment approach than existing therapies.
International PNH Interest Group (IPIG) Annual Scientific Assembly
Apellis will present new data from two Phase Ib open-label, dose-escalation clinical trials – a study assessing the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary efficacy of multiple doses of APL-2 administered by daily subcutaneous injection (SC) in patients with PNH, and a study assessing the safety, tolerability, PK and PD of single and multiple doses of APL-2 administered by SC as an add-on to standard of care in subjects with PNH.
Date: Friday, December 2, 2016
Time: 6 p.m. PT
Location: Manchester Grand Hyatt, San Diego
For more information about the IPIG Annual Scientific Assembly, visit http://www.pnhinterestgroup.org/meetings/professionals/international-pnh-interest-group-11th-annual-scientific-meeting.
American Society of Hematology (ASH) Annual Meeting
Apellis will present the following poster detailing data from Phase 1 clinical trials assessing the safety, tolerability, PK and PD of single and multiple doses of APL-2 administered by SC in healthy adult volunteers.
Title: APL-2, a Complement C3 Inhibitor for the Potential Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH): Phase I Data from Two Completed Studies in Healthy Volunteers
Abstract Number: 1251
Session: 101. Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron: Poster I
Date: Saturday, December 3, 2016
Time: 5:30-7:30 p.m. PT
Location: San Diego Convention Center, Hall GH
For more information about the ASH Annual Meeting, visit http://www.hematology.org/Annual-Meeting/.
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 subset of patients treated with the current standard of care still suffer from debilitating anemia and transfusion dependence.
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) with a particularly high potency against the alternative pathway. This comprehensive inhibition of complement-mediated pathology may have the potential to control symptoms and modify underlying disease in patients suffering from PNH.
Apellis is a clinical-stage biopharmaceutical company focused on the development of a platform of novel therapeutic compounds for the treatment of a broad range of autoimmune diseases based upon complement immunotherapy. Uncontrolled complement activation can lead to a wide range of life-threatening or debilitating disorders. Apellis is the first company to advance chronic therapy with a C3 inhibitor into clinical trials. Apellis is currently evaluating its lead product candidates in Phase 1 clinical trials in paroxysmal nocturnal hemoglobinuria (PNH) and in a Phase 2 clinical trial in geographic atrophy, the advanced form of dry age-related macular degeneration (AMD). For additional information about Apellis, please visit www.apellis.com.