Apellis Receives EMA Orphan Drug Designation for APL-2 in PNH

C3 inhibitor is in development for the treatment of PNH, both in patients not previously treated with eculizumab, and in patients who continue to experience hemolysis and require RBC transfusions despite receiving treatment with eculizumab.

Louisville, Ky. – Apellis Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company focused on inhibition of the complement system, announced today that the European Medicines Agency (EMA) has granted orphan medicinal product (“Orphan Drug”) designation to APL-2, a complement C3 inhibitor, in the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare, acquired, potentially life-threatening disease characterized by complement-mediated hemolytic anemia.

To qualify for EMA orphan designation, a sponsor must establish that the product is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting not more than 5 in 10,000 persons in the European Union (EU), and that there exists no satisfactory method of treatment of the condition that has been authorized in the EU or, if such method exists, that the product will be of significant benefit to those affected by the condition.

An orphan designation by EMA will allow Apellis to benefit from several incentives offered by the EU to companies developing medicines for rare diseases, including protocol assistance, access to the centralized authorization procedure, and market exclusivity once the medicine is on the market.

Cedric Francois, M.D., Ph.D., chief executive officer of Apellis, said: “This is another important milestone for the APL-2 program. We believe that APL-2 has the potential to offer an important, and hopefully improved, new treatment option for patients suffering with PNH. The granting of orphan designation by the EMA and the resulting incentives will benefit us, both in the near term, as we continue to advance our clinical programs, and in the longer term, as we prepare to bring APL-2 to market.”

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.

About APL-2

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.

About the Phase Ib Clinical Trials of APL-2 in PNH

Apellis is evaluating APL-2 in two Phase Ib clinical trials. PADDOCK (ClinicalTrials.gov Identifier: NCT02588833) is 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 who have not received the standard of care in the past. PHAROAH (ClinicalTrials.gov Identifier: NCT02264639) is 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 patients with PNH.

About Apellis

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.

 SOURCE Apellis Pharmaceuticals, Inc.