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Apellis’ APL-2 Receives Orphan Drug Designation from the FDA for the Treatment of Autoimmune Hemolytic Anemia

CRESTWOOD, Ky., and WALTHAM, Mass., Feb. 05, 2019 (GLOBE NEWSWIRE) — Apellis Pharmaceuticals Inc. (Nasdaq:APLS), a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds to treat diseases through inhibition of the complement system, today announced that its C3 complement inhibitor, APL-2, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of autoimmune hemolytic anemia (AIHA).

AIHA refers to both cold agglutinin disease (CAD) and warm autoimmune hemolytic anemia (wAIHA.) Both rare autoimmune disorders are caused by pathogenic Immunoglobulin G (IgG) antibodies that react with RBC and can activate the complement system leading to the premature destruction of red blood cells. Both diseases are characterized by potentially life-threatening anemia and other acute complications. There are an estimated 10,000 CAD patients and 30,000 plus wAIHA patients across the United States and Europe. C3+ wAIHA has been estimated to represent as much as two thirds of the total wAIHA population. There are currently no approved treatments for CAD or wAIHA.

By targeting C3, the protein at the center of the entire complement system, Apellis believes APL-2 has the potential to eliminate the excessive and inappropriate C3 activation that is central to AIHA, thereby protecting the cells against complement-mediated intra- and extravascular destruction more completely than other approaches with more limited effects on the complement system.  Apellis is currently conducting a Phase 2 trial of APL-2 in and 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).

“This orphan drug designation represents an important recognition by the FDA of APL-2’s potential to treat AIHA, a rare disease that can lead to potentially life-threatening anemia and hemolysis, as well as other acute complications, and for which there are no approved treatments currently for patients,” said Federico Grossi, EVP of clinical development and medical affairs. “This is an important milestone as we build out our pipeline using the systemic administration of APL-2 in rare, complement-mediated diseases, and we look forward to seeing the outcomes of our Phase 2 study.”

The FDA Orphan Drug program provides orphan designation to novel drugs that are intended for the treatment of rare diseases (those affecting fewer than 200,000 people in the United States). The designation provides sponsors with development and commercial incentives including seven years of market exclusivity in the US, consultation by FDA on clinical study design, potential for expedited drug development, and certain fee exemptions and reductions.

About APL-2 
APL-2 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). To date, APL-2 has generally been well-tolerated. No significant infections have been observed in trials involving the systemic administration of APL-2, including the trials in PNH, AIHA or other trials. 

About Apellis
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 For additional information regarding our clinical trials, visit

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