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Apellis Pharmaceuticals Presents New Data from Ongoing APL-2 Phase 2 Study in Patients with Cold Agglutinin Disease and Warm Antibody Autoimmune Hemolytic Anemia at ASH

-  New data on APL-2 demonstrate improvements in hematological parameters in cold agglutinin disease (CAD) and warm antibody autoimmune hemolytic anemia (wAIHA)

-  CAD patients treated with APL-2 had an improvement in hemoglobin within weeks and at day 56 had a normalization of mean hemoglobin, reticulocytes, bilirubin and lactate dehydrogenase

-  wAIHA patients on APL-2 with C3 loading had a meaningful improvement in hemoglobin at day 56 along with a normalization of reticulocytes and lactate dehydogenase

CRESTWOOD, Ky. and WALTHAM, Mass., Dec. 03, 2018 (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 updated data from its Phase 2 study of APL-2 in patients with autoimmune hemolytic anemia (AIHA), including cold agglutinin disease (CAD) and warm antibody autoimmune hemolytic anemia (wAIHA). Data from the PLAUDIT trial will be presented in a poster session today at the 60th American Society of Hematology (ASH) Annual Meeting and Exposition, held in San Diego, California.

In the ongoing PLAUDIT study, 12 CAD patients have been enrolled on APL-2 subcutaneous treatment, of which 5 patients have been treated for at least 56 days. Nine patients with wAIHA were enrolled, seven of which were Direct Antiglobulin Test (DAT) C3+ (C3+ wAIHA). Five of these C3+ wAIHA patients have been on APL-2 for at least 56 days. 

“CAD and wAIHA are now the third and fourth indications, along with paroxysmal nocturnal hemoglobinuria (PNH) and geographic atrophy (GA), with high unmet medical need where APL-2 has demonstrated proof of concept,” said Dr. Cedric Francois, MD, PhD, Apellis co-founder and CEO. “These additional data confirm that the unique way APL-2 targets both C3 as well as C5 via the C5 convertase supports its potential across multiple complement-mediated diseases.  We are pleased that APL-2 demonstrates a clinically meaningful benefit in these two challenging diseases that each lack an FDA-approved therapy.”

Data will be presented by Dr. Morie A. Gertz, M.D., MACP, Chair Emeritus Department of Medicine of the Department of Hematology at the Mayo Clinic in Rochester, Minnesota. Professor Gertz is the primary investigator of the PLAUDIT study.

“APL-2 reduces both intravascular and extravascular hemolysis and in this study has shown a meaningful clinical benefit in both CAD and C3+ wAIHA patients. In addition to increasing hemoglobin in both CAD and C3+ wAIHA, treatment with APL-2 has shown reductions in reticulocytes, lactate dehydrogenase and bilirubin in both of these diseases,” noted Dr. Gertz. “Neither of these conditions has an FDA-approved therapy and these patients are in need of an effective treatment. The breadth of activity in these two distinct diseases is impressive. Importantly, APL-2 appears to be safe and well-tolerated in patients with AIHA.”

Poster Presentation 2: Inhibition of C3 with APL-2 Results in Normalization of Markers of Intravascular and Extravascular Hemolysis in Patients with Autoimmune Hemolytic Anemia (AIHA)
Session Name: 101. Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron: Poster III
Date: Monday, December 3, 2018
Presentation Time: 6:00 PM - 8:00 PM
Location: San Diego Convention Center, Hall GH

PLAUDIT is a phase 2, prospective, open-label study to assess the safety, tolerability, preliminary efficacy, pharmacokinetics and pharmacodynamics of multiple doses of APL-2 in patients with AIHA including warm antibody autoimmune hemolytic anemia (wAIHA) and cold agglutinin disease (CAD).

  • Cold Agglutinin Disease (CAD)

    • Mean Hb for CAD subjects increased from 8.7 g/dL (n=12) at baseline to 12.1 g/dL (n=5) at day 56, a 3.4 g/dL increase. Two subjects have reached day 168 and showed a sustained clinical benefit as represented by a mean Hb of 12.6 g/dL.

    • Mean absolute reticulocyte count (ARC) for CAD subjects decreased from 137.5 10⁹/L (n=11) at baseline to 46.6 10⁹/L (n=5) at day 56, with sustained clinical benefit as represented by a mean ARC of 70.8 10⁹/L (n=2) at day 168

    • Mean bilirubin for CAD subjects decreased from 1.9 mg/dL (n=12) at baseline to 0.5 mg/dL (n=5) at day 56

    • Mean LDH for CAD subjects decreased from 487.8 U/L (n=12) at baseline to 173.4 U/L (n=5) at day 56 with sustained benefit as represented by mean LDH of 143.5 U/L (n=2) at day 168

  • C3+ Warm Antibody Autoimmune Hemolytic Anemia (C3+ wAIHA)

    • Mean Hb for C3+ wAIHA subjects increased from 9.3 g/dL (n=7) at baseline to 11.3 g/dL (n=5) at day 56, a 2.0 g/dL increase. 

    • Mean absolute reticulocyte count for C3 + wAIHA subjects decreased from 185.5 10⁹/L (n=7) at baseline to 61.1 10⁹/L (n=5) at day 56 

    • Mean bilirubin for C3+ wAIHA subjects decreased from 0.8 mg/dL (n=7) at baseline to 0.4 mg/dL (n=6) at day 56

    • Mean LDH for C3 + wAIHA subjects decreased from 306.9 U/L (n=7)  at baseline to 150.2 U/L (n=5) at day 56

Cold Agglutinin Disease (CAD)
Cold Agglutinin Disease (CAD) is a severe, chronic rare autoimmune disorder caused by pathogenic Immunoglobulin M (IgM) antibodies that react with red blood cells at temperatures below 30oC and leads to agglutination of the red blood cells (RBCs). Agglutinated RBCs activate a part of the body’s immune system called the complement system leading to destruction of the RBC. The disease is often characterized by chronic anemia, severe fatigue, and an increased risk of life-threatening events such as stroke. There are an estimated 10,000 CAD patients across the United States and Europe. There are currently no approved therapies for CAD.

Warm autoimmune hemolytic anemia (wAIHA)
Warm autoimmune hemolytic anemia (wAIHA) is a rare autoimmune disorder 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 at normal body temperature. The disease is often characterized by profound, and potentially life-threatening anemia and other acute complications, including severe and life-threatening hemolysis, severe weakness, enlarged spleen or liver, rapid heart rate, chest pain, heart failure and fainting. There are estimated to be more than 30,000 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 wAIHA.

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. 

Clinical trials 
Specifically in hematologic diseases, Apellis is currently evaluating APL-2 in a Phase 2 clinical trial in autoimmune hemolytic anemia (AIHA) and a Phase 2 clinical trial in complement dependent nephropathies, as well as two Phase 1b trials (PHAROAH and PADDOCK) for systemic administration in paroxysmal nocturnal hemoglobinuria (PNH). Previously reported interim data from these trials showed improvements in lactate dehydrogenase and hemoglobin levels in patients who are suboptimal responders to eculizumab and untreated patients, respectively. The company is also conducting a Phase 3 trial for patients with PNH. For additional information regarding our clinical trials, please visit

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

Forward-Looking Statements  
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 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 such as the results reported in this release 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 United States Food and Drug Administration or equivalent foreign regulatory agencies for GA, PNH 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 November 13, 2018 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.

Media Contact:
Nicole Clifford  
617.482.0042  (office)
949.533.9295  (mobile)

Investor Contact:
Alex Kane
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