Apellis Pharmaceuticals Presents Data from Ongoing APL-2 Phase 2 Study in Patients with Cold Agglutinin Disease and Warm Antibody Autoimmune Hemolytic Anemia at 24th European Hematology Association (EHA) Congress


CRESTWOOD, Ky. and WALTHAM Mass., June 15, 2019 (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 PLAUDIT 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 an oral presentation today at the 24th Annual Congress of the European Hematology Association (EHA), held in Amsterdam, the Netherlands.

In the ongoing PLAUDIT study, 13 patients with CAD have been enrolled to receive subcutaneous APL-2 treatment, of which 10 patients have been on APL-2 for at least 168 days. The trial has also enrolled 11 patients with wAIHA, 8 of which were Direct Antiglobulin Test (DAT) C3+ (C3+ wAIHA); 5 of the C3+ wAIHA patients have been on APL-2 for at least 168 days. 

“Patients with autoimmune hemolytic anemias on APL-2 have shown improvement both in important hematologic measures and in quality of life scores, particularly in patients with CAD,” said Federico Grossi, CMO, Apellis. “These data provide a clear path to move forward with a Phase 3 trial of APL-2 in patients with CAD, which we anticipate commencing in early 2020. We have also seen hematologic improvements in patients with wAIHA receiving treatment with APL-2 when there is significant involvement of the complement pathway. Apellis will continue to evaluate the potential of APL-2 for patients with C3+ wAIHA.”

Data will be presented by Bruno Fattizzo, Consultant Hematologist at Fondazione IRCCS Ca' Granda Policlinico Hospital, Milan, Italy. The presentation will be made available through the “Our Scientific Publications & Presentations” page of the Apellis website at:

“This trial demonstrates that APL-2 has the potential to significantly help patients with autoimmune hemolytic anemias when there is complement involvement,” said Dr. Fattizzo.  “APL-2 represents a promising potential therapy for these patients, the majority of whom have no access to approved treatments.”

Oral Presentation: Inhibition of C3 with APL-2 controls haemolysis and increases haemoglobin levels in subjects with autoimmune haemolytic anaemia (AIHA)  
Session Name: Hemolytic anemias: From diagnostics to treatment  
Date: Saturday, June 15
Presentation Time: 4:00 – 4:15 PM CEST
Location: RAI Amsterdam at Europaplein 24, Amsterdam, the Netherlands, Hall G102

Cold Agglutinin Disease (CAD)

Of the 10 patients who reached Day 168:

  • 70% showed a Hb increase of ≥2 g/dL, 40% had normalized Hb (≥ 12.0 g/dL) and 80% had Hb ≥11.0 g/dL at Day 168.
  • Mean Hb increased from 8.9 g/dL at baseline to 11.2 g/dL at Day 168, a 2.4 g/dL increase (normal Hb is 12-16 g/dL).
  • Mean Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Score increased from 29.4 at baseline to 39.1 at Day 168, an improvement of 9.7 points, where a clinically significant increase is 3 or more points.1
  • Mean absolute reticulocyte count (ARC) decreased from 159 X 10⁹/L at baseline to 64 X 10⁹/L at Day 168 (normal ARC is 30-100 X 10⁹/L).
  • Mean indirect bilirubin decreased from 1.6 mg/dL at baseline to 0.4 mg/dL at Day 168 (normal indirect bilirubin is 0.1-0.75 mg/dL).
  • Mean LDH decreased from 500 U/L at baseline to 183 U/L at Day 168 (normal LDH is 87-252 U/L).

Two heavily transfusion dependent patients did not respond to APL-2 and left the study at Day 56 and 108 respectively. The remaining transfusion dependent patients (n=4) did not require any transfusions during maintenance treatment with APL-2 (i.e. after one week of starting APL-2 therapy). One previously non-transfusion dependent patient received an on-study transfusion prior to APL-2 steady-state (within one week of commencement of dosing). One patient is still participating in the study and has not yet reached Day 168.

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

Of the 5 patients with C3+ wAIHA who reached Day 168:

  • Mean Hb increased from 9.0 g/dL at baseline to 11.0 g/dL at Day 168, a 2.0 g/dL increase (normal Hb is 12-16 g/dL).
  • Mean FACIT Fatigue Score increased from 38.4 at baseline to 40.8 at Day 168, an improvement of 2.4 points.
  • Mean ARC decreased from 213 X 10⁹/L at baseline to 92 X 10⁹/L at Day 168 (normal ARC is 30-100 X 10⁹/L).
  • Mean indirect bilirubin decreased from 0.8 mg/dL at baseline to 0.3 mg/dL at Day 168 (normal indirect bilirubin is 0.1-0.75 mg/dL).
  • Mean LDH decreased from 241 U/L at baseline to 142 U/L at Day 168 (normal LDH is 87-252 U/L).

Two of the eight enrolled C3+ wAIHA patients left the study due to lack of response, and one of the eight enrolled C3+ wAIHA subjects is still participating in the study and has not yet reached Day 168. The three enrolled patients who were not DAT C3+ did not show a meaningful response and two have left the study.

In both the CAD and C3+ wAIHA populations, APL-2 was generally well tolerated; no serious adverse events related to APL-2 were reported in the PLAUDIT trial.

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 (RBCs) at temperatures below 30oC and leads to agglutination of the RBCs. Agglutinated RBCs activate a part of the body’s immune system called the complement system leading to destruction of the RBCs. 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 RBCs and can activate the complement system leading to the premature destruction of RBCs 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, an investigational drug, 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). Apellis is currently evaluating APL-2 in clinical studies in patients with geographic atrophy, in patients with paroxysmal nocturnal hemoglobinuria (PNH) who are being treated with eculizumab or who are naïve to complement inhibitor treatment, in patients with AIHA and in patients with C3G and other glomerular diseases. For additional information regarding our clinical trials, visit

About APL-2 in Hematologic Diseases
Apellis is currently evaluating APL-2 in PEGASUS, a Phase 3 trial for patients with PNH as well as in two Phase 1b trials (PHAROAH and PADDOCK) for systemic administration. Previously reported interim data from these Phase 1b trials showed improvements in lactate dehydrogenase and hemoglobin levels in patients who are suboptimal responders to eculizumab and untreated patients, respectively. Apellis is also testing APL-2 in a Phase 2 open-label trial assessing the safety, tolerability, efficacy, and PK of multiple subcutaneous doses of APL-2 administered daily in patients with wAIHA or CAD.  In this trial to date, APL-2 has shown the potential to improve hemoglobin, reticulocytes, bilirubin and lactate dehydrogenase levels.

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 the Company’s clinical trials will be fully enrolled and completed when anticipated; 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 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 FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, wAIHA 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 May 7, 2019 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.

1D. Cella et al. “Combining Anchor and Distribution-Based Methods to Derive Minimal Clinically Important Differences on the Functional Assessment of Cancer Therapy (FACT) Anemia and Fatigue Scales” Journal of Pain and Symptom Management Vol. 24 No. 6 December 2002

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