- New data on APL-2 demonstrate
improvements in hematological parameters in cold agglutinin disease
(CAD) and warm antibody autoimmune hemolytic anemia (wAIHA)
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 www.apellis.com/clinical-trials.html.
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 http://www.apellis.com.
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
nclifford@denterlein.com 617.482.0042 (office)
949.533.9295 (mobile)
Investor Contact: Alex Kane
akane@w2ogroup.com 212.301.7218 (office) 929.400.2691
(mobile)
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