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 interim data from its Phase
1b study of APL-2 in treatment-naïve patients with paroxysmal
nocturnal hemoglobinuria (PNH). Data from the PADDOCK trial were
presented in a poster session today at the 60th Annual Meeting of
the American Society of Hematology (ASH).
Interim results from the ongoing PADDOCK study evaluating 270mg
subcutaneous APL-2 administered daily are presented. Data are
presented for 19 patients at baseline, 15 patients on therapy at
day 85 and 10 patients at day 169.
“There remains a significant unmet need in PNH driven by
extravascular hemolysis, which is not addressed by C5 inhibitors
such as eculizumab,” said Dr. Cedric Francois, MD, PhD, Apellis
co-founder and CEO. “A recent large study showed that over
70% of PNH patients continue to be anemic and nearly 40% had at
least one transfusion in the prior year while on treatment with
eculizumab, the only approved therapy for PNH. 1 The study also
showed that nearly all patients on eculizumab have reticulocytosis
with an average of 1.9x the upper limit of normal (ULN). In
our PADDOCK study, patients achieved transfusion independence with
an average hemoglobin increase of 4.2 g/dL by day 85 to 12.2 g/dL
and average reticulocytes decrease of 50% from 2.0x ULN to 1.0x
ULN. LDH was reduced from 9.7x ULN at baseline to 0.9x at day 85
with 80% of patients achieving normal LDH. We could not be happier
with these results as they show APL-2 represents a promising
potential improvement in treatment options for PNH patients.”
These data will be presented by Dr. Raymond SM Wong of the
Department of Medicine & Therapeutics, Prince of Wales Hospital
at The Chinese University of Hong Kong. Professor Wong is a
principal investigator for the PADDOCK study.
“APL-2 shows meaningful improvement in hematological parameters
in ways not seen with standard of care C5 inhibition,” said Dr.
Raymond Wong. “The hemoglobin increases and transfusion avoidance
are highly meaningful, as is the broader hematological correction,
including reticulocytes and bilirubin. The safety is also promising
and the subcutaneous route of administration is friendlier to
patients.”
Poster Presentation 1: Inhibition of C3 with APL-2
Results in Normalization of Markers of Intravascular and
Extravascular Hemolysis in Patients with Paroxysmal Nocturnal
Hemoglobinuria (PNH) Session Name: 101. Red Cells and
Erythropoiesis, Structure and Function, Metabolism, and Survival,
Excluding Iron: Poster II Date: Sunday, December 2, 2018
Presentation Time: 6:00 PM - 8:00 PM Location: San Diego
Convention Center, Hall GH
PADDOCK is an open-label, dose-escalation trial designed to
assess the safety, tolerability, pharmacokinetics (PK),
pharmacodynamics (PD) and efficacy of multiple doses of APL-2, in
patients with PNH, a rare and serious condition affecting the bone
marrow. Data to be presented demonstrates:
- Hemoglobin: Broad control of hemolysis, both
intravascular and extravascular, led to significant and sustained
increases in hemoglobin in the absence of transfusions. The
baseline Hb was 8.0 g/dL (n=19) which increased to 10.8 g/dL (n=19)
and 12.2 g/dL (n=15) at Days 29 and 85, respectively. In the 12
months prior to screening, subjects received an average of 8.7
units pRBCs (range 0-28). Transfusion independence was achieved
while on APL-2 maintenance therapy with the exception of one
patient with severe aplastic anemia at Day 364
- Hemolysis Control: Systemic inhibition of C3
with APL-2 controls both intravascular and extravascular hemolysis
in PNH patients as demonstrated by rapid and durable normalization
of LDH, total bilirubin, and reticulocytes, all markers of
hemolysis
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 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 In hematologic diseases,
Apellis is currently evaluating APL-2 in 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. Apellis is
also testing APL-2 for systemic administration in a Phase 2
clinical trial in autoimmune hemolytic anemia (AIHA) and a Phase 2
clinical trial in complement dependent nephropathies, as well as a
Phase 3 trial for patients with PNH. For additional information
regarding our clinical trials,
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: Tully Nicholas
tnicholas@denterlein.com 617.482.0042 (office)
860.490.0218 (mobile)
Investor Contact: Alex Kane
akane@w2ogroup.com 212.301.7218 (office) 929.400.2691
(mobile)
1 McKinley CE, Richards SJ, Munir T, Griffin M,
Mitchell LD, Arnold L, Riley K, Copeland N, Newton DJ, Hill A,
Hillmen P. Extravascular hemolysis due to C3-loading in patients
with PNH treated with eculizumab: defining the clinical syndrome.
Blood. 2017:130:3471.
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