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 positive preliminary results
in the C3 glomerulopathy (C3G) cohort of the Phase 2 DISCOVERY
study. The data showed that APL-2 (pegcetacoplan), an
investigational C3-targeted inhibitor, may have the potential to
target the underlying disease process of C3G. Results through study
day 84 (12 weeks) were presented at the American Society of
Nephrology (ASN) Kidney Week 2019 in Washington D.C., and based on
these results, Apellis plans to advance development of APL-2 in
C3G.
In the data presented on six C3G patients treated
with APL-2, there was a downward trend in mean proteinuria as
measured by the urine protein-to-creatinine ratio (uPCR). Mean (SE)
uPCR decreased from 2.03 (0.46) mg/mg to 1.05 (0.23) mg/mg at study
day 84 (normal range <0.200 mg/mg), and a corresponding
normalization of mean serum albumin was observed. Serum C3 levels
also increased in all six of these patients. No serious adverse
events and no discontinuations due to adverse events were reported
in individuals with C3G.
“There is a substantial unmet need for medicines
that can prevent the kidney failure that often results from C3G,”
said independent expert Matthew Pickering, MB BS, PhD, Professor of
Rheumatology and Wellcome Trust Senior Fellow in Clinical Science
at Imperial College London. “This novel investigational therapy
showed a reduction in mean proteinuria of nearly 50% at day 84. The
study data provide early evidence that APL-2 may have the potential
to impact disease progression in people with C3G.”
There are no medicines currently approved for C3G,
a rare disease that leads to kidney failure within five to 10 years
of diagnosis in approximately 50% of people. In C3G, a part of the
immune system known as the complement cascade is overactive, which
results in the excessive breakdown of a protein called C3. These C3
breakdown products become trapped in the kidney, causing
inflammation and damage to the organ. Proteinuria, or loss of blood
proteins (e.g. albumin) in the urine, is a common finding in people
with C3G, and an indication that the disease is damaging the
kidneys.
“We’re encouraged by these early results, which
show that APL-2 may have the potential to address the underlying
disease process of C3G,” said Federico Grossi, MD, PhD, Chief
Medical Officer of Apellis. “We look forward to speaking with
regulators in the coming months about advancing clinical
development of APL-2 in C3G.”
Detailed Preliminary Results in C3G Cohort of
DISCOVERY Trial
The DISCOVERY trial is a Phase 2, open-label study
investigating the safety and biological activity of APL-2 in four
renal diseases: C3G, immunoglobulin A (IgA) nephropathy, primary
membranous nephropathy (PMN), and lupus nephritis (LN). The primary
endpoint is change from baseline in proteinuria at week 48 as
quantified by uPCR. Secondary endpoints include analysis of serum
C3 and estimated glomerular filtration rate (eGFR).
Preliminary data presented at Kidney Week 2019
included safety and demographic data from all eight patients in the
C3G cohort through study day 84, and data on renal parameters and
complement levels in the six C3G patients who received consistent
APL-2 administration from study day 1 to study day 84. In those six
patients, the percentage reduction in mean uPCR was 48.23% (11.22),
and mean serum albumin normalized from baseline, 3.30 (0.32) g/dL,
(normal range 3.50-5.50 g/dL) to study day 84, 3.98 (0.20) g/dL.
All six of these patients also had an increase in serum C3 levels
following APL-2 administration, and mean plasma C5b-9 levels had
decreased by day 28 of APL-2 treatment. Mean serum creatinine and
eGFR, markers of kidney function, were stable through study day
84.
About APL-2
(pegcetacoplan) APL-2, an investigational drug, is
designed to inhibit the complement cascade at C3 and may have the
potential to treat a wide range of complement-mediated diseases.
APL-2 is a synthetic cyclic peptide conjugated to a polyethylene
glycol (PEG) polymer that binds specifically to C3 and C3b. Apellis
is evaluating APL-2 in clinical studies in several serious
diseases, including paroxysmal nocturnal hemoglobinuria (PNH),
geographic atrophy (GA), cold agglutinin disease (CAD) and
C3G. 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.
Dr. Pickering is providing scientific advice to
Apellis in his capacity as an international expert on C3
glomerulopathy, independently, via Imperial Consultants.
Apellis Forward-Looking
Statement
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, C3G 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
5, 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.
Media Contact:Tracy
Vineistracy.vineis@apellis.com617.420.4839
Investor Contact: Sam Martin /
Maghan MeyersArgot Partners sam@argotpartners.com /
maghan@argotpartners.com212.600.1902
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