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 that it has commenced its
Phase 3 clinical program for APL-2 in patients with geographic
atrophy (GA), consisting of two Phase 3 trials (DERBY & OAKS.)
The first patient has been dosed in the OAKS trial and the first
patient has been enrolled in the DERBY trial. Both trials will
assess the safety and efficacy of APL-2 in patients with GA, an
advanced form of age-related macular degeneration. There are
approximately 1 million patients in the United States with GA and
there are currently no FDA-approved treatments for the disease,
which leads to loss of visual function and blindness.
“Dosing the first patient in our Phase 3 program
in geographic atrophy is an exciting milestone for Apellis,
physicians, and most importantly patients suffering from GA,”
said Cedric Francois, MD, PhD, co-founder and CEO of Apellis.
“APL-2 offers hope for patients currently without an approved
treatment option.”
“Patients living with GA over time lose their
ability to drive, read and perform daily functions, so a treatment
that could potentially reduce the rate of GA lesion growth
and save visual function is important to this
population,” said Nathan Steinle, MD, Retina
Specialist at California Retina Consultants.
The Phase 3 program consists of two 600-patient
prospective, international, multicenter, randomized, double-masked,
sham-injection controlled studies (DERBY & OAKS) to assess the
efficacy and safety of multiple intravitreal (IVT) injections of
APL-2 in patients with GA. The Phase 3 trials are similar in design
to Apellis’ Phase 2 FILLY trial, including the eligibility criteria
and primary endpoint of change in GA lesion size from baseline to
month 12, compared to sham. In DERBY and OAKS, patients will
continue on therapy for an additional 12 months beyond the 12-month
primary endpoint. APL-2 met its primary endpoint in the Phase
2 FILLY trial. At 12 months, APL-2, administered monthly via
intravitreal injection, showed a 29% (p=0.008) reduction in the
rate of GA lesion growth compared to sham. With every other month
administration, a 20% (p=0.067) reduction compared to sham was
observed.
Apellis recently received Fast Track Designation
from the Food and Drug Administration (FDA) for APL-2 for the
treatment of GA.
About the DERBY and OAKS
trialsThe Derby and OAKS trials are 600-patient
prospective, international, multicenter, randomized, double-masked,
sham-injection controlled Phase 3 studies assessing the efficacy
and safety of multiple intravitreal (IVT) injections of APL-2 in
patients with Geographic Atrophy (GA) secondary to age-related
macular degeneration (AMD).
About the FILLY trial The
FILLY trial was a 246-patient Phase 2 multicenter, randomized,
single-masked, sham-controlled clinical trial of APL-2 in patients
with GA conducted at over 40 clinical sites, located in the
United States, Australia and New Zealand. APL-2 was
administered as an intravitreal injection in the study eye monthly
or every other month for 12 months, followed by six months of
monitoring without active treatment until Month 18. Eyes were
evaluated for GA by fundus autofluorescence photographs (FAF). The
rate of GA area growth was measured from baseline to Month 18. The
primary efficacy endpoint was the change in GA lesion size from
baseline to Month 12, compared to sham.
About Geographic Atrophy (GA)GA
is an advanced form of age-related macular degeneration (AMD), a
disorder of the central portion of the retina, known as the macula,
which is responsible for central vision and color perception. GA is
a chronic, progressive condition that leads to central blind spots
and permanent loss of vision. Based on published studies, the
company estimates that approximately one million people have GA
in the United States alone. There are currently no
FDA-approved treatments for GA.
About APL-2APL-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). In addition to the
DERBY and OAKS trials, Apellis is currently evaluating APL-2 in a
head-to-head Phase 3 clinical trial for systemic administration
comparing APL-2 to Soliris in PNH patients with hemoglobin levels
less than 10.5g/dL (the PEGASUS trial), a Phase 1b clinical trial
for systemic administration in treatment naïve PNH patients (the
PADDOCK trial), and a Phase 1b clinical trial for systemic
administration in eculizumab-treated PNH patients (the PHAROAH
trial).
About ApellisApellis
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.For additional information
regarding our clinical trials,
visit www.apellis.com/clinical-trials.html.
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 July 31,
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
Nicholastnicholas@denterlein.com 617.482.0042
(office)860.490.0218 (mobile)
Investor Contact: Alex
Kaneakane@w2ogroup.com212.301.7218 (office) 929.400.2691
(mobile)
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