Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global
biopharmaceutical company and leader in targeted C3 therapies,
today announced 24-month data from the Phase 1b APL2-103 study of
pegcetacoplan, an investigational targeted C3 therapy, in patients
with advanced geographic atrophy (GA) secondary to age-related
macular degeneration (AMD) and low vision. GA is a leading cause of
blindness that affects approximately five million people
worldwide1,2 and has no treatment.
The Phase 1b study, designed to enroll approximately 12 patients
with bilateral GA (disease in both eyes), was initiated to assess
the safety of the Phase 3 formulation of pegcetacoplan
(15mg/0.1mL). Patients were dosed monthly with pegcetacoplan in one
eye using the fellow eye as an untreated control.
The current post hoc analysis includes eight patients for whom
data were available for at least 24 months. In this population, the
growth rate of GA lesions in the treated eye was 46% (mean square
root) slower than the untreated fellow eye (p=0.007). It has been
shown that lesions in both eyes tend to grow at the same rate in
patients with bilateral GA.3 Of the 13 enrolled patients,
there were no reported cases of inflammation and two patients (16%)
developed new-onset exudation during the 24-month study
duration.
“I am encouraged to see the continued effect of intravitreal
pegcetacoplan in patients with GA.” stated Eleonora Lad, M.D.,
Ph.D., Associate Professor of Ophthalmology at the Duke University
Medical Center. “I believe that these findings, which demonstrate a
46% reduction in GA lesion growth at 24 months, are clinically
meaningful. I am excited to learn the Phase 3 DERBY and OAKS
results later this year.”
Post hoc analysis of Study APL2-103 at 24
months
A Media Snippet accompanying this announcement is available by
clicking on the image or link below:
The patient population enrolled in the Phase 1b study is similar
to patients enrolled in the Phase 3 DERBY and OAKS studies but
allowed for more advanced disease with a wider range of baseline
lesion size and lower baseline visual acuity. DERBY and OAKS use
the same pegcetacoplan formulation tested in this study and
top-line data are expected in the third quarter of 2021.
Patients who participated in the APL2-103 Phase 1b study will be
invited to enroll in the APL2-GA-305 GALE trial. The GALE trial is
an open-label extension study designed to evaluate the long-term
safety and efficacy of pegcetacoplan in patients with GA who
participated in the Phase 1b study or who complete DERBY and
OAKS.
About
Pegcetacoplan Pegcetacoplan is an
investigational, targeted C3 therapy designed to regulate excessive
activation of the complement cascade, part of the body’s immune
system, which can lead to the onset and progression of many serious
diseases. Pegcetacoplan is a synthetic cyclic peptide
conjugated to a polyethylene glycol polymer that binds specifically
to C3 and C3b. Pegcetacoplan is being evaluated in several clinical
studies across hematology, ophthalmology, nephrology, and
neurology. Marketing applications for pegcetacoplan for paroxysmal
nocturnal hemoglobinuria (PNH) are under review by the U.S. Food
and Drug Administration (FDA), which has granted the application
Priority Review designation, and the European Medicines Agency
(EMA). Pegcetacoplan was granted Fast Track designation by the
U.S. Food and Drug Administration (FDA) for the treatment of
geographic atrophy and received orphan drug designation for the
treatment of C3G by the FDA and European Medicines Agency. For
additional information regarding pegcetacoplan clinical
trials, visit https://apellis.com/our-science/clinical-trials.
About Geographic Atrophy (GA) GA is an
advanced form of age-related macular degeneration (AMD), a leading
cause of blindness. GA lesions affect the central portion of the
retina, known as the macula, which is responsible for central
vision. Excessive complement activation drives irreversible lesion
growth in GA4, and C3 is the only target to precisely control
complement overactivation. GA is progressive and irreversible,
leading to central visual impairment and permanent loss of vision.
Based on published studies, approximately one million people have
GA in the United States and 5 million people have GA
globally.1,2 There are currently no approved treatments
for GA.
About APL2-103 The APL2-103 study is a Phase
1b, multicenter, open label, single arm, 24-month clinical trial to
assess the safety of monthly intravitreal (IVT) injections of
pegcetacoplan in patients diagnosed with advanced geographic
atrophy (GA) secondary to age-related macular degeneration (AMD).
The primary outcome measures include incidence and severity of
ocular and systemic treatment-emergent adverse events
(TEAEs).About DERBY and OAKSDERBY (621 patients
enrolled) and OAKS (638 patients enrolled) are Phase 3,
multicenter, randomized, double-masked, sham-controlled studies
comparing the efficacy and safety of intravitreal pegcetacoplan
with sham injections in patients with GA secondary to AMD. The
primary objective of the studies is to evaluate the efficacy of
pegcetacoplan in patients with GA assessed by change in the total
area of GA lesions from baseline as measured by fundus
autofluorescence.
About GALEGALE is a Phase 3, multicenter, open
label, extension study to evaluate the long-term safety and
efficacy of intravitreal pegcetacoplan in patients with GA
secondary to AMD. The objectives of the study are to evaluate the
long-term incidence and severity of ocular and systemic treatment
emergent adverse events as well as change in the total area of GA
lesions as measured by fundus autofluorescence.
About Apellis Apellis Pharmaceuticals,
Inc. is a global biopharmaceutical company that is committed to
leveraging courageous science, creativity, and compassion to
deliver life-changing therapies. Leaders in targeted C3 therapies,
we aim to develop transformative therapies for a broad range of
debilitating diseases that are driven by excessive activation of
the complement cascade, including those within hematology,
ophthalmology, nephrology, and neurology. For more information,
please visit http://apellis.com.
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 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 pegcetacoplan will successfully advance through
the clinical trial process on a timely basis, or at all; whether
the results of the company’s clinical trials will warrant
regulatory submissions and whether pegcetacoplan will
receive approval from the FDA or equivalent foreign regulatory
agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other
indication when expected or at all; whether, if Apellis’ products
receive approval, they will be successfully distributed and
marketed; and other factors discussed in the “Risk Factors” section
of Apellis’ Annual Report on Form 10-K filed with
the Securities and Exchange Commission on February 25,
2021 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:Mark
Dolemedia@apellis.com 617.997.3484
Investor Contact:Argot
Partnersapellis@argotpartners.com 212.600.1902 ____________________________________________________________________________
1 Rudnicka AR, Jarrar Z, Wormald R, et al. Age and gender
variations in age-related macular degeneration prevalence in
populations of European ancestry: a meta
analysis. Ophthalmology 2012;119:571–580.
2 Wong WL, Su X, Li X, et al. Global prevalence of age-related
macular degeneration and disease burden projection for 2020 and
2040: a systematic review and meta-analysis. Lancet Glob
Health 2014;2:e106–116.
3 Sunness JS, et al. The long-term natural history of geographic
atrophy from age-related macular degeneration: enlargement of
atrophy and implications for interventional clinical trials.
Ophthalmology. 2007 Feb; 114(2):271-7.
4 Seddon, JM, Rosner, B. Validated prediction models for macular
degeneration progression and predictors of visual acuity loss
identify high-risk individuals. Am J
Ophthalmol 2019;198:223–261.
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