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 details about its Phase 2
FILLY study investigating intravitreal (IVT) APL-2 (pegcetacoplan)
for the treatment of geographic atrophy (GA) secondary to
age-related macular degeneration (AMD) have been published in
Ophthalmology, the journal of the American Academy of
Ophthalmology. Publication of the full study report follows the
release of topline study results earlier this year and shows that
treatment with APL-2 resulted in statistically significant
reductions in the growth of GA lesion area compared to sham at
month 12.
“There is a significant need to develop treatments for people
living with GA, a progressive, chronic disease that often results
in permanent loss of vision,” said Federico Grossi, M.D., Ph.D.,
chief medical officer of Apellis. “The FILLY study results show
preliminary positive benefits of targeting the complement cascade
at C3, which is implicated in the destruction of
eyesight-protecting retinal pigment epithelium cells, and the
potential for APL-2 to be an effective treatment option for people
living with GA.”
The FILLY study was a Phase 2, multicenter, randomized,
single-masked, sham-controlled clinical trial evaluating APL-2 in
246 patients with GA secondary to AMD conducted at over 40 clinical
sites in the United States, Australia and New
Zealand. APL-2 was administered as an intravitreal injection
monthly or every other month (EOM) for 12 months, followed by six
months of monitoring after the end of treatment. The primary
efficacy endpoint was the change in GA lesion area from baseline to
month 12 compared to sham.
At 12 months, patients treated with APL-2 showed a 29% reduction
in the growth of GA lesion area in the monthly treatment group
(p=0.008) and a 20% reduction in the EOM treatment group (p=0.067)
compared to the pooled sham group. Statistical significance was
defined as p<0.1 for this study. Post-hoc analysis showed that
the effect was more pronounced in the last six months of treatment,
with observed reductions of 45% (p=0.0004) and 33% (p=0.009) for
APL-2 monthly and EOM, respectively, compared to sham.
The administration of APL-2 was generally well tolerated. There
was an increased incidence of exudation in APL-2 treated eyes
(20.9% in the monthly group and 8.9% in the EOM group) compared to
sham-treated eyes (1.2%), which was manageable with the
administration of standard-of-care treatment.
“There are currently no approved treatments for GA, which means
that the approximately five million GA patients globally live
knowing that they will lose vision over time,” said David S.
Liao, M.D., lead author and retina specialist at the
Retina-Vitreous Associates Medical Group. “I’m very encouraged by
the FILLY data and look forward to seeing the upcoming results from
Apellis’ Phase 3 studies of APL-2 in GA.”
The U.S. Food and Drug Administration (FDA) granted APL-2 Fast
Track Designation for the treatment of GA, which facilitates the
development and expedites the review of investigational therapies
to treat serious conditions and fill an unmet medical need.
Apellis is currently enrolling two global confirmatory Phase 3
studies (DERBY and OAKS) for patients with GA. These identical,
prospective, multicenter, randomized, double-masked, sham-injection
controlled studies are designed to assess the efficacy and safety
of multiple IVT injections of APL-2 in patients with GA secondary
to AMD.
More information regarding DERBY and OAKS can be found at
https://gastudy.com/.
About APL-2 (pegcetacoplan)APL-2, an
investigational drug, 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
downstream 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). Apellis is currently evaluating APL-2 in clinical
studies in patients with geographic atrophy (GA), in patients with
paroxysmal nocturnal hemoglobinuria (PNH) who are being treated
with eculizumab or who are naïve to complement inhibitor treatment,
in patients with cold agglutinin disease (CAD) and warm autoimmune
hemolytic anemia (wAIHA), and in patients with C3 glomerulopathy
(C3G) and other glomerular diseases. For additional
information regarding our clinical trials, visit
www.apellis.com/clinical-trials.html.
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. The disease is
chronic and progressive, leading to central blind spots 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. There are currently no FDA-approved
treatments for GA.
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.
Apellis Forward-Looking StatementStatements 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 such as the ongoing Phase 3 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,
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:Ryan
Waderyan.wade@apellis.com781.209.6460 x8148 (office)781.801.5206
(mobile)
Investor Contact: Alex
Kaneakane@w2ogroup.com212.301.7218 (office)
929.400.2691 (mobile)
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