Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Swedish Orphan
Biovitrum AB (publ) (Sobi™) (STO:SOBI) today announced that
the New England Journal of Medicine (NEJM) published results from
the Phase 3 PEGASUS study, which demonstrated the superiority of
pegcetacoplan, an investigational targeted C3 therapy, in improving
hemoglobin levels and showed improvements in key clinical outcomes
compared to eculizumab, a C5 inhibitor, in adults with paroxysmal
nocturnal hemoglobinuria (PNH) at 16 weeks who had persistent
anemia following treatment with eculizumab.
“The data published in the New England Journal of Medicine
underscore the potential of pegcetacoplan to be a significant
advancement for people living with PNH,” said Peter
Hillmen, M.B., Ch.B., Ph.D, professor of
experimental haematology at Leeds Teaching Hospitals NHS
Trust and PEGASUS study author. “There is still a need for new
treatments because many patients with PNH treated with C5
inhibitors today remain anemic, resulting in moderate to severe
fatigue, with a proportion continuing to require transfusions.”
“The PEGASUS study results demonstrate that, if approved,
pegcetacoplan has the potential to elevate the standard of care for
PNH by providing more complete disease control,” said Federico
Grossi, M.D., Ph.D., chief medical officer of Apellis. “We are
working to quickly bring this potential new treatment to PNH
patients and to advance development of pegcetacoplan for many other
serious, complement-driven diseases.”
“The NEJM publication of the PEGASUS study results reflect the
significance of these data for both the medical and PNH patient
communities,” said Ravi Rao, head of research and development and
chief medical officer at Sobi. “The results further advance our
understanding of the role of C3 in PNH and our hope is to
contribute to the improvement and care for PNH patients around the
world.”
The results published in NEJM highlight that pegcetacoplan met
the study’s primary endpoint for efficacy, demonstrating
superiority to eculizumab with a statistically significant
improvement in adjusted means of 3.8 g/dL of hemoglobin at week 16
(p<0.001). Additionally, 85% of pegcetacoplan-treated patients
were transfusion free over 16 weeks versus 15% of
eculizumab-treated patients. There were meaningful improvements
across key markers of disease such as absolute reticulocyte count,
lactate dehydrogenase (LDH), and fatigue as measured by the
Functional Assessment of Chronic Illness Therapy (FACIT)-fatigue
score.
Safety was comparable between pegcetacoplan and eculizumab in
this study. Seven of 41 patients (17%) in the pegcetacoplan group
experienced a SAE, and 6 of 39 patients (15%) in the eculizumab
group experienced SAEs. No cases of meningitis and no deaths were
reported in either treatment group.
The most common adverse events reported during the 16-week,
randomized, controlled treatment period in the pegcetacoplan and
eculizumab groups, respectively, were injection site reactions (37%
vs. 3%), diarrhea (22% vs. 3%), breakthrough hemolysis (10% vs.
23%), headache (7% vs. 23%), and fatigue (5% vs. 15%).
Top-line results from the PEGASUS study at 48 weeks were
recently announced, which showed sustained improvements in key
markers of disease as well as a consistent safety profile with
previously reported data.
Marketing applications for pegcetacoplan for the treatment of
PNH are under review by the U.S. Food and Drug Administration (FDA)
and the European Medicines Agency (EMA). The FDA granted the
application Priority Review designation and set a target action
date of May 14, 2021. An opinion from the Committee for Medicinal
Products for Human Use (CHMP) is expected in 2021.
About the PEGASUS StudyThe PEGASUS study
(APL2-302; NCT03500549) is a multi-center, randomized, head-to-head
Phase 3 study in 80 adults with paroxysmal nocturnal hemoglobinuria
(PNH). The primary objective of this study was to establish the
efficacy and safety of pegcetacoplan compared to eculizumab.
Participants must have been on eculizumab (stable for at least
three months) with a hemoglobin level of <10.5 g/dL at the
screening visit. During the four-week run-in, patients were dosed
with 1080 mg of pegcetacoplan twice weekly (n=41) in addition to
their current dose of eculizumab. During the 16-week randomized,
controlled period, patients were randomized to receive either 1080
mg of pegcetacoplan twice weekly or their current dose of
eculizumab (n=39). All participants completing the randomized
controlled period (n=77) opted to enter the open-label
pegcetacoplan treatment period.
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 Paroxysmal Nocturnal Hemoglobinuria
(PNH) PNH is a rare, chronic, life-threatening blood
disorder characterized by the destruction of oxygen-carrying red
blood cells through extravascular and intravascular hemolysis.
Persistently low hemoglobin can result in frequent transfusions and
debilitating symptoms such as severe fatigue, hemoglobinuria and
difficulty breathing (dyspnea). A retrospective analysis shows
that, even on eculizumab, approximately 72% of people with PNH have
anemia, a key indicator of ongoing hemolysis.1 The analysis also
finds that 36% of patients require one or more transfusions a year
and 16% require three or more.1
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.
About SobiSobi is a specialized international
biopharmaceutical company transforming the lives of people with
rare diseases. Sobi is providing sustainable access to innovative
therapies in the areas of haematology, immunology and specialty
indications. Today, Sobi employs approximately 1,500 people
across Europe, North America, the Middle
East, Asia and North Africa. In 2020, Sobi's revenue
amounted to SEK 15.3 billion. Sobi's share (STO:SOBI) is
listed on Nasdaq Stockholm. You can find more information about
Sobi at www.sobi.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, 2020 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.
Contacts:ApellisMedia:Lissa
Pavluk media@apellis.com 617.977.6764
Investors: Argot
Partnersapellis@argotpartners.com212.600.1902
SobiPaula Treutiger, Head of Communication
& Investor Relations + 46 733 666 599
paula.treutiger@sobi.com
Maria Kruse, Corporate Communication & Investor Relations+
46 767 248 830maria.kruse@sobi.com1. McKinley C. 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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