Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Sobi today
presented post hoc data that reinforce the long-term efficacy and
safety of EMPAVELI
® (pegcetacoplan) in adults with
paroxysmal nocturnal hemoglobinuria (PNH) for up to three years.
The data were reported during an oral presentation at the American
Society of Hematology (ASH) Annual Meeting.
“These results show that treatment with EMPAVELI
can help PNH patients achieve rapid and sustained control of their
disease over the long term,” said Carlos de Castro, M.D.,
presenting author and professor of medicine, Duke University.
“Furthermore, it is impressive that the majority of patients
remained transfusion free up to three years, alleviating a
significant and common disease burden for many people living with
PNH.”
The analysis integrated data across the Phase 3
PEGASUS and PRINCE studies and the long-term extension study. After
starting treatment with EMPAVELI, key markers of disease rapidly
improved and were sustained in both treatment-naïve patients and
patients previously treated with eculizumab. Improvements in
hemoglobin reached normal or near-normal levels, and mean lactate
dehydrogenase (LDH) was maintained below the upper limit of
normal.
Additionally, 67% of treatment-naïve patients from
PRINCE were transfusion free for up to 2.5 years, and 52% of
patients from PEGASUS remained transfusion free for up to 3 years.
Less than 25% of patients were transfusion free in the year prior
to entering the PRINCE and PEGASUS studies.
The safety profile was consistent with previous
clinical study results, and no new or unexpected safety findings
were identified. Approximately 4.5% of patients experienced a
serious adverse event deemed related to treatment with
pegcetacoplan. No meningococcal infections were reported.
The prescribing information for EMPAVELI contains a
boxed warning, which states that EMPAVELI may increase the risk of
meningococcal and other serious infections caused by encapsulated
bacteria that may become rapidly life threatening or fatal if not
recognized and treated early.
“The strength of these long-term EMPAVELI results
highlight why we are seeing compliance rates of 97% with real-world
use,” said Peter Hillmen, M.B. Ch.B., Ph.D., head, rare disease
advisor, Apellis. “By achieving and maintaining normal clinical
measures over time, EMPAVELI has the potential to elevate the
standard of care for all adults with PNH."
About the Long-Term Efficacy and Safety
Extension (APL2-307) StudyThe APL2-307 study was a
nonrandomized, multicenter, open-label Phase 3 extension study of
137 adults with paroxysmal nocturnal hemoglobinuria (PNH) who
completed previous EMPAVELI®/Aspaveli® (pegcetacoplan) Phase 1
(PHAROAH, PADDOCK), Phase 2 (PALOMINO), and Phase 3 (PEGASUS,
PRINCE) clinical trials. Patients in these studies were either
anemic despite eculizumab treatment or were naïve to complement
inhibitors. During the trial, patients continued to receive 1080 mg
of EMPAVELI twice weekly or once every three days (PEGASUS, PRINCE)
or switched to 1080 mg of EMPAVELI twice weekly (PHAROAH, PADDOCK,
PALOMINO). The primary objective was to establish the long-term
efficacy and safety of EMPAVELI.
About the PEGASUS StudyThe PEGASUS
study (APL2-302; NCT03500549) was a randomized, multi-center,
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
EMPAVELI®/Aspaveli® (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 EMPAVELI 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 EMPAVELI 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 EMPAVELI treatment
period.
About the PRINCE StudyThe PRINCE
study (NCT04085601) was a randomized, multi-center, open-label,
controlled Phase 3 study in 53 treatment-naïve adults with
paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of
this study was to establish the efficacy and safety of
EMPAVELI®/Aspaveli® (pegcetacoplan) in patients who had not
received treatment with any complement inhibitor within three
months prior to screening. During the 26-week randomized,
controlled period, patients received either 1080 mg of EMPAVELI
twice weekly or standard of care therapy, which did not include
complement inhibitors. Patients in the standard of care group had
the option to escape to the EMPAVELI group if their hemoglobin
decreased by 2 g/dL or more from their baseline value.
About
EMPAVELI®/Aspaveli® (pegcetacoplan)EMPAVELI®/Aspaveli® (pegcetacoplan)
is a 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. It
is approved for the treatment of paroxysmal nocturnal
hemoglobinuria (PNH) in the United States, European Union, and
other countries globally. The therapy is also under investigation
for several other rare diseases across hematology and
nephrology.
U.S. Important Safety Information for
EMPAVELI
BOXED WARNING: SERIOUS INFECTIONS CAUSED BY
ENCAPSULATED BACTERIA
- Meningococcal infections may occur in patients treated
with EMPAVELI and may become rapidly life-threatening or fatal if
not recognized and treated early. Use of EMPAVELI may predispose
individuals to serious infections, especially those caused by
encapsulated bacteria, such as Streptococcus
pneumoniae, Neisseria
meningitidis types A, C, W, Y, and B, and
Haemophilus influenzae type
B.
- Comply with the most current Advisory Committee on
Immunization Practices (ACIP) recommendations for vaccinations
against encapsulated bacteria.
- Vaccinate patients at least 2 weeks prior to
administering the first dose of EMPAVELI unless the risks of
delaying therapy with EMPAVELI outweigh the risk of developing a
serious infection.
- Vaccination reduces, but does not eliminate, the risk
of serious infections. Monitor patients for early signs of serious
infections and evaluate immediately if infection is
suspected.
- EMPAVELI is available only through a restricted program
under a Risk Evaluation and Mitigation Strategy (REMS). Under the
EMPAVELI REMS, prescribers must enroll in the
program.
CONTRAINDICATIONS
- Hypersensitivity to pegcetacoplan or to any of the
excipients
- Not currently vaccinated against certain encapsulated bacteria,
unless the risks of delaying EMPAVELI treatment outweigh the risks
of developing a bacterial infection with an encapsulated
organism
- Unresolved serious infection caused by encapsulated bacteria
including Streptococcus pneumoniae, Neisseria meningitidis, and
Haemophilus influenzae
WARNINGS AND PRECAUTIONS
Serious Infections Caused by Encapsulated
BacteriaThe use of EMPAVELI may predispose individuals to
serious, life-threatening, or fatal infections caused by
encapsulated bacteria, including Streptococcus pneumoniae,
Neisseria meningitidis types A, C, W, Y, and B, and Haemophilus
influenzae type B (Hib). To reduce the risk of infection, all
patients must be vaccinated against these bacteria according to the
most current ACIP recommendations for patients with altered
immunocompetence associated with complement deficiencies.
Revaccinate patients in accordance with ACIP recommendations
considering the duration of therapy with EMPAVELI.
For patients without known history of vaccination,
administer required vaccines at least 2 weeks prior to receiving
the first dose of EMPAVELI. If immediate therapy with EMPAVELI is
indicated, administer required vaccine as soon as possible and
provide patients with 2 weeks of antibacterial drug
prophylaxis.
Closely monitor patients for early signs and
symptoms of serious infection and evaluate patients immediately if
an infection is suspected. Promptly treat known infections. Serious
infection may become rapidly life-threatening or fatal if not
recognized and treated early. Consider discontinuation of EMPAVELI
in patients who are undergoing treatment for serious
infections.
EMPAVELI REMSBecause of the risk
of serious infections, EMPAVELI is available only through a
restricted program under a REMS. Under the EMPAVELI REMS,
prescribers must enroll in the program and must counsel patients
about the risk of serious infection, provide the patients with the
REMS educational materials, and ensure patients are vaccinated
against encapsulated bacteria. Enrollment and additional
information are available by telephone: 1-888-343-7073 or at
www.empavelirems.com.
Infusion-Related ReactionsSystemic
hypersensitivity reactions (e.g., facial swelling, rash, urticaria)
have occurred in patients treated with EMPAVELI. One patient (less
than 1% in clinical studies) experienced a serious allergic
reaction which resolved after treatment with antihistamines. If a
severe hypersensitivity reaction (including anaphylaxis) occurs,
discontinue EMPAVELI infusion immediately, institute appropriate
treatment, per standard of care, and monitor until signs and
symptoms are resolved.
Monitoring PNH Manifestations after
Discontinuation of EMPAVELIAfter discontinuing treatment
with EMPAVELI, closely monitor for signs and symptoms of hemolysis,
identified by elevated LDH levels along with sudden decrease in PNH
clone size or hemoglobin, or reappearance of symptoms such as
fatigue, hemoglobinuria, abdominal pain, dyspnea, major adverse
vascular events (including thrombosis), dysphagia, or erectile
dysfunction. Monitor any patient who discontinues EMPAVELI for at
least 8 weeks to detect hemolysis and other reactions. If
hemolysis, including elevated LDH, occurs after discontinuation of
EMPAVELI, consider restarting treatment with EMPAVELI.
Interference with Laboratory
TestsThere may be interference between silica reagents in
coagulation panels and EMPAVELI that results in artificially
prolonged activated partial thromboplastin time (aPTT); therefore,
avoid the use of silica reagents in coagulation panels.
ADVERSE REACTIONSMost common
adverse reactions in patients with PNH (incidence ≥10%) were
injection-site reactions, infections, diarrhea, abdominal pain,
respiratory tract infection, pain in extremity, hypokalemia,
fatigue, viral infection, cough, arthralgia, dizziness, headache,
and rash.
USE IN SPECIFIC POPULATIONS
Females of Reproductive
PotentialEMPAVELI may cause embryo-fetal harm when
administered to pregnant women. Pregnancy testing is recommended
for females of reproductive potential prior to treatment with
EMPAVELI. Advise female patients of reproductive potential to use
effective contraception during treatment with EMPAVELI and for 40
days after the last dose.
Please see full
Prescribing Information, including Boxed
WARNING regarding serious infections caused by encapsulated
bacteria, and Medication
Guide.
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).
About the Apellis and Sobi
CollaborationApellis and Sobi have global co-development
rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S.
commercialization rights for systemic pegcetacoplan, and Apellis
has exclusive U.S. commercialization rights for systemic
pegcetacoplan and worldwide commercial rights for ophthalmological
pegcetacoplan, including for geographic atrophy.
About ApellisApellis
Pharmaceuticals, Inc. is a global biopharmaceutical company that
combines courageous science and compassion to develop life-changing
therapies for some of the most challenging diseases patients face.
We ushered in the first new class of complement medicine in 15
years and now have two approved medicines targeting C3. These
include the first and only therapy for geographic atrophy, a
leading cause of blindness around the world. We believe we have
only begun to unlock the potential of targeting C3 across serious
retinal, rare, and neurological diseases. For more information,
please visit http://apellis.com or follow us
on Twitter and LinkedIn.
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. The words “anticipate,”
“believe,” “continue,” “could,” “can,” “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
discussed in the “Risk Factors” section of Apellis’ Annual Report
on Form 10-K with the Securities and Exchange Commission on
February 21, 2023 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: Lissa
Pavluk media@apellis.com617.977.6764
Investor Contact: Meredith
Kaya meredith.kaya@apellis.com617.599.8178
Apellis Pharmaceuticals (NASDAQ:APLS)
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