Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global
biopharmaceutical company pioneering targeted C3 therapies, today
announced positive results from the Phase 3 PEGASUS study
evaluating pegcetacoplan (APL-2) in adults with paroxysmal
nocturnal hemoglobinuria (PNH). Top-line data show that
pegcetacoplan met the study’s primary efficacy endpoint,
demonstrating superiority to eculizumab with a statistically
significant improvement in adjusted means of 3.8 g/dL of hemoglobin
at week 16 (p<0.0001). At week 16, pegcetacoplan-treated
patients (n=41) had an adjusted mean hemoglobin increase of 2.4
g/dL from a baseline of 8.7 g/dL, compared to eculizumab-treated
patients (n=39) who had a change of -1.5 g/dL from a baseline of
8.7 g/dL.
Additionally, pegcetacoplan showed promising results in key
secondary endpoints. Pegcetacoplan met non-inferiority on
transfusion avoidance and absolute reticulocyte count.
Pegcetacoplan also showed positive trends on lactate dehydrogenase
(LDH) and fatigue as measured by the Functional Assessment of
Chronic Illness Therapy (FACIT)-fatigue score.
Key Secondary Endpoints Analysis
View full table by clicking on the image or link below:
“Pegcetacoplan is the first and only investigational therapy to
demonstrate superiority compared to eculizumab on hemoglobin
levels. We are also excited to see that 85% of patients treated
with pegcetacoplan were transfusion free,” said Federico Grossi,
M.D., Ph.D., Chief Medical Officer of Apellis. “At Apellis, we are
focused on developing groundbreaking therapies, and these results
show that pegcetacoplan has the potential to transform the lives of
people with PNH. We look forward to meeting with regulators in the
first half of the year to discuss next steps.”
“The majority of patients with PNH currently receiving
treatment with eculizumab have continuing anemia,” said Peter
Hillmen, M.B., Ch.B., Ph.D., Professor of Experimental
Hematology at the University of Leeds and an investigator in
the PEGASUS study. “The PEGASUS results show that
pegcetacoplan has the potential to become a new standard
of care for patients with PNH.”
In this study, the safety profile of pegcetacoplan was
comparable to eculizumab. Seven of 41 patients (17.1%) in the
pegcetacoplan group experienced a serious adverse event (SAE), and
6 of 39 patients (15.4%) 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 (36.6% vs. 2.6%), diarrhea (22.0% vs. 0%), headache
(7.3% vs. 20.5%) and fatigue (4.9% vs. 15.4%). Another common
adverse event was hemolysis, which was reported in four patients in
the pegcetacoplan group (9.8%) and nine patients in the eculizumab
group (23.1%). This led to the three discontinuations in
pegcetacoplan group.
All patients who completed the randomization period in both
groups (77/80) entered the 32-week open-label pegcetacoplan
treatment period.
“Going into the study, our most optimistic expectation was to
see a 2 g/dL or more change in hemoglobin and a trend on the key
secondary endpoints. Needless to say, we are thrilled with these
results,” said Cedric Francois, M.D., Ph.D., Co-Founder and Chief
Executive Officer of Apellis. “These data give us strong confidence
in the further development of pegcetacoplan as a targeted C3
inhibitor in geographic atrophy and other serious complement-driven
diseases.”
Detailed results from the PEGASUS study will be presented at a
future scientific meeting.
Conference Call and WebcastApellis will host a
conference call and webcast to discuss the results of the PEGASUS
Phase 3 clinical study today, January 7 at 8:30 a.m. ET. To access
the live call by phone please dial 866-774-0323 (domestic) or
602-563-8683 (international); the conference ID is 4199692. A live
audio webcast of the event and accompanying slides may also be
accessed through the “Events and Presentations” page of the
“Investors and Media” section of the company’s website at
http://investors.apellis.com/events-and-presentations. A replay of
the webcast will be available for 30 days following the
event.
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH is a rare, chronic, life-threatening blood disorder associated
with abnormally low hemoglobin levels due to the destruction of
oxygen-carrying red blood cells (hemolysis). Persistently low
hemoglobin can result in frequent transfusions and debilitating
symptoms such as severe fatigue and difficulty breathing (dyspnea).
Retrospective studies show that, even on eculizumab, approximately
70% of people with PNH have low hemoglobin levels,1,2 and 36%
require one or more transfusions a year.3
About the PEGASUS Study The PEGASUS study
(APL2-302; NCT03500549) is a multi-center, randomized, open-label,
active-comparator controlled Phase 3 study in 80 adults with 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 3 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 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. All participants
completing the randomized controlled period entered the open-label
pegcetacoplan treatment period where they received pegcetacoplan,
regardless of the prior treatment received in the randomized,
controlled period.
Key secondary endpoints were tested in a hierarchical manner
after statistical significance was reached for the primary
endpoint. Key secondary endpoints were tested first for
non-inferiority and, if all were met, then superiority was tested
sequentially for transfusion avoidance, absolute reticulocyte
count, and FACIT-fatigue score. Once one hypothesis was tested as
not significant, all subsequent tests were not assessed. Estimates
were computed for all key secondary and secondary endpoints
regardless of whether a hypothesis was tested.
The study was conducted in collaboration with SFJ
Pharmaceuticals, who supported the development of pegcetacoplan in
PNH. SFJ is a global drug development company, which provides a
unique and highly customized co-development partnering model for
the world’s top pharmaceutical and biotechnology companies.
About Pegcetacoplan (APL-2) Pegcetacoplan is an
investigational, targeted C3 inhibitor designed to regulate
excessive complement activation, 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. Apellis is evaluating
pegcetacoplan in several clinical studies including paroxysmal
nocturnal hemoglobinuria (PNH), geographic atrophy (GA), and C3
glomerulopathy. Pegcetacoplan was granted Fast Track designation by
the U.S. Food and Drug Administration (FDA) for the treatment of
PNH and the treatment of GA. For additional information regarding
our clinical trials,
visit www.apellis.com/clinical-trials.html.
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. By pioneering targeted C3 therapies, we
aim to develop best-in-class and first-in-class therapies for a
broad range of debilitating diseases that are driven by
uncontrolled or excessive activation of the complement cascade,
including those within hematology, ophthalmology, and nephrology.
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 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 pegcetacoplan will successfully
advance through the clinical trial process on a timely basis, or at
all; whether the results of the Pegasus or other clinical trials
will be sufficient to form the basis of regulatory submissions,
whether the Company’s clinical trials will warrant regulatory
submissions and whether pegcetacoplan will receive approval from
the United States Food and Drug Administration or equivalent
foreign regulatory agencies for GA, PNH, C3G 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 November 5, 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:Tracy
Vineismedia@apellis.com617.420.4839
Investor Contact: Sam Martin / Maghan
Meyers Argot Partners
sam@argotpartners.com / maghan@argotpartners.com
212.600.1902
1. Risitano AM, Marotta S, Ricci P, et al. (2019)
Anti-complement Treatment for Paroxysmal Nocturnal Hemoglobinuria:
Time for Proximal Complement Inhibition? A Position Paper From the
SAAWP of the EBMT. Front. Immunol. 10:1157. doi:
10.3389/fimmu.2019.01157.2. Risitano AM, Notaro R, Marando L, et
al. (2009) Complement fraction 3 binding on erythrocytes as
additional mechanism of disease in paroxysmal nocturnal
hemoglobinuria patients treated by eculizumab. Blood. 2009 Apr
23;113(17):4094-100. 3. 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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