Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and
Sobi® (STO:SOBI) today announced positive one-year results
from the Phase 2 NOBLE study investigating systemic pegcetacoplan,
a targeted C3 therapy, for the treatment of post-transplant
recurrence of C3 glomerulopathy (C3G) and primary immune complex
membranoproliferative glomerulonephritis (IC-MPGN). These data were
presented during a late-breaking oral presentation at the European
Renal Association (ERA) Congress taking place May 23-26 in
Stockholm, Sweden.
“It is exciting to see that treatment with pegcetacoplan rapidly
reduced disease activity in only 12 weeks, and the effects were
sustained over the long term,” said Fadi Fakhouri, M.D., Ph.D.,
presenting author and professor of nephrology at CHUV, Lausanne,
Switzerland. “Post-transplant C3G and IC-MPGN patients are likely
to experience disease recurrence, putting them at risk of needing
another kidney transplant or dialysis. There is a huge need for a
treatment that targets the cause of these diseases, and I am very
encouraged by these unprecedented results.”
At one year, of the 11 pegcetacoplan-treated patients with
available data:
- Seven (64%) patients showed a reduction in C3c staining by two
or more orders of magnitude of intensity from baseline.
- Six (55%) patients, including the three IC-MPGN patients,
showed zero C3c staining intensity, indicating that C3c deposits
were cleared.
- Consistent with C3c staining reduction, seven patients (64%)
showed zero inflammation as measured by the activity score of the
C3G histologic index.
Excessive C3c deposits are a marker of disease activity, which
can lead to kidney inflammation, damage, and failure. Clearance of
both C3c deposits and inflammation allow the kidney to recover and
prolong the function of the kidney.
Additionally, treatment with pegcetacoplan resulted in sustained
improvements in key measures of disease including proteinuria and
continued stabilization of kidney function.
“The NOBLE data further indicate that pegcetacoplan is treating
the underlying cause of these diseases by directly targeting C3,”
said Caroline Baumal, M.D., chief medical officer, Apellis. “Our
ongoing Phase 3 VALIANT study evaluates the potential of
pegcetacoplan in all patients with these rare kidney diseases, and
we look forward to sharing the topline results later this
year.”
Pegcetacoplan was generally well-tolerated. The majority of
adverse events were mild to moderate and consistent with previously
reported results.
"Patients with post-transplant C3G and IC-MPGN often face the
daunting reality of disease relapse, so the need for a therapeutic
approach addressing the root cause of these conditions cannot be
overstated,” said Lydia Abad-Franch, M.D., head of R&D and
medical affairs, chief medical officer, Sobi. “These findings
underscore our conviction in pegcetacoplan’s potential to address
these rare, severe, and life-threatening conditions in both native
and post-transplant kidneys."
About the Phase 2 NOBLE Study The Phase
2 NOBLE study (NCT04572854) is a multicenter, open-label,
randomized, controlled study designed to evaluate the efficacy and
safety of pegcetacoplan in 13 adults who have post-transplant
recurrence of C3G or IC-MPGN. Study participants were randomized in
a 3:1 ratio to receive pegcetacoplan (IC-MPGN: n=2; C3G: n=8) or
maintain standard of care (IC-MPGN: n=1; C3G: n=2) for 12 weeks,
and all patients received pegcetacoplan from week 13 to week 52.
The primary endpoint of the study was the proportion of patients
with reduction in C3c staining on renal biopsy after 12 weeks of
treatment with pegcetacoplan. Secondary endpoints include an
evaluation of safety, the proportion of patients with reduction in
C3c staining on renal biopsy after 52 weeks of treatment, and the
proportion of patients achieving at least a 50% reduction in
proteinuria.
About the VALIANT StudyThe VALIANT Phase 3
study (NCT05067127) is a randomized, placebo-controlled,
double-blinded, multi-center study designed to evaluate
pegcetacoplan efficacy and safety in approximately 90 patients who
are 12 years of age and older with C3G or primary IC-MPGN. It is
the only study to include both patients with native kidney disease
and patients who have recurrent disease after receiving a kidney
transplant. Study participants will be randomized to receive 1080
mg of pegcetacoplan or placebo twice weekly for 26 weeks. Following
this 26-week randomized controlled period, patients will proceed to
a 26-week open-label phase in which all patients receive
pegcetacoplan. The primary endpoint of the study is the log
transformed ratio of urine protein-to-creatinine ratio (uPCR) at
week 26 compared to baseline.
About C3 Glomerulopathy (C3G)
and Immune-Complex Membranoproliferative Glomerulonephritis
(IC-MPGN)C3G and primary IC-MPGN are rare and debilitating
kidney diseases that can lead to kidney failure. Excessive C3c
deposits are a marker of disease activity, which can lead to kidney
inflammation, damage, and failure. There are no treatments that
target the underlying cause of these diseases. Approximately 50% of
people living with C3G and IC-MPGN suffer from kidney failure
within five to 10 years of diagnosis, requiring a burdensome kidney
transplant or lifelong dialysis.1 Additionally,
two-thirds of patients who previously received a kidney transplant
will experience disease recurrence.2 The diseases
are estimated to affect 5,000 people in the United States and up to
8,000 in Europe.3
About Pegcetacoplan in Rare
DiseasesPegcetacoplan is a targeted C3 therapy designed to
regulate excessive activation of the complement cascade, a part of
the body’s immune system, which can lead to the onset and
progression of many serious diseases. Pegcetacoplan is under
investigation for rare diseases across hematology and nephrology.
Pegcetacoplan is approved for the treatment of paroxysmal nocturnal
hemoglobinuria (PNH) as EMPAVELI®/Aspaveli® in the United States,
European Union, and other countries globally.
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-ever
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 X
(Twitter) and LinkedIn.
About Sobi® Sobi® is a
specialised international biopharmaceutical company transforming
the lives of people with rare and debilitating diseases. Providing
reliable access to innovative medicines in the areas of
haematology, immunology, and specialty care, Sobi has approximately
1,800 employees across Europe, North America, the Middle East,
Asia, and Australia. In 2023, revenue amounted to SEK 22.1 billion.
Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. Learn more
about Sobi at sobi.com and LinkedIn.
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. 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 results
obtained in clinical trials will be indicative of results that will
be generated in future clinical trials; whether the results of the
company’s clinical trials in C3G and IC-MPGN will warrant
regulatory submissions and whether systemic pegcetacoplan will
receive approval for those indications from the FDA or equivalent
foreign regulatory agencies when expected or at all; and any other
factors discussed in the “Risk Factors” section of Apellis’ Annual
Report on Form 10-K with the Securities and Exchange Commission on
February 27, 2024 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:Lissa
Pavluk media@apellis.com 617.977.6764
Investors: Meredith Kaya
meredith.kaya@apellis.com 617.599.8178
ContactsFor details on how to contact the Sobi
Investor Relations Team, please click here. For Sobi Media
contacts, click here.
References1. C3 glomerulopathy. National Institute of Health,
Genetics Home
Reference. https://ghr.nlm.nih.gov/condition/c3-glomerulopathy#resources.
Accessed November 21, 2019. 2. Zand L, et al Clinical
findings, pathology, and outcomes of C3GN after kidney
transplantation. J Am Soc Nephrol. 2014 May;25(5):1110-7. doi:
10.1681/ASN.2013070715. Epub 2013 Dec 19.3. Data on file using
literature consensus.
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