Apellis Reports Top-Line Results from Phase 2 MERIDIAN Study in ALS
May 25 2023 - 7:00AM
Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that
the Phase 2 MERIDIAN study investigating systemic pegcetacoplan for
the treatment of amyotrophic lateral sclerosis (ALS) did not meet
its primary endpoint of the Combined Assessment of Function and
Survival (CAFS) rank score at Week 52. The study also did not meet
key secondary efficacy endpoints. Systemic pegcetacoplan was well
tolerated in the study, and the data were consistent with the
established safety profile.
Based on the lack of efficacy, Apellis and Sobi plan to
discontinue development of systemic pegcetacoplan for ALS. In
April, Apellis and Sobi discontinued treatment in the open-label
portion of the study, following a recommendation from an
independent data monitoring committee.
“We are disappointed in the outcome of the MERIDIAN study,
especially on behalf of the ALS community who has been waiting for
new treatments for this complex and unrelenting disease. We would
like to sincerely thank the study participants and their caregivers
from around the world who contributed to this important research,”
said Jeffrey Eisele, Ph.D., chief development officer, Apellis.
“Our hope is that the data generated from this study will continue
to support future research and development in ALS.”
The full MERIDIAN dataset is being analyzed, and detailed data
is expected to be presented at a future medical meeting.
About the Phase 2 MERIDIAN StudyThe Phase 2
MERIDIAN study (NCT04579666) is a randomized, double-blind,
placebo-controlled, multicenter study designed to evaluate the
efficacy and safety of systemic pegcetacoplan in approximately 250
adults with sporadic amyotrophic lateral sclerosis (ALS). Study
participants were randomized in a 2:1 ratio to receive
pegcetacoplan or placebo while continuing to receive their existing
standard of care treatment for ALS for 52 weeks. The primary
endpoint of the study is the Combined Assessment of Function and
Survival (CAFS) rank scores. Key secondary endpoints include
measures of overall function, survival, lung function, and muscle
strength. After 52 weeks, all study participants were to receive
pegcetacoplan. To reduce the burden on people living with ALS and
their caregivers, the study has been designed to minimize the
number of in-clinic visits.
About Amyotrophic Lateral
Sclerosis (ALS)ALS is a devastating neurodegenerative
disease that results in progressive muscle weakness and paralysis
due to the death of nerve cells, called motor neurons, in the brain
and spinal cord.1,2 The death of motor neurons leads to the
progressive loss of voluntary muscle movement required for
speaking, walking, swallowing, and breathing.1,2 There are
currently no approved treatments that stop or reverse the
progression of ALS, which impacts ~225,000 patients worldwide.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 several rare diseases across hematology,
nephrology, and neurology. Pegcetacoplan is approved for the
treatment of paroxysmal nocturnal hemoglobinuria (PNH) as
EMPAVELI® in the United States, Australia, Canada,
and Saudi Arabia and in the European Union and the United Kingdom
as Aspaveli®.
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. With nearly a dozen clinical and pre-clinical programs
underway, we believe we have only begun to unlock the potential of
targeting C3 across many serious diseases. For more information,
please visit http://apellis.com or follow us
on Twitter 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
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:Lissa
Pavluk media@apellis.com 617.977.6764
Investors: Meredith Kaya
meredith.kaya@apellis.com617.599.8178
References1 National Institute of
Neurological Disorders and Stroke. (2020). Amyotrophic Lateral
Sclerosis Fact Sheet. Retrieved from
https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Amyotrophic-lateral-Sclerosis-ALS-Fact-Sheet2 ALS
Association. What is ALS? Retrieved June 2020 from
https://www.als.org/understanding-als/what-is-als3 Arthur K et
al. Nat Commun, 2016, Vol 7, article 12408
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