Study did not meet primary endpoints of mean
change in low luminance visual acuity (LLVA) and geographic atrophy
(GA) progression
Categorical improvement in LLVA, with >15%
of elamipretide-treated patients gaining 2+ lines of vision at Week
48
Elamipretide demonstrated enhanced ellipsoid
zone preservation through reduction of progressive attenuation, an
important indicator of photoreceptor loss.
Company to host conference call and webcast
today at 8:30 a.m. ET
BOSTON, May 2, 2022
/PRNewswire/ -- Stealth BioTherapeutics (Nasdaq: MITO), a
clinical-stage biotechnology company focused on the discovery,
development and commercialization of novel therapies for diseases
involving mitochondrial dysfunction, today announced top-line data
from its Phase 2 ReCLAIM-2 trial evaluating elamipretide in
patients with geographic atrophy (GA) secondary to dry age-related
macular degeneration. Although the trial did not meet its
primary endpoints assessing mean changes in LLVA and GA, a key
secondary endpoint showed however that elamipretide categorically
improved visual function for patients with GA. Additionally,
elamipretide demonstrated proof of mechanism by reducing
progressive ellipsoid zone loss, a key biomarker of retinal
mitochondrial health which has been shown to be predictive of
long-term GA growth and development. Based on these data, the
Company currently plans to progress development initiatives
intended to better position this indication for potential
partnering consistent with previous guidance.
"The categorical improvement in LLVA is a potentially important
finding for patients with GA, who experience increasing
impairment in their daily functioning due to the otherwise
progressive and irreversible vision loss that characterizes this
disease," said Jeffrey Heier, M.D., principal investigator of
the ReCLAIM-2 study and director, retina service and director,
retinal research, Ophthalmic Consultants of Boston. "This approach shows the
possibility to improve visual function for patients with this
devastating disease, and we look forward to the Company's continued
development efforts."
ReCLAIM-2 was a randomized, double-masked, parallel-group,
placebo-controlled trial to evaluate the efficacy and safety of
elamipretide over 48 weeks in 176 patients with GA.
Elamipretide improved LLVA relative to placebo, although the
mean change (primary endpoint) was not statistically
significant. Administration of elamipretide resulted in a 2
or more line gain in LLVA for greater than 15% of patients
completing the 48-week trial (p=0.04). Although this
improvement in visual function was not associated with reduced GA
progression (primary endpoint), a significant reduction in
ellipsoid zone loss (p=0.0034) was observed.
"We are excited by this finding that elamipretide resulted in
reduction of progressive ellipsoid zone attenuation and may be a
leading indicator for progression to more advanced disease," said
Justis P. Ehlers, M.D., Advanced OCT
Analysis Director for ReCLAIM-2. "We believe that
mitochondrial dysfunction plays an important role in AMD
progression and ellipsoid zone loss on OCT is a potential important
biomarker and endpoint for both intermediate AMD and evaluating
long-term GA progression risk. Advanced technologies for
enabling the quantification of these ellipsoid zone alterations
provide new avenues for better evaluating treatment response and
disease features."
Elamipretide was generally well tolerated in ReCLAIM-2.
Injection site reactions were the most commonly observed adverse
events and in some cases led to early termination. The rate of
new-onset exudations was 5% for patients treated with elamipretide
versus 7% for patients treated with placebo.
"Although we are disappointed that the trial did not meet its
primary endpoints, we are pleased to observe that elamipretide
improved visual function for patients with GA, a finding we also
saw in our Phase 1 ReCLAIM trial," said Reenie McCarthy, CEO of Stealth
BioTherapeutics. "We are also encouraged by this evidence
that elamipretide is improving mitochondrial function in the
retina, which may offer us important enrichment strategies as we
consider pivotal trial design and, importantly, demonstrates in
vivo proof of mechanism. We are committed to making a
meaningful difference for patients living with dry AMD and other
diseases of mitochondrial dysfunction, and we believe that this
demonstration of elamipretide's potential to improve mitochondrial
health in older adults underscores the potential of our
platform."
Conference Call Information
Stealth will host a conference call and webcast today at
8:30 am ET to discuss the ReCLAIM-2
trial. The call can be accessed by dialing 1-877-407-0989
(toll free) or 1-201-389-0921 (international) and
referencing conference ID 13729736. A live audio webcast of
the event can be accessed by visiting the Investors & News
section of Stealth's Investor website,
https://investor.stealthbt.com/. A replay of the webcast will be
archived on Stealth's website for 30 days following the event.
About Geographic Atrophy
Geographic atrophy (GA) is an advanced form of dry age-related
macular degeneration (AMD), a leading cause of blindness. GA
lesions affect the central portion of the retina, known as the
macula, which is responsible for central vision. GA is progressive
and irreversible, leading to central visual impairment and
permanent loss of vision. Based on published studies, more than
five million people have GA globally including an estimated two
million people in the United States. There are currently
no approved treatments for GA.
About Stealth
We are a clinical-stage biotechnology company focused on the
discovery, development, and commercialization of novel therapies
for diseases involving mitochondrial dysfunction. Mitochondria,
found in nearly every cell in the body, are the body's main source
of energy production and are critical for normal organ function.
Dysfunctional mitochondria characterize a number of rare genetic
diseases and are involved in many common age-related diseases,
typically involving organ systems with high energy demands such as
the eye, the neuromuscular system, the heart and the brain. We
believe our lead product candidate, elamipretide, has the potential
to treat ophthalmic diseases entailing mitochondrial dysfunction,
such as dry AMD, rare neuromuscular disorders, such as primary
mitochondrial myopathy and Duchenne muscular dystrophy, and rare
cardiomyopathies, such as Barth syndrome. We are evaluating our
second-generation clinical-stage candidate, SBT-272, for rare
neurological disease indications, such as amyotrophic lateral
sclerosis and frontotemporal lobar dementia, following promising
preclinical data. We have optimized our discovery platform to
identify novel mitochondria-targeted compounds which may be
nominated as therapeutic product candidates or utilized as
mitochondria-targeted vectors to deliver other compounds to
mitochondria.
Forward-looking Statements
This press release contains
forward-looking statements within the meaning of The Private
Securities Litigation Reform Act of 1995. Such forward-looking
statements include those regarding Stealth
BioTherapeutics' expectations for its clinical development of
elamipretide for GA associated with dry AMD and other indications,
the potential benefits of elamipretide and potential regulatory
interactions. Statements that are not historical facts, including
statements about Stealth BioTherapeutics' beliefs, plans
and expectations, are forward-looking statements. The words
"anticipate," "expect," "hope," "plan," "potential," "possible,"
"will," "believe," "estimate," "intend," "may," "predict,"
"project," "would" and similar expressions are intended to identify
forward-looking statements, although not all forward-looking
statements contain these identifying words. Stealth
BioTherapeutics may not actually achieve the plans, intentions
or expectations disclosed in these forward-looking statements, and
you should not place undue reliance on these forward-looking
statements. Actual results or events could differ materially from
the plans, intentions and expectations disclosed in the
forward-looking statements as a result of known and unknown risks,
uncertainties and other important factors, including: Stealth
BioTherapeutics' ability to obtain additional funding and to
continue as a going concern; the impact of the COVID-19 pandemic;
the ability to successfully demonstrate the efficacy and safety
of Stealth BioTherapeutics' product candidates and future
product candidates; the preclinical and clinical results
for Stealth BioTherapeutics' product candidates, which
may not support further development and marketing approval; the
potential advantages of Stealth BioTherapeutics' product
candidates; the content and timing of decisions made by the FDA,
the EMA or other regulatory authorities, investigational review
boards at clinical trial sites and publication review bodies, which
may affect the initiation, timing and progress of preclinical
studies and clinical trials of Stealth BioTherapeutics product
candidates; Stealth BioTherapeutics' ability to obtain
and maintain requisite regulatory approvals and to enroll patients
in its planned clinical trials; unplanned cash requirements and
expenditures; competitive factors; Stealth
BioTherapeutics' ability to obtain, maintain and enforce
patent and other intellectual property protection for any product
candidates it is developing; and general economic and market
conditions. These and other risks are described in greater detail
under the caption "Risk Factors" included in the Stealth
BioTherapeutics' most recent Annual Report on Form 20-F filed with
the Securities and Exchange Commission ("SEC"), as well
as in any future filings with the SEC. Forward-looking
statements represent management's current expectations and are
inherently uncertain. Except as required by law, Stealth
BioTherapeutics does not undertake any obligation to update
forward-looking statements made by us to reflect subsequent events
or circumstances.
Investor Relations
Kendall Investor Relations
Adam Bero, 617-914-0008
IR@StealthBT.com
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SOURCE Stealth BioTherapeutics Inc.