BOSTON, May 5, 2021 /PRNewswire/ -- Stealth
BioTherapeutics Corp (Nasdaq: MITO), a clinical-stage biotechnology
company focused on the discovery, development, and
commercialization of novel therapies for diseases involving
mitochondrial dysfunction, today announced the presentation of new
data from a post hoc analysis of the Phase 1 ReCLAIM study
evaluating elamipretide in patients with non-central geographic
atrophy (GA) and high risk drusen associated with dry age-related
macular degeneration (AMD). The data, presented at the 2021
Association for Research in Vision and Ophthalmology (ARVO) Virtual
Annual Meeting, showed that for patients with GA treated with
elamipretide for 24-weeks, improvements from baseline in low light
visual acuity (LLVA), which is the primary efficacy endpoint in
Stealth's fully enrolled Phase 2 ReCLAIM-2 trial, were
significantly correlated to baseline assessments of mitochondrial
health in the ellipsoid zone (EZ). The EZ is a
mitochondria-rich area of the retina which supports photoreceptor
function and is attenuated in dry AMD. EZ imaging assessments
were conducted using optical coherence tomography (OCT) and
included pan-macular EZ-retinal pigment endothelium cell (RPE)
volume and macular percentage of EZ-RPE attenuation.
Improvements in LLVA were significantly correlated to both baseline
EZ-RPE volume and EZ-RPE attenuation, with eyes gaining two or more
lines showing significantly less EZ-RPE attenuation and
percentage area of macular GA at baseline.
"Based on the data presented showing an association between
outer retinal features and treatment response to elamipretide, this
approach to imaging biomarker interrogation may provide a unique
opportunity for clinical trial enrichment and identification of
eyes that are more likely to benefit from treatment," noted
Dr. Justis Ehlers, the Norman C. and Donna L. Harbert Endowed
Chair of Ophthalmic Research at Cole Eye Institute at Cleveland
Clinic. "These findings may suggest that there may be a 'retinal
reserve' that is required and potentially measurable that provides
a greater chance at treatment response."
"It is exciting to confirm the relationship between
elamipretide-mediated improvements in visual function and
mitochondrial health in patients suffering from progressive vision
loss due to GA," said Chief Executive Officer Reenie McCarthy.
"These data support our confidence in our ReCLAIM-2 trial design,
which focuses on the earlier stages of GA before irreversible
mitochondrial and RPE damage is thought to have occurred. We
are encouraged to learn that intervening at this stage of the
disease may not only slow disease progression, but also restore
visual function and visual quality of life to patients suffering
from this life-limiting disease. These learnings may also
offer potential enrichment strategies to inform Phase 3 trial
design following read-out of our ReCLAIM-2 data early next
year."
In November 2018, the U.S. Food and Drug
Administration granted Fast Track designation for elamipretide
for patients with dry AMD with GA.
For additional information on ReCLAIM, ReCLAIM-2 or
elamipretide, please refer to
Stealth's website and ClinicalTrials.gov.
About ReCLAIM and ReCLAIM-2
ReCLAIM-2 is a phase 2 randomized, double-masked,
placebo-controlled study to evaluate the efficacy and
pharmacokinetics of elamipretide in patients with dry age-related
macular degeneration (AMD) with geographic atrophy (GA). The
ReCLAIM-2 study completed enrollment with 176 patients. The primary
endpoint of the 48-week study will measure the low-luminance
best-corrected visual acuity (LLVA), which assesses visual function
under low light conditions meant to represent dusk or indoor
(artificial) lighting. Secondary functional endpoints are change in
low-luminance reading acuity and best-corrected visual acuity
(BCVA). Secondary imaging endpoints assessing the rate of
progression of the disease include GA area as measured by fundus
autofluorescence and optical coherence tomography (OCT).
ReCLAIM was a Phase 1 clinical trial which enrolled 40 patients
with dry AMD with either GA or high-risk drusen to receive
elamipretide therapy for 24 weeks. The data showed
improvements from baseline in LLVA and BCVA for patients with GA
and drusen who completed the trial. Visual quality of life
improvement under both low light and ordinary light conditions were
also reported.
About Age-Related Macular Degeneration
AMD, a progressive eye condition that is the leading cause of
blindness in adults, is estimated to affect approximately 10
million people in the U.S. AMD affects the center portion
of the retina, called the macula, which is responsible for central
vision and color perception. Although there are FDA-approved
treatments for wet AMD, which affects approximately 10 percent of
those suffering from the disease, there are no approved therapies
for dry AMD. Dry AMD with GA, an advanced form of dry AMD, is
characterized by central blind spots leading to permanent loss of
vision. The disease is a major contributor to loss of
independence and diminished quality of life in older persons.
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 heart, the eye, and the brain. We believe our lead product
candidate, elamipretide, has the potential to treat both rare
metabolic cardiomyopathies, such as Barth, Duchenne muscular
dystrophy and Friedreich's ataxia, rare mitochondrial diseases
entailing nuclear DNA mutations, as well as ophthalmic diseases
entailing mitochondrial dysfunction, such as dry age-related
macular degeneration and Leber's hereditary optic neuropathy. We
are evaluating our second-generation clinical-stage candidate,
SBT-272, and our new series of small molecules, SBT-550, for rare
neurological disease indications 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, but are not limited
to, statements relating to preliminary clinical data. 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: those regarding Stealth BioTherapeutics' plans,
strategies and expectations for its preclinical and clinical
advancement of its drug development programs, including its ongoing
clinical trials of elamipretide; whether the company's clinical
trials will be fully enrolled and completed when anticipated;
whether results obtained in preclinical studies and clinical trials
will be indicative of results that will be generated in future
clinical trials; whether elamipretide will successfully advance
through the clinical trial process on a timely basis, or at all;
whether the results of the company's clinical trials will warrant
regulatory submissions and whether elamipretide will receive
approval from the FDA or equivalent foreign regulatory agencies for
GA, AMD, Barth syndrome or any other indication when expected or at
all; whether, if Stealth BioTherapeutics' products receive
approval, they will be successfully distributed and marketed; its
expectations regarding regulatory interactions; the potential
benefits of Stealth BioTherapeutics' product candidates; its key
milestones for 2021 and 2022; and its plans regarding future data
presentations; 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 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") on April 6, 2021, 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
Stern Investor Relations
Janhavi Mohite, 212-362-1200
IR@StealthBT.com
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SOURCE Stealth BioTherapeutics Inc.