SBT-272 Phase 1 safety and tolerability data
support further clinical development
SBT-272 was neuroprotective and reduced
neuroinflammation in ALS preclinical model
SBT-272 Granted Orphan Drug Designation for
Treatment of ALS
BOSTON, Nov. 1, 2022
/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 encouraging
Phase 1 data on the safety and tolerability of SBT-272, a
next-generation small molecule designed to reach therapeutic
concentrations in the brain and to restore mitochondrial structure
and function. The Company also announced that positive
preclinical data demonstrating that treatment with SBT-272 provided
neuroprotection of upper motor neurons harboring ALS pathology will
be presented today at the Northeast Amyotrophic Lateral Sclerosis
(NEALS) conference in Clearwater,
FL. The Company recently received Orphan Drug Designation
from the US Food and Drug Administration (FDA) Office of Orphan
Products Development for SBT-272 for the treatment of patients with
ALS.
"The preclinical data demonstrate that SBT-272 improves the
stability and function of mitochondria in upper motor neurons that
are diseased with TDP-43 pathology. This also provides
neuroprotection and reduces neuroinflammation in the motor cortex
of a TDP-43 model of ALS", said Hande Ozdinler, PhD, Associate
Professor of Neurology, Feinberg School of
Medicine, Northwestern University. "There appears to be
compelling support for the therapeutic potential of targeting
mitochondria in ALS and the ongoing clinical development of
SBT-272."
Interim results from the Phase 1 study for SBT-272 demonstrated
that selected doses are anticipated to result in therapeutic
concentrations in the brain based on observed drug levels achieved
in preclinical studies. While final safety analyses are
ongoing, dose levels identified for subsequent clinical evaluation
appear generally safe and well tolerated. These data support
further clinical development of SBT-272.
"We are pleased to receive orphan drug designation from FDA for
SBT-272 for the treatment of ALS, underscoring the urgent need for
innovative new therapies for this devastating disease," said Chief
Executive Officer Reenie McCarthy.
"We are encouraged by the clinical profile of SBT-272 observed to
date and look forward to evaluating its therapeutic potential in
ALS and other neurodegenerative diseases of mitochondrial
dysfunction."
NEALS Conference November 1,
2022
Translational Pharmacology of SBT-272, A Novel
Mitochondria-Targeted Drug for the Treatment of Neurodegenerative
Disease
Poster presentation, November 1,
from 5-7pm
SBT-272 improved mitochondria structure and function and
preserved Upper Motor Neurons with TDP-43 Pathology
Platform presentation, November 3,
11:45 am
About SBT-272
SBT-272 is a novel small molecule that targets the
cardiolipin-rich inner mitochondrial membrane which is crucial for
normal mitochondrial structure and function. Preclinical SBT-272
data have demonstrated mitochondria-protective and neuroprotective
effects in models of ALS, FTD, alpha-synucleinopathy, a rare form
of dementia that is similar to Parkinson's disease, Huntington's disease and ischemic
stroke. We have also observed SBT-272-mediated improvements
in functional assessments, lifespan, inflammation, and reduction of
protein aggregates across certain nonclinical models. Data from a
Phase 1 study evaluating subcutaneous SBT-272 in healthy volunteers
supports further clinical development. We aim to develop SBT-272
for ALS and other neurological diseases of mitochondrial
dysfunction. The Orphan Drug Act was enacted in 1983 to encourage
development of drugs for rare diseases, which are diseases that
affect fewer than 200,000 persons in the
United States. Once granted, Orphan Drug Designation
provides various development benefits for an investigational drug,
including seven-year exclusivity after marketing approval is
received.
About Stealth BioTherapeutics
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.
Safe Harbor Statement
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' expectation for the preclinical and
clinical development of SBT-272 and the ability of the company to
achieve its drug discovery, development, and commercialization
goals. 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 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.
Contact
Kendall Investor Relations
Adam Bero, Ph.D.
abero@kendallir.com
IR@StealthBT.com
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SOURCE Stealth BioTherapeutics Inc.