BOSTON, Nov. 5, 2020 /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 reported financial results for the third quarter
ended September 30, 2020 and provided
a corporate update.
"As we move into the fourth quarter, we are proud that our team
has stayed focused, execution-oriented, excited about our platform
potential, and dedicated to the patients we serve during what has
been a challenging year," said Reenie
McCarthy, chief executive officer of Stealth. "We have
learned important lessons that both inform our future development
of elamipretide for diseases of mitochondrial dysfunction and stoke
our enthusiasm in our deep pipeline of mitochondrial
therapeutics. We are thrilled that with Morningside's
continued financial support under our recently announced
development funding agreement, we are well-positioned to progress
our first-in-class development efforts in 2021."
Third Quarter 2020 Program Highlights
Geographic atrophy. The ReCLAIM-2 Phase 2b clinical trial in patients with geographic
atrophy associated with dry age-related macular degeneration is
approximately 90% enrolled, with complete enrollment targeted for
year-end 2020. Several additional trial sites have been added, and
the Company has implemented best practices measures, including
availability of night and weekend visits and visiting nurses, to
alleviate COVID-19 related challenges. The Company is closely
monitoring any COVID-19 related discontinuations in light of
increased reported incidence in the
United States and may elect to upsize target enrollment if
discontinuations increase during the fourth quarter. If
enrollment is increased, completion of enrollment could extend into
the first quarter of 2021.
Barth syndrome. More than 4,250 members of the
Barth syndrome community have signed a petition asking the FDA and
the Company to work together to provide Barth syndrome patients
access to elamipretide, and for the FDA to review and approve a New
Drug Application (NDA) for elamipretide to treat this ultra-rare
disease. In their petition, patients expressed serious concern
regarding delays anticipated by the FDA's July 2020 Type C written response recommending
that the Company conduct additional controlled clinical trials
prior to NDA submission. In response, the Company has reiterated
its commitment to the Barth community and its plan to submit its
NDA by year-end.
Following FDA's July 2020
recommendation, the Company also plans to submit a protocol for a
randomized withdrawal study of the patients remaining on open-label
extension. While the Company believes that a post-approval,
long-term cardiac outcome trial would be most appropriate to
provide confirmatory evidence of efficacy in this ultra-rare
disease, the randomized withdrawal trial remains a near-term
potential approach to address FDA feedback.
Data from SPIBA-001, the Company's pivotal Phase 3 natural
history control trial, and SPIBA-201, the Company's Phase 2/3
double-blind placebo-controlled crossover trial and open label
extension, were presented at the Barth Syndrome Foundation's
July 2020 Symposium and the American
Society of Genetics in Medicine (ASGM) in October. These data
showed that long-term treatment with elamipretide resulted in
statistically significant improvements from baseline (with respect
to SPIBA-201) and compared to natural history controls (with
respect to SPIBA-001) in cardiac function, metabolic function,
skeletal muscle function, and patient and clinician reported
outcomes. Echocardiographic assessments suggest that
prolonged treatment with elamipretide may lead to cardiac reverse
remodeling. Data from SPIBA-201 were published in Genetics in
Medicine in October 2020.
SBT-272. In a Phase 1 clinical trial, orally
administered SBT-272 showed a favorable safety profile in healthy
human volunteers. With these encouraging results, the Company is
assessing drug exposure to inform decisions regarding oral
formulation and utility of exploring subcutaneous dosing.
Long term toxicology studies are expected to be conducted in 2021
to enable progression into Phase 2 trials.
As previously reported, the Company has been evaluating SBT-272
in neurological disease preclinical models due to its improved
blood brain barrier penetration and higher concentrations in the
central nervous system relative to elamipretide. Data presented at
the 2020 Annual NEALS Meeting in October demonstrated that SBT-272
improved neurite length and branching in mutant TDP43 primary upper
motor neurons. TDP43 pathology has been observed in multiple
neurodegenerative diseases, including ALS, Frontotemporal Lobar
Degeneration (FTLD), Lewy Body Dementia (LBD), Progressive
Supranuclear Palsy (PSP), and Alzheimer's Disease, and is believed
to play a role in neuronal cell death.
Third Quarter 2020 and Recent Financial Highlights
Development Funding Agreement. In November 2020, the Company announced the first
closing under a Development Funding Agreement to support the
clinical development of elamipretide. Under the terms of the
agreement, Stealth has received an upfront $20 million from Morningside Venture (I)
Investments Ltd., and expects to receive up to an additional
$15 million upon achievement of
near-term clinical and regulatory milestones associated with its
geographic atrophy and Barth programs. Additional investors may
contribute up to an additional $35
million in funding commitments at subsequent closings. The
agreement also contemplates up to an additional $35 million in funding upon mutual agreement of
the parties. Stealth is obligated to make milestone payments
following certain regulatory approvals, with most payments due in
the 5th through 7th year following regulatory
approval. No approval payments are owed should regulatory approval
not be achieved for elamipretide in the indications currently under
or planned for near-term development.
Amended Term Loan Facility with Hercules Capital, Inc. In
July 2020, the Company amended its
Term Loan Facility with Hercules Capital, Inc. to defer principal
payment until March 1, 2021.
Key Upcoming Milestones
Barth syndrome: Submission of clinical protocols to
generate post-approval-controlled data and potential NDA submission
anticipated by year-end 2020.
Geographic atrophy: Completion of enrollment of
Phase 2 clinical trial in dry AMD targeted for year-end 2020.
Data expected in early 2022.
Friedreich's ataxia: Phase 2a investigator-initiated
open-label clinical trial assessing elamipretide in a cohort of
patients affected by visual decline and/or cardiomyopathy
associated with Friedreich's ataxia expected to commence in early
2021. The Company anticipates that data from this trial will help
inform pivotal trial design.
Duchenne cardiomyopathy: Initiation of a pivotal trial in
patients with cardiomyopathy associated with Duchenne muscular
dystrophy targeted for the second half of 2021, subject to
discussions with FDA, continued planning efforts, and financing
plans.
Replisome-related mitochondrial myopathies: Initiation of
a pivotal trial in the prespecified subgroup of patients that
responded to elamipretide therapy in the Company's Phase 3 trial in
primary mitochondrial myopathy targeted for the second half of
2021, subject to discussions with FDA, continued planning
efforts, and financing plans.
SBT-272: Results from a study of SBT-272 in a model of
α-synucleinopathy, a disease pathology associated with Parkinson's
disease (PD), LBD, and multiple system atrophy (MSA), will be
presented at an upcoming conference.
SBT-550: Initiation of IND-enabling studies for neurology
candidates within SBT-550 series expected in 2021.
Financial Results for the Three Months Ended September 30, 2020
Cash Position: Cash and cash equivalents were
$19.9 million at September 30, 2020, compared to $50.8 million at December 31, 2019. The
Company received an additional $20.0
million in October 2020 upon
execution of the Development Funding Agreement with Morningside
Venture (I) Investments Limited.
Research and Development (R&D) Expenses: R&D
expenses were $6.2 million for the
three months ended September 30,
2020, compared to $9.8 million
for the same period in 2019. The decrease was primarily due to a
$3.2 million net decrease in
employee and consultant related costs attributable to the strategic
repositioning implemented in the first quarter of 2020, a
$0.7 million decrease in
contract manufacturing, and a $0.5 million decrease in preclinical costs.
These decreased costs were offset in part by a $0.8 million net increase in clinical trial costs
due to timing of trials.
General and Administrative (G&A) Expenses: G&A
expenses were $4.7 million for
the three months ended September 30,
2020, compared to $6.3 million for the same period in 2019.
The decrease in administrative expenses was attributed to a
decrease of $1.2 million in
pre-commercial activities and a $0.8
million net decrease in employee and consultant related
costs, both primarily attributable to the strategic repositioning
implemented in Q1 2020, offset in part by a $0.4 million increase in professional services
and activities attributable to the cost of various financing
transactions and increased cost of insurance.
Other Expense, Net: Other expense was $0.3 million for the three months ended
September 30, 2020, compared to
$0.4 million for the same period
in 2019. The decrease in other expense is primarily attributed to a
$0.3 million decrease in
interest expense offset in part by a $0.2 million decrease in interest
income.
Net Loss: Net loss was $11.2 million, or $0.02 basic and diluted net loss per ordinary
share, for the three months ended September
30, 2020, as compared to $16.5 million, or $0.04 basic and diluted net loss per ordinary
share, for the same period in 2019. The decreased loss was
primarily attributable to a decrease in operating costs of
$5.2 million and a net decrease
in other expenses of $0.1 million.
Conference Call
Management will host a conference call today at 8:30 am ET to discuss the financial results and
provide a general business update. The call can be accessed by
dialing (877) 407-0989 or (201) 389-0921 (international) and
referencing conference ID 13710878. 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 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, such as POLG-related disorders, 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 scaffolds 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' plans, strategies and expectations for its
preclinical and clinical advancement of its drug development
programs, including its ongoing clinical trials of elamipretide and
planned clinical trial of SBT-272; its plans for the potential
submission of an NDA; expectations regarding regulatory
interactions and funding for its plans to initiate additional
clinical trials; the potential benefits of Stealth BioTherapeutics'
product candidates; its key milestones for 2020 and 2021; its plans
regarding future data presentations; and its financial guidance
regarding the period in which it will have capital available to
fund its operations. 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 U.S.
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
Stern Investor Relations
Janhavi Mohite, 212-362-1200
IR@StealthBT.com
STEALTH
BIOTHERAPEUTICS CORP
|
|
Condensed
Consolidated Statements of Operations
|
(in thousands, except share and per share data)
|
(unaudited)
|
|
|
|
Three Months Ended
September 30,
|
|
|
Nine Months Ended
September 30,
|
|
|
|
2020
|
|
|
2019
|
|
|
2020
|
|
|
2019
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and
development
|
|
$
|
6,211
|
|
|
$
|
9,820
|
|
|
$
|
23,463
|
|
|
$
|
33,514
|
|
General and
administrative
|
|
|
4,671
|
|
|
|
6,269
|
|
|
|
14,374
|
|
|
|
16,490
|
|
Total operating
expenses
|
|
|
10,882
|
|
|
|
16,089
|
|
|
|
37,837
|
|
|
|
50,004
|
|
Loss from
operations
|
|
|
(10,882)
|
|
|
|
(16,089)
|
|
|
|
(37,837)
|
|
|
|
(50,004)
|
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Loss on extinguishment
of debt
|
|
|
—
|
|
|
|
—
|
|
|
|
—
|
|
|
|
(22,700)
|
|
Change in fair value
of derivative liability
|
|
|
—
|
|
|
|
—
|
|
|
|
—
|
|
|
|
2,782
|
|
Change in fair value
of warrant liability
|
|
|
—
|
|
|
|
—
|
|
|
|
—
|
|
|
|
(300)
|
|
Interest
income
|
|
|
2
|
|
|
|
223
|
|
|
|
139
|
|
|
|
773
|
|
Interest expense and
other
|
|
|
(330)
|
|
|
|
(664)
|
|
|
|
(1,421)
|
|
|
|
(6,009)
|
|
Total other
expense
|
|
|
(328)
|
|
|
|
(441)
|
|
|
|
(1,282)
|
|
|
|
(25,454)
|
|
Net loss attributable
to ordinary shareholders
|
|
$
|
(11,210)
|
|
|
$
|
(16,530)
|
|
|
$
|
(39,119)
|
|
|
$
|
(75,458)
|
|
Net loss per share
attributable to ordinary shareholders —
basic
and diluted
|
|
$
|
(0.02)
|
|
|
$
|
(0.04)
|
|
|
$
|
(0.07)
|
|
|
$
|
(0.21)
|
|
Weighted average
ordinary shares used in net loss per share
attributable to ordinary shareholders — basic and
diluted
|
|
|
596,900,696
|
|
|
|
420,399,807
|
|
|
|
536,558,283
|
|
|
|
355,634,626
|
|
STEALTH
BIOTHERAPEUTICS CORP
|
|
Condensed
Consolidated Balance Sheets
|
(in thousands)
|
(unaudited)
|
|
|
|
September 30,
|
|
|
December
31,
|
|
|
|
2020
|
|
|
2019
|
|
Assets
|
|
|
|
|
|
|
|
|
Current
assets:
|
|
|
|
|
|
|
|
|
Cash and cash
equivalents(a)
|
|
$
|
19,893
|
|
|
$
|
50,768
|
|
Prepaid expenses and
other current assets
|
|
|
1,759
|
|
|
|
1,630
|
|
Total current
assets
|
|
|
21,652
|
|
|
|
52,398
|
|
Property and
equipment, net
|
|
|
169
|
|
|
|
345
|
|
Deferred financing
costs and other assets
|
|
|
704
|
|
|
|
—
|
|
Total
assets
|
|
$
|
22,525
|
|
|
$
|
52,743
|
|
Liabilities and
shareholders' equity
|
|
|
|
|
|
|
|
|
Current
liabilities:
|
|
|
|
|
|
|
|
|
Accounts
payable
|
|
$
|
3,452
|
|
|
$
|
9,520
|
|
Accrued expenses and
other current liabilities
|
|
|
6,406
|
|
|
|
8,495
|
|
Accrued interest
payable
|
|
|
1,411
|
|
|
|
1,219
|
|
Current portion of
long-term debt
|
|
|
8,982
|
|
|
|
14,716
|
|
Total current
liabilities
|
|
|
20,251
|
|
|
|
33,950
|
|
Long-term debt, less
current portion
|
|
|
—
|
|
|
|
1,526
|
|
Total
liabilities
|
|
|
20,251
|
|
|
|
35,476
|
|
Total
shareholders' equity
|
|
|
2,274
|
|
|
|
17,267
|
|
Total liabilities
and shareholders' equity
|
|
$
|
22,525
|
|
|
$
|
52,743
|
|
|
|
|
|
|
|
|
|
|
|
|
(a)
|
An additional $20.0
million was received in October 2020 pursuant to the first closing
under the Development Funding Agreement with Morningside Venture
(I) Investments Limited.
|
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SOURCE Stealth BioTherapeutics Inc.