BOSTON, May 18, 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 reported financial results for the
quarter ended March 31, 2021 and
announced recent business highlights.
"Our team is energized by the potential of both elamipretide and
our deep pipeline of novel mitochondrial-targeted compounds to
address the devastating and typically progressive disease burden
associated with diseases of mitochondrial dysfunction and we are
thrilled to have secured additional financial resources to expand
these development efforts," said Reenie
McCarthy, Chief Executive Officer at Stealth. "We are
pleased with our progress towards alignment with the Division of
Cardiology and Nephrology at the FDA on a randomized withdrawal
trial of the patients remaining in our open-label extension trial,
which preserves a potential near-term path to NDA submission for
Barth cardiomyopathy, and we are eager to engage with DRDMG
regarding our Phase 3 protocol for the group of patients with nPMD
who responded to therapy in our previous primary mitochondrial
myopathy trial. As we complete dosing in our fully enrolled Phase 2
trial in geographic atrophy, we are encouraged by new data
correlating visual improvement in our Phase 1 trial with
mitochondrial function, which further supports our Phase 2
enrollment criteria. We are expanding our development efforts both
clinically and preclinically, with continued progress toward
initiating trials in Friedreich's ataxia and cardiomyopathy
associated with Duchenne muscular dystrophy, to explore the full
potential of our candidates, including several new pipeline
compounds central to our burgeoning neurology franchise."
First Quarter 2021 and Recent Highlights
- Barth syndrome. The Company has had
multiple recent meetings with senior Food and Drug Administration
(FDA) officials at the Office level and at the Division of
Cardiology and Nephrology (DCN), regarding its Barth cardiomyopathy
program, with meetings requested by the Company held in
November 2020 and February 2021, and a meeting requested by the FDA
held in April 2021. After further discussions with Barth
Syndrome Foundation and the principal investigator following the
April 2021 meeting, the Company has
agreed with DCN's recommendation to conduct another Phase 3 trial
in which eligible patients remaining in the Company's open label
extension trial would be randomized to elamipretide or placebo
until a minimum number of clinical events occur (i.e., events
leading to treatment failure), whereupon patients would be allowed
to resume elamipretide therapy. The Company has advised DCN that
its recommendation to enroll additional patients other than those
already in the Company's open label extension trial would not be
practicable due to the ultra-rare nature of the disease. The
Company expects to enroll the trial during the third quarter of
this year, which could lead to data as early as the first quarter
of 2022 subject to the timing of requisite clinical
events.
- The Company is undertaking initiatives to advance toward an
anticipated Marketing Authorization Application (MAA) to the EMA's
Committee for Medicinal Products for Human Use (CHMP). These
efforts are supportive of the Company's plans to partner European
rights to elamipretide for the treatment of Barth to a partner best
suited to ensure that European patients affected by this disease
will have timely access to therapy, if approved.
- The Company recently announced a moderated poster presentation
of its Barth syndrome data showing improved cardiac function after
long-term treatment with elamipretide at the American College of
Cardiology's 2021 Scientific Session and Expo in May 2021.
- Geographic atrophy. In February
2021, the Company announced that it had completed enrollment
of 176 patients in its ReCLAIM-2 Phase 2b clinical trial in patients with geographic
atrophy associated with dry age-related macular degeneration. This
milestone triggered the payment of an additional $10 million of development funding to the Company
under the Development Funding Agreement announced in November 2020. Design and preliminary baseline
demographics for the ReCLAIM-2 trial were presented at
Angiogenesis, Exudation, and Degeneration in February 2021.
More recently, data presented at the Association for Research in
Vision and Ophthalmology (ARVO) in May
2021 demonstrated that in the Company's Phase 1 ReCLAIM
clinical trial, the relative health of the ellipsoid zone, a
mitochondria-rich portion of the retina, was associated with
patients whose vision improved after six months of elamipretide
therapy. We believe these results connect mitochondrial viability
with the potential therapeutic benefit of elamipretide and supports
the inclusion criteria for ReCLAIM-2. Data from ReCLAIM-2 is
expected during the first half of 2022.
- nPMD. The Company has a Type C meeting scheduled
with DRDMG during the third quarter of 2021 to discuss its planned
Phase 3 clinical trial in patients with mitochondrial disease
associated with pathogenic nDNA mutations (nPMD), the prespecified
subgroup in which a favorable response was observed in the
Company's MMPOWER-3 clinical trial. The Company plans to initiate
this Phase 3 global trial by the end of 2021.
- Financial updates. In February
2021, the Company received gross proceeds of $4.7 million from the registered direct offering
of its ADSs. In April 2021, the
Company amended its Term Loan Facility with Hercules Capital, Inc.
to defer ongoing amortization payments and extend the maturity date
until January 2022. In May
2021, the Company announced additional commitments of
$30.0 million under the Development
Funding Agreement with Morningside Venture (I) Investments Limited
("Morningside"). The additional funding was triggered by new
analyses of data from the Company's ReCLAIM clinical trial and
MMPOWER-3 clinical trial which the Company and Morningside agreed
was positive and supportive of further development efforts in
geographic atrophy, where intravitreal formulation work is ongoing,
and nPMD, where a Phase 3 trial is planned.
Key Upcoming Milestones
- Barth: Enrollment of a Phase 3 randomized
withdrawal trial is expected during the third quarter 2021 and data
is anticipated as early as year-end (subject to timing of clinical
events).
- Geographic atrophy: Data from the Company's
ReCLAIM-2 trial is expected during the second quarter of
2022. The Company also expects to have further data regarding
feasibility of its ongoing intravitreal formulation development
efforts commensurate with Phase 2 data read-out.
- Initiation of Phase 3 clinical trial in nPMD: The
Company is scheduled to meet with DRDMG during the third quarter of
2021 prior to initiating a Phase 3 clinical trial in the
prespecified subgroup of patients with nPMD who appeared to respond
to elamipretide therapy in the Company's MMPOWER-3 trial. The
Company expects to initiate a new Phase 3 trial in this enriched
patient population by year end.
- Expansion of cardiomyopathy franchise: A 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 is
expected to commence next quarter. The Company anticipates that
data from this trial will help inform pivotal trial design. A
meeting with the FDA to discuss protocol design for a trial to
evaluate elamipretide in patients with cardiomyopathy associated
with Duchenne muscular dystrophy is expected during the second half
of 2021.
- Expansion of neurology franchise: The Company is
continuing to advance its neurology pipeline expansion efforts with
SBT-272 and a group of compounds from its SBT-550 series and
expects to announce results of SBT-272 preclinical studies and
initiation of SBT-550 pre-IND enabling studies during 2021.
Financial Results for the quarter ended March 31, 2021
Cash Position: Cash and cash equivalents were
$32.1 million at March 31, 2021, compared to $32.8 million at December
31, 2020. In May 2021,
the Company received an additional commitment of $30.0 million under the Development Funding
Agreement with Morningside, payable $8.0 million during the second quarter of 2021
and $22.0 million during the fourth
quarter of 2021. The Company is also eligible to receive an
additional $5.0 million upon
submission of its Barth NDA, which is currently anticipated during
the first half of 2022. The Company expects that its cash, cash
equivalents and investments as of March 31,
2021, together with the $30
million in expected proceeds to be received under the
amendment to the Development Funding Agreement, will be sufficient
to enable it to fund its planned operations into the second quarter
of 2022.
Research and Development (R&D) Expenses: R&D
expenses were $6.1 million for the
three months ended March 31, 2021,
compared to $9.8 million for the same
period in 2020.
The decrease was due to a net decrease of $1.7 million in clinical costs primarily driven
by the closeout of our Primary Mitochondrial Myopathy development
efforts, a decrease of $1.3 million
in employee related costs due to the strategic repositioning in
2020 and a decrease of $1.0 million
in manufacturing costs offset in part by a $0.2 million net increase in regulatory costs and
a $0.1 million increase in
preclinical costs.
General and Administrative (G&A)
Expenses: G&A expenses were $5.0 million for the three months ended
March 31, 2021, compared to
$5.2 million for the same period in
2020. The decrease was primarily attributable to a net
decrease of $0.5 million in employee
and consultant related costs offset in part by $0.3 million for increased costs of
insurance.
Other Income (Expense): Other income was
$3.4 million for the three months
ended March 31, 2021, compared to
other expense of $0.5 million for the
same period in 2020. Other income in 2021 consisted of a
$3.7 million gain due to the change
in fair value of the derivative liability offset by $0.3 million in interest expense. Other expense
in 2020 consisted of a $0.6 million
in interest expense offset by $0.1
million in interest income.
Net Loss: Net loss was $7.7
million, or $0.01 basic and
diluted net loss per ordinary share, for the three months ended
March 31, 2021, as compared to
$15.5 million, or $0.04 basic and diluted net loss per ordinary
share, for the same period in 2020.
Conference Call
Management will host the conference call with a slide deck
presentation 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 (domestic) or (201)-389-0921
(international) and referencing conference ID 13719019. Management
will be presenting slides and can be accessed by
visiting https://www.webcast-eqs.com/register/stealthbio05182021/en.
All dial-in information can be accessed by visiting the Investor
& 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, 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 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; its expectations regarding regulatory
interactions, including its belief that the existing data and the
data from the withdrawal protocol may provide sufficient evidence
to support NDA review; the potential benefits of Stealth
BioTherapeutics' product candidates; its key milestones for 2021
and 2022; 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 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 Balance Sheets
|
(in thousands)
|
(unaudited)
|
|
|
March
31,
|
|
December
31,
|
|
|
2021
|
|
2020
|
Assets
|
|
|
|
|
Current
assets:
|
|
|
|
|
Cash and cash
equivalents
|
|
$
32,060
|
|
$
32,787
|
Prepaid expenses and
other current assets
|
|
1,927
|
|
2,253
|
Total current
assets
|
|
33,987
|
|
35,040
|
Property and
equipment, net
|
|
92
|
|
106
|
Deferred financing
costs and other non-current assets
|
|
702
|
|
702
|
Total
assets
|
|
$
34,781
|
|
$
35,848
|
Liabilities and
shareholders' deficit
|
|
|
|
|
Current
liabilities:
|
|
|
|
|
Accounts
payable
|
|
$
4,620
|
|
$
3,526
|
Accrued expenses and
other current liabilities
|
|
4,123
|
|
7,024
|
Accrued interest
payable
|
|
227
|
|
1,499
|
Current portion of
debt
|
|
7,236
|
|
9,000
|
Total current
liabilities
|
|
16,206
|
|
21,049
|
Long-term deferred
rent, less current portion
|
|
11
|
|
16
|
Development
derivative liability - related party
|
|
30,643
|
|
25,155
|
Total
liabilities
|
|
46,860
|
|
46,220
|
Total shareholders'
deficit
|
|
(12,079)
|
|
(10,372)
|
Total liabilities
and shareholders' deficit
|
|
$
34,781
|
|
$
35,848
|
STEALTH
BIOTHERAPEUTICS CORP
|
Condensed
Consolidated Statements of Operations
|
(in thousands, except share and per share data)
|
(unaudited)
|
|
|
Three months
ending March 31,
|
|
|
2021
|
|
2020
|
|
|
|
|
|
Operating
expenses:
|
|
|
|
|
Research and
development
|
|
$
6,099
|
|
$
9,847
|
General and
administrative
|
|
4,979
|
|
5,180
|
Total operating
expenses
|
|
11,078
|
|
15,027
|
Loss from
operations
|
|
(11,078)
|
|
(15,027)
|
Other income
(expense):
|
|
|
|
|
Change in fair value
of derivative liability
|
|
3,688
|
|
—
|
Interest
income
|
|
1
|
|
123
|
Interest expense and
other
|
|
(300)
|
|
(636)
|
Total other income
(expense)
|
|
3,389
|
|
(513)
|
Net loss attributable
to ordinary shareholders
|
|
$
(7,689)
|
|
$
(15,540)
|
Net loss per share
attributable to ordinary shareholders — basic and
diluted
|
|
$
(0.01)
|
|
$
(0.04)
|
Weighted average
ordinary shares used in net loss per share attributable to ordinary
shareholders — basic and diluted
|
|
652,807,323
|
|
436,720,810
|
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SOURCE Stealth BioTherapeutics Inc.