U.S. Food and Drug Administration (FDA) Grants
Fast Track Designation for Belzupacap Sarotalocan (bel-sar) for the
Treatment of Choroidal Metastasis, Bel-sar’s Second Ocular Oncology
Indication to Receive this Designation
Global Phase 3 Trial in Primary Choroidal
Melanoma on Track to Begin Dosing in 1H 2023
Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage
biotechnology company developing a novel class of virus-like drug
conjugate (VDC) therapies for multiple oncology indications, today
reported financial results for the fourth quarter and year ended
December 31, 2022, and provided clinical development and
operational highlights.
“2023 is off to a strong start with positive interim Phase 2
safety and efficacy data with suprachoroidal administration in
early-stage choroidal melanoma. In February, at the Macula
Society’s 46th Annual Meeting, we presented average nine-month
interim data which strongly supports the assumptions for the
success of the global Phase 3 trial, which is on track to dose the
first patient in the first half of this year,” said Elisabet de los
Pinos, Ph.D., Chief Executive Officer of Aura. “We are also excited
to initiate clinical development in our second ocular oncology
indication, choroidal metastasis, and to report early Phase 1 data
in our non-muscle invasive bladder cancer program in the second
half of this year. Our recently strengthened balance sheet
positions us well to execute and advance our pipeline to meaningful
clinical milestones across ocular and urologic oncology.”
“We are excited that bel-sar was granted Fast Track Designation
for choroidal metastasis. There is an important opportunity to
develop a new standard of care as we see a large number of patients
with this type of metastasis,” said Dr. Cadmus Rich, Chief Medical
Officer of Aura Biosciences. “This is the second Fast Track
Designation bel-sar has obtained for an ocular oncology indication,
which highlights the need for vision preserving treatment
options.”
Recent Pipeline Developments
- Bel-sar is being developed for the first-line treatment of
early-stage choroidal melanoma (CM), a life-threatening rare
disease with no approved therapies.
- Positive interim Phase 2 data evaluating suprachoroidal (SC)
administration of bel-sar for the first-line treatment of adult
patients with early-stage CM was presented at the Macula Society
46th Annual Meeting. The results reported as of January 10,
2023, with an average of nine months of follow up in patients
similar to the planned Phase 3 population, who received three
cycles of therapy (n=8), showed a statistically significant
reduction in the tumor growth rate (-0.289 mm/yr, p = <0.0001)
compared to each patient’s documented growth rate at study entry,
and a 100% tumor control rate. In addition, only one patient lost
visual acuity in these cohorts, with the majority of patients being
at high-risk for vision loss with tumors close to fovea or optic
disk. The overall tolerability profile of bel-sar was generally
favorable, with no dose-limiting toxicities, treatment-related
serious adverse effects (SAEs) or significant adverse events (AEs).
There was no posterior inflammation and mild anterior inflammation
(Grade 1) in 25% of the patients. Treatment-related AEs were
predominantly mild and resolved without sequalae. We believe these
interim results indicate that bel-sar may offer a targeted, vision
preserving therapy for the first-line treatment of early-stage CM,
where 80% of patients are diagnosed early and have no approved
therapies to date.
- Aura finalized the global Phase 3 trial design in alignment
with regulatory agencies and selected SC route of administration to
evaluate the efficacy and safety of bel-sar in early-stage CM.
The global Phase 3 trial is randomized and masked and will include
three arms, where the primary endpoint will be a time to event
composite endpoint that will compare the tumor control and visual
acuity of the intervention group with high dose of bel-sar to sham.
Aura is planning to enroll approximately 85 adult patients with
early-stage CM, including patients with indeterminate lesions and
small choroidal melanoma. Patients with documented growth will be
enrolled as an enrichment strategy intended to increase the
efficiency of the trial, which will also include an adaptive design
to further increase the probability of success.
- Aura is enrolling a Phase 1 clinical trial of bel-sar for
the treatment of non-muscle invasive bladder cancer (NMIBC).
This represents an area of high unmet need with approximately
80,000 patients diagnosed in the United States every year. Aura
received Fast Track Designation from the Oncology Division of the
FDA for this indication in June 2022.
- The Phase 1 multi-center, open-label clinical trial is expected
to enroll approximately 23 adult patients. The trial is designed to
assess the safety and tolerability of bel-sar as a single agent.
The primary endpoint of the Phase 1 clinical trial is the incidence
and severity of treatment-related AEs and SAEs and the incidence of
dose-limiting toxicities. The goal of this study is to demonstrate
distribution, local necrosis and evidence of immune activation.
Aura expects to report initial Phase 1 data in the second half of
2023.
- Beyond early-stage CM, Aura continues to build its ocular
oncology franchise. Aura’s goal is to initiate clinical
development in choroidal metastasis, an indication with an unmet
medical need and no approved therapies, as the second ocular
oncology indication. Aura received Fast Track Designation from the
Oncology Division of the FDA for this indication in February 2023,
and the Investigational Drug application was opened in January
2023.
- Nonclinical data supporting bel-sar’s broad tumor targeting
potential and immune mediated mechanism of action was presented at
the 22nd EURETINA Congress. Preclinical results highlighted
bel-sar’s targeted cytotoxicity towards tumor cells derived from
the most common cancer types known to metastasize to the choroid,
supporting its potential use for the treatment of choroidal
metastasis, a key second ocular oncology indication. The
presentation also included nonclinical data that supported the
activity of bel-sar as a single agent as well as in combination
with checkpoint inhibitors, highlighting the possibility to treat
not only primary tumors in the eye but also potentially distant
metastases by an abscopal effect.
Recent Corporate Events
- Raised Gross Proceeds of $92.5 Million in Oversubscribed
Follow-on Public Offering. In December 2022, Aura announced the
closing of an oversubscribed underwritten follow-on public offering
yielding aggregate gross proceeds of approximately $92.5 million.
All of the shares in the offering were offered by Aura.
Full Year and Fourth Quarter 2022 Financial Results
- As of December 31, 2022, Aura had cash and cash equivalents and
marketable securities totaling $188.8 million. Aura believes its
current cash and cash equivalents and marketable securities are
sufficient to fund its operations into 2025.
- Research and development expenses increased to $13.2 million
and $42.2 million for the three months and full year ended December
31, 2022, respectively, from $8.0 million and $25.2 million for the
three months and full year ended December 31, 2021, respectively,
primarily due to ongoing clinical costs associated with the
progression of Aura’s Phase 2 study and clinical research
organization costs associated with the start of Aura’s Phase 3
global trial, manufacturing and development costs for bel-sar, and
higher personnel expenses from growing headcount.
- General and administrative expenses increased to $4.5 million
and $18.1 million for the three months and full year ended December
31, 2022, respectively, from $3.6 million and $10.1 million for the
three months and full year ended December 31, 2021, respectively.
General and administrative expenses include $1.1 million and $0.9
million of stock-based compensation for the three months ended
December 31, 2022 and 2021, respectively. The increase was
primarily driven by personnel expenses, as well as increases in
general corporate expenses related to a full year of operating as a
public company.
- Net loss for the three months and full year ended December 31,
2022, was $16.6 million and $58.8 million, respectively, compared
to $11.6 million and $35.3 million for the three months and full
year ended December 31, 2021, respectively.
About Aura Biosciences
Aura Biosciences, Inc., is a clinical-stage biotechnology
company developing virus-like drug conjugates (VDCs), a novel class
of therapies, for the treatment of multiple oncology indications.
Aura’s lead VDC candidate, belzupacap sarotalocan (bel-sar;
AU-011), consists of a virus-like particle conjugated with an
anti-cancer agent. Bel-sar is designed to selectively target and
destroy cancer cells and activate the immune system with the
potential to create long-lasting anti-tumor immunity. Bel-sar is
currently in development for ocular cancers, and Aura has initiated
activities for the global Phase 3 trial evaluating first-line
treatment of early-stage choroidal melanoma, a vision- and
life-threatening form of eye cancer where standard of care with
radiotherapy leaves patients with severe comorbidities, including
major vision loss. Aura plans to pursue development of bel-sar
across its ocular oncology franchise including for the treatment of
patients with choroidal metastasis. In addition, leveraging Aura’s
technology platform, Aura is developing bel-sar more broadly across
multiple cancers, including in patients with non-muscle invasive
bladder cancer. Aura is headquartered in Boston, MA.
For more information, visit aurabiosciences.com, or follow us on
Twitter and LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995, as amended, and other federal securities laws. Any statements
that are not statements of historical fact may be deemed to be
forward looking statements. Words such as “may,” “will,” “could”,
“should,” “expects,” “intends,” “plans,” “anticipates,” “believes,”
“estimates,” “predicts,” “projects,” “seeks,” “endeavor,”
“potential,” “continue” or the negative of such words or other
similar expressions that can be used to identify forward-looking
statements. These forward looking statements include express or
implied statements regarding Aura’s future expectations, plans and
prospects, including, without limitation, statements regarding the
therapeutic potential of bel-sar for the treatment of cancers
including choroidal melanoma, non-muscle invasive bladder cancer
and choroidal metastasis; any express or implied statements
regarding the Company’s expectations for the Phase 2 and Phase 3
clinical trials of bel-sar for early-stage choroidal melanoma and
the Phase 1 trial of bel-sar for non-muscle invasive bladder
cancer; and Aura’s expectations regarding the estimated patient
populations and related market opportunities for bel-sar.
The forward-looking statements in this press release are neither
promises nor guarantees, and investors should not place undue
reliance on these forward-looking statements because they involve
known and unknown risks, uncertainties, and other factors, many of
which are beyond Aura’s control and which could cause actual
results to differ materially from those expressed or implied by
these forward-looking statements, including, without limitation, an
improved quality of life of patients after treatment with bel-sar;
a potential paradigm shift in the approach to the treatment of
choroidal melanoma; the urgent need for a vision preserving
targeted therapy; the potential of bel-sar compared to the existing
standard of care for patients with choroidal melanoma;
uncertainties inherent in clinical trials and in the availability
and timing of data from ongoing clinical trials; the expected
timing for submissions for regulatory approval or review by
governmental authorities; the risk that the results of Aura’s
clinical trials may not be predictive of future results in
connection with future clinical trials; the risk that interim data
from ongoing clinical trials may not be predictive of final data
from completed clinical trials; whether Aura will receive
regulatory approvals to conduct trials or to market products;
whether Aura’s cash resources will be sufficient to fund its
foreseeable and unforeseeable operating expenses and capital
expenditure requirements; risks, assumptions and uncertainties
regarding the impact of the continuing COVID-19 pandemic on Aura’s
business, operations, strategy, goals and anticipated timelines;
Aura’s ongoing and planned pre-clinical activities; and Aura’s
ability to initiate, enroll, conduct or complete ongoing and
planned clinical trials. These risks, uncertainties, and other
factors include those risks and uncertainties described under the
heading “Risk Factors” in Aura’s most recent Annual Report on Form
10-K filed with the U.S. Securities and Exchange Commission (SEC)
and in subsequent filings made by Aura with the SEC, which are
available on the SEC’s website at www.sec.gov. Except as required
by law, Aura disclaims any intention or responsibility for updating
or revising any forward-looking statements contained in this press
release in the event of new information, future developments or
otherwise. These forward-looking statements are based on Aura’s
current expectations and speak only as of the date hereof and no
representations or warranties (express or implied) are made about
the accuracy of any such forward-looking statements.
Aura Biosciences, Inc.
Consolidated Statement of
Operations and Comprehensive Loss
(in thousands, except share
and per share amounts)
Year Ended December
31,
2022
2021
Operating Expenses:
Research and development
$
42,238
$
25,161
General and administrative
18,057
10,089
Total operating expenses
60,295
35,250
Total operating loss
(60,295
)
(35,250
)
Other income (expense):
Interest income, including amortization
and accretion income
1,864
13
Realized loss on marketable securities
(9
)
—
Loss on disposal of assets
(318
)
(3
)
Other expense
(5
)
(11
)
Total other income (expense)
1,532
(1
)
Net loss
(58,763
)
$
(35,251
)
Net loss attributable to common
stockholders—basic and diluted
(58,763
)
(46,193
)
Net loss per share attributable to common
stockholders—basic and diluted
(1.96
)
(8.95
)
Weighted average common stock
outstanding—basic and diluted
29,937,228
5,159,973
Comprehensive loss:
Net loss
$
(58,763
)
$
(35,251
)
Other comprehensive items:
Unrealized loss on marketable
securities
(72
)
—
Total other comprehensive loss
(72
)
—
Total comprehensive loss
$
(58,835
)
$
(35,251
)
Aura Biosciences, Inc.
Consolidated Balance
Sheets
(in thousands, except share
and per share amounts)
December 31,
2022
2021
Assets
Current assets:
Cash and cash equivalents
$
121,582
$
149,063
Marketable securities
67,229
—
Restricted cash and deposits
20
23
Prepaid expenses and other current
assets
7,871
4,618
Total current assets
196,702
153,704
Restricted cash and deposits, net of
current portion
768
125
Right of use assets - operating lease
20,671
950
Other long-term assets
423
—
Property and equipment, net
5,371
5,251
Total Assets
$
223,935
$
160,030
Liabilities and Stockholders’
Equity
Current liabilities:
Accounts payable
2,921
2,401
Short-term operating lease liability
2,963
615
Accrued expenses and other current
liabilities
4,573
4,339
Total current liabilities
10,457
7,355
Long-term operating lease liability
17,895
360
Total Liabilities
28,352
7,715
Commitments and Contingencies
Stockholders’ Equity:
Common stock, $0.00001 par value,
150,000,000 authorized at December 31, 2022 and December 31, 2021,
and 37,771,918 and 29,211,643 shares issued and outstanding at
December 31, 2022 and December 31, 2021, respectively
—
—
Additional paid-in capital
406,555
304,452
Accumulated deficit
(210,900
)
(152,137
)
Accumulated other comprehensive loss
(72
)
—
Total Stockholders’ Equity
195,583
152,315
Total Liabilities and Stockholders’
Equity
$
223,935
$
160,030
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230315005674/en/
Investor and Media:
Alex Dasalla Head of Investor Relations and Corporate
Communications adasalla@aurabiosciences.com
Argot Partners Matthew DeYoung aura@argotpartners.com
Aura Biosciences (NASDAQ:AURA)
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