U.S. Food and Drug Administration (FDA)
Guidance in Type C Meeting Supports Global Phase 3 Trial in
Early-stage Choroidal Melanoma
Enrollment Complete in Phase 2 Trial in
Choroidal Melanoma Using Suprachoroidal Route of Administration
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 first quarter ended March 31,
2023, and provided clinical development and operational
highlights.
“We are encouraged by our recent interactions with the FDA in
support of our global Phase 3 trial designed to enable us to
develop the first vision preserving targeted therapy for the
treatment of patients with early-stage choroidal melanoma, a
disease with a high unmet medical need and no approved therapies,”
said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura.
“With a strong balance sheet, we are well-positioned to execute and
advance our pipeline to meaningful clinical milestones.”
Recent Pipeline Developments
- Aura is planning to initiate a potentially
registration-enabling Phase 3 clinical trial in 1H 2023 to evaluate
the safety and efficacy of Belzupacap Sarotalocan (bel-sar) for the
first-line treatment of adult patients with early-stage choroidal
melanoma (CM), a life-threatening rare disease with no approved
therapies.
- The Phase 3 clinical trial design incorporates guidance and
feedback from the FDA following a recent Type C meeting.
- The FDA recommended that the Phase 3 trial follow a standard
three-arm randomized, controlled and masked design. The trial is
intended to enroll approximately 100 patients and it will be
randomized 2:1:2 to receive investigational therapeutic regimen
bel-sar, low dose regimen bel-sar or a sham control. The primary
efficacy analysis is planned to be a time to event composite
endpoint that will compare the tumor control and visual acuity of
the therapeutic regimen group to sham when the last patient meets
12 months of follow up.
- Enrollment is complete in the Phase 2 trial evaluating
suprachoroidal (SC) administration of bel-sar for the first-line
treatment of adult patients with early-stage CM. Updated
interim data of patients treated with the therapeutic regimen
intended to be used in the Phase 3 trial is on track to be
presented in 2H 2023.
- Enrollment is ongoing for the Phase 1 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 trial is the incidence and
severity of treatment-related adverse events, serious adverse
events and/or the incidence of dose-limiting toxicities. The trial
will provide histopathological evaluation after the local treatment
to support bel-sar’s biological activity. Aura expects to report
initial Phase 1 data in 2H 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 a high
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 New Drug application was opened in January
2023. Aura is on track to initiate start-up activities for the
Phase 2 trial in 2H 2023.
Recent Corporate Events
- Enhanced Senior Leadership Team. In March 2023, Aura
appointed Patrick Nealon as SVP, Clinical Development Operations.
Mr. Nealon brings over 20 years of biopharmaceutical industry
experience, leading the clinical development of therapeutics across
multiple disease areas. Mr. Nealon will be responsible for
overseeing all aspects of clinical operations as Aura transitions
into late-stage clinical development.
First Quarter 2023 Financial Results
- As of March 31, 2023, Aura had cash and cash equivalents and
marketable securities totaling $173.5 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 $14.4 million
for the three months ended March 31, 2023 from $8.3 million for the
three months ended March 31, 2022, primarily due to ongoing
clinical costs associated with the progression of our Phase 2 study
and CRO costs associated with the start of our global Phase 3
trial, manufacturing and development costs for bel-sar, and higher
personnel expenses from growing headcount.
- General and administrative expenses increased to $5.0 million
for the three months ended March 31, 2023 from $4.5 million for the
three months ended March 31, 2022. General and administrative
expenses include $1.1 million and $1.0 million of stock-based
compensation for the three months ended March 31, 2023 and 2022,
respectively. The increase was primarily driven by personnel
expenses, as well as increases in general corporate expenses
related to growth of the Company.
- Net loss for the three months ended March 31, 2023 was $17.5
million compared to $12.8 million for the three months ended March
31, 2022.
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; the potential approvability of bel-sar; the Phase 2 trial
of bel-sar for choroidal metastasis; Aura’s expectations regarding
the estimated patient populations and related market opportunities
for bel-sar; and Aura’s expectations regarding cash runway.
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,
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; the risk that governmental
authorities may disagree with Aura’s clinical trial designs, even
after governmental authorities have reviewed and commented on such
clinical trial designs; 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 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.
Condensed Consolidated
Statement of Operations and Comprehensive Loss
(in thousands, except share
and per share amounts)
Three Months Ended
March 31,
2023
2022
Operating Expenses:
Research and development
$
14,405
$
8,276
General and administrative
5,039
$
4,535
Total operating expenses
19,444
12,811
Total operating loss
(19,444
)
(12,811
)
Other income (expense):
Interest income, including amortization
and accretion income
1,991
25
Loss on disposal of assets
0
(5
)
Other income (expense)
(13
)
(44
)
Total other income (expense)
1,978
(24
)
Net loss
(17,466
)
(12,835
)
Net loss per common share—basic and
diluted
(0.46
)
(0.44
)
Weighted average common stock
outstanding—basic and diluted
37,784,282
29,213,632
Comprehensive loss:
Net loss
$
(17,466
)
$
(12,835
)
Other comprehensive items:
Unrealized gain (loss) on marketable
securities
$
27
$
(5
)
Total other comprehensive gain (loss)
27
(5
)
Total comprehensive loss
$
(17,439
)
$
(12,840
)
Aura Biosciences, Inc.
Condensed Consolidated Balance
Sheets
(in thousands, except share
and per share amounts)
March 31, 2023
December 31, 2022
Assets
Current assets:
Cash and cash equivalents
$
38,492
$
121,582
Marketable securities
135,030
67,229
Restricted cash and deposits
20
20
Prepaid expenses and other current
assets
5,579
7,871
Total current assets
179,121
196,702
Restricted cash and deposits, net of
current portion
768
768
Right of use assets - operating lease
20,340
20,671
Other long-term assets
623
423
Property and equipment, net
5,167
5,371
Total Assets
$
206,019
$
223,935
Liabilities and Stockholders’
Equity
Current liabilities:
Accounts payable
1,055
2,921
Short-term operating lease liability
2,985
2,963
Accrued expenses and other current
liabilities
4,067
4,573
Total current liabilities
8,107
10,457
Long-term operating lease liability
17,654
17,895
Total Liabilities
25,761
28,352
Commitments and Contingencies
Stockholders’ Equity:
Common stock, $0.00001 par value,
150,000,000 authorized at March 31, 2023 and December 31, 2022, and
37,800,102 and 37,771,918 shares issued and outstanding at March
31, 2023 and December 31, 2022, respectively
—
—
Additional paid-in capital
408,669
406,555
Accumulated deficit
(228,366)
(210,900)
Accumulated other comprehensive loss
(45)
(72)
Total Stockholders’ Equity
180,258
195,583
Total Liabilities and Stockholders’
Equity
$
206,019
$
223,935
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230511005608/en/
Investor and Media Contact:
Alex Dasalla Head of Investor Relations and Corporate
Communications adasalla@aurabiosciences.com
Aura Biosciences (NASDAQ:AURA)
Historical Stock Chart
From Mar 2024 to Apr 2024
Aura Biosciences (NASDAQ:AURA)
Historical Stock Chart
From Apr 2023 to Apr 2024