Received FDA Agreement Under Special Protocol
Assessment (SPA) for the CoMpass Phase 3 Clinical Trial
Positive Clinical Efficacy Updates of Bel-sar
for Early-Stage Choroidal Melanoma from the Ongoing Phase 2
Clinical Trial with Suprachoroidal Administration Presented at AAO
2023
Preliminary Data from Phase 1 Trial in Bladder
Cancer – First Patient Utilizing a Single Dose of Bel-sar with
Light Activation Demonstrated a Clinical Complete Response
Raised Gross Proceeds of $99.0 Million in an
Underwritten Public Offering
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 third quarter ended September
30, 2023, and provided clinical development and operational
highlights.
“In the third quarter we made meaningful progress across our
portfolio,” said Elisabet de los Pinos, Ph.D., Chief Executive
Officer of Aura. “We are encouraged by the agreement with the FDA
under the SPA, as it reinforces the plan for our CoMpass trial and
further supports our goal of having the first approved
vision-preserving therapy for patients with early-stage choroidal
melanoma. The Phase 2 data presented at AAO, with 90% of patients
at twelve months follow-up, show results that are highly consistent
with, and strongly support, the assumptions for the design of the
CoMpass Phase 3 trial.”
Dr. de los Pinos added, “We are pleased with the preliminary
data from our ongoing Phase 1 trial in bladder cancer, where the
first patient who received a single dose of bel-sar with light
activation demonstrated a clinical complete response evidenced by
absence of cancer cells on histopathology, and we look forward to
additional data from the program in mid-2024. With a strong balance
sheet from our recent financing, we are well-positioned to execute
our pipeline to meaningful clinical milestones.”
Recent Pipeline Developments
- The CoMpass trial is designed as a superiority trial comparing
bel-sar versus sham. The trial is a global Phase 3, randomized,
multi-center, masked study, and it is intended to enroll
approximately 100 patients randomized 2:1:2 to receive high dose
regimen of bel-sar, low dose regimen of bel-sar with suprachoroidal
(SC) administration, or a sham control.
- The Company received written agreement from the FDA under an
SPA for the overall design of the CoMpass trial. The primary
endpoint is time to tumor progression and the first key secondary
endpoint is a composite time to event analysis that will compare
the tumor control and visual acuity of the bel-sar high dose
regimen to sham when the last patient completes their 15 months of
follow up.
- The Company is planning to dose the first patient in Q4
2023.
Positive updated Phase 2 data evaluating SC administration of
bel-sar for the first-line treatment of adult patients with
early-stage CM was presented at AAO 2023.
- The results, with 90% of patients at twelve months of follow-up
who received three cycles of therapy in Cohorts 5 and 6, and who
match the criteria for the global Phase 3 trial, showed a tumor
control rate of 80% (8/10) and the visual acuity preservation rate
was 90% (9/10), with the majority of patients being at high-risk
for vision loss with tumors close to the fovea or optic disk. For
the 80% of patients that responded, data showed a statistically
significant reduction in tumor growth rate (-0.382 mm/yr, p =
<0.0001) compared to each patient’s documented growth rate at
study entry. The overall tolerability profile of bel-sar was
favorable, with no dose-limiting toxicities, treatment-related SAEs
or significant AEs reported as of August 3, 2023. There was no
posterior inflammation and only mild anterior inflammation (Grade
1) in ~18% of the patients which was self-limited or resolved with
a short course of topical steroids. Treatment-related AEs were
predominantly mild and resolved without sequelae.
- Beyond early-stage CM, the Company continues to build its
ocular oncology franchise. The Company’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. The Company is on track to initiate the
Phase 2 trial in 2024, with initial data by year-end 2024.
- The Phase 1 trial of bel-sar for the treatment of muscle
invasive bladder cancer (MIBC) and non-muscle invasive bladder
cancer (NMIBC) is currently ongoing, and the Company expects to
report data in mid-2024. This represents an area of high unmet
need with approximately 80,000 patients diagnosed in the United
States every year. The Company received Fast Track Designation from
the Oncology Division of the FDA for NMIBC in June 2022.
- The FDA has allowed an amendment to the protocol of the ongoing
Phase 1 trial allowing the inclusion of adult patients with MIBC,
in addition to NMIBC.
- The ongoing Phase 1 multi-center, open-label clinical trial is
expected to enroll approximately 19 adult patients. The trial is
designed to assess the safety and tolerability of bel-sar as a
single agent. The trial will provide histopathological evaluation
after the local treatment to assess bel-sar’s biological activity.
The trial has completed enrollment of the cohort that received
bel-sar injection without light activation. Protocol mandated
safety review found no safety issues and the study has proceeded to
the bel-sar injection plus light activation cohorts. Preliminary
data from the first patient in the light activated cohort of the
trial, utilizing a single dose of bel-sar with light activation,
demonstrated a clinical complete response demonstrated by absence
of cancer cells on histopathology with evidence of extensive
necrosis and immune activation.
- The Company expects to provide additional data in
mid-2024.
Recent Corporate Events
- Raised Gross Proceeds of $99.0 million in an underwritten
public offering. In November 2023, the Company announced the
pricing of an underwritten public offering of 11,000,000 shares of
its common stock at a price to the public of $9.00 per share. The
offering closed on November 9, 2023. In addition, Aura has granted
the underwriters a 30-day option to purchase up to 1,650,000
additional shares of common stock at the public offering price,
less the underwriting discount.
- Strengthened the clinical leadership team with the following
key appointments:
- J. Jill Hopkins, M.D., appointed as Chief Medical Officer and
President of Research and Development. Dr. Hopkins previously
served as Senior Vice President, Global Head of Ophthalmology and
Exploratory Development at Novartis, and Chief Executive Officer of
Gyroscope Therapeutics, a Novartis Company. Dr. Hopkins brings over
30 years of cross-sector experience in ophthalmology, spanning
clinical care, academia, education, industry, advocacy and
innovation.
- Mark Plavsic, Ph.D., appointed as Chief Technology Officer. Dr.
Plavsic previously served as Chief Technology Officer at Fate
Therapeutics, a clinical-stage biopharmaceutical company dedicated
to bringing a first-in-class pipeline stem celled-derived cellular
immunotherapies to patients with cancer and autoimmune disorders.
Dr. Plavsic brings 30 years of global biopharmaceutical experience
including end-to-end technical operations in the United States,
Europe, and Australasia and successful translation and scale-up of
complex biologics from preclinical development through commercial
launch and distribution.
Third Quarter 2023 Financial Results
- As of September 30, 2023, the Company had cash and cash
equivalents and marketable securities totaling $149.1 million. This
excludes the net proceeds from the underwritten public offering
received in November 2023. The Company believes its current cash
and cash equivalents, marketable securities, and proceeds from the
underwritten public offering are sufficient to fund its operations
into the second half of 2026.
- Research and development expenses increased to $15.4 million
for the three months ended September 30, 2023 from $11.3 million
for the three months ended September 30, 2022, primarily due to
ongoing clinical costs associated with the progression of the
Company’s Phase 2 study and CRO costs associated with the start of
the Company’s Global Phase 3 trial, and manufacturing and
development costs for bel-sar.
- General and administrative expenses increased to $5.1 million
for the three months ended September 30, 2023 from $4.8 million for
the three months ended September 30, 2022. General and
administrative expenses include $1.2 million and $1.1 million of
stock-based compensation for the three months ended September 30,
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 September 30, 2023 was
$18.5 million compared to $15.9 million for the three months ended
September 30, 2022.
About Aura Biosciences
Aura Biosciences, Inc. is a clinical-stage biotechnology company
developing 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 the
standard of care with radiotherapy leaves patients with severe
comorbidities, including significant 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 and 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, MIBC, NMIBC, 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 MIBC and NMIBC; the potential approvability of
bel-sar; the Phase 2 trial of bel-sar for choroidal metastasis; and
the Company’s 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
where Aura has obtained agreement with governmental authorities on
the design of such trials, such as the Phase 3 SPA agreement with
FDA; 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; Aura’s ongoing and
planned preclinical 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 and Quarterly Report
on Form 10-Q 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)
Three Months Ended September
30,
Nine Months Ended September
30,
2023
2022
2023
2022
Operating Expenses:
Research and development
$
15,428
$
11,293
$
44,952
$
29,079
General and administrative
5,060
$
4,762
15,256
13,603
Total operating expenses
20,488
16,055
60,208
42,682
Total operating loss
(20,488
)
(16,055
)
(60,208
)
(42,682
)
Other income (expense):
Interest income, including amortization
and accretion income
1,981
483
5,981
802
Other income (expense)
(5
)
(329
)
(50
)
(324
)
Total other income
1,976
154
5,931
478
Net loss
(18,512
)
(15,901
)
(54,277
)
(42,204
)
Net loss per common share—basic and
diluted
(0.48
)
(0.54
)
(1.43
)
(1.44
)
Weighted average common stock
outstanding—basic and diluted
38,185,197
29,273,577
37,943,139
29,246,449
Comprehensive loss:
Net loss
$
(18,512
)
$
(15,901
)
$
(54,277
)
$
(42,204
)
Other comprehensive items:
Unrealized gain (loss) on marketable
securities
$
89
(19
)
(62
)
(147
)
Total other comprehensive income
(loss)
89
(19
)
(62
)
(147
)
Total comprehensive loss
$
(18,423
)
$
(15,920
)
$
(54,339
)
$
(42,351
)
Aura Biosciences, Inc. Consolidated
Balance Sheets (in thousands, except share and per share
amounts)
September 30, 2023
December 31, 2022
Assets
Current assets:
Cash and cash equivalents
$
55,584
$
121,582
Marketable securities
93,472
67,229
Restricted cash and deposits
20
20
Prepaid expenses and other current
assets
4,812
7,871
Total current assets
153,888
196,702
Restricted cash and deposits, net of
current portion
768
768
Right of use assets - operating lease
19,569
20,671
Other long-term assets
685
423
Property and equipment, net
4,856
5,371
Total Assets
$
179,766
$
223,935
Liabilities and Stockholders’
Equity
Current liabilities:
Accounts payable
1,490
2,921
Short-term operating lease liability
3,035
2,963
Accrued expenses and other current
liabilities
6,369
4,573
Total current liabilities
10,894
10,457
Long-term operating lease liability
17,154
17,895
Total Liabilities
28,048
28,352
Commitments and Contingencies
Stockholders’ Equity:
Common stock, $0.00001 par value,
150,000,000 authorized at September 30, 2023 and December 31, 2022,
and 38,216,717 and 37,771,918 shares issued and outstanding at
September 30, 2023 and December 31, 2022, respectively
—
—
Additional paid-in capital
417,029
406,555
Accumulated deficit
(265,177
)
(210,900
)
Accumulated other comprehensive loss
(134
)
(72
)
Total Stockholders’ Equity
151,718
195,583
Total Liabilities and Stockholders’
Equity
$
179,766
$
223,935
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231109599988/en/
Investor and Media: Alex Dasalla Head of Investor
Relations and Corporate Communications
adasalla@aurabiosciences.com
Aura Biosciences (NASDAQ:AURA)
Graphique Historique de l'Action
De Avr 2024 à Mai 2024
Aura Biosciences (NASDAQ:AURA)
Graphique Historique de l'Action
De Mai 2023 à Mai 2024