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
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
announced it has received agreement from the U.S. Food and Drug
Administration (FDA) under an SPA for the design and planned
analysis of CoMpass, the Company’s global Phase 3 clinical trial of
bel-sar for the first-line treatment of adult patients with
early-stage choroidal melanoma (CM). The Company also announced the
presentation of positive Phase 2 safety and efficacy data of
bel-sar with 90% of patients at twelve months of follow-up
evaluating two key clinical endpoints: tumor control and visual
acuity preservation using suprachoroidal (SC) route of
administration for the first-line treatment of adult patients with
early-stage CM. The results were presented at the American Academy
of Ophthalmology (AAO) 2023 Annual Meeting in San Francisco,
California.
“This written agreement from the FDA is consistent with our
regulatory strategy and reaffirms that the design and planned
analysis of the CoMpass Phase 3 clinical trial, if successful,
adequately address the objectives necessary to support a biologics
license application submission for bel-sar for the treatment of
early-stage CM,” said Dr. Jill Hopkins, Chief Medical Officer and
President of R&D of Aura Biosciences. “We are excited with the
momentum in the ocular oncology community to participate in what
would be the first ever registration-enabling clinical trial for
early-stage CM.”
“The Phase 2 data presented today, 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 trial. We have observed 80% tumor control and 90% visual
acuity preservation for patients that have been treated with the
therapeutic regimen of bel-sar and meet the Phase 3 enrollment
criteria. The safety profile continues to be favorable with no
treatment-related serious adverse events or significant adverse
events. This is very encouraging as most of these patients had
tumors close to the fovea or optic disk and would have likely
experienced severe and irreversible vision loss with the current
standard of care with radiotherapy,” said Dr. Carol Shields, Chief
of the Ocular Oncology Service at Wills Eye Hospital and Professor
of Ophthalmology at Thomas Jefferson University (USA). “We are
excited for the possibility to have for the first time a vision
preserving therapy, expanding the possibility to treat patients
earlier than we do today.”
Agreement from the FDA Under an SPA for the Global Phase 3
CoMpass Trial:
The Company received written agreement from the FDA under an SPA
for the design and planned analysis of the Global Phase 3 CoMpass
trial indicating concurrence by the FDA with the adequacy of the
study, if successful, to address the objectives necessary to
support the Company’s planned biologics license application
submission. The Phase 3 trial is designed as a superiority trial
comparing bel-sar versus sham. The trial is a global, 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
with SC administration, low dose regimen of bel-sar with SC
administration, or a sham control. 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
trial is powered at greater than 90%. The Company is on track to
dose the first patient in Q4 2023.
Updated Safety and Efficacy Data from the Phase 2 Trial with
SC Administration
The Phase 2 trial (NCT04417530) is assessing the safety and
preliminary efficacy of single- and multiple ascending-doses of
bel-sar up to three cycles of treatment via SC administration for
the first-line treatment of early-stage CM. A total of 22 adult
patients have been enrolled in the trial including the single dose
Cohorts 1-3 (n=6) and multiple dose escalation Cohorts 4-6 (n=16).
Cohorts 5 and 6 (n=13) received up to three cycles of therapy,
which was considered the therapeutic regimen for evaluation. One
patient in Cohort 5 (n=3) received two cycles of therapy and two
patients in Cohort 5 received three cycles of therapy (40 μg/dose).
All patients from Cohort 6 (n=10) were assigned to receive three
cycles of therapy at the highest dose (80 μg/dose). One patient
from Cohort 6, who discontinued after one cycle due to unrelated
serious adverse events (SAEs), is not included in the analysis. All
patients in Cohorts 5 and 6 had active tumor growth at study entry,
as an enrichment strategy to evaluate preliminary efficacy. This
group of patients with active growth treated at the therapeutic
regimen of three cycles was evaluated for tumor control, visual
acuity preservation and tumor growth rate as the defined clinical
endpoints to evaluate preliminary efficacy. 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). 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
sequalae. We believe these updated results indicate that bel-sar
may offer a targeted, vision preserving therapy for the first-line
treatment of primary CM, where 80% of patients are diagnosed at an
early stage and have no approved therapies to date.
The presentation can be accessed on the Company’s website
here.
Preliminary Data from Phase 1 Trial in Bladder Cancer
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.
Bel-sar is a novel investigational agent designed with a dual
mechanism of action that includes targeted cytotoxicity and immune
activation. 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. In addition, the FDA has allowed an amendment to the
protocol of the Company’s ongoing Phase 1 trial evaluating bel-sar,
allowing the inclusion of adult patients with muscle invasive
bladder cancer in addition to non-muscle invasive bladder cancer.
The Company expects to provide more data mid-2024.
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, muscle invasive bladder cancer,
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
muscle invasive bladder cancer and non-muscle invasive bladder
cancer; and the potential approvability of bel-sar; the Phase 2
trial of bel-sar for choroidal metastasis.
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.
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Investor and Media Contact: Alex Dasalla Head of Investor
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adasalla@aurabiosciences.com
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