Aura Biosciences Reports Fourth Quarter and Full Year 2023 Financial Results and Business Highlights
- Enrollment in a global Phase 3 CoMpass trial for small choroidal melanoma and indeterminate lesions
- Strong cash position until the second half of 2026
- Expansion of bel-sar therapy into bladder cancer to address unmet medical needs in ocular oncology
- Positive Phase 2 data for bel-sar in small choroidal melanoma and indeterminate lesions
- Increase in research and development expenses
- Net loss for the full year ended December 31, 2023
Insights
The initiation and progression of the Phase 3 trial for small choroidal melanoma (CM) and indeterminate lesions (ILs) by Aura Biosciences represents a significant milestone in ocular oncology. The trial's design, which includes a comparison against a sham control arm, is set to provide robust data regarding the efficacy of bel-sar. The involvement of the FDA through the Special Protocol Assessment (SPA) agreement adds a layer of credibility and regulatory assurance to the trial's design and endpoints, which is critical for investor confidence. The positive Phase 2 data showing high tumor control and visual acuity preservation rates indicate a strong potential for bel-sar to meet an urgent medical need, which could translate into a significant market opportunity given the estimated patient population in the US and EU.
From a financial perspective, the company's reported strong cash position extending into the second half of 2026 is noteworthy. This financial runway is important for supporting ongoing clinical trials and potentially expanding into new indications, such as bladder cancer. The Fast Track Designation for non-muscle invasive bladder cancer (NMIBC) underscores the unmet need and the FDA's interest in accelerating the development of promising therapies. The preliminary data from the Phase 1 trial in bladder cancer, showing a complete response in a patient, is promising and could be a harbinger of the therapy's efficacy in a solid tumor indication outside of ocology.
Aura Biosciences' financial results for the fourth quarter and full year of 2023 reflect an increase in research and development expenses, which is aligned with the company's clinical advancements. The uptick in expenses is expected as the company progresses from Phase 2 to a global Phase 3 trial and continues to invest in the development of bel-sar. The increase in general and administrative expenses is modest and can be attributed to the natural growth of the company. The reported net loss is consistent with the developmental stage of the company, where investment in research outpaces revenue.
The successful closure of an underwritten public offering, raising $99.0 million, is a strong indicator of market confidence and provides the capital necessary to fund the company's ambitious clinical programs. The cash reserves of $226.2 million provide a substantial buffer for the company to reach key clinical milestones without the immediate need for further capital raises, which is reassuring for investors.
The clinical advancements of Aura Biosciences in developing bel-sar, particularly for small choroidal melanoma, addresses a significant gap in current treatment options. Radiotherapy, the current standard of care, often results in vision loss, highlighting the necessity for organ-sparing therapies. The trial's primary endpoint of time to tumor progression is a direct measure of therapeutic efficacy and will be critical in determining bel-sar's potential to alter the treatment landscape. The SPA agreement with the FDA suggests that the endpoints and trial design are well-constructed to meet regulatory expectations, which is an essential consideration for clinical success.
Entering the bladder cancer space, bel-sar's mechanism of inducing a tissue and tumor-specific immune response represents an innovative approach to treating NMIBC and MIBC. The Fast Track Designation indicates the therapy's potential to address the high unmet need in bladder cancer. However, it's important to note that early Phase 1 results, while promising, must be interpreted with caution until more comprehensive data is available.
Enrolling Patients in Global Phase 3 CoMpass Trial in Small Choroidal Melanoma and Indeterminate Lesions; Granted SPA Agreement by FDA
Data Expected Mid-2024 from Ongoing Phase 1 Trial in Bladder Cancer
(Non-Muscle Invasive Bladder Cancer and Muscle Invasive Bladder Cancer)
Strong Cash Position into Second Half of 2026
“We’re excited to expand bel-sar into bladder cancer, as we leverage our therapy’s unique mechanism of action in additional solid cancer indications with major unmet medical need,” said Elisabet de los Pinos, Ph.D., CEO of Aura Biosciences. “Bel-sar is a potential vision-and-organ sparing therapy that we believe will change the standard of care in ocular oncology, in particular choroidal melanoma, where there are no treatment options except radiotherapy which leads to vision loss, or surgical removal of the eye. There is an urgent need to develop vision-sparing therapies, as all eye cancers represent an estimated 60,000 patients annually in the US and EU. This is a multi-billion dollar market, which includes choroidal melanoma, choroidal metastases, and ocular surface cancers. Our financial strength allows us to fund multiple clinical programs through major inflection points while enabling the flexibility to expand bel-sar into additional indications, starting with bladder cancer.”
Recent Pipeline Developments
Global Phase 3 CoMpass trial actively enrolling patients for the treatment of small choroidal melanoma (CM) and indeterminate lesions (ILs).
- The trial is a superiority trial comparing treatment with bel-sar versus a sham control arm. The trial is a global Phase 3, randomized, multi-center, masked study, and is intended to enroll approximately 100 patients randomized 2:1:2 to receive three cycles of treatment with either high or low doses of bel-sar, or a sham control.
-
The Company received written agreement from the
U.S. Food and Drug Administration (FDA) under an SPA for the overall design, statistical analysis plan and clinical endpoints. The primary endpoint is time to tumor progression when the last patient completes 15 months of follow up. -
The trial is actively enrolling in the
U.S. with a strong endorsement from the ocular oncology community. The Company is on track to activate sites and enroll patients globally throughout 2024.
Positive Phase 2 data evaluating SC administration of bel-sar for patients with small CM and ILs was presented at the American Academy of Ophthalmology (AAO) 2023 Annual Meeting.
Results:
-
Patients who received the therapeutic regimen with three cycles of therapy showed a tumor control rate of
80% (8/10) and a visual acuity preservation rate of90% (9/10), with most of the patients (>90% ) being at 12 months of follow up. Final study results with all patients at 12 months will be presented by year end 2024. -
The overall tolerability profile of bel-sar was favorable, with no dose-limiting toxicities, treatment-related serious adverse events (SAEs) or significant adverse events (AEs) reported as of August 3, 2023. There was no posterior segment inflammation and only mild anterior inflammation (Grade 1) in approximately
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. - These patients match the criteria for enrollment in the ongoing Phase 3 trial which is highly powered based on the Phase 2 results.
Bel-sar is being evaluated in additional ocular oncology indications with a collective incidence of approximately 60,000 patients/year in the US/EU per year. The Company’s plan is to initiate clinical development in choroidal metastasis (Cmets), an indication with a high unmet medical need where bel-sar has the potential to be the first approved therapy that is vision and organ sparing. Cmets is the second potential ocular oncology indication for bel-sar affecting over 20,000 patients in the US/EU annually. The Company is on track to initiate a Phase 2 trial in 2024.
The Phase 1 trial of bel-sar for the treatment of non-muscle invasive bladder cancer (NMIBC) and muscle invasive bladder cancer (MIBC) 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
- The ongoing Phase 1 multi-center, open-label clinical trial is expected to enroll approximately 21 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 which will include the evaluation of focal necrosis and immune activation.
- 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, demonstrated a clinical complete response demonstrated by absence of cancer cells on histopathology with evidence of extensive necrosis and immune activation after a single administration of bel-sar followed by light activation.
Recent Corporate Events
-
Raised Gross Proceeds of
$99.0 million $9.00
Full Year and Fourth Quarter 2023 Financial Results
-
As of December 31, 2023, Aura had cash and cash equivalents and marketable securities totaling
$226.2 million -
Research and development expenses increased to
$20.3 million $65.2 million $13.2 million $42.2 million -
General and administrative expenses increased to
$4.5 million $19.8 million $4.5 million $18.1 million $1.2 million $1.1 million -
Net loss for the three months and full year ended December 31, 2023, was
$22.1 million $76.4 million $16.6 million $58.8 million
About Aura Biosciences
Aura Biosciences is a clinical-stage biotechnology company developing precision immunotherapies to treat solid tumors designed to preserve the function of the afflicted organ with cancer. Our lead candidate bel-sar is in late-stage clinical development for the treatment of patients with primary choroidal melanoma, and other ocular oncology indications as well as in early-stage clinical development in bladder cancer. We are evaluating the safety and efficacy of bel-sar as a potential vision-sparing therapy in an ongoing global Phase 3 CoMpass trial for the first-line treatment of adult patients with early-stage choroidal melanoma. Bel-sar is also being evaluated in additional solid cancers, including bladder cancer. Our mission is to develop vision and organ-sparing therapies to improve patient outcomes in cancer. Aura is headquartered in
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, 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 choroidal melanoma and indeterminate legions and the Phase 1 trial of bel-sar for non-muscle invasive bladder cancer and 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; 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 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 and Quarterly Report on Form 10-Q filed with the
Aura Biosciences, Inc. Consolidated Statement of Operations and Comprehensive Loss (in thousands, except share and per share amounts) |
||||||||
|
|
Year Ended
|
|
|||||
|
|
2023 |
|
|
2022 |
|
||
Operating Expenses: |
|
|
|
|
|
|
||
Research and development |
|
$ |
65,232 |
|
|
$ |
42,238 |
|
General and administrative |
|
|
19,759 |
|
|
|
18,057 |
|
Total operating expenses |
|
|
84,991 |
|
|
|
60,295 |
|
Total operating loss |
|
|
(84,991 |
) |
|
|
(60,295 |
) |
Other income (expense): |
|
|
|
|
|
|
||
Interest income, including amortization and accretion income |
|
|
8,588 |
|
|
|
1,864 |
|
Gain (loss) from disposal of assets |
|
|
208 |
|
|
|
(318 |
) |
Other income (expense) |
|
|
(76 |
) |
|
|
(14 |
) |
Total other income |
|
|
8,720 |
|
|
|
1,532 |
|
Loss before income taxes |
|
|
(76,271 |
) |
|
|
(58,763 |
) |
Income tax benefit (provision), net |
|
|
(137 |
) |
|
|
— |
|
Net loss |
|
|
(76,408 |
) |
|
|
(58,763 |
) |
Net loss per common share—basic and diluted |
|
|
(1.93 |
) |
|
|
(1.96 |
) |
Weighted average common stock outstanding—basic and diluted |
|
|
39,620,036 |
|
|
|
29,937,228 |
|
Comprehensive loss: |
|
|
|
|
|
|
||
Net loss |
|
$ |
(76,408 |
) |
|
$ |
(58,763 |
) |
Other comprehensive items: |
|
|
|
|
|
|
||
Unrealized gain (loss) on marketable securities |
|
|
611 |
|
|
|
(72 |
) |
Total other comprehensive income (loss) |
|
|
611 |
|
|
|
(72 |
) |
Total comprehensive loss |
|
$ |
(75,797 |
) |
|
$ |
(58,835 |
) |
Aura Biosciences, Inc. Consolidated Balance Sheets (in thousands, except share and per share amounts) |
||||||||
|
|
December 31,
|
|
|
December 31,
|
|
||
Assets |
|
|
|
|
|
|
||
Current assets: |
|
|
|
|
|
|
||
Cash and cash equivalents |
|
$ |
41,063 |
|
|
$ |
121,582 |
|
Marketable securities |
|
|
185,087 |
|
|
|
67,229 |
|
Restricted cash and deposits |
|
|
19 |
|
|
|
20 |
|
Prepaid expenses and other current assets |
|
|
5,625 |
|
|
|
7,871 |
|
Total current assets |
|
|
231,794 |
|
|
|
196,702 |
|
Restricted cash and deposits, net of current portion |
|
|
768 |
|
|
|
768 |
|
Right of use assets - operating lease |
|
|
18,854 |
|
|
|
20,671 |
|
Other long-term assets |
|
|
509 |
|
|
|
423 |
|
Property and equipment, net |
|
|
3,150 |
|
|
|
5,371 |
|
Total Assets |
|
$ |
255,075 |
|
|
$ |
223,935 |
|
Liabilities and Stockholders’ Equity |
|
|
|
|
|
|
||
Current liabilities: |
|
|
|
|
|
|
||
Accounts payable |
|
|
1,787 |
|
|
|
2,921 |
|
Short-term operating lease liability |
|
|
2,687 |
|
|
|
2,963 |
|
Accrued expenses and other current liabilities |
|
|
7,883 |
|
|
|
4,573 |
|
Total current liabilities |
|
|
12,357 |
|
|
|
10,457 |
|
Long-term operating lease liability |
|
|
16,870 |
|
|
|
17,895 |
|
Total Liabilities |
|
|
29,227 |
|
|
|
28,352 |
|
Commitments and Contingencies |
|
|
|
|
|
|
||
Stockholders’ Equity: |
|
|
|
|
|
|
||
Common stock, |
|
|
— |
|
|
|
— |
|
Additional paid-in capital |
|
|
512,617 |
|
|
|
406,555 |
|
Accumulated deficit |
|
|
(287,308 |
) |
|
|
(210,900 |
) |
Accumulated other comprehensive loss |
|
|
539 |
|
|
|
(72 |
) |
Total Stockholders’ Equity |
|
|
225,848 |
|
|
|
195,583 |
|
Total Liabilities and Stockholders’ Equity |
|
$ |
255,075 |
|
|
$ |
223,935 |
|
View source version on businesswire.com: https://www.businesswire.com/news/home/20240327005499/en/
Investor and Media:
Alex Dasalla
Head of Investor Relations and Corporate Communications
IR@aurabiosciences.com
Source: Aura Biosciences, Inc.
FAQ
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