Aura Biosciences (AURA) FY 2025 Conference May 20, 2025 02:00 PM ET Speaker0 Great. I'd like to welcome everybody back to our afternoon session of the H. C. Wainwright BioConnect Investor Conference at NASDAQ. My name is Andres Maldonado. I'm a covering analyst here at the firm. And I'd like to welcome our next presenters, Aura Biosciences. And today we're joined by Jill Hopkins, Chief Medical Officer, President of R and D as well and Tony Gibney, Chief Financial and Business Officer. Welcome on behalf of H ...

Core Viewpoint - Aura Biosciences, Inc. has announced a public offering of common stock and warrants, aiming to raise approximately $75 million to support its clinical programs and general corporate purposes [2][3]. Group 1: Offering Details - The public offering consists of 11,735,565 shares of common stock and warrants to purchase 2,933,891 shares of common stock, along with pre-funded warrants for up to 3,571,435 shares at an exercise price of $0.00001 [1]. - The combined offering price for each share of common stock and accompanying warrant is set at $4.90, while the pre-funded warrant and accompanying warrant are priced at $4.89999 [1]. - The offering is expected to close on or about May 16, 2025, subject to customary closing conditions [2]. Group 2: Use of Proceeds - The net proceeds from the offering will be utilized to advance clinical programs in early-stage choroidal melanoma, metastases to the choroid, cancers of the ocular surface, and non-muscle invasive bladder cancer [3]. Group 3: Company Overview - Aura Biosciences is a clinical-stage biotechnology company focused on developing precision therapies for solid tumors, with a mission to transform the lives of patients [7]. - The company's lead candidate, bel-sar (AU-011), is in late-stage development for primary choroidal melanoma and early-stage development for other ocular oncology indications and bladder cancer [7]. Group 4: Management and Underwriters - Leerink Partners and Evercore ISI are acting as joint bookrunning managers for the offering, with LifeSci Capital also serving as a bookrunning manager [4]. - Citizens Capital Markets and Scotiabank are co-managers for the offering [4]. Group 5: Regulatory Information - A shelf registration statement for the offering was filed with the SEC on March 27, 2024, and was declared effective on April 5, 2024 [5]. - A preliminary prospectus supplement was filed with the SEC on May 15, 2025, and will be available on the SEC's website [5].

Company Overview - Aura Biosciences, Inc. is a clinical-stage biotechnology company focused on developing precision therapies for solid tumors aimed at preserving organ function [5] - The company's lead candidate, bel-sar (AU-011), is in late-stage development for primary choroidal melanoma and early-stage development for other ocular oncology indications and bladder cancer [5] Public Offering Announcement - Aura announced the launch of an underwritten public offering of shares of its common stock, including pre-funded warrants and accompanying warrants [1] - The offering is subject to market conditions, and there is no assurance regarding its completion or the actual size and terms [1] Management and Underwriters - Leerink Partners and Evercore ISI are acting as joint bookrunning managers for the offering, with LifeSci Capital also serving as a bookrunning manager [2] - Citizens Capital Markets and Scotiabank are co-managers for the offering [2] Regulatory Compliance - The offering is made pursuant to a shelf registration statement filed with the SEC on March 27, 2024, and declared effective on April 5, 2024 [3] - A preliminary prospectus supplement and accompanying prospectus will be filed with the SEC and made available on their website [3] Investor Relations - For inquiries, investors can contact Alex Dasalla, Head of Investor Relations and Corporate Communications at Aura [7]

Clinical Development - The lead candidate bel-sar is in late-stage clinical development for primary choroidal melanoma and other solid tumors, with enrollment for the Phase 3 trial expected to complete by the end of 2025 [88]. - In a Phase 2 study, bel-sar achieved an 80% tumor control rate among Phase 3-eligible patients, with a post-treatment average growth rate of 0.011 mm/yr compared to 0.351 mm/yr prior to study entry (p<0.0001) and 90% visual acuity preservation [89]. - The ongoing Phase 1b/2 trial for non-muscle invasive bladder cancer (NMIBC) will evaluate bel-sar in approximately 26 patients, with initial data expected by year-end 2025 [93]. - The company plans to initiate a Phase 1 trial in cancers of the ocular surface in 2025, targeting an incidence rate of 35,000 patients per year in the U.S. and Europe [90]. Financial Performance - As of March 31, 2025, the company reported net losses of $27.5 million and an accumulated deficit of $401.7 million [95]. - The company has not generated any revenue since inception and does not expect to do so in the foreseeable future [99]. - Research and development expenses are expected to increase as the company continues clinical development for bel-sar and other product candidates [102]. - Research and development expenses increased to $23.3 million for the three months ended March 31, 2025, up from $17.1 million in the same period of 2024, primarily due to ongoing clinical and CRO costs associated with the global Phase 3 trial of bel-sar [109]. - General and administrative expenses rose to $5.7 million for the three months ended March 31, 2025, compared to $5.3 million in the same period of 2024, driven by higher personnel expenses [110]. - Total operating expenses for the three months ended March 31, 2025, were $29.0 million, an increase of $6.7 million from $22.3 million in the same period of 2024 [108]. - The net loss for the three months ended March 31, 2025, was $27.5 million, compared to a net loss of $19.7 million for the same period in 2024, reflecting an increase of $7.8 million [108]. Cash and Funding - The company has raised approximately $420.0 million since inception, with $99.0 million raised from the 2023 Follow-On Offering at $9.00 per share [94]. - The company had cash and cash equivalents of $128.0 million as of March 31, 2025, which is expected to fund operations into the second half of 2026 [98]. - Cash, cash equivalents, and marketable securities as of March 31, 2025, totaled $128.0 million, expected to fund operations into the second half of 2026 [118]. - Net cash used in operating activities was $23.4 million for the three months ended March 31, 2025, compared to $24.5 million for the same period in 2024 [112]. - Net cash provided by investing activities was $29.8 million for the three months ended March 31, 2025, significantly higher than $9.4 million in the same period of 2024 [115]. - The company raised approximately $420.0 million in gross proceeds through the sale of equity and convertible preferred stock up to March 31, 2025 [111]. Operating Expenses and Commitments - The company has incurred significant operating losses since 2009 and anticipates needing substantial additional funding to support ongoing operations and growth strategies [96]. - Operating lease commitments total $26.836 million, with $3.331 million due within 1 year, $10.6 million due in 1 to 3 years, and $7.598 million due in 3 to 5 years [123]. - The company has no long-term debt or finance leases, and contracts with service providers are generally cancelable [125]. - Payments due upon cancellation of contracts consist only of services provided or expenses incurred, with no material non-cancelable purchase commitments [125]. Regulatory and Accounting Matters - The company remains classified as an "emerging growth company" until it exceeds $1.235 billion in annual revenue or meets other specified criteria [129]. - The market value of the company's stock held by non-affiliates is less than $700 million, qualifying it as a "smaller reporting company" [130]. - The company has not experienced material changes to its critical accounting policies as of March 31, 2025 [126]. - No recent accounting pronouncements have had a material impact on the company's financial statements [127]. - The company has elected to use the extended transition period under the JOBS Act for compliance with new accounting standards [128]. - The company is not required to provide quantitative and qualitative disclosures about market risk due to its status as a smaller reporting company [132]. - The lease for office and laboratory space in Boston, MA, has an initial term of 10 years with a renewal option for an additional 7 years [124]. - The income tax provision for the year ended December 31, 2024, was recorded at $0.1 million, with accumulated net operating loss carryforwards of approximately $209.8 million [107]. - The company anticipates a substantial increase in expenses related to ongoing research and development activities and operating as a public company [117].

Financial Performance - Aura Biosciences reported a net loss of $27.5 million for Q1 2025, compared to a net loss of $19.7 million for Q1 2024, reflecting an increase of approximately 39% year-over-year [21]. - The comprehensive loss for Q1 2025 was $27.6 million, compared to $20.2 million in Q1 2024 [24]. - General and administrative expenses increased to $5.7 million in Q1 2025 from $5.3 million in Q1 2024, driven by higher personnel costs [21]. Research and Development - Research and development expenses rose to $23.3 million in Q1 2025 from $17.1 million in Q1 2024, primarily due to ongoing clinical trial costs [17]. - The ongoing Phase 1b/2 trial for NMIBC will evaluate bel-sar in approximately 26 patients, with initial efficacy data expected by year-end 2025 [12]. - The CoMpass trial for early-stage choroidal melanoma has enrolled over 220 patients in a pre-screening tool since June 2024, with completion expected by the end of 2025 [4]. - The company expects initial data from the Phase 2 trial for metastases to the choroid in 2025, with a focus on a basket study approach [7]. - Bel-sar is being explored for three ocular oncology indications, collectively affecting over 60,000 patients annually in the U.S. and Europe [6]. - The company has filed a patent application for a new formulation of bel-sar for bladder cancer, aimed at enhancing in-office urologist procedures [10]. Financial Position - As of March 31, 2025, the company had cash and cash equivalents totaling $128.0 million, sufficient to fund operations into the second half of 2026 [17]. - Total assets decreased from $182,503 million as of December 31, 2024, to $155,401 million as of March 31, 2025, representing a decline of approximately 14.85% [26]. - Total current assets decreased from $160,623 million to $134,517 million, a reduction of about 16.26% [26]. - Stockholders' equity decreased from $151,970 million to $127,966 million, reflecting a decline of approximately 15.77% [26]. - Cash and cash equivalents increased from $31,693 million to $38,226 million, an increase of about 20.04% [26]. - Total liabilities decreased from $30,533 million to $27,435 million, a decrease of approximately 10.14% [26]. - Total current liabilities decreased from $14,913 million to $12,163 million, a decrease of about 18.43% [26]. - Long-term operating lease liability slightly decreased from $15,620 million to $15,272 million, a reduction of about 2.23% [26]. - Additional paid-in capital increased from $525,934 million to $529,571 million, a growth of about 0.25% [26]. - Accumulated deficit increased from $(374,227) million to $(401,710) million, indicating a worsening of approximately 7.36% [26]. - Accumulated other comprehensive income decreased from $263 million to $105 million, a decline of approximately 60.00% [26].

Core Insights - Aura Biosciences, Inc. has initiated a multi-dose Phase 1b/2 trial of bel-sar for Non-Muscle-Invasive Bladder Cancer (NMIBC), with initial data expected by the end of 2025 [1][14] - The company has strengthened its leadership team by appointing Tony Gibney as Chief Financial and Business Officer [1][18] - Aura is making significant progress in its ocular and urologic oncology programs, particularly with the Phase 3 CoMpass trial for early-stage choroidal melanoma [2][3] Recent Pipeline Developments - The CoMpass trial is the first registration-enabling study for early-stage choroidal melanoma, actively enrolling patients globally [3][4] - Over 220 patients have been pre-screened for the CoMpass trial since June 2024, indicating a strong demand for vision-preserving therapies [4] - Bel-sar is also being explored for metastases to the choroid and cancers of the ocular surface, with a collective incidence of over 60,000 patients annually in the U.S. and Europe [6][10] Clinical Trials and Data - A Phase 2 trial for bel-sar in metastases to the choroid has been initiated, with plans to broaden inclusion criteria to include various solid tumors [7] - Positive results from a completed Phase 1 trial for NMIBC showed multiple clinical complete responses and a favorable safety profile [12] - The ongoing Phase 1b/2 trial for NMIBC will evaluate two approaches, with endpoints including complete response rates and recurrence-free survival [13][14] Financial Overview - As of March 31, 2025, Aura reported cash and cash equivalents totaling $128.0 million, sufficient to fund operations into the second half of 2026 [18] - Research and development expenses increased to $23.3 million in Q1 2025 from $17.1 million in Q1 2024, driven by clinical trial costs [19] - The net loss for Q1 2025 was $27.5 million, compared to $19.7 million in Q1 2024, reflecting increased operational expenses [24]

Summary of Aura Biosciences Conference Call Company Overview - **Company**: Aura Biosciences (AURA) - **Technology**: Novel virus-like drug conjugates with a dual mechanism of action for cancer treatment, focusing on local early-stage diseases with a good safety profile [3][60] Core Industry Insights Uveal Melanoma - **Lead Indication**: Primary uveal melanoma, a rare and life-threatening disease with an incidence of 11,000 patients annually in the U.S. [4][61] - **Market Opportunity**: Approximately 66,000 patients in ocular oncology with no approved drugs, primarily treated with surgery or radiotherapy [4][61] - **Current Treatment Limitations**: Existing treatments often lead to blindness; Aura's drug, Belzar, aims to preserve vision in over 90% of patients [8][64] Treatment Administration - **Administration Method**: Belzar is administered via a suprachoroidal injection, which is safer than intravitreal injections and can be done in an office setting without the need for hospitalization [12][68] - **Treatment Regimen**: Nine treatments over three months, with a focus on achieving a durable local cure [14][70] Clinical Development - **Current Phase**: Phase III clinical trials with SPA agreement, aiming for BLA submission [16][72] - **Efficacy Data**: 80% efficacy reported in previous trials, with a focus on patients with actively growing lesions to ensure quick differentiation from sham treatments [19][74] Bladder Cancer Insights - **Emerging Opportunity**: Similar to ocular oncology, bladder cancer presents a high incidence of pre-metastatic disease [31][82] - **Efficacy in Trials**: Initial trials showed 80% complete response rates with a single dose, with plans for further dose escalation [84][87] - **Mechanism of Action**: The treatment aims to prime the immune system for an anti-tumoral response, contrasting with traditional adjuvant treatments that may not effectively target residual tumors [85][86] Market Dynamics - **Competitive Landscape**: Aura operates in a white space with no direct competition for its lead indication, positioning itself for a successful market launch [5][61] - **Physician Adoption**: There may be initial resistance from urologists who are accustomed to surgical interventions; however, the economic incentives favor office-based treatments [42][91] Future Outlook - **Cash Position**: Aura has sufficient funding to support clinical programs into the second half of 2026 [56][56] - **Upcoming Milestones**: Key data milestones expected within the next six months, including bladder cancer durability data and ocular metastasis results [56][57] Additional Considerations - **Tumor Agnostic Potential**: The technology is designed to treat various solid tumors, expanding the potential market significantly beyond ocular and bladder cancers [52][53] - **Patient-Centric Approach**: The treatment strategy emphasizes patient safety and efficacy, aiming to provide options that do not compromise quality of life [42][92]

BOSTON, April 02, 2025 (GLOBE NEWSWIRE) -- Aura Biosciences, Inc. (NASDAQ:AURA), a clinical-stage biotechnology company developing precision therapies for solid tumors designed to preserve organ function, today announced the appointment of Teresa Bitetti, President of the Global Oncology Business Unit at Takeda, to the Company's Board of Directors, effective March 31, 2025. "I am pleased to welcome Teresa to our Board of Directors," said Elisabet de los Pinos, Chief Executive Officer of Aura Biosciences. "S ...

Financial Performance and Capital Needs - The company incurred net losses of $86.9 million and $76.4 million for the years ended December 31, 2024 and 2023, respectively, with an accumulated deficit of $374.2 million as of December 31, 2024[205]. - The company has not generated any revenue from product sales to date and does not expect to do so in the foreseeable future[205]. - The company will require substantial additional capital to finance operations, particularly for the development and commercialization of bel-sar, and may need to raise additional funds sooner than planned[210]. - Existing cash and cash equivalents are expected to fund operations into the second half of 2026, but will not be sufficient to fund bel-sar through regulatory approval[211]. - The company has federal gross operating loss carryforwards of approximately $209.8 million, state gross operating loss carryforwards of $183.6 million, and foreign gross operating loss carryforwards of $0.7 million as of December 31, 2024[386]. - The company has never declared or paid cash dividends on its capital stock, intending to retain future earnings to finance business growth[387]. Product Development and Regulatory Approval - Significant research and development expenses are anticipated to increase as the company continues clinical development for bel-sar and other product candidates[205]. - The company is heavily dependent on the success of bel-sar, its only product candidate to date, and its business relies on its successful development and commercialization[221]. - The ability to generate revenue and achieve profitability depends on successful completion of clinical development and obtaining marketing approvals for bel-sar and future product candidates[218]. - The company faces risks related to the regulatory approval process, which could delay or prevent the commercialization of bel-sar[222]. - The company has not yet received regulatory approval for any of its product candidates, which significantly impacts its ability to generate revenue[225]. - The process of obtaining regulatory approvals is expensive and may take many years, with potential delays due to additional clinical trials or changes in regulations[226]. - The company is currently conducting a global Phase 3 trial for bel-sar, but has not yet completed pivotal clinical trials necessary for marketing approval[232]. - The FDA may require an Advisory Committee to evaluate the safety and efficacy data, which could further delay the approval process[229]. - The company plans to submit a single Biologics License Application (BLA) for bel-sar in combination with the SCS Microinjector and laser[235]. - The lengthy and unpredictable nature of the drug approval process poses significant risks to the company's business and operational prospects[227]. Clinical Trials and Market Risks - The company faces challenges in recruiting sufficient patients for clinical trials, which could delay regulatory approvals[237]. - Changes in manufacturing methods or formulations during development may lead to increased costs and delays in clinical trials[236]. - The acceptance of data from foreign clinical trials by the FDA is subject to specific conditions, and failure to meet these could necessitate additional costly trials[240]. - Regulatory approval in one jurisdiction does not guarantee approval in others, which could negatively impact market potential[241]. - The marketing approval process in other countries may involve additional testing and could lead to significant delays[243]. - The company may face significant delays and costs in obtaining foreign regulatory approvals, impacting market introduction[244]. - Adverse events or side effects from product candidates could delay or prevent marketing approval, impacting commercialization[250]. - The number of patients required for clinical trials may be larger than anticipated, leading to slower enrollment and potential dropout rates[256]. - The company may face delays in clinical trials due to regulatory suspensions or terminations, which could impact the approval of product candidates[257]. Competition and Market Acceptance - Market acceptance of bel-sar and future product candidates is uncertain, which could limit revenue generation and delay profitability[291]. - Significant resources may be required to educate the medical community on the benefits of bel-sar, and failure to achieve market acceptance could hinder profitability[292]. - The company faces intense competition in the biopharmaceutical industry, with potential competitors having greater financial and technical resources[298]. - The total addressable market for the product candidate bel-sar may be smaller than estimated, adversely affecting revenue and profitability[301]. - The acceptance of the company's product candidates may be inhibited by price competition and reluctance from physicians to switch from existing treatments[299]. Intellectual Property and Legal Risks - The ability to protect proprietary rights is crucial for commercial success, as competition may arise from third parties if patents are not adequately maintained[310]. - Patent protection for products like bel-sar is uncertain due to potential challenges from third parties and the complexity of patent laws[312]. - The company may face significant expenses related to maintaining its patent portfolio, including maintenance fees and renewal costs[313]. - Legal actions to enforce patent rights can be costly and may divert management resources, impacting overall business operations[314]. - The company may need to license intellectual property from third parties, which may not be available on commercially reasonable terms[315]. - The company faces risks from potential patent infringement claims by third parties, which could lead to substantial costs and delays in product development[328]. Management and Operational Risks - The company is highly dependent on key management and scientific personnel, and losing them could impair its business strategy and competitiveness[341]. - The competitive hiring environment in the Boston area poses challenges for the company in attracting and retaining qualified personnel[342]. - The company anticipates significant growth in employee numbers and operational scope, particularly in sales, marketing, finance, and accounting[378]. - The company may face increased operating expenses and cash requirements due to anticipated growth[379]. Cybersecurity and Compliance Risks - Significant disruptions to information technology systems or cybersecurity incidents could lead to material disruptions in product development programs, potentially increasing costs and delaying regulatory approvals[352]. - Cybersecurity incidents may result in unauthorized access to confidential information, leading to financial, legal, and reputational harm to the company[352]. - The company faces stringent and evolving privacy and information security laws, which could increase compliance costs and impact operational results[356]. - The company must navigate complex regulatory environments across different jurisdictions, which may complicate compliance and increase operational costs[361]. Financial Market and Economic Risks - The trading market for the company's common stock may decline if analysts publish unfavorable research or cease coverage[384]. - The company anticipates fluctuations in quarterly operating results, which may affect stock price and investor expectations[389]. - Access to funding sources and credit arrangements may be significantly impaired due to liquidity constraints or disruptions in the financial services industry[402]. - Any decline in available funding could adversely impact the company's ability to meet operating expenses and financial obligations[403].

Financial Performance - Aura Biosciences reported a net loss of $86.9 million for the full year ended December 31, 2024, compared to a net loss of $76.4 million in 2023, representing a 13.5% increase in losses[22] - Total current assets decreased from $231,794 million in 2023 to $160,623 million in 2024, a decline of approximately 30.7%[24] - Cash and cash equivalents dropped from $41,063 million in 2023 to $31,693 million in 2024, representing a decrease of about 22.8%[24] - Total liabilities increased slightly from $29,227 million in 2023 to $30,533 million in 2024, an increase of approximately 4.5%[24] - Stockholders' equity decreased significantly from $225,848 million in 2023 to $151,970 million in 2024, a decline of around 32.6%[24] - The accumulated deficit widened from $287,308 million in 2023 to $374,227 million in 2024, indicating a deterioration in financial health[24] - Additional paid-in capital increased from $512,617 million in 2023 to $525,934 million in 2024, reflecting a growth of about 2.6%[24] - Total assets decreased from $255,075 million in 2023 to $182,503 million in 2024, a reduction of approximately 28.5%[24] - Current liabilities rose from $12,357 million in 2023 to $14,913 million in 2024, an increase of about 20.6%[24] - Long-term operating lease liability decreased from $16,870 million in 2023 to $15,620 million in 2024, a decline of approximately 7.4%[24] - Right-of-use assets for operating lease decreased from $18,854 million in 2023 to $17,379 million in 2024, a reduction of about 7.8%[24] Research and Development - Research and development expenses increased to $73.3 million for the full year 2024, up from $65.2 million in 2023, reflecting ongoing clinical trial costs[22] - The Phase 3 CoMpass trial for early-stage choroidal melanoma is actively enrolling, with over 175 patients registered in pre-screening since June 2024[6] - Positive data from the Phase 1 trial in non-muscle invasive bladder cancer (NMIBC) demonstrated clinical complete responses and robust cell-mediated immunity across the disease spectrum[11] - The company has initiated a Phase 2 clinical trial for metastases to the choroid, with initial data expected in 2025[8] Regulatory and Market Position - The company received Orphan Drug Designation and Fast Track designation from the FDA for early-stage choroidal melanoma, indicating significant regulatory support[4] - The collective incidence of ocular oncology indications being targeted by bel-sar exceeds 60,000 patients annually in the U.S. and Europe[7] - The company anticipates that bel-sar could transform treatment paradigms for multiple rare oncology indications, starting with ocular cancers[3] General and Administrative Expenses - General and administrative expenses rose to $22.8 million for the full year 2024, compared to $19.8 million in 2023, driven by personnel and corporate expenses[22]