On May 15, 2025 Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing precision therapies for solid tumors designed to preserve organ function, reported financial results for the first quarter ended March 31, 2025, and provided recent business highlights (Press release, Aura Biosciences, MAY 15, 2025, View Source [SID1234653160]).

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"Aura has started 2025 with strong momentum, making meaningful strides across both our ocular and urologic oncology programs," said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura. "Our global Phase 3 CoMpass trial in early-stage choroidal melanoma continues to advance, and we enrolled the first patient in our multi-dose Phase 1b/2 trial in NMIBC. At Aura, we remain deeply focused on transforming the treatment landscape in ocular and urologic cancers—two areas where patients urgently need innovative therapies."

Recent Pipeline Developments

Early-Stage Choroidal Melanoma

Update on Ongoing Phase 3 CoMpass Trial: CoMpass is the first registration-enabling study in early-stage choroidal melanoma. The study is a global, Phase 3, randomized trial evaluating bel-sar treatment against a sham control arm and includes an enrichment strategy to enroll approximately 100 patients with documented tumor growth.

The CoMpass trial is actively enrolling globally. To identify appropriate patients to meet the enrichment strategy of documented growth, the Company has enabled a pre-screening ‘run in’ period. Globally, since June 2024, investigators have registered over 220 patients in a pre-screening tool as having met initial enrollment criteria for the study, highlighting the global need for a frontline vision-preserving therapy. Given the momentum in the study globally, the Company believes study enrollment may be completed as early as the end of 2025.

The Company previously received Orphan Drug Designation from the FDA and the European Medicines Agency and Fast Track designation from the FDA for the treatment of early-stage choroidal melanoma. The CoMpass trial is under a Special Protocol Assessment agreement with the FDA.

Additional Ocular Oncology Indications

In addition to early-stage choroidal melanoma, bel-sar is being explored for metastases to the choroid and cancers of the ocular surface. These three ocular oncology indications have a collective incidence of greater than 60,000 patients annually in the United States and Europe.

Metastases to the Choroid

Metastases to the choroid is an indication with high unmet medical need and no approved therapies. Bel-sar has the potential to treat a wide variety of tumor types that metastasize from several primary tumors. The Company has initiated a Phase 2 clinical trial in metastases to the choroid from breast and lung cancer and have activated sites with patients in prescreening in the United States. The Company is currently implementing a protocol amendment for the Phase 2 trial to broaden the inclusion criteria beyond breast and lung cancer to include all metastases from different solid tumors as a basket study approach. The Company believes that this approach, in addition to advancing bel-sar in metastases to the choroid, can provide clinical insights into multiple tumor types that could be impacted by bel-sar. The Company expects initial data from this trial in 2025.

Metastases to the choroid represents the second potential ocular oncology indication for bel-sar, affecting approximately 20,000 patients annually in the United States and Europe. The Company previously received FDA Fast Track designation for bel-sar in this indication.

Cancers of the Ocular Surface

The Company’s third potential ocular oncology indication is cancers of the ocular surface, which affects approximately 35,000 patients in the United States and Europe annually and has no approved therapies. We continue to advance pre-clinical activities in cancers of the ocular surface, and we plan to initiate a Phase 1 trial in 2025.

Bladder Cancer

Patent Application Filed for New Formulation of Bel-sar for Use in Bladder Cancer: The Company has filed a patent application for a new formulation of bel-sar for use in urologic oncology. This new formulation is designed to enable convenient in-office urologist procedures with enhanced storage and handling at refrigerator temperatures, as well as an adjusted volume and concentration.

Positive Data from Completed Phase 1 Window-of-Opportunity Trial: In the completed Phase 1 window-of-opportunity trial for NMIBC, the administration of a single, low dose of the ocular formulation of bel-sar resulted in multiple clinical complete responses among patients with intermediate and high-risk NMIBC. These histopathologic outcomes highlight robust cell-mediated immunity and a urothelial field effect. Additionally, the study demonstrated a favorable safety profile, with only grade 1 drug-related adverse events occurring in less than 10% of patients. Detailed data can be accessed here: link. Based on these findings, the Company believes bel-sar has the potential to transform treatment of patients with intermediate and high-risk NMIBC with its immune-ablative, front-line approach.

Ongoing Phase 1b/2 Trial: Based on the positive data from the Phase 1 window of opportunity trial, the Company is advancing the development of bel-sar in NMIBC. The ongoing Phase 1b/2 trial will evaluate additional doses and cycles of bel-sar in approximately 26 intermediate and high-risk patients. The trial will evaluate two approaches: an immune ablative design and a multimodal neoadjuvant design. In the immune ablative approach, bel-sar will be administered in two cycles without the need for a transurethral resection of the bladder tumor (TURBT). In the multimodal neoadjuvant cohorts, bel-sar will be administered in two cycles ahead of TURBT. For both approaches, patients will be monitored for response assessments and recurrence at 3, 6, 9, and 12 months.

Endpoints of this trial include multiple efficacy assessments, such as complete response rate at 3 months and durability of response up to 12 months in the immune ablative cohorts and recurrence-free survival in the neoadjuvant cohorts. Patients will also be monitored for safety. The Company expects initial efficacy data at 3 months by year-end 2025.

The Company has filed a patent application with the U.S. Patent and Trademark Office covering the new formulation, which if issued, would provide patent coverage for this formulation into 2046.

Corporate Updates

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The Company strengthened the leadership team with the appointment of Tony Gibney as Chief Finance and Business Officer. Mr. Gibney is an experienced biotechnology leader and former investment banker who brings over 30 years of experience dedicated to leading and advising biotechnology companies across their businesses, including corporate strategy, business development, finance and investor relations, among others. Following his investment banking career, he has worked as Chief Business Officer at Achillion Pharmaceuticals, Inc. and Iveric Bio, Inc. and as Chief Business and Financial Officer at Fog Pharmaceuticals, Inc.

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The Company hosted a virtual urologic oncology investor event on March 24, 2025. A replay of the webcast is available on the "Investors & Media" page under the "Events & Presentations" section of Aura’s website at View Source

First Quarter 2025 Financial Results

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As of March 31, 2025, Aura had cash and cash equivalents and marketable securities totaling $128.0 million. The Company believes its current cash and cash equivalents and marketable securities are sufficient to fund its operations into the second half of 2026.

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Research and development expenses increased to $23.3 million for the three months ended March 31, 2025 from $17.1 million for the three months ended March 31, 2024, primarily due to ongoing clinical and contract research organization costs associated with the progression of the Company’s Phase 3 trial of bel-sar in early-stage choroidal melanoma and manufacturing and development costs for bel-sar.

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General and administrative expenses increased to $5.7 million for the three months ended March 31, 2025 from $5.3 million for the three months ended March 31, 2024. General and administrative expenses include $1.6 million and $1.4 million of stock-based compensation for the three months ended March 31, 2025 and 2024, respectively. The increase was primarily driven by higher personnel expenses related to the growth of the Company.

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Net loss for the three months ended March 31, 2025 was $27.5 million compared to $19.7 million for the three months ended March 31, 2024.