On March 15, 2023 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, reported financial results for the fourth quarter and year ended December 31, 2022, and provided clinical development and operational highlights (Press release, Aura Biosciences, MAR 15, 2023, View Source [SID1234628777]).
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"2023 is off to a strong start with positive interim Phase 2 safety and efficacy data with suprachoroidal administration in early-stage choroidal melanoma. In February, at the Macula Society’s 46th Annual Meeting, we presented average nine-month interim data which strongly supports the assumptions for the success of the global Phase 3 trial, which is on track to dose the first patient in the first half of this year," said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura. "We are also excited to initiate clinical development in our second ocular oncology indication, choroidal metastasis, and to report early Phase 1 data in our non-muscle invasive bladder cancer program in the second half of this year. Our recently strengthened balance sheet positions us well to execute and advance our pipeline to meaningful clinical milestones across ocular and urologic oncology."
"We are excited that bel-sar was granted Fast Track Designation for choroidal metastasis. There is an important opportunity to develop a new standard of care as we see a large number of patients with this type of metastasis," said Dr. Cadmus Rich, Chief Medical Officer of Aura Biosciences. "This is the second Fast Track Designation bel-sar has obtained for an ocular oncology indication, which highlights the need for vision preserving treatment options."
Recent Pipeline Developments
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Bel-sar is being developed for the first-line treatment of early-stage choroidal melanoma (CM), a life-threatening rare disease with no approved therapies
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Positive interim Phase 2 data evaluating suprachoroidal (SC) administration of bel-sar for the first-line treatment of adult patients with early-stage CM was presented at the Macula Society 46th Annual Meeting. The results reported as of January 10, 2023, with an average of nine months of follow up in patients similar to the planned Phase 3 population, who received three cycles of therapy (n=8), showed a statistically significant reduction in the tumor growth rate (-0.289 mm/yr, p = <0.0001) compared to each patient’s documented growth rate at study entry, and a 100% tumor control rate. In addition, only one patient lost visual acuity in these cohorts, with the majority of patients being at high-risk for vision loss with tumors close to fovea or optic disk. The overall tolerability profile of bel-sar was generally favorable, with no dose-limiting toxicities, treatment-related serious adverse effects (SAEs) or significant adverse events (AEs). There was no posterior inflammation and mild anterior inflammation (Grade 1) in 25% of the patients. Treatment-related AEs were predominantly mild and resolved without sequalae. We believe these interim results indicate that bel-sar may offer a targeted, vision preserving therapy for the first-line treatment of early-stage CM, where 80% of patients are diagnosed early and have no approved therapies to date.
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Aura finalized the global Phase 3 trial design in alignment with regulatory agencies and selected SC route of administration to evaluate the efficacy and safety of bel-sar in early-stage CM. The global Phase 3 trial is randomized and masked and will include three arms, where the primary endpoint will be a time to event composite endpoint that will compare the tumor control and visual acuity of the intervention group with high dose of bel-sar to sham. Aura is planning to enroll approximately 85 adult patients with early-stage CM, including patients with indeterminate lesions and small choroidal melanoma. Patients with documented growth will be enrolled as an enrichment strategy intended to increase the efficiency of the trial, which will also include an adaptive design to further increase the probability of success.
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Aura is enrolling a Phase 1 clinical trial of bel-sar for the treatment of non-muscle invasive bladder cancer (NMIBC). This represents an area of high unmet need with approximately 80,000 patients diagnosed in the United States every year. Aura received Fast Track Designation from the Oncology Division of the FDA for this indication in June 2022.
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The Phase 1 multi-center, open-label clinical trial is expected to enroll approximately 23 adult patients. The trial is designed to assess the safety and tolerability of bel-sar as a single agent. The primary endpoint of the Phase 1 clinical trial is the incidence and severity of treatment-related AEs and SAEs and the incidence of dose-limiting toxicities. The goal of this study is to demonstrate distribution, local necrosis and evidence of immune activation. Aura expects to report initial Phase 1 data in the second half of 2023.
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Beyond early-stage CM, Aura continues to build its ocular oncology franchise. Aura’s goal is to initiate clinical development in choroidal metastasis, an indication with an unmet medical need and no approved therapies, as the second ocular oncology indication. Aura received Fast Track Designation from the Oncology Division of the FDA for this indication in February 2023, and the Investigational Drug application was opened in January 2023
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Nonclinical data supporting bel-sar’s broad tumor targeting potential and immune mediated mechanism of action was presented at the 22nd EURETINA Congress. Preclinical results highlighted bel-sar’s targeted cytotoxicity towards tumor cells derived from the most common cancer types known to metastasize to the choroid, supporting its potential use for the treatment of choroidal metastasis, a key second ocular oncology indication. The presentation also included nonclinical data that supported the activity of bel-sar as a single agent as well as in combination with checkpoint inhibitors, highlighting the possibility to treat not only primary tumors in the eye but also potentially distant metastases by an abscopal effect.
Recent Corporate Events
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Raised Gross Proceeds of $92.5 Million in Oversubscribed Follow-on Public Offering. In December 2022, Aura announced the closing of an oversubscribed underwritten follow-on public offering yielding aggregate gross proceeds of approximately $92.5 million. All of the shares in the offering were offered by Aura.
Full Year and Fourth Quarter 2022 Financial Results
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As of December 31, 2022, Aura had cash and cash equivalents and marketable securities totaling $188.8 million. Aura believes its current cash and cash equivalents and marketable securities are sufficient to fund its operations into 2025.
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Research and development expenses increased to $13.2 million and $42.2 million for the three months and full year ended December 31, 2022, respectively, from $8.0 million and $25.2 million for the three months and full year ended December 31, 2021, respectively, primarily due to ongoing clinical costs associated with the progression of Aura’s Phase 2 study and clinical research organization costs associated with the start of Aura’s Phase 3 global trial, manufacturing and development costs for bel-sar, and higher personnel expenses from growing headcount.
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General and administrative expenses increased to $4.5 million and $18.1 million for the three months and full year ended December 31, 2022, respectively, from $3.6 million and $10.1 million for the three months and full year ended December 31, 2021, respectively. General and administrative expenses include $1.1 million and $0.9 million of stock-based compensation for the three months ended December 31, 2022 and 2021, respectively. The increase was primarily driven by personnel expenses, as well as increases in general corporate expenses related to a full year of operating as a public company.
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Net loss for the three months and full year ended December 31, 2022, was $16.6 million and $58.8 million, respectively, compared to $11.6 million and $35.3 million for the three months and full year ended December 31, 2021, respectively.