Core Insights - Benitec Biopharma Inc. is advancing its BB-301 clinical development program for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD) and expects to provide interim clinical study results for Cohort 1 in Q4 2025 [1][4][2] Corporate Highlights - The Independent Data Safety Monitoring Board (DSMB) recommended the continuation of subject enrollment into the BB-301 Phase 1b/2a Treatment Study after the safe treatment of the sixth subject in Cohort 1, with enrollment for Cohort 2 expected to begin in Q4 2025 [3][2] Financial Highlights - For the year ended June 30, 2025, total expenses were $41.8 million, up from $22.5 million in 2024, with research and development expenses increasing to $18.3 million from $15.6 million [5][6] - General and administrative expenses rose to $23.4 million in 2025 from $7.0 million in 2024, primarily due to higher share-based compensation and increased legal and consulting fees [6] - The net loss attributable to shareholders for the year was $37.9 million, or $1.05 per share, compared to a net loss of $22.4 million, or $1.22 per share, in the previous year [7][10] Balance Sheet Overview - As of June 30, 2025, the company had cash and cash equivalents of $97.7 million, an increase from $50.9 million in 2024 [9] - Total assets were reported at $99.6 million, compared to $52.2 million in the previous year, indicating significant growth in the company's financial position [9] About BB-301 - BB-301 utilizes a modified AAV9 capsid to promote co-expression of a functional version of the PABPN1 protein while silencing the expression of the faulty mutant PABPN1 gene, positioning it as a potential therapy for OPMD [11] About Benitec Biopharma, Inc. - Benitec Biopharma is a clinical-stage biotechnology company focused on developing novel genetic medicines, particularly for chronic and life-threatening conditions like OPMD, using its proprietary "Silence and Replace" platform [12]

Core Viewpoint - Benitec Biopharma Inc. reported its financial results for the third fiscal quarter ended March 31, 2025, highlighting significant increases in expenses and net loss, while also providing updates on its clinical development of BB-301 for Oculopharyngeal Muscular Dystrophy (OPMD) [1][3][4]. Financial Highlights - Total expenses for Q3 2025 were $10.2 million, up from $4.1 million in Q3 2024, marking a 148.8% increase [3]. - Research and development expenses were $6.0 million compared to $2.6 million in the same quarter of the previous year, reflecting a 130.8% increase, primarily due to ongoing clinical development of BB-301 [3]. - General and administrative expenses rose to $4.2 million from $1.6 million year-over-year, an increase of 162.5% [3]. - The loss from operations for Q3 2025 was $10.2 million, compared to a loss of $4.1 million in Q3 2024, indicating a 148.8% increase in operational losses [4]. - Net loss attributable to shareholders for Q3 2025 was $9.4 million, or $0.24 per share, compared to a net loss of $4.3 million, or $0.23 per share, in Q3 2024 [4][7]. - As of March 31, 2025, the company had $103.6 million in cash and cash equivalents, a significant increase from $50.9 million as of June 30, 2024 [4][5]. Clinical Development Updates - The sixth and final subject of Cohort 1 was treated with a low dose of BB-301 in April 2025, with plans to enroll additional subjects at a higher dose later in the year [2]. - BB-301 is designed to treat dysphagia in OPMD patients by promoting co-expression of a functional version of the PABPN1 protein while silencing the expression of the faulty mutant version [8]. Company Overview - Benitec Biopharma Inc. is a clinical-stage biotechnology company focused on developing novel genetic medicines using its proprietary "Silence and Replace" DNA-directed RNA interference platform [9]. - The company aims to address chronic and life-threatening conditions, including OPMD, through innovative therapeutic approaches that combine RNA interference with gene therapy [9].