Benitec Biopharma(BNTC) - 2025 Q4 - Annual Results

Company Overview & Recent Developments Executive Summary & Key Announcements Benitec Biopharma announced FY2025 results and operational updates, anticipating BB-301 Cohort 1 interim results and Cohort 2 enrollment in Q4 2025, utilizing its "Silence and Replace" ddRNAi platform - Interim clinical study results for Cohort 1 of the BB-301 Phase 1b/2a Treatment Study are anticipated in Q4 2025[1] - Enrollment of the first subject into Cohort 2 of the BB-301 Phase 1b/2a Treatment Study is expected in Q4 2025[1] - Benitec Biopharma is a clinical-stage, gene therapy-focused biotechnology company developing novel genetic medicines based on its proprietary "Silence and Replace" DNA-directed RNA interference ("ddRNAi") platform[1] Corporate Highlights & Clinical Program Update Following DSMB recommendation, Benitec Biopharma plans to continue BB-301 Cohort 2 enrollment in Q4 2025 and provide additional Cohort 1 interim results - The Independent Data Safety Monitoring Board (DSMB) formally recommended continuing subject enrollment into the Phase 1b/2a Treatment Study, expected to begin in Q4 2025[3] - Additional interim clinical study results for Cohort 1 Subjects are expected in the fourth calendar quarter of this year[4] - The CEO expressed strong enthusiasm for BB-301's potential as a safe and efficacious therapy for improving swallowing in OPMD patients with dysphagia[2] About Benitec Biopharma, Inc. Benitec Biopharma Inc. is a clinical-stage biotechnology company advancing novel genetic medicines using its proprietary "Silence and Replace" ddRNAi platform, primarily focused on Oculopharyngeal Muscular Dystrophy (OPMD) - Benitec Biopharma Inc. is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines[12] - The proprietary "Silence and Replace" DNA-directed RNA interference platform combines RNA interference with gene therapy to facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes[12] - The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions, including Oculopharyngeal Muscular Dystrophy (OPMD)[12] Financial Performance Full Year 2025 Financial Highlights Benitec Biopharma reported increased total expenses, R&D, and G&A costs for FY2025, leading to a higher net loss, yet cash and cash equivalents nearly doubled year-over-year Key Financial Metrics (Year Ended June 30, in thousands) | Metric | FY2025 | FY2024 | Change (YoY) | | :----------------------------------- | :----- | :----- | :----------- | | Total Expenses | $41,765 | $22,490 | +85.7% | | Research and Development Expenses | $18,332 | $15,609 | +17.4% | | General and Administrative Expenses | $23,433 | $6,989 | +235.3% | | Loss from Operations | $(41,765) | $(22,490) | +85.7% | | Net Loss Attributable to Shareholders | $(37,917) | $(22,370) | +69.5% | | Net Loss per Share (Basic & Diluted) | $(1.05) | $(1.22) | -13.9% | | Cash and Cash Equivalents (as of June 30) | $97,744 | $50,866 | +92.2% | - The increase in research and development expenses was primarily related to the ongoing clinical development of BB-301 for OPMD, reflecting contract manufacturing and OPMD Natural History and Dosing study payments[5] - The increase in general and administrative expenses was primarily driven by $14.5 million higher share-based compensation, alongside increases in legal, consulting, travel, and salary expenses[6] Consolidated Balance Sheets As of June 30, 2025, Benitec Biopharma reported substantial increases in cash, total assets, and stockholders' equity, while total liabilities decreased over 50% year-over-year Consolidated Balance Sheet Highlights (as of June 30, in thousands) | Item | 2025 | 2024 | Change (YoY) | | :------------------------- | :----- | :----- | :----------- | | Cash and cash equivalents | $97,744 | $50,866 | +92.2% | | Total current assets | $98,518 | $51,674 | +90.7% | | Total assets | $99,592 | $52,210 | +90.7% | | Total current liabilities | $1,802 | $4,924 | -63.4% | | Total liabilities | $2,297 | $4,962 | -53.7% | | Additional paid-in capital | $326,308 | $238,398 | +36.9% | | Accumulated deficit | $(228,176) | $(190,259) | +20.0% | | Total stockholders' equity | $97,295 | $47,248 | +106.0% | Consolidated Statements of Operations and Comprehensive Loss For FY2025, Benitec Biopharma reported no revenues, a $37.9 million net loss (up from $21.8 million), driven by increased operating expenses despite higher interest income Consolidated Statements of Operations Highlights (Year Ended June 30, in thousands) | Item | 2025 | 2024 | Change (YoY) | | :----------------------------------- | :----- | :----- | :----------- | | Revenues | $0 | $0 | 0% | | Royalties and license fees | $0 | $(108) | N/A | | Research and development | $18,332 | $15,609 | +17.4% | | General and administrative | $23,433 | $6,989 | +235.3% | | Total operating expenses | $41,765 | $22,490 | +85.7% | | Loss from operations | $(41,765) | $(22,490) | +85.7% | | Interest income (expense), net | $3,286 | $904 | +263.5% | | Net loss | $(37,917) | $(21,751) | +74.3% | | Net loss attributable to common shareholders | $(37,917) | $(22,370) | +69.5% | | Basic and diluted net loss per share | $(1.05) | $(1.22) | -13.9% | | Weighted average shares outstanding | 36,209,271 | 18,364,386 | +97.2% | - A gain on extinguishment of liabilities of $764 thousand was recognized in 2025, compared to none in 2024[10] Product & Technology About BB-301 BB-301 is a novel gene therapy for OPMD, using a modified AAV9 capsid to deliver a bifunctional construct that simultaneously silences the mutant PABPN1 gene and replaces it with a functional version - BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct[11] - The construct promotes co-expression of codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against mutant PABPN1, the causative gene for OPMD[11] - The "silence and replace" mechanism of BB-301 is uniquely positioned for OPMD treatment by halting mutant expression while providing a functional replacement protein[11] Forward-Looking Information Forward Looking Statements This section outlines forward-looking statements regarding Benitec's product development, clinical trials, regulatory processes, and ddRNAi platform benefits, which are subject to inherent risks and uncertainties - Forward-looking statements include plans for product candidate development and commercialization, timing of preclinical and clinical trials, data availability, patient enrollment, regulatory filings, and the clinical utility and potential attributes of ddRNAi and Benitec's product candidates[13] - These statements are based on current expectations and are subject to risks and uncertainties, including unanticipated developments in clinical trials, regulatory approvals, intellectual property, financing needs, and market conditions[14][15] - The Company disclaims any intent or obligation to update these forward-looking statements[15] Additional Information Investor Relations Contact This section provides contact details for investor relations inquiries - Investor Relations Contact: Irina Koffler, LifeSci Advisors, LLC[16] - Contact details: (917) 734-7387, ikoffler@lifesciadvisors.com[16]