Benitec Biopharma(BNTC) - 2024 Q2 - Quarterly Report

PART I - FINANCIAL INFORMATION This section presents the company's financial statements, management's discussion and analysis, market risk disclosures, and controls and procedures Item 1. Financial Statements The financial statements for the period ended December 31, 2023, reflect a substantial increase in cash and cash equivalents to $20.4 million due to financing activities, alongside a widened net loss of $12.8 million driven by increased research and development expenses Consolidated Balance Sheets As of December 31, 2023, cash and cash equivalents significantly increased to $20.4 million, driving total assets to $21.4 million and stockholders' equity to $15.5 million, primarily due to capital raises Consolidated Balance Sheet Highlights (in thousands) | Balance Sheet Item | Dec 31, 2023 (Unaudited) | June 30, 2023 | | :--- | :--- | :--- | | Assets | | | | Cash and cash equivalents | $20,374 | $2,477 | | Total current assets | $20,796 | $3,729 | | Total assets | $21,353 | $4,464 | | Liabilities & Equity | | | | Total current liabilities | $5,746 | $3,978 | | Total liabilities | $5,883 | $4,262 | | Total stockholders' equity | $15,470 | $202 | Consolidated Statements of Operations and Comprehensive Loss For the six months ended December 31, 2023, the company reported no revenue, with net loss widening to $12.8 million due to increased research and development expenses Statement of Operations Summary (in thousands, except per share data) | Metric | Six Months Ended Dec 31, 2023 | Six Months Ended Dec 31, 2022 | | :--- | :--- | :--- | | Total revenues | $0 | $14 | | Research and development | $9,531 | $6,421 | | General and administrative | $3,375 | $3,783 | | Loss from operations | $(12,801) | $(10,190) | | Net loss | $(12,752) | $(10,504) | | Net loss per share (Basic & Diluted) | $(5.39) | $(9.30) | Consolidated Statements of Stockholders' Equity Stockholders' equity significantly increased from $0.2 million to $15.5 million for the six months ended December 31, 2023, primarily driven by $27.9 million in capital raised from stock and warrant issuances - Total stockholders' equity increased from $202 thousand at June 30, 2023, to $15.47 million at December 31, 2023[122] - The company raised $27.9 million from the issuance of common stock and warrants, net of $2.96 million in offering costs, during the six months ended December 31, 2023[122] Consolidated Statements of Cash Flows For the six months ended December 31, 2023, net cash used in operating activities was $9.9 million, offset by $28.0 million provided by financing activities, resulting in a $17.9 million net increase in cash Cash Flow Summary (in thousands) | Cash Flow Activity | Six Months Ended Dec 31, 2023 | Six Months Ended Dec 31, 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | $(9,942) | $(9,888) | | Net cash provided by financing activities | $27,958 | $16,015 | | Net increase in cash | $17,898 | $6,475 | | Cash at end of period | $20,388 | $10,551 | Notes to Consolidated Financial Statements (Unaudited) The notes detail the company's genetic medicine focus, a 1-for-17 reverse stock split, and the impact of a $27.9 million public offering on liquidity, which is expected to fund operations for at least twelve months - The company's business focuses on developing novel genetic medicines using its proprietary DNA-directed RNA interference (ddRNAi) platform[125] - A 1-for-17 reverse stock split of common stock was effected on July 26, 2023. All share and per-share amounts in the report reflect this split[127] - As of December 31, 2023, the company had $20.4 million in cash and cash equivalents, which is estimated to be sufficient to fund operations for at least the next twelve months[167] - In August 2023, the company closed an underwritten public offering, raising net proceeds of $27.9 million through the sale of common stock, pre-funded warrants, and common warrants[174] Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations Management discusses the company's clinical-stage biotechnology focus, the advancement of its lead program BB-301 for OPMD, and the significant strengthening of its financial condition by a $27.9 million capital raise, providing a cash runway for at least 12 months Company Overview and Strategy Benitec is a clinical-stage biotechnology company focused on developing genetic medicines using its proprietary ddRNAi and "silence and replace" technology, aiming to develop and commercialize treatments for genetic disorders - Benitec is a clinical-stage biotechnology company focused on advancing novel genetic medicines using its proprietary DNA-directed RNA interference (ddRNAi) platform[210] - The company's unique "silence and replace" approach combines RNA interference (RNAi) with gene therapy to silence disease-causing genes while simultaneously delivering wildtype replacement genes[210] - The company's strategy is to develop proprietary programs, explore partnerships with large biopharmaceutical companies, and actively protect its intellectual property[239] Our Pipeline: BB-301 for OPMD BB-301, the company's lead AAV-based gene therapy for OPMD, received FDA IND approval in June 2023, with the first patient dosed in its Phase 1b/2a clinical trial in November 2023, alongside an ongoing Natural History study - BB-301 is the lead investigational agent for Oculopharyngeal Muscular Dystrophy (OPMD), a degenerative muscle disorder[15][16] - The Investigational New Drug (IND) application for BB-301 was approved by the FDA in June 2023[15][27] - The first subject in the BB-301 Phase 1b/2a clinical trial was dosed in November 2023[15][29] - The clinical development program includes a Natural History (NH) study to establish baseline dysphagia progression before subjects are enrolled into the treatment study. As of January 2024, 23 subjects are enrolled in the NH study[29][282] Intellectual Property, Manufacturing, and Competition Benitec's intellectual property for OPMD includes five patent families, the company relies on third-party CMOs for manufacturing, and despite intense competition, it is unaware of other gene therapy approaches specifically for OPMD - The patent portfolio for OPMD includes five patent families covering the therapeutic candidate BB-301, the 'silence and replace' treatment strategy, and the AAV delivery system[31] - The company does not own or operate manufacturing facilities and relies on contract manufacturing organizations for the production of its product candidates under cGMP conditions[36] - The company faces intense competition but is not aware of any other companies developing a gene therapy or gene silencing approach for OPMD[290] Government Regulation The company's gene therapy products are subject to extensive FDA regulation, requiring IND and BLA submissions, with BB-301 having received Orphan Drug Designation in the U.S. and E.U., and post-approval regulations including cGMP - Gene therapy products are regulated as biologics by the FDA's Center for Biologics Evaluation and Research (CBER)[67] - The approval process requires submitting an Investigational New Drug (IND) application before human trials and a Biologics License Application (BLA) for marketing approval[68][263] - The FDA has created the Regenerative Medicine Advanced Therapy (RMAT) designation to expedite the development of certain gene therapies[67] - BB-301 has received Orphan Drug Designation in the United States and the European Union, which provides benefits such as market exclusivity upon approval[16][215][325] Results of Operations For the six months ended December 31, 2023, the company reported no revenue, with research and development expenses increasing to $9.5 million due to BB-301 clinical development, resulting in a total operating loss of $12.8 million Operating Expenses (in thousands) | Expense Category | Six Months Ended Dec 31, 2023 | Six Months Ended Dec 31, 2022 | | :--- | :--- | :--- | | Royalties and License Fees | $(105) | $0 | | Research and development | $9,531 | $6,421 | | General and administrative | $3,375 | $3,783 | | Total operating expenses | $12,801 | $10,204 | - The increase in R&D expenses is primarily related to the ongoing clinical development of BB-301 for the treatment of OPMD[344] - The decrease in G&A expenses for the six-month period was mainly due to lower stock-based compensation ($70k) and legal fees ($396k)[103] Liquidity and Capital Resources As of December 31, 2023, the company had $20.4 million in cash and cash equivalents, with liquidity significantly bolstered by a $28.0 million public offering, which management believes is sufficient to fund operations for at least the next twelve months - As of December 31, 2023, the company had cash and cash equivalents of approximately $20.4 million[346] - Net cash used in operating activities was $9.9 million for the six months ended December 31, 2023[91] - Net cash provided by financing activities was $28.0 million for the six months ended December 31, 2023, primarily from the August 2023 public offering[360] - The company estimates that its current cash and cash equivalents will be sufficient to fund operations for at least the next twelve months from the date of the report[92] Item 3. Quantitative and Qualitative Disclosures About Market Risk As a smaller reporting company, Benitec Biopharma Inc. is not required to provide the information requested under this item - The company is not required to provide information for this item as it qualifies as a smaller reporting company[111] Item 4. Controls and Procedures Management concluded that the company's disclosure controls and procedures were effective as of December 31, 2023, with no material changes to internal control over financial reporting during the quarter - Based on an evaluation as of the end of the period, the principal executive officer and principal financial officer concluded that the company's disclosure controls and procedures are effective[95] - There were no changes in internal controls over financial reporting during the quarter ended December 31, 2023, that have materially affected, or are reasonably likely to materially affect, internal control over financial reporting[353] PART II - OTHER INFORMATION This section covers legal proceedings, risk factors, equity sales, defaults, mine safety, other information, and exhibits Item 1. Legal Proceedings The company reports that it is not currently a party to any material legal proceedings - The company is not currently a party to any material legal proceedings[113][354] Item 1A. Risk Factors There have been no material changes to the risk factors previously disclosed in the company's Annual Report on Form 10-K for the fiscal year ended June 30, 2023 - There have been no material changes to the risk factors disclosed in the Company's Annual Report on Form 10-K for the fiscal year ended June 30, 2023[366] Item 2. Unregistered Sales of Equity Securities and Use of Proceeds This section was noted in the table of contents but no specific information regarding unregistered sales was provided in the corresponding text - The report indicates this item but does not provide specific details on unregistered sales of equity securities in the reviewed chunks[9][115] Item 3. Defaults Upon Senior Securities The company reports that there were no defaults upon senior securities during the period - None reported[97][369] Item 4. Mine Safety Disclosures This item is not applicable to the company - Not applicable[100] Item 5. Other Information The company reports that none of its directors or officers adopted, modified, or terminated a Rule 10b5-1 trading arrangement or a non-Rule 10b5-1 trading arrangement during the quarter ended December 31, 2023 - During the quarter ended December 31, 2023, none of the company's directors or officers adopted, modified, or terminated a Rule 10b5-1 trading arrangement or a non-Rule 10b5-1 trading arrangement[367] Item 6. Exhibits This section lists the exhibits filed with the Form 10-Q, including amendments to compensation plans, CEO/CFO certifications pursuant to the Sarbanes-Oxley Act, and Inline XBRL documents - Exhibits filed include the Second Amendment to the 2020 Equity and Incentive Compensation Plan, CEO and CFO certifications (Sections 302 and 906), and various Inline XBRL documents[368]