Benitec Biopharma(BNTC)

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Benitec Biopharma(BNTC) - 2025 Q1 - Quarterly Report
2024-11-14 21:32
Product Development and Technology - Benitec Biopharma is developing BB-301, an AAV-based gene therapy for Oculopharyngeal Muscular Dystrophy (OPMD), which has received Orphan Drug Designation in both the United States and the European Union[84]. - The silence and replace approach aims to permanently silence disease-causing genes while simultaneously replacing them with wildtype genes, potentially improving treatment outcomes for chronic diseases[86]. - The proprietary ddRNAi technology allows for sustained silencing of disease-causing genes through a single administration, reducing the frequency of drug administration typically required for chronic disease management[84]. - Benitec's technology could facilitate the development of therapeutics for over 20,000 human genes, addressing significant unmet medical needs in genetic disorders[87]. - The silence and replace technology combines RNAi with gene therapy to overcome limitations of traditional gene silencing approaches, enhancing the efficacy of treatments for genetic disorders[91]. - Benitec's proprietary AAV vector technology improves the delivery of genetic constructs, enhancing the potential for effective gene therapy applications[94]. - BB-301 employs a "silence and replace" mechanism, achieving 75% silencing of mutant PABPN1 and 26% replacement of wild type PABPN1 activity in preclinical models[104]. - BB-301 demonstrated robust intracellular silencing of PABPN1 protein production and restoration of muscle strength in the A17 mouse model[104]. - BB-301 is designed to be administered via direct intramuscular injection, enhancing the precision of treatment delivery[108]. - The BB-301 Pilot Dosing Study in Beagle dogs confirmed dose-dependent transduction efficiency and gene expression levels, with significant biological activity observed[108]. - BB-301 demonstrated a 248-fold improvement (+24,650%) in transduction of the HP muscle and a 111-fold improvement (+11,027%) in transduction of the TP muscle compared to previous studies[112]. Clinical Development and Regulatory Progress - BB-301 is in clinical development for Oculopharyngeal Muscular Dystrophy (OPMD) with the first study subject treated in November 2023 and subsequent subjects treated in February and October 2024[101]. - The Investigational New Drug (IND) application for BB-301 was approved by the U.S. FDA in June 2023, indicating regulatory progress[101]. - The BB-301 clinical development program includes approximately 76 weeks of follow-up, with a 6-month pre-treatment observation period for evaluating baseline dysphagia[113]. - The primary endpoint of the Phase 1b/2a study is safety, with secondary endpoints evaluating swallowing efficiency and muscle function[114]. - Subject 1 experienced a 35% reduction in SSQ Total Score and a 33% reduction in VFSS TPR for thin liquids post-BB-301 treatment[118]. - Subject 2 showed an 89% reduction in SSQ Total Score and a 92% reduction in the frequency of low-volume sequential swallows post-BB-301 treatment[120]. - The NH Study has enrolled 23 subjects as of January 2024, assessing dysphagia progression through quantitative measures[114]. Financial Performance and Funding - The Company raised approximately $27.9 million in net proceeds from an underwritten public offering on August 11, 2023, selling 875,949 shares of common stock and warrants[132]. - For the three months ended September 30, 2024, the Company incurred research and development expenses of $3.6 million, a decrease from $4.4 million in the same period in 2023[145]. - General and administrative expenses increased to $2.2 million for the three months ended September 30, 2024, compared to $1.6 million in the same period in 2023[146]. - The Company reported total other income of $732 thousand for the three months ended September 30, 2024, compared to a loss of $80 thousand in the same period in 2023[147]. - As of September 30, 2024, the Company had accumulated losses of $195 million since inception[148]. - The Company had cash and cash equivalents of approximately $67.8 million as of September 30, 2024[150]. - Net cash used in operating activities was $4.6 million for both the three months ended September 30, 2024 and 2023[152]. - The Company provided $21.7 million in net cash from financing activities for the three months ended September 30, 2024, down from $27.9 million in the same period in 2023[153]. - The Company has not generated any revenue from product sales and does not expect to do so until regulatory approval is obtained[155]. - The Company entered into a Sales Agreement on October 11, 2024, allowing for the sale of up to $75 million of common stock[135]. - The company anticipates continued losses for the foreseeable future, with expectations that losses will increase as product candidates are developed and prepared for commercialization[156]. - Cash and cash equivalents are estimated to be sufficient to fund operations for at least the next twelve months[156]. - Future funding requirements will depend on various factors, including clinical trial costs and regulatory approval outcomes[157]. Corporate Strategy and Partnerships - The company plans to utilize its cash reserves for advancing BB-301 and other product candidates, as well as for general corporate purposes and strategic growth opportunities[92]. - The company aims to establish partnerships with global biopharmaceutical companies to co-develop and commercialize its ddRNAi-based products, leveraging its unique scientific platform[95]. - Manufacturing of gene therapy products is complex, and the company is exploring long-term alliances for production[121]. - The company has not yet established sales or marketing operations, pending clinical trial results and regulatory approvals[123]. Internal Controls and Compliance - A material weakness in internal control over financial reporting has been identified, affecting the ability to process complex transactions[171]. - The company plans to retain additional accounting experts to assist in remediating the identified material weakness[171]. - No changes in internal controls over financial reporting have materially affected the company during the quarter ended September 30, 2024[173]. - The company is currently evaluating the impact of recently issued accounting standards on its financial disclosures[167].
Benitec Biopharma Releases First Quarter 2025 Financial Results and Provides Operational Update
GlobeNewswire News Room· 2024-11-14 21:30
-Positive 270-day Interim Clinical Study Data for the First Subject and Positive 180-day Interim Clinical Study Data for the Second Subject Treated with the Low-Dose of BB-301 in the Phase 1b/2a Clinical Treatment Study Reported in October as a Late-Breaking Oral Presentation at the 29th Annual Congress of the World Muscle Society- -Third Subject Safely Treated with the Low-Dose of BB-301 in October 2024, and Fourth Subject Expected to Receive Treatment with the Low-Dose of BB-301 in December 2024 HAYWARD, ...
Benitec Biopharma CEO to Participate in the Guggenheim Securities Healthcare Conference
GlobeNewswire News Room· 2024-11-04 13:00
Core Insights - 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 [3] Group 1: Company Overview - Benitec is headquartered in Hayward, California and specializes in gene therapy aimed at chronic and life-threatening conditions, including Oculopharyngeal Muscular Dystrophy (OPMD) [3] - The "Silence and Replace" platform combines RNA interference with gene therapy to enable sustained silencing of disease-causing genes while delivering wildtype replacement genes in a single administration [3] Group 2: Upcoming Events - CEO Jerel A. Banks will participate in the Guggenheim Securities Healthcare Conference in Boston from November 11-13, 2024 [1] - The presentation will include a fireside chat and one-on-one meetings, with a live webcast available for interested parties [2]
Benitec Biopharma: OPMD Program Continues To Advance With Catalysts
Seeking Alpha· 2024-10-17 19:35
Group 1 - Benitec Biopharma Inc. (NASDAQ: BNTC) reported positive results from its phase 1b/2a clinical study using BB-301 for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD) [1] - The positive interim results were reported at two time points during the study [1] Group 2 - The article is authored by Terry Chrisomalis, who runs the Biotech Analysis Central pharmaceutical service on Seeking Alpha Marketplace [1] - The Biotech Analysis Central service offers a deep-dive analysis of various pharmaceutical companies, including a library of over 600 biotech investing articles and a model portfolio of small and mid-cap stocks [1]
Benitec Biopharma Announces Updated Investor Webcast Information
GlobeNewswire News Room· 2024-10-14 10:00
-Management plans to host an investor webcast on October 14th at 8:30 am EDT to provide an update for the BB-301 Phase 1b/2a Clinical Study, details below- -The webcast access information has been revised from the conference call information provided on October 12th- HAYWARD, Calif., Oct. 14, 2024 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) ("Benitec" or "Company"), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary "Silenc ...
Benitec Biopharma Reports Positive Data from Two Subjects Treated with Low-Dose BB-301 in Phase 1b/2a Study Presented at 29th Annual Congress of the World Muscle Society
GlobeNewswire News Room· 2024-10-12 10:30
-Subject 1 and Subject 2 experienced durable, clinically meaningful improvements in swallowing at 9-months and 6-months post-BB-301 treatment, respectively, with Subject 2 achieving a Sydney Swallow Questionnaire Score Representative of Clinically Normal Swallowing- -Management plans to host a conference call on October 14 at 8:30 am EDT to discuss the interim results, details below- HAYWARD, Calif., Oct. 12, 2024 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) ("Benitec" or "Company"), a clinical ...
Benitec Biopharma(BNTC) - 2024 Q4 - Annual Results
2024-09-26 20:43
Exhibit 99.1 LOGO Benitec Biopharma Releases Full Year 2024 Financial Results and Provides Operational Update -Positive 90-day and 180-day Interim Clinical Trial Data for the First Oculopharyngeal Muscular Dystrophy (OPMD) Subject Dosed with the Low-Dose of BB-301 in the Phase 1b/2a Clinical Treatment Study Reported in April and July- -Second Subject Dosed with the Low-Dose of BB-301 in February 2024, and Third Subject Expected to Receive the Low-Dose of BB-301 in Calendar Quarter Four of 2024 -Closed an Ov ...
Benitec Biopharma(BNTC) - 2024 Q4 - Annual Report
2024-09-26 20:30
Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ Annual Report Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 For the fiscal year ended June 30, 2024 ☐ Transition Report under Section 13 or 15(d) of the Securities Exchange Act of 1934 For the transition period from to Commission File Number: 001-39267 Benitec Biopharma Inc. (Exact name of registrant as specified in its charter) Delaware 84-462-0206 (State or other jurisd ...
Benitec Biopharma Releases Full Year 2024 Financial Results and Provides Operational Update
GlobeNewswire News Room· 2024-09-26 20:30
-Positive 90-day and 180-day Interim Clinical Trial Data for the First Oculopharyngeal Muscular Dystrophy (OPMD) Subject Dosed with the Low-Dose of BB-301 in the Phase 1b/2a Clinical Treatment Study Reported in April and July- -Second Subject Dosed with the Low-Dose of BB-301 in February 2024, and Third Subject Expected to Receive the Low-Dose of BB-301 in Calendar Quarter Four of 2024- -Closed an Oversubscribed Private Placement Financing of $40.0 Million on April 22nd, Cash Runway Extended Through 2025- H ...
Benitec Biopharma Announces Late Breaking Oral Abstract Presentation on BB-301 Phase 1b/2a Clinical Study at the 29th Annual Congress of the World Muscle Society
GlobeNewswire News Room· 2024-09-18 12:00
HAYWARD, Calif., Sept. 18, 2024 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) ("Benitec" or the "Company"), 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, today announced that a Late Breaking Abstract on BB-301 was accepted for oral presentation at the 29th Annual Congress of the World Muscle Society, taking place in Prague, Czech Republic from Octob ...