-Interim clinical study results for the BB-301 Phase 1b/2a Treatment Study including 12-month follow-up results for the first four Cohort 1 completers, 24-month clinical study results for the first Cohort 1 Patient, and interim clinical study results for the first Cohort 2 Patient will be reported in a late-breaking poster presentation at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference on March 9, 2026- HAYWARD, Calif., Feb. 23, 2026 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDA ...

Core Insights - Benitec Biopharma Inc. announced positive results from the BB-301 Phase 1b/2a treatment study, with all four patients in Cohort 1 demonstrating durable responses at the 12-month follow-up [1][4] - The company plans to engage with the FDA in mid-2026 to confirm the pivotal study design for BB-301, which has received Fast Track and Orphan Drug Designations [2][8] - Financial results for the second fiscal quarter ended December 31, 2025, showed total expenses of $13.4 million, an increase from $10.8 million in the same quarter of the previous year [9][10] Clinical Development - All four patients in Cohort 1 were formal responders to BB-301, indicating a durable response at the 12-month follow-up [4] - Patient 1 from Cohort 1 showed continued disease-modifying effects at the 24-month follow-up, with improvements in post-swallow residue and dysphagic symptom burden [6] - Enrollment for Cohort 2 is ongoing, with the first patient treated with a higher dose in Q4 2025, and an interim results update is expected in mid-2026 [7] Responder Analysis - A Responder Analysis was developed to evaluate BB-301 efficacy, including multiple response categories such as patient-reported outcomes and videofluoroscopic swallowing study assessments [3] - The analysis framework combines results from various response categories to assess clinical benefits achieved by each patient [3][11] Financial Performance - Total expenses for the quarter ended December 31, 2025, were $13.4 million, with research and development expenses at $5.8 million, consistent with the previous year [9] - The net loss attributable to shareholders for the quarter was $11.8 million, or $(0.26) per share, unchanged from the previous year [10][14] - As of December 31, 2025, the company had $189 million in cash and cash equivalents [10][13] Corporate Updates - The company is focused on advancing its proprietary "Silence and Replace" DNA-directed RNA interference platform for the treatment of Oculopharyngeal Muscular Dystrophy [16][17] - Benitec has received Orphan Drug Designation from both the EMA and FDA for BB-301, highlighting its potential in treating rare diseases [8][16]

Financial Performance - Total expenses for Q2 2026 were $13.4 million, up from $10.8 million in Q2 2025, reflecting a 24% increase year-over-year[9] - Net loss attributable to shareholders for Q2 2026 was $11.8 million, or $(0.26) per share, compared to a net loss of $9.6 million, or $(0.26) per share in Q2 2025[10] - Total stockholders' equity increased to $187.4 million as of December 31, 2025, compared to $97.3 million as of June 30, 2025[12] - As of December 31, 2025, the company had $189 million in cash and cash equivalents, a significant increase from $97.7 million as of June 30, 2025[12] - There are ongoing losses and future revenue uncertainties, with a need for additional financing depending on market conditions[18] Research and Development - Research and development expenses were $5.8 million, consistent with $5.4 million in Q2 2025, indicating a 7% increase[9] - The company is focused on developing and potentially commercializing its product candidates, with ongoing clinical trials and preclinical studies[18] - The company is exploring the development of novel AAV vectors and potential future out-licenses and collaborations[18] - The company is assessing the efficacy and safety of its products, as well as those of its collaboration partners, in the marketplace[18] Clinical Trials and Studies - All four patients in Cohort 1 of the BB-301 study were formal responders at the 12-month follow-up, demonstrating a durable response[4] - Patient 1 from Cohort 1 showed continued disease-modifying effects at the 24-month follow-up, with improvements in post-swallow residue and dysphagic symptom burden[6] - The first patient in Cohort 2 was treated with a higher dose of BB-301 in Q4 2025, with interim results expected in mid-2026[7] - The FDA granted Fast Track Designation for BB-301 in November 2025, following positive interim clinical results[8] - The company plans to engage with the FDA in mid-2026 to confirm the pivotal study design for BB-301[8] Risks and Challenges - There are risks related to patient enrollment and dosing in future clinical trials, which may affect the timing of data availability[18] - Regulatory approvals and determinations by the FDA and other authorities are critical to the company's future operations[18] - The company is addressing identified material weaknesses in its internal controls over financial reporting[18] - The company is dependent on relationships with collaboration partners and third parties for its success[18] - Market competition and sales, marketing, manufacturing, and distribution requirements are significant factors impacting the company's performance[18] - The company disclaims any obligation to update forward-looking statements, highlighting the inherent uncertainties in its projections[18]

Product Development - BB-301 is the only clinical-stage therapeutic agent in development for Oculopharyngeal Muscular Dystrophy (OPMD), with a commercial opportunity exceeding $1 billion[153]. - The Investigational New Drug (IND) application for BB-301 was approved by the FDA in June 2023, with the first study subject treated in November 2023[147]. - BB-301 utilizes a modified AAV serotype 9 (AAV9) capsid to express a bifunctional construct targeting both functional and mutant PABPN1 genes[154]. - BB-301 has been granted Orphan Drug Designation in both the United States and the European Union, providing potential commercial exclusivity[153]. - The Phase 1b/2a clinical trial for BB-301 is ongoing, with dosing for Cohort 1 completed in Q2 2025 and Cohort 2 initiated in Q4 2025[147]. - The silence and replace approach aims to permanently silence disease-causing genes while delivering functional genes, potentially providing a permanent solution with a single administration[126]. - BB-301 clinical development program includes a 76-week follow-up with a 6-month pre-treatment observation period and a 52-week post-dosing evaluation[157]. - All six subjects in Cohort 1 and the first subject in Cohort 2 have been safely treated with BB-301, with no treatment-related severe adverse events reported[158]. - Interim results from the BB-301 Phase 1b/2a trial show significant improvements in dysphagic symptoms and swallowing function, with the first patient in Cohort 1 demonstrating robust outcomes at 24 months[159]. Financial Performance - Research and development expenses for the three months ended December 31, 2025, were $5.8 million, an increase from $5.4 million in the same period in 2024[175]. - General and administrative expenses totaled $7.5 million for the three months ended December 31, 2025, compared to $5.4 million for the same period in 2024, reflecting an increase in share-based compensation[176]. - The company incurred total operating expenses of $13.4 million for the three months ended December 31, 2025, compared to $10.8 million for the same period in 2024[175]. - Other income for the three months ended December 31, 2025, was $1.5 million, an increase from $1.3 million in the same period in 2024, driven by higher interest income[177]. - The company has accumulated losses of $249 million as of December 31, 2025, with expectations of increased research and development expenses for the OPMD program[178]. - As of December 31, 2025, the company has cash and cash equivalents of approximately $188.8 million, primarily held in bank accounts[180]. - For the six months ended December 31, 2025, net cash used in operating activities was $7.1 million, an improvement from $12.3 million in the same period of 2024[182]. - Net cash provided by financing activities for the six months ended December 31, 2025, was $98.2 million, significantly higher than $39.5 million in 2024, primarily due to the issuance of common stock[184]. - The total number of warrants issued by the company as of December 31, 2025, is 20,179,428 shares of common stock[179]. - The company has not generated any revenue from product sales and does not expect to do so until regulatory approval is obtained for its product candidates[186]. - The company anticipates continued losses for the foreseeable future as it develops product candidates and prepares for commercialization[187]. - The company estimates that its cash and cash equivalents will be sufficient to fund operations for at least the next twelve months[187]. - The company entered into a sales agreement on October 11, 2024, allowing for the sale of up to $75 million of common stock in "at-the-market offerings"[180]. Strategic Focus - The company plans to use cash and cash equivalents for advancing BB-301 and other product candidates, as well as for strategic growth opportunities[145]. - The company is focused on leveraging ddRNAi technology to develop differentiated therapeutics for genetic disorders[143]. - The silence and replace technology overcomes limitations of traditional siRNA approaches, offering robust gene silencing capabilities[137]. - The company is exploring long-term manufacturing alliances to support future commercialization efforts, as it currently does not own manufacturing facilities[160]. - The company anticipates facing intense competition in the biopharmaceutical industry, particularly in the development of gene therapy and gene silencing products[165]. - The company has no products approved for sale and is actively seeking licensing partners for its ddRNAi technology[185]. - The company has ongoing operating leases for office spaces in California, with commitments extending through December 2027 and January 2028[189]. Patient Demographics - OPMD typically presents in patients aged 40-50 and is characterized by progressive swallowing difficulties, with significant geographical clustering noted[146].

Core Viewpoint - Benitec Biopharma Inc. (BNTC) is focused on developing BB-301, a genetic medicine aimed at treating OPMD-related dysphagia, a progressive condition that can lead to severe complications [1]. Group 1: Company Overview - Benitec Biopharma Inc. is a clinical-stage company specializing in genetic medicine [1]. - The lead asset, BB-301, targets a specific medical condition related to OPMD [1]. Group 2: Educational Background of Key Personnel - Myriam Hernandez Alvarez holds multiple degrees, including an Electronics and Telecommunication Engineering degree, a M.Sc. in computer science, a graduate degree in Business Management, and a Ph.D. in computer applications [1].

Core Insights - Benitec Biopharma Inc. announced positive results from the 24-month follow-up of the first patient treated with BB-301 in a clinical study for oculopharyngeal muscular dystrophy (OPMD), showing robust disease-modifying outcomes [1][3][4] Group 1: Clinical Study Results - Patient 1 demonstrated deepening improvements in post-swallow pharyngeal residue and total dysphagic symptom burden at the 24-month follow-up compared to both pre-treatment and 12-month follow-up assessments [1][3][4] - All four patients in Cohort 1 completed the 12-month follow-up, with each showing durable responses to BB-301, meeting pre-specified statistical criteria for improvement [1][4][8] - Patient 1 exhibited a 27% improvement in Pharyngeal Area at Maximum Constriction (PhAMPC) at the 12-month mark, which was maintained at the 24-month follow-up [5] - Patient 1 showed a 35% reduction in post-swallow residue in the vallecular region at 12 months, improving to a 60% reduction at 24 months [7] - Total Pharyngeal Residue (TPR) improved by 32% at 12 months and 39% at 24 months, indicating enhanced swallowing efficiency over time [7] Group 2: Patient-Reported Outcomes - The Sydney Swallow Questionnaire (SSQ) total score indicated a 64% reduction in total dysphagic symptom burden at 12 months, improving to a 78% reduction at 24 months [7] - The Responder Analysis framework was utilized to evaluate the efficacy of BB-301, with all four patients classified as formal responders based on their improvements across multiple assessment categories [6][8] Group 3: Company Overview and Future Plans - Benitec Biopharma is focused on developing novel genetic medicines using its proprietary "Silence and Replace" platform, targeting chronic and life-threatening conditions like OPMD [10][11] - The company plans to engage with the U.S. Food and Drug Administration (FDA) in mid-2026 to confirm the pivotal study design for BB-301 and will present interim clinical results at future medical conferences [2]

Core Viewpoint - Benitec Biopharma Limited (BNTC) has shown a modest gain of 0.2% over the past four weeks, closing at $12.96, with analysts suggesting a potential upside of 83.9% based on a mean price target of $23.83 [1] Price Targets and Analyst Estimates - The mean estimate for BNTC comprises six short-term price targets with a standard deviation of $6.15, indicating variability among analysts; the lowest estimate is $17.00 (31.2% increase), while the highest is $32.00 (146.9% increase) [2] - A low standard deviation suggests a strong agreement among analysts regarding the stock's price movement, which can serve as a starting point for further research [9] Earnings Estimates and Analyst Optimism - Analysts have shown growing optimism regarding BNTC's earnings prospects, as indicated by a positive trend in earnings estimate revisions; the Zacks Consensus Estimate for the current year has increased by 23% over the past month [11][12] - BNTC holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimates, which further supports the stock's potential upside [13] Caution on Price Targets - While price targets are a common metric for investors, relying solely on them may not be prudent due to concerns about analysts' biases and the potential for inflated targets [3][8][10]

Group 1 - Benitec Biopharma Limited (BNTC) closed at $13.18, with a 9.8% gain over the past four weeks, and a mean price target of $24.71 suggests an 87.5% upside potential [1] - The mean estimate includes seven short-term price targets with a standard deviation of $6.07, indicating variability among analysts; the lowest estimate is $17.00 (29% increase), while the highest is $32.00 (142.8% increase) [2] - Analysts show strong agreement in revising earnings estimates higher, which correlates with potential stock price movements [11] Group 2 - The Zacks Consensus Estimate for the current year has increased by 52.3%, with four estimates moving higher and no negative revisions in the last 30 days [12] - BNTC holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimates [13] - While the consensus price target may not be a reliable indicator of the extent of potential gains, it does provide a directional guide for price movement [14]

Core Viewpoint - Benitec Biopharma Limited (BNTC) is experiencing significant selling pressure, with a 25.8% decline over the past four weeks, but is now positioned for a potential trend reversal due to being in oversold territory and positive earnings expectations from analysts [1]. Group 1: Technical Indicators - The Relative Strength Index (RSI) is a momentum oscillator that indicates whether a stock is oversold, with readings below 30 typically signaling this condition [2]. - BNTC's current RSI reading is 28.26, suggesting that the heavy selling may be exhausting itself and a trend reversal could occur soon [5]. Group 2: Fundamental Indicators - There is a strong consensus among sell-side analysts that BNTC will report better earnings than previously predicted, leading to a 55.9% increase in the consensus EPS estimate over the last 30 days [7]. - BNTC holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimate revisions and EPS surprises, indicating a strong potential for a turnaround [8].

Core Insights - Benitec Biopharma Inc. (NASDAQ:BNTC) is identified as a promising investment opportunity following a price target increase from $20 to $22 by Citizens JMP, supported by positive clinical trial results for its treatment BB-301 for Oculopharyngeal Muscular Dystrophy (OPMD) [1][2] - The treatment demonstrated a 100% response rate in Phase 1b/2a trials, leading to the FDA granting Fast Track designation for the gene therapy [2][3] Group 1 - The price target for Benitec Biopharma was raised to $22 from $20, with an Outperform rating reiterated by Citizens JMP [1] - The clinical trial results showed a 100% response rate for BB-301 in treating OPMD, a rare genetic muscle-wasting disease [1][2] - The treatment has effectively reduced swallowing difficulties and shortened the time needed to consume liquids in patients [2] Group 2 - The CEO of Benitec Biopharma expressed excitement over the potential impact of BB-301 on OPMD, highlighting the strength of the clinical data and the urgency of the unmet medical need [3] - Suvretta Capital Management announced a $20 million investment in Benitec Biopharma, indicating confidence in the company's prospects [3] - Benitec Biopharma is a clinical-stage biotechnology company utilizing a proprietary DNA-directed RNA interference (ddRNAi) platform to develop genetic medicines [3]