BNTC
Benitec BiopharmaDDocument history
Earnings documents stored for BNTC.
Investor releaseQuarter not tagged2026-05-14Benitec Biopharma Releases Third Quarter 2026 Financial Results and Provides Operational Update
GlobeNewswire
Benitec Biopharma Releases Third Quarter 2026 Financial Results and Provides Operational Update
Late-breaking abstract for BB-301 presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in March demonstrated durable responses to low dose BB-301 at 12-months and 24-months post-treatment and further improvements in depth of response following treatment with high dose BB-301 at the 3-month post-treatment follow-up time-point, with both doses demonstrating disease modifying outcomes BB-301 Phase 1b/2a interim clinical study results selected for oral presentation at the 2026 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting on May 15 Advancement of Cohort 2 enrollment continues as Patient 1 and Patient 2 were both safely treated with high dose BB-301 in the ongoing Phase 1b/2a clinical study of BB-301; Benitec remains on track to provide updated interim clinical study results in 2H-2026 The Company remains on track to engage with U.S. Food and Drug Administration (FDA) mid-year to formalize the BB-301 pivotal study design Benitec is well-capitalized to advance BB-301 through completion of the BB-301 pivotal study with cash as of March 31, 2026, of approximately $184.8 million. HAYWARD, Calif., May 14, 2026 (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 financial results for its third fiscal quarter ended March 31, 2026, and provided a review of recent clinical study results and anticipated upcoming regulatory and clinical study milestones. “This has been a critical period of continued advancements for the BB-301 clinical development program, marked by strongly encouraging clinical progress for patients treated with low dose BB-301 in Cohort 1 and the progression of enrollment for Cohort 2 with the safe treatment of Patient 1 and Patient 2 with high dose BB-301,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “We look forward to the meaningful milestones ahead, including expanding visibility of our interim clinical study results and engaging with the FDA to confirm the pivotal study design required to support BB-301 approval. We are well-positioned to continue to advance BB-301, the first and only potential disease-mod…Read full documentShow less
Late-breaking abstract for BB-301 presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in March demonstrated durable responses to low dose BB-301 at 12-months and 24-months post-treatment and further improvements in depth of response following treatment with high dose BB-301 at the 3-month post-treatment follow-up time-point, with both doses demonstrating disease modifying outcomes BB-301 Phase 1b/2a interim clinical study results selected for oral presentation at the 2026 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting on May 15 Advancement of Cohort 2 enrollment continues as Patient 1 and Patient 2 were both safely treated with high dose BB-301 in the ongoing Phase 1b/2a clinical study of BB-301; Benitec remains on track to provide updated interim clinical study results in 2H-2026 The Company remains on track to engage with U.S. Food and Drug Administration (FDA) mid-year to formalize the BB-301 pivotal study design Benitec is well-capitalized to advance BB-301 through completion of the BB-301 pivotal study with cash as of March 31, 2026, of approximately $184.8 million. HAYWARD, Calif., May 14, 2026 (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 financial results for its third fiscal quarter ended March 31, 2026, and provided a review of recent clinical study results and anticipated upcoming regulatory and clinical study milestones. “This has been a critical period of continued advancements for the BB-301 clinical development program, marked by strongly encouraging clinical progress for patients treated with low dose BB-301 in Cohort 1 and the progression of enrollment for Cohort 2 with the safe treatment of Patient 1 and Patient 2 with high dose BB-301,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “We look forward to the meaningful milestones ahead, including expanding visibility of our interim clinical study results and engaging with the FDA to confirm the pivotal study design required to support BB-301 approval. We are well-positioned to continue to advance BB-301, the first and only potential disease-modifying therapy for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD)-related dysphagia and are now one step closer to reaching the patients and families of the OPMD community.” Recent Clinical Study Highlights The Company continues to advance the BB-301 Phase 1b/2a Clinical Study evaluating the safety and clinical efficacy of locally-delivered BB-301 for the treatment of OPMD-related dysphagia. Key interim clinical study results were presented at the 2026 MDA Clinical & Scientific Conference demonstrating durable responses to low dose BB-301 at 12-months and 24-months and further improvements in depth of response following treatment with high dose BB-301 at the 3-month post-treatment follow-up time-point. BB-301 Phase 1b/2a interim clinical study results have been selected for oral presentation at the 2026 ASGCT Annual Meeting on May 15 in Boston, MA. BB-301 Development Program Updates All six Cohort 1 patients have safely completed the 12-month post-BB-301-treatment follow-up period and continue to experience robust responses to the low dose of BB-301. The first two Cohort 2 patients have been safely treated with the high dose of BB-301, and available interim clinical results continue to demonstrate deep, disease-modifying efficacy for the high dose of BB-301. No treatment-related severe adverse events have been observed. Upcoming Regulatory and Clinical Milestones The Company remains on track to engage with FDA mid-year to formalize the BB-301 pivotal study design. Benitec remains on track to provide updated interim clinical study results for Cohort 1 patients and Cohort 2 patients in 2H-2026. Financial Highlights Third Quarter 2026 Financial Results Total Expenses for the quarter ended March 31, 2026, were $13.6 million compared to $15.3 million for the quarter ended March 31, 2025. The Company incurred $6.3 million of research and development expenses which was in line with $6.5 million for the comparable quarter ended March 31, 2025. Research and development expenses relate primarily to ongoing clinical development of BB-301 for the treatment of OPMD. General and administrative expenses were $7.3 million compared to $8.8 million for the quarter ended March 31, 2025. The loss from operations for the quarter ended March 31, 2026, was $13.6 million compared to a loss of $15.3 million for the quarter ended March 31, 2025. Net loss attributable to shareholders for the quarter ended March 31, 2026, was $11.9 million, or $(0.24) per basic and diluted share, compared to a net loss of $14.5 million, or $(0.38) per basic and diluted share for the quarter ended March 31, 2025. As of March 31, 2026, the Company had $184.8 million in cash and cash equivalents, which includes $0.5 million from the exercise of warrants during the nine-month period ended March 31, 2026. About BB-301 BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against mutant PABPN1 (the causative gene for OPMD). The two siRNAs are modeled into microRNA backbones to silence expression of faulty mutant PABPN1, while allowing expression of the codon-optimized PABPN1 to replace the mutant with a functional version of the protein. We believe the silence and replace mechanism of BB-301 is uniquely positioned for the treatment of OPMD by halting mutant PABPN1 expression while providing a functional replacement protein. BB-301 has received Orphan Drug Designation from the EMA and Orphan Drug and Fast Track Designations from the FDA. About Benitec Biopharma, Inc. Benitec Biopharma Inc. (“Benitec” or the “Company”) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary “Silence and Replace” DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview of the Company can be found on Benitec’s website at www.benitec.com. Forward Looking Statements Except for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec’s plans to develop and commercialize its product candidates, the timing of the completion of preclinical and clinical trials, the timing of the availability of data from our clinical trials, the timing and sufficiency of patient enrollment and dosing in clinical trials, the timing of expected regulatory filings and other regulatory steps, and the clinical utility and potential attributes and benefits of ddRNAi and Benitec’s product candidates, and other forward looking statements. These forward-looking statements are based on the Company’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: the success of our plans to develop and potentially commercialize our product candidates; the timing of the completion of preclinical studies and clinical trials; the timing and sufficiency of patient enrollment and dosing in any future clinical trials; the timing of the availability of data from our clinical trials; the timing and outcome of regulatory filings and approvals; the development of novel AAV vectors; our potential future out-licenses and collaborations; the plans of licensees of our technology; the clinical utility and potential attributes and benefits of ddRNAi and our product candidates, including the potential duration of treatment effects and the potential for a “one shot” cure; our intellectual property position and the duration of our patent portfolio; expenses, ongoing losses, future revenue, capital needs and needs for additional financing, and our ability to access additional financing given market conditions and other factors; the length of time over which we expect our cash and cash equivalents to be sufficient to execute on our business plan; unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities and other regulatory developments; the Company’s ability to protect and enforce its patents and other intellectual property rights; the Company’s dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Company’s products and the products of the Company’s collaboration partners; the acceptance of the Company’s products and the products of the Company’s collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the impact of, and our ability to remediate, the identified material weakness in our internal control over financial reporting; the Company’s ability to satisfy its capital needs through increasing revenue and obtaining additional financing; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company’s reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements. Investor Relations Contact: Candice Masse astr partners [email protected] Media Contact: Audra Friis Sam Brown Healthcare Communications (917) 519-9577 [email protected] Source: Benitec Biopharma Inc.
Investor releaseQuarter not tagged2026-04-28Benitec Biopharma Announces Oral Presentation of Interim Phase 1b/2a Clinical Study Results for High Dose BB-301 and Continued Durable Improvements for Low Dose BB-301 at the 2026 American Society of Gene and Cell Therapy Annual Meeting
GlobeNewswire
Benitec Biopharma Announces Oral Presentation of Interim Phase 1b/2a Clinical Study Results for High Dose BB-301 and Continued Durable Improvements for Low Dose BB-301 at the 2026 American Society of Gene and Cell Therapy Annual Meeting
- Interim clinical results for the BB-301 Phase 1b/2a study include 12-month post-treatment follow-up results for the first four Cohort 1 completers, 24-month post-treatment follow-up results for the first Cohort 1 Patient, and interim clinical results for the first Cohort 2 Patient - BB-301 is the only clinical-stage therapeutic agent in development for the treatment of dysphagia in patients diagnosed with OPMD HAYWARD, Calif., April 28, 2026 (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 “Silence and Replace” DNA-directed RNA interference ("ddRNAi") platform, today announced the acceptance of the interim clinical results from the ongoing BB-301 Phase 1b/2a first-in-human study for Oculopharyngeal Muscular Dystrophy (OPMD) with moderate dysphagia for oral presentation at the 2026 American Society of Gene and Cell Therapy Annual Meeting (ASGCT). Interim clinical results for patients enrolled into Cohort 1 and Cohort 2 will be discussed in an oral presentation at ASGCT, being held in Boston, MA from May 11 – 15, 2026. Details regarding the BB-301 Oral Presentation are as follows: Session Name: Long-term safety and durable efficacy in cell and gene therapy Presentation Date and Time: 05/15/2026, 09:15 AM - 09:30 AM Location: MCEC Room 257AB (Level 2) Abstract ID: 448 Presenter: Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer, Benitec Biopharma About OPMD There are currently no approved therapies for OPMD, a rare autosomal-dominant degenerative muscle disorder, that impacts nearly 15,000 patients in North America, Europe and Israel. OPMD is caused by a mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene; PABPN1 is a ubiquitous protein that controls the length of mRNA poly(A) tails, mRNA export from the nucleus and alternative poly(A) site usage. OPMD is a debilitating progressive disease that weakens the pharyngeal muscles, causing severe swallowing difficulties (dysphagia).1 Progressive dysphagia impacts 97% of OPMD patients and is a severe, life-threatening complication of OPMD which can lead to chronic choking, malnutrition, aspiration pneumonia and death. About BB-301 BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct prom…Read full documentShow less
- Interim clinical results for the BB-301 Phase 1b/2a study include 12-month post-treatment follow-up results for the first four Cohort 1 completers, 24-month post-treatment follow-up results for the first Cohort 1 Patient, and interim clinical results for the first Cohort 2 Patient - BB-301 is the only clinical-stage therapeutic agent in development for the treatment of dysphagia in patients diagnosed with OPMD HAYWARD, Calif., April 28, 2026 (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 “Silence and Replace” DNA-directed RNA interference ("ddRNAi") platform, today announced the acceptance of the interim clinical results from the ongoing BB-301 Phase 1b/2a first-in-human study for Oculopharyngeal Muscular Dystrophy (OPMD) with moderate dysphagia for oral presentation at the 2026 American Society of Gene and Cell Therapy Annual Meeting (ASGCT). Interim clinical results for patients enrolled into Cohort 1 and Cohort 2 will be discussed in an oral presentation at ASGCT, being held in Boston, MA from May 11 – 15, 2026. Details regarding the BB-301 Oral Presentation are as follows: Session Name: Long-term safety and durable efficacy in cell and gene therapy Presentation Date and Time: 05/15/2026, 09:15 AM - 09:30 AM Location: MCEC Room 257AB (Level 2) Abstract ID: 448 Presenter: Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer, Benitec Biopharma About OPMD There are currently no approved therapies for OPMD, a rare autosomal-dominant degenerative muscle disorder, that impacts nearly 15,000 patients in North America, Europe and Israel. OPMD is caused by a mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene; PABPN1 is a ubiquitous protein that controls the length of mRNA poly(A) tails, mRNA export from the nucleus and alternative poly(A) site usage. OPMD is a debilitating progressive disease that weakens the pharyngeal muscles, causing severe swallowing difficulties (dysphagia).1 Progressive dysphagia impacts 97% of OPMD patients and is a severe, life-threatening complication of OPMD which can lead to chronic choking, malnutrition, aspiration pneumonia and death. About BB-301 BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against mutant PABPN1 (the causative gene for OPMD). The two siRNAs are modeled into microRNA backbones to silence expression of faulty mutant PABPN1, while allowing expression of the codon-optimized PABPN1 to replace the mutant with a functional version of the protein. We believe the silence and replace mechanism of BB-301 is uniquely positioned for the treatment of OPMD by halting mutant PABPN1 expression while providing a functional replacement protein. BB-301 has received Orphan Drug Designation from the EMA and Orphan Drug and Fast Track Designations from the FDA. About Benitec Biopharma Inc. Benitec Biopharma Inc. (“Benitec” or the “Company”) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary “Silence and Replace” DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview of the Company can be found on Benitec’s website at www.benitec.com. Forward Looking Statements Except for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec’s plans to develop and commercialize its product candidates, the timing of the completion of pre-clinical and clinical trials, the timing of the availability of data from our clinical trials, the timing and sufficiency of patient enrollment and dosing in clinical trials, the timing of expected regulatory filings and other regulatory steps, and the clinical utility and potential attributes and benefits of ddRNAi and Benitec’s product candidates, and other forward-looking statements. These forward-looking statements are based on the Company’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: the success of our plans to develop and potentially commercialize our product candidates; the timing of the completion of preclinical studies and clinical trials; the timing and sufficiency of patient enrollment and dosing in any future clinical trials; the timing of the availability of data from our clinical trials; the timing and outcome of regulatory filings and approvals; the development of novel AAV vectors; our potential future out-licenses and collaborations; the plans of licensees of our technology; the clinical utility and potential attributes and benefits of ddRNAi and our product candidates, including the potential duration of treatment effects and the potential for a “one shot” cure; our intellectual property position and the duration of our patent portfolio; expenses, ongoing losses, future revenue, capital needs and needs for additional financing, and our ability to access additional financing given market conditions and other factors; the length of time over which we expect our cash and cash equivalents to be sufficient to execute on our business plan; unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities and other regulatory developments; the Company’s ability to protect and enforce its patents and other intellectual property rights; the Company’s dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Company’s products and the products of the Company’s collaboration partners; the acceptance of the Company’s products and the products of the Company’s collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the impact of, and our ability to remediate, the identified material weakness in our internal controls over financial reporting; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company’s reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements. References: Investor Relations Contact: Candice Masse Astr Partners (978) 879-7273 [email protected] Media Contact: Audra Friis Sam Brown Healthcare Communications (917) 519-9577 [email protected]
Investor releaseQuarter not tagged2026-03-09Benitec Biopharma Announces Positive Interim Phase 1b/2a Results for High Dose BB-301 and Continued Durable Improvements for Low Dose BB-301 Treatment at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference
GlobeNewswire
Benitec Biopharma Announces Positive Interim Phase 1b/2a Results for High Dose BB-301 and Continued Durable Improvements for Low Dose BB-301 Treatment at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference
- Oculopharyngeal Muscular Dystrophy (OPMD) Patients treated with low dose BB-301 and high dose BB-301 experienced significant improvements in throat closure, throat emptying, and total dysphagic symptom burden - OPMD Patients treated with low dose BB-301 experienced highly durable improvements, with clinical and radiographic improvements continuing to deepen two years post BB-301 treatment -The first OPMD Patient treated with high dose BB-301 experienced an extraordinarily robust dose-response at an early interim follow-up time-point, indicating the continued potential for BB-301 to achieve disease-modifying outcomes for OPMD patients with dysphagia - BB-301 is the only clinical-stage therapeutic in development designed to treat dysphagia in patients with OPMD HAYWARD, Calif., March 09, 2026 (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 “Silence and Replace” DNA-directed RNA interference ("ddRNAi") platform, today announced promising interim clinical results from the BB-301 Phase 1b/2a first-in-human study (NCT06185673) evaluating low dose and high dose BB-301 treatment for Oculopharyngeal Muscular Dystrophy (OPMD) with moderate dysphagia. Interim and long-term clinical results for patients enrolled into Cohort 1 (low dose BB-301 ), and interim clinical results for the first patient enrolled into Cohort 2 (high dose BB-301) in the ongoing clinical trial will be presented as a late-breaking poster presentation at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, in Orlando, Florida on March 9, 2026. “We are strongly encouraged by the 100% response rate and the depth and durability of the responses that have been observed for all patients treated with BB-301 to date,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “We are incredibly excited to share these interim clinical results which demonstrate positive, clinically meaningful improvements across the most critical radiographic, functional, and patient-reported assessments of swallowing function. With no currently approved treatments for OPMD patients, the results presented today represent an important step towards the management of the unmet medical need that exists in the OPMD…Read full documentShow less
- Oculopharyngeal Muscular Dystrophy (OPMD) Patients treated with low dose BB-301 and high dose BB-301 experienced significant improvements in throat closure, throat emptying, and total dysphagic symptom burden - OPMD Patients treated with low dose BB-301 experienced highly durable improvements, with clinical and radiographic improvements continuing to deepen two years post BB-301 treatment -The first OPMD Patient treated with high dose BB-301 experienced an extraordinarily robust dose-response at an early interim follow-up time-point, indicating the continued potential for BB-301 to achieve disease-modifying outcomes for OPMD patients with dysphagia - BB-301 is the only clinical-stage therapeutic in development designed to treat dysphagia in patients with OPMD HAYWARD, Calif., March 09, 2026 (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 “Silence and Replace” DNA-directed RNA interference ("ddRNAi") platform, today announced promising interim clinical results from the BB-301 Phase 1b/2a first-in-human study (NCT06185673) evaluating low dose and high dose BB-301 treatment for Oculopharyngeal Muscular Dystrophy (OPMD) with moderate dysphagia. Interim and long-term clinical results for patients enrolled into Cohort 1 (low dose BB-301 ), and interim clinical results for the first patient enrolled into Cohort 2 (high dose BB-301) in the ongoing clinical trial will be presented as a late-breaking poster presentation at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, in Orlando, Florida on March 9, 2026. “We are strongly encouraged by the 100% response rate and the depth and durability of the responses that have been observed for all patients treated with BB-301 to date,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “We are incredibly excited to share these interim clinical results which demonstrate positive, clinically meaningful improvements across the most critical radiographic, functional, and patient-reported assessments of swallowing function. With no currently approved treatments for OPMD patients, the results presented today represent an important step towards the management of the unmet medical need that exists in the OPMD community. We remain committed to advancing the BB-301 program, and we are deeply grateful to the patients, their families, and our investigators whose unyielding commitment continues to make this progress possible.” BB-301 Phase 1b/2a Clinical Treatment Study Background: The BB-301 Phase 1b/2a clinical study (NCT06185673) is an open-label, dose escalation study evaluating the safety and clinical activity of intramuscular doses of BB-301 to treat moderate dysphagia in patients with OPMD. The following parameters represent the core assessments of BB-301 efficacy for each study participant: Sydney Swallow Questionnaire (SSQ), pharyngeal area at maximum constriction (PhAMPC), total pharyngeal residue (TPR) and normalized residue ratio scale-valleculae (NRRSv). The SSQ is a validated 17-item patient-reported outcome instrument that assesses the total dysphagic symptom burden experienced by a patient. The PhAMPC is assessed by videofluoroscopic swallowing studies (VFSS) and serves as a surrogate for the functional capacity of the pharyngeal constrictor muscles during the swallowing cycle. The TPR is assessed by VFSS and represents the quantity of food and liquid material (residue) remaining in the throat upon completion of a swallow (post-swallow residue). The NRRSv is assessed by VFSS and represents the quantity of food and liquid material (residue) remaining in the vallecular region of the throat upon completion of a swallow (post-swallow residue). Key interim clinical study results to be presented at the 2026 MDA Clinical & Scientific Conference include: Interim Clinical Results for High Dose BB-301 (Cohort 2): High dose BB-301 is being evaluated for the potential to facilitate more rapid clinical improvements and/or greater magnitudes of clinical improvements across the core functional, anatomical, and symptom-focused elements of the dysphagic symptom burden experienced by OPMD patients Patient B (Cohort 2, high dose BB-301) safely received the high dose of BB-301 with no treatment-related SAEs Patient B (Cohort 2, high dose BB-301) and Patient A (Cohort 1, low dose BB-301) had comparable baseline functional, anatomical, and symptom-focused deficits prior to the administration of BB-301 When comparing 3-month post-BB-301-treatment clinical results for Patient A and Patient B, Patient B experienced a significant improvement in depth of response to high dose BB-301 Patient B, the first OPMD Patient treated with high dose BB-301, experienced an extraordinarily robust dose-response at an early interim follow-up time-point, indicating the continued potential for BB-301 to achieve disease-modifying outcomes for OPMD patients with dysphagia. When comparing the interim clinical results for the low dose BB-301 treatment and the high dose BB-301 treatment at the 3-month post-treatment time-point in Patients with comparable pre-treatment baseline deficits, the high dose BB-301 treatment demonstrated significantly improved results across all radiographic and patient-reported assessments employed in the BB-301 Phase 1b/2a Clinical Treatment Study: Significantly differentiated levels of dysphagic symptom burden reduction were observed, with a ~7% reduction in total dysphagic symptom burden (SSQ) achieved with low dose BB-301 as compared to a ~68% reduction in SSQ achieved with high dose BB-301 Significantly differentiated levels of throat closure were observed, with an ~8% improvement in throat closure (PhAMPC) achieved with low dose BB-301 as compared to a ~19% improvement in PhAMPC achieved with high dose BB-301 Significantly differentiated levels of overall throat emptying were observed, with a ~6% worsening in overall throat emptying (TPR) observed with low dose BB-301 as compared to a ~44% improvement in TPR achieved with high dose BB-301 Significantly differentiated levels of throat emptying from the vallecular region were observed, with a ~3% improvement in throat emptying from the vallecular region (NRRSv) achieved with low dose BB-301 as compared to a ~57% improvement in NRRSv with high dose BB-301 Interim Clinical Results for Low Dose BB-301 (Cohort 1): All Study Completers in Cohort 1 (low dose BB-301) are formal Responders to BB-301 Completers are Patients that have reached the 12-month post-BB-301-treatment assessment time-point in the BB-301 Phase 1b/2a Clinical Treatment Study Long-term efficacy trends for low dose BB-301 at 24-months post BB-301 treatment continue to demonstrate robust disease-modifying outcomes for throat closure, throat emptying, and total dysphagic symptom burden Late-Breaking Poster Presentation An interim clinical study update for the Phase 1b/2a Clinical Treatment Study of BB-301 in OPMD subjects with moderate dysphagia will be provided in a late-breaking poster presentation, (poster number 501 LB) entitled “Durable Responses to Low Dose BB-301 in Oculopharyngeal Muscular Dystrophy at 12- and 24-months and Improved Depth of Response to High Dose BB-301” during poster sessions from 10:15-10:45 am, 12:00-1:30 pm, 3:30-4:00 pm and 6:00-8:00 pm Eastern Time on March 9th in the Exhibit Hall at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference. The poster is available on the Benitec website, and a link to the poster is found here. About OPMD There are currently no approved therapies for OPMD, a rare autosomal-dominant degenerative muscle disorder, that impacts nearly 15,000 patients in North America, Europe and Isreal. OPMD is caused by a mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene; PABPN1 is a ubiquitous protein that controls the length of mRNA poly(A) tails, mRNA export from the nucleus and alternative poly(A) site usage. OPMD is a debilitating progressive disease that weakens the pharyngeal muscles, causing severe swallowing difficulties (dysphagia).1 Progressive dysphagia impacts 97% of OPMD patients and is a severe, life-threatening complication of OPMD which can lead to chronic choking, malnutrition, aspiration pneumonia and death. About BB-301 BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against mutant PABPN1 (the causative gene for OPMD). The two siRNAs are modeled into microRNA backbones to silence expression of faulty mutant PABPN1, while allowing expression of the codon-optimized PABPN1 to replace the mutant with a functional version of the protein. We believe the silence and replace mechanism of BB-301 is uniquely positioned for the treatment of OPMD by halting mutant PABPN1 expression while providing a functional replacement protein. BB-301 has received Orphan Drug Designation from the EMA and Orphan Drug and Fast Track Designations from the FDA. About Benitec Biopharma Inc. Benitec Biopharma Inc. (“Benitec” or the “Company”) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary “Silence and Replace” DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview of the Company can be found on Benitec’s website at www.benitec.com. Forward Looking Statements Except for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec’s plans to develop and commercialize its product candidates, the timing of the completion of pre-clinical and clinical trials, the timing of the availability of data from our clinical trials, the timing and sufficiency of patient enrollment and dosing in clinical trials, the timing of expected regulatory filings and other regulatory steps, and the clinical utility and potential attributes and benefits of ddRNAi and Benitec’s product candidates, and other forward-looking statements. These forward-looking statements are based on the Company’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: the success of our plans to develop and potentially commercialize our product candidates; the timing of the completion of preclinical studies and clinical trials; the timing and sufficiency of patient enrollment and dosing in any future clinical trials; the timing of the availability of data from our clinical trials; the timing and outcome of regulatory filings and approvals; the development of novel AAV vectors; our potential future out-licenses and collaborations; the plans of licensees of our technology; the clinical utility and potential attributes and benefits of ddRNAi and our product candidates, including the potential duration of treatment effects and the potential for a “one shot” cure; our intellectual property position and the duration of our patent portfolio; expenses, ongoing losses, future revenue, capital needs and needs for additional financing, and our ability to access additional financing given market conditions and other factors; the length of time over which we expect our cash and cash equivalents to be sufficient to execute on our business plan; unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities and other regulatory developments; the Company’s ability to protect and enforce its patents and other intellectual property rights; the Company’s dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Company’s products and the products of the Company’s collaboration partners; the acceptance of the Company’s products and the products of the Company’s collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the impact of, and our ability to remediate, the identified material weakness in our internal controls over financial reporting; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company’s reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements. References: Media Contact: Audra Friis Sam Brown Healthcare Communications (917) 519-9577 [email protected] Investor Relations Contact: Irina Koffler LifeSci Advisors, LLC (917) 734-7387 [email protected] A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/898abdaf-6d60-4063-8b30-b75497df9259
Investor releaseQuarter not tagged2026-02-12Benitec Biopharma Releases Second Quarter 2026 Financial Results and Provides Operational Update
GlobeNewswire
Benitec Biopharma Releases Second Quarter 2026 Financial Results and Provides Operational Update
-The first 4 Patients enrolled into Cohort 1 of the BB-301 Phase 1b/2a treatment study have completed the 12-month statistical follow-up period, and all 4 Completers were formal Responders to BB-301 at the 12-month follow-up timepoint demonstrating durable response to BB-301- -Patient 1 of Cohort 1 completed the 24-month follow-up timepoint, and at the 24-month post-treatment timepoint Patient 1 continued to experience the disease-modifying effects of BB-301, with deepening improvements in post-swallow residue and total dysphagic symptom burden as compared to the 12-month follow-up timepoint- - An update on the Interim clinical results for Cohort 2 is planned for mid-2026- -FDA meeting to formalize the pivotal BB-301 study design expected mid-year- HAYWARD, Calif.,, Feb. 12, 2026 (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 financial results for its second fiscal quarter ended December 31, 2025. The Company has filed its quarterly report on Form 10-Q with the U.S. Securities and Exchange Commission. “We continue to be encouraged by the benign safety profile and the durability of efficacy demonstrated in our BB-301 clinical development program,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “We look forward to engaging with the U.S. Food and Drug Administration (FDA) in mid-2026 to confirm the BB-301 pivotal study design and continuing to present interim clinical results at future medical conferences. I want to sincerely thank our investigators, our clinical advisors, and—most importantly—the patients and families who have made this progress possible.” The recent and upcoming key milestones related to the development of BB-301 for the treatment of Oculopharyngeal Muscular Dystrophy-related Dysphagia, are outlined below: Responder Analysis for Study Completers A Responder Analysis was developed to facilitate standardized evaluation of BB-301 efficacy for each Patient. The Responder Analysis consists of multiple discrete response categories that collectively assess the dysphagic symptom burden in patients with OPMD. These response categories include: Patient-Reporte…Read full documentShow less
-The first 4 Patients enrolled into Cohort 1 of the BB-301 Phase 1b/2a treatment study have completed the 12-month statistical follow-up period, and all 4 Completers were formal Responders to BB-301 at the 12-month follow-up timepoint demonstrating durable response to BB-301- -Patient 1 of Cohort 1 completed the 24-month follow-up timepoint, and at the 24-month post-treatment timepoint Patient 1 continued to experience the disease-modifying effects of BB-301, with deepening improvements in post-swallow residue and total dysphagic symptom burden as compared to the 12-month follow-up timepoint- - An update on the Interim clinical results for Cohort 2 is planned for mid-2026- -FDA meeting to formalize the pivotal BB-301 study design expected mid-year- HAYWARD, Calif.,, Feb. 12, 2026 (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 financial results for its second fiscal quarter ended December 31, 2025. The Company has filed its quarterly report on Form 10-Q with the U.S. Securities and Exchange Commission. “We continue to be encouraged by the benign safety profile and the durability of efficacy demonstrated in our BB-301 clinical development program,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “We look forward to engaging with the U.S. Food and Drug Administration (FDA) in mid-2026 to confirm the BB-301 pivotal study design and continuing to present interim clinical results at future medical conferences. I want to sincerely thank our investigators, our clinical advisors, and—most importantly—the patients and families who have made this progress possible.” The recent and upcoming key milestones related to the development of BB-301 for the treatment of Oculopharyngeal Muscular Dystrophy-related Dysphagia, are outlined below: Responder Analysis for Study Completers A Responder Analysis was developed to facilitate standardized evaluation of BB-301 efficacy for each Patient. The Responder Analysis consists of multiple discrete response categories that collectively assess the dysphagic symptom burden in patients with OPMD. These response categories include: Patient-Reported Outcome: Patient-reported oral-pharyngeal dysphagia as assessed by the Sydney Swallow Questionnaire (SSQ) total score Videofluoroscopic Swallowing Study (VFSS) Assessments: Pharyngeal constrictor muscle function as estimated by the Pharyngeal Area at Maximum Constriction (PhAMPC) Swallowing efficiency as measured by NRRSv and Total Pharyngeal Residue %(C2-4)2 Frequency of pathologic sequential swallows (SEQ) Functional Swallowing Capacity: Cold-Water Timed Drinking Test (CWDT) The evaluation is completed as follows: Following completion of the 12-month post-treatment follow-up timepoint, each discrete response category is evaluated for each study Completer using prespecified statistical criteria Results of the statistical characterization of each response category are combined into a single scoring framework that facilitates the overall assessment of clinical benefit achieved by each Completer following treatment with BB-301 A total Score of 5 is possible Responder status for each Completer will be assigned based on the achievement of statistical criteria for at least 2 out of 5 discrete response categories (≥40%) Responder Analysis for Study Completers: All four Cohort 1 Completers were formal responders to BB-301, demonstrating durable response to BB-301 at the conclusion of the 12-month statistical follow-up period. 24-Month Post-Treatment Follow-Up for Patient 1 of Cohort 1 At the 24-month post-BB-301 treatment follow-up timepoint, Patient 1 of Cohort 1 continued to demonstrate robust, disease-modifying outcomes. Patient 1 demonstrated deepening improvements in post-swallow pharyngeal residue as compared to the final pre-treatment timepoint and as compared to the 12-month post-treatment follow-up timepoint as assessed by VFSS. Patient 1 also experienced deepening improvements in total dysphagic symptom burden as assessed by the SSQ. Enrollment into the BB-301 Phase 1b/2a Clinical Treatment Study is Ongoing: The first Patient in Cohort 2 was safely treated with the higher-dose of BB-301 in 4Q of 2025 and an update on the interim clinical results of Cohort 2 is planned for mid-2026. Corporate Updates: The Company plans to engage with the FDA in mid-2026 to confirm the BB-301 pivotal study design. In November 2025, Fast Track Designation was granted for BB-301 following FDA review of positive interim clinical study results and the proprietary Responder Analysis planned for use in the BB-301 pivotal study. Previously, BB-301 has received Orphan Drug Designation from the EMA and the FDA. Financial Highlights Second Quarter 2026 Financial Results Total Expenses for the quarter ended December 31, 2025 were $13.4 million compared to $10.8 million for the quarter ended December 31, 2024. The Company incurred $5.8 million of research and development expenses which was in line with $5.4 million for the comparable quarter ended December 31, 2024. Research and development expenses relate primarily to ongoing clinical development of BB-301 for the treatment of OPMD. General and administrative expenses were $7.5 million compared to $5.4 million for the quarter ended December 31, 2024. The loss from operations for the quarter ended December 31, 2025, was $13.4 million compared to a loss of $10.8 million for the quarter ended December 31, 2024. Net loss attributable to shareholders for the quarter ended December 31, 2025, was $11.8 million, or $(0.26) per basic and diluted share, compared to a net loss of $9.6 million, or $(0.26) per basic and diluted share for the quarter ended December 31, 2024. As of December 31, 2025, the Company had $189 million in cash and cash equivalents, which includes $0.1 million from the exercise of warrants during the six-month period ended December 31, 2025. About BB-301 BB-301 is a silence and replace-based genetic medicine currently under development by Benitec. BB-301 uses DNA-directed RNA interference (ddRNAi) to simultaneously silence the mutant gene and replace it with a functional gene, potentially providing a permanent solution with a single administration. This fundamental therapeutic approach to disease management is called “silence and replace.” The silence and replace mechanism offers the potential to restore the normative physiology of diseased cells and tissues and to improve treatment outcomes for patients suffering from the chronic, and potentially fatal, effects of OPMD. BB-301 has been granted Orphan Drug Designation from the EMA and Orphan Drug and Fast Track Designations from the FDA. About Benitec Biopharma, Inc. Benitec Biopharma Inc. (“Benitec” or the “Company”) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary “Silence and Replace” DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview of the Company can be found on Benitec’s website at www.benitec.com. Forward Looking Statements Except for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec’s plans to develop and commercialize its product candidates, the timing of the completion of pre-clinical and clinical trials, the timing of the availability of data from our clinical trials, the timing and sufficiency of patient enrollment and dosing in clinical trials, the timing of expected regulatory filings and other regulatory steps, and the clinical utility and potential attributes and benefits of ddRNAi and Benitec’s product candidates, and other forward-looking statements. These forward-looking statements are based on the Company’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: the success of our plans to develop and potentially commercialize our product candidates; the timing of the completion of preclinical studies and clinical trials; the timing and sufficiency of patient enrollment and dosing in any future clinical trials; the timing of the availability of data from our clinical trials; the timing and outcome of regulatory filings and approvals; the development of novel AAV vectors; our potential future out-licenses and collaborations; the plans of licensees of our technology; the clinical utility and potential attributes and benefits of ddRNAi and our product candidates, including the potential duration of treatment effects and the potential for a “one shot” cure; our intellectual property position and the duration of our patent portfolio; expenses, ongoing losses, future revenue, capital needs and needs for additional financing, and our ability to access additional financing given market conditions and other factors; the length of time over which we expect our cash and cash equivalents to be sufficient to execute on our business plan; unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities and other regulatory developments; the Company’s ability to protect and enforce its patents and other intellectual property rights; the Company’s dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Company’s products and the products of the Company’s collaboration partners; the acceptance of the Company’s products and the products of the Company’s collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the impact of, and our ability to remediate, the identified material weakness in our internal controls over financial reporting; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company’s reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements. Investor Relations Contact: Irina Koffler LifeSci Advisors, LLC (917) 734-7387 [email protected] A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/8aeb15e8-1b57-4093-98b6-8d84522b188c
Investor releaseQuarter not tagged2026-01-11Benitec Biopharma Provides Positive Long-Term Clinical Study Results for BB-301 Phase 1b/2a Clinical Trial Demonstrating Robust Efficacy and Continued Durability of Response
GlobeNewswire
Benitec Biopharma Provides Positive Long-Term Clinical Study Results for BB-301 Phase 1b/2a Clinical Trial Demonstrating Robust Efficacy and Continued Durability of Response
• Patient 1 of Cohort 1 has now completed the 24-month follow-up timepoint, and at month-24 post-treatment Patient 1 continued to demonstrate the powerful disease-modifying effects of BB-301, with deepening improvements in post-swallow residue and total dysphagic symptom burden as compared to the 12-month follow-up timepoint • Patient 4 of Cohort 1 continued to experience strong response to BB-301 at the 12-month follow-up timepoint • The first 4 patients enrolled into Cohort 1 have completed the 12-month statistical follow-up period, and all 4 Completers were formal Responders to BB-301 at the month-12 follow-up timepoint demonstrating durable response to BB-301 HAYWARD, Calif., Jan. 11, 2026 (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 “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today announced that the first patient treated in Cohort 1 of the BB-301 Phase 1b/2a clinical study (NCT06185673) evaluating BB-301 for the treatment of dysphagia in oculopharyngeal muscular dystrophy (OPMD) has completed the 24-month post-treatment assessment. At the 24-month follow-up timepoint, Patient 1 continued to demonstrate robust, disease-modifying outcomes. At the 24-month follow-up timepoint, Patient 1 demonstrated deepening improvements in post-swallow pharyngeal residue as compared to the final pre-treatment timepoint and the 12-month post-treatment follow-up timepoint as assessed by x-ray-based swallowing studies. Additionally, Patient 1 experienced deepening improvements in total dysphagic symptom burden as assessed by the Sydney Swallow Questionnaire (SSQ). The first 4 patients in Cohort 1 have now completed the 12-month statistical follow-up period for the Phase 1b/2a study, and all 4 Completers continued to demonstrate durable response to BB-301. All 4 Cohort 1 Completers met the pre-specified statistical criteria for response to BB-301 defined by Benitec which require improvement across 2 or more of the 5 categories of assessment comprising the Responder Analysis1. “Progressive dysphagia is the most severe, life-threatening complication of OPMD, and we are extremely excited to observe safe, durable, disease-modifying outcomes for the patients treated with BB-301,” said Jerel A…Read full documentShow less
• Patient 1 of Cohort 1 has now completed the 24-month follow-up timepoint, and at month-24 post-treatment Patient 1 continued to demonstrate the powerful disease-modifying effects of BB-301, with deepening improvements in post-swallow residue and total dysphagic symptom burden as compared to the 12-month follow-up timepoint • Patient 4 of Cohort 1 continued to experience strong response to BB-301 at the 12-month follow-up timepoint • The first 4 patients enrolled into Cohort 1 have completed the 12-month statistical follow-up period, and all 4 Completers were formal Responders to BB-301 at the month-12 follow-up timepoint demonstrating durable response to BB-301 HAYWARD, Calif., Jan. 11, 2026 (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 “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today announced that the first patient treated in Cohort 1 of the BB-301 Phase 1b/2a clinical study (NCT06185673) evaluating BB-301 for the treatment of dysphagia in oculopharyngeal muscular dystrophy (OPMD) has completed the 24-month post-treatment assessment. At the 24-month follow-up timepoint, Patient 1 continued to demonstrate robust, disease-modifying outcomes. At the 24-month follow-up timepoint, Patient 1 demonstrated deepening improvements in post-swallow pharyngeal residue as compared to the final pre-treatment timepoint and the 12-month post-treatment follow-up timepoint as assessed by x-ray-based swallowing studies. Additionally, Patient 1 experienced deepening improvements in total dysphagic symptom burden as assessed by the Sydney Swallow Questionnaire (SSQ). The first 4 patients in Cohort 1 have now completed the 12-month statistical follow-up period for the Phase 1b/2a study, and all 4 Completers continued to demonstrate durable response to BB-301. All 4 Cohort 1 Completers met the pre-specified statistical criteria for response to BB-301 defined by Benitec which require improvement across 2 or more of the 5 categories of assessment comprising the Responder Analysis1. “Progressive dysphagia is the most severe, life-threatening complication of OPMD, and we are extremely excited to observe safe, durable, disease-modifying outcomes for the patients treated with BB-301,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec Biopharma Inc. “Durable improvements in the dysphagic symptom burden can have a profound impact on the lives of patients living with OPMD, and we remain singularly focused on advancing BB-301 through development to improve the lives of all OPMD patients. We look forward to engaging with the U.S. Food and Drug Administration (FDA) in mid-2026 to confirm the BB-301 pivotal study design and continuing to present interim clinical results at future medical conferences. We extend our deepest gratitude to the patients and families participating in the clinical study and to the investigators and clinical teams for their dedication to advancing new treatment options.” Updated Interim Clinical Study Results 24-Month Post-Treatment Follow-Up for Patient 1 of Cohort 1 At the 24-month post-BB-301 treatment follow-up timepoint, Patient 1 of Cohort 1 continued to demonstrate robust, disease-modifying outcomes. At the 24-month follow-up timepoint, Patient 1 demonstrated deepening improvements in post-swallow pharyngeal residue as compared to the final pre-treatment timepoint and as compared to the 12-month post-treatment follow-up timepoint as assessed by videofluoroscopic swallowing studies (VFSS). Patient 1 also experienced deepening improvements in total dysphagic symptom burden as assessed by the Sydney Swallow Questionnaire (SSQ). Pharyngeal Area at Maximum Constriction (“PhAMPC”), as assessed by VFSS, represents the functional capacity of the pharyngeal constrictor muscles during the swallowing cycle At the 24-month post-treatment timepoint, Patient 1 demonstrated durable functional improvement of pharyngeal constrictor-mediated throat closure, as the 12-month post-treatment improvements were perfectly maintained at the 24-month post-treatment timepoint As compared to the final pre-treatment visit, Patient 1 demonstrated a 27% improvement in PhAMPC (throat closure) at the 12-month post-treatment timepoint (27% improvement in throat closure at the peak of swallowing as compared to the final pre-treatment assessment), and the 27% improvement in PhAMPC was maintained at the 24-month post treatment timepoint (27% improvement in throat closure at the peak of swallowing as compared to the final pre-treatment assessment), indicating durable improvement in pharyngeal muscle function during swallowing Normalized Residue Ratio Scale-Valleculae (NRRSv), as assessed by VFSS, represents the quantity of food and liquid material (residue) remaining in the vallecular region of the throat upon completion of a swallow (post-swallow residue) Elevated levels of post-swallow residue in the vallecular region of the throat have been shown to correlate with increased risk of aspiration events At the 24-month post-treatment timepoint, the throat-emptying ability of Patient 1 continued to significantly improve for liquids and solid food As compared to the final pre-treatment visit, Patient 1 demonstrated a 35% improvement in NRRSv at the 12-month post-treatment timepoint (35% reduction in post-swallow residue in the vallecular region as compared to the final pre-treatment assessment), and at the 24-month post-treatment timepoint Patient 1 demonstrated a 60% improvement in NRRSv (60% reduction in post-swallow residue in the vallecular region as compared to the final pre-treatment assessment) exemplifying a deepening of the improvement in swallowing efficiency over time following the administration of BB-301 Total Pharyngeal Residue (TPR), as assessed by VFSS, represents the quantity of food and liquid material (residue) remaining in the throat upon completion of a swallow (post-swallow residue) At the 24-month post-treatment timepoint, the throat-emptying ability of Patient 1 continued to significantly improve for liquids and solid food As compared to the final pre-treatment visit, Patient 1 demonstrated a 32% improvement in TPR at the 12-month post-treatment timepoint (32% reduction in post-swallow residue as compared to the final pre-treatment assessment), and at the 24-month post-treatment timepoint Patient 1 demonstrated a 39% improvement in TPR (39% reduction in post-swallow residue as compared to the final pre-treatment assessment) exemplifying a deepening of the improvement in swallowing efficiency over time following the administration of BB-301 Sydney Swallow Questionnaire, a validated 17-item patient-reported outcome instrument, represents the total dysphagic symptom burden experienced by a patient At the 24-month post-treatment timepoint, the total dysphagic symptom burden continued to significantly decline As compared to the final pre-treatment visit, Patient 1 experienced a 64% improvement in SSQ total score at the 12-month post-treatment timepoint (64% reduction in total dysphagic symptom burden as compared to the final pre-treatment assessment), and at the 24-month post-treatment timepoint Patient 1 experienced a 78% improvement in SSQ total score (78% reduction in total dysphagic symptom burden as compared to the final pre-treatment assessment) exemplifying a deepening of the improvement in total dysphagic symptom burden over time following the administration of BB-301 Analysis of Study Completers (12-Month Post-Treatment Follow-Up) A Responder Analysis was developed to facilitate standardized evaluation of BB-301 efficacy for each patient. The Responder Analysis consists of multiple discrete response categories that collectively assess the dysphagic symptom burden in patients with OPMD. These response categories include: Patient-Reported Outcome: Patient-reported oropharyngeal dysphagia as assessed by the Sydney Swallow Questionnaire (SSQ) total score Videofluoroscopic Swallowing Study (VFSS) Assessments: Pharyngeal constrictor muscle function as estimated by the Pharyngeal Area at Maximum Constriction (PhAMPC) Swallowing efficiency as measured by NRRSv and Total Pharyngeal Residue %(C2-4)2 Frequency of pathologic sequential swallows (SEQ) Functional Swallowing Capacity: Cold-Water Timed Drinking Test (CWDT) Following completion of the 12-month post-treatment follow-up timepoint, each discrete response category is evaluated for each study Completer using prespecified statistical criteria Results of the statistical characterization of each response category are combined into a single scoring framework that facilitates the overall assessment of clinical benefit achieved by each patient following treatment with BB-301 A total Score of 5 is possible Responder status for each patient will be assigned based on the achievement of statistical criteria for at least 2 out of 5 discrete response categories (≥40%) Responder Analysis for Study Completers: Patients 1-to-4 of Cohort 1 All 4 Cohort 1 Completers were formal Responders to BB-301, demonstrating durable response to BB-301 at the conclusion of the 12-month statistical follow-up period. About BB-301 BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against mutant PABPN1 (the causative gene for OPMD). The two siRNAs are modeled into microRNA backbones to silence expression of faulty mutant PABPN1, while allowing expression of the codon-optimized PABPN1 to replace the mutant with a functional version of the protein. We believe the silence and replace mechanism of BB-301 is uniquely positioned for the treatment of OPMD by halting mutant expression while providing a functional replacement protein. BB-301 has received Orphan Drug Designation from the EMA and Orphan Drug and Fast Track Designations from the FDA. About Benitec Biopharma, Inc. Benitec Biopharma Inc. (“Benitec” or the “Company”) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary “Silence and Replace” DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview of the Company can be found on Benitec’s website at www.benitec.com. Forward Looking Statements Except for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec’s plans to develop and commercialize its product candidates, the timing of the completion of pre-clinical and clinical trials, the timing of the availability of data from our clinical trials, the timing and sufficiency of patient enrollment and dosing in clinical trials, the timing of expected regulatory filings, and the clinical utility and potential attributes and benefits of ddRNAi and Benitec’s product candidates, and other forward-looking statements. These forward-looking statements are based on the Company’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: the success of our plans to develop and potentially commercialize our product candidates; the timing of the completion of preclinical studies and clinical trials; the timing and sufficiency of patient enrollment and dosing in any future clinical trials; the timing of the availability of data from our clinical trials; the timing and outcome of regulatory filings and approvals; the development of novel AAV vectors; our potential future out-licenses and collaborations; the plans of licensees of our technology; the clinical utility and potential attributes and benefits of ddRNAi and our product candidates, including the potential duration of treatment effects and the potential for a “one shot” cure; our intellectual property position and the duration of our patent portfolio; expenses, ongoing losses, future revenue, capital needs and needs for additional financing, and our ability to access additional financing given market conditions and other factors; the length of time over which we expect our cash and cash equivalents to be sufficient to execute on our business plan; unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities and other regulatory developments; the Company’s ability to protect and enforce its patents and other intellectual property rights; the Company’s dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Company’s products and the products of the Company’s collaboration partners; the acceptance of the Company’s products and the products of the Company’s collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the impact of, and our ability to remediate, the identified material weakness in our internal controls over financial reporting; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company’s reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements. Investor Relations Contact: Irina Koffler LifeSci Advisors, LLC (917) 734-7387 [email protected] 1 (Final+Webcast+Slides)BENITEC,+2025NOV01.pdf A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/f653cced-806c-4f5a-a3ea-991da27438a7
Investor releaseQuarter not tagged2025-11-18Citizens Lifts Benitec Biopharma Inc. (BNTC) Price Target on Topline Results for Oculopharyngeal Muscular Dystrophy Treatment
Insider Monkey
Citizens Lifts Benitec Biopharma Inc. (BNTC) Price Target on Topline Results for Oculopharyngeal Muscular Dystrophy Treatment
Benitec Biopharma Inc. (NASDAQ:BNTC) is one of the best breakout stocks to invest in. On November 4, an analyst at Citizens JMP raised the stock’s price target to $22 from $20 while reiterating an Outperform rating. The price target hike follows topline clinical data results that showed a 100% response rate for BB-301 in Phase 1b/2a of the treatment for Oculopharyngeal Muscular Dystrophy (OPMD), a rare genetic muscle-wasting disease. The US Food and Drug Administration has already granted the company Fast Track designation for the gene therapy following the positive interim results. In the trials, the treatment successfully reduced swallowing difficulties in patients and shortened the time required to consume liquids. Meanwhile, on November 11, Suvretta Capital Management and related entities announced a $20 million investment in Benitec Biopharma. Benitec Biopharma Inc. (NASDAQ:BNTC) is a clinical-stage biotechnology company that develops novel genetic medicines using its proprietary DNA-directed RNA interference (ddRNAi) platform. Its platform combines gene therapy and RNA interference to create treatments that silence disease-causing genes and simultaneously replace them with functional, wild-type genes after a single administration. While we acknowledge the potential of BNTC as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 13 Best AI Stocks to Buy Under $20 and Top 6 Steel Stocks to Buy Amid US Tariffs. Disclosure: None. This article is originally published at Insider Monkey.
Investor releaseQuarter not tagged2025-11-15Benitec Biopharma Releases First Quarter 2026 Financial Results and Provides Operational Update
GlobeNewswire
Benitec Biopharma Releases First Quarter 2026 Financial Results and Provides Operational Update
–Fast Track Designation was granted for BB-301 following U.S. Food and Drug Administration (FDA) review of positive interim clinical study results and proprietary Responder Analysis planned for use in pivotal study for BB-301– –Positive interim clinical study results from the Phase 1b/2a trial of BB-301, showed a 100% responder rate, with all six patients in Cohort 1 meeting the formal statistical criteria for response to BB-301– –First patient of Cohort 2 successfully treated with BB-301 in Q4 of 2025– –The Company raised approximately $100 million in an oversubscribed public offering of common stock, which is expected to fund advancement of the BB-301 Oculopharyngeal Muscular Dystrophy (OMPD) registrational program and associated regulatory filing activities– HAYWARD, Calif., Nov. 14, 2025 (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 financial results for its first fiscal quarter ended September 30, 2025. The Company has filed its quarterly report on Form 10-Q for the quarter ended September 30, 2025 with the U.S. Securities and Exchange Commission (SEC). “We are fortunate to have achieved several important milestones this year. We previously announced positive interim clinical study results from the Phase 1b/2a trial of BB-301, with a 100% responder rate to BB-301 in Cohort 1. BB-301 was also granted Fast Track Designation by the FDA, and we completed a significant capital raise to fund the advancement of BB-301, which has the potential to become the first approved therapy for the treatment of the central clinical symptom plaguing OPMD patients,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “We remain extremely grateful for the strong support from the OPMD community and their families, the clinical research community, and the investment community. We look forward to continued collaboration with the FDA in 2026 to advance the development of BB-301.” Operational Updates The key milestones related to the development of BB-301 for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD)-related dysphagia, are outlined below: On November 3rd, the Company announc…Read full documentShow less
–Fast Track Designation was granted for BB-301 following U.S. Food and Drug Administration (FDA) review of positive interim clinical study results and proprietary Responder Analysis planned for use in pivotal study for BB-301– –Positive interim clinical study results from the Phase 1b/2a trial of BB-301, showed a 100% responder rate, with all six patients in Cohort 1 meeting the formal statistical criteria for response to BB-301– –First patient of Cohort 2 successfully treated with BB-301 in Q4 of 2025– –The Company raised approximately $100 million in an oversubscribed public offering of common stock, which is expected to fund advancement of the BB-301 Oculopharyngeal Muscular Dystrophy (OMPD) registrational program and associated regulatory filing activities– HAYWARD, Calif., Nov. 14, 2025 (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 financial results for its first fiscal quarter ended September 30, 2025. The Company has filed its quarterly report on Form 10-Q for the quarter ended September 30, 2025 with the U.S. Securities and Exchange Commission (SEC). “We are fortunate to have achieved several important milestones this year. We previously announced positive interim clinical study results from the Phase 1b/2a trial of BB-301, with a 100% responder rate to BB-301 in Cohort 1. BB-301 was also granted Fast Track Designation by the FDA, and we completed a significant capital raise to fund the advancement of BB-301, which has the potential to become the first approved therapy for the treatment of the central clinical symptom plaguing OPMD patients,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “We remain extremely grateful for the strong support from the OPMD community and their families, the clinical research community, and the investment community. We look forward to continued collaboration with the FDA in 2026 to advance the development of BB-301.” Operational Updates The key milestones related to the development of BB-301 for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD)-related dysphagia, are outlined below: On November 3rd, the Company announced positive interim clinical study results from its Phase 1b/2a trial of BB-301. Following administration of BB-301, Cohort 1 patients experienced significant and sustained improvements across multiple clinical measures of swallowing function. All 6 patients in Cohort 1 met the formal statistical criteria for response to BB-301, representing a 100% responder rate. Following review of the positive interim clinical study results from the Phase 1b/2a trial of BB-301, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation (FTD) to BB-301 for the treatment of dysphagia associated with Oculopharyngeal Muscular Dystrophy. Benitec also announced the first patient in Cohort 2 of the BB-301 Phase 1b/2a trial has been successfully treated with BB-301. The Company hosted a webcast to discuss the positive interim clinical study results from its Phase 1b/2a trial of BB-301 on Monday November 3rd, and a replay of that event can be found here. As announced on July 9th, in accordance with the protocol for the BB-301 Phase 1b/2a trial, a meeting of the independent data safety monitoring board (DSMB) was convened following the completion of the 28-day post BB-301 dosing visit for the sixth patient enrolled into Cohort 1. The DSMB recommended continuation of subject enrollment for the Phase 1b/2a Clinical Treatment Study. Corporate Updates: On November 3rd, Benitec announced the addition of Sharon Mates, Ph.D., to its Board of Directors. Dr. Mates served as the Chairman and Chief Executive Officer, and was Co-founder of Intra-Cellular Therapies, a mental health company, from June 2002 until it was acquired for $14.6 billion by Johnson & Johnson in 2025. On November 5th, Benitec announced the pricing of its underwritten public offering of 5,930,000 shares of its common stock and a concurrent registered direct offering of 1,481,481 shares of its common stock with long-term investor Suvretta Capital. Each share of common stock was sold at an offering price of $13.50. In addition, the Company has granted the underwriters a 30-day option to purchase up to an additional 889,500 shares of common stock on the same terms and conditions. Benitec plans to use the net proceeds from this financing, together with existing cash on hand, to support the continued development and registration of its BB-301 candidate in OPMD, working capital and other general corporate purposes. Financial Highlights First Quarter 2026 Financial Results Total Expenses for the quarter ended September 30, 2025, were $9.8 million compared to $5.8 million for the quarter ended September 30, 2024. The Company incurred $3.4 million of research and development expenses for the quarter ended September 30, 2025 compared to $3.6 million for the quarter ended September 30, 2024. Research and development expenses relate primarily to ongoing clinical development of BB-301 for the treatment of OPMD-related Dysphagia. General and administrative expenses were $6.4 million for the quarter ended September 30, 2025 compared to $2.2 million for the quarter ended September 30, 2024. The total comprehensive loss from operations for the quarter ended September 30, 2025, was $8.9 million compared to a loss of $5.2 million for the quarter ended September 30, 2024. Net loss attributable to shareholders for the quarter ended September 30, 2025, was $9.0 million, or $(0.22) per basic and diluted share, compared to a net loss of $5.1 million, or $(0.18) per basic and diluted share for the quarter ended September 30, 2024. As of September 30, 2025, the Company had $94.5 million in cash and cash equivalents. On November 5, 2025, Benitec concluded an equity financing grossing approximately $100 million before deducting costs. About BB-301 BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against mutant PABPN1 (the causative gene for OPMD). The two siRNAs are modeled into microRNA backbones to silence expression of faulty mutant PABPN1, while allowing expression of the codon-optimized PABPN1 to replace the mutant with a functional version of the protein. We believe the silence and replace mechanism of BB-301 is uniquely positioned for the treatment of OPMD by halting mutant expression while providing a functional replacement protein. BB-301 has received Orphan Drug Designation from the EMA and Orphan Drug and Fast Track Designations from the FDA. About Benitec Biopharma, Inc. Benitec Biopharma Inc. (“Benitec” or the “Company”) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary “Silence and Replace” DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview of the Company can be found on Benitec’s website at www.benitec.com. Forward Looking Statements Except for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec’s plans to develop and commercialize its product candidates, the timing of the completion of pre-clinical and clinical trials, the timing of the availability of data from our clinical trials, the timing and sufficiency of patient enrollment and dosing in clinical trials, the timing of expected regulatory filings, and the clinical utility and potential attributes and benefits of ddRNAi and Benitec’s product candidates, and other forward-looking statements. These forward-looking statements are based on the Company’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: the success of our plans to develop and potentially commercialize our product candidates; the timing of the completion of preclinical studies and clinical trials; the timing and sufficiency of patient enrollment and dosing in any future clinical trials; the timing of the availability of data from our clinical trials; the timing and outcome of regulatory filings and approvals; the development of novel AAV vectors; our potential future out-licenses and collaborations; the plans of licensees of our technology; the clinical utility and potential attributes and benefits of ddRNAi and our product candidates, including the potential duration of treatment effects and the potential for a “one shot” cure; our intellectual property position and the duration of our patent portfolio; expenses, ongoing losses, future revenue, capital needs and needs for additional financing, and our ability to access additional financing given market conditions and other factors; the length of time over which we expect our cash and cash equivalents to be sufficient to execute on our business plan; unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities and other regulatory developments; the Company’s ability to protect and enforce its patents and other intellectual property rights; the Company’s dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Company’s products and the products of the Company’s collaboration partners; the acceptance of the Company’s products and the products of the Company’s collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the impact of, and our ability to remediate, the identified material weakness in our internal controls over financial reporting; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company’s reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements. Investor Relations Contact: Irina Koffler LifeSci Advisors, LLC (917) 734-7387 [email protected]
Investor releaseQuarter not tagged2025-11-03Benitec Biopharma Provides Positive Interim Clinical Study Results for BB-301 Phase 1b/2a Clinical Trial and Receives FDA Fast Track Designation for BB-301
GlobeNewswire
Benitec Biopharma Provides Positive Interim Clinical Study Results for BB-301 Phase 1b/2a Clinical Trial and Receives FDA Fast Track Designation for BB-301
Fast Track Designation was granted for BB-301 following FDA review of positive interim clinical study results and proprietary Responder Analysis planned for use in pivotal study for BB-301 BB-301 has also been granted Orphan Drug Designation from both FDA and EMA All six patients enrolled into Cohort 1 met the formal statistical criteria for response to BB-301, representing a 100% response rate Following the administration of BB-301, Cohort 1 patients experienced significant continuing reductions in dysphagic symptom burden, post-swallow residue accumulation, time required to consume fixed volumes of liquid, and improved pharyngeal closure during swallowing First patient in Cohort 2 successfully treated with BB-301 in fourth quarter of 2025 Benitec plans to meet with the FDA in 2026 to confirm the BB-301 pivotal study design Dr. Sharon Mates, who served as Chairman, Chief Executive Officer, and Co-founder of Intra-Cellular Therapies Inc., appointed to the Benitec Biopharma Board of Directors as previously disclosed HAYWARD, Calif., Nov. 03, 2025 (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 “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today provides positive interim clinical results for the BB-301 Phase 1b/2a Clinical Trial. Following administration of BB-301, Cohort 1 patients demonstrated significant and sustained improvements across multiple clinical measures including dysphagic symptom burden, post-swallow residue accumulation, time required to consume fixed volumes of liquid, as well as improved pharyngeal closure during swallowing. All six patients enrolled into Cohort 1 met the formal statistical criteria for response to BB-301, representing a 100% response rate. Following review of these encouraging interim data, the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to BB-301 for the treatment of OPMD with dysphagia. BB-301 was also previously granted Orphan Drug Designation from both the FDA and European Medical Association (EMA). “Progressive dysphagia is a severe, life-threatening complication of OPMD which impacts 97% of OPMD patients, often leading to serious health risks, such as chronic choking, malnutrition, aspiration pneumonia, and…Read full documentShow less
Fast Track Designation was granted for BB-301 following FDA review of positive interim clinical study results and proprietary Responder Analysis planned for use in pivotal study for BB-301 BB-301 has also been granted Orphan Drug Designation from both FDA and EMA All six patients enrolled into Cohort 1 met the formal statistical criteria for response to BB-301, representing a 100% response rate Following the administration of BB-301, Cohort 1 patients experienced significant continuing reductions in dysphagic symptom burden, post-swallow residue accumulation, time required to consume fixed volumes of liquid, and improved pharyngeal closure during swallowing First patient in Cohort 2 successfully treated with BB-301 in fourth quarter of 2025 Benitec plans to meet with the FDA in 2026 to confirm the BB-301 pivotal study design Dr. Sharon Mates, who served as Chairman, Chief Executive Officer, and Co-founder of Intra-Cellular Therapies Inc., appointed to the Benitec Biopharma Board of Directors as previously disclosed HAYWARD, Calif., Nov. 03, 2025 (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 “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today provides positive interim clinical results for the BB-301 Phase 1b/2a Clinical Trial. Following administration of BB-301, Cohort 1 patients demonstrated significant and sustained improvements across multiple clinical measures including dysphagic symptom burden, post-swallow residue accumulation, time required to consume fixed volumes of liquid, as well as improved pharyngeal closure during swallowing. All six patients enrolled into Cohort 1 met the formal statistical criteria for response to BB-301, representing a 100% response rate. Following review of these encouraging interim data, the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to BB-301 for the treatment of OPMD with dysphagia. BB-301 was also previously granted Orphan Drug Designation from both the FDA and European Medical Association (EMA). “Progressive dysphagia is a severe, life-threatening complication of OPMD which impacts 97% of OPMD patients, often leading to serious health risks, such as chronic choking, malnutrition, aspiration pneumonia, and death. We are excited by the profound effect that BB-301 can potentially have on this progressive disease as demonstrated by the interim clinical trial results for Cohort 1, where 100% of patients were responders” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec Biopharma Inc. “Securing Fast Track designation for BB-301 reflects the strength of our clinical data and the urgency of the unmet need in OPMD. This recognition validates our team’s scientific and strategic execution, and we look forward to continued collaboration with the FDA as we advance toward a pivotal clinical trial.” The pre-treatment data for Cohort 1 patients reflect the first six months of Natural History Study follow-up and the final pre-treatment visit (i.e., the Phase 1 Screening Visit) The interim post-treatment data for Cohort 1 patients reflect the following: 12-months of post-BB-301-treatment follow-up for Patient 1 and Patient 2 9-months of post-BB-301-treatment follow-up for Patient 3 6-months of post-BB-301-treatment follow-up for Patient 4 and Patient 5; and 3-months of post-BB-301-treatment follow-up for Patient 6 As the total dysphagic symptom burden experienced by OPMD patients has several known underlying contributors, the development of a multi-component composite endpoint to evaluate the potential treatment effects of BB-301 allows for incorporation of multiple discrete assessments that, in total, assess disease progression and treatment benefit of BB-301. The BB-301 Responder Analysis (the multi-component composite endpoint) is comprised of a combination of patient-reported outcome results, objective assessment results, and swallowing capacity assessment results: Patient-Reported Outcome assessment results include: Sydney Swallow Questionnaire or “SSQ” results Objective Assessment Results include: Videofluoroscopic swallowing study results (Pharyngeal Area at Maximum Constriction or “PhAMPC”, Post-Swallow Pharyngeal Residue as measured by Total Pharyngeal Residue or “TPR” and Normalized Residue Ratio Scale or “NRRS”, Frequency of sequential swallows or “SEQ”) Functional Swallowing Capacity Assessment Results include: Clinically administered drinking assessment results (as measured by the cold-water timed drinking test or “CWDT”) Following the administration of BB-301, Cohort 1 patients experienced clinically significant reductions, and met the formal statistical criteria for response, in the following assessments: Summary of Cohort 1 Results To date, the Benitec OPMD Natural History Study and the BB-301 Phase 1b/2a Clinical Trial represent the only clinical studies ever conducted which employ serial evaluation of the dysphagic symptom burden of OPMD patients and serial radiographic evaluation of the anatomical and functional elements of swallowing in OPMD patients at a frequency of approximately every 3-months. Positive interim clinical study results demonstrate the significant and durable clinical benefit achieved by patients treated with BB-301. Company Webcast Information: Webcast title: Interim BB-301 Phase 1b/2a Clinical Study Update A live webcast of the interim clinical data presentation, will be held at 8:00 AM ET on Monday, November 3, 2025, and can be accessed by clicking here. The event replay and corresponding slides will be placed on the News & Events tab on the Investor page of the Benitec website. About BB-301 BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against mutant PABPN1 (the causative gene for OPMD). The two siRNAs are modeled into microRNA backbones to silence expression of faulty mutant PABPN1, while allowing expression of the codon-optimized PABPN1 to replace the mutant with a functional version of the protein. We believe the silence and replace mechanism of BB-301 is uniquely positioned for the treatment of OPMD by halting mutant expression while providing a functional replacement protein. BB-301 has received Orphan Drug Designation from the EMA and Orphan Drug and Fast Track Designations from the FDA. About Benitec Biopharma, Inc. Benitec Biopharma Inc. (“Benitec” or the “Company”) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary “Silence and Replace” DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview of the Company can be found on Benitec’s website at www.benitec.com. Forward Looking Statements Except for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec’s plans to develop and commercialize its product candidates and the clinical utility and potential attributes and benefits of ddRNAi and Benitec’s product candidates, and other forward-looking statements. These forward-looking statements are based on the Company’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: the success of our plans to develop and potentially commercialize our product candidates; the timing of the completion of preclinical studies and clinical trials; the timing and sufficiency of patient enrollment and dosing in any future clinical trials; the timing of the availability of data from our clinical trials; the timing and outcome of regulatory filings and approvals; the development of novel AAV vectors; our potential future out-licenses and collaborations; the plans of licensees of our technology; the clinical utility and potential attributes and benefits of ddRNAi and our product candidates, including the potential duration of treatment effects and the potential for a “one shot” cure; our intellectual property position and the duration of our patent portfolio; expenses, ongoing losses, future revenue, capital needs and needs for additional financing, and our ability to access additional financing given market conditions and other factors; the length of time over which we expect our cash and cash equivalents to be sufficient to execute on our business plan; unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of patients in clinical trials; determinations made by the FDA and other governmental authorities and other regulatory developments; the Company’s ability to protect and enforce its patents and other intellectual property rights; the Company’s dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Company’s products and the products of the Company’s collaboration partners; the acceptance of the Company’s products and the products of the Company’s collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the impact of, and our ability to remediate, the identified material weakness in our internal controls over financial reporting, the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company’s reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements. Investor Relations Contact: Irina Koffler LifeSci Advisors, LLC (917) 734-7387 [email protected] A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/82289b3c-6338-4e34-9274-b0507edf6346
Investor releaseQuarter not tagged2025-09-23Benitec Biopharma Releases Full Year 2025 Financial Results and Provides Operational Update
GlobeNewswire
Benitec Biopharma Releases Full Year 2025 Financial Results and Provides Operational Update
Interim Clinical Study Results for Cohort 1 of the BB-301 Phase 1b/2a Treatment Study Anticipated in Q4 2025 Enrollment of the First Subject into Cohort 2 of the BB-301 Phase 1b/2a Treatment Study Expected in Q4 2025 HAYWARD, Calif., Sept. 22, 2025 (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 “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today announced financial results for its full year ended June 30, 2025. The Company has filed its annual report on Form 10-K with the U.S. Securities and Exchange Commission. “We remain incredibly thankful for our continued close collaboration with families, clinical researchers, and healthcare providers as we advance the BB-301 clinical development program. With increasing durations of clinical follow-up for Subjects enrolled into Cohort 1 of the BB-301 Phase 1b/2a Treatment Study, our enthusiasm continues to be strong for the potential to develop BB-301 as a safe and efficacious therapy for the improvement of swallowing in patients diagnosed with OPMD with dysphagia,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “We are also grateful to have received a favorable recommendation to continue enrollment into Cohort 2 from our Independent Data Safety Monitoring Board following the safe treatment of the sixth and final Subject of Cohort 1. We look forward to beginning enrollment of Subjects into Cohort 2 of the BB-301 Phase 1b/2a Treatment Study, as well as providing additional interim clinical study results for Cohort 1 Subjects, in the fourth calendar quarter of this year.” Corporate Highlights In accordance with the protocol for the BB-301 Phase 1b/2a Treatment Study, a meeting of the Independent Data Safety Monitoring Board (DSMB) was convened following the completion of the 28-day post BB-301 dosing visit for the sixth Subject enrolled into Cohort 1. At the conclusion of the meeting, the DSMB formally recommended the continuation of subject enrollment into the Phase 1b/2a Treatment Study, which is expected to begin in calendar Q4 2025. We look forward to providing additional interim clinical study results for Cohort 1 Subjects in the fourth calendar quarter of this year. Financial Highlights…Read full documentShow less
Interim Clinical Study Results for Cohort 1 of the BB-301 Phase 1b/2a Treatment Study Anticipated in Q4 2025 Enrollment of the First Subject into Cohort 2 of the BB-301 Phase 1b/2a Treatment Study Expected in Q4 2025 HAYWARD, Calif., Sept. 22, 2025 (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 “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today announced financial results for its full year ended June 30, 2025. The Company has filed its annual report on Form 10-K with the U.S. Securities and Exchange Commission. “We remain incredibly thankful for our continued close collaboration with families, clinical researchers, and healthcare providers as we advance the BB-301 clinical development program. With increasing durations of clinical follow-up for Subjects enrolled into Cohort 1 of the BB-301 Phase 1b/2a Treatment Study, our enthusiasm continues to be strong for the potential to develop BB-301 as a safe and efficacious therapy for the improvement of swallowing in patients diagnosed with OPMD with dysphagia,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “We are also grateful to have received a favorable recommendation to continue enrollment into Cohort 2 from our Independent Data Safety Monitoring Board following the safe treatment of the sixth and final Subject of Cohort 1. We look forward to beginning enrollment of Subjects into Cohort 2 of the BB-301 Phase 1b/2a Treatment Study, as well as providing additional interim clinical study results for Cohort 1 Subjects, in the fourth calendar quarter of this year.” Corporate Highlights In accordance with the protocol for the BB-301 Phase 1b/2a Treatment Study, a meeting of the Independent Data Safety Monitoring Board (DSMB) was convened following the completion of the 28-day post BB-301 dosing visit for the sixth Subject enrolled into Cohort 1. At the conclusion of the meeting, the DSMB formally recommended the continuation of subject enrollment into the Phase 1b/2a Treatment Study, which is expected to begin in calendar Q4 2025. We look forward to providing additional interim clinical study results for Cohort 1 Subjects in the fourth calendar quarter of this year. Financial Highlights Full Year 2025 Financial Results For the year ended June 30, 2025, the Company reported total expenses of $41.8 million compared to $22.5 million for the year ended June 30, 2024. Research and development expenses were $18.3 million in 2025, up from $15.6 million in 2024, and were primarily related to the ongoing clinical development of BB-301 for the treatment of OPMD. The increase in research and development expenses reflected the timing of contract manufacturing activities and payments associated with the OPMD Natural History and Dosing study. General and administrative expenses totaled $23.4 million in 2025 compared to $7.0 million in 2024. The increase was primarily driven by higher share-based compensation of $14.5 million, as well as increases in legal fees of $492,000, consulting fees of $605,000, travel expenses of $219,000, and salaries and wages of $685,000. The loss from operations for the year ended June 30, 2025, was $37.9 million compared to $21.8 million for the prior year. Net loss attributable to shareholders was $37.9 million, or $1.05 per basic and diluted share, compared to a net loss of $22.4 million, or $1.22 per basic and diluted share, for the year ended June 30, 2024. As of June 30, 2025, the Company had $97.7 million in cash and cash equivalents. About BB-301 BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against mutant PABPN1 (the causative gene for OPMD). The two siRNAs are modeled into microRNA backbones to silence expression of faulty mutant PABPN1, while allowing expression of the codon-optimized PABPN1 to replace the mutant with a functional version of the protein. We believe the silence and replace mechanism of BB-301 is uniquely positioned for the treatment of OPMD by halting mutant expression while providing a functional replacement protein. About Benitec Biopharma, Inc. Benitec Biopharma Inc. (“Benitec” or the “Company”) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary “Silence and Replace” DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview of the Company can be found on Benitec’s website at www.benitec.com. Forward Looking Statements Except for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec’s plans to develop and commercialize its product candidates, the timing of the completion of pre-clinical and clinical trials, the timing of the availability of data from our clinical trials, the timing and sufficiency of patient enrollment and dosing in clinical trials, the timing of expected regulatory filings, and the clinical utility and potential attributes and benefits of ddRNAi and Benitec’s product candidates, and other forward-looking statements. These forward-looking statements are based on the Company’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: the success of our plans to develop and potentially commercialize our product candidates; the timing of the completion of preclinical studies and clinical trials; the timing and sufficiency of patient enrollment and dosing in any future clinical trials; the timing of the availability of data from our clinical trials; the timing and outcome of regulatory filings and approvals; the development of novel AAV vectors; our potential future out-licenses and collaborations; the plans of licensees of our technology; the clinical utility and potential attributes and benefits of ddRNAi and our product candidates, including the potential duration of treatment effects and the potential for a “one shot” cure; our intellectual property position and the duration of our patent portfolio; expenses, ongoing losses, future revenue, capital needs and needs for additional financing, and our ability to access additional financing given market conditions and other factors; the length of time over which we expect our cash and cash equivalents to be sufficient to execute on our business plan; unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities and other regulatory developments; the Company’s ability to protect and enforce its patents and other intellectual property rights; the Company’s dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Company’s products and the products of the Company’s collaboration partners; the acceptance of the Company’s products and the products of the Company’s collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the impact of, and our ability to remediate, the identified material weakness in our internal controls over financial reporting; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company’s reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements. Investor Relations Contact: Irina Koffler LifeSci Advisors, LLC (917) 734-7387 [email protected]
Investor releaseQuarter not tagged2025-05-14Benitec Biopharma Releases Third Quarter 2025 Financial Results
GlobeNewswire
Benitec Biopharma Releases Third Quarter 2025 Financial Results
HAYWARD, Calif., May 14, 2025 (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 “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today announced financial results for its third fiscal quarter ended March 31, 2025. The Company has filed its quarterly report on Form 10-Q with the U.S. Securities and Exchange Commission. “We are profoundly honored to be closely engaged with the OPMD patient community and are thankful for the support of the Subjects and their families as we remain focused on the continued development of BB-301 for the treatment of dysphagia in OPMD patients,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “The sixth and final Subject of Cohort 1 was safely treated with the low dose of BB-301 in April 2025. We look forward to enrolling additional Subjects at the next, higher dose of BB-301 later this year. Additional clinical study updates for Subjects enrolled in Cohort 1 are planned for the fourth calendar quarter of this year.” Financial Highlights Third Fiscal Quarter 2025 Financial Results Total Expenses for the quarter ended March 31, 2025, were $10.2 million compared to $4.1 million for the quarter ended March 31, 2024. The Company incurred $6.0 million of research and development expenses compared to $2.6 million for the comparable quarter ended March 31, 2024. Research and development expenses relate primarily to ongoing clinical development of BB-301 for the treatment of OPMD. General and administrative expenses were $4.2 million compared to $1.6 million for the quarter ended March 31, 2024. The loss from operations for the quarter ended March 31, 2025, was $10.2 million compared to a loss of $4.1 million for the quarter ended March 31, 2024. Net loss attributable to shareholders for the quarter ended March 31, 2025, was $9.4 million, or $0.24 per basic and diluted share, compared to a net loss of $4.3 million, or $0.23 per basic and diluted share for the quarter ended March 31, 2024. The basic earnings per share calculation has been revised to include pre-funded warrants in the weighted number of shares outstanding for the current period and the comparative periods. As of March 31, 2025, the Company had $103.6 mil…Read full documentShow less
HAYWARD, Calif., May 14, 2025 (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 “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today announced financial results for its third fiscal quarter ended March 31, 2025. The Company has filed its quarterly report on Form 10-Q with the U.S. Securities and Exchange Commission. “We are profoundly honored to be closely engaged with the OPMD patient community and are thankful for the support of the Subjects and their families as we remain focused on the continued development of BB-301 for the treatment of dysphagia in OPMD patients,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “The sixth and final Subject of Cohort 1 was safely treated with the low dose of BB-301 in April 2025. We look forward to enrolling additional Subjects at the next, higher dose of BB-301 later this year. Additional clinical study updates for Subjects enrolled in Cohort 1 are planned for the fourth calendar quarter of this year.” Financial Highlights Third Fiscal Quarter 2025 Financial Results Total Expenses for the quarter ended March 31, 2025, were $10.2 million compared to $4.1 million for the quarter ended March 31, 2024. The Company incurred $6.0 million of research and development expenses compared to $2.6 million for the comparable quarter ended March 31, 2024. Research and development expenses relate primarily to ongoing clinical development of BB-301 for the treatment of OPMD. General and administrative expenses were $4.2 million compared to $1.6 million for the quarter ended March 31, 2024. The loss from operations for the quarter ended March 31, 2025, was $10.2 million compared to a loss of $4.1 million for the quarter ended March 31, 2024. Net loss attributable to shareholders for the quarter ended March 31, 2025, was $9.4 million, or $0.24 per basic and diluted share, compared to a net loss of $4.3 million, or $0.23 per basic and diluted share for the quarter ended March 31, 2024. The basic earnings per share calculation has been revised to include pre-funded warrants in the weighted number of shares outstanding for the current period and the comparative periods. As of March 31, 2025, the Company had $103.6 million in cash and cash equivalents. About BB-301 BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against mutant PABPN1 (the causative gene for OPMD). The two siRNAs are modeled into microRNA backbones to silence expression of faulty mutant PABPN1, while allowing expression of the codon-optimized PABPN1 to replace the mutant with a functional version of the protein. We believe the silence and replace mechanism of BB-301 is uniquely positioned for the treatment of OPMD by halting mutant expression while providing a functional replacement protein. About Benitec Biopharma, Inc. Benitec Biopharma Inc. (“Benitec” or the “Company”) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary “Silence and Replace” DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview of the Company can be found on Benitec’s website at www.benitec.com. Forward Looking Statements Except for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec’s plans to develop and commercialize its product candidates, the timing of the completion of pre-clinical and clinical trials, the timing of the availability of data from our clinical trials, the timing and sufficiency of patient enrollment and dosing in clinical trials, the timing of expected regulatory filings, and the clinical utility and potential attributes and benefits of ddRNAi and Benitec’s product candidates, and other forward-looking statements. These forward-looking statements are based on the Company’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: the success of our plans to develop and potentially commercialize our product candidates; the timing of the completion of preclinical studies and clinical trials; the timing and sufficiency of patient enrollment and dosing in any future clinical trials; the timing of the availability of data from our clinical trials; the timing and outcome of regulatory filings and approvals; the development of novel AAV vectors; our potential future out-licenses and collaborations; the plans of licensees of our technology; the clinical utility and potential attributes and benefits of ddRNAi and our product candidates, including the potential duration of treatment effects and the potential for a “one shot” cure; our intellectual property position and the duration of our patent portfolio; expenses, ongoing losses, future revenue, capital needs and needs for additional financing, and our ability to access additional financing given market conditions and other factors, including our capital structure; the length of time over which we expect our cash and cash equivalents to be sufficient to execute on our business plan; unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities and other regulatory developments; the Company’s ability to protect and enforce its patents and other intellectual property rights; the Company’s dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Company’s products and the products of the Company’s collaboration partners; the acceptance of the Company’s products and the products of the Company’s collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the impact of, and our ability to remediate, the identified material weakness in our internal controls over financial reporting; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company’s reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements. Investor Relations Contact: Irina KofflerLifeSci Advisors, LLC(917) [email protected]
Investor releaseQuarter not tagged2025-03-19Benitec Biopharma Reports Positive Interim Clinical Results for Three Subjects Treated with BB-301 in Phase 1b/2a Study to be Presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference
GlobeNewswire
Benitec Biopharma Reports Positive Interim Clinical Results for Three Subjects Treated with BB-301 in Phase 1b/2a Study to be Presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference
-Durable, Clinically Significant Improvements in Swallowing Function Achieved 12-months Post-Treatment with BB-301 for Subject 1- -Durable, Clinically Significant Improvements in Swallowing Function Achieved 12-months Post-Treatment with BB-301 for Subject 2, with Subject 2 Achieving a Clinically Normal Swallowing Profile Following the Significant Reduction in Total Dysphagic Symptom Burden- -Clinically Significant Improvements in Swallowing Function Achieved 3-months Post-Treatment with BB-301 for Subject 3, with Subject 3 Achieving a Clinically Normal Swallowing Profile Following the Significant Reduction in Total Dysphagic Symptom Burden- -Positive Interim Clinical Study Results to be Reported as a Late-Breaking Oral Presentation at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference- HAYWARD, Calif., March 19, 2025 (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 “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today announces continued durable improvements in swallowing function and reductions in total dysphagic symptom burden following administration of the low-dose of BB-301 in the first three Subjects treated in the BB-301 Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study (NCT06185673) in Oculopharyngeal Muscular Dystrophy (OPMD). Interim clinical study results will be presented today in an oral late-breaking podium presentation at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference, taking place in Dallas, Texas. The interim clinical study update to be presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference will detail the 12-month (365-day) post-treatment results for the first Subject, the 12-month (365-day) post-treatment results for the second Subject, and the 3- month (90-day) post-treatment results for the third Subject, each of whom have been safely treated with BB-301. The key radiographic efficacy endpoints that will be described during the presentation include serial videofluoroscopic swallowing study (“VFSS”) assessments of Swallowing Efficiency (via characterization of post swallow accumulation of food and liquid material or “Total Pharyngeal Residue”) and VFSS as…Read full documentShow less
-Durable, Clinically Significant Improvements in Swallowing Function Achieved 12-months Post-Treatment with BB-301 for Subject 1- -Durable, Clinically Significant Improvements in Swallowing Function Achieved 12-months Post-Treatment with BB-301 for Subject 2, with Subject 2 Achieving a Clinically Normal Swallowing Profile Following the Significant Reduction in Total Dysphagic Symptom Burden- -Clinically Significant Improvements in Swallowing Function Achieved 3-months Post-Treatment with BB-301 for Subject 3, with Subject 3 Achieving a Clinically Normal Swallowing Profile Following the Significant Reduction in Total Dysphagic Symptom Burden- -Positive Interim Clinical Study Results to be Reported as a Late-Breaking Oral Presentation at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference- HAYWARD, Calif., March 19, 2025 (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 “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today announces continued durable improvements in swallowing function and reductions in total dysphagic symptom burden following administration of the low-dose of BB-301 in the first three Subjects treated in the BB-301 Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study (NCT06185673) in Oculopharyngeal Muscular Dystrophy (OPMD). Interim clinical study results will be presented today in an oral late-breaking podium presentation at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference, taking place in Dallas, Texas. The interim clinical study update to be presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference will detail the 12-month (365-day) post-treatment results for the first Subject, the 12-month (365-day) post-treatment results for the second Subject, and the 3- month (90-day) post-treatment results for the third Subject, each of whom have been safely treated with BB-301. The key radiographic efficacy endpoints that will be described during the presentation include serial videofluoroscopic swallowing study (“VFSS”) assessments of Swallowing Efficiency (via characterization of post swallow accumulation of food and liquid material or “Total Pharyngeal Residue”) and VFSS assessments of Swallowing Effectiveness (via characterization of the frequency of pathologic sequential swallows which comprise rapid involuntary contractions of the pharyngeal muscles without restoration of the resting pharyngeal diameter between pharyngeal contractions). The key subject-reported efficacy endpoints that will be described during the presentation include serial assessments of total dysphagic symptom burden via the use of the Sydney Swallow Questionnaire or “SSQ” (a 17-question patient-reported outcome instrument). The average post-treatment results for each Subject will be compared to their average pre-treatment results (as evaluated during the five clinical assessment visits conducted during enrollment in the Benitec-sponsored OPMD Natural History Study). A detailed description of the interim clinical study results will be found here on the Company website following the completion of the formal presentation at 1:30 pm Central Time. “We are extremely grateful for the extraordinary commitment of the Subjects and their families to the BB-301 clinical development program. We are highly encouraged by the clinically significant improvements observed for the first three Subjects treated with BB-301, with Subject 2 and Subject 3 each achieving clinically normal swallowing profiles based on the results of the respective reductions in their total dysphagic symptom burdens,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “The sixth and final Subject of Cohort 1 will be treated with BB-301 in the second calendar quarter of this year, and we are highly optimistic about the potential for continued benefit in Subjects enrolled in the ongoing clinical study. We look forward to enrolling additional Subjects at the next, higher dose of BB-301 later this year.” OPMD is a rare, autosomal dominant, late-onset degenerative muscle disorder presenting in patients at 40-60 years of age. OPMD is principally characterized by severe progressive dysphagia, impacting 97% of patients, which can lead to chronic choking, malnutrition, aspiration pneumonia and, in severe cases, death. OPMD is caused by a mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene. There is no effective drug therapy available for OPMD. Current clinical interventions are limited to palliative surgical procedures and dietary modifications, which do not address the underlying cause of the disease. BB-301, a novel investigational gene therapy designed to improve the dysphagic symptoms of OPMD, is being evaluated in a Phase 1b/2a, open-label dose escalation study (NCT06185673) to assess safety and clinical activity. Subjects Enrolled into the BB-301 Clinical Development Program are Impacted by Two Discrete Drivers of Total Dysphagic Symptom Burden: OPMD Subjects enrolled into the OPMD Natural History Study and the BB-301 Phase 1b/2a Clinical Treatment Study can be impacted by the post swallow accumulation of food and liquid (“Inefficient Swallowing”). OPMD Subjects enrolled into the OPMD Natural History Study and the BB-301 Phase 1b/2a Clinical Treatment Study can be impacted by pathologic sequential swallows comprising rapid involuntary contractions of the pharyngeal muscles without restoration of the resting pharyngeal diameter between pharyngeal contractions (“Ineffective Swallowing”). Clinical Utility, Sensitivity, and Specificity of the Key Assessment Methods: In the BB-301 Phase 1b/2a Clinical Treatment Study, Radiologists and Speech Language Pathologists employ serial VFSS to objectively characterize the nature and severity of anatomical and functional abnormalities present in each Subject during the pre-treatment period and the post-treatment period. Serial SSQ assessments are employed to characterize the contribution of the VFSS findings to the total symptom burden experienced by each Subject, thus, linking the VFSS findings to the changes in Subject-reported symptom burden for the pre-treatment period and the post-treatment period. The SSQ has been used in conjunction with VFSS in several controlled clinical studies which compared the results for healthy subjects with those of dysphagic patients: The clinical studies demonstrated strong correlations between the results of VFSS-based assessments and SSQ-based assessments and facilitated the identification of SSQ cut-off values of 111.01 and 118.52, below which swallowing is clinically normal. Audag, et al.2 obtained a sensitivity of 93% and a specificity of 82% with the use of an SSQ cut-off score of 118.5. Additionally, these clinical studies provide robust support for the discriminant validity of the SSQ which is critical to its use in the accurate characterization of responses to treatment and the establishment of efficacy for a given treatment. Subjects in the BB-301 Phase 1b/2a Treatment Study are blinded to their SSQ Total Scores and VFSS (TPR and pathologic sequential swallowing frequency) assessment results, and the Central Reader for the VFSS assessments is blinded to the SSQ Total Scores for each Subject. Summary of the Interim Clinical Study Results for Subject 1, Subject 2, and Subject 3: Three Subjects with distinct causes of their respective dysphagic symptom burdens were safely treated with BB-301 (1.2e13 vg/Subject) and experienced significant, clinically meaningful improvements in swallowing function. There were no Severe Adverse Events. All three Subjects experienced significant reductions in their total dysphagic symptom burdens: Subject 1, plagued by Inefficient Swallowing, experienced clinically significant reductions in post swallow accumulation of foods and liquids per the VFSS Total Pharyngeal Residue (TPR) results and achieved a correspondingly significant reduction in total dysphagic symptom burden per the Total SSQ Scores 12-months post-BB-301 administration. This Subject has completed the statistical follow-up period of the BB-301 Phase 1b/2a Treatment Study. Subject 2, plagued by Ineffective Swallowing, experienced an almost complete resolution of pathologic sequential swallows per the VFSS results and achieved a correspondingly significant reduction in total dysphagic symptom burden per the Total SSQ Scores, achieving an SSQ score indicative of a clinically normal swallowing profile 12-months post-BB-301 administration. This Subject has completed the statistical follow-up period of the BB-301 Phase 1b/2a Treatment Study. Subject 3, plagued by Ineffective Swallowing, experienced complete resolution of pathologic sequential swallows per the VFSS results and achieved a correspondingly significant reduction in total dysphagic symptom burden per the Total SSQ Score, achieving an SSQ score indicative of a clinically normal swallowing profile 3-months post BB-301 administration. Clinical Study Results for Subject 1 (365-Days Post Treatment with BB-301): Subject 1, plagued by Inefficient Swallowing, experienced significant, clinically meaningful reductions of post swallow residue across all food and liquid consistencies 12-months post treatment with BB-301 per the VFSS results, and the VFSS results were accompanied by significant reductions in total dysphagic symptom burden. Subject 1 displayed significant reductions (i.e., improvements) in VFSS TPR (37% reduction for Thin Liquid, 18% reduction for Solid Food, and 29% reduction for Thick Liquids) following the administration of the low-dose of BB-301 as compared to the average values recorded for Subject 1 during the pre-treatment period. Subject 1 also displayed continued clinically meaningful reductions (i.e., improvements) in total dysphagic symptom burden with an average 12-month post-treatment SSQ Total Score demonstrating a 41% reduction as compared to the average values recorded for Subject 1 during the pre-treatment period. Clinical Study Results for Subject 2 (365-Days Post Treatment with BB-301): Subject 2, plagued by Ineffective Swallowing, experienced significant, clinically meaningful reductions in the frequency of pathologic sequential swallows 12-months post treatment with BB-301 per the VFSS results, and the VFSS results were accompanied by significant reductions in total dysphagic symptom burden with Subject 2 achieving an SSQ score indicative of a clinically normal swallowing profile. During the fifteen pre-treatment VFSS assessments conducted for Thin Liquid in the OPMD Natural History Study, Subject 2 experienced a high frequency of pathologic sequential swallows (observed during 80% of the swallowing assessments). During the twelve post-treatment VFSS assessments conducted for Thin Liquid in the Phase 1b/2a Clinical Treatment Study, Subject 2 experienced a significantly lower frequency of pathologic sequential swallows (observed during 17% of the swallowing assessments). Critically, the magnitude of reduction in the frequency of pathologic sequential swallows reported for Thin Liquid at the 6-month post-treatment interim clinical update in October 2024 (observed during 17% of the swallowing assessments) was maintained at month 12 (again observed during 17% of the swallowing assessments). Subject 2 also displayed continued clinically meaningful reductions (i.e., improvements) in total dysphagic symptom burden with an average 12-month post-treatment SSQ Total Score demonstrating a 91% reduction as compared to the average values recorded for Subject 2 during the pre-treatment period. The 12-month post-treatment average SSQ value of 68 units for Subject 2 represents a clinically normal swallowing profile. Interim Clinical Study Results for Subject 3 (90-Days Post Treatment with BB-301): Subject 3, plagued by Ineffective Swallowing, experienced significant, clinically meaningful reductions in the frequency of pathologic sequential swallows 3-months post treatment with BB-301 per the VFSS results, and the VFSS results were accompanied by a significant reduction in total dysphagic symptom burden with Subject 3 achieving an SSQ score indicative of a clinically normal swallowing profile. During the twenty-five pre-treatment VFSS assessments conducted for Thin Liquid and Thick Liquids in the OPMD Natural History Study, Subject 3 experienced a high frequency of pathologic sequential swallows (observed during 84% of the swallowing assessments). During the five post-treatment VFSS assessment conducted for Thin Liquid and Thick Liquids in the Phase 1b/2a Clinical Treatment Study, Subject 3 experienced no pathologic sequential swallows (observed during 0% of the swallowing assessments). Subject 3 also displayed a clinically meaningful reduction (i.e., improvement) in total dysphagic symptom burden with a 3-month post-treatment SSQ Total Score demonstrating a 68% reduction as compared to the average values recorded for Subject 3 during the pre-treatment period. The 3-month post-treatment SSQ value of 70 units for Subject 3 represents a clinically normal swallowing profile. The Subjects were blinded to their SSQ Total Scores and VFSS (TPR and pathologic sequential swallowing frequency) assessment results, and the Central Reader for the VFSS assessments was blinded to the SSQ Total Scores for each Subject. Enrollment Into the BB-301 Phase 1b/2a Clinical Treatment Study is Ongoing: Five Subjects have been safely treated with the low-dose of BB-301, and the sixth and final Subject of Cohort 1 is anticipated to receive the low-dose of BB-301 in 2Q 2025. Adverse Events: No Severe Adverse Events have been observed for the Subjects treated with BB-301. About Benitec Biopharma Inc. Benitec Biopharma Inc. (“Benitec” or the “Company”) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary “Silence and Replace” DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview of the Company can be found on Benitec’s website at www.benitec.com. Forward Looking Statements Except for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec’s plans to develop and potentially commercialize its product candidates, the timing of completion of pre-clinical and clinical trials, the timing of the availability of data from our clinical trials, the timing and sufficiency of patient enrollment and dosing in clinical trials, the timing of expected regulatory filings, the clinical utility and potential attributes and benefits of ddRNAi and Benitec’s product candidates, the intellectual property position, and other forward- looking statements. These forward-looking statements are based on the Company’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities; the Company’s ability to protect and enforce its patents and other intellectual property rights; the Company’s dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Company’s products and the products of the Company’s collaboration partners; the acceptance of the Company’s products and the products of the Company’s collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the Company’s ability to satisfy its capital needs through increasing its revenue and obtaining additional financing, given market conditions and other factors, including our capital structure; our ability to continue as a going concern; the length of time over which the Company expects its cash and cash equivalents to be sufficient to execute on its business plan; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company’s reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements. Investor Relations Contact: Irina KofflerLifeSci Advisors, LLC(917) 734-7387 [email protected] Source: Benitec Biopharma Inc. 1 Bua, B.A. and Bülow, M., BMC Research Notes (2014) 7:742; 2 Audag N., et al., Dysphagia (2019) 34:556-566

