BNTC

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...

- 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...

- 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...

-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...

• 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...

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.

–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...

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...

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...

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...

-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...