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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

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Information

• Patient 1 of Cohort 1 has now completed the 24-month follow-up timepoint, and at month-24 post-tre...

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AI Summary

Benitec Biopharma reported strong efficacy data from its BB-301 clinical trial for OPMD, with significant improvement in patient symptoms at 24 months. All patients demonstrated durable responses, enhancing the credibility of BB-301's potential as a treatment. Anticipated engagement with the FDA in 2026 could expedite further development.

Sentiment Rationale

Strong clinical results enhance investor confidence and may lead to increased stock value, similar to past biotech successes like CRISPR (CRSP) after positive trial data releases.

Trading Thesis

BNTC presents a buying opportunity, driven by promising clinical trial results and FDA engagement outlook.

Market-Moving

  • Positive clinical results may lead to increased investor interest in BNTC.
  • Engagement with FDA could accelerate regulatory approval timelines.
  • Upcoming medical conference presentations might boost visibility and stock demand.
  • Potential expansion of indications for BB-301 could drive future revenue forecasts.

Key Facts

  • Patient 1 shows significant improvement at 24-month follow-up of BB-301.
  • All four patients completed 12-month follow-up, demonstrating durable responses.
  • Results indicate robust disease-modifying effects of BB-301 in trial patients.
  • Management plans to engage with FDA on BB-301 study design in mid-2026.
  • BB-301 received FDA Fast Track and Orphan Drug designations.

Companies Mentioned

  • CRSP (CRSP)
  • EDIT (EDIT)
  • NVTA (NVTA)

Corporate Developments

The updates fit within 'Corporate Developments' as they showcase pivotal clinical trial advancements for BNTC's lead product, BB-301, crucial for potential regulatory approval and market success.

Benitec Biopharma Reports Positive Results from BB-301 Phase 1b/2a Clinical Trial

HAYWARD, Calif., Jan. 11, 2026 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) has announced significant advancements in the Phase 1b/2a clinical study of BB-301, a novel gene therapy aimed at treating dysphagia in patients with oculopharyngeal muscular dystrophy (OPMD). The results demonstrate long-lasting efficacy, with patients showing marked improvements in swallowing conditions over a 24-month follow-up period.

Key Findings from the Study

The latest interim results from Cohort 1 of the BB-301 clinical trial highlight the following:

  • Patient 1 has completed the 24-month follow-up, exhibiting ongoing disease-modifying effects, including reduced post-swallow residue and diminished dysphagic symptoms.
  • All four patients in Cohort 1 successfully concluded the 12-month statistical follow-up, with each participant categorized as a formal responder to BB-301.
  • Durability of response in these patients was confirmed, showing improvements over time across multiple evaluation parameters.

Significant Clinical Improvements

Patient 1's progress was specifically assessed using videofluoroscopic swallowing studies (VFSS) and the Sydney Swallow Questionnaire (SSQ). Notably:

  • At the 24-month follow-up, Patient 1 achieved a 60% reduction in post-swallow residue as per the Normalized Residue Ratio Scale-Valleculae (NRRSv).
  • A stable 27% improvement in the pharyngeal area at maximum constriction (PhAMPC) has also been maintained, indicating sustained functionality in the swallowing process.
  • The total dysphagic symptom burden measured by the SSQ revealed a dramatic decline, with a 78% improvement noted at the 24-month assessment compared to pre-treatment levels.

Executive Insights

Dr. Jerel A. Banks, Executive Chairman and CEO of Benitec Biopharma, expressed enthusiasm regarding the results, stating, “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.” He further emphasized the potential impact on patients' lives and the company's commitment to advancing BB-301 through regulatory processes, looking forward to discussions with the U.S. Food and Drug Administration (FDA) in mid-2026.

Implications for Future Research

The results from this study provide a strong basis for further clinical development of BB-301, as Benitec Biopharma aims to refine its pivotal study design. As the clinical team prepares to present ongoing findings at upcoming medical conferences, the feedback from these discussions is expected to enhance the understanding of BB-301’s effectiveness.

About Benitec Biopharma

Benitec Biopharma Inc. (NASDAQ: BNTC) is at the forefront of biotechnology, focusing on gene therapy solutions that leverage its proprietary “Silence and Replace” DNA-directed RNA interference (ddRNAi) platform. The company's goal is to transform the treatment landscape for challenging conditions such as OPMD.

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