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Benitec Biopharma Announces Positive Interim Phase 1b/2a Results for High Dose BB-301 and Continued Durable Improvements for Low Dose BB-301

Globe Newswire•28/10/2026•08:00 ET
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Key Highlights

  • ➤100% of nine BB-301-treated patients were formal responders across low- and high-dose cohorts
  • ➤High-dose Cohort 2 showed greater Month 3 throat-emptying improvements than low-dose Cohort 1
  • ➤High-dose Cohort 2 reached 67% Month 3 response for functional swallowing capacity
  • ➤No serious adverse events occurred in any BB-301-treated patient to date
  • ➤Benitec targets initiating BB-301 pivotal study in mid-2027

Expert Statements

Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec

“Progressive loss of swallowing function has a devastating impact on OPMD Patients, yet there are no approved therapies to address this critical unmet medical need”

Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec

“The progress we continue to see regarding both the safety and efficacy of BB-301 reinforces our conviction in the program and the potential to establish a new treatment paradigm for Patients suffering from OPMD-related dysphagia.”

Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec

“As we advance toward our pivotal study in mid-2027, we remain focused on the promise of delivering a potentially transformative therapy to the OPMD community.”

- Both low dose and high dose BB-301 continue to drive robust improvements in swallowing function for dysphagic Patients with Oculopharyngeal Muscular Dystrophy (OPMD)

- 100% of Patients treated with BB-301 across both doses are formal Responders to therapy

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