Benitec Biopharma Announces Regulatory Update Following Type C Meeting with FDA for BB-301 Pivotal Study
Key Highlights
- ➤FDA requested additional data, including Benitec’s OPMD Natural History Study data
- ➤BB-301 pivotal study design expected confirmation in 1Q 2027
- ➤BB-301 pivotal trial enrollment remains planned for mid-2027
- ➤Benitec will present Phase 1b/2a interim results at ESGCT October 27-30, 2026
Expert Statements
Jerel A. Banks, Executive Chairman and Chief Executive Officer of Benitec
“We greatly appreciate the collaboration from the FDA and are encouraged by the constructive nature of our discussions”
Jerel A. Banks, Executive Chairman and Chief Executive Officer of Benitec
“The OPMD natural history study was designed from the outset to provide a rigorous foundation for the clinical development of BB-301, and the additional available data requested by the Agency represent an important final component of this ongoing development work.”
Jerel A. Banks, Executive Chairman and Chief Executive Officer of Benitec
“We look forward to providing these data to the FDA in the coming weeks and confirming the pivotal study design for BB-301 in the first quarter of 2027.”
Jerel A. Banks, Executive Chairman and Chief Executive Officer of Benitec
“With the comprehensive body of clinical and natural history data generated to date, we remain on track to initiate the pivotal study in mid-2027 and are focused on advancing BB-301 toward becoming the first approved disease-modifying therapy for patients living with OPMD-related dysphagia.”
* The BB-301 pivotal trial remains on track to begin in mid-2027 * Discussions regarding an open-label, single-arm, pivotal study for BB-301 are ongoing, and based on these discussions, the FDA requested additional available data, including data from the Benitec-sponsored OPMD Natural History Study * Final pivotal study design expected to be confirmed in 1Q 2027 * Benitec will present interim clinical results from the ongoing BB-301 Phase 1b/2a study as a late-breaking poster presentation, which will encompass 12-month follow-up data for all six Cohort 1 patients and interim follow-up data for all three Cohort 2 patients, at the European Society of Gene and Cell Therapy (ESGCT) Annual Congress in Hamburg, Germany, October 27-30, 2026 HAYWARD, Calif., Oct. 05, 2026 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or the “Company”), a clinical-stage biotechnology company developing disease-modifying genetic medicines based on its proprietary “Silence and Replace” DNA-directed RNA interference (ddRNAi) platform, today announced the receipt of formal feedback from the U.S. Food and Drug Administration (FDA) following the Type C meeting in 3Q 2026.
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