Benitec Biopharma Announces Regulatory Update Following Type C Meeting with FDA for BB-301 Pivotal Study (Form 8-K)

Benitec Biopharma Announces Regulatory Update Following Type C Meeting with FDA for BB-301 Pivotal Study

HAYWARD, Calif., October 5, 2026 - 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.

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. The FDA has agreed to review the relevant information on a timeline that would allow Benitec to confirm the final pivotal study design in 1Q 2027.

Timelines for the BB-301 pivotal study remain on track. Patient enrollment is planned to begin in mid-2027.

"We greatly appreciate the collaboration from the FDA and are encouraged by the constructive nature of our discussions" said Jerel A. Banks, M.D., Ph.D., 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. 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. 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."

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.

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. BB-301 is administered locally, in a one-time procedure, to the muscles in the throat that drive the swallowing process, an approach designed to maximize local benefit and minimize systemic exposure. We believe the silence and replace mechanism of BB-301 is uniquely positioned for the treatment of OPMD by halting mutant PABPN1 expression while simultaneously 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 and is currently being evaluated in a Phase 1b/2a, first-in-human, open-label dose escalation study to evaluate the safety and clinical activity of intramuscular doses of BB-301 administered to subjects with OPMD (NCT06185673).

About Benitec Biopharma Inc.

Benitec Biopharma Inc. (NASDAQ: BNTC) is a clinical-stage biotechnology company developing disease-modifying genetic medicines designed to improve the lives of people with life-threatening, genetically defined diseases. The company's proprietary "Silence and Replace" DNA-directed RNA interference (ddRNAi) platform combines RNA interference, or RNAi, with gene therapy to create medicines designed to facilitate sustained silencing of disease-causing genes and simultaneous delivery of replacement genes that restore normal cellular function following a single administration. The company's lead investigational candidate, BB-301, is the first and only disease-modifying genetic medicine in clinical development for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD)-related dysphagia.