Benitec Biopharma Inc.

Clinical Highlights The Company continues to advance BB-301 through clinical development in the ongoing Phase 1b/2a Clinical Study evaluating the safety and clinical efficacy of locally-administered BB-301 for the treatment of OPMD-related dysphagia.

Interim Clinical Study Update

• Clinically meaningful improvements (combined with a favorable safety profile) were observed across patient-reported swallowing symptom evaluations and X-ray based swallowing function evaluations, including improved throat closing ability, throat emptying ability, throat muscle relaxation, and functional swallowing ability

Regulatory Update

Upcoming Catalysts

• Presentation of interim clinical results from the Phase 1b/2a study at the ESGCT Annual Congress in Hamburg, Germany, October 27-30, 2026.

• Benitec anticipates initiating the BB-301 pivotal trial in mid-2027.

Financial Highlights

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 slowing or 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.