Benitec Biopharma (BNTC) Corporate presentation summary

Disease overview and unmet need

• Oculopharyngeal muscular dystrophy (OPMD) is a rare, progressive, autosomal-dominant muscle disorder caused by a mutation in the PABPN1 gene, leading to severe dysphagia and life-threatening complications with no approved therapies.

• Dysphagia in OPMD results from weakened pharyngeal muscles, causing choking, malnutrition, aspiration pneumonia, and increased mortality risk.

BB-301 mechanism and administration

• BB-301 is a gene therapy using DNA-directed RNA interference (ddRNAi) to silence the mutant gene and replace it with a functional gene, aiming for a permanent solution with a single administration.

• Delivered via direct intramuscular injection to the pharyngeal constrictor muscles, BB-301 is designed to increase muscle mass and function, reducing dysphagia.

Clinical development and study design

• The clinical program includes a natural history study and a Phase 1b/2a open-label dose escalation study, with up to 30 patients across three doses.

• Primary endpoint is safety; secondary endpoints assess swallowing function using patient-reported outcomes and objective measures.

• No treatment-related severe adverse events have been reported to date.