Benitec Biopharma (BNTC) Corporate presentation summary

Disease overview and unmet need

• Oculopharyngeal muscular dystrophy (OPMD) is a rare, progressive, autosomal-dominant muscle disorder with no approved therapies, affecting about 15,000 patients in North America, Europe, and Israel.

• OPMD typically presents in the 40s–50s with ptosis, dysphagia, and progressive muscle weakness, leading to severe complications such as malnutrition and aspiration pneumonia.

• Current management strategies, including dietary modifications and surgical interventions, do not halt disease progression.

Therapeutic platform and mechanism

• The ddRNAi "Silence and Replace" platform enables permanent silencing of mutated genes and replacement with wildtype genes in target tissues from a single administration.

• BB-301 is a gene therapy designed to treat OPMD by inhibiting production of the mutant PABPN1 protein and delivering a functional version, aiming to restore muscle function.

Clinical development and study design

• The BB-301 Phase 1b/2a open-label dose escalation study (NCT06185673) enrolled up to 30 subjects, with six treated as of January 2024 and no treatment-related serious adverse events observed.

• Efficacy endpoints include the Sydney Swallow Questionnaire (SSQ) and Videofluoroscopic Swallowing Studies (VFSS), assessed at multiple time points post-treatment.

• Key assessments focus on subject-reported dysphagia severity and objective measures of swallowing efficiency and effectiveness.