Benitec Biopharma (BNTC) Corporate presentation summary

Transformative approach for OPMD-related dysphagia

• BB-301 combines gene silencing and gene replacement in a single vector, targeting the root cause of OPMD-related dysphagia with a one-time intramuscular injection to the pharyngeal muscles.

• The therapy silences the mutant PABPN1 gene and delivers a functional gene, aiming for permanent disease modification and minimal systemic exposure.

• OPMD is a rare, progressive muscle disorder with no approved therapies, affecting about 15,000 patients in North America, Europe, and Israel.

• BB-301 is the only clinical-stage therapy in development for OPMD-related dysphagia, addressing a significant unmet need.

Clinical efficacy and safety results

• In Phase 1b/2a, 100% of Cohort 1 patients (n=6) met responder criteria, showing significant improvements in swallowing function and quality of life.

• Key efficacy endpoints included patient-reported outcomes, pharyngeal muscle function, swallowing efficiency, and functional swallowing capacity.

• Statistically significant improvements were observed in SSQ scores, PhAMPC, TPR, and NRRSv, with up to 69% decline in abnormal residue measures.

• No treatment-related severe adverse events or deaths were reported in Cohort 1.

• The first patient in Cohort 2 was successfully treated, and pivotal study planning is underway.

Regulatory milestones and market opportunity

• BB-301 received Fast Track and Orphan Drug Designations from the FDA and EMA, validating its clinical and regulatory differentiation.

• The proprietary responder analysis and natural history data informed pivotal study design and supported regulatory submissions.

• If approved, BB-301 could address up to 90% of the 15,000 OPMD patients, representing a high-value, durable rare disease market.

• Manufacturing capabilities include GMP-compliant, commercial-scale batches, and the company holds $188.6M in cash and equivalents as of December 2025.

• Plans are in place for a 2026 FDA meeting to confirm pivotal trial details.