Benitec Biopharma Releases Second Quarter 2026 Financial Results and Provides Operational Update

Core Insights - Benitec Biopharma Inc. announced positive results from the BB-301 Phase 1b/2a treatment study, with all four patients in Cohort 1 demonstrating durable responses at the 12-month follow-up [1][4] - The company plans to engage with the FDA in mid-2026 to confirm the pivotal study design for BB-301, which has received Fast Track and Orphan Drug Designations [2][8] - Financial results for the second fiscal quarter ended December 31, 2025, showed total expenses of $13.4 million, an increase from $10.8 million in the same quarter of the previous year [9][10] Clinical Development - All four patients in Cohort 1 were formal responders to BB-301, indicating a durable response at the 12-month follow-up [4] - Patient 1 from Cohort 1 showed continued disease-modifying effects at the 24-month follow-up, with improvements in post-swallow residue and dysphagic symptom burden [6] - Enrollment for Cohort 2 is ongoing, with the first patient treated with a higher dose in Q4 2025, and an interim results update is expected in mid-2026 [7] Responder Analysis - A Responder Analysis was developed to evaluate BB-301 efficacy, including multiple response categories such as patient-reported outcomes and videofluoroscopic swallowing study assessments [3] - The analysis framework combines results from various response categories to assess clinical benefits achieved by each patient [3][11] Financial Performance - Total expenses for the quarter ended December 31, 2025, were $13.4 million, with research and development expenses at $5.8 million, consistent with the previous year [9] - The net loss attributable to shareholders for the quarter was $11.8 million, or $(0.26) per share, unchanged from the previous year [10][14] - As of December 31, 2025, the company had $189 million in cash and cash equivalents [10][13] Corporate Updates - The company is focused on advancing its proprietary "Silence and Replace" DNA-directed RNA interference platform for the treatment of Oculopharyngeal Muscular Dystrophy [16][17] - Benitec has received Orphan Drug Designation from both the EMA and FDA for BB-301, highlighting its potential in treating rare diseases [8][16]