Benitec Completes High-Dose Cohort and Holds FDA Pivotal Meeting
Cash and cash equivalents stood at $180.0 million as of June 30, 2026, as the company planned a BB-301 pivotal trial in mid-2027.
Benitec Biopharma Inc. (BNTC), a clinical-stage biotechnology company developing genetic medicines for genetically defined diseases, completed enrollment of Cohort 2 in its BB-301 Phase 1b/2a study and held a Type C meeting with the U.S. Food and Drug Administration on pivotal study design in the third quarter of 2026.
All six Cohort 1 patients, who received a low dose of BB-301, finished the full 12-month post-treatment follow-up period. Cohort 2, the high-dose group, is fully enrolled, with all three patients safely treated. Two months earlier, only the first two Cohort 2 patients had been treated. 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 throat closing, throat emptying, throat muscle relaxation and functional swallowing ability.
The Type C meeting, held in the third quarter of 2026, was intended to discuss the BB-301 pivotal study design. Meeting minutes are anticipated in October. In May, the company remained on track to engage the FDA mid-year to formalize that design. Benitec anticipates initiating the pivotal trial in mid-2027.
Interim clinical results for Cohort 1 and Cohort 2 will be presented at the 33rd Annual Congress of the European Society of Gene and Cell Therapy in Hamburg, Germany, October 27-30, 2026. Interim results were previously presented orally at the American Society of Gene and Cell Therapy annual meeting on May 15, 2026.
BB-301 is a modified AAV9 vector expressing a construct that co-expresses codon-optimized PABPN1 and two small inhibitory RNAs against mutant PABPN1, the causative gene for oculopharyngeal muscular dystrophy. It is administered locally, in a one-time procedure, to throat muscles. The drug has Orphan Drug Designation from the EMA and Orphan Drug and Fast Track Designations from the FDA.
For the fiscal year ended June 30, 2026, the company reported total expenses of $51.2 million, compared with $41.8 million for the year ended June 30, 2025. Research and development expenses were $23.4 million, up from $18.3 million, and were primarily related to ongoing clinical development of BB-301. The increase in research and development expenses primarily reflected higher share-based compensation of $6.3 million and increased payroll of $2.2 million, partially offset by a reduction in contract manufacturing activity of $3.8 million. General and administrative expenses were $27.8 million, compared with $23.4 million a year earlier, an increase primarily driven by higher share-based compensation of $2.7 million and an increase in payroll of $0.8 million.
The net loss from operations for the year ended June 30, 2026, was $51.2 million, compared with $41.8 million for the prior year. Net loss attributable to shareholders was $45.5 million, or $0.98 a share, compared with $37.9 million, or $1.05 a share, for the year ended June 30, 2025. Weighted average shares outstanding were 46.6 million, compared with 36.2 million a year earlier. Interest income, net, was $5.6 million, compared with $3.3 million.
Cash and cash equivalents were $180.0 million as of June 30, 2026. That compared with approximately $184.8 million as of March 31, 2026. Common shares outstanding were 34.4 million at June 30, 2026, compared with 26.3 million a year earlier.
Executive Chairman and Chief Executive Officer Jerel A. Banks said the year included completion of Cohort 1 follow-up, full enrollment of Cohort 2, and continued regulatory interactions to prepare BB-301 for a pivotal trial. Benitec looks forward to building on that momentum as it works toward the planned initiation of the pivotal study in mid-2027.