Benitec Biopharma Inc.

Benitec Biopharma Announces Positive Interim Phase 1b/2a Results for High Dose BB-301 and Continued Durable Improvements for Low Dose BB-301 

Benitec reported positive interim Phase 1b/2a results for BB-301 in OPMD-related dysphagia, with 100% of the nine treated patients formal responders to therapy. High-dose Cohort 2 showed greater improvements than low-dose Cohort 1 at three months, including 100% vs 67% meeting response criteria for improved throat emptying, with no serious adverse events to date. The company plans to advance BB-301 into a pivotal study in mid-2027; BB-301 holds FDA Orphan Drug and Fast Track designations and EMA Orphan Drug Designation.

•12d ago

Benitec Biopharma Releases Full Year 2026 Financial Results and Provides Operational Update

Benitec Biopharma reported fiscal 2026 (ended June 30, 2026) results with a net loss of $45.5 million, or $0.98 per basic and diluted share, versus a $37.9 million loss, or $1.05 per share, in fiscal 2025. Total expenses rose to $51.2 million from $41.8 million, led by R&D of $23.4 million and G&A of $27.8 million tied to the BB-301 program. The company ended the year with $180.0 million in cash and cash equivalents, nearly double the $97.7 million held a year earlier, and describes itself as well-capitalized. On the clinical side, all six Cohort 1 (low-dose BB-301) patients completed the full 12-month follow-up and Cohort 2 (high dose) is fully enrolled with all three patients safely treated, with clinically meaningful swallowing-function improvements and a favorable safety profile reported in the ongoing Phase 1b/2a OPMD dysphagia study. A Type C meeting with the FDA on the BB-301 pivotal study design was held in 3Q2026 with minutes expected in October; interim data will be presented at the ESGCT congress in Hamburg on October 27-30, 2026, and the pivotal trial is planned to begin in mid-2027.

•1mo ago

Benitec Biopharma Announces Oral Presentation of Interim Phase 1b/2a Clinical Study Results for High Dose BB-301 and Continued Durable Improvements for Low Dose BB-301 at the 2026 American Society of Gene and Cell Therapy Annual Meeting

Benitec Biopharma announced that interim Phase 1b/2a clinical results for high dose and low dose BB-301 have been accepted for oral presentation at the ASGCT Annual Meeting. The data set includes 12-month follow-up for four Cohort 1 completers, 24-month results for the first Cohort 1 patient, and interim results for the first Cohort 2 patient, demonstrating continued durable improvements. BB-301 utilizes the company's proprietary Silence and Replace platform and holds Orphan Drug and Fast Track designations for OPMD, a rare disease with no approved therapies affecting approximately 15,000 patients in North America, Europe, and Israel.

•6mo ago

Seaport Therapeutics Adds Intra-Cellular Therapies Founder and CEO, Dr. Sharon Mates, to its Board of Directors

Seaport Therapeutics appointed Dr. Sharon Mates, the founder and former CEO of Intra-Cellular Therapies, to its Board of Directors. Dr. Mates brings decades of experience, having guided Intra-Cellular through the commercialization of CAPLYTA and its eventual $14.6 billion acquisition by Johnson & Johnson. Seaport CEO Daphne Zohar emphasized that Dr. Mates' strategic depth and industry relationships will support the company's mission to develop neuropsychiatric medicines.

•6mo ago