–Positive interim clinical study results from the Phase 1b/2a trial of BB-301, showed a 100% responder rate, with all six patients in Cohort 1 meeting the formal statistical criteria for response to BB-301–
–First patient of Cohort 2 successfully treated with BB-301 in Q4 of 2025–
–The Company raised approximately $100 million in an oversubscribed public offering of common stock, which is expected to fund advancement of the BB-301 Oculopharyngeal Muscular Dystrophy (OMPD) registrational program and associated regulatory filing activities–
HAYWARD, Calif., Nov. 14, 2025 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ:BNTC) ("Benitec" or the "Company"), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary "Silence and Replace" DNA-directed RNA interference ("ddRNAi") platform, today announced financial results for its first fiscal quarter ended September 30, 2025. The Company has filed its quarterly report on Form 10-Q for the quarter ended September 30, 2025 with the U.S. Securities and Exchange Commission (SEC).
"We are fortunate to have achieved several important milestones this year. We previously announced positive interim clinical study results from the Phase 1b/2a trial of BB-301, with a 100% responder rate to BB-301 in Cohort 1. BB-301 was also granted Fast Track Designation by the FDA, and we completed a significant capital raise to fund the advancement of BB-301, which has the potential to become the first approved therapy for the treatment of the central clinical symptom plaguing OPMD patients," said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. "We remain extremely grateful for the strong support from the OPMD community and their families, the clinical research community, and the investment community. We look forward to continued collaboration with the FDA in 2026 to advance the development of BB-301."
Operational Updates
The key milestones related to the development of BB-301 for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD)-related dysphagia, are outlined below:
On November 3rd, the Company announced positive interim clinical study results from its Phase 1b/2a trial of BB-301. Following administration of BB-301, Cohort 1 patients experienced significant and sustained improvements across multiple clinical measures of swallowing function. All 6 patients in Cohort 1 met the formal statistical criteria for response to BB-301, representing a 100% responder rate.
Following review of the positive interim clinical study results from the Phase 1b/2a trial of BB-301, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation (FTD) to BB-301 for the treatment of dysphagia associated with Oculopharyngeal Muscular Dystrophy.
Benitec also announced the first patient in Cohort 2 of the BB-301 Phase 1b/2a trial has been successfully treated with BB-301.
The Company hosted a webcast to discuss the positive interim clinical study results from its Phase 1b/2a trial of BB-301 on Monday November 3rd, and a replay of that event can be found here.
As announced on July 9th, in accordance with the protocol for the BB-301 Phase 1b/2a trial, a meeting of the independent data safety monitoring board (DSMB) was convened following the completion of the 28-day post BB-301 dosing visit for the sixth patient enrolled into Cohort 1. The DSMB recommended continuation of subject enrollment for the Phase 1b/2a Clinical Treatment Study.
Corporate Updates:
On November 3rd, Benitec announced the addition of Sharon Mates, Ph.D., to its Board of Directors. Dr. Mates served as the Chairman and Chief Executive Officer, and was Co-founder of Intra-Cellular Therapies, a mental health company, from June 2002 until it was acquired for $14.6 billion by Johnson & Johnson in 2025.
On November 5th, Benitec announced the pricing of its underwritten public offering of 5,930,000 shares of its common stock and a concurrent registered direct offering of 1,481,481 shares of its common stock with long-term investor Suvretta Capital. Each share of common stock was sold at an offering price of $13.50. In addition, the Company has granted the underwriters a 30-day option to purchase up to an additional 889,500 shares of common stock on the same terms and conditions. Benitec plans to use the net proceeds from this financing, together with existing cash on hand, to support the continued development and registration of its BB-301 candidate in OPMD, working capital and other general corporate purposes.
Financial Highlights
First Quarter 2026 Financial Results
Total Expenses for the quarter ended September 30, 2025, were $9.8 million compared to $5.8 million for the quarter ended September 30, 2024. The Company incurred $3.4 million of research and development expenses for the quarter ended September 30, 2025 compared to $3.6 million for the quarter ended September 30, 2024. Research and development expenses relate primarily to ongoing clinical development of BB-301 for the treatment of OPMD-related Dysphagia. General and administrative expenses were $6.4 million for the quarter ended September 30, 2025 compared to $2.2 million for the quarter ended September 30, 2024.
The total comprehensive loss from operations for the quarter ended September 30, 2025, was $8.9 million compared to a loss of $5.2 million for the quarter ended September 30, 2024. Net loss attributable to shareholders for the quarter ended September 30, 2025, was $9.0 million, or $(0.22) per basic and diluted share, compared to a net loss of $5.1 million, or $(0.18) per basic and diluted share for the quarter ended September 30, 2024. As of September 30, 2025, the Company had $94.5 million in cash and cash equivalents. On November 5, 2025, Benitec concluded an equity financing grossing approximately $100 million before deducting costs.
BENITEC BIOPHARMA INC.
Consolidated Balance Sheets
(in thousands, except par value and share amounts)
September 30,
June 30,
2025
2025
(Unaudited)
Assets
Current assets:
Cash and cash equivalents
$
94,479
$
97,744
Restricted Cash
113
113
Trade and other receivables
7
33
Prepaid and other assets
480
628
Total current assets
95,079
98,518
Property and equipment, net
118
131
Deposits
55
55
Other assets
20
28
Right-of-use assets
755
860
Total assets
$
96,027
