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HAYWARD, Calif. - Benitec Biopharma Inc. (NASDAQ:BNTC), a clinical-stage biotechnology company with a market capitalization of $331 million, announced Wednesday that its Phase 1b/2a clinical trial for BB-301, a gene therapy treatment for Oculopharyngeal Muscular Dystrophy (OPMD), has received approval to continue enrollment following a safety review. According to InvestingPro data, the company maintains a strong liquidity position with current assets significantly exceeding short-term obligations.
The company completed treatment of the sixth and final subject in Cohort 1 with the low dose of BB-301 in April. An independent Data Safety Monitoring Board (DSMB) reviewed safety data from all six subjects and recommended continuation of the trial, according to the press release statement.
Following this positive recommendation, Benitec plans to begin enrollment for Cohort 2 in the fourth quarter of 2025.
"We are extremely thankful and humbled to have the opportunity to continue our collaborative development work for BB-301 with the OPMD patient community and the OPMD clinical community," said Jerel A. Banks, Executive Chairman and Chief Executive Officer of Benitec. The company’s conservative financial approach is reflected in its minimal debt-to-equity ratio of 0.01, as reported by InvestingPro.
BB-301 uses Benitec’s proprietary "Silence and Replace" DNA-directed RNA interference (ddRNAi) platform. The treatment is designed to silence the expression of mutant PABPN1 protein, which causes OPMD, while simultaneously delivering a functional replacement protein.
The company noted that the direct intramuscular delivery method for BB-301 enables lower dosing compared to systemic administration routes used in other gene therapy programs.
Benitec plans to provide additional clinical updates on subjects enrolled in Cohort 1 in the fourth quarter of this year.
In other recent news, Benitec BioPharma has been the focus of several analyst updates and clinical developments. TD Cowen initiated coverage on Benitec BioPharma with a Buy rating, highlighting the company’s innovative DNA-directed "Silence and Replace" platform and the promising initial clinical data from its lead asset, BB-301, for oculopharyngeal muscular dystrophy (OPMD). Analysts from Citizens JMP maintained a Market Outperform rating and a $20 price target, expressing confidence in the company’s ongoing research following positive clinical updates. JMP Securities also raised their price target for Benitec BioPharma to $20, citing increased confidence in the BB-301 program after observing significant clinical improvements in patients.
Benitec BioPharma has successfully dosed all six patients in the low-dose cohort of its study without safety concerns, and an update on this cohort is expected in the fourth quarter of 2025. The company is preparing to start enrollment for the high-dose cohort of BB-301 later in 2025, with the safety of this treatment being a critical factor. Analysts have noted the importance of Benitec’s efforts to identify patient subgroups within the OPMD community, which could aid in future discussions with the FDA. These developments mark significant progress in Benitec’s gene therapy program.
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