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HAYWARD, Calif. - Benitec Biopharma Inc. (NASDAQ: BNTC), a clinical-stage biotechnology company with a market capitalization of $225 million, announced today that it will present interim data from its ongoing Phase 1b/2a clinical study of BB-301 for treating Oculopharyngeal Muscular Dystrophy (OPMD) at the upcoming Muscular Dystrophy Association Clinical and Scientific Conference. According to InvestingPro data, the company's stock has shown remarkable strength with a 246% return over the past year, despite recent market volatility. The oral abstract, which will provide clinical updates on the first three subjects treated with BB-301, is scheduled for March 19, 2025.
BB-301 is a novel gene therapy candidate utilizing a "Silence and Replace" strategy through DNA-directed RNA interference (ddRNAi). This approach aims to silence the mutant gene responsible for OPMD while simultaneously replacing it with a functional version. The interim clinical data previously shared in October 2024 showed that the first two subjects treated with BB-301 experienced durable and clinically meaningful improvements in swallowing function, a severe complication associated with OPMD. InvestingPro analysis indicates the company maintains a strong financial position with more cash than debt on its balance sheet, which is crucial for supporting ongoing clinical development.
The company's Executive Chairman and CEO, Dr. Jerel A. Banks, expressed optimism about the potential benefits of BB-301 for OPMD patients, emphasizing the goal of developing a safe and effective treatment to reduce the burden of dysphagia. Further updates on the clinical study are expected in the fourth quarter of this year.
Benitec's proprietary platform combines RNA interference with gene therapy to create treatments that aim for sustained silencing of disease-causing genes and delivery of replacement genes with a single therapeutic construct. The company focuses on developing therapies for chronic and life-threatening conditions.
The forward-looking statements in the press release are based on Benitec's current expectations and are subject to risks and uncertainties that may cause actual results to differ. These include the success and timing of clinical trials, regulatory filings, and potential commercialization of product candidates. While analysts have set price targets ranging from $17 to $35, InvestingPro subscribers can access comprehensive analysis including 8 additional ProTips, detailed financial metrics, and exclusive Fair Value calculations through the platform's Pro Research Report, available for over 1,400 US stocks.
The information for this article is based on a press release statement from Benitec Biopharma Inc.
In other recent news, Benitec BioPharma has been the subject of several significant developments. H.C. Wainwright initiated coverage on the company, giving it a Buy rating and a price target of $28.00, citing the potential of its gene therapy, particularly the lead candidate, BB-301, for treating oculopharyngeal muscular dystrophy (OPMD). Baird also initiated coverage on Benitec, setting a price target of $30.00, highlighting the potential of BB-301.
Simultaneously, Benitec has announced substantial changes to its executive team, including the appointment of a new Chief Operating Officer and adjustments to the roles and compensation of existing officers. The company's board also approved the issuance of up to 5 million shares of preferred stock, potentially providing more flexibility in financing its operations and pursuing growth opportunities.
These recent developments follow the company's 2024 Annual Meeting of Stockholders, where several key proposals were approved, including the election of directors and the authorization of preferred stock issuance. These updates, along with the analysts' positive outlook on Benitec's gene therapy, indicate a period of significant activity for the company.
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