HAYWARD, Calif. - Benitec Biopharma Inc. (NASDAQ: BNTC), a biotechnology company focusing on gene therapy, reported continued positive effects from its BB-301 therapy in a patient with Oculopharyngeal Muscular Dystrophy (OPMD). The company's recent update highlighted sustained improvements in swallowing function at the 180-day mark post-treatment.
OPMD is a rare genetic disorder characterized by progressive muscle weakness, leading to difficulties in swallowing (dysphagia) and other symptoms. Benitec's BB-301 therapy has been granted Orphan Drug designation by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
The interim clinical study results showed that the total amount of food and liquid residue remaining in the patient's throat after swallowing, known as Total Pharyngeal Residue (TPR), was significantly reduced after receiving a low dose of BB-301. This reduction was observed across three of the four food types used in the study, with the fourth food type showing similar results to the lowest observed pre-treatment values.
In addition to the videofluoroscopic swallowing study (VFSS) assessments, the patient-reported outcome measure, Sydney Swallow Questionnaire (SSQ), also indicated continued symptom reduction. The SSQ scores, which reflect the patient's subjective experience of swallowing difficulties, were lower at the 180-day post-treatment timepoint than at any time during the pre-treatment observation period.
This data is consistent with the 90-day post-treatment results previously reported and suggests a durable response to BB-301 therapy. No Serious Adverse Events (SAEs) have been reported for the two subjects treated with the therapy to date, with only transient Grade 2 Gastroesophageal Reflux Disease (GERD) noted.
Benitec's Executive Chairman and CEO, Jerel A. Banks, M.D., Ph.D., expressed optimism about the findings, emphasizing the importance of reducing pharyngeal residue to improve swallowing and reduce the risk of aspiration. The company is preparing to dose a third subject and plans to present further analyses at medical conferences and webcasts in early 2025.
BB-301 utilizes a "Silence and Replace" mechanism, aiming to halt the expression of the mutant PABPN1 gene responsible for OPMD while providing a functional replacement protein. This therapeutic approach is being developed as a potential long-term treatment for the disease.
The information in this article is based on a press release statement from Benitec Biopharma Inc.
In other recent news, Benitec Biopharma reported the appointment of Kishen Mehta, a portfolio manager at Suvretta Capital, to its Board of Directors following a $40 million private investment by Suvretta Capital.
Analysts have expressed confidence in the company's prospects. Piper Sandler initiated coverage on Benitec BioPharma, assigning an Overweight rating, while JMP Securities maintained a Market Outperform rating and increased the price target from $10.00 to $16.00, both citing the success of BB-301.
InvestingPro Insights
Benitec Biopharma Inc. (NASDAQ: BNTC) continues to make strides in the biotechnology sector with its gene therapy advancements. As the company reports positive clinical trial results, investors and stakeholders are keeping a close eye on the company's financial health and market performance. Here are some key insights based on real-time data and InvestingPro Tips:
InvestingPro Data indicates that Benitec Biopharma has a market capitalization of $88.71 million, reflecting its current valuation in the market. Despite the company's innovative strides, it holds a negative Price/Earnings (P/E) ratio of -0.78, suggesting that investors are awaiting profitability. Additionally, the company's Price/Book (P/B) ratio stands at 7.71, which may indicate a premium valuation compared to its book value.
Among the InvestingPro Tips, two particularly stand out for Benitec Biopharma. Firstly, the company is noted to be holding more cash than debt on its balance sheet, which is a positive sign of financial stability and potential for future investments in research and development. Secondly, analysts do not anticipate the company to be profitable this year, which aligns with the negative P/E ratio observed.
These financial metrics are crucial for investors who are considering the long-term potential of Benitec Biopharma. The company's research progress, particularly with BB-301 therapy, could be a significant driver for future growth, but current financial health and market performance are equally important to assess the investment risk.
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