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HAYWARD, Calif. - Benitec Biopharma Inc. (NASDAQ: BNTC), a biotechnology company focused on gene therapy with a market capitalization of $364 million and an impressive 206% return over the past year, has reported sustained improvements in swallowing function in subjects treated with its investigational gene therapy BB-301. According to InvestingPro data, the company maintains a strong financial health score of 2.62, rated as "GOOD," with more cash than debt on its balance sheet. The findings come from a Phase 1b/2a study for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD), a rare degenerative muscle disorder with no effective drug therapy currently available.
The interim results, presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference in Dallas, Texas, showed that the first two subjects in the study maintained clinically significant improvements in swallowing function 12 months after treatment. A third subject also showed significant improvements three months post-treatment. These results were measured by reductions in total dysphagic symptom burden, as assessed by the Sydney Swallow Questionnaire (SSQ) and videofluoroscopic swallowing study (VFSS).
Subject 1, who had issues with inefficient swallowing, showed significant reductions in post-swallow residue across various food and liquid consistencies. Subject 2, who had ineffective swallowing, exhibited a significant reduction in the frequency of pathologic sequential swallows, achieving a clinically normal swallowing profile. Subject 3, also with ineffective swallowing, experienced complete resolution of pathologic sequential swallows, with an SSQ score indicative of normal swallowing function.
The study is designed to assess the safety and clinical activity of BB-301, a novel gene therapy, by escalating doses in a single-arm, open-label format. To date, five subjects have been treated with a low dose of BB-301, and a sixth is expected to be treated in the second quarter of 2025. No severe adverse events have been observed. Wall Street analysts maintain a positive outlook on the company, with price targets ranging from $17 to $35 per share. Get deeper insights into BNTC’s valuation and 11 additional key ProTips with InvestingPro.
OPMD is characterized by progressive dysphagia, which affects 97% of patients and can lead to choking, malnutrition, and potentially fatal aspiration pneumonia. The disease is caused by a mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene.
Benitec’s proprietary DNA-directed RNA interference (ddRNAi) platform is being used to develop BB-301 and other therapies for chronic and life-threatening conditions. Further details of the study will be available on the company’s website following the conference presentation.
This article is based on a press release statement from Benitec Biopharma Inc. The company’s stock currently trades near its 52-week high of $15.97, reflecting strong investor confidence in its clinical progress. For comprehensive analysis including Fair Value estimates and detailed financial metrics, access the full Pro Research Report available exclusively on InvestingPro.
In other recent news, Benitec BioPharma has been actively advancing its clinical trials and research efforts. H.C. Wainwright analysts have maintained a Buy rating with a $28.00 price target, reflecting confidence in the ongoing Phase 1b/2a trial of Benitec’s lead clinical candidate, BB-301. The trial has achieved significant milestones with the treatment of additional patients, showing promising results without severe adverse events. Meanwhile, Citizens JMP reiterated its Market Outperform rating and an $18.00 price target, highlighting the progress in Benitec’s FiH OPMD trial and its potential to substantiate the company’s "silence and replace" strategy.
JMP analysts also maintained their Market Outperform rating and $18.00 price target, in anticipation of Benitec’s presentation at the upcoming Muscular Dystrophy Association Conference. The company plans to present interim data from its Phase 1b/2a clinical study of BB-301, which has shown clinically meaningful improvements in swallowing function for patients with Oculopharyngeal Muscular Dystrophy. Benitec’s innovative approach combines RNA interference with gene therapy, aiming for sustained gene silencing and replacement. These developments are critical as Benitec continues to navigate its clinical trials and potential future commercialization.
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