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Benitec Biopharma Reports Positive Data from Two Subjects Treated with Low-Dose BB-301 in Phase 1b/2a Study Presented at 29th Annual Congress of the World Muscle Society
-Subject 1 and Subject 2 experienced durable, clinically meaningful improvements in swallowing at 9-months and 6-months post-BB-301 treatment, respectively,
About this update from Benitec Biopharma Inc.
[{"type":"text","content":"-Subject 1 and Subject 2 experienced durable, clinically meaningful improvements in swallowing at 9-months and 6-months post-BB-301 treatment, respectively, with Subject 2 achieving a Sydney Swallow Questionnaire Score Representative of Clinically Normal Swallowing- -Management plans to host a conference call on October 14 at 8:30 am EDT to discuss the interim results, details below- HAYWARD, Calif., Oct. 12, 2024 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “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 announces continued durable improvements in swallowing following administration of the low-dose of BB-301 in the study’s first two subjects treated in the BB-301 Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study (NCT06185673) in Oculopharyngeal Muscular Dystrophy (OPMD). Interim clinical study data were presented today in an oral late-breaking podium presentation at the 29th Annual Congress of the World Muscle Society, taking place in Prague, Czech Republic by the study’s principal investigator, Professor Milan R. Amin, M.D., Department of Otolaryngology-Head and Neck Surgery, New York University Grossman School of Medicine, Director, New York University Langone Voice Center. The interim clinical study update presented at the Annual Congress of the World Muscle Society detailed the 9-month (270-day) post-dose results for the first subject and the 6-month (180-day) post-dose results for the second subject, both of whom have been safely treated with BB-301. Key efficacy endpoints presented included videofluoroscopic swallowing study (“VFSS”) assessments of Total Pharyngeal Residue (“TPR”, i.e., the amount of solid food or liquid material remaining in the pharynx after the first swallow) and the Subject-Reported Outcome Instrument (i.e., the Sydney Swallow Questionnaire or “SSQ”). The post-dose results were compared to the average pre-dose results for each subject as evaluated during the five clinical assessment visits conducted during their enrollment in the Benitec-sponsored OPMD Natural History (NH) Study. The post-dose results for each subject were also compared to the pre-dose result derived from the final pre-...