PABPN1

Benitec Biopharma Reports Positive Interim Clinical Trial Data for First OPMD Subject Treated with BB-301 in Phase 1b/2a Study

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星期四, 四月 18, 2024
FDA, Dysphagia, Mutation, EDT, Nature, COMP, Oculopharyngeal muscular dystrophy, Hope, Food, News, Death, Yogurt, Pharmacotherapy, Observation, PABPN1, Disease, OPMD, Subject, Ptosis, Patient, EMA, Caregiver, Committee, Clinical trial, Trial of the century, Orphan drug, European Medicines Agency, Pharmaceutical industry

HAYWARD, Calif., April 18, 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 positive interim clinical data from the 90-day timepoint following the administration of BB-301 to the study’s first subject (Subject 1) treated in the BB-301 Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study (NCT06185673) in Oculopharyngeal Muscular Dystrophy (OPMD). BB-301 has been granted Orphan Drug designation by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP).

Key Points: 
  • “To date, no clinical studies have systematically demonstrated a clinical improvement in OPMD patients across both objective and subjective measures of swallowing.
  • We are, therefore, pleased to report positive interim clinical data from multiple radiographic measures as well as subject-reported outcome measures from the first subject treated with BB-301,” said Jerel A.
  • Videofluoroscopic swallowing studies represent the gold standard analytical method for the quantitative assessment of dysphagia (swallowing difficulty) in the clinical setting.
  • The event replay will be placed on the News & Events tab on the Investor page of the Benitec website.