HAYWARD, Calif. — 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 announced the acceptance of a late-breaking abstract for the BB-301 Phase 1b/2a Clinical Treatment Study ongoing in Oculopharyngeal Muscular Dystrophy Patients (OPMD) with moderate dysphagia. Interim clinical study results for Patients enrolled into Cohort 1 and Cohort 2 will be discussed in a poster presentation at the Muscular Dystrophy Association Clinical and Scientific Conference, being held in Orlando, Florida on March 9, 2026.
“We are pleased to present long-term clinical study results for Patients treated with BB-301 in Cohort 1 and interim clinical study results for the first Patient treated with BB-301 in Cohort 2 at the Muscular Dystrophy Association Clinical & Scientific Conference,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “We are grateful to the OPMD community and investigators, and we look forward to providing updates on the ongoing clinical study and future discussions with the FDA as we work toward confirming the pivotal study path for BB-301.”
Late-Breaking Poster Presentation:
An interim clinical study update for the Phase 1b/2a Clinical Treatment Study of BB-301 in OPMD subjects with moderate dysphagia will be provided in a late-breaking poster presentation, (poster number 501 LB) entitled “Durable Responses to Low-Dose BB-301 in Oculopharyngeal Muscular Dystrophy at 12- and 24-months and Improved Depth of Response to High-Dose BB-301” during poster sessions from 10:15-10:45 am, 12:00-1:30 pm, 3:30-4:00 pm and 6:00-8:00 pm Eastern Time on March 9th in the Exhibit Hall at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference.
About BB-301
BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against mutant PABPN1 (the causative gene for OPMD). The two siRNAs are modeled into microRNA backbones to silence expression of faulty mutant PABPN1, while allowing expression of the codon-optimized PABPN1 to replace the mutant with a functional version of the protein. We believe the silence and replace mechanism of BB-301 is uniquely positioned for the treatment of OPMD by halting mutant expression while providing a functional replacement protein. BB-301 has received Orphan Drug Designation from the EMA and Orphan Drug and Fast Track Designations from the FDA.
About Benitec Biopharma Inc.
Benitec Biopharma Inc. (“Benitec” or the “Company”) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary “Silence and Replace” DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview of the Company can be found on Benitec’s website at www.benitec.com.
Investor Relations Contact
Irina Koffler
LifeSci Advisors, LLC
(917) 734-7387
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