Pabparna (BB-301) / Benitec BioPharma - LARVOL DELTA

Fast Track Designation was granted for BB-301 following FDA review of positive interim clinical study results and proprietary Responder Analysis planned for use in pivotal study for BB-301 (Benitec Press Release) - "BB-301 has also been granted Orphan Drug Designation from both FDA and EMA...All six patients enrolled into Cohort 1 met the formal statistical criteria for response to BB-301, representing a 100% response rate...First patient in Cohort 2 successfully treated with BB-301 in fourth quarter of 2025...Benitec plans to meet with the FDA in 2026 to confirm the BB-301 pivotal study design"

Fast track • New trial • Orphan drug • P1/2 data • Muscular Dystrophy

Benitec Biopharma…announced that it will provide an update on the six treated patients from Cohort 1 of its Phase 1b/2a Clinical Study of BB-301 being investigated for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD) (GlobeNewswire) - "Investor webcast to be held on Monday November 3, 2025 at 8:00 am EST."

P1/2 data • Muscular Dystrophy

Benitec Biopharma Reports Positive Interim Clinical Results for Three Subjects Treated with BB-301 in Phase 1b/2a Study to be Presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference (GlobeNewswire) - P1b/2a | N=30 | NCT06185673 | Sponsor: Benitec Biopharma, Inc. | "Benitec Biopharma Inc...today announces continued durable improvements in swallowing function and reductions in total dysphagic symptom burden following administration of the low-dose of BB-301 in the first three Subjects treated in the BB-301...Interim clinical study results will be presented today in an oral late-breaking podium presentation at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference...'We look forward to enrolling additional Subjects at the next, higher dose of BB-301 later this year.'...Three Subjects with distinct causes of their respective dysphagic symptom burdens were safely treated with BB-301 (1.2e13 vg/Subject) and experienced significant, clinically meaningful improvements in swallowing function...final Subject of Cohort 1 is anticipated to receive the low-dose of BB-301 in 2Q 2025."

P1/2 data • Trial status • Muscular Dystrophy

Benitec Biopharma Announces Acceptance of Late- Breaking Oral Abstract for the BB-301 Phase 1b/2a Clinical Study at the Muscular Dystrophy Association Clinical and Scientific Conference (GlobeNewswire) - "Benitec Biopharma Inc...today announced the acceptance of a late-breaking oral abstract for the BB-301 Phase 1b/2a Clinical Treatment Study ongoing in Subjects diagnosed with Oculopharyngeal Muscular Dystrophy (OPMD) with moderate dysphagia. Interim clinical study updates for the first three Subjects will be discussed in an oral presentation at the Muscular Dystrophy Association Clinical and Scientific Conference on March 19, 2025 at 1:15 pm Central Time...'Additional clinical updates for Subjects enrolled in the BB-301 Phase 1b/2a Clinical Treatment Study are planned for the fourth calendar quarter of this year.'"

Late-breaking abstract • P1/2 data • Muscular Dystrophy

Benitec Biopharma Releases Second Quarter 2025 Financial Results and Provides Operational Update (GlobeNewswire) - "Enrollment into the BB-301 Phase 1b/2a Clinical Treatment Study is Ongoing...The sixth Subject is expected to be treated with the low-dose of BB-301 in 2Q 2025. Benitec expects to begin treating a second cohort of OPMD Subjects with a higher dose of BB-301 later in the year."

Trial status • Muscular Dystrophy

A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia (clinicaltrials.gov) - P1/2 | N=30 | Recruiting | Sponsor: Benitec Biopharma, Inc.

New P1/2 trial • Gastrointestinal Disorder • Muscular Dystrophy

BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy. (PubMed, Mol Ther Nucleic Acids) - "A single intramuscular injection of BB-301 results in robust inhibition of mutant PABPN1 and concomitant replacement of the codon-optimized PABPN1 protein. The treatment restores muscle strength and muscle weight to wild-type levels as well as improving other physiological hallmarks of the disease in a mouse model of OPMD."

Journal • Gene Therapies • Muscular Dystrophy