Exhibit 99.1
Benitec Biopharma Releases Second Quarter 2024 Financial Results and Provides Operational Update
-First Subject Dosed with BB-301 in Phase 1b/2a Clinical Treatment Study (NCT06185673) in November
2023-
-Data Safety Monitoring Board Review for First Subject Completed After Day 28 Study Visit-
-Second Subject Scheduled to Receive BB-301 in February 2024-
-23 Subjects Enrolled into the Oculopharyngeal Muscular Dystrophy Natural History Study with
Multiple Subjects Eligible for entry into the Phase 1b/2a Clinical Treatment Study in 2024-
HAYWARD, Calif., February 13, 2024 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 financial results for its second fiscal quarter ended December 31, 2023. The Company has filed its quarterly report on Form 10-Q with the U.S. Securities and
Exchange Commission.
In 2023 we formally began the journey towards clinical validation of our Silence and Replace-based approach to the management
of genetically defined disorders, and our central goals remain focused on the improvement of the lives of patients suffering from Oculopharyngeal Muscular Dystrophy, said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive
Officer of Benitec. No dose-limiting toxicities have been observed in the first subject treated with BB-301, and the Data Safety Monitoring Board recommended the continuation of subject enrollment for
the Phase 1b/2a Clinical Treatment Study without any modifications to the study. We are grateful to have the opportunity to continue the clinical evaluation of BB-301, and we look forward to reporting interim
safety data and efficacy data in mid-2024.
Operational Updates
The key milestones related to the development of BB-301 for the treatment of Oculopharyngeal Muscular Dystrophy
(OPMD)-related Dysphagia, along with other corporate updates, are outlined below:
BB-301 Clinical
Development Program Overview:
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The BB-301 Phase 1b/2a clinical development program is currently underway
in the United States, and the primary elements of the program are summarized below: |
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The program comprises approximately 76 weeks of follow-up which we
anticipate will consist of: |
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The OPMD Natural History (NH) Study: 6-month pre-treatment observation periods for the evaluation of baseline disposition and natural history of OPMD-related dysphagia (swallowing impairment) in each study participant. |
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Dosing with BB-301:
1-day of BB-301 dosing to initiate participation in the Phase 1b/2a single-arm, open-label, sequential, dose-escalation
cohort study (NCT06185673). BB-301 will be delivered directly into the pharyngeal constrictor muscles of each study subject. |