The BB-301 clinical development program will be conducted in France, Canada, and the United States, and the primary elements of the clinical development plan are summarized below.

The clinical development plan will begin in 2022 and comprise approximately 76 weeks of follow-up:

6-month pre-treatment observation periods for the evaluation of baseline disposition and natural history of OPMD-derived dysphagia (swallowing impairment) in each study participant.

1-day of BB-301 dosing to initiate participation in the Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study.

52-weeks of post-dosing follow-up for conclusive evaluation of the primary and secondary endpoints of the Phase 1b/2a BB-301 treatment study.

The OPMD Natural History (NH) Study (i.e., the 6-month pre-treatment observation period) will facilitate the characterization of OPMD patient disposition at baseline and assess subsequent rates of progression of dysphagia via the use of the following quantitative radiographic measures (i.e., videofluoroscopic swallowing studies or “VFSS”), with the VFSS outlined below collectively providing objective assessments of global swallowing function and the function of the pharyngeal constrictor muscles (i.e., the muscles whose functional deterioration drives disease progression in OPMD):

Total Pharyngeal Residue %(C2-4)²

Pharyngeal Area at Maximum Constriction (PhAMPC)

Dynamic Imaging Grade of Swallowing Toxicity Scale (DIGEST)

Vallecular Residue %(C2-4)², Pyriform Sinus Residue %(C2-4)², and Other Pharyngeal Residue %(C2-4)²

Normalized Residue Ratio Scale (NRRS_v, NRRS_p)

Pharyngeal Construction Ratio (PCR)

The NH study will also employ clinical measures of global swallowing capacity and oropharyngeal dysphagia, along with two distinct patient-reported outcome instruments targeting the assessment of oropharyngeal dysphagia.

Upon the achievement of 6-months of follow-up in the NH Study, participants will be eligible for enrollment onto the BB-301 Phase 1b/2a treatment study.

BB-301 Phase 1b/2a Treatment Study:

This first-in-human (FIH) study will be a Phase 1b/2a, open-label, dose-escalation study to evaluate the safety and clinical activity of intramuscular doses of BB-301 administered to subjects with OPMD.

Upon rollover onto the Phase 1b/2a BB-301 treatment study, the follow-up of OPMD study participants will continue for 52 weeks.

The primary endpoints of the FIH study will be safety and tolerability, with secondary endpoints comprising quantitative radiographic measures of global swallowing function and pharyngeal constrictor muscle function (i.e., VFSS) as well as clinical assessments (all of which will be equivalent to those employed for the NH study). These endpoints will be evaluated during each 90-day period following BB-301 intramuscular injection (Day 1).

The natural history of dysphagia observed for each OPMD study participant, as characterized by the VFSS and clinical swallowing assessments carried out during the NH Study, will serve as the baseline for comparative assessment of safety and efficacy of BB-301 upon rollover from the NH Study onto the Treatment Study.

In the Second Calendar Quarter 2022, the NH Study Protocol, the Patient Informed Consent Form, the patient-facing documents and the physician-facing documents were completed; primary clinical study sites were formally qualified; Principal Investigators and Sub-Investigators were identified at the clinical study sites; the core VFSS site was selected and qualified; and the central reader for the VFSS data was selected.

Upon completion of these operational milestones, a formal submission was made to the Central Ethics Committee (CEC) in France.

In the Third Calendar Quarter 2022, Benitec received feedback from the CEC advising minor changes to the text of the Patient Informed Consent form (ICF).

The suggested updates were incorporated into the ICF, and the formal re-submission process for CEC final review has been completed.

Approval of the updated ICF, and the overall NH Study trial package, by the CEC allows for clinical study site activation and OPMD patient screening and enrollment to begin in France.

The final CEC decision is expected in October 2022 (allowing for NH Study screening visits and patient enrollment to begin in the Fourth Calendar Quarter 2022).

Formal submission of the comprehensive NH Study trial package to the Institutional Review Board (IRB) at the lead clinical study site in the United States was completed in August 2022.

Approval of the overall NH Study trial package by the IRB allows for clinical study site activation and OPMD patient screening and enrollment to begin in the United States.

The IRB decision is expected in October 2022 (allowing for NH Study screening visits and patient enrollment to begin in the Fourth Calendar Quarter 2022).

Benitec plans to complete the formal submission of the comprehensive NH Study trial package to the Research Ethics Board (REB) for the lead clinical site in Canada in the Fourth Calendar Quarter 2022.

Approval of the overall NH Study trial package by the REB allows for clinical study site activation and OPMD patient screening and enrollment to begin in Canada.

Clinical Trial Application (CTA) and Investigational New Drug (IND) filings are anticipated in the First Calendar Quarter 2023.

CTA and IND filings are required to initiate the BB-301 Phase 1b/2a Treatment Study in France, Canada, and the United States.

The first NH Study subject is anticipated to be eligible for BB-301 administration in the Second Calendar Quarter 2023 (following 6-months of NH Study enrollment and follow-up and final confirmation of eligibility for the BB-301 Phase 1b/2a Treatment Study).

Interim safety and efficacy data for subjects enrolled onto the BB-301 Phase 1b/2a Treatment Study are anticipated to become available for disclosure every 90-days following BB-301 administration.

In preclinical proof-of-concept studies for BB-301, the OPMD disease phenotype was reversed at 14 weeks (i.e., approximately 90-days) after the administration of BB-301 (with the strength of BB-301 injected muscles being restored in the diseased animals and robust evidence of anatomical and microscopic improvement of the BB-301 injected muscle tissues being observed).

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