The BB-301 clinical development program will be conducted in the United States, Canada, and France, 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.

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

The submission package included: the NH Study Protocol, the subject Informed Consent Form, the patient-facing clinical documents and recruitment documents, the physician-facing clinical and laboratory manuals and recruitment documents.

Additionally, the primary clinical study site was formally qualified, the Principal Investigator and Sub-Investigators were identified at the clinical study site, the core VFSS site was selected and qualified, and the Central Reader for the VFSS data was selected.

The IRB for the lead clinical site in the United States approved the NH Study trial package in September 2022.

Following the favorable IRB decision, the Site Initiation Visit was completed in October 2022, and the screening of OPMD subjects for enrollment into the OPMD Natural History Study will begin at the lead clinical site in the United States in November 2022.

Planning for the formal submission of the comprehensive NH Study trial package to the Research Ethics Board (REB) for the lead clinical site in Canada is ongoing.

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.

In the Third Calendar Quarter 2022, Benitec submitted responses to the Central Ethics Committee (CEC) related to minor non-clinical queries.

In November 2022 two additional non-clinical queries have been received from the CEC regarding the General Data Protection Regulation (GDPR) in the European Union.

GDPR is a regulation that requires businesses to protect the personal data and privacy of European Union (EU) citizens.

GDPR became effective on May 25, 2018, and is applicable to organizations within the EU that use personal data, as well as international organizations that provide goods and services to individuals in the EU or monitor their behavior.

Approval of 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

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

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

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 approximately 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).

Assets

Current assets:

Cash and cash equivalents

Restricted cash

Trade and other receivables

Prepaid and other assets

Total current assets

Property and equipment, net

Deposits

Other assets

Right-of-use assets

Total assets

Liabilities and stockholders’ equity

Current liabilities:

Trade and other payables

Accrued employee benefits

Lease liabilities, current portion

Total current liabilities

Lease liabilities, less current portion

Total liabilities

Commitments and contingencies (Note 10)

Stockholders’ equity:

Common stock, $0.0001 par value-40,000,000 shares authorized; 25,809,533 shares and 8,171,690 shares issued and outstanding at September 30, 2022 and June 30, 2022, respectively

Additional paid-in capital

Accumulated deficit

Accumulated other comprehensive loss

Total stockholders’ equity

Total liabilities and stockholders’ equity

Revenue:

Licensing revenues from customers

Total revenues

Operating expenses

Research and development

General and administrative

Total operating expenses

Loss from operations

Other income (loss):

Foreign currency transaction loss

Interest expense, net

Unrealized gain on investment

Total other income (loss), net

Net loss

Other comprehensive income:

Unrealized foreign currency translation gain

Total other comprehensive income

Total comprehensive loss

Net loss

Net loss per share: basic and diluted

Weighted average number of shares outstanding: basic and diluted