Benitec Biopharma Inc.

Previous wording

You should read the following discussion and analysis of financial condition and operating results together with our consolidated financial statements and the related notes and other financial information included elsewhere in this document.

Current wording

You should read the following discussion and analysis of financial condition and operating results together with our consolidated financial statements and the related notes and other financial information included elsewhere in this document. See also “Special Note Regarding Forward-Looking Statements” immediately prior to Part I, Item 1 in this Quarterly Report on Form 10-Q.

Previous wording

BB-301 is a silence and replace-based genetic medicine currently under development by Benitec. BB-301 uses DNA-directed RNA interference (ddRNAi) to simultaneously silence the mutant gene and replace it with a functional gene, potentially providing a permanent solution with a single administration. This fundamental therapeutic approach to disease management is called “silence and replace.” The silence and replace mechanism offers the potential to restore the normative physiology of diseased cells and tissues and to improve treatment outcomes for patients suffering from the chronic, and potentially fatal, effects of OPMD. BB-301 has been granted Orphan Drug Designation in the United States and the European Union.

Current wording

BB-301 is a silence and replace-based genetic medicine currently under development by Benitec. BB-301 uses DNA-directed RNA interference ("ddRNAi") to simultaneously silence the mutant gene and replace it with a functional gene, potentially providing a permanent solution with a single administration. This fundamental therapeutic approach to disease management is called “silence and replace.” The silence and replace mechanism offers the potential to restore the normative physiology of diseased cells and tissues and to improve treatment outcomes for patients suffering from the chronic, and potentially fatal, effects of OPMD. BB-301 has been granted Orphan Drug Designation in the European Union and Orphan Drug Designation and Fast Track Designation in the United States.

Previous wording

ddRNAi is designed to produce permanent silencing of disease-causing genes, by combining RNA interference, or RNAi, with viral delivery agents typically associated with the field of gene therapy (i.e., viral vectors). Modified AAV vectors are employed to deliver genetic constructs which encode short hairpin RNAs that are, then, serially expressed and processed to produce siRNA molecules within the transduced cell for the duration of the life of the target cell. These newly introduced siRNA molecules drive permanent silencing of the expression of the disease-causing gene. The silence and replace approach further bolsters the biological benefits of permanent silencing of disease-causing genes by incorporating multifunctional genetic constructs within the modified AAV vectors to create an AAV-based gene therapy agent that is designed to silence the expression of disease-causing genes (to slow, or halt, the underlying mechanism of disease progression) and to simultaneously replace the mutant genes with normal, functional genes (to drive restoration of function in diseased cells). This fundamentally distinct therapeutic approach to disease management offers the potential to restore the underlying physiology of the treated tissues and, in the process, improve treatment outcomes for patients suffering from the chronic and, potentially, fatal effects of diseases like Oculopharyngeal Muscular Dystrophy (OPMD).

Current wording

ddRNAi is designed to produce permanent silencing of disease-causing genes, by combining RNA interference, or RNAi, with viral delivery agents typically associated with the field of gene therapy (i.e., viral vectors). Modified adeno-associated viral (“AAV”) vectors are employed to deliver genetic constructs which encode short hairpin RNAs that are, then, serially expressed and processed to produce siRNA molecules within the transduced cell for the duration of the life of the target cell. These newly introduced siRNA molecules drive permanent silencing of the expression of the disease-causing gene. The silence and replace approach further bolsters the biological benefits of permanent silencing of disease-causing genes by incorporating multifunctional genetic constructs within the modified AAV vectors to create an AAV-based gene therapy agent that is designed to silence the expression of disease-causing genes (to slow, or halt, the underlying mechanism of disease progression) and to simultaneously replace the mutant genes with normal, functional genes (to drive restoration of function in diseased cells). This fundamentally distinct therapeutic approach to disease management offers the potential to restore the underlying physiology of the treated tissues and, in the process, improve treatment outcomes for patients suffering from the chronic and, potentially, fatal effects of diseases like OPMD.

Previous wording

The mutation of a single gene can cause a chronic disease via the resulting intracellular production of a disease-causing protein (i.e., an abnormal form of the protein of interest), and many chronic and/or fatal disorders are known to result from the inappropriate expression of a single gene or multiple genes. In some cases, genetic disorders of this type can be treated exclusively by “silencing” the intracellular production of the disease-causing protein through well-validated biological approaches like RNA interference (“RNAi”). RNAi employs small nucleic acid molecules to activate an intracellular enzyme complex, and this biological pathway temporarily reduces the production of the disease-causing protein. In the absence of the disease-causing protein, normal cellular function is restored and the chronic disease that initially resulted from the presence of the mutant protein is partially or completely resolved. RNAi is potentially applicable to over 20,000 human genes and a large number of disease-causing microorganism-specific genes.

Current wording

The mutation of a single gene can cause a chronic disease via the resulting intracellular production of a disease-causing protein (i.e., an abnormal form of the protein of interest), and many chronic and/or fatal disorders are known to result from the inappropriate expression of a single gene or multiple genes. In some cases, genetic disorders of this type can be treated exclusively by “silencing” the intracellular production of the disease-causing protein through well-validated biological approaches like RNAi. RNAi employs small nucleic acid molecules to activate an intracellular enzyme complex, and this biological pathway temporarily reduces the production of the disease-causing protein. In the absence of the disease-causing protein, normal cellular function is restored and the chronic disease that initially resulted from the presence of the mutant protein is partially or completely resolved. RNAi is potentially applicable to over 20,000 human genes and a large number of disease-causing microorganism-specific genes.

Previous wording

A small double stranded RNA, or dsRNA, molecule (A, Figure 1), comprising one strand known as the sense strand and another strand known as the antisense strand, which are complementary to each other, is synthesized in the laboratory. These small dsRNAs are called small interfering RNAs, or siRNAs. The sequence of the sense strand corresponds to a short region of the target gene mRNA. The siRNA is delivered to the target cell (B, Figure 1), where a group of enzymes, referred to as the RNA-Induced Silencing Complex, or RISC, process the siRNA (C, Figure 1), where one of the strands (usually the sense strand) is released (D, Figure 1). RISC uses the antisense strand to find the mRNA that has a complementary sequence (E, Figure 1) leading to the cleavage of the target mRNA (F, Figure 1). As a consequence, the output of the mRNA (protein production) does not occur (G, Figure 1). Several companies, including Alnylam Pharmaceuticals Inc. (“Alnylam”), utilize this approach in their RNAi product candidates.

Current wording

A small double stranded RNA, or dsRNA, molecule (A, Figure 1), comprising one strand known as the sense strand and another strand known as the antisense strand, which are complementary to each other, is synthesized in the laboratory. These small dsRNAs are called small interfering RNAs, or siRNAs. The sequence of the sense strand corresponds to a short region of the target gene mRNA. The siRNA is delivered to the target cell (B, Figure 1), where a group of enzymes, referred to as the RNA-Induced Silencing Complex, or RISC, process the siRNA (C, Figure 1), where one of the strands (usually the sense strand) is released (D, Figure 1). RISC uses the antisense strand to find the mRNA that has a complementary sequence (E, Figure 1) leading to the cleavage of the target mRNA (F, Figure 1). As a consequence, the output of the mRNA (protein production) does not occur (G, Figure 1). Several companies, including Alnylam Pharmaceuticals Inc., utilize this approach in their RNAi product candidates.

Previous wording

The silence and replace approach to the treatment of genetic disorders employs adeno-associated viral vectors (“AAVs”) to deliver genetic constructs which may, after a single administration to the target tissues:

Current wording

The silence and replace approach to the treatment of genetic disorders employs adeno-associated viral (“AAVs”) vectors to deliver genetic constructs which may, after a single administration to the target tissues:

Previous wording

Chronically express RNAi molecules inside of the target, diseased, cells (to serially silence the intracellular production of mutant, disease-causing, protein and the functional protein of interest);

Current wording

Chronically express RNAi molecules inside the target, diseased, cells (to serially silence the intracellular production of mutant, disease-causing, protein and the functional protein of interest);

Previous wording

The Investigational New Drug (IND) application for BB-301 was approved to proceed by the U.S. Food and Drug Administration in June 2023. The first study subject was safely treated in Cohort 1 of the BB-301 Phase 1b/2a clinical trial (NCT06185673) in November 2023. Cohort 1 dosing was safely completed in the second calendar quarter of 2025. Cohort 2 dosing was safely initiated in the fourth calendar quarter of 2025 . BB-301 is the lead investigational gene therapy agent under development by Benitec. BB-301 has been granted Orphan Drug Designation in the United States and the European Union and the key attributes of BB-301 are outlined in Figure 3.

Current wording

The Investigational New Drug (IND) application for BB-301 was approved to proceed by the U.S. Food and Drug Administration in June 2023. The first study subject was safely treated in Cohort 1 of the BB-301 Phase 1b/2a clinical trial (NCT06185673) in November 2023. All Cohort 1 subjects have safely completed the 12-month post-BB-301-treatment follow-up period. The first two Cohort 2 subjects have been safely treated. No treatment-related severe adverse events have been observed. BB-301 is the lead investigational gene therapy agent under development by Benitec. BB-301 has been granted Orphan Drug Designation in the European Union and Orphan Drug Designation and Fast Track Designation in the United States, and the key attributes of BB-301 are outlined in Figure 3.

Previous wording

There are currently no therapeutic interventions approved for OPMD. BB-301 is the only clinical-stage therapeutic agent in development designed to treat dysphagia in patients with OPMD. Additionally, there are no surgical interventions available to OPMD patients that modify the natural history of the disease, which is principally comprised of chronic deterioration of swallowing function. BB-301 has received Orphan Drug Designation in the United States and the European Union and, upon achievement of regulatory approval for BB-301 in these respective jurisdictions, the Orphan Drug Designations would provide commercial exclusivity independent of intellectual property protection. While OPMD is a rare disorder, we believe the commercial opportunity for a safe and efficacious therapeutic agent in this clinical indication exceeds $1 billion over the course of the commercial life of the product.

Current wording

There are currently no therapeutic interventions approved for OPMD. BB-301 is the only clinical-stage therapeutic agent in development designed to treat dysphagia in patients with OPMD. Additionally, there are no surgical interventions available to OPMD patients that modify the natural history of the disease, which is principally comprised of chronic deterioration of swallowing function. BB-301 has received Orphan Drug Designation in the European Union and Orphan Drug Designation and Fast Track Designation in the United States and, upon achievement of regulatory approval for BB-301 in these respective jurisdictions, the Orphan Drug Designations would provide commercial exclusivity independent of intellectual property protection. While OPMD is a rare disorder, we believe the commercial opportunity for a safe and efficacious therapeutic agent in this clinical indication exceeds $1 billion over the course of the commercial life of the product.

Previous wording

Total Pharyngeal Residue %(C2-4)2 o

Current wording

Total Pharyngeal Residue %(C2-4)2

Previous wording

All six subjects in Cohort 1 have been safely treated with BB-301, and the first subject of Cohort 2 has been safely treated with BB-301.No treatment-related Severe Adverse Events have been observed for the Subjects treated with BB-301.

Current wording

The first study subject was safely treated in Cohort 1 of the BB-301 Phase 1b/2a clinical trial (NCT06185673) in November 2023. All six Cohort 1 subjects have safely completed the 12-month post-BB-301-treatment follow-up period. The first two Cohort 2 subjects have been safely treated with BB-301. No treatment-related severe adverse events have been observed.

Previous wording

The Company’s functional currency and reporting currency is the United States dollar. BBL’s functional currency is the Australian dollar (AUD). Assets and liabilities are translated at the exchange rate in effect at the balance sheet date. Revenues and expenses are translated at the average rate of exchange prevailing during the reporting period. Equity transactions are translated at each historical transaction date spot rate. Translation adjustments arising from the use of different exchange rates from period to period are included as a component of stockholders’ equity in accumulated other comprehensive income (loss) and the Consolidated Statements of Comprehensive Income (Loss). Other comprehensive income (loss) for all periods presented consists entirely of foreign currency translation gains and losses.

Current wording

The Company’s functional currency and reporting currency is the United States dollar. BBL’s functional currency is the Australian dollar (AUD). Assets and liabilities are translated at the exchange rate in effect at the balance sheet date. Revenues and expenses are translated at the average rate of exchange prevailing during the reporting period. Equity transactions are translated at each historical transaction date spot rate. Translation adjustments arising from the use of different exchange rates from period to period are included as a component of stockholders’ equity in accumulated other comprehensive income (loss) and the Consolidated Statements of Comprehensive Loss. Other comprehensive income (loss) for all periods presented consists entirely of foreign currency translation gains and losses.

Previous wording

We anticipate that our general and administrative expenses may increase as we focus on the continued development of the clinical OPMD program. We also anticipate an increase in expenses relating to accounting, legal and regulatory-related services associated with maintaining compliance with exchange listing and SEC requirements, director and officer insurance premiums and other similar costs.

Current wording

We anticipate that our general and administrative expenses may increase as we focus on the continued development of the clinical OPMD program. We also anticipate an increase in expenses relating to accounting, legal and regulatory-related services associated with maintaining compliance with stock exchange listing and SEC requirements, director and officer insurance premiums and other similar costs.

Previous wording

The following tables sets forth a summary of our expenses for each of the periods:

Current wording

The following tables set forth a summary of our expenses for each of the periods:

Previous wording

During the three and six months ended December 31, 2025, we incurred $5.8 million and $9.2 million in research and development expenses, respectively, as compared to $5.4 million and $9.0 million for the comparable period ended December 31, 2024. Research and development expenses relate primarily to ongoing clinical development of BB-301 for the treatment of OPMD. The increase for the three and six months ended December 31, 2025 reflects increase in share based compensation of $2.2 million and $3.0 million, respectively, offset by the timing of contract manufacturing activity and the timing of payments for the OPMD Natural History and Dosing study.

Current wording

During the three and nine months ended March 31, 2026, we incurred $6.3 million and $15.5 million in research and development expenses, respectively, as compared to $6.5 million and $15.5 million for the comparable period ended March 31, 2025. Research and development expenses relate primarily to ongoing clinical development of BB-301 for the treatment of OPMD. The decrease for the three months ended March 31, 2026 was due to the timing of contract manufacturing activity and the timing of payments for the OPMD Natural History and Dosing study, partially offset by an increase in share based compensation of $2.0 million. In the nine months ended March 31, 2026 and 2025, research and development expense remained flat.

Previous wording

General and administrative expense totaled $7.5 million and $14.0 million for the three and six months ended December 31, 2025, compared to $5.4 million and $7.6 million for the comparable period ended December 31, 2024. The increase for the three and six months ended December 31, 2025, relates primarily to an increase in share based compensation of $2.0 million and $6.0 million and salaries and wages of $0.6 million and $0.9 million, offset by a decrease in legal fees of $0.4 million and $0.5 million, respectively.

Current wording

General and administrative expense totaled $7.3 million and $21.3 million for the three and nine months ended March 31, 2026, compared to $8.8 million and $16.5 million for the comparable period ended March 31, 2025. The decrease for the three months ended March 31, 2026 relates primarily to a decrease in share based compensation of $2.1 million, partially offset by increases in salaries and wages of $0.4 million. The increase for the nine months ended March 31, 2026 primarily related to increases in share based compensation of $3.9 million and salaries and wages of $0.8 million.

Previous wording

The following tables sets forth a summary of our other income (loss) for each of the periods:

Current wording

The following tables set forth a summary of our other income (loss) for each of the periods:

Previous wording

Other income (loss), net during the three and six months ended December 31, 2025, which consists of foreign currency transaction gain (loss), interest income, and other income (expense), net, totaled $1.5 million and $2.4 million, respectively. Other income (loss), net during the three and six months ended December 31, 2025, consists of foreign currency transaction gain (loss), interest income, and other expense, net, totaled $1.3 million and $2.0 million, respectively. Foreign currency transaction gains and losses reflect changes in foreign exchange rates. Net interest income for the three and six months ended December 31, 2025, in comparison to the three and six months ended December 31, 2024, reflects the increase in the Company’s cash and cash equivalent balances. Gain on extinguishment of liabilities is due to the settlement with a vendor of an outstanding trade payable balance and an accrued clinical development project cost balance totaling $1.3 million for $0.5 million due to a dispute regarding contract performance and deliverables. This settlement resulted in a gain of $0.8 million in the three and six months ended December 31, 2024.

Current wording

Other income (loss), net during the three and nine months ended March 31, 2026, consists of foreign currency transaction gain (loss), interest income, net, and other expense, net, totaled $1.6 million and $4.0 million, respectively. Other income (loss), net during the three and nine months ended March 31, 2025, consists of foreign currency transaction gain (loss), interest income, net, gain on extinguishment of liabilities, and other expense, net, totaled $0.8 million and $2.8 million, respectively. Foreign currency transaction gains and losses reflect changes in foreign exchange rates. The increase in net interest income for the three and nine months ended March 31, 2026, in comparison to the three and nine months ended March 31, 2025, reflects the increase in the Company’s cash and cash equivalent balances. Gain on extinguishment of liabilities in the prior period is due to the settlement with a vendor of an outstanding trade payable balance and an accrued clinical development project cost balance totaling $1.3 million for $0.5 million due to a dispute regarding contract performance and deliverables. This settlement resulted in a gain of $0.8 million in the nine months ended March 31, 2025.

Previous wording

The Company has incurred cumulative losses and negative cash flows from operations since our predecessor’s inception in 1995. The Company had accumulated losses of $249 million as of December 31, 2025. We expect that our research and development expenses will increase due to the continued development of the OPMD program.

Current wording

The Company has incurred cumulative losses and negative cash flows from operations since our predecessor’s inception in 1995. The Company had accumulated losses of $261 million as of March 31, 2026. We expect that our research and development expenses will increase due to the continued development of the OPMD program.

Previous wording

As of December 31, 2025, the Company has issued warrants allowing holders to purchase 20,179,428 shares of Common Stock, consisting of the following:

Current wording

As of March 31, 2026, the Company has issued warrants allowing holders to purchase 20,080,001 shares of Common Stock, consisting of the following:

Previous wording

As of December 31, 2025, we had cash and cash equivalents of approximately $188.8 million. Cash in excess of immediate requirements is invested in accordance with our investment policy, primarily with a view to liquidity and capital preservation. Currently, our cash and cash equivalents are held in bank accounts.

Current wording

As of March 31, 2026, we had cash and cash equivalents of approximately $184.8 million. Cash in excess of immediate requirements is invested in accordance with our investment policy, primarily with a view to liquidity and capital preservation. Currently, our cash and cash equivalents are held in bank accounts.

Previous wording

Net cash used in operating activities for the six months ended December 31, 2025 and 2024 was $7.1 million and $12.3 million, respectively. Net cash used in operating activities was primarily the result of our net loss, partially offset by non-cash expenses, and changes in working capital primarily in trade and other payables.

Current wording

Net cash used in operating activities for the nine months ended March 31, 2026 and 2025 was $11.6 million and $15.4 million, respectively. Net cash used in operating activities was primarily the result of our net loss, partially offset by non-cash expenses, and changes in working capital primarily in trade and other payables.

Previous wording

Net cash used in investing activities six months ended December 31, 2025 and 2024 was $11 thousand and $12 thousand, respectively.

Current wording

Net cash used in investing activities nine months ended March 31, 2026 and 2025 was $26 thousand and $18 thousand, respectively.

Previous wording

Net cash provided by financing activities was $98.2 million and $39.5 million for the six months ended December 31, 2025 and 2024, respectively. The financing activities for the six months ended December 31, 2025 primarily related to the issuance of common stock with gross proceeds of $104.5 million, offset by $6.3 million in share issuance costs. Cash from financing activities in the six months ended December 31, 2024 was related to the issuance of common stock from the exercise of Series 2 warrants and common stock warrants with gross proceeds of $39.5 million.

Current wording

Net cash provided by financing activities was $98.7 million and $67.7 million for the nine months ended March 31, 2026 and 2025, respectively. The financing activities for the nine months ended March 31, 2026 primarily related to the issuance of common stock with gross proceeds of $104.5 million and from the exercise of common stock warrants with gross proceeds of $0.5 million, offset by $6.3 million in share issuance costs. Cash from financing activities in the nine months ended March 31, 2025 was related to the issuance of common stock with gross proceeds of $30.5 million and issuance of common stock from the exercise of Series 2 warrants and common stock warrants with gross proceeds of $39.5 million, offset by $2.2 million in share issuance costs.

Previous wording

On October 1, 2016, the Company entered into an operating lease for office space in Hayward, California with multiple amendments extending the lease through December 2027. The Company also entered into a new lease in Los Angeles, California for office space with an initial expiration date in July 2026. The Company entered into a lease amendment for the Los Angeles office that extended the lease through January 2028. See Note 8 of the Notes to Consolidated Financial Statements included in this Quarterly Report on Form 10-Q.

Current wording

On October 1, 2016, the Company entered into an operating lease for office space in Hayward, California with multiple amendments extending the lease through December 2027. The Company also entered into a lease in Los Angeles, California for office space with an initial expiration date in July 2026. The Company entered into a lease amendment for the Los Angeles office that extended the lease through January 2028. See Note 8 of the Notes to Consolidated Financial Statements included in this Quarterly Report on Form 10-Q for additional information.

Previous wording

Estimates and assumptions about future events and their effects cannot be determined with certainty. The Company bases its estimates on historical experience and on various other assumptions believed to be applicable and reasonable under the circumstances. These estimates may change as new events occur, as additional information is obtained and as the Company’s operating environment changes. These changes have historically been minor and have been included in the consolidated financial statements as soon as they became known. In addition, management is periodically faced with uncertainties, the outcomes of which are not within its control and will not be known for prolonged periods of time. These uncertainties are discussed in the section above entitled “Risk Factors.” Based on a critical assessment of its accounting policies and the underlying judgments and uncertainties affecting the application of those policies, management believes that the Company’s consolidated financial statements are fairly stated in accordance with accounting principles generally accepted in the United States of America and provide a meaningful presentation of the Company’s financial condition and results of operations.

Current wording

Estimates and assumptions about future events and their effects cannot be determined with certainty. The Company bases its estimates on historical experience and on various other assumptions believed to be applicable and reasonable under the circumstances. These estimates may change as new events occur, as additional information is obtained and as the Company’s operating environment changes. These changes have historically been minor and have been included in the consolidated financial statements as soon as they became known. In addition, management is periodically faced with uncertainties, the outcomes of which are not within its control and will not be known for prolonged periods of time. These uncertainties are discussed in the section entitled “Risk Factors” in our Annual Report on Form 10-K for the year ended June 30, 2025. Based on a critical assessment of its accounting policies and the underlying judgments and uncertainties affecting the application of those policies, management believes that the Company’s consolidated financial statements are fairly stated in accordance with accounting principles generally accepted in the United States of America and provide a meaningful presentation of the Company’s financial condition and results of operations.