10-Q: Quarterly report pursuant to Section 13 or 15(d)
Published on May 15, 2023
Table of Contents
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QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(D) OF THE SECURITIES EXCHANGE ACT OF 1934 |
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TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(D) OF THE SECURITIES EXCHANGE ACT OF 1934 |
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(State or other jurisdiction of incorporation or organization) |
(IRS Employer Identification No.) |
Title of each class |
Trading Symbol(s) |
Name of each exchange on which registered |
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| Large accelerated filer | ☐ | Accelerated filer | ☐ | |||
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Non-accelerated filer |
☒ | Smaller reporting company | ||||
| Emerging growth company | ||||||
Table of Contents
BENITEC BIOPHARMA INC.
INDEX TO FORM 10-Q
| 1 | ||||||
| 2 | ||||||
| ITEM 1. |
2 | |||||
| Consolidated Balance Sheets as of March 31, 2023 (Unaudited) and June 30, 2022 |
2 | |||||
| 3 | ||||||
| 4 | ||||||
| Consolidated Statements of Cash Flows for the Nine Months Ended March 31, 2023 and 2022 (Unaudited) |
5 | |||||
| 6 | ||||||
| ITEM 2. |
Management’s Discussion and Analysis of Financial Condition and Results of Operations |
15 | ||||
| ITEM 3. |
29 | |||||
| ITEM 4. |
29 | |||||
| ITEM 1. |
30 | |||||
| ITEM 1A. |
30 | |||||
| ITEM 2. |
31 | |||||
| ITEM 3. |
31 | |||||
| ITEM 4. |
31 | |||||
| ITEM 5. |
31 | |||||
| ITEM 6. |
32 | |||||
| 33 | ||||||
Table of Contents
SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
This Report contains forward-looking statements that are subject to a number of risks and uncertainties, many of which are beyond our control. Our forward-looking statements relate to future events or our future performance and include, but are not limited to, statements concerning our business strategy, future commercial revenues, market growth, capital requirements, new product introductions, expansion plans and the adequacy of our funding. All statements, other than statements of historical fact included in this Report, are forward-looking statements. When used in this Report, the words “could,” “believe,” “anticipate,” “intend,” “estimate,” “expect,” “may,” “continue,” “predict,” “potential,” “project,” or the negative of these terms, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain such identifying words. These statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements.
Some of the risks and uncertainties that may cause our actual results, performance or achievements to differ materially from those expressed or implied by forward-looking statements include the following:
| • | the success of our plans to develop and potentially commercialize our product candidates; |
| • | the timing of the initiation and completion of preclinical studies and clinical trials; |
| • | the timing and sufficiency of patient enrollment and dosing in any future clinical trials; |
| • | the timing of the availability of data from clinical trials; |
| • | the timing and outcome of regulatory filings and approvals; |
| • | unanticipated delays; |
| • | sales, marketing, manufacturing and distribution requirements; |
| • | market competition and the acceptance of our products in the marketplace; |
| • | regulatory developments in the United States of America, France and Canada; |
| • | the development of novel AAV vectors; |
| • | the plans of licensees of our technology; |
| • | the clinical utility and potential attributes and benefits of ddRNAi and our product candidates, including the potential duration of treatment effects and the potential for a “one shot” cure; |
| • | our dependence on our relationships with collaborators and other third parties; |
| • | expenses, ongoing losses, future revenue, capital needs and needs for additional financing, and our ability to access additional financing given market conditions and other factors, including our capital structure; |
| • | our ability to continue as a going concern; |
| • | our ability to meet the Nasdaq listing standards; |
| • | the length of time over which we expect our cash and cash equivalents to be sufficient to execute on our business plan; |
| • | our intellectual property position and the duration of our patent portfolio; |
| • | the impact of local, regional, and national and international economic conditions and events; and |
| • | the impact of the current COVID-19 pandemic, the disease caused by the SARS-CoV-2 virus, which may adversely impact our business and preclinical and future clinical trials; |
as well as other risks detailed under the caption “Risk Factors” in this Report and in other reports filed with the SEC. Although we believe that we have a reasonable basis for each forward-looking statement contained in this Report, we caution you that these statements are based on a combination of facts and important factors currently known by us and our expectations of the future, about which we cannot be certain. We have based the forward-looking statements included in this Report on information available to us on the date of this Report or on the date thereof. Except as required by law we undertake no obligation to revise or update any forward-looking statements, whether as a result of new information, future events or otherwise. You are advised to consult any additional disclosures that we may make directly to you or through reports that we, in the future, may file with the SEC, including annual reports on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K.
All forward-looking statements included herein or in documents incorporated herein by reference are expressly qualified in their entirety by the cautionary statements contained or referred to elsewhere in this Report.
1
Table of Contents
| ITEM 1. | Financial Statements |
| March 31, | June 30, | |||||||
| 2023 | 2022 | |||||||
| (Unaudited) | ||||||||
Assets |
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Current assets: |
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Cash and cash equivalents |
$ | $ | ||||||
Restricted cash |
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Trade and other receivables |
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Prepaid and other assets |
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Total current assets |
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Property and equipment, net |
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Deposits |
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Other assets |
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Right-of-use |
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Total assets |
$ | $ | ||||||
Liabilities and stockholders’ equity |
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Current liabilities: |
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Trade and other payables |
$ | $ | ||||||
Accrued employee benefits |
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Lease liabilities, current portion |
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Total current liabilities |
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Lease liabilities, less current portion |
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Total liabilities |
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Commitments and contingencies (Note 11) |
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Stockholders’ equity: |
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Common stock, $ value- shares authorized; |
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Additional paid-in capital |
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Accumulated deficit |
( |
) | ( |
) | ||||
Accumulated other comprehensive loss |
( |
) | ( |
) | ||||
Total stockholders’ equity |
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Total liabilities and stockholders’ equity |
$ | $ | ||||||
Three Months Ended |
Nine Months Ended |
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March 31, |
March 31, |
|||||||||||||||
2023 |
2022 |
2023 |
2022 |
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Revenue: |
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Licensing revenues from customers |
$ | $ | $ | $ | ||||||||||||
Total revenues |
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Operating expenses |
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Research and development |
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General and administrative |
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Total operating expenses |
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Loss from operations |
( |
) | ( |
) | ( |
) | ( |
) | ||||||||
Other income (loss): |
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Foreign currency transaction gain (loss) |
( |
) | ( |
) | ||||||||||||
Interest expense, net |
( |
) | ( |
) | ( |
) | ( |
) | ||||||||
Other income, net |
|
( |
) | ( |
) | |||||||||||
Unrealized loss on investment |
( |
) | ( |
) | ( |
) | ( |
) | ||||||||
Total other income (loss), net |
( |
) | ( |
) | ( |
) | ||||||||||
Net loss |
$ | ( |
) | $ | ( |
) | $ | ( |
) | $ | ( |
) | ||||
Other comprehensive income: |
||||||||||||||||
Unrealized foreign currency translation gain (loss) |
( |
) | ( |
) | ||||||||||||
Total other comprehensive income (loss) |
( |
) | ( |
) | ||||||||||||
Total comprehensive loss |
$ | ( |
) | $ | ( |
) | $ | ( |
) | $ | ( |
) | ||||
Net loss |
$ | ( |
) | $ | ( |
) | $ | ( |
) | $ | ( |
) | ||||
Net loss per share: |
||||||||||||||||
Basic and diluted |
$ | ( |
) | $ | ( |
) | $ | ( |
) | $ | ( |
) | ||||
Weighted average number of shares outstanding: basic and diluted |
||||||||||||||||
Common Stock |
Additional Paid-in |
Accumulated |
Accumulated Other Comprehensive |
Total Stockholders’ |
||||||||||||||||||||
Shares |
Amount |
Capital |
Deficit |
Loss |
Equity |
|||||||||||||||||||
| Balance at June 30, 2021 |
$ | $ | $ | ( |
) | $ | ( |
) | $ | |||||||||||||||
| Share-based compensation |
— | — | — | — | ||||||||||||||||||||
| Foreign currency translation gain |
— | — | — | — | ||||||||||||||||||||
| Net loss |
— | — | — | ( |
) | ( |
) | |||||||||||||||||
| |
|
|
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|
|
|
|
|
|
|||||||||||||
| Balance at September 30, 2021 |
( |
) | ( |
) | ||||||||||||||||||||
| |
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|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Share-based compensation |
— | — | — | — | ||||||||||||||||||||
| Foreign currency translation loss |
— | — | — | — | ( |
) | ( |
) | ||||||||||||||||
| Net loss |
— | — | — | ( |
) | — | ( |
) | ||||||||||||||||
| |
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Balance at December 31, 2021 |
( |
) | ( |
) | ||||||||||||||||||||
| Share-based compensation |
— | — | — | — | ||||||||||||||||||||
| Foreign currency translation loss |
— | — | — | — | ( |
) | ( |
) | ||||||||||||||||
| Net loss |
— | — | — | ( |
) | — | ( |
) | ||||||||||||||||
| |
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Balance at March 31,2022 |
$ | $ | $ | ( |
) | $ | ( |
) | $ | |||||||||||||||
| |
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Balance at June 30, 2022 |
$ | $ | $ | ( |
) | $ | ( |
) | $ | |||||||||||||||
| Issuance of common stock, pre-funded warrants, and common warrants sold for cash, net of offering costs of $ |
— | — | ||||||||||||||||||||||
| Share-based compensation |
— | — | — | — | ||||||||||||||||||||
| Foreign currency translation gain |
— | — | — | — | ||||||||||||||||||||
| Net loss |
— | — | — | ( |
) | — | ( |
) | ||||||||||||||||
| |
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Balance at September 30, 2022 |
( |
) | ( |
) | ||||||||||||||||||||
| Exercise of pre-funded warrants |
— | — | — | — | — | |||||||||||||||||||
| Share-based compensation |
— | — | ( |
) | — | — | ( |
) | ||||||||||||||||
| Foreign currency translation loss |
— | — | — | — | ( |
) | ( |
) | ||||||||||||||||
| Net loss |
— | — | — | ( |
) | — | ( |
) | ||||||||||||||||
| |
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|
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|
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|
|
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|
|
|
| Balance at December 31, 2022 |
( |
) | ( |
) | ||||||||||||||||||||
| Share-based compensation |
— | — | — | — | ||||||||||||||||||||
| Foreign currency translation gain |
— | — | — | — | ||||||||||||||||||||
| Net loss |
— | — | — | ( |
) | — | ( |
) | ||||||||||||||||
| |
|
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| Balance at March 31, 2023 |
$ |
$ |
$ |
( |
) | $ |
( |
) | $ |
|||||||||||||||
| |
|
|
|
|
|
|
|
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|
|||||||||||||
| Nine Months Ended March 31, |
||||||||
| 2023 | 2022 | |||||||
Cash flows from operating activities: |
||||||||
Net loss |
$ | ( |
) | $ | ( |
) | ||
Adjustments to reconcile net loss to net cash used in operating activities: |
||||||||
Depreciation and amortization |
||||||||
Amortization of right-of-use |
||||||||
Unrealized loss on investment |
||||||||
Share-based compensation expense |
||||||||
Changes in operating assets and liabilities: |
||||||||
Trade and other receivables |
( |
) | ||||||
Prepaid expense and other assets |
( |
) | ||||||
Trade and other payables |
||||||||
Accrued employee benefits |
||||||||
Lease liabilities |
( |
) | ( |
) | ||||
Net cash used in operating activities |
( |
) | ( |
) | ||||
Cash flows from financing activities: |
||||||||
Proceeds from issuance of common stock, pre-funded warrants, and common warrants |
— | |||||||
Shares and pre-funded warrant issuance costs |
( |
) | — | |||||
Net cash provided by financing activities |
— | |||||||
Effects of exchange rate changes on cash, cash equivalents, and restricted cash |
( |
) | ||||||
Net increase (decrease) in cash, cash equivalents, and restricted cash |
( |
) | ||||||
Cash, cash equivalents, and restricted cash, beginning of period |
||||||||
Cash, cash equivalents, and restricted cash, end of period |
$ | $ | ||||||
Supplemental disclosure of cash flow information: |
||||||||
Re-measurement of operating lease right-of-use |
$ |
|
$ | |||||
| Principal place of business/country of incorporation |
||
Benitec Biopharma Proprietary Limited (“BBL”) |
||
Benitec Australia Proprietary Limited |
||
Benitec Limited |
||
Benitec, Inc. |
||
Benitec LLC |
||
RNAi Therapeutics, Inc. |
||
Tacere Therapeutics, Inc. |
||
Benitec IP Holdings, Inc. |
| Level 1: | Observable inputs such as quoted prices (unadjusted) in active markets for identical assets or liabilities. | |
| Level 2: | Inputs, other than quoted prices that are observable, either directly or indirectly. These include quoted prices for similar assets or liabilities in active markets and quoted prices for identical or similar assets or liabilities in markets that are not active. | |
| Level 3: | Unobservable inputs in which little or no market data exists, therefore developed using estimates and assumptions developed by us, which reflect those that a market participant would use. | |
| Software |
|
|
| Lab equipment |
|
|
| Computer hardware |
|
|
| Leasehold improvements |
| March 31, | June 30, | |||||||
| (US$’000) | 2023 | 2022 | ||||||
| Cash at bank |
$ | $ | ||||||
| Restricted cash |
||||||||
| |
|
|
|
|
|
|
|
|
| Total |
$ | $ | ||||||
| |
|
|
|
|
|
|
|
|
(US$’000) |
March 31, 2023 |
June 30, 2022 |
||||||
| Prepaid expenses |
$ | $ | ||||||
| Prepaid expenses—OPMD Program |
||||||||
| Market value of listed shares |
||||||||
| Total other assets |
||||||||
| Less: non-current portion |
( |
) | ( |
) | ||||
| |
|
|
|
|
|
|
|
|
| Current portion |
$ | $ | ||||||
| |
|
|
|
|
|
|
|
|
(US$’000) |
March 31, 2023 |
June 30, 2022 |
||||||
| Software |
$ |
$ |
||||||
| Lab equipment |
||||||||
| Computer hardware |
||||||||
| Leasehold improvements |
||||||||
| |
|
|
|
|||||
| Total property and equipment, gross |
||||||||
| Accumulated depreciation and amortization |
( |
) |
( |
) | ||||
| |
|
|
|
|||||
| Total property and equipment, net |
$ |
$ |
||||||
| |
|
|
|
|||||
| (US$’000) | March 31, 2023 |
June 30, 2022 |
||||||
| Trade payable |
$ | $ | ||||||
| Accrued license fees |
||||||||
| Accrued professional fees |
||||||||
| Accrued OPMD project costs |
||||||||
| Accrued consultant fees |
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| Other payables |
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| |
|
|
|
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| Total |
$ | $ | ||||||
| |
|
|
|
|
|
|
|
|
| (US$’000) | Operating lease right- of- use assets |
|||
| Balance at July 1, 2022 |
$ | |||
| Amortization of right of use asset |
( |
) | ||
| |
|
|||
| Operating lease right-of-use |
$ | |||
| |
|
|||
| (US$’000) | Operating lease liabilities |
|||
| Balance at July 1, 2022 |
$ | |||
| Principal payments on operating lease liabilities |
( |
) | ||
| |
|
|||
| Operating lease liabilities at March 31, 2023 |
||||
| Less: non-current portion |
( |
) | ||
| |
|
|||
| Current portion at March 31, 2023 |
$ | |||
| |
|
|||
| (US$’000) | March 31, 2023 |
|||
| 2023 |
$ | |||
| 2024 |
||||
| 2025 |
||||
| |
|
|||
| Total operating lease payments |
||||
| Less imputed interest |
( |
) | ||
| |
|
|||
| Present value of operating lease liabilities |
$ | |||
| |
|
|||
| Weighted- | ||||||||
| average | ||||||||
| Exercise | ||||||||
| Common | Price | |||||||
| Stock from | (per | |||||||
| Warrants | share) | |||||||
| Outstanding at July 1, 2022 |
$ | |||||||
| Pre-funded warrants issued September 15, 2022 |
$ | |||||||
| Series 2 Warrants issued September 15, 2022 |
$ | |||||||
| |
|
|
|
|||||
| Outstanding at September 30, 2022 |
$ | |||||||
| Pre-funded warrants exercised |
$ | |||||||
| |
|
|
|
|||||
| Outstanding at March 31, 2023 |
$ | |||||||
| |
|
|
|
|||||
| Exercisable at March 31, 2023 |
$ | |||||||
| |
|
|
|
|||||
| Weighted- | ||||||||||||||||
| Weighted- | average | |||||||||||||||
| average | Remaining | Aggregate | ||||||||||||||
| Stock | Exercise | Contractual | Intrinsic | |||||||||||||
| Options | Price | Term | Value | |||||||||||||
Outstanding at June 30, 2022 |
$ |
|
$ | — | ||||||||||||
Expired |
$ | |||||||||||||||
Granted |
$ |
|
||||||||||||||
Outstanding at March 31, 2023 |
$ |
|
$ | |||||||||||||
Exercisable at March 31, 2023 |
$ |
|
$ | — | ||||||||||||
| Three Months Ended | Nine Months Ended | |||||||||||||||
| March 31, 2023 | ||||||||||||||||
| (US$’000) | 2023 | 2022 | 2023 | 2022 | ||||||||||||
Research and development |
$ | $ | $ | $ | ||||||||||||
General and administrative |
||||||||||||||||
Total share-based compensation expense |
$ | $ | $ | $ | ||||||||||||
Table of Contents
| Item 2. | Management’s Discussion and Analysis of Financial Condition and Results of Operations |
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.
Overview
We endeavor to become the leader in discovery, development, and commercialization of therapeutic agents capable of addressing significant unmet medical need via the application of the silence and replace approach to the treatment of genetic disorders.
Benitec Biopharma Inc. (“Benitec” or the “Company” or in the third person, “we” or “our”) is a development-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary platform, called DNA-directed RNA interference, or ddRNAi, combines RNA interference, or RNAi, with gene therapy to create medicines that facilitate sustained silencing of disease- causing genes following a single administration. The Company is developing a ddRNAi-based therapeutic (BB-301) for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD), a chronic, life-threatening genetic disorder.
BB-301 is a ddRNAi-based genetic medicine currently under development by Benitec. BB-301 is an AAV-based gene therapy designed to simultaneously silence the expression of the mutant, disease-causing gene (to slow, or halt, the biological mechanisms underlying disease progression in OPMD) and replace the mutant gene with a wild type gene (to drive restoration of function in diseased cells). 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.
The targeted gene silencing effects of RNAi, in conjunction with the durable transgene expression achievable via the use of modified viral vectors, imbues the silence and replace approach with the potential to produce long-term silencing of disease-causing genes along with simultaneous replacement of wild type gene function following a single administration of the proprietary genetic medicine. We believe that this novel mechanistic profile of the current and future investigational agents developed by Benitec could facilitate the achievement of robust and durable clinical activity while greatly reducing the frequency of drug administration traditionally expected for medicines employed for the management of chronic diseases.
Additionally, the achievement of long-term gene silencing and gene replacement may significantly reduce the risk of patient non-compliance during the course of medical management of potentially fatal clinical disorders.
COVID-19
COVID-19 has been declared a pandemic by the World Health Organization and has spread to nearly every country, including Australia and the United States. The impact of this pandemic has been, and will likely continue to be, extensive in many aspects of society, which has resulted in, and will likely continue to result in, significant disruptions to businesses and capital markets around the world. The extent to which the coronavirus impacts us will depend on future developments, which are highly uncertain and cannot be predicted, including new information which may emerge concerning the severity of the coronavirus and its variants, and the actions to contain the coronavirus or treat its impact, including the effectiveness and adoption of vaccines for the virus, among others.
Certain elements of our research and development efforts are conducted globally, including the ongoing development of our silence and replace therapeutic for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD), and will be dependent upon our ability to complete preclinical studies and initiate clinical studies despite the ongoing COVID-19 pandemic.
As we endeavor to complete our development programs, including the ongoing Toxicology and Biodistribution and Natural History studies for BB-301, we are in close contact with our principal investigators, contract research organizations, and preclinical trial sites, which are located in the United States, Canada, and France, and are assessing the impact of COVID-19 on our studies and the expected development timelines and costs on an ongoing basis. In light of developments relating to the COVID-19 global pandemic since the beginning of the outbreak, the focus of healthcare providers and hospitals on fighting the virus, and consistent with the FDA’s industry guidance for conducting clinical trials, we have experienced delays to the original timeline regarding the initiation and anticipated completion of the ongoing BB-301 Clinical Trial Application (CTA)-enabling and Investigational New Drug Application (IND)-enabling development work. The initiation of the BB-301 Pilot Dosing Study in Beagle dogs, which represents a key component of the CTA- enabling and IND-enabling work, was delayed by several months, however, the study has been completed without incident. The acquisition of chemical reagents, biological reagents and laboratory supplies which are essential for the conduct of basic laboratory research, the conduct of nonclinical studies and the
15
Table of Contents
completion of GMP manufacturing of BB-301, has also become challenging due to the disruption of global supply chains inherent to the production of these materials. We will continue to evaluate the impact of the COVID-19 pandemic on our business and we expect to reevaluate the timing of our anticipated preclinical and clinical milestones as we learn more and the impact of COVID-19 on our industry becomes clearer.
We have also implemented a halt of non-essential business travel and a rotation system whereby staff work from home and attend the laboratory on designated days which may result in a reduction of laboratory work. As we transition our employees back to our premises, there is a risk that COVID-19 infections occur at our offices or laboratory facilities and significantly affect our operations. Additionally, if any of our critical vendors are impacted, our business could be affected if we become unable to procure essential equipment in a timely manner or obtain supplies or services in adequate quantities and at acceptable prices.
Public Equity Offering
On September 15, 2022, we closed an underwritten public offering in which we issued and sold (i) 17,637,843 shares of the Company’s common stock, (ii) 12,171,628 pre-funded warrants, with each pre-funded warrant immediately exercisable for one share of common stock at an exercise price of $0.0001 per share until exercised in full and (iii) 29,809,471 common warrants, with each common warrant accompanying each issued share of common stock and/or pre-funded warrant and exercisable for one share of common stock at an exercise price of $0.66 per share (the “September 2022 Capital Raise”). The common warrants sold in the offering became exercisable on December 9, 2022, the date by which the Company (a) received approval from its stockholders to increase the number of shares of common stock it is authorized to issue and (b) effected such stockholder approval by filing with the Secretary of State of the State of Delaware a certificate of amendment to its amended and restated certificate of incorporation, and will expire on the fifth anniversary of such initial exercise date. The combined purchase price for each share of common stock and accompanying common warrant was $0.60, which was allocated as $0.59 per share of common stock and $0.01 per common warrant.
The net proceeds to the Company from the public offering were approximately $16 million, after deducting underwriting discounts and commissions and public offering expenses payable by the Company, and excluding any proceeds the Company may receive upon exercise of the pre-funded warrants or the common warrants. The Company currently intends to use the net proceeds for the clinical development of BB-301, including the natural history lead-in study and the Phase 1b/2a BB-301 treatment study, for the continued advancement of development activities for other existing and new product candidates, for general corporate purposes and for strategic growth opportunities. The Company will have broad discretion in determining how the proceeds of the public offering will be used, and its discretion is not limited by the aforementioned possible uses.
Nasdaq Listing
On September 6, 2022, we received a letter from the Listing Qualifications Department of the Nasdaq Stock Market notifying us that the minimum bid price per share for our common stock fell below $1.00 for a period of 30 consecutive business days and therefore we did not meet the minimum bid price requirement set forth in Nasdaq Listing Rule 5550(a)(2).
The letter also states that we will be provided 180 calendar days, or until March 6, 2023, to regain compliance with the minimum bid price requirement. In accordance with Rule 5810(c)(3)(A), we can regain compliance if at any time during the 180-day period the closing bid price of our common stock is at least $1.00 for a minimum of 10 consecutive business days. On March 7, 2023, Nasdaq notified the Company in writing that while the Company had not regained compliance with the minimum bid price requirement, it was eligible for an additional 180 calendar day period, or until September 5, 2023, to regain compliance with the minimum bid price requirement. Nasdaq’s determination was based on the Company having met the continued listing requirement for market value of publicly held shares and all other applicable requirements for initial listing on The Nasdaq Capital Market, with the exception of the minimum bid price requirement and on the Company’s written notice to Nasdaq of its intention to cure the deficiency during the second compliance period by effecting a reverse stock split, if necessary. If at any time during this additional time period the closing bid price of the Company’s common stock is at least $1.00 per share for a minimum of 10 consecutive business days, Nasdaq will provide written confirmation of compliance and the matter will be closed.
We intend to monitor the closing bid price of our common stock and may, if appropriate, consider implementing available options to regain compliance with the minimum bid price requirement. These options include completing a reverse stock split of our common stock for the purpose of meeting the closing bid price requirement. We received stockholder approval to effect a reverse stock split at our Annual Meeting of Stockholders on December 7, 2022 but currently have no plans to proceed. Such approval allows our board of directors, in its discretion, to elect to implement such reverse stock split at any time prior to December 7, 2023. Completing a reverse stock split will not, in of itself, cause us to remain in compliance with Nasdaq’s listing standards.
Development Programs
Our Pipeline
The following table sets forth the current product candidate and the development status:
Table 1. Pipeline: Oculopharyngeal Muscular Dystrophy
BB-301
We are developing BB-301 for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD). BB-301 is the lead investigational agent under development by Benitec, and the key attributes of OPMD and BB-301 are outlined in Figure 3.
Figure 3. Overview of the BB-301 Program
BB-301 is a first-in-class genetic medicine employing the “silence and replace” approach for the treatment of OPMD. OPMD is an insidious, autosomal- dominant, late-onset, degenerative muscle disorder that typically presents in patients at 40-to-50 years of age. The disease is characterized by progressive swallowing difficulties (dysphagia) and eyelid drooping (ptosis). OPMD is caused by a specific mutation in the poly(A)-binding protein nuclear 1 gene (PABPN1).
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OPMD is a rare disease, however, patients have been diagnosed with OPMD in at least 33 countries. Patient populations suffering from OPMD are well- identified, and significant geographical clustering has been noted for patients with this disorder. Each of these attributes could facilitate efficient clinical development and global commercialization of BB-301.
PABPN1 is a ubiquitous factor that promotes the interaction between the poly(A) polymerase and CPSF (cleavage and polyadenylation specificity factor) and, thus, controls the length of mRNA poly(A) tails, mRNA export from the nucleus, and alternative poly(A) site usage. The characteristic genetic mutation underlying OPMD results in trinucleotide repeat expansion(s) within exon 1 of PABPN1 and results in an expanded poly-alanine tract at the N-terminal end of PABPN1. The mutation generates a protein with an N-terminal expanded poly-alanine tract of up to 18 contiguous alanine residues, and the mutant protein is prone to the formation of intranuclear aggregates designated as intranuclear inclusions (INIs). The INIs that sequester wildtype PABPN1 may contribute to the “loss of function” phenotype associated with OPMD.
No therapeutic agents are approved for the treatment of 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 medical 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.
Benitec has previously outlined the core CTA-enabling and IND-enabling studies required by global regulatory agencies to support the initiation of BB-301 clinical trials in OPMD patients, and these studies include a BB-301 Pilot Dosing Study (the “Pilot Dosing Study”) in large animals and a classical 12-week GLP Toxicology and Biodistribution Study for BB-301. In these large animal studies, BB-301 is directly injected into the pharyngeal muscles known to underlie the morbidity and mortality which characterizes the natural history of OPMD in human subjects.
As referenced above, the BB-301 Pilot Dosing Study in large animals was the first of two CTA-enabling and IND-enabling studies conducted by Benitec. This study was carried out under the guidance of the scientific team at Benitec, with key elements of the design and execution of the study conducted in close collaboration with a team of experts in both medicine and surgery that have been deeply engaged in the treatment of OPMD patients for decades. The BB-301 Pilot Dosing Study and the GLP Toxicology and Biodistribution Study for BB-301 were conducted in canine subjects in order to:
| • | Support the validation and optimization of the newly designed route and method of BB-301 administration, |
| • | Confirm the efficiency of vector transduction and transgene expression in the key tissue compartments underlying the morbidity and mortality that comprises the natural history of OPMD, |
| • | Confirm the optimal BB-301 doses in advance of initiation of human clinical studies, |
| • | Facilitate the observation of key toxicological data-points. |
The BB-301 Pilot Dosing Study was designed as an 8-week study in Beagle dogs to confirm the transduction efficiency of BB-301 upon administration via direct intramuscular injection into specific anatomical regions of the pharynx through the use of an open surgical procedure. This new method and route of BB-301 administration was developed in collaboration with key surgical experts in the field of Otolaryngology, and this novel method of BB-301 dosing will significantly enhance the ability of treating physicians to accurately administer the AAV-based investigational agent to the muscles that underlie the characteristic deficits associated with disease progression in OPMD. It is important to note that prior BB-301 non-clinical studies have reproducibly validated the robust biological activity achieved following direct intramuscular injection of the AAV-based agent. As an example, direct injection of BB-301 into the tibialis anterior muscles of A17 mice facilitated robust transduction of the targeted skeletal muscle cells and supported complete remission of the OPMD disease phenotype in this animal model.
Benitec conducted the BB-301 Pilot Dosing Study in Beagle dog subjects to demonstrate that direct intramuscular injection of BB-301 via the use of a proprietary dosing device in an open surgical procedure could safely achieve the following goals:
| • | Biologically significant and dose-dependent levels of BB-301 tissue transduction (i.e., delivery of the multi-functional BB-301 genetic construct into the target pharyngeal muscle cells), |
| • | Broad-based and dose-dependent expression of the three distinct genes comprising the BB-301 gene construct within the pharyngeal muscle cells, and |
| • | Biologically significant levels of target gene knock-down (i.e., inhibition of the expression of the gene of interest) within the pharyngeal muscle cells. |
The Pilot Dosing Study evaluated the safety and biological activity of two concentrations of BB-301 (1.0+E13 vg/mL and 3.0+E13 vg/mL) across three distinct doses (1.0+E13 vg/mL and 3.0+E13 vg/mL with a low injection volume, and 3.0+E13
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vg/mL with a high injection volume) following direct intramuscular injection into the Hypopharyngeus (HP) muscles and the Thyropharyngeus (TP) muscles of Beagle dogs via the use of a proprietary delivery device employed in an open surgical procedure. The HP muscle in Beagle dogs corresponds to the Middle Pharyngeal Constrictor muscle in human subjects, and the TP muscle in Beagle dogs corresponds to the Inferior Pharyngeal Constrictor muscle in human subjects. BB-301 was injected only on Day 1 of the Pilot Dosing Study, and the corresponding canine pharyngeal muscles were harvested for molecular analyses after 8 weeks of observation post-injection. BB-301 dosing was carried out independently by a veterinary surgeon and an Otolaryngologist with extensive experience regarding the provision of palliative surgical care for OPMD patients.
Molecular analyses have been completed for the canine subjects treated in the BB-301 Pilot Dosing Study. Key interim data-sets derived from the analyses of pharyngeal muscle tissues isolated from 16 Beagle dog subjects (of the 24-subject Beagle dog study population) are highlighted below. The final data-set derived from the completed molecular analyses of the pharyngeal muscle tissues of the canine subjects treated on the Pilot Dosing Study will be presented in a peer-reviewed format.
The key interim data-sets are summarized below:
Figure 4. Pharyngeal Muscle Tissue Transduction Levels Achieved by BB-301
Regarding Gene Expression Levels Observed for BB-301 Within the Pharyngeal Muscle Tissues (Figure 5, Figure 6, Figure 7):
| • | BB-301 encodes two distinct siRNA species (i.e., siRNA13 and siRNA17) which are each, independently, capable of inhibiting (i.e., “silencing”) the expression of the mutant form of the PABPN1 protein and the wild type (i.e., endogenous) form of the PABPN1 protein (importantly, the mutant form of the PABPN1 protein underlies the development, and progression, of OPMD). |
| • | BB-301 also codes for a wild type version of the PABPN1 protein whose intracellular expression is unaffected by the inhibitory activities of siRNA13 and siRNA17; this “codon optimized” transcript drives the expression of a PABPN1 protein (i.e., coPABPN1) which serves to replenish the endogenous form of the PABPN1 protein and to replace the mutant form of PABPN1 that underlies the development and progression of OPMD in diseased tissues. |
| • | For comparative purposes, it should be noted that the average range of expression for wild type PABPN1 within the pharyngeal muscle cells of Beagle dogs is 4.5 copies per cell-to-7.8 copies per cell. |
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Figure 5. siRNA13 Expression Levels Achieved by BB-301 within Pharyngeal Muscle Tissues
Figure 6. siRNA17 Expression Levels Achieved by BB-301 within Pharyngeal Muscle Tissues
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Figure 7. coPABPN1 Expression Levels Achieved by BB-301 within Pharyngeal Muscle Tissues
Regarding Wild Type PABPN1 Silencing (i.e., target “knock-down”) Observed for BB-301 Within the Pharyngeal Muscle Tissues (Figure 8):
| • | As noted above, BB-301 encodes two distinct siRNA species (i.e., siRNA13 and siRNA17) which are each, independently, capable of inhibiting (i.e., “silencing”) the expression of all forms of the PABPN1 protein (siRNA13 and siRNA17 silence the expression of both wild type PABPN1 [wtPABPN1] and mutant PABPN1). |
| • | While the Beagle dog subjects treated in the BB-301 Pilot Dosing Study do not express mutant PABPN1, the level of BB-301-driven gene silencing for the PABPN1 target can be indirectly assessed in these study subjects due to the equivalent inhibitory effects of siRNA13 and siRNA17 on both wtPABPN1 and mutant PABPN1. |
| • | Thus, the wtPABPN1 silencing activity observed in the BB-301 Pilot Dosing Study serves as a surrogate for the silencing activity that would be anticipated in the presence of mutant PABPN1. |
| • | BB-301 has been evaluated in prior non-clinical studies in animals that express mutant PABPN1 and, as a result, manifest the symptomatic phenotype of OPMD; in the symptomatic animal model of OPMD (i.e. the A17 mouse model), the achievement of PABPN1 silencing levels of 31% inhibition (or higher) following BB-301 administration led to resolution of OPMD disease symptoms and the elimination of the histopathological hallmarks of OPMD. |
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Figure 8. PABPN1 Silencing (i.e., “target knock-down”) Achieved by BB-301 within Pharyngeal Muscle Tissues
There are key methodological distinctions between the current BB-301 Pilot Dosing Study conducted by Benitec as compared to the prior BB-301 Beagle dog dosing study carried out independently by the previous BB-301 licensee. The BB-301 dosing study conducted by the prior BB-301 licensee employed non-ideal routes and methods of BB-301 administration to the target pharyngeal muscle tissues and employed similarly limited analytical methods at the completion of the dosing phase of the study. Subsequently, the Benitec team worked to optimize the route and method of administration of BB-301 and to refine the core analytical methods employed following the completion of dosing of the large animal subjects.
The current proprietary method of BB-301 delivery to the key pharyngeal muscles of study subjects, and the proprietary molecular analytical methods employed to assay the pharyngeal muscle tissues of study subjects, with both methods having been developed by the Benitec team, led to the observation of broad-based transduction of the targeted pharyngeal muscle tissues (Figure 9, represents individual sections of the TP muscle following BB-301 dosing). Critically, the Benitec-developed methods also facilitated the achievement of a 228-fold improvement (+22,647%) in BB-301 transduction of the HP muscle and a 113-fold improvement (+11,163%) in BB-301 transduction of the TP muscle relative to the levels of BB-301 transduction observed by the previous BB-301 licensee at identical BB-301 doses in identical canine study populations (Figure 10).
Figure 9. BB-301 Transduction Levels Achieved for Individual Sections of the TP Muscle Following BB-301 dosing
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Figure 10. Impact of the Methodological Improvements to the BB-301 Large Animal Dosing Study Design on the Relative Pharyngeal Muscle Tissue Transduction Levels Achieved by Benitec vs. the Former BB-301 Licensee
Following the disclosure of the positive interim BB-301 Pilot Dosing Study results, Benitec completed pre-CTA and pre-IND meetings with regulatory agencies in France, Canada, and the United States.
Summary of Regulatory Interactions:
| • | Benitec successfully completed the regulatory interactions required to support initiation of the OPMD clinical development program in 2022 |
| • | Successful regulatory engagement comprised the completion of the following meetings: |
| • | Pre-Clinical Trial Application (Pre-CTA) Consultation Meeting with Health Canada |
| • | Scientific Advice Meeting with The National Agency for the Safety of Medicines and Health Products in France (L’Agence nationale de sécurité du médicament et des produits de santé or “ANSM”) |
| • | Type C Meeting with the U.S. Food and Drug Administration (“FDA”) |
In December 2022 Benitec began screening OPMD subjects at the first clinical trial site in the United States in support of the OPMD clinical development program. In January 2023 Benitec disclosed the enrollment of the first OPMD subject into the Natural History Study (i.e., the 6-month pre-treatment observation period described below). Enrollment of OPMD subjects into the Natural History Study at the U.S. clinical trial site is proceeding at a rapid pace and supports our central clinical development goals of: (1) administering BB-301 to OPMD subjects in 2H2023, and (2) disclosing interim safety and efficacy data in 2H2023 for one or more subjects that have received BB-301.
Summary of the BB-301 Clinical Development Program:
| • | The BB-301 clinical development program began in 2022, and the conduct of the development program will comprise approximately 76-weeks of follow-up for each OPMD study participant, inclusive of: |
| • | 6-month pre-treatment observation periods employing quantitative radiographic imaging techniques for evaluation of the baseline disposition and natural history of OPMD-derived dysphagia 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 screening of OPMD subjects for enrollment into the OPMD Natural History Study began in December 2022, with the first OPMD subject being formally enrolled in January 2023 and brisk enrollment having continued over the subsequent months. This observational study will facilitate the characterization of OPMD patient disposition at baseline and assess subsequent rates of progression of dysphagia (swallowing impairment) in subjects with OPMD via the use of quantitative radiographic measures of global swallowing function and pharyngeal constrictor muscle function along with clinical assessments and patient-reported self-assessments of swallowing function |
| • | Videofluoroscopic Swallowing Studies (VFSS) will be conducted to complete the following methodological assessments: |
| • | Dynamic Imaging Grade of Swallowing Toxicity Scale (DIGEST) |
| • | Pharyngeal Area at Maximum Constriction (PhAMPC) |
| • | Pharyngeal Constriction Ratio (PCR) |
| • | Clinical measures of global swallowing capacity and oropharyngeal dysphagia |
| • | Patient-reported measures of oropharyngeal dysphagia |
| • | The natural history of dysphagia observed for each OPMD study participant in the Natural History Study, as characterized by the quantitative radiographic measures and the clinical and patient self-reported assessments outlined above, will serve as the baseline for comparative assessments of safety and efficacy of BB-301 upon rollover of OPMD study subjects from the Natural History Study onto the Phase 1b/2a BB-301 treatment study |
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| • | Upon the achievement of 6-months of follow-up in the Natural History Study, OPMD Natural History Study participants can become eligible for enrollment onto the Phase 1b/2a treatment study with the investigational genetic medicine, BB-301, which uses an AAV9-based gene therapy approach for the treatment of OPMD-derived dysphagia |
| • | This first-in-human (FIH) clinical trial 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 the pharyngeal muscles of subjects with OPMD |
| • | Upon rollover from the Natural History Study onto the Phase 1b/2a BB-301 treatment study, the follow-up of OPMD study participants will continue for 52-weeks, and the primary endpoints (safety) and secondary endpoints (comprising the quantitative radiographic measures of global swallowing function and pharyngeal constrictor muscle function, and the clinical and patient-reported assessments noted above) will be evaluated during each 90-day period following Day 1 (Day 1 represents the day of BB-301 intramuscular injection). |
Royalties, milestone payments and other license fees
We are required to pay royalties, milestone payments and other license fees in connection with our licensing of intellectual property from third parties, including as discussed below.
We have collaborated with Biomics Biotechnologies Co., Ltd., or Biomics, pursuant to several collaboration agreements in relation to single-stranded RNA and shRNA sequences for treatment of hepatitis B. In July 2015, we entered into an earn-out agreement with Biomics which confirmed Benitec’s ownership of certain patents resulting from the collaboration in exchange for an upfront payment and equity issuance to Biomics and a share of certain future licensing revenue received by Benitec.
October 2020 Capital Raise
On October 6, 2020, the Company announced the closing of an underwritten public offering of common stock and common stock equivalents. The Company received gross proceeds of approximately $11.5 million and net proceeds of approximately $9.9 million from the offering.
April 2021 Capital Raise
On April 30, 2021, the Company announced the closing of an underwritten public offering of common stock and common stock equivalents. The Company received gross proceeds of approximately $14.3 million and net proceeds of approximately $12.7 million from the offering.
September 2022 Capital Raise
On September 15, 2022, the Company announced the closing of an underwritten public offering of common stock and common stock equivalents. The Company received gross proceeds of approximately $17.9 million and net proceeds of approximately $16.0 million from the offering.
Results of Operations
Revenues
The Company has not generated any revenues from the sales of products. Revenues from licensing fees are included in the revenue from customers line item on our consolidated statements of operations and comprehensive loss. Our licensing fees have been generated through the licensing of our ddRNAi technology to biopharmaceutical companies. The Company recognized licensing fee revenues totaling $68 thousand and $73 thousand during the nine months ended March 31, 2023 and March 31, 2022, respectively. Licensing fee revenue for the three-month periods ended March 31, 2023 and March 31, 2022 totaled $54 thousand and $48 thousand, respectively.
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Royalties and License Fees
Royalties and license fees consist primarily of payments we are required to remit for royalties and other payments related to in-licensed intellectual property. Under our in-license agreements, we may pay up-front fees and milestone payments and be subject to future royalties. We cannot precisely predict the amount, if any, of royalties we will owe in the future, and if our calculations of royalty payments are incorrect, we may owe additional royalties, which could negatively affect our results of operations. As our product sales increase, we may, from time to time, disagree with our third-party collaborators as to the appropriate royalties owed, and the resolution of such disputes may be costly, may consume management’s time, and may damage our relationship with our collaborators. Furthermore, we may enter into additional license agreements in the future, which may also include royalty, milestone and other payments.
Expenses
Research and Development Expenses
Research and development expenses relate primarily to the cost of conducting clinical and preclinical trials. Preclinical and clinical development costs are a significant component of research and development expenses. The Company records accrued liabilities for estimated costs of research and development activities conducted by third-party service providers, which include the conduct of preclinical studies and clinical trials, and contract manufacturing activities. The Company records the estimated costs of research and development activities based upon the estimated amount of services provided but not yet invoiced and includes these costs in trade and other payables on the consolidated balance sheets and within research and development expenses on the consolidated statements of operations and comprehensive loss.
The Company accrues for these costs based on factors such as estimates of the work completed and in accordance with agreements established with its third-party service providers. The Company makes significant judgments and estimates in determining the accrued liabilities balance at the end of each reporting period. As actual costs become known, the Company adjusts its accrued liabilities. The Company has not experienced any material differences between accrued costs and actual costs incurred.
General and Administrative Expenses
General and administrative expenses consist primarily of salaries, related benefits, travel, and equity-based compensation expense. General and administrative expenses also include facility expenses, professional fees for legal, consulting, accounting and audit services and other related costs.
We anticipate that our general and administrative expenses may increase as the Company focuses on the continued development of the preclinical OPMD program. The Company also anticipates 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.
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Operating Expenses
The following tables sets forth a summary of our expenses for each of the periods:
| Three Months Ended | Nine Months Ended | |||||||||||||||
| March 31, | March 31, | |||||||||||||||
| 2023 | 2022 | 2023 | 2022 | |||||||||||||
| (US$’000) | ||||||||||||||||
| Operating Expenses: |
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| Research and development |
$ | 3,167 | $ | 2,171 | $ | 9,588 | $ | 8,096 | ||||||||
| General and administrative |
1,228 | 1,337 | 5,011 | 5,093 | ||||||||||||
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| Total operating expenses |
$ | 4,395 | $ | 3,508 | $ | 14,599 | $ | 13,189 | ||||||||
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During the three and nine months ended March 31, 2023, we incurred $3.2 million and $9.6 million in research and development expenses, respectively, as compared to $2.2 million and $8.1 million for the comparable periods ended March 31, 2022. Research and development expenses relate primarily to the OPMD project. The year-over-year increases for the nine-month period reflect conclusion of the BB-301 Regulatory Toxicology Study and the Parallel Assay Method Development, Qualification, and Validation project, and the continuation of the GMP manufacturing project and Natural History Study.
General and administrative expense totaled $1.2 million and $5.0 million for the three and nine months ended March 31, 2023, compared to $1.3 million and $5.1 million for the comparable periods ended March 31, 2022. The year-over-year decrease for the three-month periods ended March 31 relates to lower listing and filing fees and stock-based compensation. See Note 9 of the Notes to Consolidated Financial Statements included in this Quarterly Report on Form 10-Q.
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Other Income (Loss)
The following tables sets forth a summary of our other income (loss) for each of the periods:
| Three Months Ended | Nine Months Ended | |||||||||||||||
| March 31, | March 31, | |||||||||||||||
| 2023 | 2022 | 2023 | 2022 | |||||||||||||
| (US$’000) | ||||||||||||||||
| Other Income (Loss): |
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| Foreign currency transaction gain (loss) |
$ | (45 | ) | $ | 229 | $ | (391 | ) | $ | 36 | ||||||
| Interest expense, net |
(7 | ) | (10 | ) | (25 | ) | (22 | ) | ||||||||
| Other income, net |
— | (29 | ) | 50 | (29 | ) | ||||||||||
| Unrealized loss on investment |
(4 | ) | (5 | ) | (4 | ) | (10 | ) | ||||||||
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| Total other income (loss), net |
$ | (56 | ) | $ | 185 | $ | (370 | ) | $ | (25 | ) | |||||
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Other income (loss), net during the three and nine months ended March 31, 2023, which consists of foreign currency transaction gain (loss), interest expense, other income, and unrealized loss on investment, totaled $(56) thousand and $(370) thousand, respectively. During the three and nine months ended March 31, 2022, respectively, other income (loss) totaled $185 thousand and $(25) thousand. The swings to foreign currency transaction losses during the three- and nine-month month periods ended March 31 reflect changes in foreign exchange rates. Other income for the nine-month period ended March 31, 2023 relates to recognition of a refund. Unrealized loss on investment decreased for the three- and nine- month periods ended March 31, 2023, compared to the same prior year periods.
Liquidity and Capital Resources
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 $163 million as of March 31, 2023. We expect that our research and development expenses may increase due to the continued development of the OPMD program. It is also likely that there will be an increase in the general and administrative expenses due to the obligations of being a domestic public company in the United States.
We had no borrowings as of March 31, 2023 and do not currently have a credit facility.
As of March 31, 2023, we had cash and cash equivalents of approximately $6.6 million. Cash in excess of immediate requirements is invested in accordance with our investment policy, primarily with a view to liquidity and capital preservation. Currently, our cash and cash equivalents are held in bank accounts.
The following table sets forth a summary of the net cash flow activity for each of the periods set forth below:
| Nine Months Ended | ||||||||
| March 31, | ||||||||
| 2023 | 2022 | |||||||
| (US$’000) | ||||||||
| Net cash provided by (used in): |
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| Operating activities |
$ | (13,917 | ) | $ | (11,044 | ) | ||
| Financing activities |
16,015 | — | ||||||
| Effects of exchange rate changes on cash and cash equivalents |
391 | (95 | ) | |||||
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| Net increase (decrease) in cash, cash equivalents, and restricted cash |
$ | 2,489 | $ | (11,139 | ) | |||
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Operating activities
Net cash used in operating activities for the nine months ended March 31, 2023 and 2022 was $13.9 million and $11.0 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, including an increase in payables.
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Financing activities
Net cash provided by financing activities was $16.0 million and $0 for the nine months ended March 31, 2023 and 2022, respectively. Cash from financing activities during the nine months ended March 31 2023, was related to the issuance of common stock, pre-funded warrants, and Series 2 warrants, including $17.9 million in gross proceeds from the September 2022 Capital Raise, partially offset by $1.9 million in share issuance costs.
The future of the Company as an operating business will depend on its ability to manage operating costs and budgeted amounts and obtain adequate financing. While we continue to progress discussions and advance opportunities to engage with pharmaceutical companies and continue to seek licensing partners for ddRNAi in disease areas that are not our focus, there can be no assurance as to whether we will enter into such arrangements or what the terms of any such arrangement could be.
While we have established some licensing arrangements, we do not have any products approved for sale and have not generated any revenue from product sales. We do not know when, or if, we will generate any revenue from product sales. We do not expect to generate significant revenue from product sales unless and until we obtain regulatory approval of and commercialize one of our current or future product candidates.
Unless and until we establish significant revenues from licensing programs, strategic alliances or collaboration arrangements with pharmaceutical companies, or from product sales, we anticipate that we will continue to generate losses for the foreseeable future, and we expect the losses to increase as we continue the development of product candidates and begin to prepare to commercialize any product that receives regulatory approval. We are subject to the risks inherent in the development of new gene therapy products, and we may encounter unforeseen expenses, difficulties, complications, delays, and other unknown factors that may adversely affect our business. The Company has performed a review of its cash flow forecasts and has concluded that substantial doubt exists as to its ability to continue as a going concern. The Company does not have sufficient liquidity to fund its operations for the next twelve months without raising additional funds and the success of raising such additional capital is not solely within the Company’s control.
We have based our projections of operating capital requirements on assumptions that may prove to be incorrect and we may use all of our available capital resources sooner than we expect. Because of the numerous risks and uncertainties associated with research, development, and commercialization of pharmaceutical products, we are unable to estimate the exact amount of our operating capital requirements. Our future funding requirements will depend on many factors, including, but not limited to:
| • | the timing and costs of our planned clinical trials for our ddRNAi and silence and replace product candidates; |
| • | the timing and costs of our planned preclinical studies for our ddRNAi and silence and replace product candidates; |
| • | the number and characteristics of product candidates that we pursue; |
| • | the outcome, timing, and costs of seeking regulatory approvals; |
| • | revenue received from commercial sales of any of our product candidates that may receive regulatory approval; |
| • | the terms and timing of any future collaborations, licensing, consulting, or other arrangements that we may establish; |
| • | the amount and timing of any payments we may be required to make, or that we may receive, in connection with the licensing, filing, prosecution, defense and enforcement of any patents or other intellectual property rights; |
| • | the costs of preparing, filing and prosecuting patent applications, maintaining and protecting our intellectual property rights and defending against intellectual property related claims; and |
| • | the extent to which we need to in-license or acquire other products and technologies. |
Contractual Obligations and Commercial Commitments
On October 1, 2016, the Company entered into an operating lease for office space in Hayward, California that originally expired in April 2018. The Company has entered into lease amendments that extended the lease through June 2025. See Note 8 of the Notes to Consolidated Financial Statements included in this Quarterly Report on Form 10-Q.
The Company enters into contracts in the normal course of business with third-party contract research organizations, contract development and manufacturing organizations and other service providers and vendors. These contracts generally provide for termination on notice and, therefore, are cancellable contracts and not considered contractual obligations and commitments.
Critical Accounting Policies and Significant Accounting Estimates
The preparation of consolidated financial statements and related disclosures in conformity with accounting principles generally accepted in the United States of America requires management to make judgments, assumptions and estimates that affect the amounts reported. Note 2 of the Notes to Consolidated Financial Statements included in this Quarterly Report on Form 10-Q describes the significant accounting policies used in the preparation of the consolidated financial statements. Certain of these significant accounting policies are considered to be critical accounting policies.
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A critical accounting policy is defined as one that is both material to the presentation of the Company’s consolidated financial statements and requires management to make difficult, subjective, or complex judgments that could have a material effect on the Company’s financial condition or results of operations. Specifically, these policies have the following attributes: (1) the Company is required to make assumptions about matters that are highly uncertain at the time of the estimate; and (2) different estimates the Company could reasonably have used, or changes in the estimate that are reasonably likely to occur, would have a material effect on the Company’s financial condition or results of operations.
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.
Management believes that the following are critical accounting policies:
Research and Development Expense
Research and development expenses relate primarily to the cost of conducting clinical and preclinical trials. Preclinical and clinical development costs are a significant component of research and development expenses. The Company records accrued liabilities for estimated costs of research and development activities conducted by third-party service providers, which include the conduct of preclinical studies and clinical trials, and contract manufacturing activities. The Company records the estimated costs of research and development activities based upon the estimated amount of services provided but not yet invoiced and includes these costs in trade and other payables on the consolidated balance sheets and within research and development expenses on the consolidated statements of operations and comprehensive loss.
The Company accrues for these costs based on factors such as estimates of the work completed and in accordance with agreements established with its third-party service providers. The Company makes significant judgments and estimates in determining the accrued liabilities balance at the end of each reporting period. As actual costs become known, the Company adjusts its accrued liabilities. The Company has not experienced any material differences between accrued costs and actual costs incurred.
Share-based Compensation Expense
The Company records share-based compensation in accordance with ASC 718, Stock Compensation. ASC 718 requires the fair value of all share-based employee compensation awarded to employees and non-employees to be recorded as an expense over the shorter of the service period or the vesting period. The Company determines employee and non-employee share-based compensation based on grant-date fair value using the Black-Scholes Option Pricing Model.
Recent Accounting Pronouncements
Accounting Standards recently adopted
None.
New Accounting Standards and Interpretations not yet mandatory or early adopted
ASU 2016-13-In June 2016, the FASB issued ASU No. 2016-13: “Financial Instruments-Credit Losses (Topic 326)”. This ASU represents a significant change in the accounting for credit losses model by requiring immediate recognition of management’s estimates of current expected credit losses (CECL). Under the prior model, losses were recognized only as they were incurred. The Company has determined that it has met the criteria of a smaller reporting company (“SRC”) as of November 15, 2019. As such, ASU 2019-10: “Financial Instruments-Credit Losses, Derivatives and Hedging, and Leases: Effective Dates” amended the effective date for the Company to be for reporting periods beginning after December 15, 2022. The Company will adopt this ASU effective July 1, 2023.
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| Item 3. | Quantitative and Qualitative Disclosures About Market Risk |
As a smaller reporting company, we are not required to provide the information pursuant to this Item.
| Item 4. | Controls and Procedures |
We have established disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended). As of the end of the period covered by this Report we carried out an evaluation under the supervision and with the participation of our management, including our principal executive officer and principal financial and accounting officer, of the effectiveness of our disclosure controls and procedures pursuant to Rule 13a-15 of the Securities and Exchange Act of 1934, as amended. Based upon that evaluation, our principal executive officer and principal financial and accounting officer concluded that our disclosure controls and procedures are effective.
There were no changes in our internal controls over financial reporting during the quarter ended March 31, 2023 that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.
We do not expect that our disclosure controls and procedures or our internal controls will prevent all errors and all fraud. A control system, no matter how well conceived and operated, can provide only reasonable, not absolute, assurance that the objectives of the control system are met. Further, the design of a control system must reflect the fact that there are resource constraints, and the benefits of controls must be considered relative to their costs. Because of the inherent limitations in all control systems, no evaluation of controls can provide absolute assurance that all control issues and instances of fraud, if any, have been detected.
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PART II
OTHER INFORMATION
| Item 1. | Legal Proceedings |
We are currently not a party to any material legal proceedings.
| Item 1A. | Risk Factors |
There have been no material changes to the risk factors disclosed in Item 1A of the Company’s Annual Report on Form 10-K for the fiscal year ended June 30, 2022 other than as set forth below.
We may fail to continue to meet the listing standards of The Nasdaq Capital Market and our failure to maintain the listing of our common stock with a U.S. national securities exchange could adversely affect the liquidity off our common stock.
Our common stock is currently listed on The Nasdaq Capital Market. In order to maintain that listing, we must satisfy minimum financial and other continued listing requirements and standards, including maintaining a minimum share price. For example, the current continued listing requirements of Nasdaq provide, among other things, that a company may be delisted if the bid price of its stock drops below $1.00 for a period of 30 consecutive business days.
On September 6, 2022, we received a letter from the Listing Qualifications Department of the Nasdaq Stock Market notifying us that the minimum bid price per share for our common stock fell below $1.00 for a period of 30 consecutive business days and that therefore we did not meet the minimum bid price requirement set forth in Nasdaq Listing Rule 5550(a)(2). The letter stated that we will be provided 180 calendar days, or until March 6, 2023 to regain compliance with the minimum bid price requirement.
On March 7, 2023, Nasdaq notified the Company in writing that while the Company had not regained compliance with the minimum bid price requirement, it was eligible for an additional 180 calendar day period, or until September 5, 2023, to regain compliance with the minimum bid price requirement. Nasdaq’s determination was based on the Company having met the continued listing requirement for market value of publicly held shares and all other applicable requirements for initial listing on The Nasdaq Capital Market, with the exception of the minimum bid price requirement and on the Company’s written notice to Nasdaq of its intention to cure the deficiency during the second compliance period by effecting a reverse stock split, if necessary. If at any time during this additional time period the closing bid price of the Company’s common stock is at least $1.00 per share for a minimum of 10 consecutive business days, Nasdaq will provide written confirmation of compliance and the matter will be closed.
If the Company does not regain compliance by September 5, 2023, then Nasdaq will provide written notification that the Company’s common stock will be delisted, at which point the Company may appeal Nasdaq’s determination to a Hearings Panel. The Company would remain listed on Nasdaq pending the Hearings Panel’s decision. There can be no assurance that, if the Company does appeal the delisting determination by Nasdaq to the Hearings Panel, that such appeal would be successful.
In the event of a delisting, we would take actions to restore our compliance with Nasdaq’s listing requirements, but we can provide no assurance that any such action taken by us would allow our common stock to become listed again, stabilize the market price or improve the liquidity of our securities, prevent our common stock from dropping below the Nasdaq minimum share price requirement or prevent future non-compliance with Nasdaq’s listing requirements.
We intend to monitor the closing bid price of our common stock and may, if appropriate, consider implementing available options to regain compliance with the minimum bid price requirement. These options include completing a reverse stock split of our common stock for the purpose of meeting the closing bid price requirement. Our stockholders approved a reverse stock split at our stockholder meeting on December 7, 2022.
If our common stock were to be delisted from Nasdaq, our common stock could begin to trade on one of the markets operated by OTC Markets Group, including OTCQX, OTCQB or OTC Pink (formerly known as the “pink sheets”), as the case may be. In such event, our common stock could be subject to the “penny stock” rules which, among other things, require brokers or dealers to approve investors’ accounts, receive written agreements and determine investor suitability for transactions and disclose risks relating to investing in the penny stock market. Any such delisting of our common stock could have an adverse effect on the market price of, and the efficiency of the trading market for our common stock, not only in terms of the number of shares that can be bought and sold at a given price, but also through delays in the timing of transactions and less coverage of us by securities analysts, if any. Also, if in the future we were to determine that we need to seek additional equity capital, it could have an adverse effect on our ability to raise capital in the public or private equity markets. In addition, there can be no assurance that our common stock would be eligible for trading on any such alternative exchange or markets.
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We will need to continue our efforts to raise additional funding, which may not be available on acceptable terms, or at all. Failure to obtain capital when needed may negatively impact our ability to continue as a going concern.
Developing ddRNAi products is expensive, and we expect our research and development expenses to increase substantially in connection with our ongoing activities, particularly as we advance our product candidates in preclinical studies and in future clinical trials and as we undertake preclinical studies of new product candidates. As of March 31, 2023, our cash and cash equivalents were $6.6 million. We estimate that without additional financing, our existing cash and cash equivalents will not be sufficient to fund our operations for the next twelve months and the success of raising such additional capital is not solely within the Company’s control. Our operating plan may require changes and delays as a result of such liquidity issues. We may need to seek additional funds sooner than planned, through public or private equity or debt financings, government grants or other third-party funding, strategic alliances and licensing arrangements or a combination of these approaches. In addition, because the length of time and activities associated with successful development of our product candidates is highly uncertain, we are unable to estimate the actual funds we will require for development and any approved marketing and commercialization activities. In any event, we will require additional capital to obtain regulatory approval for our product candidates and to commercialize any product candidates that receive regulatory approval.
Any additional fundraising efforts may divert our management from their day-to-day activities, which may compromise our ability to develop and commercialize our product candidates. In addition, we cannot guarantee that future financing will be available in sufficient amounts or on terms acceptable to us, if at all. Moreover, the terms of any financing may adversely affect the holdings or the rights of our shareholders, and the issuance of additional securities, whether equity or debt, by us, or the possibility of such issuance, may cause the market price of our common stock to decline. If we incur indebtedness we may be required to agree to restrictive covenants, such as limitations on our ability to incur additional debt, limitations on our ability to acquire, sell or license intellectual property rights and other operating restrictions that could compromise our ability to conduct our business. We could also seek financing through arrangements with collaborative partners at an earlier stage than would otherwise be desirable and we may be required to relinquish rights to some or all of our technologies or product candidates or otherwise agree to terms unfavorable to us.
If we are unable to obtain funding on a timely basis or on acceptable terms, we may be required to significantly curtail, delay or discontinue one or more of our research or development programs or the commercialization of any approved product candidates.
| Item 2. | Unregistered Sales of Equity Securities and Use of Proceeds |
None.
| Item 3. | Defaults Upon Senior Securities |
None.
| Item 4. | Mine Safety Disclosures |
None.
| Item 5. | Other Information |
None.
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| Item 6. | Exhibits. |
| Number | Description of Document | |
| 31.1 | Statement of CEO Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002* | |
| 31.2 | Statement of CFO Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002* | |
| 32.1 | Statement of CEO Pursuant to Section 906 of the Sarbanes-Oxley Act of 2002** | |
| 32.2 | Statement of CFO Pursuant to Section 906 of the Sarbanes-Oxley Act of 2002** | |
| 101.INS | Inline XBRL Instance Document* | |
| 101.SCH | Inline XBRL Taxonomy Extension Schema Document* | |
| 101.CAL | Inline XBRL Calculation Linkbase Document* | |
| 101.DEF | Inline XBRL Taxonomy Extension Definition Linkbase Document* | |
| 101.LAB | Inline XBRL Label Linkbase Document* | |
| 101.PRE | Inline XBRL Taxonomy Presentation Linkbase Document* | |
| 104 | Cover Page Interactive Data File—the cover page interactive data file does not appear in the Interactive Data File because its XBRL tags are embedded within the Inline XBRL document | |
| * | Filed herewith. |
| ** | Furnished, not filed. |
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SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has duly caused this report to be signed on our behalf by the undersigned thereunto duly authorized.
| Benitec Biopharma Inc. | ||||||
| Dated: May 15, 2023 | /s/ Jerel Banks |
|||||
| Jerel Banks | ||||||
| Executive Chairman and Chief Executive Officer | ||||||
| (principal executive officer) | ||||||
| /s/ Megan Boston |
||||||
| Megan Boston | ||||||
| Executive Director (principal financial and accounting officer) | ||||||
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