BNTC Benitec Biopharma Inc.

3.25
0  0%
Previous Close 3.25
Open 3.2
52 Week Low 2.3
52 Week High 10.49
Market Cap $26,557,993
Shares 8,171,690
Float 8,161,493
Enterprise Value $11,784,992
Volume 7,545
Av. Daily Volume 138,052
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Drug Pipeline

Drug Stage Notes
BB-301
Oculopharyngeal muscular dystrophy (OPMD)
Phase 1
Phase 1
Phase 1 data provide continued support for the Phase 1b/2a study and Phase 1b/2a trial to be initiated in 2022.
TT-034
Hepatitis C
Phase 1/2
Phase 1/2
Announced late February 2016 plans to discontinue development

Latest News

  1. HAYWARD, Calif., Nov. 15, 2021 /PRNewswire/ -- Benitec Biopharma Inc. (NASDAQ:BNTC) ("Benitec" or "the Company"), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on the proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced the financial results for its Fiscal Year Q1 ended September 30, 2021. The Company has filed its quarterly report on Form 10-Q for the quarter ended September 30, 2021, with the U.S. Securities and Exchange Commission.  

    Operational Updates

    The key milestones related to the investigational agents under development by the Company and other corporate updates are outlined below:

    BB-301 (Oculopharyngeal Muscular Dystrophy Program) 

    • On September 8, 2021, Benitec provided three key updates related to the progress of the BB-301 development program, including: updated results for the BB-301 Pilot Dosing Study in large animals, updates on European and North American regulatory interactions for the BB-301 development program, and a comprehensive overview of the design of, and key primary and secondary endpoints for, the Phase 1b/2a clinical trial which is planned for initiation in 2022. All of the updates were positive and demonstrated the significant progress that has been achieved for the BB-301 development program; below is a summary of each update:
      • BB-301 Pilot Dosing Study in Large Animals: On September 8th the Company disclosed updated analyses for the animal subjects dosed with BB-301 in the Pilot Dosing Study. The updated data continued to demonstrate dose-dependent transduction of the target pharyngeal muscle tissues by BB-301, dose-dependent gene expression for the three distinct components of the therapeutic BB-301 transgene, and biologically significant levels of gene silencing ("knock-down") of the target PABPN1 protein. These updated data provide continued support for the planned advancement of BB-301 into the Phase 1b/2a clinical study in 2022.
      • Regulatory Interactions in Europe: Following the disclosure in February 2021 of the positive interim data from the BB-301 Pilot Dosing Study in large animals, Benitec completed a 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") in the first half of 2021. At the conclusion of the meeting:
        • The BB-301 Pilot Dosing Study in large animals was viewed as an appropriate dose range finding study.
        • The design of the ongoing GLP Toxicology and Biodistribution study was viewed as appropriate to support Phase 1b/2a testing of BB-301.
        • The manufacturing plan for clinical grade BB-301 drug product can be conducted under GMP conditions with a production process analogous to that employed in prior large-scale production runs for BB-301.
        • The design of the Phase 1b/2a clinical trial can support the evaluation of BB-301 safety and clinical efficacy in key populations of OPMD patients.
      • Regulatory Interactions in the United States: Benitec has been granted a Type C Meeting with the U.S. Food and Drug Administration ("FDA") in the fourth quarter of 2021.
      • Regulatory Interactions in Canada: Benitec has been granted a Pre-CTA Consultation Meeting with Health Canada in the fourth quarter of 2021.
      • BB-301 Phase 1b/2a Clinical Study Design: On September 8th the Company provided a comprehensive overview of the key design elements of the upcoming BB-301 Phase 1b/2a clinical trial. The Phase 1b/2a study is planned for 2022. In addition to the determination of the safety and tolerability profiles of BB-301, the secondary endpoints of the trial will facilitate the accurate and reproducible characterization of the key physiological processes underlying the successful completion of the pharyngeal phase of swallowing. The core analytical tools and methods that will be employed during the clinical study will focus on functional measures of swallowing efficiency for OPMD patients during the pharyngeal phase of swallowing.

    Financial Highlights

    Total Revenues for the quarter ended September 30, 2021 were $0 compared to fifty-five thousand dollars in total revenue for three months ended September 30, 2020. The decrease in revenues from customers is due to the decrease in licensing and royalty revenues in the current period.

    Total Operating Expenses were $4.8 million for the quarter ended September 30, 2021 compared to $2.7 million for the comparable period in 2020. For the quarters ended September 30, 2021 and 2020, respectively, Benitec incurred $0 and $134 thousand in royalties and license fees. During the three months ended September 30, 2021 and 2020, respectively, the Company incurred $2.8 million and $0.8 million in research and development expenses. The increase in research and development expenses is primarily related to the commencement of the BB-301 GLP Toxicology and Biodistribution Study in large animals at Charles River Laboratories. Additionally, the Company incurred expenses related to the production of GMP-grade drug product to facilitate submissions of the Clinical Trial Applications outside of the United States and the Investigational New Drug Application in the United States, as these activities are critical to the planned initiation of the Phase 1b/2a clinical trial for BB-301 in 2022. General and administrative expenses were $2.0 million and $1.8 million for the three months ended September 30, 2021 and 2020, respectively. The increase during this three-month period was due to small increases in insurance costs, consultant fees, and legal and accounting fees.

    Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec Biopharma commented, "Our team continues to focus on the core development activities that will ensure the advancement of BB-301 into the first-in-human study. Our primary goal has been, and will continue to be, the improvement of the lives of patients suffering from genetic disorders for which no curative interventions exist. We believe that the initiation of the Phase 1b/2a study is critical for patients in the Oculopharyngeal Muscular Dystrophy community and represents an important milestone with respect to our long-term goals as a company."  

    PART I – FINANCIAL INFORMATION

    ITEM 1. Financial Statements



    BENITEC BIOPHARMA INC.

    Consolidated Balance Sheets

    (in thousands, except par value and share amounts)

























    September 30,





    June 30,







    2021





    2021







    (Unaudited)









    Assets

















    Current assets:

















    Cash and cash equivalents



    $

    15,727





    $

    19,769



    Trade and other receivables





    3







    25



    Prepaid and other assets





    642







    814



    Total current assets





    16,372







    20,608



    Property and equipment, net





    323







    375



    Deposits





    25







    9



    Other assets





    169







    185



    Right-of-use assets





    947







    202



    Total assets



    $

    17,836





    $

    21,379



    Liabilities and Stockholders' Equity

















    Current liabilities:

















    Trade and other payables



    $

    1,106





    $

    880



    Accrued employee benefits





    301







    276



    Lease liabilities, current portion





    194







    213



    Total current liabilities





    1,601







    1,369



    Lease liabilities, less current portion





    760









    Total liabilities





    2,361







    1,369



    Commitments and contingencies (Note 12)

















    Stockholders' equity:

















    Common stock, $0.0001 par value—10,000,000 shares authorized; 8,171,690 shares issued and outstanding at September 30, 2021 and June 30, 2021, respectively





    1







    1



    Additional paid-in capital





    151,854







    151,583



    Accumulated deficit





    (135,164)







    (130,119)



    Accumulated other comprehensive loss





    (1,216)







    (1,455)



    Total stockholders' equity





    15,475







    20,010



    Total liabilities and stockholders' equity



    $

    17, 836





    $

    21,379























    The accompanying notes are an integral part of these consolidated financial statements.

     

    BENITEC BIOPHARMA INC.

    Consolidated Statements of Operations and Comprehensive Loss

    (Unaudited)

    (in thousands, except share and per share amounts)

























    Three Months Ended September 30,







    2021





    2020



    Revenue:

















    Revenues from customers



    $





    $

    55



    Total revenues











    55



    Operating expenses

















    Royalties and license fees











    134



    Research and development





    2,780







    774



    General and administrative





    2,042







    1,837



    Total operating expenses





    4,822







    2,745



    Loss from operations





    (4,822)







    (2,690)



    Other income (loss):

















    Foreign currency transaction loss





    (240)







    (54)



    Interest expense, net





    (1)







    (1)



    Other income, net











    27



    Unrealized gain on investment





    18









    Total other loss, net





    (223)







    (28)



    Net loss



    $

    (5,045)





    $

    (2,718)



    Other comprehensive income:

















    Unrealized foreign currency translation gain





    239







    178



    Total other comprehensive income





    239







    178



    Total comprehensive loss



    $

    (4,806)





    $

    (2,540)



    Net loss



    $

    (5,045)





    $

    (2,718)



    Net loss per share:

















    Basic and diluted



    $

    (0.62)





    $

    (2.45)



    Weighted-average shares outstanding:

















    Basic and diluted





    8,171,690







    1,108,374























    The accompanying notes are an integral part of these consolidated financial statements.

    About Benitec Biopharma Inc.

    Benitec Biopharma Inc. ("Benitec" or the "Company") 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 ddRNAi-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD), and Chronic Hepatitis B. A comprehensive overview of the Company can be found on Benitec's website at www.benitec.com.

    Forward Looking Statements

    Except for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec's plans to develop and commercialize its product candidates, the timing of the initiation and completion of preclinical and clinical trials, the timing of patient enrolment and dosing in clinical trials, the timing of expected regulatory filings, the clinical utility and potential attributes and benefits of ddRNAi and Benitec's product candidates, potential future out-licenses and collaborations, the intellectual property position and the ability to procure additional sources of financing, and other forward-looking statements.

    These forward-looking statements are based on the Company's current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities; the Company's ability to protect and enforce its patents and other intellectual property rights; the Company's dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Company's products and the products of the Company's collaboration partners; the acceptance of the Company's products and the products of the Company's collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the Company's ability to satisfy its capital needs through increasing its revenue and obtaining additional financing; the impact of the current COVID-19 pandemic, the disease caused by the SARS-CoV-2 virus, which may adversely impact the Company's business and preclinical and future clinical trials; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company's reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements. 

    Media & Investor Relations Contact:

    Jay A. Morakis

    CEO of M Group Strategic Communications (for Benitec Biopharma, Inc.)

    Phone: 646-859-5951

    Email:

     

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/benitec-biopharma-discloses-q1-2022-financial-results-301423819.html

    SOURCE Benitec Biopharma Inc.

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  2. HAYWARD, Calif., Sept. 20, 2021 /PRNewswire/ -- Benitec Biopharma Inc. (NASDAQ:BNTC) ("Benitec" or "the Company"), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on the proprietary DNA-directed RNA interference ("ddRNAi") platform, today provided an operational update and announced the financial results for its fiscal year ended June 30, 2021. The Company has filed its annual report on Form 10-K for the year ended June 30, 2021, with the U.S. Securities and Exchange Commission.  

    Operational Updates

    The key milestones related to the investigational agents under development by the Company and other corporate updates are outlined below:

    BB-301 (Oculopharyngeal Muscular Dystrophy Program) 

    • On September 8, 2021, Benitec announced three key updates related to the progress of the BB-301 development program, including: updated results for the BB-301 Pilot Dosing Study in large animals, updates on European and North American regulatory interactions for the BB-301 development program, and a comprehensive overview of the design of, and key primary and secondary endpoints for, the Phase 1b/2a clinical trial which is planned for initiation in 2022. All of the updates were positive and demonstrated the significant progress that has been achieved for the BB-301 development program; below is a summary of each update:
      • BB-301 Large Animal Pilot Dosing Study: On September 8th the Company disclosed updated analyses that continue to demonstrate robust, dose-dependent target tissue transduction for BB-301, dose-dependent gene expression for the three distinct components of the therapeutic transgene, and biologically significant knock-down of the target PABPN1 protein. These updated data provide continued support for the planned advancement of BB-301 into the Phase 1b/2a clinical study in 2022.
      • European Regulatory Interaction: Following the disclosure in February 2021 of the positive interim data from the BB-301 Pilot Dosing Study in large animals, Benitec completed a 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") in the first half of 2021. At the conclusion of the meeting:
        • The BB-301 Pilot Dosing Study was viewed as an appropriate dose range finding study.
        • The design of the ongoing GLP Biodistribution and Toxicology study was viewed as appropriate to support Phase 1b/2a testing of BB-301.
        • The manufacturing plan for clinical grade BB-301 drug product can be conducted under GMP conditions with a production process analogous to that that employed in prior large-scale production runs for BB-301.
        • The design of the Phase 1b/2a clinical trial can support the evaluation of BB-301 safety and clinical efficacy in key populations of OPMD patients.
      • North American Regulatory Interaction: Benitec has been granted a Type C Meeting with the U.S. Food and Drug Administration ("FDA") in the fourth quarter of 2021.
      • BB-301 Phase 1b/2a Clinical Study Design: On September 8th the Company provided a comprehensive overview of the key design elements of the upcoming BB-301 Phase 1b/2a clinical trial. The Phase 1b/2a study is planned for 2022. In addition to the determination of the safety and tolerability profiles of BB-301, the secondary endpoints of the trial will facilitate the accurate and reproducible characterization of the key physiological processes underlying the successful completion of the pharyngeal phase of swallowing. The core analytical tools and methods that will be employed during the clinical study will focus on functional measures of swallowing efficiency for OPMD patients during the pharyngeal phase of swallowing.

    Corporate Updates

    • On April 30, 2021, the Company announced the closing of an underwritten public offering of common stock and common stock equivalents (the "April 2021 Capital Raise"). The Company received gross proceeds of approximately $14.3 million and net proceeds of approximately $12.7 million from the offering.
    • On October 6, 2020, the Company announced the closing of an underwritten public offering of common stock and common stock equivalents (the "October 2020 Capital Raise"). The Company received gross proceeds of approximately $11.5 million and net proceeds of approximately $9.9 million from the offering.

    Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec Biopharma commented, "Our team has made significant progress towards the goal of advancing BB-301 into clinical studies, and we remain focused on improving the lives of patients suffering from Oculopharyngeal Muscular Dystrophy. In the near future, we will begin to apply the ddRNAi platform to the treatment of other fatal genetic disorders with significant unmet medical need."

    Financial Highlights

    Total Revenues for the year ended June 30, 2021 were fifty-nine thousand dollars compared to $102,000 for the year ended June 30, 2020. Benitec recognized fifty-nine thousand dollars in customer revenues for the year ended June 30, 2021 compared to $97,000 for the comparable year ended June 30, 2020. The decrease in revenues from customers is due to the decrease in licensing and royalty revenues in the current year. During the year ended June 30, 2021, the Company recognized no revenue from government research and development grants, as compared $5 million for the comparable year ended June 30, 2020. The decrease in grant revenue is a result of Benitec no longer claiming the grant from the Australian government due to the Re-domiciliation of the Company to the United States of America.

    Total expenses were $13.7 million for the year ended June 30, 2021 compared to $8.4 million for the comparable period in 2020. Royalty and license fees, research and development costs, and general and administrative costs comprise the primary corporate expenses. For the year ended June 30, 2021, Benitec incurred one hundred twenty-three thousand dollars in royalties and license fees compared to a gain of one hundred eighty-five thousand dollars for the comparable year ended June 30, 2020. The change is primarily due to a reversal of an accrual which created the negative balance for the year ended June 30, 2020. Benitec incurred $7 million of research and development expenses compared to $3 million for the comparable year ended June 30, 2020. The increase in research and development expenses are related to the pre-clinical studies associated with BB-301 as well as an increase in research and development costs related to stock-based compensation expenses. General and administrative expenses were $6.5 million and $5.5 million for the years ended June 30, 2021 and 2020, respectively. The increase for the year was due to the increase in insurance, consultants, legal and accounting fees.

    The loss from operations for fiscal 2021 was $13.9 million compared to a loss of $8.3 million for fiscal 2020. Net loss attributable to shareholders for the fiscal year ended 2021 was $13.9 million, or $3.23 per basic and diluted share, compared to a net loss of $8.3 million, or $8.10 per basic and diluted share in earnings for the fiscal year ended 2020. At the end of fiscal year 2021 the Company had $19.8 million in cash and cash equivalents. 

    BENITEC BIOPHARMA INC.

    Consolidated Statements of Operations and Comprehensive Loss

    (in thousands, except share and per share amounts)





    Year Ended June 30,



    2021



    2020

    Revenue:

         
    Revenues from customers

    $                 59



     

    $               97

              Government research and development grants



    5

        Total revenues

    59



    102

    Operating expenses

         
    Royalties and license fees

     

    123



     

    (185)

              Research and development

    7,020



    3,001

              General and administrative

    6,512



    5,567

    Total operating expenses

    13,655



    8,383

    Loss from operations

    (13,596)



    (8,281)

    Other income (loss):

          
    Foreign currency transaction loss

     

    (333)



     

    (88)

           Interest income (expense), net

    (6)



    62

           Other income, net

    37



    34

           Unrealized gain (loss) on investment

    16



    (1)

       Total other income (loss), net

    (286)



    7

      Net loss

    $         (13,882)



    $         (8,274)

    Other comprehensive income (loss):

          
    Unrealized foreign currency translation gain (loss)

     

    498



     

    (89)

    Total other comprehensive income (loss)

    498



    (89)

    Total comprehensive loss

    $ (13,384)



    $         (8,363)

       Net loss

    $ (13,882)



    $         (8,274)

    Net loss per share:

          
    Basic and diluted 

     

    $             (3.23)



     

    $           (8.10)

    Weighted-average shares outstanding:

           Basic and diluted

    4,295,416



    1,021,193



    The accompanying notes are an integral part of these consolidated financial statements

     

    BENITEC BIOPHARMA INC.

    Consolidated Balance Sheets

    (in thousands, except par value and share amounts)





    June 30,

    2021



    June 30,

    2020

    Assets







    Current assets:







           Cash and cash equivalents

    $    19,769



    $      9,801

           Trade and other receivables

    25



    59

           Prepaid and other assets

    814



    949

    Total current assets

    20,608



    10,809

    Property and equipment, net

    375



    374

       Deposits

    9



    9

    Other assets

    185



       Right-of-use assets

    202



    395

    Total assets  

    $ 21,379



    $ 11,587

    Liabilities and Stockholders' Equity







    Current liabilities:







           Trade and other payables

    $         880



    $        741

           Accrued employee benefits

    276



    203

           Lease liabilities, current portion

    213



    192

    Total current liabilities

    1,369



    1,136

    Lease liabilities, less current portion



    213

    Total liabilities

    1,369



    1,349

    Commitments and contingencies (Note 12)







    Stockholders' equity:







    Common stock, $0.0001 par value—10,000,000 shares authorized; 8,171,690 and

        1,108,374 shares issued and outstanding at June 30, 2021 and 2020,

        respectively

    1



    0

             Additional paid-in capital

    151,583



    128,827

             Accumulated deficit

    (130,119)



    (116,636)

             Accumulated other comprehensive loss

    (1,455)



    (1,953)

    Total stockholders' equity

    20,010



    10,238

    Total liabilities and stockholders' equity

    $    21,379



    $    11,587



    The accompanying notes are an integral part of these consolidated financial statements.

    About Benitec Biopharma Inc.

    Benitec Biopharma Inc. ("Benitec" or the "Company") 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 ddRNAi-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD), and Chronic Hepatitis B. A comprehensive overview of the Company can be found on Benitec's website at www.benitec.com.

    Forward Looking Statements

    Except for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec's plans to develop and commercialize its product candidates, the timing of the initiation and completion of preclinical and clinical trials, the timing of patient enrolment and dosing in clinical trials, the timing of expected regulatory filings, the clinical utility and potential attributes and benefits of ddRNAi and Benitec's product candidates, potential future out-licenses and collaborations, the intellectual property position and the ability to procure additional sources of financing, and other forward-looking statements.

    These forward-looking statements are based on the Company's current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities; the Company's ability to protect and enforce its patents and other intellectual property rights; the Company's dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Company's products and the products of the Company's collaboration partners; the acceptance of the Company's products and the products of the Company's collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the Company's ability to satisfy its capital needs through increasing its revenue and obtaining additional financing; the impact of the current COVID-19 pandemic, the disease caused by the SARS-CoV-2 virus, which may adversely impact the Company's business and preclinical and future clinical trials; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company's reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements.

    Media & Investor Relations Contact:

    Jay A. Morakis

    CEO of M Group Strategic Communications (for Benitec Biopharma, Inc.)

    Phone: 646-859-5951

    Email:

     

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/benitec-biopharma-provides-operational-update-and-releases-its-2021-fiscal-year-end-financial-results-301380191.html

    SOURCE Benitec Biopharma Inc.

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  3. HAYWARD, Calif., Sept. 13, 2021 /PRNewswire/ -- Benitec Biopharma, Inc. (NASDAQ:BNTC), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on the proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced that Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec Biopharma will present at the H.C. Wainwright 23rd Annual Global Investment Conference. The relevant details are outlined below: 

    Date: 

    Wednesday, September 13, 2021

    Event:

    H.C. Wainwright 23rd Annual Global Investment Conference

    Time:

    7:00 P.M. ET

    Link:

    www.hcwevents.com

    Please visit the link above to register for the presentation which will be presented via webcast during the virtual conference. The September 2021 Corporate Presentation is also viewable on the Investor Relations section of the Company's website, here: https://ir.benitec.com/company-information/presentations

    If you are an institutional investor, and would like to attend the Company's presentation, click on the following link (www.hcwevents.com) to register. Once your registration is confirmed, you will be prompted to log into the conference website to request a one-on-one meeting with the Company.

    About Benitec Biopharma, Inc.

    Benitec Biopharma, Inc. ("Benitec" or the "Company") 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 ddRNAi-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD), and Chronic Hepatitis B. A comprehensive overview of the Company can be found on Benitec's website at www.benitec.com.

    Forward Looking Statement

    Except for the historical information set forth herein, the matters set forth in this press release represent forward-looking statements, including statements regarding Benitec's plans to develop and commercialize its product candidates, the timing of the initiation and completion of preclinical and clinical trials, the timing of patient enrolment and dosing in clinical trials, the timing of expected regulatory filings, the clinical utility and potential attributes and benefits of ddRNAi and Benitec's product candidates, potential future out-licenses and collaborations, the intellectual property position and the ability to procure additional sources of financing, and other forward-looking statements.

    These forward-looking statements are based on the Company's current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA; the Company's dependence on its relationships with its collaboration partners; the efficacy or safety of the Company's products and the products of the Company's collaboration partners; the acceptance of the Company's products and the products of the Company's collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; and other risks detailed from time to time in the Company's reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements.

    Media & Investor Relations Contact:

    Jay A. Morakis

    CEO of M Group Strategic Communications (for Benitec Biopharma, Inc.)

    Phone: 646-859-5951

    Email:

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/benitec-biopharma-to-present-at-hc-wainwrights-23rd-annual-global-investment-conference-301375670.html

    SOURCE Benitec Biopharma Inc.

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  4. HAYWARD, Calif., Sept. 8, 2021 /PRNewswire/ -- Benitec Biopharma Inc. (NASDAQ:BNTC), a development-stage biotechnology company focused on the advancement of novel genetic medicines, today provided an overview of the key elements of the BB-301 Phase 1b/2a clinical trial design. The clinical trial is planned for 2022.

    BB-301 is a novel investigational gene therapy under development for the treatment of patients with Oculopharyngeal Muscular Dystrophy (OPMD). OPMD is a chronic, life-threatening genetic disorder affecting approximately 15,000 patients in the United States, Canada, Western Europe, and Israel.  OPMD is caused by a mutation in the gene encoding poly(A) binding protein nuclear 1 (PABPN1). Patients with OPMD lose the ability to swallow liquids and solids, and the natural history of the disorder is characterized by chronic malnutrition, aspiration, and fatal episodes of aspiration pneumonia.  Currently, no therapeutic agents are approved for the treatment of OPMD. Additionally, no surgical interventions capable of altering the long-term natural history of OPMD are available. 

    Goal of the BB-301 Phase 1b/2a Study:

    • Benitec is focused on the accurate and reproducible characterization of the key physiological processes underlying the successful completion of the pharyngeal phase of swallowing
    • In this regard, the core analytical tools and methods employed during the clinical study will focus on functional measures of swallowing efficiency for OPMD patients during the pharyngeal phase of swallowing

    Summary and Background for the BB-301 Phase 1b/2a Study:

    Study Title:

    A Phase 1b/2a, Open-Label Dose Escalation Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects with OPMD

    Study Rationale:

    The purpose of this clinical investigation is to evaluate the safety, tolerability, and clinical activity of ascending doses of BB-301 administered to subjects with OPMD via local intramuscular (IM) injection into the middle pharyngeal constrictor (MPC) muscles and the inferior pharyngeal constrictor (IPC) muscles following open surgical dissection of the pharyngeal region under general anesthesia.

    Study Design:

    This Phase 1b/2a, first-in-human (FIH) study is a single arm, open-label, sequential, dose escalation cohort study with a six-month pre-treatment observation period and a 52-week post injection follow-up period in up to 24 subjects diagnosed with OPMD.

    BB-301 will be administered in three sequential escalating dose cohorts. After the initial three cohorts have been dosed, additional subjects may be added at the Maximally Effective Dose (MED), or at an alternate dose as determined by review of cumulative safety and efficacy data.

    At 76 weeks all subjects will be enrolled into a long-term safety follow-up study.

    Criteria for Study Subject Evaluation:

    Primary Endpoint (assessed at screening and through end of treatment):

    • To assess the safety and tolerability of ascending IM doses of BB-301 administered locally to the MPC muscles and the IPC muscles of subjects with OPMD following open surgical dissection of the pharyngeal region under general anesthesia

    Secondary Endpoints (assessed at Day -180, Day -90, Day -2, Day 90, Day 180, Day 270, Day 360):

    • To evaluate the impact of ascending IM doses of BB-301 on key functional elements of the pharyngeal phase of swallowing as measured by the following videofluoroscopic assessments:
      • Global swallowing function as measured by the Dynamic Imaging Swallowing Toxicity (DIGEST) scale
      • Pharyngeal constrictor muscle force generation as measured by the Pharyngeal Area at Maximum Constriction (PAMC)
      • Pharyngeal constrictor muscle force generation as measured by the Pharyngeal Constriction Ratio (PCR)
    • To evaluate the impact of ascending IM doses of BB-301 on dysphagia severity as measured by the cold-water timed drinking test
    • To evaluate the impact of ascending IM doses of BB-301 on patient reported dysphagia (Sydney Swallow Questionnaire)
    • To evaluate the impact of ascending IM doses of BB-301 on functional oral intake (International Dysphagia Diet Standardization Initiative Functional Diet Scale)

    Rationale for the Use of Videofluoroscopy to Assess Core Metrics of Pharyngeal Function and Pharyngeal Constrictor Muscle Strength:

    • The key Secondary Endpoints outlined above will rely on the implementation of several videofluoroscopy-based methods
      • Videofluoroscopy (or modified barium swallow) is viewed as the gold standard for the assessment of swallowing function
    • Recall that the core biological outcome of the BB-301 proof-of-concept studies in the A17 mouse model (Strings-Ufombah, V., et al., Molecular Therapy: Nucleic Acids, 2021; Malerba, A., et al. Nature Communications, 2017) was the increase in the cross-sectional area, and the increase in strength, of the skeletal muscles injected with BB-301
      • In this regard, Benitec's primary goal with respect to the functional assessment of OPMD patients enrolled onto the Phase 1b/2a study is to accurately and reproducibly document the impact of the administration of BB-301 on the strength and function of the injected pharyngeal constrictor muscles
    • In the Phase 1b/2a study Benitec endeavors to use analytical methods that directly measure, or are robust surrogates for, pharyngeal constrictor muscle strength and swallowing efficiency for OPMD patients during the pharyngeal phase of swallowing
      • The videofluoroscopy-based methods outlined above (and further detailed below) have been demonstrated to measure the core biological and functional attributes that Benitec aims to address with the administration of BB-301 (i.e. improved efficiency of the pharyngeal phase of swallowing via increases in pharyngeal constrictor muscle strength)

    Key Standardization Measures:

    The following measures will be put into place to minimize variability in the study:

    • Guidelines will be produced for videofluoroscopic swallowing studies to ensure standardization across all sites
    • A central blinded reader will receive the videofluoroscopy data and will be responsible for calculating the DIGEST score, Pharyngeal Area at Maximum Constriction (PAMC), and Pharyngeal Constriction Ratio (PCR)

    The BB-301 Phase 1b/2a study is designed in a manner that could, potentially, demonstrate clinical benefit for OPMD patients via three distinct scenarios:

    • Stabilization of the rate of the loss of function for the pharyngeal phase of swallowing relative to the baseline rate of disease progression observed over the course of the 6-month pre-treatment observation period
    • Slowing of the rate of the loss of function for the pharyngeal phase of swallowing relative to the baseline rate of disease progression observed over the course of the 6-month pre-treatment observation period
    • Improvement in function for the pharyngeal phase of swallowing relative to the baseline rate of disease progression observed over the course of the 6-month pre-treatment observation period

    Methodological Background for the Key Analytical Tools and Methods:

    Summary:

    • Prior studies employing high resolution manometry and videofluoroscopy have demonstrated that OPMD patients exhibit significant dysfunction with respect to the pharyngeal phase of swallowing
      • Studies employing high resolution manometry have illustrated significantly lower pharyngeal pressures during swallowing in OPMD patients (Castell, J. et al., Dysphagia, 1995)
      • Additionally, videofluoroscopy studies have demonstrated significant pharyngeal residue post-swallow (references detailed below)
    • As a consequence, in the BB-301 Phase 1b/2a study, Benitec will focus on imaging modalities and diagnostic protocols that accurately and reproducibly characterize the efficiency of the pharyngeal phase of swallowing

    Overview of the Dynamic Imaging Grade of Swallowing Toxicity (DIGEST) Scale:

    • DIGEST is a 5-point scale that allows clinical researchers to grade pharyngeal dysphagia in a uniform manner in clinical studies (0 = no dysphagia; 1 = mild dysphagia; 2 = moderate dysphagia; 3 = severe dysphagia; 4 = life threatening dysphagia)
    • The DIGEST scale is a validated tool developed by the NCI to assess dysphagia related to the pharyngeal phase of swallowing (Hutcheson, K., et al., Cancer, 2017)
    • DIGEST was developed to allow clinical researchers that study Head and Neck Cancer to grade the dysphagia that may result from the common surgical and radiological treatment interventions
    • Video fluoroscopy (or modified barium swallow) is viewed as the gold standard for the assessment of swallowing function
      • In this regard, the NCI sought to develop and validate a video fluoroscopy-based scale that could be used by Head and Neck Cancer clinical researchers to monitor and report dysphagia-related toxicity (and in Head and Neck Cancer, the pharyngeal phase of swallowing is most often disturbed)
    • DIGEST focuses on two elements of the pharyngeal phase of swallowing: 1. swallowing efficiency, and 2. swallowing safety; swallowing inefficiency leads to weight loss and malnutrition and problems with swallowing safety can lead to aspiration
    • Grades for swallowing efficiency are combined with grades for swallowing safety to calculate the final DIGEST score

    Overview of DIGEST Scale Use for the Evaluation of OPMD Patients: 

    • OPMD patients experience dysphagia, and, primarily, it is the pharyngeal phase of swallowing that is disturbed
    • Videofluoroscopy examinations have been conducted by investigators in 22 patients with OPMD, and the results were employed to grade the levels of dysphagia for these patients with the DIGEST method (Tabor, L., et al., Neurogastroenterology & Motility, 2017)
    • Based on the DIGEST method, 96% of these 22 OPMD patients had dysphagia (i.e. a DIGEST score of 1 or greater), and, therefore, DIGEST accurately identified patients with dysphagia
    • 50% of the patients had Mild dysphagia (DIGEST = 1), 15% had Moderate dysphagia (DIGEST = 2), and 31% had Severe dysphagia (DIGEST = 3)

    Overview of the Pharyngeal Constriction Ratio:

    • The Pharyngeal Constriction Ratio (PCR) was created to quantify the efficiency of the pharyngeal phase of swallowing via videofluoroscopy
    • The PCR was demonstrated to be a surrogate for pharyngeal muscle strength (Leonard, R. et al., Dysphagia, 2011)
    • The PCR compares the amount of free air-space visible in the pharynx at the point of maximal pharyngeal constriction during swallowing to the amount of free air-space visible in the pharynx in its resting state (i.e. the area of the free air-space during maximal constriction is the numerator and the area of the free air-space at rest is the denominator)
    • Ideally, the pharynx should close completely during swallowing (as it does in young, healthy individuals) and this would result in a very low PCR (i.e. 1% or 0%)
    • If a patient experiences difficulty while attempting to close the pharynx (i.e. patients that have difficulty swallowing due to pharyngeal-focused dysphagia), then the PCR will be much higher than that observed for young, healthy individuals (i.e. the PCR can be 25% or higher in patients with dysphagia)
    • Leonard, et al. validated the correlation between PCR and pharyngeal pressure generated during swallowing as measured via high resolution manometry

    Overview of the Pharyngeal Area at Maximum Constriction:

    • Videofluoroscopy was employed to evaluate the pharyngeal phase of swallowing in 11 OPMD patients to characterize abnormalities in the key elements underlying the successful completion of the pharyngeal phase of swallowing (Waito, A., et al., Dysphagia, 2018)
    • In this study, pharyngeal constriction was found to be the most significant abnormality in this OPMD patient population as illustrated by the abnormal Pharyngeal Area at Maximum Constriction (PAMC) values obtained in 91% of the swallows during the study
    • PAMC uses the pixelated area of the pharynx at the point of maximum constriction during swallowing as the numerator and the C2-C4 length squared (i.e. C2-C42) is used as the denominator
      • Healthy individuals without dysphagia do not have PAMC values above 3% (Steele, C. et al., Journal of Speech, Language, and Hearing Research, 2019)
    • In the Waito, et al. study, 91% of the swallows exhibited PAMC values above 4%

    Overview of the Sydney Swallow Questionnaire:

    • The Sydney Swallow Questionnaire is a 17-question self-report inventory that is used by a patient to describe the severity of dysphagia
    • This tool was developed and validated as a self-report inventory

    Overview of the International Dysphagia Diet Standardization Initiative (IDDSI) Functional Diet Scale:

    • IDDSI is a newly developed functional outcome scale intended to capture the severity of oropharyngeal dysphagia as represented by the degree of diet texture restriction recommended for the patient

    About Benitec Biopharma, Inc.

    Benitec Biopharma, Inc. ("Benitec" or the "Company") is a development-stage biotechnology company focused on the advancement of novel genetic medicines with its 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 ddRNAi-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD), and Chronic Hepatitis B. A comprehensive overview of the Company can be found on Benitec's website at www.benitec.com.

    Forward Looking Statements

    Except for the historical information set forth herein, the matters set forth in this press release represent forward-looking statements, including statements regarding BB-301, Benitec's plans to develop and commercialize its product candidates, the timing of the initiation and completion of preclinical and clinical trials, the timing of patient enrolment and dosing in clinical trials, the timing of expected regulatory filings, the clinical utility and potential attributes and benefits of ddRNAi and Benitec's product candidates, potential future out-licenses and collaborations, the intellectual property position and the ability to procure additional sources of financing, and other forward-looking statements. In addition, preliminary results or other preliminary analyses do not in any way ensure that later or final results in a clinical trial or in similar clinical trials will replicate those interim results.

    These forward-looking statements are based on the Company's current expectations and subject to risks and uncertainties that may cause actual results to differ materially  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;
    • 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;
    • 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 our reports filed with the SEC from time to time. Any forward-looking statements in this release speak only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

    Media & Investor Relations Contact:

    Jay A. Morakis

    CEO of M Group Strategic Communications (for Benitec Biopharma, Inc.)

    Phone: 646-859-5951

    Email:

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  5. HAYWARD, Calif., Sept. 8, 2021 /PRNewswire/ -- Benitec Biopharma Inc. (NASDAQ:BNTC), a development-stage biotechnology company focused on the advancement of novel genetic medicines, today provided an update on the ongoing analyses of the large animal subjects treated with BB-301 in the Pilot Dosing Study.  Interim data from the BB-301 Pilot Dosing Study were disclosed in February 2021 via press release and presentation and subsequently discussed with European regulators in the first half of 2021. The updated BB-301 analyses outlined today continue to demonstrate robust, dose-dependent target tissue transduction, dose-dependent transgene expression, and biologically significant knock-down of the target protein.  These data provide continued support…

    HAYWARD, Calif., Sept. 8, 2021 /PRNewswire/ -- Benitec Biopharma Inc. (NASDAQ:BNTC), a development-stage biotechnology company focused on the advancement of novel genetic medicines, today provided an update on the ongoing analyses of the large animal subjects treated with BB-301 in the Pilot Dosing Study.  Interim data from the BB-301 Pilot Dosing Study were disclosed in February 2021 via press release and presentation and subsequently discussed with European regulators in the first half of 2021. The updated BB-301 analyses outlined today continue to demonstrate robust, dose-dependent target tissue transduction, dose-dependent transgene expression, and biologically significant knock-down of the target protein.  These data provide continued support for the planned advancement of BB-301 into the Phase 1b/2a study in 2022.

    BB-301 is a novel investigational gene therapy under development for the treatment of patients with Oculopharyngeal Muscular Dystrophy (OPMD). OPMD is a chronic, life-threatening genetic disorder affecting approximately 15,000 patients in the United States, Canada, Western Europe, and Israel.  OPMD is caused by a mutation in the gene encoding poly(A) binding protein nuclear 1 (PABPN1). Patients with OPMD lose the ability to swallow liquids and solids, and the natural history of the disorder is characterized by chronic malnutrition, aspiration, and fatal episodes of aspiration pneumonia.  Currently, no therapeutic agents are approved for the treatment of OPMD. Additionally, no surgical interventions capable of altering the long-term natural history of OPMD are available.

    Benitec has previously disclosed key data-points related to the completed non-clinical studies and the planned non-clinical studies for BB-301 that were anticipated to support the filing of Clinical Trial Applications in Europe and in Canada and, similarly, to facilitate the filing of an Investigational New Drug Application in the United States. The BB-301 Pilot Dosing Study in large animals (Beagle dogs) and the GLP Toxicology and Biodistribution Study represent two of the core non-clinical studies. BB-301 is directly injected into the pharyngeal muscles known to underlie the morbidity and mortality characterizing the natural history of OPMD. Against this backdrop, the BB-301 Pilot Dosing Study in large animal subjects was conducted to demonstrate that direct intramuscular injection of BB-301 via the use of a proprietary dosing procedure could safely achieve the following goals:

    • Biologically significant, dose-dependent levels of BB-301 tissue transduction (i.e., delivery of the multi-functional genetic construct into the target pharyngeal muscle cells)
    • Broad-based, dose-dependent expression within the pharyngeal muscle cells of the three distinct genes comprising the BB-301 gene construct
    • 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 BB-301 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, 3.0+E13 vg/mL with a low injection volume, and 3.0+E13 vg/mL with a high injection volume) following direct intramuscular injection into the Hypopharyngeus (HP) muscles and the Thyropharyngeus (TP) muscles of Beagle dogs. 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 analysis after 8 weeks of follow-up. BB-301 dosing was carried out by both a veterinary surgeon and a practicing otolaryngologist who has extensive experience with the provision of palliative surgical care for OPMD patients.

    The initial interim data-points from the BB-301 Pilot Dosing Study presented in February 2021 via press release and presentation were derived from completed analyses of the pharyngeal muscle tissues isolated from 6 Beagle dog subjects. The updated analyses of the BB-301 Pilot Dosing Study subjects outlined today comprise data-points derived from 16 Beagle dog subjects. Further data analyses are ongoing for the canine subjects treated in the 24-subject BB-301 Pilot Dosing Study. The key updates are summarized below.

    Pharyngeal Muscle Tissue Transduction for BB-301 (Figure 1):

    Figure 1

    Gene Expression Within the Pharyngeal Muscle Tissues (Figure 2, Figure 3, Figure 4):

    • 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 encodes a wild type version of the PABPN1 protein whose intracellular expression is unaffected by the inhibitory activities of siRNA13 and siRNA17, and this codon optimized PABPN1 protein (i.e. coPABPN1) 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 level of expression for wild type PABPN1 within the pharyngeal muscle cells of Beagle dogs is 4.5-to-7.8 copies per cell

     

    Figure 2

     

    Figure 3

     

    Figure 4

    Wild Type PABPN1 Knock-Down Within the Pharyngeal Muscle Tissues (Figure 5):

    • 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 current BB-301 Pilot Dosing Study do not express mutant PABPN1, the level of BB-301-driven gene silencing for the PABPN1 target can be accurately assessed via the equivalent inhibitory effects of siRNA13 and siRNA17 on both wtPABPN1 and mutant PABPN1
    • Thus, the wtPABPN1 silencing activity observed in the current BB-301 Pilot Dosing Study serves as a surrogate for the 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 manifest the key signs and symptoms of OPMD and, in these animal models of OPMD, the achievement of PABPN1 silencing levels of 31% or higher led to complete resolution of OPMD disease symptoms and correction of the histological hallmarks of the disease

     

    Figure 5

    Ubiquity of Target Tissue Transduction Following Implementation of the Proprietary Dosing Procedure (Figure 6, Figure 7):

    It is critical to highlight the 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. 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.

    The newly developed proprietary methods of BB-301 delivery, as well as the analytical methods developed to assay the key target tissues of the Beagle dog subjects, led to broad-based transduction of the pharyngeal muscle target tissues (Figure 6, individual sections of the TP muscle following BB-301 dosing) and demonstrated 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 (Figure 7).

    Figure 6

     

    Figure 7

    About Benitec Biopharma, Inc.

    Benitec Biopharma, Inc. ("Benitec" or the "Company") is a development-stage biotechnology company focused on the advancement of novel genetic medicines with its 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 ddRNAi-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD), and Chronic Hepatitis B. A comprehensive overview of the Company can be found on Benitec's website at www.benitec.com.

    Forward Looking Statements

    Except for the historical information set forth herein, the matters set forth in this press release represent forward-looking statements, including statements regarding BB-301, Benitec's plans to develop and commercialize its product candidates, the timing of the initiation and completion of preclinical and clinical trials, the timing of patient enrolment and dosing in clinical trials, the timing of expected regulatory filings, the clinical utility and potential attributes and benefits of ddRNAi and Benitec's product candidates, potential future out-licenses and collaborations, the intellectual property position and the ability to procure additional sources of financing, and other forward-looking statements. In addition, preliminary results or other preliminary analyses do not in any way ensure that later or final results in a clinical trial or in similar clinical trials will replicate those interim results.

    These forward-looking statements are based on the Company's current expectations and subject to risks and uncertainties that may cause actual results to differ materially  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;
    • 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;
    • 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 our reports filed with the SEC from time to time. Any forward-looking statements in this release speak only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

    Media & Investor Relations Contact:

    Jay A. Morakis

    CEO of M Group Strategic Communications (for Benitec Biopharma, Inc.)

    Phone: 646-859-5951

    Email:

     

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    SOURCE Benitec Biopharma Inc.

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