1. NEW YORK and CLEVELAND, March 25, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced the appointment of two new independent members to its Board of Directors with significant biotechnology and life sciences experience: Faith L. Charles, Partner, Corporate Transactions and Securities at Thompson Hine LLP and Mark Alvino, President at life sciences strategic advisory firm Hudson Square Capital LLC.

    "In addition to the experience and backgrounds of the current Abeona directors, our two new board members represent a group of highly qualified and diverse executives who bring fresh perspectives, relevant expertise and leadership experience, positioning Abeona extremely…

    NEW YORK and CLEVELAND, March 25, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced the appointment of two new independent members to its Board of Directors with significant biotechnology and life sciences experience: Faith L. Charles, Partner, Corporate Transactions and Securities at Thompson Hine LLP and Mark Alvino, President at life sciences strategic advisory firm Hudson Square Capital LLC.

    "In addition to the experience and backgrounds of the current Abeona directors, our two new board members represent a group of highly qualified and diverse executives who bring fresh perspectives, relevant expertise and leadership experience, positioning Abeona extremely well to fulfill our mission of bringing our novel gene and cell therapies to patients who currently have no approved treatment options," said Steven H. Rouhandeh, Chairman of Abeona's Board of Directors. "The addition of Faith and Mark is part of an ongoing strategic effort to add relevant leadership experience to Abeona's Board of Directors to support the company's focus on driving future growth, enhancing its corporate governance, and creating additional shareholder value."

    Faith L. Charles, Partner, Corporate Transactions and Securities for Thompson Hine LLP

    Ms. Charles is a corporate transactions and securities partner at Thompson Hine with 30 years of legal experience. She leads Thompson Hine's Life Sciences practice, advising public and emerging biotech and pharmaceutical companies in the U.S. and internationally. Ms. Charles negotiates complex private and public financing transactions, mergers and acquisitions, licensing transactions and strategic collaborations. She serves as outside counsel to a myriad of life sciences companies and is known in the industry as an astute business advisor, providing valuable insights into capital markets, corporate governance and strategic development.

    Ms. Charles serves on the Board of Directors of Entera Bio (NASDAQ:ENTX) as a member of the Audit Committee and Chair of the Compensation Committee. She also serves on the Board of Directors of several private life science companies. Ms. Charles founded the Women in Bio Metro New York chapter and chaired the chapter for five years. She currently serves on the national board of Women in Bio. Ms. Charles is also a member of the board of Gilda's Club New York City. She has been recognized as a Life Sciences Star by Euromoney's LMG Life Sciences, has been named a BTI Client Service All-Star, and was named by Crain's New York Business to the list of 2020 Notable Women in the Law. Ms. Charles holds a J.D degree from The George Washington University Law School and a B.A. in Psychology from Barnard College, Columbia University. Ms. Charles is a graduate of Women in Bio's Boardroom Ready Program, an Executive Education Program taught by The George Washington University School of Business.

    Mark Alvino, President at Hudson Square Capital LLC

    Mr. Alvino has provided leadership and experience in the areas of financial management and business strategy as a member of the Board of Directors of multiple life sciences companies, including PainQx, a company with a patented method for objective, quantitative assessment of chronic pain, and previously serving on Abeona's Board of Directors. He has more than 25 years of experience in the pharmaceutical and biotechnology industries through a range of disciplines including healthcare investment banking, communications, business consulting, market research and health and medical marketing communications. In his role at Hudson Square Capital, Mr. Alvino provides strategic advisory to emerging growth and turnaround biotechnology and medical technology companies. Mr. Alvino is also Founder and Chief Executive Officer of Bridge Back Foundation, focused on addressing the broad underlying issues behind the deadly opioid crisis in America. Previously, he led the Life Sciences team at broker dealer Bradley Woods & Co. Ltd. and served as Managing Director at Griffin Securities and at SCO Financial Group, LLC. Earlier in his career, he spent several years working with Wall Street brokerages including Ladenburg, Thalmann & Co. and Martin Simpson & Co. Mr. Alvino received a B.B.A in Economics and Public Policy from the George Washington University.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical needs. Abeona's novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. We have attempted to identify forward-looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Company's most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.



    Investor and Media Contact:
    
    Greg Gin
    VP, Investor Relations and Corporate Communications
    Abeona Therapeutics
    +1 (646) 813-4709
    
    

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  2. Fourth patient treated in pivotal Phase 3 VIITAL™ study of EB-101 in RDEB after successful Type B meeting with FDA

    Positive new interim data from MPS IIIA and MPS IIIB programs presented at 17th Annual WORLDSymposium

    $95 million in cash, cash equivalents and short-term investments as of December 31, 2020

    Conference call scheduled for Thursday, March 25, 2021 at 8:30 a.m. ET

    NEW YORK and CLEVELAND, March 24, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced financial results for the fourth quarter and full year 2020, and provided an update on recent operational progress.

    "In 2020, Abeona advanced our three clinical programs toward bringing urgently…

    Fourth patient treated in pivotal Phase 3 VIITAL™ study of EB-101 in RDEB after successful Type B meeting with FDA

    Positive new interim data from MPS IIIA and MPS IIIB programs presented at 17th Annual WORLDSymposium

    $95 million in cash, cash equivalents and short-term investments as of December 31, 2020

    Conference call scheduled for Thursday, March 25, 2021 at 8:30 a.m. ET

    NEW YORK and CLEVELAND, March 24, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced financial results for the fourth quarter and full year 2020, and provided an update on recent operational progress.

    "In 2020, Abeona advanced our three clinical programs toward bringing urgently needed treatments to patients with recessive dystrophic epidermolysis bullosa (RDEB) and Sanfilippo syndrome type A (MPS IIIA) and type B (MPS IIIB) despite the macro disruptions that impacted the world," said Michael Amoroso, Chief Executive Officer of Abeona. "We remain laser focused on executing our strategy and achieving upcoming milestones. Our recent momentum is highlighted by a successful Type B meeting with the FDA where we aligned on co-primary endpoints for the pivotal Phase 3 VIITAL study of EB-101 in RDEB, treatment of the fourth patient in the VIITAL study, and reporting new positive clinical data for both ABO-102 in MPS IIIA and ABO-101 in MPS IIIB at the WORLDSymposium. We look forward to continuing to propel our clinical programs forward and bringing our gene and cell therapies to patients who currently have no approved treatment options. We believe we have sufficient cash resources to build on our momentum and fund our current development and operating plan through the achievement of key anticipated milestones, including the potential for multiple regulatory submissions."

    Recent Highlights

    Corporate Developments

    • In March 2021, Michael Amoroso, Executive Vice President, Chief Operating Officer (COO) and principal executive officer at Abeona, was promoted to President, Chief Executive Officer (CEO) and a member of the company's Board of Directors.

    EB-101 (Autologous, Gene-Corrected Cell Therapy)

    • The fourth patient was treated in Abeona's EB-101 pivotal Phase 3 VIITAL™ study for recessive dystrophic epidermolysis bullosa (RDEB). The company currently anticipates completing study enrollment in 2021 of 10 to 15 patients with RDEB, comprising approximately 35 large chronic wound sites treated in total.
    • Abeona held a successful Type B meeting with the U.S. Food and Drug Administration (FDA) to align with the Agency on the company's proposal regarding co-primary endpoints of partial wound closure and mean pain reduction for the Phase 3 VIITAL™ study of EB-101 in RDEB.

    ABO-102 and ABO-101 (AAV-based Gene Therapies)

    • Reported new positive interim data from the ABO-102 Transpher A study for MPS IIIA and the ABO-101 Transpher B study for MPS IIIB. The data was presented in late-breaking platform oral presentations at the 17th Annual WORLDSymposium™ in February 2021. The presented results from the high dose cohort 3 in the Transpher A study showed evidence of preservation of neurocognitive development within normal range of a non-afflicted child for 2.5 years to 3 years after treatment with ABO-102 in the three young patients treated before 30 months of age. In addition, the data showed a dose-related and sustained reduction in cerebrospinal fluid (CSF) levels of heparan sulfate, denoting transgene expression in the CNS, and a durable reduction of liver volume. The presented results from the Transpher B study continued to show signals of biologic effect with reduction of disease-specific biomarkers in the CSF, plasma and urine and reduction in liver volumes. Abeona expects additional follow-up visits and neurocognitive assessments of patients treated in the high dose cohort 3 in the Transpher A study and additional clinical updates from the Transpher B study in 2021.
    • The FDA accepted Abeona's request for a meeting to discuss the data-to-date from the Transpher A study and the potential path to a Biologics License Application (BLA) submission for ABO-102 in MPS IIIA.
    • As previously reported, target enrollment in the ABO-102 Transpher A study has been achieved. Abeona continues to enroll patients into the study given the lack of treatment options for MPS IIIA and encouraging efficacy and safety data from the high dose cohort 3. To date, 19 patients have been dosed in the Transpher A study, including 13 patients dosed in cohort 3.
    • The ABO-101 Transpher B study for MPS IIIB is ongoing, and to date, 11 patients have been dosed, including 4 patients dosed in cohort 3.

    Preclinical Pipeline

    • Initiated preclinical research in December 2020 and are assessing AAV capsids in six undisclosed ophthalmic disorders and intend to advance toward IND-enabling studies in two to three indications in 2022. Previously reported preclinical data showed the potential for AIM™ AAV vectors to efficiently target the retinal epithelium after intravitreal injection, creating the potential for new pipeline candidates that can address multiple ophthalmic disorders.

    Fourth Quarter and Full Year 2020 Financial Results

    Cash, cash equivalents and short-term investments totaled $95.0 million as of December 31, 2020, compared to $129.3 million as of December 31, 2019. Net cash used in operating activities was $35.0 million for the full year of 2020, compared to $62.8 million for the full year 2019.

    License and other revenues for the fourth quarter and full year of 2020 were $3.0 million and $10.0 million, respectively, compared to zero revenues in the same periods in 2019. The increase in revenue was comprised of initial proceeds from agreements with Taysha Gene Therapies in August 2020 relating to ABO-202, a potential gene therapy for for CLN1 disease, and in October 2020 relating to intellectual property directed to a potential gene therapy for Rett syndrome.

    Research and development (R&D) expenses were $9.2 million for the fourth quarter of 2020 and $30.1 million for the full year of 2020, compared to $9.6 million and $48.6 million in the same periods in 2019. The decrease in R&D expenses was primarily due to decreased clinical and development work for the company's gene and cell therapy product candidates as a result of scaled back manufacturing, clinical and non-clinical development activities impacted by the COVID-19 pandemic, and cost savings from the decision to internally manufacture retrovirus for the EB-101 program.

    General and administrative (G&A) expenses were $7.4 million for the fourth quarter of 2020 and $23.8 million for the full year of 2020, compared to $4.7 million and $20.7 million in the same periods in 2019. The increase in G&A expenses in the fourth quarter of 2020 was primarily due to increased professional fees and increased share-based compensation expense. The increase in G&A expenses for full year 2020 was primarily due to severance costs associated with management changes and increased professional fees.

    Net loss was $15.8 million for the fourth quarter of 2020 and $84.2 million for the full year of 2020, compared to net loss of $16.4 million and $76.3 million for the same periods in 2019. The increase in the full year 2020 net loss is primarily due to the licensed technology non-cash impairment charge of $32.9 million related to the termination of the license agreement with REGENXBIO, partially offset by increased license and other revenues along with decreased clinical and development expenses.

    Conference Call Details

    Abeona Therapeutics will host a conference call and webcast tomorrow, Thursday, March 25, 2021 at 8:30 a.m. ET, to discuss its fourth quarter 2020 financial results and business update. To access the call, dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) and Entry Code: 251720 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona's website at www.abeonatherapeutics.com. The archived webcast replay will be available for 30 days following the call.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical needs. Abeona's novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. We have attempted to identify forward-looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Company's most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

     
     
    Abeona Therapeutics Inc. and Subsidiaries
    Consolidated Statements of Operations and Comprehensive Loss
    (unaudited)
            
     For the three months ended December 31, For the years ended December 31,
      2020   2019   2020   2019 
    Revenues:       
    License and other revenues$3,000,000  $-  $10,000,000  $- 
    Total revenues 3,000,000   -   10,000,000   - 
            
    Expenses:       
    Research and development 9,243,000   9,605,000   30,139,000   48,566,000 
    General and administrative 7,397,000   4,734,000   23,779,000   20,705,000 
    Depreciation and amortization 840,000   2,072,000   4,586,000   7,819,000 
    Licensed technology impairment charge -   -   32,916,000   - 
    Total expenses 17,480,000   16,411,000   91,420,000   77,090,000 
            
    Loss from operations (14,480,000)  (16,411,000)  (81,420,000)  (77,090,000)
            
    Interest and miscellaneous income 40,000   380,000   1,301,000   1,208,000 
    Interest and other expense (1,388,000)  (400,000)  (4,115,000)  (400,000)
    Net loss$(15,828,000) $(16,431,000) $(84,234,000) $(76,282,000)
            
    Basic and diluted loss per common share$(0.17) $(0.30) $(0.91) $(1.51)
            
    Weighted average number of common       
    shares outstanding – basic and diluted 92,869,775   54,718,776   92,663,574   50,354,596 
            
    Other comprehensive loss:       
    Change in unrealized losses related to available-for-sale debt securities (27,000)  -   (10,000)  - 
    Comprehensive loss$(15,855,000) $(16,431,000) $(84,244,000) $(76,282,000)



     
    Abeona Therapeutics Inc. and Subsidiaries
    Consolidated Balance Sheets
    (unaudited)
        
    ASSETSDecember 31,

    2020
     December 31,

    2019
    Current assets:   
    Cash and cash equivalents$12,596,000  $129,258,000 
    Short-term investments 82,438,000   - 
    Prepaid expenses and other current assets 2,708,000   3,132,000 
    Total current assets 97,742,000   132,390,000 
        
    Property and equipment, net 11,322,000   13,157,000 
    Right-of-use lease assets 7,032,000   8,047,000 
    Licensed technology, net 1,500,000   36,178,000 
    Goodwill 32,466,000   32,466,000 
    Other assets and restricted cash 1,136,000   1,144,000 
    Total assets$151,198,000  $223,382,000 
        
    LIABILITIES AND STOCKHOLDERS' EQUITY   
    Current liabilities:   
    Accounts payable$4,695,000  $3,763,000 
    Accrued expenses 3,410,000   5,543,000 
    Current portion of lease liability 1,713,000   1,699,000 
    Current portion of loan payable 330,000   - 
    Payable to licensor 31,515,000   27,400,000 
    Deferred revenue 296,000   296,000 
    Total current liabilities 41,959,000   38,701,000 
        
    Loan payable 1,428,000   - 
    Long-term lease liabilities 5,260,000   6,251,000 
    Total liabilities 48,647,000   44,952,000 
        
    Commitments and contingencies -   - 
    Stockholders' equity:   
            
    Common stock - $0.01 par value; authorized

    200,000,000 shares; issued and outstanding

    96,131,678 at December 31, 2020; issued and

    outstanding 83,622,135 at December 31, 2019
     961,000   836,000 
            
    Additional paid-in capital 672,304,000   664,064,000 
    Accumulated deficit (570,704,000)  (486,470,000)
    Accumulated other comprehensive loss (10,000)  - 
    Total stockholders' equity 102,551,000   178,430,000 
    Total liabilities and stockholders' equity$151,198,000  $223,382,000 



    Investor and Media Contact:
    Greg Gin
    VP, Investor Relations and Corporate Communications
    Abeona Therapeutics
    +1 (646) 813-4709
    

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  3. NEW YORK and CLEVELAND, March 22, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that Michael Amoroso, Executive Vice President, Chief Operating Officer (COO) and principal executive officer at Abeona, has been promoted to President, Chief Executive Officer (CEO) and a member of the company's Board of Directors, effective immediately.

    "Michael's promotion to CEO reflects his unwavering commitment and outstanding operational leadership during a period of important transition for the company, and the Board fully supports him and the Abeona senior management team," said Steven H. Rouhandeh, Chairman of Abeona's Board of Directors. "Under Michael's stewardship…

    NEW YORK and CLEVELAND, March 22, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that Michael Amoroso, Executive Vice President, Chief Operating Officer (COO) and principal executive officer at Abeona, has been promoted to President, Chief Executive Officer (CEO) and a member of the company's Board of Directors, effective immediately.

    "Michael's promotion to CEO reflects his unwavering commitment and outstanding operational leadership during a period of important transition for the company, and the Board fully supports him and the Abeona senior management team," said Steven H. Rouhandeh, Chairman of Abeona's Board of Directors. "Under Michael's stewardship, Abeona has remained focused on the strategy of providing our novel gene and cell therapies to patients who currently have no approved treatment options, delivering on meaningful milestones across our three clinical development programs during the last six months."

    Most recently, Mr. Amoroso has been responsible for overseeing the operational management of Abeona, including research and clinical development, regulatory, medical, commercial, corporate affairs and business development as COO and principal executive officer. Mr. Amoroso joined Abeona in 2020, bringing extensive experience in leading teams across clinical development, regulatory and medical affairs, corporate affairs, and commercial, both in the U.S and globally, with direct operational experience in major world markets. For 20 years, he has led and been part of teams responsible for launches and delivering profit and loss results for major biopharmaceutical products across most therapeutic areas. Mr. Amoroso has worked with companies in the small molecule, biologic, and cell and gene therapy spaces, with his deepest areas of expertise in rare oncological diseases.

    Prior to joining Abeona, Mr. Amoroso held various senior level executive positions at leading biopharmaceutical companies, including Kite, Eisai Inc., Celgene Corporation (now a subsidiary of Bristol-Myers Squibb Company), and began his biopharmaceutical career at Aventis (now Sanofi). Mr. Amoroso earned his Executive M.B.A. in Management from the Stern School of Business, New York University, and his B.A. in Biological Sciences, summa cum laude, from Rider University.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical needs. Abeona's novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company exploring all strategic options, including the sale of some or all of its assets or sale of the Company. We have attempted to identify forward-looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, the outcome of the Company's ongoing strategic review, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Company's most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.



    Investor and Media Contact:
    
    Greg Gin
    VP, Investor Relations and Corporate Communications
    Abeona Therapeutics
    +1 (646) 813-4709
    
    

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  4. NEW YORK and CLEVELAND, Feb. 22, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that the company will present at the 10th Annual SVB Leerink Global Healthcare Conference on Wednesday, February 24, 2021 at 11:20 am EST.

    A live webcast of the presentation will be available on the investor section of the Abeona Therapeutics website, www.abeonatherapeutics.com, and an archived replay will be accessible for up to 30 days.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy…

    NEW YORK and CLEVELAND, Feb. 22, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that the company will present at the 10th Annual SVB Leerink Global Healthcare Conference on Wednesday, February 24, 2021 at 11:20 am EST.

    A live webcast of the presentation will be available on the investor section of the Abeona Therapeutics website, www.abeonatherapeutics.com, and an archived replay will be accessible for up to 30 days.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical needs. Abeona's novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company exploring all strategic options, including the sale of some or all of its assets or sale of the Company. We have attempted to identify forward-looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, the outcome of the strategic review, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Company's most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.



    Investor and Media Contact:
    
    Greg Gin
    VP, Investor Relations and Corporate Communications
    Abeona Therapeutics
    +1 (646) 813-4709
    
    

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  5. In addition to preservation of neurocognitive development with ABO-102 in MPS IIIA, new clinical results of ABO-102 in MPS IIIA and ABO-101 in MPS IIIB continue to show dose-dependent and sustained reductions in disease-specific biomarkers, denoting clear biologic effects

    In addition, ABO-102 and ABO-101 continue to show favorable safety profile in ongoing studies

    Abeona to host investor webinar on Tuesday, February 16, 2021 at 1:00 p.m. EST

    NEW YORK and CLEVELAND, Feb. 12, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced new positive data from two ongoing Phase 1/2 clinical trials of the company's investigational AAV-based gene therapies ABO-102 and ABO-101…

    In addition to preservation of neurocognitive development with ABO-102 in MPS IIIA, new clinical results of ABO-102 in MPS IIIA and ABO-101 in MPS IIIB continue to show dose-dependent and sustained reductions in disease-specific biomarkers, denoting clear biologic effects

    In addition, ABO-102 and ABO-101 continue to show favorable safety profile in ongoing studies

    Abeona to host investor webinar on Tuesday, February 16, 2021 at 1:00 p.m. EST

    NEW YORK and CLEVELAND, Feb. 12, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced new positive data from two ongoing Phase 1/2 clinical trials of the company's investigational AAV-based gene therapies ABO-102 and ABO-101 in MPS IIIA and MPS IIIB, respectively. The interim data was presented in late-breaking platform oral presentations at the 17th Annual WORLDSymposium™. The presentation slides are available on the company's website at www.abeonatherapeutics.com.

    Michael Amoroso, Principal Executive and Chief Operating Officer of Abeona, stated, "We are excited to share updated positive efficacy and safety results that continue to suggest ABO-102 has the potential to be a life-altering treatment option for children with MPS IIIA, a rare, debilitating condition with no approved treatment that leads to progressive neurodevelopmental and physical decline, and often results in death early in life. We have requested a meeting with the FDA later this quarter to discuss the ABO-102 data and the potential path towards a Biologics License Application filing for ABO-102. In addition, the new results from the Transpher B study continue to support ABO-101's biologic activity in patients with MPS IIIB."

    The updated results from the Transpher A study evaluating ABO-102 in Sanfilippo syndrome type A (MPS IIIA) demonstrated that neurocognitive development was preserved within normal range of a non-afflicted child for 2.5 years to 3 years (the latest time point measured) after treatment with ABO-102 (3x1013 vg/kg) in three young patients in the high-dose cohort 3. The three young patients were treated with ABO-102 at ages 27 months, 19 months, and 12 months and are now at ages ranging from 3.5 years to 5+ years, the timepoint at which patients with MPS IIIA have already started to experience neurocognitive decline based on the natural history of disease progression. Dose-dependent and statistically significant reductions in cerebrospinal fluid heparan sulfate, denoting enzyme activity in the central nervous system (CNS), and liver volume were sustained for two years after treatment. ABO-102 has been well-tolerated with long-term safety remaining favorable 24-55 months following treatment. There have been no treatment-related severe adverse events and no clinically significant adverse events reported.

    Kevin Flanigan, M.D., Director, Center for Gene Therapy at AWRI at Nationwide Children's and Transpher A study principal investigator, said, "The results presented today show a single intravenous dose of ABO-102 can help preserve neurocognitive development for up to 3 years following treatment in young MPS IIIA patients during early stages of their disease. The data shows ABO-102's ability to deliver a functional copy of the disease-causing SGSH gene to cells of the CNS and peripheral organs, as evidenced by the clinical benefits in neurocognition and biophysical measures and improvements in disease-specific biomarkers."

    Results from the Transpher B study evaluating ABO-101 in Sanfilippo syndrome type B (MPS IIIB) showed that treatment with ABO-101 is associated with a dose-dependent and sustained improvement in central nervous system and systemic biomarkers, indicating the potent biologic effect of ABO-101 in patients with MPS IIIB. ABO-101 has been well-tolerated with no infusion related or early acute reactions and no clinically significant adverse events or laboratory abnormalities.

    Maria Jose de Castro, M.D., Hospital Clínico Universitario Santiago de Compostela and Transpher B study investigator, said, "The results from the Transpher B study provide evidence of ABO-101's impact on disease biomarkers and potential to break down the accumulation of glycosaminoglycans that underlie MPS IIIB pathology. We look forward to continued follow-up to assess ABO-101's potential to preserve neurocognitive development in patients with MPS IIIB."

    Investor Webinar on MPS III Gene Therapy Programs

    Abeona management along with Dr. Flanigan and Dr. de Castro will host an investor webinar on February 16, 2021 at 1:00 p.m. EST. The live webinar, including audio and presentation slides, will be accessible at https://investors.abeonatherapeutics.com/events at the time of the meeting. To register in advance for the live webinar, click here.

    An archived replay of the webinar will be available after the conclusion of the live event at https://investors.abeonatherapeutics.com/events.

    About the Annual WORLDSymposium

    The WORLDSymposium™ is designed for basic, translational and clinical researchers, patient advocacy groups, clinicians, and all others who are interested in learning more about the latest discoveries related to lysosomal diseases and the clinical investigation of these advances. For additional information on the 17th Annual WORLDSymposium™, please visit https://worldsymposia.org/.

    About the Transpher A Study

    The Transpher A Study (NCT02716246) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). The study, also known as ABT-001, is intended for patients 6 months to 2 years of age, or patients older than 2 years with a cognitive developmental quotient of 60% or above. ABO-102 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

    About the Transpher B Study

    The Transpher B Study (NCT03315182) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-101 for the treatment of patients with Sanfilippo syndrome type B (MPS IIIB). The study, also known as ABT-002, is intended for patients 6 months to 2 years of age, or patients older than 2 years with a cognitive developmental quotient of 60% or above. ABO-101 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

    About ABO-102

    ABO-102 is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). ABO-102 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations for the ABO-102 clinical program. In the EU, the Company holds PRIME and Orphan medicinal product designations.

    About ABO-101

    ABO-101 is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type B (MPS IIIB), a rare lysosomal storage disease with no approved therapy that primarily affects the central nervous system (CNS). ABO-101 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the NAGLU gene to cells of the CNS and peripheral tissues. The therapy is designed to address the underlying NAGLU enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Fast Track and Rare Pediatric Disease designations for ABO-101 and Orphan Drug designation in both the U.S. and EU.

    About Sanfilippo Syndrome Type A (MPS IIIA)

    Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the CNS and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIA present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive facial features with thick eyebrows or a unibrow, full lips and excessive body hair for one's age, and liver/spleen enlargement are also present in early childhood. MPS IIIA is caused by genetic mutations that lead to a deficiency in the SGSH enzyme responsible for breaking down glycosaminoglycans, which accumulate in cells throughout the body resulting in rapid health decline associated with the disorder.

    About Sanfilippo syndrome type B (MPS IIIB)

    Sanfilippo syndrome type B (MPS IIIB) is a rare and fatal lysosomal storage disease with no approved therapy that primarily affects the central nervous system and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIB present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive signs such as facial features with thick eyebrows or a unibrow, full lips and excessive body hair for one's age and liver/spleen enlargement are also present. The underlying cause of MPS IIIB is a deficiency in the NAGLU enzyme responsible for breaking down glycosaminoglycans, which accumulate throughout the body resulting in rapid decline associated with the disorder.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical needs. Abeona's novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company exploring all strategic options, including the sale of some or all of its assets or sale of the Company. We have attempted to identify forward-looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, the outcome of the strategic review, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Company's most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.



    Investor and Media Contact:
    Greg Gin
    VP, Investor Relations and Corporate Communications
    Abeona Therapeutics
    +1 (646) 813-4709
    

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  6. NEW YORK and CLEVELAND, Feb. 02, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that it will host a live webinar for the investment community on Tuesday, February 16, 2021 at 1:00 p.m. EST to review clinical data on the company's investigational AAV-based gene therapies ABO-102 and ABO-101 presented at the 17th Annual WORLDSymposium™.

    Speakers will include Kevin Flanigan, M.D., Director, Center for Gene Therapy at AWRI at Nationwide Children's and Transpher A study principal investigator, Maria Jose de Castro, M.D., Hospital Clínico Universitario Santiago de Compostela and Transpher B study investigator, and Michael Amoroso, Principal Executive and Chief…

    NEW YORK and CLEVELAND, Feb. 02, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that it will host a live webinar for the investment community on Tuesday, February 16, 2021 at 1:00 p.m. EST to review clinical data on the company's investigational AAV-based gene therapies ABO-102 and ABO-101 presented at the 17th Annual WORLDSymposium™.

    Speakers will include Kevin Flanigan, M.D., Director, Center for Gene Therapy at AWRI at Nationwide Children's and Transpher A study principal investigator, Maria Jose de Castro, M.D., Hospital Clínico Universitario Santiago de Compostela and Transpher B study investigator, and Michael Amoroso, Principal Executive and Chief Operating Officer of Abeona.

    To register in advance for the live webinar, please visit this registration link. The live webinar, including audio and presentation slides, will be accessible at https://investors.abeonatherapeutics.com/events at the time of the meeting. An archived replay of the webinar will be available after the conclusion of the live event at https://investors.abeonatherapeutics.com/events.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical needs. Abeona's novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company exploring all strategic options, including the sale of some or all of its assets or sale of the Company. We have attempted to identify forward-looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, the outcome of the strategic review, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Company's most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.



    Investor and Media Contact:
    Greg Gin
    VP, Investor Relations and Corporate Communications
    Abeona Therapeutics
    +1 (646) 813-4709
    

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  7. NEW YORK and CLEVELAND, Jan. 25, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that the company held a successful Type B meeting with the U.S. Food and Drug Administration (FDA) to align with the Agency on the company's proposal regarding co-primary endpoints for the pivotal Phase 3 VIITAL™ study of EB-101 in recessive dystrophic epidermolysis bullosa (RDEB). Following the meeting, Abeona is proceeding with its plan to enroll between 10 to 15 patients with RDEB, comprising approximately 35 large chronic wound sites treated in total.

    "We appreciate the clarity provided by the FDA and we are pleased to be aligned with the Agency on the co-primary endpoints…

    NEW YORK and CLEVELAND, Jan. 25, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that the company held a successful Type B meeting with the U.S. Food and Drug Administration (FDA) to align with the Agency on the company's proposal regarding co-primary endpoints for the pivotal Phase 3 VIITAL™ study of EB-101 in recessive dystrophic epidermolysis bullosa (RDEB). Following the meeting, Abeona is proceeding with its plan to enroll between 10 to 15 patients with RDEB, comprising approximately 35 large chronic wound sites treated in total.

    "We appreciate the clarity provided by the FDA and we are pleased to be aligned with the Agency on the co-primary endpoints for the Phase 3 VIITAL™ study," said Michael Amoroso, Principal Executive and Chief Operating Officer of Abeona. "Following the successful completion of the FDA meeting, we continue with all necessary steps to enroll our next patient in the VIITAL™ study and aim to complete enrollment in 2021."

    The co-primary endpoints of the study are: 1) the proportion of RDEB wound sites with greater than or equal to 50% healing from baseline, comparing treated with untreated wound sites at Week 24 (Month 6) as determined by direct investigator assessment; and 2) pain reduction associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds at Week 24 (Month 6).

    As previously announced, data from a Phase 1/2a clinical trial presented at the 2020 Society for Pediatric Dermatology Annual Meeting showed that wound healing of 50% or greater following EB-101 treatment in patients with RDEB was associated with no pain at treated sites at three-, four- and five-years post-treatment, compared with presence of pain in 53% of wound sites at baseline.

    Jodie Gillon, Vice President and Chief Patient Officer of Abeona commented, "We greatly appreciate the level of clarity we received from the FDA as we continue to work with our clinical study partners at Stanford University Medical Center and patient advocacy groups to enroll additional patients in the VIITAL™ study."

    Investigators at Stanford University Medical Center are currently enrolling eligible patients into the VIITAL™ study. Additional information about the trial, including eligibility criteria, is available at https://www.abeonatherapeutics.com/clinical-trials/rdeb and https://clinicaltrials.gov/ (Identifier: NCT04227106).

    About Recessive Dystrophic Epidermolysis Bullosa

    Recessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning type VII collagen which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

    About EB-101

    EB-101 is an autologous, gene-corrected cell therapy currently being investigated in the pivotal Phase 3 VIITAL™ study for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. The EB-101 VIITAL™ study is a randomized clinical trial enrolling 10 to 15 RDEB patients with approximately 30 large, chronic wound sites treated in total. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into a patient's own skin cells (keratinocytes and its progenitors) and transplanting them back to the patient to enable normal Type VII collagen expression and facilitate wound healing. Abeona produces EB-101 for the VIITAL™ study at its fully-functional gene and cell therapy manufacturing facility in Cleveland, OH. In a Phase 1/2a clinical trial, EB-101 provided durable wound healing for RDEB patients lasting 2+ to 5+ years, including for the largest, most challenging wounds that affect the majority of the RDEB population.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical needs. Abeona's novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company's aim to complete enrollment of patients in our VIITAL study in 2021. We have attempted to identify forward-looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, the outcome of our announced strategic review, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Company's most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.



    Investor and Media Contact:
    Greg Gin
    VP, Investor Relations and Corporate Communications
    Abeona Therapeutics
    +1 (646) 813-4709
    

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  8. NEW YORK and CLEVELAND, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that abstracts detailing new interim results from its ABO-102 Phase 1/2 Transpher A study for MPS IIIA and ABO-101 Phase 1/2 Transpher B study for MPS IIIB have been accepted for platform oral presentations during the late-breaking abstract session at the 17th Annual WORLDSymposium™ being held February 8-12, 2021.

    "Children born with MPS IIIA and MPS IIIB experience progressive neurodevelopmental decline and loss of motor function that is life-threatening," said Michael Amoroso, Chief Operating Officer of Abeona. "We are excited to share new analyses from the Transpher A study…

    NEW YORK and CLEVELAND, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that abstracts detailing new interim results from its ABO-102 Phase 1/2 Transpher A study for MPS IIIA and ABO-101 Phase 1/2 Transpher B study for MPS IIIB have been accepted for platform oral presentations during the late-breaking abstract session at the 17th Annual WORLDSymposium™ being held February 8-12, 2021.

    "Children born with MPS IIIA and MPS IIIB experience progressive neurodevelopmental decline and loss of motor function that is life-threatening," said Michael Amoroso, Chief Operating Officer of Abeona. "We are excited to share new analyses from the Transpher A study that will add to the understanding of the potential for ABO-102 to help preserve neurocognitive development in patients with MPS IIIA when they are treated at a young age, and new results from the Transpher B study that will provide insights into ABO-101's biologic effect in patients with MPS IIIB."

    Presentation Details

    Title: Updated Results of Transpher A, a Multicenter, Single-Dose, Phase 1/2 Clinical Trial of ABO-102 Gene Therapy for Sanfilippo Syndrome Type A (Mucopolysaccharidosis IIIA)

    Abstract Number: 390

    Presenter: Kevin Flanigan, M.D., Center for Gene Therapy at Nationwide Children's Hospital

    Date/Time: Friday, February 12, 2021, time to be determined

    Title: Updated Results of Transpher B, a Multicenter, Single-Dose, Phase 1/2 Clinical Trial of ABO-101 Gene Therapy for Sanfilippo Syndrome Type B (Mucopolysaccharidosis IIIB)

    Abstract Number: 407

    Presenter: Maria Jose de Castro, M.D., Hospital Clínico Universitario Santiago de Compostela

    Date/Time: Friday, February 12, 2021, time to be determined

    About the Annual WORLDSymposium

    The WORLDSymposium™ is designed for basic, translational and clinical researchers, patient advocacy groups, clinicians, and all others who are interested in learning more about the latest discoveries related to lysosomal diseases and the clinical investigation of these advances. For additional information on the 17th Annual WORLDSymposium™, please visit https://worldsymposia.org/.

    About the Transpher A Study

    The Transpher A Study (NCT02716246) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). The study, also known as ABT-001, is intended for patients from birth to 2 years of age, or patients older than 2 years with a cognitive developmental quotient of 60% or above. ABO-102 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment changes and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

    About the Transpher B Study

    The Transpher B Study (NCT03315182) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-101 for the treatment of patients with Sanfilippo syndrome type B (MPS IIIB). The study, also known as ABT-002, is intended for patients from birth to 2 years of age, or patients older than 2 years with a cognitive developmental quotient of 60% or above. ABO-101 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment changes and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

    About ABO-102

    ABO-102 is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). ABO-102 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations for the ABO-102 clinical program. In the EU, the Company holds PRIME and Orphan medicinal product designations.

    About ABO-101

    ABO-101 is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type B (MPS IIIB), a rare lysosomal storage disease with no approved therapy that primarily affects the central nervous system (CNS). ABO-101 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the NAGLU gene to cells of the CNS and peripheral tissues. The therapy is designed to address the underlying NAGLU enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Fast Track and Rare Pediatric Disease designations for ABO-101 and Orphan Drug designation in both the U.S. and EU.

    About Sanfilippo Syndrome Type A (MPS IIIA)

    Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the CNS and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIA present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive facial features with thick eyebrows or a unibrow, full lips and excessive body hair for one's age, and liver/spleen enlargement are also present in early childhood. MPS IIIA is caused by genetic mutations that lead to a deficiency in the SGSH enzyme responsible for breaking down glycosaminoglycans, which accumulate in cells throughout the body resulting in rapid health decline associated with the disorder.

    About Sanfilippo syndrome type B (MPS IIIB)

    Sanfilippo syndrome type B (MPS IIIB) is a rare and fatal lysosomal storage disease with no approved therapy that primarily affects the central nervous system and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIB present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive signs such as facial features with thick eyebrows or a unibrow, full lips and excessive body hair for one's age and liver/spleen enlargement are also present. The underlying cause of MPS IIIB is a deficiency in the NAGLU enzyme responsible for breaking down glycosaminoglycans, which accumulate throughout the body resulting in rapid decline associated with the disorder.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical needs. Abeona's novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company exploring all strategic options, including the sale of some or all of its assets or sale of the Company. We have attempted to identify forward-looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, the outcome of the strategic review, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Company's most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.



    Investor and Media Contact:
    Greg Gin
    VP, Investor Relations
    Abeona Therapeutics
    +1 (646) 813-4709
    

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  9. NEW YORK and CLEVELAND, Nov. 09, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced financial results for the third quarter 2020 and recent business progress.

    "Abeona remains committed to pursuing the development of our portfolio of advanced and early stage programs toward providing our novel gene and cell therapies to patients who currently have no approved treatment options," said Michael Amoroso, Chief Operating Officer of Abeona. "In particular, we are continuing to propel our clinical programs in recessive dystrophic epidermolysis bullosa (RDEB) and Sanfilippo syndrome type A (MPS IIIA) and type B (MPS IIIB) to drive Abeona's long-term growth and unlock…

    NEW YORK and CLEVELAND, Nov. 09, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced financial results for the third quarter 2020 and recent business progress.

    "Abeona remains committed to pursuing the development of our portfolio of advanced and early stage programs toward providing our novel gene and cell therapies to patients who currently have no approved treatment options," said Michael Amoroso, Chief Operating Officer of Abeona. "In particular, we are continuing to propel our clinical programs in recessive dystrophic epidermolysis bullosa (RDEB) and Sanfilippo syndrome type A (MPS IIIA) and type B (MPS IIIB) to drive Abeona's long-term growth and unlock shareholder value."

    Third Quarter and Recent Highlights

    Corporate and Business Development Updates

    • In August 2020, Abeona entered into definitive agreements with Taysha Gene Therapies to license its rights to ABO-202, an AAV-based gene therapy for CLN1 disease (also known as infantile Batten disease). As part of the transaction, Abeona received initial cash proceeds of $7.0 million in October 2020. Abeona is also eligible to receive up to $56.0 million upon the achievement of certain clinical, regulatory and sales milestones, plus royalty-based payments based on net sales.
    • In October 2020, Abeona entered into a license agreement with Taysha Gene Therapies with respect to certain intellectual property rights, materials, and know-how relating to a potential AAV-based gene therapy for Rett syndrome. In connection with the agreement, Abeona received a one-time upfront payment of $3.0 million and is eligible to receive up to $56.5 million upon the achievement of certain clinical, regulatory and sales milestones, plus royalty-based payments based on net sales.
    • In October 2020, Michael Amoroso, Senior Vice President and Chief Commercial Officer at Abeona, was promoted to Chief Operating Officer. In this newly created role, Mr. Amoroso's responsibilities are broadened to include oversight and leadership of all operations.
    • Abeona continues to work with Jefferies LLC as its financial advisor in the review of strategic options focused on advancing the Company's mission and maximizing stakeholder value.

    EB-101 (Autologous, Gene-Corrected Cell Therapy)

    • Patient enrollment is ongoing for Abeona's EB-101 pivotal Phase 3 VIITAL™ study for RDEB. The Company currently anticipates completing enrollment in the VIITAL study in the first half of 2021, depending upon the impact from the COVID-19 pandemic, including travel restrictions and safety concerns.

    ABO-102 and ABO-101 (AAV-based Gene Therapies)

    • Target enrollment in the ABO-102 Transpher A study for MPS IIIA (15 to 22 patients) has been achieved. To date, 18 patients have been dosed in the Transpher A study, including 12 patients dosed in cohort 3. Abeona intends to continue enrolling patients into the study through the first quarter of 2021 given the lack of treatment options for MPS IIIA and encouraging efficacy and safety data from cohort 3.
    • In the ABO-101 Transpher B study for MPS IIIB, 11 patients have been dosed to date, including 4 patients dosed in cohort 3. The Company anticipates completing target enrollment in the Transpher B study (15 to 20 patients) in the first quarter of 2021.
    • In the third quarter, Abeona presented its plan toward registration of ABO-102 for MPS IIIA (Sanfilippo syndrome type A) during a kick-off meeting under the European Medicines Agency's (EMA) PRIority MEdicines (PRIME) program that offers a path for accelerated assessment of promising therapies targeting unmet medical needs. Based on the meeting, along with previous input from the Committee for Medicinal Products for Human Use and the Pediatric Committee of the EMA, Abeona anticipates submitting a marketing authorization application for EU conditional approval of ABO-102 for MPS IIIA after completion of two-year follow-up of the last patient treated in the Transpher A study. Regarding the U.S. regulatory path for ABO-102 in MPS IIIA, Abeona plans to request a meeting with the FDA to take place in the first quarter of 2021, depending on FDA's availability.

    Manufacturing Activities

    • Process development at the Company's GMP manufacturing facility in Cleveland, Ohio is ongoing that will enable production of the retrovirus used for EB-101 manufactured at the facility, allowing for increased control of the supply chain and product quality, as well as reduced costs. In addition, Abeona continues process development activities to enable in-house manufacturing of commercial supply of ABO-102 and ABO-101.

    Third Quarter Financial Results

    Cash, cash equivalents, receivables and short-term investments totaled $103.9 million as of September 30, 2020, compared to $129.3 million as of December 31, 2019. Accounts receivable were $7.0 million at September 30, 2020, which was paid by the customer in October 2020. Net cash used in operating activities was $10.7 million for the third quarter of 2020.

    License and other revenues for the third quarter of 2020 were $7.0 million, comprised of initial proceeds from the ABO-202 transaction, compared to zero revenues in the year-ago quarter.  

    Research and development (R&D) expenses were $8.0 million for the third quarter of 2020 and $20.9 million for the nine months ended September 30, 2020, compared to $10.9 million and $39.0 million in the comparable periods in 2019. The decrease in R&D expenses was primarily due to decreased manufacturing, clinical and non-clinical development activities arising from the effects of the COVID-19 pandemic, and cost savings associated with the decision to internally manufacture retrovirus for the EB-101 program.

    General and administrative (G&A) expenses were $4.4 million for the third quarter of 2020 and $16.4 million for the nine months ended September 30, 2020, compared to $4.7 million and $16.0 million in the comparable periods in 2019. The decrease in G&A expenses in the third quarter of 2020 was primarily due to decreased salary and related costs. The increase in G&A expenses in the nine months ended September 30, 2020 was primarily from severance costs associated with management changes, partially offset by decreased professional fees.

    Net loss was $7.2 million for the third quarter of 2020 and $68.4 million for the nine months ended September 30, 2020, compared to net loss of $17.4 million and $59.9 million for the comparable periods in 2019. The increase in net loss for the nine months ended September 30, 2020 includes the non-cash impairment charge of $32.9 million related to the termination of the license agreement with REGENXBIO.

    Conference Call Details

    Abeona Therapeutics will host a conference call and webcast tomorrow, Tuesday, November 10, 2020 at 8:30 a.m. ET, to discuss its third quarter 2020 financial results and business update. To access the call, dial 844-369-8770 (U.S. toll-free) or 862-298-0840 (international) five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona's website at www.abeonatherapeutics.com. The archived webcast replay will be available for 30 days following the call.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical needs. Abeona's novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.   

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.  These statements include statements about the Company's clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Company's goals and objectives. We have attempted to identify forward looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, our ongoing strategic review of our business and pre-clinical and clinical programs, including the sale of the Company or potential partnering or sale of such programs or the suspension of one or more of our pre-clinical or clinical programs, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws. 



    Abeona Therapeutics Inc. and Subsidiaries 
    Condensed Consolidated Statements of Operations and Comprehensive Loss 
    (unaudited) 
                    
     For the three months ended

    September 30,
     For the nine months ended

    September 30,
      2020   2019   2020   2019 
    Revenues:       
    License and other revenues$7,000,000  $-  $7,000,000  $- 
    Total revenues 7,000,000   -   7,000,000   - 
            
    Expenses:       
    Research and development 7,969,000   10,917,000   20,896,000   38,961,000 
    General and administrative 4,432,000   4,700,000   16,382,000   15,971,000 
    Depreciation and amortization 847,000   2,032,000   3,746,000   5,747,000 
    Licensed technology impairment charge -   -   32,916,000   - 
    Total expenses 13,248,000   17,649,000   73,940,000   60,679,000 
            
    Loss from operations (6,248,000)  (17,649,000)  (66,940,000)  (60,679,000)
            
    Interest and miscellaneous income 338,000   277,000   1,261,000   828,000 
    Interest and other expense (1,327,000)  -   (2,727,000)  - 
    Net loss$(7,237,000) $(17,372,000) $(68,406,000) $(59,851,000)
            
    Basic and diluted loss per common share$(0.08) $(0.35) $(0.73) $(1.22)
            
    Weighted average number of common       
    shares outstanding – basic and diluted 93,772,712   49,721,753   93,199,679   48,883,883 
            
    Other comprehensive income/(loss):       
    Change in unrealized (losses)/gains related to available-for-sale debt securities (116,000)  -   17,000   - 
            
    Comprehensive loss$(7,353,000) $(17,372,000) $(68,389,000) $(59,851,000)





    Abeona Therapeutics Inc. and Subsidiaries
    Condensed Consolidated Balance Sheets
    (unaudited)
        
        
    ASSETSSeptember 30,

    2020
     December 31,

    2019
        
    Current assets:   
    Cash and cash equivalents$8,424,000  $129,258,000 
    Receivables 7,000,000   - 
    Short-term investments 88,447,000   - 
    Prepaid expenses and other current assets 689,000   3,132,000 
    Total current assets 104,560,000   132,390,000 
        
    Property and equipment, net 12,095,000   13,157,000 
    Right-of-use lease assets 7,295,000   8,047,000 
    Licensed technology, net 1,881,000   36,178,000 
    Goodwill 32,466,000   32,466,000 
    Other assets and restricted cash 1,068,000   1,144,000 
    Total assets$159,365,000  $223,382,000 
        
    LIABILITIES AND STOCKHOLDERS' EQUITY   
        
    Current liabilities:   
    Accounts payable$1,262,000  $3,763,000 
    Accrued expenses 3,585,000   5,543,000 
    Loan payable 1,758,000   - 
    Current portion of lease liability 1,709,000   1,699,000 
    Payable to licensor 30,127,000   27,400,000 
    Deferred revenue 296,000   296,000 
    Total current liabilities 38,737,000   38,701,000 
        
    Long-term lease liability 5,517,000   6,251,000 
    Total liabilities 44,254,000   44,952,000 
        
    Commitments and contingencies -   - 
    Stockholders' equity:   
    Common stock - $0.01 par value; authorized 200,000,000 shares; issued and outstanding 84,516,161 at September 30, 2020 and 83,622,135 at December 31, 2019 845,000   836,000 
    Additional paid-in capital 669,125,000   664,064,000 
    Accumulated deficit (554,876,000)  (486,470,000)
    Accumulated other comprehensive income 17,000   - 
    Total stockholders' equity 115,111,000   178,430,000 
    Total liabilities and stockholders' equity$159,365,000  $223,382,000 
    Investor and Media Contact:
    
    Greg Gin
    VP, Investor Relations
    Abeona Therapeutics
    +1 (646) 813-4709
    

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  10. NEW YORK and CLEVELAND, Oct. 27, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that Michael Amoroso, Senior Vice President and Chief Commercial Officer at Abeona, has been promoted to Chief Operating Officer and will serve as the Company's principal executive and operating officer, effective November 1, 2020. In this newly created role at Abeona, Mr. Amoroso's responsibilities will broaden to include oversight and leadership of all operations; including but not limited to, research and clinical development, regulatory, medical, commercial, corporate affairs and business development.

    "Michael has more than 20 years of strategic and operational leadership…

    NEW YORK and CLEVELAND, Oct. 27, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that Michael Amoroso, Senior Vice President and Chief Commercial Officer at Abeona, has been promoted to Chief Operating Officer and will serve as the Company's principal executive and operating officer, effective November 1, 2020. In this newly created role at Abeona, Mr. Amoroso's responsibilities will broaden to include oversight and leadership of all operations; including but not limited to, research and clinical development, regulatory, medical, commercial, corporate affairs and business development.

    "Michael has more than 20 years of strategic and operational leadership experience in the biotechnology and pharmaceutical industries, including working within commercialization teams across clinical development, regulatory affairs, and medical affairs in major global markets," said Steven H. Rouhandeh, Chairman of Abeona's Board of Directors. "His recent and highly relevant experience with the commercial development of novel therapies for underserved patient populations will serve Abeona well as the Company seeks to further advance its clinical programs toward providing novel gene and cell therapies to patients who currently have no approved treatment options."

    Mr. Amoroso stated, "Abeona has a significant foundation in place with a compelling vision, great people, deep science and a robust pipeline of advanced and early stage programs. The experienced and talented team at Abeona remains committed to serving the patients with recessive dystrophic epidermolysis bullosa (RDEB) and Sanfilippo syndrome, and we are focused on completing enrollment in our EB-101 pivotal Phase 3 study for RDEB and Phase 1/2 studies of ABO-102 and ABO-101 for Sanfilippo syndrome type A (MPS IIIA) and Sanfilippo syndrome type B (MPS IIIB), respectively."

    Mr. Amoroso has served as Senior Vice President and Chief Commercial Officer of Abeona since July 2020. Prior to joining Abeona, Mr. Amoroso held various senior level commercial positions at leading biopharmaceutical companies, including Kite, Eisai Inc., Celgene Corporation (now a subsidiary of Bristol-Myers Squibb Company), and Sanofi. At Kite, he was responsible for the company's worldwide commercial organization leading the commercialization efforts for the autologous CAR T-cell therapy, YESCARTA®, and the future cell therapy pipeline. Before Kite, Mr. Amoroso was Senior Vice President, Americas for Eisai's Commercial Oncology Business Group, where he was accountable for teams charged with creating and driving commercial strategy and implementation for the company's approved products and earlier-stage assets. Previously, Mr. Amoroso worked at Celgene for six years in several commercial roles before serving as the organization's Commercial Lead for CAR T-cell therapy programs. In this capacity, he helped Celgene develop an organizational model to commercialize cell therapies including specialized manufacturing and customer services for patients with lymphoma and myeloma. Before joining Celgene, Mr. Amoroso held various marketing and sales leadership positions over his 10-plus year tenure at Sanofi. Mr. Amoroso earned his M.B.A. in Management from the Stern School of Business, New York University, and his B.A. in Biological Sciences, summa cum laude, from Rider University.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio of AAV-based gene therapies also features ABO-201 for CLN3 disease. Abeona's novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company exploring all strategic options, including the sale of some or all of its assets or sale of the Company. We have attempted to identify forward-looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, the outcome of the strategic review, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Company's most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

     

    Investor and Media Contact:
    Greg Gin
    VP, Investor Relations
    Abeona Therapeutics
    +1 (646) 813-4709
    

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  11. Steven H. Rouhandeh Appointed as Chairman of the Board of Directors

    Jefferies LLC Retained as Financial Advisor to Assist with the Review of Strategic Options

    NEW YORK and CLEVELAND, Oct. 06, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that its Board of Directors has formed a Special Committee to oversee and advise the executive leadership team on the operations of the Company, leveraging the respective medical, business development and financial expertise of its members. The Special Committee, which consists of current board members, is also working closely with the executive leadership team to develop the Company's strategic direction and leadership…

    Steven H. Rouhandeh Appointed as Chairman of the Board of Directors

    Jefferies LLC Retained as Financial Advisor to Assist with the Review of Strategic Options

    NEW YORK and CLEVELAND, Oct. 06, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that its Board of Directors has formed a Special Committee to oversee and advise the executive leadership team on the operations of the Company, leveraging the respective medical, business development and financial expertise of its members. The Special Committee, which consists of current board members, is also working closely with the executive leadership team to develop the Company's strategic direction and leadership plan as it continues to advance its clinical programs toward providing novel gene and cell therapies to patients who currently have no approved treatment options. The Company also announced the appointment of Steven H. Rouhandeh as Chairman of its Board of Directors.

    Strategic Review

    Abeona has retained Jefferies LLC as its financial advisor to assist with the review of strategic options focused on advancing the Company's mission and maximizing stakeholder value. There can be no assurance this strategic review will result in the completion of any particular course of action. There is no defined timeline for completion of the review process and the Company does not intend to comment further unless a specific initiative is approved by the Board of Directors, the review process is concluded, or it is otherwise determined that other disclosure is appropriate.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio of AAV-based gene therapies also features ABO-201 for CLN3 disease. Abeona's novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company exploring all strategic options, including the sale of some or all of its assets or sale of the Company. We have attempted to identify forward-looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, the outcome of the strategic review, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Company's most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor and Media Contact:
    Greg Gin
    VP, Investor Relations
    Abeona Therapeutics
    +1 (646) 813-4709
    

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  12. NEW YORK and CLEVELAND, Sept. 28, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced changes in its executive leadership and Board of Directors. João Siffert, M.D. resigned from his positions as CEO, Head of Research and Development, and Chief Medical Officer, as well as member of the Board of Directors, on September 23, 2020. On September 27, 2020, Brian Pereira, M.D., Stefano Buono, Stephen B. Howell, M.D., George Migausky and Shawn Tomasello each announced that they had stepped down from the Board, effective immediately. No director gave any reason for their resignation and no director indicated any disagreements with the Company.

    The Company is reviewing…

    NEW YORK and CLEVELAND, Sept. 28, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced changes in its executive leadership and Board of Directors. João Siffert, M.D. resigned from his positions as CEO, Head of Research and Development, and Chief Medical Officer, as well as member of the Board of Directors, on September 23, 2020. On September 27, 2020, Brian Pereira, M.D., Stefano Buono, Stephen B. Howell, M.D., George Migausky and Shawn Tomasello each announced that they had stepped down from the Board, effective immediately. No director gave any reason for their resignation and no director indicated any disagreements with the Company.

    The Company is reviewing and exploring all strategic options and alternatives focused on advancing the Company's mission and maximizing stakeholder value, including the sale of some or all of its assets or sale of the Company. There can be no assurance this strategic review will result in the completion of any particular course of action. There is no defined timeline for completion of the review process.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio of AAV-based gene therapies also features ABO-201 for CLN3 disease. Abeona's novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company exploring all strategic options, including the sale of some or all of its assets or sale of the Company. We have attempted to identify forward looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Company's most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Greg Gin
    VP, Investor Relations
    Abeona Therapeutics
    +1 (646) 813-4709
    tics.com
    
    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843
    

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  13. NEW YORK and CLEVELAND, Sept. 08, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that the Company will participate in the following upcoming investor conferences.

    • João Siffert, M.D., Chief Executive Officer, will conduct virtual one-on-one meetings with institutional investors at Citi's 15th Annual BioPharma Virtual Conference on Wednesday, September 9, 2020.

    • Dr. Siffert will present at the Wells Fargo Virtual Healthcare Conference on Thursday, September 10, 2020 at 10:00 a.m. ET. A live webcast of the presentation will be available on the investor section of the Abeona Therapeutics website at www.abeonatherapeutics.com. The webcast replay will be available…

    NEW YORK and CLEVELAND, Sept. 08, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that the Company will participate in the following upcoming investor conferences.

    • João Siffert, M.D., Chief Executive Officer, will conduct virtual one-on-one meetings with institutional investors at Citi's 15th Annual BioPharma Virtual Conference on Wednesday, September 9, 2020.



    • Dr. Siffert will present at the Wells Fargo Virtual Healthcare Conference on Thursday, September 10, 2020 at 10:00 a.m. ET. A live webcast of the presentation will be available on the investor section of the Abeona Therapeutics website at www.abeonatherapeutics.com. The webcast replay will be available within 24 hours of the live presentation and will be accessible for 30 days.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio of AAV-based gene therapies also features ABO-201 for CLN3 disease. Abeona's novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

    Investor Contact:

    Greg Gin

    VP, Investor Relations

    Abeona Therapeutics

    +1 (646) 813-4709

    Media Contact:

    Scott Santiamo

    Director, Corporate Communications

    Abeona Therapeutics

    +1 (718) 344-5843

    Primary Logo

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  14. NEW YORK and CLEVELAND and DALLAS, Aug. 17, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, and Taysha Gene Therapies, a patient-centric gene therapy company with a mission to eradicate monogenic CNS disease, today announced that they have entered into license and inventory purchase agreements for ABO-202, an adeno-associated virus (AAV) gene therapy for CLN1 disease (also known as infantile Batten disease). The agreements grant Taysha worldwide exclusive rights to intellectual property developed by scientists at the University of North Carolina at Chapel Hill (UNC) and Abeona, and Abeona know-how relating to the research, development, and manufacture of ABO-202. The therapy…

    NEW YORK and CLEVELAND and DALLAS, Aug. 17, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, and Taysha Gene Therapies, a patient-centric gene therapy company with a mission to eradicate monogenic CNS disease, today announced that they have entered into license and inventory purchase agreements for ABO-202, an adeno-associated virus (AAV) gene therapy for CLN1 disease (also known as infantile Batten disease). The agreements grant Taysha worldwide exclusive rights to intellectual property developed by scientists at the University of North Carolina at Chapel Hill (UNC) and Abeona, and Abeona know-how relating to the research, development, and manufacture of ABO-202. The therapy was originally developed in the lab of Steven Gray, Ph.D., Associate Professor at UT Southwestern Medical Center (formerly with UNC) and Chief Scientific Advisor for Taysha Gene Therapies. Abeona continued to progress the program, including development of the Phase 1/2 clinical trial protocol and manufacturing process, and received FDA clearance of its Investigational New Drug (IND) Application for a Phase 1/2 clinical trial that is anticipated to enter the clinic in 2021.

    Under the terms of the agreement, Taysha will make initial cash payments to Abeona of $7 million, comprised of a $3 million upfront license fee and $4 million inventory purchase price, including GMP-sourced CLN1 plasmid from Abeona. In addition, Abeona is eligible to receive up to $56 million from Taysha upon the achievement of certain clinical, regulatory and sales milestones, plus high single-digit royalties on net sales of Taysha's CLN1 product.

    "We are excited to partner with Taysha in their further development of a potential treatment for children living with Batten disease," said João Siffert, M.D., Chief Executive Officer of Abeona. "At the same time, these agreements allow Abeona to continue to focus its resources on advancing its key clinical programs in RDEB, MPS IIIA and MPS IIIB towards Biologics License Application submissions with the goal of providing safe and effective gene and cell therapies to patients who currently have no approved treatment options."

    "CLN1 is a progressive monogenic CNS disease with significant unmet medical need, and we believe the ABO-202 data generated thus far demonstrate great translational potential and offer hope to children suffering from this devastating disorder," said RA Session II, President, Chief Executive Officer and Founder of Taysha. "We are excited to continue working closely with Dr. Gray to rapidly advance this promising gene therapy into the clinic."

    ABO-202 is a one-time, self-complementary AAV (serotype 9) gene therapy designed to deliver a functional copy of the palmitoyl-protein thioesterase 1 (PPT1) gene. Preclinical studies in a CLN1 animal model demonstrated that ABO-202 normalized survival and led to improvement of neurological function in affected mice.  The therapy has been granted Orphan Drug and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA) and has received Orphan Medicinal Product Designation from the European Medicines Agency.

    About CLN1 disease (Infantile Batten disease)

    CLN1 disease, also known as Infantile Neuronal Ceroid Lipofuscinosis or infantile Batten disease, is a rapidly progressing rare lysosomal storage disease with no approved treatment. It primarily affects the central nervous system and typically manifests during the first year of life with vision impairment that can progress to blindness, progressive motor and cognitive decline, seizures and ultimately early death. The underlying cause of the disorder is a defect in the PPT1 gene that encodes the enzyme of the same name, resulting in the accumulation of lipopigments within cells, leading to neuroinflammation and neurodegeneration. Some patients with CLN1 disease develop symptoms later in childhood or in adulthood; these variants are called late-infantile, juvenile, or adult-onset CLN1.

    About ABO-202

    ABO-202 is a novel, one-time gene therapy for CLN1 disease, a rapidly progressing rare lysosomal storage disease with no approved therapy. ABO-202 delivers a functional copy of the PPT1 gene to address the underlying gene and enzyme deficiency that leads to abnormal buildup of lipopigments and results in neuroinflammation and neurodegeneration.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio of AAV-based gene therapies also features ABO-201 for CLN3 disease. Abeona's novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

    About Taysha Gene Therapies

    Taysha Gene Therapies is a patient-centric gene therapy company with a mission to eradicate monogenic CNS disease. We are focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the CNS in both rare and large patient populations. We were founded in partnership with The University of Texas Southwestern Medical Center, or UT Southwestern, to develop and commercialize transformative gene therapy treatments. Together with UT Southwestern, we are advancing a deep and sustainable product portfolio of 18 gene therapy product candidates, with exclusive options to acquire four additional development programs. By combining our management team's proven experience in gene therapy drug development and commercialization with UT Southwestern's world-class gene therapy research capabilities, we believe we have created a powerful engine to develop transformative therapies to dramatically improve patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.  These statements include statements about the Company's clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Company's goals and objectives.  We have attempted to identify forward looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Company's most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission.  The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Abeona Therapeutics Contacts:

    Investors:

    Greg Gin

    VP, Investor Relations

    Abeona Therapeutics

    +1 (646) 813-4709

    Media:

    Scott Santiamo

    Director, Corporate Communications

    Abeona Therapeutics

    +1 (718) 344-5843

    Taysha Gene Therapies Contacts:

    Company:

    Niren Shah, PharmD, MBA

    Taysha Gene Therapies

    Media:

    Carolyn Hawley

    Canale Communications

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  15. Additional patients dosed in RDEB, MPS IIIA and MPS IIIB clinical programs

    General alignment with CHMP on plans for EU registration of ABO-102 for MPS IIIA

    Data from RDEB and MPS III programs presented at recent medical meetings

    Strengthened leadership team with appointment of experienced Chief Commercial Officer and two independent Board members

    Conference call on Tuesday, August 11, 2020 at 8:30 a.m. ET

    NEW YORK and CLEVELAND, Aug. 10, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced financial results for the second quarter 2020 and recent business progress.

    "During the past several months, Abeona has delivered on our goals in clinical development…

    Additional patients dosed in RDEB, MPS IIIA and MPS IIIB clinical programs

    General alignment with CHMP on plans for EU registration of ABO-102 for MPS IIIA

    Data from RDEB and MPS III programs presented at recent medical meetings

    Strengthened leadership team with appointment of experienced Chief Commercial Officer and two independent Board members

    Conference call on Tuesday, August 11, 2020 at 8:30 a.m. ET

    NEW YORK and CLEVELAND, Aug. 10, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced financial results for the second quarter 2020 and recent business progress.

    "During the past several months, Abeona has delivered on our goals in clinical development, manufacturing, and regulatory affairs toward bringing urgently needed treatments to patients with RDEB and MPS III," said João Siffert, M.D., Chief Executive Officer of Abeona. "Notably, new patients have been treated in our RDEB and MPS IIIA clinical programs, and we expect additional patient enrollment across our clinical programs in the coming weeks. Concurrent with the increased clinical activities, we resumed internal manufacturing operations at our Cleveland campus in June. We have made significant advancements in process development for retrovirus and AAV manufacturing in-house, anticipated to start in late-2020 and early-2021, respectively. In addition, we recently reached general alignment with the CHMP on our proposed path toward a European marketing authorization application for ABO-102 in MPS IIIA, anticipated in 2023. Looking ahead to the potential commercial launches of EB-101 in late-2022 and ABO-102 the following year, we have also strengthened our leadership team, bringing on board a Chief Commercial Officer with significant relevant experience and expertise."

    Second Quarter and Recent Highlights

    EB-101 (Autologous, Gene-Corrected Cell Therapy)

    • A second patient was treated in Abeona's EB-101 pivotal Phase 3 VIITAL™ study for recessive dystrophic epidermolysis bullosa (RDEB) after enrollment resumed in June 2020 following a pause due to the COVID-19 pandemic. Additional patients are expected to be treated in the coming weeks, with completion of enrollment in the VIITAL study expected in early-2021.
    • Two posters were presented at the Society for Pediatric Dermatology 45th Annual Meeting. The first poster highlighted data showing that EB-101 treatment of large RDEB wounds resulted in up to five years of durable healing, which was associated with long-term pain relief. A separate poster characterized the significant disease burden of RDEB on patients and their families.

    ABO-102 and ABO-101 (AAV-based Gene Therapies)

    • In July 2020, Abeona held a kick-off meeting under the European Medicines Agency's (EMA) PRIority MEdicines (PRIME) program, which included members of the Committee for Advanced Therapies (CAT) and the Committee for Medicinal Products for Human Use (CHMP) of the EMA. The Company presented its plan toward registration of ABO-102 for MPS IIIA (Sanfilippo syndrome type A), taking advantage of the PRIME designation that offers a path for accelerated assessment of promising therapies targeting unmet medical needs. Based on the PRIME meeting, along with previous input from the CHMP and the Pediatric Committee (PDCO) of the EMA, Abeona anticipates submitting a marketing authorization application for EU conditional approval of ABO-102 for MPS IIIA in 2023. The Company continues to seek guidance from the FDA on the U.S. regulatory path for ABO-102 in MPS IIIA, but acknowledges that the FDA is currently focused on matters related to COVID-19 and other life-threatening conditions, as reflected in its issued guidance in May 2020.
    • Total enrollment to date in the ABO-102 Transpher A study for MPS IIIA is 17 patients, including 11 patients dosed in cohort 3. Total enrollment to date in the ABO-101 Transpher B study for MPS IIIB (Sanfilippo syndrome type B) is 9 patients, including 2 patients dosed in cohort 3. Abeona anticipates completing enrollment in both the Transpher A and Transpher B studies by the end of 2020.
    • Updated positive interim data from the Transpher A and Transpher B studies were presented at the American Society of Gene & Cell Therapy 23rd Annual Meeting. The findings support previously reported data showing preservation of neurocognitive skills among three young patients treated in dose cohort 3 of the Transpher A study. Improvements in multiple disease-specific biomarkers, denoting clear biologic effects, and a favorable safety profile in MPS IIIA and MPS IIIB patients after treatment with ABO-102 and ABO-101, respectively, were observed in both studies.

    Manufacturing Activities

    • In June 2020, Abeona fully resumed operations at its state-of-the-art GMP manufacturing facility in Cleveland, Ohio, manufacturing EB-101 drug product for the Phase 3 VIITAL™ study. The Company initiated process development at the facility that will enable production of the retrovirus used for EB-101 manufacture, allowing for increased control of the supply chain and product quality, as well as reduced costs. Abeona also resumed process development activities to enable in-house manufacturing of commercial supply of ABO-101 and ABO-102.

    Corporate Update

    • The Company further strengthened its leadership team with the appointment of Michael Amoroso as Chief Commercial Officer, and the addition of George Migausky and Paul Mann as independent members of its Board of Directors. In addition to their Board service, Mr. Migausky serves as Chairman of the Company's Audit Committee and Mr. Mann serves as a member of the Audit Committee.

    Second Quarter Financial Results

    Cash, cash equivalents and short-term investments totaled $107.9 million as of June 30, 2020, compared to $129.3 million as of December 31, 2019. Net cash used in operating activities was $9.5 million for the second quarter 2020.

    Research and development (R&D) spending was $6.1 million for the second quarter of 2020 and $12.9 million for the six months ended June 30, 2020, compared to $16.3 million and $28.0 million in the comparable periods in 2019. The decrease in R&D expenses was primarily due to decreased manufacturing, clinical and non-clinical development activities arising from the effects of the COVID-19 pandemic, and cost savings associated with the decision to internally manufacture retrovirus for the EB-101 program.

    General and administrative (G&A) expenses were $5.5 million for the second quarter of 2020 and $12.0 million for the six months ended June 30, 2020, compared to $5.6 million and $11.3 million in the comparable periods in 2019. The increase in G&A expenses for the six months ended June 30, 2020 was largely due to increases in salary and related costs partially offset by decreased professional fees.

    Net loss was $13.0 million for the second quarter of 2020 and $61.2 million for the six months ended June 30, 2020, compared to net loss of $23.9 million and $42.5 million for the comparable periods in 2019.

    Conference Call Details

    Abeona Therapeutics will host a conference call and webcast tomorrow, Tuesday, August 11, 2020 at 8:30 a.m. ET, to discuss its second quarter 2020 financial results and provide an update on the company's business. To access the call, dial 844-369-8770 (U.S. toll-free) or 862-298-0840 (international) and provide conference ID 18965539 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona's website at www.abeonatherapeutics.com. The archived webcast replay will be available for 30 days following the call.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio of AAV-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona's novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.  

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.  These statements include statements about the Company's clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Company's goals and objectives.  We have attempted to identify forward looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.





    Abeona Therapeutics Inc. and Subsidiaries
    Condensed Consolidated Statements of Operations and Comprehensive Loss
    (unaudited)
             
             
      For the three months ended June 30, For the six months ended June 30,
       2020   2019   2020   2019 
             
    Revenues $  -   $  -   $  -   $  -  
             
    Expenses:        
    Research and development    6,109,000     16,307,000     12,927,000     28,044,000 
    General and administrative    5,538,000     5,612,000     11,950,000     11,271,000 
    Depreciation and amortization    834,000     2,057,000     2,899,000     3,715,000 
    Licensed technology impairment charge    -      -      32,916,000     -  
    Total expenses    12,481,000     23,976,000     60,692,000     43,030,000 
             
    Loss from operations    (12,481,000)    (23,976,000)    (60,692,000)    (43,030,000)
             
    Interest and miscellaneous income    271,000     52,000     923,000     551,000 
    Interest expense    (800,000)    -      (1,400,000)    -  
    Net loss $  (13,010,000) $  (23,924,000) $  (61,169,000) $  (42,479,000)
             
    Basic and diluted loss per common share $  (0.14) $  (0.49) $  (0.66) $  (0.88)
             
    Weighted average number of common        
      shares outstanding – basic and diluted    93,180,837     48,961,988     92,910,014     48,458,004 
             
    Other comprehensive income/(loss):        
    Change in unrealized (losses) gains related to available-for-sale debt securities    (253,000)    -      133,000     -  
    Comprehensive loss $  (13,263,000) $  (23,924,000) $  (61,036,000) $  (42,479,000)



     
    Abeona Therapeutics Inc. and Subsidiaries
    Condensed Consolidated Balance Sheets
    (unaudited)
    ASSETSJune 30,

    2020
     December 31,

    2019
        
    Current assets:   
    Cash and cash equivalents $  14,542,000  $  129,258,000 
    Short-term investments   93,337,000     -  
    Prepaid expenses and other current assets   924,000     3,132,000 
            Total current assets   108,803,000     132,390,000 
        
    Property and equipment, net   12,628,000     13,157,000 
    Right-of-use lease assets   7,551,000     8,047,000 
    Licensed technology, net   1,924,000     36,178,000 
    Goodwill   32,466,000     32,466,000 
    Other assets and restricted cash   1,006,000     1,144,000 
         Total assets$  164,378,000  $  223,382,000 
        
    LIABILITIES AND STOCKHOLDERS' EQUITY   
        
    Current liabilities:   
    Accounts payable$  1,979,000  $  3,763,000 
    Accrued expenses   3,017,000     5,543,000 
    Loan payable   1,758,000     -  
    Current portion of lease liability   1,706,000     1,699,000 
    Payable to licensor   28,800,000     27,400,000 
    Deferred revenue   296,000     296,000 
            Total current liabilities   37,556,000     38,701,000 
        
    Long-term lease liabilities   5,768,000     6,251,000 
           Total liabilities   43,324,000     44,952,000 
        
    Commitments and contingencies   
    Stockholders' equity:   
    Common stock - $0.01 par value; authorized 200,000,000 shares;   
      issued and outstanding 84,781,241 at June 30, 2020

      and 83,622,135 at December 31, 2019
       848,000     836,000 
    Additional paid-in capital   667,712,000     664,064,000 
    Accumulated deficit   (547,639,000)    (486,470,000)
    Accumulated other comprehensive income   133,000     -  
           Total stockholders' equity   121,054,000     178,430,000 
           Total liabilities and stockholders' equity$  164,378,000  $  223,382,000 



    Investor Contact:

    Greg Gin

    VP, Investor Relations

    Abeona Therapeutics

    +1 (646) 813-4709

    Media Contact:

    Scott Santiamo

    Director, Corporate Communications

    Abeona Therapeutics

    +1 (718) 344-5843

    Primary Logo

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    • EB-101 treatment of large, chronic wounds is associated with durable healing and pain relief in patients with RDEB

    • Literature review of 65 studies confirms and expands understanding of substantial disease burden of RDEB with considerable clinical, economic and humanistic impact on patients and their families

    NEW YORK and CLEVELAND, July 10, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that two poster presentations related to its clinical program for recessive dystrophic epidermolysis bullosa (RDEB) were featured at the Society for Pediatric Dermatology (SPD) 45th Annual Meeting. The first poster includes a detailed analysis of patients with RDEB in the EB-101…

    • EB-101 treatment of large, chronic wounds is associated with durable healing and pain relief in patients with RDEB



    • Literature review of 65 studies confirms and expands understanding of substantial disease burden of RDEB with considerable clinical, economic and humanistic impact on patients and their families

    NEW YORK and CLEVELAND, July 10, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that two poster presentations related to its clinical program for recessive dystrophic epidermolysis bullosa (RDEB) were featured at the Society for Pediatric Dermatology (SPD) 45th Annual Meeting. The first poster includes a detailed analysis of patients with RDEB in the EB-101 Phase 1/2a trial showing that wound healing following EB-101 treatment was associated with improved long-term pain relief. A separate poster provides insights on the significant disease burden associated with RDEB, highlighting data from a literature review on the clinical characteristics, humanistic consequences and economic impact of living with RDEB on patients and their families.

    "The large wounds of RDEB cause substantial pain, and only palliative treatments are currently available," said João Siffert, M.D., Chief Executive Officer of Abeona. "The data presented at SPD showed that EB-101 treatment of large, chronic wounds resulted in considerable and durable reduction in wound burden, which was associated with long-term pain relief for up to five years. The second poster at SPD helps to characterize the disease burden and management of RDEB, providing an important reminder of the extraordinary toll RDEB takes on quality of life, and underscores the need for therapies that reduce wound burden and the associated humanistic and economic impact."

    EB-101 Treatment of Large, Chronic Wounds Is Associated with Durable Healing and Pain Reduction in Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB)

    Jean Tang, M.D., Ph.D., Professor of Dermatology, Stanford University Medical Center and Principal Investigator of the EB-101 pivotal Phase 3 VIITALTM study, presented long-term outcomes following EB-101 treatment for large, chronic wounds in patients with RDEB. EB-101 treatment resulted in considerable and durable reduction in wound burden in the range of three to five years in a Phase 1/2a study. Wound healing of 50% or greater following EB-101 treatment was associated with no pain at treated sites at three years, four years and five years post-treatment, compared with presence of pain in 53% of wound sites at baseline. The ongoing VIITALTM study will further characterize the relationship between reduction of wound burden and pain relief following EB-101 treatment.

    The Full Burden of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

    M. Peter Marinkovich, M.D., Bullous Disease Clinic Director, Stanford University Medical Center, and Investigator in the VIITALTM study, presented findings from a literature review of 65 studies that provide new insights on the disease burden from the perspective of patients with RDEB and their families. Key observations of the clinical, humanistic and economic burden of RDEB include:

    • Large, chronic wounds comprise a major clinical burden of RDEB and are correlated with pain.
    • Many patients experience anxiety and depression.
    • Parents of children with RDEB reported negative effects on their relationship, choosing to not have more children.
    • 50% of U.S. families characterized the economic impact of managing RDEB as "high" or "severe."

    Abeona's posters from the SPD 45th Annual Meeting are available on the "News/Events" page under the "Investors & Media" section of Abeona's website at www.abeonatherapeutics.com.

    About Recessive Dystrophic Epidermolysis Bullosa

    Recessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning type VII collagen, which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

    About EB-101

    EB-101 is an autologous, gene-corrected cell therapy currently being investigated in the pivotal Phase 3 VIITALTM study for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. The EB-101 VIITALTM study is a multi-center, randomized clinical trial enrolling 10 to 15 RDEB patients with approximately 30 large, chronic wound sites treated in total. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into a patient's own skin cells (keratinocytes and their progenitors) and transplanting them back to the patient to enable normal Type VII collagen expression and facilitate wound healing. Abeona produces EB-101 for the VIITALTM study at its fully-functional gene and cell therapy manufacturing facility in Cleveland, OH. In a Phase 1/2a clinical trial, EB-101 provided durable wound healing for RDEB patients lasting 2+ to 5+ years, including for the largest, most challenging wounds that affect the majority of the RDEB population. More information on the clinical trials of EB-101 can be found at https://www.abeonatherapeutics.com/clinical-trials/rdeb and ClinicalTrials.gov (Identifier: NCT04227106).

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio of AAV-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona's novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITALTM study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.  These statements include statements about the Company's clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Company's goals and objectives.  We have attempted to identify forward looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:

    Greg Gin

    VP, Investor Relations

    Abeona Therapeutics

    +1 (646) 813-4709

    Media Contact:

    Scott Santiamo

    Director, Corporate Communications

    Abeona Therapeutics

    +1 (718) 344-5843

    Primary Logo

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  16. NEW YORK and CLEVELAND, July 09, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced the appointment of Michael Amoroso as Senior Vice President and Chief Commercial Officer (CCO), effective immediately. Mr. Amoroso brings to Abeona over 20 years of product commercialization experience in the biotechnology and pharmaceutical industries, most recently as Senior Vice President and Head of Worldwide Commercial, Cell Therapy at Kite, a Gilead Company.

    Mr. Amoroso will have overall responsibility for building the Company's commercial organization, developing the commercialization strategy for EB-101, its autologous, gene-corrected cell therapy for the treatment of…

    NEW YORK and CLEVELAND, July 09, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced the appointment of Michael Amoroso as Senior Vice President and Chief Commercial Officer (CCO), effective immediately. Mr. Amoroso brings to Abeona over 20 years of product commercialization experience in the biotechnology and pharmaceutical industries, most recently as Senior Vice President and Head of Worldwide Commercial, Cell Therapy at Kite, a Gilead Company.

    Mr. Amoroso will have overall responsibility for building the Company's commercial organization, developing the commercialization strategy for EB-101, its autologous, gene-corrected cell therapy for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) and its lead product candidate, as well as leading pre-commercial planning for its investigational adeno-associated virus vector (AAV)-based gene therapies.

    "Michael is a highly accomplished commercial leader with a focus on cell and gene therapies and a proven track record of launching innovative drugs for rare diseases," said João Siffert, M.D., Chief Executive Officer of Abeona. "As we advance our pivotal Phase 3 VIITAL™ study of EB-101 in RDEB, Michael's proven track record in commercialization, supply chain management for personalized, autologous cell therapies, experience in developing novel launch plans, working closely with governments around the world to ensure patients have access, and ability for building commercial and organizational capabilities will lay the groundwork for our potential go-to-market strategy for EB-101. Furthermore, his history of integrating commercial perspective into pipeline programs will be instrumental in positioning our investigational AAV gene therapies to shape the treatment paradigm for patients with MPS IIIA, MPS IIIB, and other rare genetic diseases."

    Prior to joining Abeona, Mr. Amoroso held various senior level commercial positions at leading biopharmaceutical companies, including Kite, Eisai Inc., Celgene Corporation (now a subsidiary of Bristol-Myers Squibb Company), and Sanofi. At Kite, he was responsible for the company's worldwide commercial organization leading the commercialization efforts for the autologous CAR T-cell therapy, YESCARTA®, and the future cell therapy pipeline. Before Kite, Mr. Amoroso was Senior Vice President, Americas for Eisai's Commercial Oncology Business Group, where he was accountable for teams charged with creating and driving commercial strategy and implementation for the company's approved products and earlier-stage assets. Previously, Mr. Amoroso worked at Celgene for six years in several commercial roles before serving as the organization's Commercial Lead for CAR T-cell therapy programs. In this capacity, he helped Celgene develop an organizational model to commercialize cell therapies including specialized manufacturing and customer services for patients with lymphoma and myeloma. Before joining Celgene, Mr. Amoroso held various marketing and sales leadership positions over his 10-plus year tenure at Sanofi. Mr. Amoroso earned his M.B.A. in Management from the Stern School of Business, New York University, and his B.A. in Biological Sciences, summa cum laude, from Rider University.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio of AAV-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona's library of novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITALTM study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.  These statements include statements about the Company's clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Company's goals and objectives.  We have attempted to identify forward looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:

    Greg Gin

    VP, Investor Relations

    Abeona Therapeutics

    +1 (646) 813-4709

    Media Contact:

    Scott Santiamo

    Director, Corporate Communications

    Abeona Therapeutics

    +1 (718) 344-5843

    Primary Logo

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  17. NEW YORK and CLEVELAND, July 08, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced recent updates on its clinical programs, highlighted by the restart of patient enrollment in the Company's pivotal Phase 3 VIITAL™ study of EB-101, its autologous, gene-corrected cell therapy, for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Enrollment in the VIITAL™ study at Stanford University Medical Center was paused in March 2020 in order to redirect healthcare resources to COVID-19 patients and to ensure the safety of study participants and site staff.

    "RDEB is a debilitating and life-threatening rare genetic disorder without an approved treatment…

    NEW YORK and CLEVELAND, July 08, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced recent updates on its clinical programs, highlighted by the restart of patient enrollment in the Company's pivotal Phase 3 VIITAL™ study of EB-101, its autologous, gene-corrected cell therapy, for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Enrollment in the VIITAL™ study at Stanford University Medical Center was paused in March 2020 in order to redirect healthcare resources to COVID-19 patients and to ensure the safety of study participants and site staff.

    "RDEB is a debilitating and life-threatening rare genetic disorder without an approved treatment option. Reinitiating enrollment in the VIITAL study is important for patients with RDEB and brings us a step closer toward concluding the clinical development of EB-101," said João Siffert, M.D., Chief Executive Officer of Abeona. "The unprecedented COVID-19 global health crisis has had a broad impact across our industry, and we will continue to monitor the pandemic to ensure the safety of trial participants, healthcare professionals and our employees."

    Abeona's MPS III Phase 1/2 Clinical Trials Continue Despite COVID-19 Pandemic

    Abeona also announced that two ongoing MPS III Phase 1/2 clinical trials of its investigational adeno-associated virus (AAV)-based gene therapies, ABO-102 and ABO-101, have continued to treat patients, with additional enrollment expected in the programs in the coming weeks. In June 2020, the 10th patient was dosed in cohort 3 of the ABO-102 Transpher A study for MPS IIIA (Sanfilippo syndrome type A), bringing the total to 16 patients. The Company previously reported in May 2020 that additional patients were treated in dose cohort 3 of the Transpher A study and the ABO-101 Transpher B study for MPS IIIB (Sanfilippo syndrome type B), bringing the total enrollment in the Transpher B study to 9 children to date.

    Dr. Siffert added, "We have worked closely with sites in our MPS III clinical studies to continue to enroll patients given the urgent need to treat children with Sanfilippo syndrome as early as possible. We are encouraged by the number of new pre-screened patients that may meet enrollment criteria and could allow dosing in the coming weeks. We also hope to resume on-site follow-up study visits that include neurocognitive assessments of already enrolled patients." 

    About Recessive Dystrophic Epidermolysis Bullosa

    Recessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning type VII collagen which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

    About EB-101

    EB-101 is an autologous, gene-corrected cell therapy currently being investigated in the pivotal Phase 3 VIITALTM study for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. The EB-101 VIITALTM study is a multi-center, randomized clinical trial enrolling 10 to 15 RDEB patients with approximately 30 large, chronic wound sites treated in total. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into a patient's own skin cells (keratinocytes and its progenitors) and transplanting them back to the patient to enable normal Type VII collagen expression and facilitate wound healing. Abeona produces EB-101 for the VIITALTM study at its fully-functional gene and cell therapy manufacturing facility in Cleveland, OH. In a Phase 1/2a clinical trial, EB-101 provided durable wound healing for RDEB patients lasting 2+ to 5+ years, including for the largest, most challenging wounds that affect the majority of the RDEB population. More information on the clinical trials of EB-101 can be found at https://www.abeonatherapeutics.com/clinical-trials/rdeb and ClinicalTrials.gov (Identifier: NCT04227106).

    About the VIITAL™ Phase 3 Study

    The VIITAL™ Phase 3 study is a multi-center, randomized clinical trial assessing EB-101 in up to 15 RDEB patients, with approximately 30 large, chronic wound sites treated in total. The primary outcome measure is wound healing, comparing treated with untreated wound sites on the same patient. Secondary endpoints include the assessments of pain, as well as other patient reported outcomes. Investigators at Stanford University Medical Center are currently enrolling eligible patients into the VIITALTM study. Additional information about the trial is available at https://www.abeonatherapeutics.com/clinical-trials/rdeb and ClinicalTrials.gov (Identifier: NCT04227106).  

    About The Transpher A Study

    The Transpher A Study (NCT02716246) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). The study, also known as ABT-001, is intended for patients 6 months to 2 years of age, or patients older than 2 years with a cognitive Developmental Quotient of 60% or above. The gene therapy ABO-102 is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

    About The Transpher B Study

    The Transpher B Study (NCT03315182) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-101 for the treatment of patients with Sanfilippo syndrome type B (MPS IIIB). The study, also known as ABT-002, is intended for patients from birth to 2 years of age, or patients older than 2 years with a cognitive Developmental Quotient of 60% or above. The gene therapy ABO-101 is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio of AAV-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona's library of novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITALTM study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.  These statements include statements about the Company's clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Company's goals and objectives.  We have attempted to identify forward looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:

    Greg Gin

    VP, Investor Relations

    Abeona Therapeutics

    +1 (646) 813-4709

    Media Contact:

    Scott Santiamo

    Director, Corporate Communications

    Abeona Therapeutics

    +1 (718) 344-5843

     

    Primary Logo

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  18. NEW YORK and CLEVELAND, June 18, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that two abstracts related to its clinical program for recessive dystrophic epidermolysis bullosa (RDEB) will be presented at the upcoming Society for Pediatric Dermatology (SPD) 45th Annual Meeting, to be held virtually during July 10-12, 2020. RDEB is a rare connective tissue disorder without an approved treatment, in which patients suffer from severe epidermal wounds that severely impact their lives.

    The first abstract features a more detailed examination than previously disclosed of data on long-term pain relief following durable healing of wounds in RDEB patients treated…

    NEW YORK and CLEVELAND, June 18, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that two abstracts related to its clinical program for recessive dystrophic epidermolysis bullosa (RDEB) will be presented at the upcoming Society for Pediatric Dermatology (SPD) 45th Annual Meeting, to be held virtually during July 10-12, 2020. RDEB is a rare connective tissue disorder without an approved treatment, in which patients suffer from severe epidermal wounds that severely impact their lives.

    The first abstract features a more detailed examination than previously disclosed of data on long-term pain relief following durable healing of wounds in RDEB patients treated with EB-101 in a Phase 1/2 study. Separately, a literature review on the clinical, humanistic and economic burden of RDEB will be presented.

    The posters will be presented by Jean Tang, M.D., Ph.D., Professor of Dermatology, Stanford University Medical Center and Principal Investigator of the EB-101 pivotal Phase 3 VIITALTM study, and M. Peter Marinkovich, M.D., Investigator on the VIITALTM study and Bullous Disease Clinic Director, Stanford University Medical Center. Details for the presentations are as follows:

    Title: Durable Healing and Pain Reduction in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Following EB-101 Treatment of Large, Chronic Wounds

    Presenter: Jean Tang, M.D. Ph.D.

    Virtual Session Date: July 10-12, 2020

    Title: The Full Burden of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

    Presenter: M. Peter Marinkovich, M.D.

    Virtual Session Date: July 10-12, 2020

    Following the conclusion of each virtual presentation, the posters will be available on the "Events" page under the "Investors & Media" section of Abeona's website at www.abeonatherapeutics.com.

    More details about the programs for the SPD Annual Meeting are available at https://pedsderm.net/meetings/annual-meeting/.

    About EB-101

    EB-101 is an autologous, gene-corrected cell therapy currently being investigated in the pivotal Phase 3 VIITALTM study for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. The EB-101 VIITALTM study is a multi-center, randomized clinical trial enrolling 10 to 15 RDEB patients with approximately 30 large, chronic wound sites treated in total. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into a patient's own skin cells (keratinocytes and its progenitors) and transplanting them back to the patient to enable normal Type VII collagen expression and facilitate wound healing. Abeona produces EB-101 for the VIITALTM study at its fully-functional gene and cell therapy manufacturing facility in Cleveland, OH. In a Phase 1/2a clinical trial, EB-101 provided durable wound healing for RDEB patients lasting 2+ to 5+ years, including for the largest, most challenging wounds that affect the majority of the RDEB population. More information on the clinical trials of EB-101 can be found at https://www.abeonatherapeutics.com/clinical-trials/rdeb and ClinicalTrials.gov.

    About Recessive Dystrophic Epidermolysis Bullosa

    Recessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning type VII collagen which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio of AAV-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona's library of novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITALTM study and is capable of clinical and commercial production of AAV gene therapies. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company's clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Company's goals and objectives. We have attempted to identify forward looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:

    Greg Gin

    VP, Investor Relations

    Abeona Therapeutics

    +1 (646) 813-4709

    Media Contact:

    Scott Santiamo

    Director, Corporate Communications

    Abeona Therapeutics

    +1 (718) 344-5843

    Primary Logo

    View Full Article Hide Full Article
  19. NEW YORK and CLEVELAND, June 17, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced Board of Director appointments that strengthen and expand the Company's leadership. George Migausky and Paul Mann were appointed as independent directors, effective June 17, 2020. In addition to their Board service, Mr. Migausky will serve as Chairman of the Company's Audit Committee and Mr. Mann will serve as a member of the Audit Committee.

    "George and Paul are highly regarded professionals with substantial financial management, operational, business development and capital raising experience in the biotechnology industry, bringing essential knowledge and expertise to Abeona…

    NEW YORK and CLEVELAND, June 17, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced Board of Director appointments that strengthen and expand the Company's leadership. George Migausky and Paul Mann were appointed as independent directors, effective June 17, 2020. In addition to their Board service, Mr. Migausky will serve as Chairman of the Company's Audit Committee and Mr. Mann will serve as a member of the Audit Committee.

    "George and Paul are highly regarded professionals with substantial financial management, operational, business development and capital raising experience in the biotechnology industry, bringing essential knowledge and expertise to Abeona," said Brian Pereira, M.D., Executive Chairman of Abeona. "The addition of George and Paul complements the skills and experiences of Abeona's existing Board members and leadership team, and we are confident that they will provide valuable perspective and guidance as we continue to advance our gene and cell therapies aimed at addressing urgent unmet needs."

    Mr. Migausky brings more than 30 years of experience in senior financial management to Abeona's Board, having served as Chief Financial Officer (CFO) of several public biopharmaceutical and clinical diagnostic companies. Most recently, Mr. Migausky served as interim CFO of Ocular Therapeutix, Inc. and previously served as Executive Vice President and CFO of Dyax Corp. for eight years through its acquisition by Shire plc for $6.4 billion. Mr. Migausky's previous roles include CFO of Wellstat Management Company, as well as CFO at IGEN International and BioVeris Corporation through their respective acquisitions by F. Hoffman LaRoche, and Manager, Emerging Business Services at Deloitte & Touche. Mr. Migausky currently serves on the Board of Directors and Chair of the Audit Committee at Immunovant, Inc., is an independent member of the Board of Directors at Hyperion Catalysis International, and a trustee for the Massachusetts Eye and Ear Institute. He received his MBA from Babson College and his BS from Boston College.

    Mr. Mann has over 20 years of experience in the financial and biotechnology industries. Most recently, he served as CFO at PolarityTE, Inc., a biotechnology and regenerative biomaterials company, where he was responsible for all financial operations. Prior to that, Mr. Mann was the Healthcare Portfolio Manager at Highbridge Capital Management and has held positions with Soros Fund Management, UBS Investment Group, Morgan Stanley and Deutsche Bank. Mr. Mann began his career as a scientist at Procter and Gamble and is named as an inventor on patents for skincare compounds and technologies. Mr. Mann has an MA (Cantab) and an MEng from Cambridge University where he studied Natural Sciences and Chemical Engineering.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio of AAV-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona's library of novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and commercial production of AAV gene therapies. For more information, visit www.abeonatherapeutics.com

    Forward-Looking Statements

    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company's clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Company's goals and objectives. We have attempted to identify forward looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:

    Greg Gin

    VP, Investor Relations

    Abeona Therapeutics

    +1 (646) 813-4709

    Media Contact:

    Scott Santiamo

    Director, Corporate Communications

    Abeona Therapeutics

    +1 (718) 344-5843

    Primary Logo

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  20. NEW YORK and CLEVELAND, May 28, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that João Siffert, M.D., Chief Executive Officer, will present at the Jefferies Virtual Healthcare Conference on Thursday, June 4, 2020 at 10:00 a.m. ET.

    A live webcast of the presentation will be available on the investor section of the Abeona Therapeutics website at www.abeonatherapeutics.com. The webcast replay will be available within 24 hours of the live presentation and will be accessible for 90 days.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical…

    NEW YORK and CLEVELAND, May 28, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that João Siffert, M.D., Chief Executive Officer, will present at the Jefferies Virtual Healthcare Conference on Thursday, June 4, 2020 at 10:00 a.m. ET.

    A live webcast of the presentation will be available on the investor section of the Abeona Therapeutics website at www.abeonatherapeutics.com. The webcast replay will be available within 24 hours of the live presentation and will be accessible for 90 days.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). www.abeonatherapeutics.com

    Investor Contact:
    Greg Gin
    VP, Investor Relations
    Abeona Therapeutics
    +1 (646) 813-4709

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

    Primary Logo

    View Full Article Hide Full Article
  21. First patient treated in ongoing pivotal Phase 3 VIITAL™ study evaluating EB-101 for RDEB

    Additional patients dosed in the Phase 1/2 gene therapy studies for MPS IIIA and MPS IIIB

    Positive interim data from MPS III gene therapy programs presented at WORLDSymposium™; updated results to be presented at ASGCT 2020

    Appointed two industry leaders to Board of Directors and strengthened management team

    Company to host investor conference call Thursday, May 7, 2020 at 8:30 a.m. ET

    NEW YORK and CLEVELAND, May 06, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced first quarter 2020 financial results, which will be discussed on a conference call scheduled for Thursday…

    First patient treated in ongoing pivotal Phase 3 VIITAL™ study evaluating EB-101 for RDEB

    Additional patients dosed in the Phase 1/2 gene therapy studies for MPS IIIA and MPS IIIB

    Positive interim data from MPS III gene therapy programs presented at WORLDSymposium™; updated results to be presented at ASGCT 2020

    Appointed two industry leaders to Board of Directors and strengthened management team

    Company to host investor conference call Thursday, May 7, 2020 at 8:30 a.m. ET

    NEW YORK and CLEVELAND, May 06, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced first quarter 2020 financial results, which will be discussed on a conference call scheduled for Thursday, May 7, 2020 at 8:30 a.m. ET. Interested parties are invited to participate in the call by dialing 844-455-1352 (U.S. toll-free) or 509-844-0155 (international), and reference conference ID 5352629, or via webcast at https://investors.abeonatherapeutics.com/ir-calendar.

    "During these challenging times, our priority remains to ensure the safety of our employees and patients, while supporting continuity of our business and clinical operations," said João Siffert, M.D., Chief Executive Officer of Abeona. "We have made considerable progress in our clinical programs in the first quarter of 2020, and are working with our investigators to minimize the impact of the COVID-19 pandemic. It is our intention to restore full patient access to our clinical programs as soon as possible. Our gene and cell therapies in development aim at addressing urgent unmet needs, and have the potential to provide durable benefit to patients who have no approved treatments."

    First Quarter and Recent Highlights

    • First patient treated in pivotal Phase 3 VIITAL™ study evaluating EB-101 for recessive dystrophic epidermolysis bullosa (RDEB). An additional 10 patients have been prescreened for this study.
    • Additional patients treated in dose cohort 3 of the Transpher A study and the Transpher B study.
    • Presented positive interim data from the Transpher A study of ABO-102 at WORLDSymposium™ demonstrating improved neurocognitive skills 18 months to two years post-treatment in MPS IIIA patients younger than 30 months, sustained, dose-related biomarker improvements, and a favorable safety profile.
    • Presented positive interim data from the Transpher B study of ABO-101 at WORLDSymposium™ demonstrating initial improvement in multiple disease-specific biomarkers, denoting clear biologic effects, and a favorable safety profile among MPS IIIB patients.
    • Updated interim results from the Transpher A and Transpher B studies to be presented during the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting, which will take place online May 12-15, 2020.
    • Two U.S. patents issued for adeno-associated virus (AAV) capsids exclusively licensed by Abeona from the University of North Carolina ("UNC"), generated using UNC's AIM™ vector platform.
    • Announced key appointments of industry leaders to its Board of Directors in April. Dr. Brian J. G. Pereira was appointed as Executive Chairman and Ms. Shawn Tomasello as an Independent Board Member. Dr. Pereira is a seasoned biopharmaceutical and healthcare leader with experience in financing and growing companies, including the clinical development and commercialization of innovative drug products. Ms. Tomasello has substantial commercial and strategic experience, including serving as Chief Commercial Officer at cell therapy pioneer Kite Pharma, which was acquired by Gilead Sciences.
    • Strengthened its leadership team with the appointments of Gregory Gin as Vice President, Investor Relations and Dr. Dan Rudin as Vice President, Clinical Development, focusing on the EB-101 program. Mr. Gin brings more than 25 years of investor relations, communications, and capital markets experience with small- and mid-cap biotechnology and specialty pharmaceutical companies developing novel treatments for orphan diseases and areas of high unmet medical need. Dr. Rudin has substantial research and development experience gained in industry and academia with focus on rare diseases, including lysosomal storage diseases. He has led several programs through the lifecycle of clinical development supporting multiple product approvals.

    Dr. Siffert continued, "We look forward to working with our new Executive Chairman, Dr. Brian Pereira and Independent Board Member, Ms. Shawn Tomasello. Both bring invaluable experience guiding biotech companies from clinical development through commercial launch. In addition, with the appointments of Greg and Dan, we have strengthened our leadership in investor relations and clinical development, respectively."

    COVID-19 Impact Mitigation

    The ongoing COVID-19 pandemic has caused meaningful disruptions to the global healthcare system, including the conduct of clinical trials as healthcare institutions shift their focus and resources to  treating COVID-19 patients. In response to the unprecedented challenges related to the COVID-19 pandemic, Abeona has taken several measures to protect and support the health of its employees and their families, healthcare partners and patients participating in its clinical trials. At the same time, the Company has implemented measures to maintain continuity of its operations and to preserve financial flexibility for the future.

    First Quarter Financial Results

    Cash, cash equivalents and marketable securities as of March 31, 2020, were $116 million compared to $129 million as of December 31, 2019. The decrease in cash of $13 million was driven by R&D expenses across our programs along with supporting administrative costs.

    The net loss was $0.52 per share for the first quarter of 2020, compared to $0.39 per share in the comparable period in 2019. The increase in the net loss per share results primarily from the non-cash impairment charge on the termination of the REGENXBIO license of $32.9 million, or $0.36 per share.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). www.abeonatherapeutics.com

    Forward-Looking Statements
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.  These statements include statements about the Company's clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Company's goals and objectives.  We have attempted to identify forward looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Greg Gin
    VP, Investor Relations
    Abeona Therapeutics
    +1 (646) 813-4709

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

    Primary Logo

    View Full Article Hide Full Article
  22. NEW YORK and CLEVELAND, April 21, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced it granted options to Brian Pereira, M.D., the newly-appointed Executive Chairman of the Company, to purchase 930,000 shares of Abeona common stock. The stock options have a $2.50 exercise price per share, which is equal to the closing price of Abeona's common stock on the grant date. The options were offered as a material inducement to Dr. Pereira's appointment as Executive Chairman.

    The options were granted outside of the Company's 2015 Equity Incentive Plan but will have terms and conditions consistent with those set forth in that plan. The options were approved by the Abeona…

    NEW YORK and CLEVELAND, April 21, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced it granted options to Brian Pereira, M.D., the newly-appointed Executive Chairman of the Company, to purchase 930,000 shares of Abeona common stock. The stock options have a $2.50 exercise price per share, which is equal to the closing price of Abeona's common stock on the grant date. The options were offered as a material inducement to Dr. Pereira's appointment as Executive Chairman.

    The options were granted outside of the Company's 2015 Equity Incentive Plan but will have terms and conditions consistent with those set forth in that plan. The options were approved by the Abeona Compensation Committee of the Board of Directors in reliance on the employment inducement exception under Nasdaq Listing Rule 5635(c)(4), which requires that grants relying on this exception be disclosed promptly in a press release.

    The options have a 10-year term and will vest 25% on the one-year anniversary of the grant date and in equal monthly installments over the following three years. Additionally, 100% of the options will be accelerated in the event of certain qualifying terminations of Dr. Pereira's relationship following a change of control of the Company.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). www.abeonatherapeutics.com

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics Inc.
    +1 (718) 344-5843

    Investor Contact:
    Dan Ferry
    LifeSci Advisors, LLC
    +1 (617) 535-7746

    Primary Logo

    View Full Article Hide Full Article
  23. NEW YORK and CLEVELAND, April 21, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced appointments of Brian Pereira, M.D. and Shawn Tomasello, M.B.A. as Executive Chairman and Independent Board Member, respectively.

    "On behalf of the Board, I am pleased to welcome Brian and Shawn, and look forward to collaborating with them as we guide Abeona to success," said Steven H. Rouhandeh, former Executive Chairman and current Director. "Each brings seasoned insights, leadership, and an array of knowledge that will be extremely valuable as the Company evolves from a clinical to commercial-stage organization." 

    Dr. Pereira, President and CEO of Visterra, Inc. (ACQ: Otsuka…

    NEW YORK and CLEVELAND, April 21, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced appointments of Brian Pereira, M.D. and Shawn Tomasello, M.B.A. as Executive Chairman and Independent Board Member, respectively.

    "On behalf of the Board, I am pleased to welcome Brian and Shawn, and look forward to collaborating with them as we guide Abeona to success," said Steven H. Rouhandeh, former Executive Chairman and current Director. "Each brings seasoned insights, leadership, and an array of knowledge that will be extremely valuable as the Company evolves from a clinical to commercial-stage organization." 

    Dr. Pereira, President and CEO of Visterra, Inc. (ACQ: Otsuka America, Inc.), brings proven leadership to Abeona fostered during a distinguished career in guiding several biopharma companies to success. Equally, Ms. Tomasello has extensive experience in several leadership roles, most notably as Chief Commercial Officer (CCO) at cell therapy pioneer Kite Pharmaceuticals (ACQ: Gilead).

    "With an impressive clinical pipeline of gene and cell therapies, a novel capsid platform, and world-class GMP manufacturing operations, Abeona has tremendous potential to be a leader in the exciting field of genetic medicine that is making treatment for those with rare genetic diseases a reality," said Dr. Pereira. "Fulfilling the promise of Abeona's mission, led by the most advanced gene therapy program in Recessive Dystrophic Epidermolysis Bullosa, is central to my commitment to the company and its investors."

    Ms. Tomasello said, "My past experiences have several parallels to the opportunity at Abeona, most notably at Kite, which was a true innovator in the fields of autologous cell therapy, biotech, and medicine as a whole. Moreover, Abeona's fully-integrated business has a range of unique qualities rarely found in a company of its size, and has it poised to reach its full potential." 

    Dr. Pereira and Ms. Tomasello were nominated by funds affiliated with Great Point Partners, LLC (GPP) pursuant to the previously disclosed letter agreement entered into as part of the Company's December 24, 2019 underwritten public offering, which was supported by a considerable investment from GPP. As contemplated by that agreement, former Executive Chairman Steven H. Rouhandeh has remained as a Director and Richard Van Duyne and Mark Alvino have resigned from the Board. In conjunction with a leading executive search firm, the Board has formed a search committee to identify a new, highly-qualified Independent Director to add to the Board who would also serve as Chair of the Audit Committee. Dr. Pereira and Ms. Tomasello are each independent of GPP.

    Dr. Pereira continued, "On behalf of the Board and all Abeona employees, I am grateful to Steve, Dick, and Mark for their service and dedication to the company. We wish Dick and Mark well and welcome continued leadership from Steve as a Board Director. I look forward to working with Joao Siffert, CEO, and his team in making the promise of Abeona a reality."

    Dr. Pereira brings over 30 years of expertise to Abeona as a veteran biopharmaceutical leader. His experience in financing and growing companies is seen at Visterra, where he continues to serve as President and CEO following the Company's 2018 acquisition by Otsuka America, Inc. Previously, Dr. Pereira served as the President and CEO of AMAG Pharmaceuticals where he built the clinical development, manufacturing, supply-chain, and commercial infrastructure for Feraheme®, underpinned by four financing rounds at increasing valuations. Previously, he held senior roles at Tufts Medical Center including President and CEO of a Physician Organization. Currently, Dr. Pereira serves as Chairman of the Board of Directors of Africa Healthcare Network and serves on the Boards of KalVista, Cullinan Pearl, and the American India Foundation. He earned a medical degree (M.B.B.S.) from St. John's Medical College, M.D. (Medicine), D.M. (Nephrology) from the Post Graduate Institute, and M.B.A. from Kellogg Graduate School of Management at Northwestern University.

    Ms. Tomasello brings over 30 years of experience in biopharmaceutical industry to Abeona. As CCO, she was a key executive in the $11.9 billion Kite Pharma/Gilead acquisition in 2017. Prior to joining Kite, Ms. Tomasello was the CCO of Commercial and Medical Affairs at Pharmacyclics, surging its market cap from $8 billion to $19 billion, and culminating in the 2015 acquisition by AbbVie for $21 billion. She brings extensive strategic experience in building world class organizations encompassing commercial, compliance, regulatory, pricing, and medical affairs functions. Ms. Tomasello built the foundation for her career with leadership roles at Celgene Corporation, Genentech, Pfizer Laboratories, Miles Pharmaceuticals and Proctor & Gamble. She currently serves on the Board of Principia BioPharma, Urogen Pharma, Mesoblast Ltd., Gamida Cell, and Centrexion Therapeutics. Ms. Tomasello earned a B.S. in Marketing from the University of Cincinnati and M.B.A. from Murray State University, KY.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). www.abeonatherapeutics.com

    Forward Looking Statements
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company's pipeline of product candidates and clinical trials, including its gene therapy designed to address Recessive Dystrophic Epidermolysis Bullosa; commercialization prospects; the Company's novel capsid platform; the Company's manufacturing operations; and the ongoing search for a suitable candidate to join the Company's Board of Directors and Audit Committee. We have attempted to identify forward-looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the ongoing effects of the coronavirus (COVID-19) pandemic, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise these forward-looking statements or update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics Inc.
    +1 (718) 344-5843

    Investor Contact:
    Dan Ferry
    LifeSci Advisors, LLC
    +1 (617) 535-7746

    Primary Logo

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  24.  

     

     

    UNITED STATES

    SECURITIES AND EXCHANGE COMMISSION

    Washington, DC 20549

     

    FORM 8-K

     

    CURRENT REPORT

    PURSUANT TO SECTION 13 OR 15(d) OF THE

    SECURITIES EXCHANGE ACT OF 1934

     

    Date of report (Date of earliest event reported): March 31, 2020

     

    ABEONA THERAPEUTICS INC.

    (Exact name of registrant as specified in its charter)

     

    Delaware   001-15771   83-0221517

    (State or other jurisdiction

    of incorporation)

     

    (Commission

    File Number)

     

    (I.R.S. Employer

    Identification No.)

     

    1330 Avenue of the Americas, 33rd Floor,

    New York, NY 10019

    (Address of principal executive offices) (Zip Code)

     

    (646) 813-4712
    (Registrant’s telephone

     

     

     

    UNITED STATES

    SECURITIES AND EXCHANGE COMMISSION

    Washington, DC 20549

     

    FORM 8-K

     

    CURRENT REPORT

    PURSUANT TO SECTION 13 OR 15(d) OF THE

    SECURITIES EXCHANGE ACT OF 1934

     

    Date of report (Date of earliest event reported): March 31, 2020

     

    ABEONA THERAPEUTICS INC.

    (Exact name of registrant as specified in its charter)

     

    Delaware   001-15771   83-0221517

    (State or other jurisdiction

    of incorporation)

     

    (Commission

    File Number)

     

    (I.R.S. Employer

    Identification No.)

     

    1330 Avenue of the Americas, 33rd Floor,

    New York, NY 10019

    (Address of principal executive offices) (Zip Code)

     

    (646) 813-4712
    (Registrant’s telephone number, including area code)

     

    N/A

    (Former name or former address, if changed since last report)

     

    Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):

     

    [  ] Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

     

    [  ] Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

     

    [  ] Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

     

    [  ] Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

     

    Securities registered pursuant to Section 12(b) of the Act:

     

    Title of Each Class   Trading Symbol   Name of each exchange on which registered
    Common Stock, $0.01 par value   ABEO   Nasdaq Capital Market

     

    Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

     

    Emerging growth company [  ]

     

    If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. [  ]

     

     

     

     
     

     

    Item 5.02. Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers; Compensatory Arrangements of Certain Officers.

     

    On March 31, 2020, Abeona Therapeutics Inc. (the “Company”), and Steven H. Rouhandeh, Executive Chairman executed an amendment (the “Amendment”) to Mr. Rouhandeh’s separation agreement with the Company dated January 2, 2020, as amended (the “Separation Agreement”). Pursuant to the Amendment, Mr. Rouhandeh’s effective date of separation from the Company was amended to be the earlier of (i) June 30, 2020 or (ii) the date that the Board of Directors of the Company appoints a new Executive Chairman of the Company. All other material terms of the Separation Agreement remain as previously disclosed.

     

    2
     

     

    SIGNATURE

     

    Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

     

      Abeona Therapeutics Inc.
      (Registrant)
         
      By: /s/ Edward G. Carr
      Name:  Edward G. Carr
      Title: Chief Accounting Officer

     

    Date: April 6, 2020

     

    3

     

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  25. NEW YORK and CLEVELAND, March 27, 2020 (GLOBE NEWSWIRE) -- The COVID-19 pandemic has created extraordinary challenges across all aspects of healthcare. Many institutions and their brave staff of providers are now focused on caring for patients stricken by the virus.  We at Abeona are thankful for their sacrifices and efforts, and stand behind these healthcare institutions whose resources are focused on the greater good. We are dedicated to being a good global citizen in doing our part by following guidance from local health officials in New York, Cleveland, and Madrid, and where remote employees reside. 

    Above all, we will take every reasonable measure to ensure the safety of our patients and employees, while sustaining our business operations…

    NEW YORK and CLEVELAND, March 27, 2020 (GLOBE NEWSWIRE) -- The COVID-19 pandemic has created extraordinary challenges across all aspects of healthcare. Many institutions and their brave staff of providers are now focused on caring for patients stricken by the virus.  We at Abeona are thankful for their sacrifices and efforts, and stand behind these healthcare institutions whose resources are focused on the greater good. We are dedicated to being a good global citizen in doing our part by following guidance from local health officials in New York, Cleveland, and Madrid, and where remote employees reside. 

    Above all, we will take every reasonable measure to ensure the safety of our patients and employees, while sustaining our business operations during this uncertain time. Abeona is fully focused on getting through the pandemic by working closely with our clinical trial sites to ensure that patient safety remains paramount. 

    Clinical Development
    We remain committed to advancing our clinical programs, but recognize delays are inevitable in these uncertain times, especially as healthcare resources are justly redirected to those who need them most. We are continually assessing the dynamic situation and implementing plans to minimize disruption. We are also constantly reviewing these plans and associated processes and policies to ensure our patients and employees are safe, and continuity in our scaled back operations remains.

    While the full impact on our clinical programs cannot be quantified at this point, what we do know is that all current clinical trial sites remain active. We can say with certainty that some sites have paused screening and delays are expected as the situation evolves globally. What we won't know for the foreseeable future is the long-term impact. However, we remain dedicated to communicating frequently and openly with our stakeholders as more information becomes available, including updates on material changes to prior guidance as we continue to follow applicable government, regulatory and institutional guidelines.

    Business Operations
    Looking inward, the safety of our employees is a top priority. We have instituted additional protective measures since news of COVID-19 broke, and we frequently assess and improve our safety practices and policies. Operations at our Cleveland manufacturing facility have been significantly scaled back to ensure that employees and those around them have the best chance to remain safe, and to accommodate reduced clinical development activities. Only employees deemed essential by senior management to maintaining the manufacturing operation are entering the facility and under strict safety protocols to mitigate their risk. More traditional means of risk mitigation including a global work from home policy and suspended business travel are also in place.

    As we try to rationalize the unprecedented developments sweeping the globe, please be assured that Abeona is doing all it can to protect our patients, employees, and the communities around us. This includes company operations that underpin our aspirations to bring new medicines to patients in need.

    Abeona is about great Science and great People, and it's important we continue to do everything we can to preserve both.

    We will get through this – but only if we work together.


    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). www.abeonatherapeutics.com

    Investor Contact:
    Dan Ferry
    LifeSci Advisors, LLC
    +1 (617) 535-7746

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

    Primary Logo

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  26. Majority of potential study participants have been pre-screened

    EB-101 successfully manufactured at Abeona and transplanted at Stanford University Medical Center

    NEW YORK and CLEVELAND, March 17, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that investigators at Stanford University Medical Center have treated the first patient in the pivotal phase III VIITAL™ study evaluating EB-101, the Company's gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (RDEB).

    "Treating the first patient in our pivotal Phase III VIITAL™ study is an important achievement for the EB-101 program, now the most advanced gene therapy program in RDEB," said…

    Majority of potential study participants have been pre-screened

    EB-101 successfully manufactured at Abeona and transplanted at Stanford University Medical Center

    NEW YORK and CLEVELAND, March 17, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that investigators at Stanford University Medical Center have treated the first patient in the pivotal phase III VIITAL™ study evaluating EB-101, the Company's gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (RDEB).

    "Treating the first patient in our pivotal Phase III VIITAL™ study is an important achievement for the EB-101 program, now the most advanced gene therapy program in RDEB," said João Siffert, M.D., Chief Executive Officer. "This achievement confirms that Abeona can deliver EB-101 in a study setting that closely parallels its potential real-world application. We remain confident that VIITALTM will replicate results from the Phase I/II trial demonstrating that EB-101 treatment resulted in sustained and durable wound healing with a favorable safety profile."

    The VIITAL™ Phase III study is a multi-center, randomized clinical trial assessing EB-101 in up to 15 RDEB patients, with approximately 30 large, chronic wound sites treated in total. The primary outcome measure is wound healing, comparing treated with untreated wound sites on the same patient. Secondary endpoints include the assessments of pain, as well as other patient reported outcomes. Investigators at Stanford University Medical Center are currently enrolling eligible patients into the VIITALTM study and preparations for an additional clinical site initiation are ongoing. Additional information about the trial is available at abeonatherapeutics.com/clinical-trials/rdeb.

    Abeona is producing EB-101 for the VIITALTM study at the Elisa Linton Center for Rare Disease Therapies, its fully-functional gene and cell therapy manufacturing facility centrally-located in Cleveland, OH. The 26,000 ft2 center is housing large-scale cGMP capacity for AAV gene therapy and EB-101 cell therapy manufacturing, and state-of-the-art laboratories to support CMC development for process and analytics, all of which is validated and governed by comprehensive quality systems and overseen by experienced staff.

    About EB-101
    EB-101 is an autologous, gene-corrected cell therapy in late-stage clinical development for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into a patient's own skin cells (keratinocytes and its progenitors) and transplanting them back to the patient to enable normal Type VII collagen expression and facilitate wound healing. Data from a Phase I/IIa clinical trial conducted by Stanford University evaluating EB-101 showed that the gene-corrected cell therapy provided durable wound healing for RDEB patients lasting 2+ to 5+ years, including for the largest, most challenging wounds that affect the majority of the RDEB population. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Breakthrough Therapy, and Rare Pediatric designations for EB-101 and Orphan Drug designation in both the U.S. and EU.

    About Recessive Dystrophic Epidermolysis Bullosa
    Recessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning Type VII collagen which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). www.abeonatherapeutics.com

    Forward Looking Statement
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company's clinical trials, including the timing and success thereof; the Company's products and product candidates; EB-101 can provide durable healing in large, chronic wounds that afflict many RDEB patients; future regulatory interactions with regulatory authorities; and the Company's goals and objectives. We have attempted to identify forward-looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise these forward-looking statements or update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Dan Ferry
    LifeSci Advisors, LLC
    +1 (617) 535-7746

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

    Primary Logo

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  27. On track to treat first RDEB patient in pivotal Phase 3 VIITAL™ study of EB-101 in Q1 2020

    Positive interim data from MPS IIIA and MPS IIIB programs presented at WORLDSymposium™;
    Completion of cohort 2 and first patient enrolled in cohort 3 of MPS IIIB study

    Issuance of U.S. Patents for AIM™ AAV capsids

    Closing of $103.5 million underwritten public offering

    Company to host investor conference call Tuesday, March 17 at 10:00 a.m. ET

    NEW YORK and CLEVELAND, March 16, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced fourth quarter and full year 2019 financial results, as well as business updates, which will be discussed on a conference call scheduled for Tuesday…

    On track to treat first RDEB patient in pivotal Phase 3 VIITAL™ study of EB-101 in Q1 2020

    Positive interim data from MPS IIIA and MPS IIIB programs presented at WORLDSymposium™;
    Completion of cohort 2 and first patient enrolled in cohort 3 of MPS IIIB study

    Issuance of U.S. Patents for AIM™ AAV capsids

    Closing of $103.5 million underwritten public offering

    Company to host investor conference call Tuesday, March 17 at 10:00 a.m. ET

    NEW YORK and CLEVELAND, March 16, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced fourth quarter and full year 2019 financial results, as well as business updates, which will be discussed on a conference call scheduled for Tuesday, March 17 at 10:00 a.m. ET. Interested parties are invited to participate in the call by dialing 844-369-8770 (domestic) or 862-298-0840 (international) or via webcast at https://www.webcaster4.com/Webcast/Page/1818/33644.

    "We have started 2020 strongly by opening enrollment in the pivotal Phase 3 VIITAL™ study evaluating EB-101 for RDEB and we remain on track to dose the first patient in the first quarter," said João Siffert, M.D., Chief Executive Officer of Abeona. "A majority of study participants have been pre-screened and interest from the RDEB community has been high. The recently published long-term follow-up data leave us confident that VIITAL™ will demonstrate EB-101 has the unique potential to durably heal the most challenging large and chronic wounds faced by RDEB patients."

    Dr. Siffert continued, "We have also made great strides in our MPS III gene therapy programs. Positive interim data from the ongoing Phase 1/2 Transpher A study demonstrated that ABO-102 preserved neurocognitive skills up to two years after treatment in MPS IIIA patients treated early in life. In MPS IIIB, dose-dependent and sustained reductions in disease-specific biomarkers demonstrated a clear biologic effect of ABO-101 treatment in the Phase 1/2 Transpher B study, which is now enrolling a third dose cohort. Finally, our $103.5 million gross underwritten public offering fully funds operations well into 2021 and enables the continued progress and momentum across our robust clinical pipeline."

    Fourth Quarter and Recent Highlights
    Opening of enrollment in the Phase 3 VIITAL™ study evaluating EB-101 for RDEB, which is on track to treat the first patient in Q1. The majority of study participants have been pre-screened and preparations for an additional clinical site initiation are ongoing.

    Presentation of positive interim data from ABO-102 gene therapy program in MPS IIIA at WORLDSymposium™:

    • Three young patients treated in high-dose cohort 3 (at ages 27 months, 19 months, and 12 months) continued to show preserved neurocognitive skills 18 months to two years post treatment, compared with natural history.
    • Across all cohorts (n=14), biomarker improvements included rapid and sustained, dose-related reductions in CSF-HS that reached lower limit of quantitation in Cohort 3 (n=2); a reduction in plasma HS levels; and a durable, dose-dependent reduction in liver volume with up to 2 years of follow up.
    • ABO-102 has been well-tolerated, with long-term safety remaining favorable 15-45 months post treatment. There have been no treatment-related severe adverse events and no clinically-significant adverse events reported.

    Presentation of positive interim data from ABO-101 gene therapy program in MPS IIIB at WORLDSymposium™:

    • Initial improvements in multiple disease-specific biomarkers including decreased cerebrospinal fluid heparan sulfate (HS) levels, reduced plasma and urine HS and glycosaminoglycans, reduced liver volume.
    • ABO-101 has been well-tolerated to date, with no treatment-related severe adverse events and no clinically-significant adverse events reported (n=8).

    Completion of cohort 2 and enrollment in cohort 3 of the ABO-101 Transpher B study in MPS IIIB.

    Closing of $103.5 million underwritten public offering strengthening balance sheet to support the VIITAL™ study through data readouts and the advancement of additional clinical programs.

    Receipt of EMA PRIME designation for ABO-102 program in MPS IIIA.

    Issuance of two U.S. Patents for AIM™ AAV capsids.

    Completion of strategic review.

    Fourth Quarter and Full Year Summary Financial Results
    Cash, cash equivalents and marketable securities as of December 31, 2019 were $129.3 million, compared to $47.9 million as of September 30, 2019. The increase in cash of $81.4 million was driven primarily by $103.5 million gross underwritten public offering.

    Net loss was $0.30 per share for the fourth quarter of 2019, compared to $0.36 per share in the comparable period in 2018. For the twelve months ended December 31, 2019, net loss was $1.51 per share compared to $1.19 per share in the same period in 2018.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). www.abeonatherapeutics.com

    Forward-Looking Statements
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.  These statements include statements about the Company's clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Company's goals and objectives.  We have attempted to identify forward looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Dan Ferry
    LifeSci Advisors, LLC
    +1 (617) 535-7746

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

    Primary Logo

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  28. NEW YORK and CLEVELAND, Feb. 26, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that João Siffert, M.D., Chief Executive Officer, will present at the 9th Annual SVB Leerink Global Healthcare Conference in New York City on Thursday, February 27, 2020 at 11:00 a.m. ET.

    A live webcast of the presentation will be available on the investor section of the Abeona Therapeutics website, www.abeonatherapeutics.com, and an archived replay will be accessible for up to 30 days.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include…

    NEW YORK and CLEVELAND, Feb. 26, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that João Siffert, M.D., Chief Executive Officer, will present at the 9th Annual SVB Leerink Global Healthcare Conference in New York City on Thursday, February 27, 2020 at 11:00 a.m. ET.

    A live webcast of the presentation will be available on the investor section of the Abeona Therapeutics website, www.abeonatherapeutics.com, and an archived replay will be accessible for up to 30 days.

    About Abeona Therapeutics

    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). www.abeonatherapeutics.com

    Investor Contact:
    Dan Ferry
    LifeSci Advisors, LLC
    +1 (617) 535-7746

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics Inc.
    +1 (718) 344-5843

    Primary Logo

    View Full Article Hide Full Article
  29. Neurocognitive development of young MPS IIIA patients preserved up to two years post ABO-102 treatment

    Dose-dependent and sustained reductions in disease-specific biomarkers denotes clear biologic effects of ABO-102 and ABO-101

    First patient treated in cohort 3 of ABO-101 MPS IIIB trial; total enrollment eight patients

    Favorable safety profile observed in both studies

    NEW YORK and CLEVELAND, Feb. 12, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that researchers from the Abigail Wexner Research Institute (AWRI) at Nationwide Children's Hospital presented positive interim data from two ongoing Phase I/II clinical trials evaluating ABO-102 and ABO-101, the…

    Neurocognitive development of young MPS IIIA patients preserved up to two years post ABO-102 treatment

    Dose-dependent and sustained reductions in disease-specific biomarkers denotes clear biologic effects of ABO-102 and ABO-101

    First patient treated in cohort 3 of ABO-101 MPS IIIB trial; total enrollment eight patients

    Favorable safety profile observed in both studies

    NEW YORK and CLEVELAND, Feb. 12, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that researchers from the Abigail Wexner Research Institute (AWRI) at Nationwide Children's Hospital presented positive interim data from two ongoing Phase I/II clinical trials evaluating ABO-102 and ABO-101, the Company's investigational gene therapies for MPS IIIA and MPS IIIB, respectively, at WORLDSymposium™. Results from the Transpher A study demonstrated that MPS IIIA patients younger than 30 months treated with ABO-102 in dose cohort 3 continue to show neurocognitive development 18 months to two years after treatment. Reductions in cerebrospinal fluid (CSF) heparan sulfate (HS), denoting enzyme activity in the central nervous system, and liver volume reductions remain stable two years after treatment. Results from the Transpher B study showed that ABO-101 also improved multiple disease biomarkers providing clear evidence of a biologic effect in patients with MPS IIIB. Dosing in cohort 2 is complete and the first patient in cohort 3 was treated in late January, with a total of 8 patients treated to date. Both therapies have been well-tolerated to date. Abeona licensed the AAV9-based gene therapy technology underpinning ABO-102 and ABO-101 from AWRI at Nationwide Children's where it was developed.

    Today's presentations are available on abeonatherapeutics.com by following this link: https://investors.abeonatherapeutics.com/news-events

    "In total, the new results continue to show that early treatment with ABO-102 can help preserve neurodevelopment in children with MPS IIIA. These data will inform our ongoing discussions with the FDA and EMA, as we work towards providing a regulatory update in the second quarter," said João Siffert, M.D., Chief Executive Officer. "For ABO-101, the reductions in disease-specific biomarkers are encouraging and demonstrate a clear biologic effect, which parallels that seen in the MPS IIIA study. We look forward to enrolling the Transpher B study as expeditiously as possible."

    Results from the Transpher A study, an ongoing Phase I/II clinical trial with ABO-102 showed that:

    • Three young patients treated in high-dose cohort 3 (at ages 27 months, 19 months, and 12 months) continued to show improved neurocognitive skills 18 months to two years post treatment, compared with natural history.
    • Across all cohorts (n=14), biomarker improvements included rapid and sustained, dose-related reductions in CSF-HS that reached lower limit of quantitation in Cohort 3 (n=2); a reduction in plasma HS levels; and a durable, dose-dependent reduction in liver volume with up to 2 years of follow up.
    • ABO-102 has been well-tolerated, with long-term safety remaining favorable 15-45 months post treatment. There have been no treatment-related severe adverse events and no clinically-significant adverse events reported.

    "The interim results presented today add to evidence suggesting a single intravenous dose of ABO-102 AAV9-based gene therapy has the potential to help MPS IIIA patients sustain neurocognitive development when they are treated at a young age," said Kevin Flanigan, M.D., Director, Center for Gene Therapy at AWRI at Nationwide Children's and Transpher A study investigator. "These data showed that ABO-102 can deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs, as evidenced by the clinical benefits in neurocognition and biophysical measures and improvements in disease-specific biomarkers." 

    Sites in the U.S., Spain, and Australia continue to enroll eligible patients into the Transpher A study. Additional information about the trial is available at AbeonaTrials.com and ClinicalTrials.gov.

    Results from cohorts 1 and 2 (n=7) of the Transpher B study, an ongoing Phase I/II clinical trial showed that ABO-101 treatment demonstrated biologic effect in patients with MPS IIIB, as evidenced by initial improvements in multiple disease biomarkers associated with abnormal accumulation of glycosaminoglycans (GAGs) in the brain and throughout the body:

    • Initial improvements in multiple disease-specific biomarkers include:
      • Decreased CSF-HS levels
      • Reduction in plasma and urine HS and GAGs
      • Reduction in liver volume
    • ABO-101 has been well-tolerated to date, with no treatment-related severe adverse events and no clinically-significant adverse events reported (n=8)

    "The Transpher B study provides hope that we may one day alter the course of this devastating disease," said Kim McBride, M.D., Principal Investigator at AWRI at Nationwide Children's and co-investigator for the Transpher B study. "The impact on disease biomarkers in the early stages of follow up suggest the potential of ABO-101 gene therapy to break down the accumulation of glycosaminoglycans that underlie MPS IIIB pathology. I look forward to working with fellow investigators to gather more data from the study, including results from high-dose cohort 3."   

    Dose cohort 2 has been completed and dosing is underway in cohort 3 (n=1). Sites in the U.S., Spain, and France continue to enroll eligible patients into the Transpher B study. Additional information about the trial is available at abeonatherapeutics.com/clinical-trials and ClinicalTrials.gov.

    About The Transpher A Study
    The Transpher A Study (NCT02716246) is an ongoing, two-year, open-label, dose-escalation, Phase I/II global clinical trial assessing ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). The study, also known as ABT-001, is intended for patients 6 months to 2 years of age, or patients older than 2 years with a cognitive Developmental Quotient of 60% or above. The study has enrolled 14 patients to date across three dose-escalating cohorts (N=3, N=3, N=8) and remains open for enrollment. The gene therapy ABO-102 is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

    About The Transpher B Study
    The Transpher B Study (NCT03315182) is an ongoing, two-year, open-label, dose-escalation, Phase I/II global clinical trial assessing ABO-101 for the treatment of patients with Sanfilippo syndrome type B (MPS IIIB). The study, also known as ABT-002, is intended for patients 6 months to 2 years of age, or patients older than 2 years with a cognitive Developmental Quotient of 60% or above. The study has enrolled 8 patients to date across three dose-escalating cohorts (N=2, N=5, N=1) and remains open for enrollment. The gene therapy ABO-101 is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

    About ABO-102
    ABO-102 is a novel gene therapy in Phase I/II development for Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). ABO-102 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations for the ABO-102 clinical program. In the EU, the Company holds PRIME and Orphan medicinal product designations.

    About ABO-101
    ABO-101 is a novel gene therapy in Phase I/II development for Sanfilippo syndrome type B (MPS IIIB), a rare lysosomal storage disease with no approved therapy that primarily affects the central nervous system (CNS). ABO-101 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the NAGLU gene to cells of the CNS and peripheral tissues. The therapy is designed to address the underlying NAGLU enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Fast Track and Rare Pediatric Disease designations for ABO-101 and Orphan Drug designation in both the U.S. and EU.

    About Sanfilippo Syndrome Type A (MPS IIIA)
    Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the CNS and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIA present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive facial features with thick eyebrows or a unibrow, full lips and excessive body hair for one's age, and liver/spleen enlargement are also present in early childhood. MPS IIIA is caused by genetic mutations that lead to a deficiency in the SGSH enzyme responsible for breaking down glycosaminoglycans, which accumulate in cells throughout the body resulting in rapid health decline associated with the disorder.

    About Sanfilippo syndrome type B (MPS IIIB)
    Sanfilippo syndrome type B (MPS IIIB) is a rare and fatal lysosomal storage disease with no approved therapy that primarily affects the central nervous system and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIB present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive signs such as facial features with thick eyebrows or a unibrow, full lips and excessive body hair for one's age and liver/spleen enlargement are also present. The underlying cause of MPS IIIB is a deficiency in the NAGLU enzyme responsible for breaking down glycosaminoglycans, which accumulate throughout the body resulting in rapid decline associated with the disorder.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). www.abeonatherapeutics.com

    Forward Looking Statement
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.  These statements include statements about the Company's clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Company's goals and objectives.  We have attempted to identify forward looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Dan Ferry
    LifeSci Advisors, LLC
    +1 (617) 535-7746

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

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  30. NEW YORK and CLEVELAND, Jan. 21, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced its participation in the first World Congress on Epidermolysis Bullosa (EB2020), which is convening the world's leading experts on epidermolysis bullosa (EB) research and clinical management in London, January 19-23, 2020. Organized by The Global EB Alliance, EB2020 is a forum to share state-of-the art knowledge on this devastating disease and to debate the direction of future research.

    Dr. Jean Tang of Stanford University Medical Center will present updated recessive dystrophic epidermolysis bullosa (RDEB) natural history data on large wounds and highlights from a completed Phase…

    NEW YORK and CLEVELAND, Jan. 21, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced its participation in the first World Congress on Epidermolysis Bullosa (EB2020), which is convening the world's leading experts on epidermolysis bullosa (EB) research and clinical management in London, January 19-23, 2020. Organized by The Global EB Alliance, EB2020 is a forum to share state-of-the art knowledge on this devastating disease and to debate the direction of future research.

    Dr. Jean Tang of Stanford University Medical Center will present updated recessive dystrophic epidermolysis bullosa (RDEB) natural history data on large wounds and highlights from a completed Phase I/II clinical trial of Abeona's investigational EB-101 gene therapy for RDEB. The presentation is part of the Clinical Trials and Research Program track at the congress. Details of the presentation are as follows:
    Large wounds: an update on natural history data and EB-101
    Jean Tang, M.D., Ph.D.
    Professor of Dermatology, Stanford University Medical Center
    Tuesday, January 21
    14:25 GMT

    About Recessive Dystrophic Epidermolysis Bullosa
    Recessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning Type VII collagen which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

    About EB-101
    EB-101 is an autologous, gene-corrected cell therapy in late-stage clinical development for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. Treatment with EB-101 involves gene transfer to deliver COL7A1 genes into a patient's own skin cells (keratinocytes) and transplanting them back to the patient to enable normal Type VII collagen expression and facilitate wound healing. Data from a Phase I/IIa clinical trial conducted by Stanford University evaluating EB-101 showed that the gene-corrected cell therapy provided durable wound healing for RDEB patients lasting several 2+ to 5+ years, including for the largest, most challenging wounds that affect the majority of the RDEB population. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Breakthrough Therapy, and Rare Pediatric designations for EB-101 and Orphan Drug designation in both the U.S. and EU.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). www.abeonatherapeutics.com

    Forward Looking Statement
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements regarding our pipeline including the potential use of investigational product EB-101 in the treatment of recessive dystrophic epidermolysis bullosa (RDEB). We have attempted to identify forward-looking statements by such terminology as "may," "will," "anticipate," "believe," "estimate," "expect," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the risk of whether or when the Company will complete its Phase 3 clinical trial for EB-101 and any potential results thereof, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as maybe detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Dan Ferry
    LifeSci Advisors, LLC
    +1 (617) 535-7746

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

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  31. NEW YORK and CLEVELAND, Jan. 15, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that the United States Patent and Trademark Office (USPTO) issued a patent covering next-generation adeno-associated virus (AAV) capsids generated using the University of North Carolina's (UNC) AIM™ vector platform and that the USPTO allowed the claims of a companion patent application, which will issue in the next several weeks.  Both patents are exclusively licensed by the Company from UNC.

    "The issuance of these patents strengthens intellectual property around our library of AIM™ capsids that have shown potential to improve tropism profiles for a variety of devastating diseases…

    NEW YORK and CLEVELAND, Jan. 15, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that the United States Patent and Trademark Office (USPTO) issued a patent covering next-generation adeno-associated virus (AAV) capsids generated using the University of North Carolina's (UNC) AIM™ vector platform and that the USPTO allowed the claims of a companion patent application, which will issue in the next several weeks.  Both patents are exclusively licensed by the Company from UNC.

    "The issuance of these patents strengthens intellectual property around our library of AIM™ capsids that have shown potential to improve tropism profiles for a variety of devastating diseases," said João Siffert, M.D., Chief Executive Officer. "It is also a step forward in the development of next-generation AAV technology that could make re-treatment with gene therapy possible for patients who have been previously treated using certain AAV-based vectors."

    On January 14, 2020, the USPTO issued UNC's U.S. Patent No. 10,532,110, entitled "AAV Vectors Targeted to the Central Nervous System." The patent provides protection for methods of delivering Abeona's AAV204-based gene therapy vectors to the central nervous system until November 2036. 

    On January 2, 2020, the USPTO allowed the claims of a companion UNC patent application covering Abeona's AAV204-based gene therapy vector compositions.  A patent is expected to be issued for the application in the next several weeks, with an expected expiration date in November 2035.

    Abeona continues to develop the AIM™ capsid library and to generate improved vectors for use in gene therapies. AIM™ capsids use AAV biology to selectively target delivery of genetic payloads to the central nervous system (including the retina), lungs, eye, muscle, liver and other tissues with potentially improved tropism profiles key to enable treatment of a variety of devastating diseases. AIM™ vectors are non-virus-producing and have shown the potential to evade the immune response generated by exposure to naturally-occurring AAV vectors. The Company's AIM™ library contains more than 100 capsids with tissue tropisms selected for their potential to target a wide range of organs and multiple routes of delivery.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). www.abeonatherapeutics.com

    Forward Looking Statement
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company's ability to secure patents for its technology, the Company's products, product candidates including AIM platform; and the Company's goals and objectives. We have attempted to identify forward-looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise these forward-looking statements or update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Dan Ferry
    LifeSci Advisors, LLC
    +1 (617) 535-7746

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

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  32. Study open for enrollment and patient screening underway at Stanford University

    Company expects first patient to be treated in the first quarter of 2020

    EB-101 manufactured at Abeona facility in Cleveland

    NEW YORK and CLEVELAND, Jan. 13, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that it has received Institutional Review Board (IRB) approval from Stanford University to commence the VIITAL™ study, the Company's pivotal Phase 3 clinical trial evaluating EB-101 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The majority of patients targeted for enrollment have completed the pre-screening process at Stanford, and the Company expects…

    Study open for enrollment and patient screening underway at Stanford University

    Company expects first patient to be treated in the first quarter of 2020

    EB-101 manufactured at Abeona facility in Cleveland

    NEW YORK and CLEVELAND, Jan. 13, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that it has received Institutional Review Board (IRB) approval from Stanford University to commence the VIITAL™ study, the Company's pivotal Phase 3 clinical trial evaluating EB-101 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The majority of patients targeted for enrollment have completed the pre-screening process at Stanford, and the Company expects the first patient in the VIITAL™ study to be treated in the first quarter of 2020.

    "We expect 2020 to be a transformational year at Abeona, and we are proud to start it with the initiation of our pivotal Phase 3 study evaluating EB-101 in RDEB," said João Siffert, M.D., Chief Executive Officer. "We look forward to the first patient receiving EB-101 this quarter, setting in motion the final stages of this important program. We have devoted significant effort to establish and validate our independent, fully-functional GMP facility that will produce EB-101 for the VIITAL™ study and has capacity to support commercial launch. EB-101 has the potential to be the first approved therapy for RDEB and the only durable treatment to address large chronic wounds, which are the most painful and debilitating."

    The VIITAL™ Phase 3 study is a multi-center, randomized clinical trial assessing EB-101 in up to 15 RDEB patients, with approximately 30 large, chronic wound sites treated in total. The primary study endpoint is the proportion of wounds with greater than 50% healing at three months, comparing treated with untreated wound sites on the same patient. Secondary endpoints include the patient's global impression of change from baseline in pain as well as other patient reported outcomes assessing pain during dressing changes, pain impact and physical function. Investigators at Stanford University Medical Center are currently enrolling eligible patients into the VIITAL™ study, with additional study sites expected to be added in the coming months. Additional information about the trial is available at abeonatherapeutics.com/clinical-trials/rdeb.

    Abeona will produce EB-101 for the pivotal VIITAL™ study at the Elisa Linton Center for Rare Disease Therapies, its fully-functional gene and cell therapy manufacturing facility, centrally-located in Cleveland, OH. The center is a 26,000 ft2 facility housing large-scale cGMP capacity and state-of-the-art laboratories to support CMC development for process and analytics, all of which is validated and governed by comprehensive quality systems and overseen by experienced staff. The facility is also capable of clinical production of the Company's AAV gene therapies.

    About EB-101
    EB-101 is an autologous, gene-corrected cell therapy in late-stage clinical development for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. Treatment with EB-101 involves gene transfer to deliver COL7A1 genes into a patient's own skin cells (keratinocytes) and transplanting them back to the patient to enable normal Type VII collagen expression and facilitate wound healing. Data from a Phase I/IIa clinical trial conducted by Stanford University evaluating EB-101 showed that the gene-corrected cell therapy provided durable wound healing for RDEB patients lasting several 2+ to 5+ years, including for the largest, most challenging wounds that affect the majority of the RDEB population. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Breakthrough Therapy, and Rare Pediatric designations for EB-101 and Orphan Drug designation in both the U.S. and EU.

    About Recessive Dystrophic Epidermolysis Bullosa
    Recessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning Type VII collagen which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Its preclinical assets include ABO-401, which uses a novel vector from Abeona's AIM™ AAV capsid platform to address all mutations of cystic fibrosis. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). www.abeonatherapeutics.com

    Forward Looking Statement
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company's clinical trials, including the timing and success thereof; the Company's products and product candidates; EB-101 can provide durable healing in large, chronic wounds that afflict many RDEB patients; future regulatory interactions with regulatory authorities; and the Company's goals and objectives. We have attempted to identify forward-looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise these forward-looking statements or update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Dan Ferry
    LifeSci Advisors, LLC
    +1 (617) 535-7746

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

    Primary Logo

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  33. NEW YORK and CLEVELAND, Dec. 24, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced the closing of its underwritten public offering, with a gross offering size of approximately $103.5 million, which includes the full exercise of the underwriters' option to purchase 5,400,000 additional shares of common stock, at a public offering price of $2.50 per share. In addition, as part of the offering, Abeona sold to Great Point Partners ("GPP"), an existing investor, pre-funded warrants to purchase up to an aggregate of 9,017,055 shares of common stock at a purchase price of $2.4999 per pre-funded warrant, which equals the public offering price per share of the common…

    NEW YORK and CLEVELAND, Dec. 24, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced the closing of its underwritten public offering, with a gross offering size of approximately $103.5 million, which includes the full exercise of the underwriters' option to purchase 5,400,000 additional shares of common stock, at a public offering price of $2.50 per share. In addition, as part of the offering, Abeona sold to Great Point Partners ("GPP"), an existing investor, pre-funded warrants to purchase up to an aggregate of 9,017,055 shares of common stock at a purchase price of $2.4999 per pre-funded warrant, which equals the public offering price per share of the common stock less the $0.0001 per share exercise price of each pre-funded warrant.

    The Company has granted to affiliates of GPP the right to nominate two Board members to Abeona's Board of Directors, including a new Executive Chairman, due to GPP's considerable investment in the transaction. As a result, Steven H. Rouhandeh will step down as Executive Chairman and will retain a seat on the Board, while Mark J. Alvino and Richard Van Duyne will exit the Board. These changes will be effective upon the Board's qualification and election of GPP's nominees. Such replacement members will be independent of GPP.

    "Today's event strengthens our financial position, providing cash runway into 2021 and resources that will allow us to fund continued clinical development of pipeline products, including the initiation and enrollment of the EB-101 pivotal Phase 3 VIITALTM study, and achieve critical near-term milestones," said João Siffert, M.D., Chief Executive Officer of Abeona. "On behalf of the Board and all Abeona employees, I am grateful to our outgoing members for their service and dedication to the company, and particularly to Steven, who has served as Chairman of our Board of Directors for a number of years. Finally, I would like to thank our shareholders and new investors for their ongoing support and confidence in our pipeline and the Abeona team."

    "We are excited to lead this recapitalization of Abeona," said David Kroin, Managing Director of Great Point Partners.  "The Company is one of the world leaders in gene therapy technology, developing products using retrovirus, adeno-associated viruses and next generation capsids with potentially improved tropism profiles for a variety of devastating diseases. This funding greatly enhances Abeona's financial position, and we believe it can now reach its full potential, as other gene therapy companies in which Great Point Partners has invested have been able to do.  We intend to recruit the highest caliber people to guide the Company at the Board level in order to unlock the potential of a fully functioning manufacturing facility in Cleveland, Ohio, a late stage pivotal program in Recessive Dystrophic Epidermolysis Bullosa that has BreakThrough and RMAT Designations from the FDA, a pipeline of exciting neurology programs, and a wonderful team of professionals."

    Concurrently, the Company announced that its review of strategic options announced on September 3, 2019 has been completed. The Board of Directors concluded that it is in the best interest of the Company and its shareholders to develop its pipeline products independently, and with the additional funding and planned leadership nominations announced today. While the Board determined that this pathway was the best course of action to advance the Company's mission and maximize stakeholder value, it was not due to a lack of interested partners, and Abeona continues to entertain strategic alternatives consistent with standard industry practices.

    Jefferies LLC and SVB Leerink LLC acted as joint book-running managers and underwriters for the offering.

    The securities described above were offered pursuant to a shelf registration statement on Form S-3 (File No. 333-224867) that was filed with the Securities and Exchange Commission (the "SEC") on May 11, 2018 and amended on June 1, 2018, and that was declared effective by the SEC on June 7, 2018. The offering was made only by means of the written prospectus and prospectus supplement that form a part of the registration statement. The preliminary prospectus supplement and the accompanying prospectus that form a part of the registration statement has been filed with the SEC and is available on the SEC's website at www.sec.gov. Copies of the preliminary prospectus supplement and the accompanying prospectus may also be obtained by contacting Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, via telephone at (877) 821-7388, or email at: ; or SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6132, or by e-mail at .

    The securities described above have not been qualified under any state blue sky laws. This press release does not constitute an offer to sell or the solicitation of offers to buy any securities of Abeona being offered, and shall not constitute an offer, solicitation or sale of any security in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Its preclinical assets include ABO-401, which uses a novel vector from Abeona's AIM™ AAV capsid platform to address all mutations of cystic fibrosis. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102).

    Forward-Looking Statements
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.  These statements include statements about the Company's future liquidity and capital resources; the initiation and enrollment of the EB-101 pivotal Phase 3 VIITALTM study; the Company's clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Company's goals and objectives.  We have attempted to identify forward looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Dan Ferry
    LifeSci Advisors, LLC
    +1 (617) 535-7746

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

    Primary Logo

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  34. NEW YORK and CLEVELAND, Dec. 20, 2019 (GLOBE NEWSWIRE) --  Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced the pricing of its public offering of 26,982,945 shares of its common stock at a public offering price of $2.50 per share and in lieu of common stock, pre-funded warrants to purchase 9,017,055 shares of its common stock at a purchase price of $2.4999 per pre-funded warrant, which equals the public offering price per share of the common stock less the $0.0001 per share exercise price of each pre-funded warrant. The aggregate gross proceeds from the offering are expected to be $90 million, before deducting the underwriting discounts and commissions and other offering expenses payable…

    NEW YORK and CLEVELAND, Dec. 20, 2019 (GLOBE NEWSWIRE) --  Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced the pricing of its public offering of 26,982,945 shares of its common stock at a public offering price of $2.50 per share and in lieu of common stock, pre-funded warrants to purchase 9,017,055 shares of its common stock at a purchase price of $2.4999 per pre-funded warrant, which equals the public offering price per share of the common stock less the $0.0001 per share exercise price of each pre-funded warrant. The aggregate gross proceeds from the offering are expected to be $90 million, before deducting the underwriting discounts and commissions and other offering expenses payable by Abeona. All of the shares of common stock and pre-funded warrants are being offered by Abeona. In addition, Abeona has granted the underwriters a 30-day option to purchase up to an additional 5,400,000 shares of its common stock from Abeona at the public offering price, less the underwriting discounts and commissions. The offering is expected to close on December 24, 2019, subject to the satisfaction of customary closing conditions.

    An existing holder of the Company's common stock, Great Point Partners ("GPP"), has agreed to purchase approximately $31 million in the offering, including pre-funded warrants in lieu of common stock, subject to allocation by the underwriters and market and other conditions. The Company has granted GPP the right to nominate two directors, including a new Executive Chairman, to Abeona's Board of Directors. GPP has indicated that it expects such director nominees would be industry professionals not affiliated with GPP. As a result, Steven H. Rouhandeh will step down as Executive Chairman and will retain a seat on the Board, while Mark J. Alvino and Richard Van Duyne will exit the Board. These changes will be effective upon the Board's qualification and election of GPP's nominees.

    Jefferies LLC and SVB Leerink LLC are acting as book-running managers and underwriters for the offering.

    Abeona intends to use the net proceeds of the offering to fund continued clinical development of pipeline products, as well as for working capital and corporate purposes.

    The securities described above are being offered pursuant to a shelf registration statement on Form S-3 (File No. 333-224867) that was filed with the Securities and Exchange Commission (the "SEC") on May 11, 2018 and amended on June 1, 2018, and that was declared effective by the SEC on June 7, 2018. The offering will be made only by means of the written prospectus and prospectus supplement that form a part of the registration statement. The preliminary prospectus supplement and the accompanying prospectus that form a part of the registration statement has been filed with the SEC and is available on the SEC's website at www.sec.gov. Copies of the preliminary prospectus supplement and the accompanying prospectus may also be obtained by contacting Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, via telephone at (877) 821-7388, or email at: ; or SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6132, or by e-mail at .

    The securities described above have not been qualified under any state blue sky laws. This press release does not constitute an offer to sell or the solicitation of offers to buy any securities of Abeona being offered, and shall not constitute an offer, solicitation or sale of any security in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Its preclinical assets include ABO-401, which uses a novel vector from Abeona's AIM™ AAV capsid platform to address all mutations of cystic fibrosis. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102).

    Forward-Looking Statements
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.  These statements include statements about the offering and the Company's intended use of proceeds generated from the offering.  We have attempted to identify forward looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Dan Ferry
    LifeSci Advisors, LLC
    +1 (617) 535-7746

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

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  35. NEW YORK and CLEVELAND, Dec. 20, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to the Company's ABO-102 program studying its adeno-associated virus 9 (AAV9) gene therapy for Sanfilippo syndrome type A (MPS IIIA). The PRIME designation is based on nonclinical data and clinical data from the Transpher A Study, a global Phase 1/2 clinical trial evaluating a single-dose of ABO-102 for the treatment of children with MPS IIIA.

    "EMA's PRIME designation for the ABO-102 program recognizes the urgent need for a treatment option for children suffering from MPS IIIA, and underscores…

    NEW YORK and CLEVELAND, Dec. 20, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to the Company's ABO-102 program studying its adeno-associated virus 9 (AAV9) gene therapy for Sanfilippo syndrome type A (MPS IIIA). The PRIME designation is based on nonclinical data and clinical data from the Transpher A Study, a global Phase 1/2 clinical trial evaluating a single-dose of ABO-102 for the treatment of children with MPS IIIA.

    "EMA's PRIME designation for the ABO-102 program recognizes the urgent need for a treatment option for children suffering from MPS IIIA, and underscores the potential of ABO-102 to modify the course of this devastating lysosomal storage disease," said João Siffert, M.D., Chief Executive Officer.

    The Transpher A Study is enrolling patients at sites in the U.S., Spain, and Australia. Additional information about the trial is available at AbeonaTrials.com and ClinicalTrials.gov (NCT02716246).

    The PRIME initiative provides access to enhanced support for the development of medicines targeting an unmet medical need. The designation affords sponsors with enhanced interaction and early dialogue regarding promising medicines, as well as the possibility of accelerated assessment of medicines applications. PRIME is intended to optimize development plans and speed up evaluation so these medicines can help patients to benefit as early as possible from therapies that may significantly improve their quality of life.

    About ABO-102
    ABO-102 is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). ABO-102 is dosed in a one-time intravenous infusion using an AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations for the ABO-102 clinical program. In the EU, the Company holds PRIME and Orphan medicinal product designations.

    About The Transpher A Study
    The Transpher A Study (NCT02716246) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). The study, also known as ABT-001, is intended for patients 6 months to 2 years of age and patients older than 2 years with a cognitive Developmental Quotient of 60% or above. The study has enrolled 14 patients to date across three dose cohorts (N=3, N=3, N=8) and remains open for enrollment. The ABO-102 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study's primary endpoints are neurodevelopment and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

    About Sanfilippo Syndrome Type A (MPS IIIA)
    Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the CNS and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIA present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive facial features with thick eyebrows or a unibrow, full lips and excessive body hair for one's age, and liver/spleen enlargement are also present in early childhood. MPS IIIA is caused by genetic mutations that lead to a deficiency in the SGSH enzyme responsible for breaking down glycosaminoglycans, which accumulate in cells throughout the body resulting in rapid health decline associated with the disorder.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Its preclinical assets include ABO-401, which uses a novel vector from Abeona's AIM™ AAV capsid platform to address all mutations of cystic fibrosis. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102).

    Forward Looking Statement
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.  These statements include statements regarding the potential of ABO-102 to modify the course of lysosomal storage disease; our pipeline including the therapeutic potential for ABO-202 in the treatment of CLN1; the ability to obtain regulatory marketing approvals; and the Company's goals and objectives.  We have attempted to identify forward looking statements by such terminology as "may," will," "anticipate," "believe," "estimate," "expect," "intend," and similar expressions.

    Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to: continued interest in our rare disease portfolio, our ability to initiate and enroll patients in clinical trials, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's annual reports on Form 10-K and quarterly reports on Form 10-Q and other reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise the forward-looking statements or update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Dan Ferry
    LifeSci Advisors, LLC
    +1 (617) 535-7746

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843


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  36. NEW YORK and CLEVELAND, Dec. 19, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that it intends to offer and sell shares of its common stock, and pre-funded warrants in lieu of common stock, in an underwritten public offering pursuant to an existing shelf registration statement. All of the securities in the offering are to be sold by Abeona. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

    An existing holder of the Company's common stock, Great Point Partners ("GPP"), has indicated its intention to purchase up to $33 million in…

    NEW YORK and CLEVELAND, Dec. 19, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that it intends to offer and sell shares of its common stock, and pre-funded warrants in lieu of common stock, in an underwritten public offering pursuant to an existing shelf registration statement. All of the securities in the offering are to be sold by Abeona. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

    An existing holder of the Company's common stock, Great Point Partners ("GPP"), has indicated its intention to purchase up to $33 million in the offering, including pre-funded warrants in lieu of common stock, subject to allocation by the underwriter and market and other conditions, including the ability to nominate two directors, including a new Executive Chairman, to Abeona's Board of Directors. GPP has indicated that it expects such director nominees would be industry professionals not affiliated with such holder. As a result, Steven H. Rouhandeh would step down as Executive Chairman and would retain a seat on the Board, while Mark J. Alvino and Richard Van Duyne would exit the Board. These changes would be effective upon the Board's qualification and election of GPP's nominees.

    Jefferies LLC is acting as sole book-running manager and underwriter for the offering. Abeona intends to grant the underwriter a 30-day option to purchase additional shares of its common stock in an amount up to 15% of the securities sold in the offering, on the same terms and conditions.

    Abeona intends to use the net proceeds of the offering to fund continued clinical development of pipeline products, as well as for working capital and corporate purposes.

    The securities described above are being offered pursuant to a shelf registration statement on Form S-3 (File No. 333-224867) that was filed with the Securities and Exchange Commission (the "SEC") on May 11, 2018 and amended on June 1, 2018, and that was declared effective by the SEC on June 7, 2018. The offering will be made only by means of the written prospectus and prospectus supplement that form a part of the registration statement. The preliminary prospectus supplement and the accompanying prospectus that form a part of the registration statement has been filed with the SEC and is available on the SEC's website at www.sec.gov. Copies of the preliminary prospectus supplement and the accompanying prospectus may also be obtained by contacting Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, via telephone at (877) 821-7388, or email at: 

    The securities described above have not been qualified under any state blue sky laws. This press release does not constitute an offer to sell or the solicitation of offers to buy any securities of Abeona being offered, and shall not constitute an offer, solicitation or sale of any security in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Its preclinical assets include ABO-401, which uses a novel vector from Abeona's AIM™ AAV capsid platform to address all mutations of cystic fibrosis. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102).

    Forward Looking Statement
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.  We have attempted to identify forward looking statements by such terminology as "may," "will," "anticipate," "believe," "estimate," "expect," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. These statements include statements about the offering, the Company's intention to grant the underwriter a 30-day option to purchase additional shares in the offering, and the Company's intended use of proceeds generated from the offering.  Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and other reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Dan Ferry
    LifeSci Advisors, LLC
    +1 (617) 535-7746

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

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  37. FDA removes clinical hold; Company may proceed with VIITAL™ study

    Company expects to initiate study in the first quarter of 2020

    Primary endpoint confirmed as proportion of wounds with greater than 50% healing at 3 months vs control wounds

    Majority of potential subjects have been pre-screened for the study

    NEW YORK and CLEVELAND, Dec. 09, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that the U.S. Food and Drug Administration (FDA) has removed its clinical hold and provided clearance to proceed with the VIITAL™ study, the Company's pivotal Phase 3 clinical trial evaluating EB-101 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The…

    FDA removes clinical hold; Company may proceed with VIITAL™ study

    Company expects to initiate study in the first quarter of 2020

    Primary endpoint confirmed as proportion of wounds with greater than 50% healing at 3 months vs control wounds

    Majority of potential subjects have been pre-screened for the study

    NEW YORK and CLEVELAND, Dec. 09, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that the U.S. Food and Drug Administration (FDA) has removed its clinical hold and provided clearance to proceed with the VIITAL™ study, the Company's pivotal Phase 3 clinical trial evaluating EB-101 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The FDA removed the clinical hold following the Company's submission of additional data points on transport stability of EB-101 to clinical sites. Abeona expects to initiate the VIITAL™ study in first quarter of 2020.

    "The Abeona team has worked diligently to provide a prompt and thorough response to the FDA, enabling us to proceed with our pivotal Phase 3 trial for EB-101," said João Siffert, M.D., Chief Executive Officer of Abeona. "Recently published long-term follow up data from our Phase 1/2 trial leaves us increasingly confident that EB-101 can provide durable healing for large, chronic wounds that afflict many RDEB patients. We are now focused on initiating the VIITAL™ study in the first quarter of 2020. The success in building and qualifying a state-of-the-art GMP manufacturing facility also represents a critical step toward bringing this novel product to patients in dire need of effective treatment."

    With two to five years of follow-up, data from a Phase 1/2 clinical trial conducted by Stanford University evaluating EB-101 showed that the gene-corrected cell therapy provided durable wound healing for RDEB patients, including for the largest, most challenging wounds that constitute the majority of wounds in this population.

    About The VIITAL™ Study
    The VIITAL™ Phase 3 study will be a multi-center, randomized clinical trial assessing EB-101 in 10 to 15 RDEB patients, with approximately 30 chronic wound sites treated in total. The primary study endpoint will be the proportion of wounds with greater than 50% healing at three months, comparing treated with untreated wound sites on the same patient.  Secondary endpoints include the patient's global impression of change in pain from baseline as well as other patient reported outcomes assessing pain during dressing changes, pain impact and physical function.

    About EB-101
    EB-101 is an autologous, gene-corrected cell therapy in late-stage clinical development for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into a patient's own skin cells (keratinocytes) and transplanting them back to the patient to enable normal Type VII collagen expression and facilitate wound healing. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Breakthrough Therapy, and Rare Pediatric designations for EB-101 and Orphan Drug designation in both the U.S. and EU.

    About Recessive Dystrophic Epidermolysis Bullosa
    Recessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning Type VII collagen which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Its preclinical assets include ABO-401, which uses the novel AIM™ AAV vector platform to address all mutations of cystic fibrosis. Abeona has received twenty regulatory designations from the FDA and EMA for its pipeline candidates. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company's clinical trials, including the timing and success thereof; the Company's products and product candidates; EB-101 can provide durable healing in large, chronic wounds that afflict many RDEB patients; future regulatory interactions with regulatory authorities; and the Company's goals and objectives. We have attempted to identify forward-looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise these forward-looking statements or update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Dan Ferry
    LifeSci Advisors, LLC
    +1 (617) 535-7746

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

    Primary Logo

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    • Submitted additional EB-101 transport stability data to FDA in response to Clinical Hold Letter; CMC clearance for pivotal VIITALTM Phase 3 trial anticipated in Q4 2019
    • Publication of positive long-term data for EB-101 demonstrated sustained wound healing, relief of pain and itch, and favorable safety profile three years post-treatment
    • Ongoing strategic review of broad range of initiatives
    • Company to host investor conference call Wednesday, November 13 at 10:00 a.m. ET

    NEW YORK and CLEVELAND, Nov. 12, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced third quarter 2019 financial results and business updates, which will be discussed on a conference call scheduled…

    • Submitted additional EB-101 transport stability data to FDA in response to Clinical Hold Letter; CMC clearance for pivotal VIITALTM Phase 3 trial anticipated in Q4 2019
    • Publication of positive long-term data for EB-101 demonstrated sustained wound healing, relief of pain and itch, and favorable safety profile three years post-treatment
    • Ongoing strategic review of broad range of initiatives
    • Company to host investor conference call Wednesday, November 13 at 10:00 a.m. ET

    NEW YORK and CLEVELAND, Nov. 12, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced third quarter 2019 financial results and business updates, which will be discussed on a conference call scheduled for Wednesday, November 13 at 10:00 a.m. ET. Interested parties are invited to participate in the call by dialing 844-602-0380 (toll-free domestic) or 862-298-0970 (international) or via webcast at https://www.investornetwork.com/event/presentation/56788.

    "Abeona has made progress on our lead clinical program, EB-101, for the treatment of recessive dystrophic epidermolysis bullosa," said João Siffert, M.D., Chief Executive Officer of Abeona. "We continue to anticipate receiving CMC clearance for this pivotal trial by year-end following the recent submission of additional transport stability data for EB-101, in response to the Clinical Hold Letter received in the quarter, as well as the submission of updated clinical and comparability protocols for the VIITALTM Phase 3 study."

    Dr. Siffert continued, "We were also very pleased with the publication of positive long-term data from our Phase 1/2a clinical trial, reinforcing the potential of EB-101 to safely provide durable healing for the most challenging to treat, large and painful RDEB wounds. Three years post treatment, the majority of EB-101-treated wounds remained healed and without pain. We believe that EB-101 is uniquely positioned to address the needs of the majority of RDEB patients who suffer from these types of chronic wounds and we remain dedicated to delivering this therapy to the community."

    Third Quarter Financial Results:
    Cash, cash equivalents and marketable securities as of September 30, 2019, were $47.9 million compared to $62.5 million as of June 30, 2019. The decrease in cash was driven primarily by the net cash used in operating activities of $18.3 million.

    Research and development expenses for the third quarter ended September 30, 2019 were $10.9 million compared to $13.2 million in the same period of 2018. The decrease in R&D expense was primarily attributable to decreased clinical and development work, partially offset by increased salary and related costs from the hiring of additional clinical, regulatory, manufacturing and quality staff.

    General and administrative expenses for the third quarter ended September 30, 2019 were $4.7 million compared to $5.0 million in the same period of 2018. The decrease in G&A expenses was primarily due to decreased rental, recruiting, professional fee and salary related costs.

    Net loss was $0.35 per share for the third quarter of 2019 compared to $0.34 per share in the same period of 2018.

    Third Quarter and Recent Highlights:

    • Submitted additional transport stability data for EB-101 in response to September 17 FDA Clinical Hold Letter regarding the planned Phase 3 VIITAL™ study.
    • Submitted Phase 3 VIITAL™ clinical trial protocol with updated PRO assessments, and submitted the retrovirus comparability protocol to FDA.
    • Presentation of data from the Transpher A Study, the Company's ongoing Phase 1/2 clinical trial evaluating ABO-102 for the treatment of MPS IIIA, and research updates from its library of novel AIMTM adeno-associated virus capsids at the 27th European Society of Gene and Cell Therapy Congress.
    • Publication of positive long-term safety and efficacy data from the Phase 1/2a clinical trial of EB-101 in JCI Insight with collaborators from Stanford University School of Medicine.
      • Three years after treatment with EB-101, a majority of RDEB patients had sustained wound healing, with 80% (16/20) of wounds achieving ≥50% healing, and 70% (14/20) achieving ≥75%
      • Two years after treatment, only 1 of 6 untreated (17%), prospectively selected control wounds, had ≥50% healing
      • 50% or greater wound healing was associated with no pain (0/16) and no itch (0/16) at treated sites three years post-treatment, compared with presence of pain in 53% (20/38) and itch in 61% (23/38) of wound sites at baseline
      • EB-101 was associated with long-term molecular expression of type VII collagen protein, which plays an important role in anchoring the dermal and epidermal layers of the skin
      • No serious treatment-related adverse events were observed during the three-year observation period
    • Retained Jefferies LLC as its financial advisor to assist with the review of strategic initiatives focused on advancing the Company's mission and maximizing stakeholder value.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Its preclinical assets include ABO-401, which uses the novel AIM™ AAV vector platform to address all mutations of cystic fibrosis. Abeona has received twenty regulatory designations from the FDA and EMA for its pipeline candidates. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.  These statements include statements about the timing for CMC clearance for the VIITAL™ trial and the Company's beliefs relating thereto, the Company's ability to provide additional transport stability data points in response to the FDA clinical hold letter and the timing thereof, the Company's belief that completion of its CMC work and the durable safety and efficacy data will ultimately be critical to support a future Biologics License Application,  the ability of its management team to lead the Company and deliver on key strategies, the Company's clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Company's goals and objectives.  We have attempted to identify forward looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, risk of whether or when the FDA will lift the clinical hold respecting the Company's planned Phase 3 clinical trial for EB-101, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Sofia Warner
    Senior Director, Investor Relations
    Abeona Therapeutics
    +1 (646) 813-4710

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

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  38. NEW YORK and CLEVELAND, Oct. 21, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced the presentations of data from the Transpher A Study, the Company's ongoing Phase 1/2 clinical trial evaluating ABO-102 for the treatment of Sanfilippo syndrome type A (MPS IIIA), and research updates from its library of novel AIMTM adeno-associated virus (AAV) capsids at the 27th European Society of Gene and Cell Therapy (ESGCT) Congress, to be held October 22-25, 2019 in Barcelona, Spain.  

    The data will be presented as follows:

    Safety, Tolerability, Biopotency and Neurocognitive Data of ABO-102 in Transpher A, an Open-Label, Multicenter, Single-Dose, Dose-Escalation, Phase

    NEW YORK and CLEVELAND, Oct. 21, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced the presentations of data from the Transpher A Study, the Company's ongoing Phase 1/2 clinical trial evaluating ABO-102 for the treatment of Sanfilippo syndrome type A (MPS IIIA), and research updates from its library of novel AIMTM adeno-associated virus (AAV) capsids at the 27th European Society of Gene and Cell Therapy (ESGCT) Congress, to be held October 22-25, 2019 in Barcelona, Spain.  

    The data will be presented as follows:

    Safety, Tolerability, Biopotency and Neurocognitive Data of ABO-102 in Transpher A, an Open-Label, Multicenter, Single-Dose, Dose-Escalation, Phase 1/2 Clinical Trial in Sanfilippo Syndrome type A (Mucopolysaccharidosis IIIA)
    Oral Presentation #039
    Presenter: Maria Jose de Castro, M.D., Hospital Universitario Santiago de Compostela
    Session 4c: Metabolic and Genetic Diseases
    Date/Time: Friday, October 25, 2019, 9:00 a.m. to 11:00 a.m. CEST
    Location: Room 112

    Novel AAV Capsids Show Increased Evasion to Neutralizing Antibodies Against Natural Serotypes
    Poster #P347
    Session Title: Poster Session I
    Date/Time: Wednesday, October 23, 2019, 1:00 p.m. to 3:00 p.m. CEST
    Location: Multipurpose Hall

    Development of an Improved Novel AAV Capsids for Intramuscular Delivery
    Poster #P027
    Session Title: Poster Session I
    Date/Time: Wednesday, October 23, 2019, 1:00 p.m. to 3:00 p.m. CEST
    Location: Multipurpose Hall

    Novel AAV Capsids for Delivery to the Retina by Intravitreal Administration
    Poster #P009
    Session Title: Poster Session I
    Date/Time: Wednesday, October 23, 2019, 1:00 p.m. to 3:00 p.m. CEST
    Location: Multipurpose Hall

    Development of a Novel AAV Capsid with Improved PNS Tropism for Treating Pompe Disease by Intravenous Administration
    Poster #P007
    Session Title: Poster Session I
    Date/Time: Wednesday, October 23, 2019, 1:00 p.m. to 3:00 p.m. CEST
    Location: Multipurpose Hall

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Its preclinical assets include ABO-401, which uses the novel AIM™ AAV vector platform to address all mutations of cystic fibrosis. Abeona has received twenty regulatory designations from the FDA and EMA for its pipeline candidates. For more information, visit www.abeonatherapeutics.com.

    Forward-Looking Statements
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.  These statements include statements about the Company's clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Company's goals and objectives.  We have attempted to identify forward looking statements by such terminology as "may," "will," "believe," "estimate," "expect," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Sofia Warner
    Senior Director, Investor Relations
    Abeona Therapeutics
    +1 (646) 813-4710

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

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  39. Sustained wound healing and favorable safety profile observed at three years post-treatment

    Durable wound healing in large, disabling, chronic wounds

    EB-101 associated with long-term molecular expression of Type VII collagen protein

    NEW YORK and CLEVELAND, Oct. 15, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced positive long term efficacy and safety results from its Phase 1/2a clinical trial evaluating EB-101, a gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (RDEB). Treatment with EB-101 resulted in sustained wound healing with a favorable safety profile at three years post-treatment. These long-term follow-up data in seven RDEB…

    Sustained wound healing and favorable safety profile observed at three years post-treatment

    Durable wound healing in large, disabling, chronic wounds

    EB-101 associated with long-term molecular expression of Type VII collagen protein

    NEW YORK and CLEVELAND, Oct. 15, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced positive long term efficacy and safety results from its Phase 1/2a clinical trial evaluating EB-101, a gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (RDEB). Treatment with EB-101 resulted in sustained wound healing with a favorable safety profile at three years post-treatment. These long-term follow-up data in seven RDEB participants with 42 treated wounds were published in the peer-reviewed journal JCI Insight. Full text of the publication can be accessed here: https://insight.jci.org/articles/view/130554

    "These results bolster our belief that EB-101 is a safe and effective gene-corrected cell therapy capable of providing durable, long-lasting healing for the most disabling wounds in patients with RDEB," said João Siffert, M.D., Chief Executive Officer of Abeona. "These results are particularly significant, as EB-101 treatment led to wound healing even in the most challenging to treat large and painful chronic wounds. Given the average RDEB chronic wound size is over 118 cm2, it is essential that potential new treatments are capable of addressing these wounds to improve quality of life. We thank our collaborators at Stanford and the patients who volunteered to participate in this study and look forward to building upon this strong clinical foundation with the initiation of the pivotal Phase 3 VIITALTM Study evaluating EB-101 for the treatment of RDEB." 

    Key Study findings include:

    • Wounds selected for treatment were present for a mean of 11.2 years (range 3-20 years)
       
    • Three years after treatment with EB-101, a majority of RDEB patients had sustained wound healing, with 80% (16/20) of wounds achieving ³50% healing, and 70% (14/20) achieving ≥75%
       
    • Two years after treatment, only 1 of 6 untreated (17%), prospectively selected control wounds, had ≥50% healing
       
    • 50% or greater wound healing was associated with no pain (0/16) and no itch (0/16) at treated sites three years post-treatment, compared with presence of pain in 53% (20/38) and itch in 61% (23/38) of wound sites at baseline
       
    • EB-101 was associated with long-term molecular expression of type VII collagen protein, which plays an important role in anchoring the dermal and epidermal layers of the skin
       
    • No serious treatment-related adverse events were observed during the three-year observation period
       
    • No replication competent virus was present at any time point

    Researchers from Stanford University School of Medicine conducted the Phase 1/2a single-center, open-label clinical trial to evaluate the long-term wound healing and safety of EB-101 in seven adult patients with severe generalized RDEB and to assess patient-reported outcomes following treatment. Chronic open wounds, defined as wounds present and unhealed for at least 12 weeks, with a total area of at least 100 cm2, were required for enrollment. In the trial, gene-corrected EB-101 skin grafts (35 cm2 each) were transplanted onto six wound sites in each of the seven adult participants (n= 42 sites total) and wounds selected for treatment had been present for a mean of 11.2 years (range: 3-20 years). Participants were followed for two to five years after transplantation of EB-101 and received standard of care therapies including iron supplementation and esophageal dilations during the study.

    Abeona is currently continuing preparations for the pivotal Phase 3 VIITALTM Study evaluating EB-101 for the treatment of RDEB pending the anticipated receipt of Chemical, Manufacturing and Controls (CMC) clearance from the U.S. Food and Drug Administration expected in Q4 2019.

    About EB-101
    EB-101 is an investigational, autologous, gene-corrected cell therapy poised to enter late-stage development for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into a patient's own skin cells (keratinocytes) and transplanting them back to the patient to enable normal type VII collagen expression and facilitate wound healing. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Breakthrough Therapy, and Rare Pediatric designations for EB-101 and Orphan Drug designation in both the U.S. and EU.

    About Recessive Dystrophic Epidermolysis Bullosa
    Recessive dystrophic epidermolysis bullosa, or RDEB, is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning type VII collagen which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Its preclinical assets include ABO-401, which uses the novel AIM™ AAV vector platform to address all mutations of cystic fibrosis. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates and is the only company with Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). For more information, visit www.abeonatherapeutics.com.

    Forward Looking Statement
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the timing for CMC clearance for the VIITAL™ trial and the Company's beliefs relating thereto, the Company's ability to provide additional transport stability data points in response to the FDA clinical hold letter and the timing thereof, the Company's belief that completion of its CMC work and the durable safety and efficacy data will ultimately be critical to support a future Biologics License Application, the ability of its management team to lead the Company and deliver on key strategies, the market opportunities for the Company's products and product candidates, and the Company's goals and objectives. We have attempted to identify forward-looking statements by such terminology as "may," "will," "anticipate," "believe," "estimate," "expect," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the risk of whether or when the FDA will lift the clinical hold respecting the Company's planned Phase 3 clinical trial for EB-101, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as maybe detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Sofia Warner
    Senior Director, Investor Relations
    Abeona Therapeutics
    +1 (646) 813-4710

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

     

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    • Company to provide additional transport stability data points in response to FDA Clinical Hold Letter
    • CMC clearance for VIITAL™ trial anticipated in Q4 2019
    • Investor conference call today, Monday, September 23 at 09:30 a.m. ET

    NEW YORK and CLEVELAND, Sept. 23, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that it has recently received a clinical hold letter from the U.S. Food and Drug Administration (FDA) clarifying that the FDA will not provide approval for the Company to begin its planned Phase 3 clinical trial for EB-101 until it submits to the FDA additional data points on transport stability of EB-101 to clinical sites. Over the last 12 months, the…

    • Company to provide additional transport stability data points in response to FDA Clinical Hold Letter
    • CMC clearance for VIITAL™ trial anticipated in Q4 2019
    • Investor conference call today, Monday, September 23 at 09:30 a.m. ET

    NEW YORK and CLEVELAND, Sept. 23, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that it has recently received a clinical hold letter from the U.S. Food and Drug Administration (FDA) clarifying that the FDA will not provide approval for the Company to begin its planned Phase 3 clinical trial for EB-101 until it submits to the FDA additional data points on transport stability of EB-101 to clinical sites. Over the last 12 months, the Company has worked closely with the FDA to address and narrow open Chemical, Manufacturing and Controls (CMC) items and has been working to resolve this one item identified in the FDA Clinical Hold Letter. The Company continues to anticipate receiving CMC clearance for VIITAL™ trial in Q4 2019.

    "Initiating the VIITAL™ pivotal Phase 3 trial for EB-101 is the Company's top priority," said João Siffert, M.D., Chief Executive Officer of Abeona. "Efforts to gather supplemental data points on transport stability of EB-101 are ongoing and we are confident that the requested additional data will be submitted to the FDA promptly. Looking ahead, we believe that completion of our CMC work coupled with the durable safety and efficacy data, now out to five years in some patients, will ultimately be critical to support a future Biologics License Application. We remain deeply committed to advancing EB-101 to provide a desperately needed treatment for RDEB patients."

    The Company will hold a conference call scheduled for Monday, September 23 at 09:30 a.m. ET. Interested parties are invited to participate in the call by dialing 844-369-8770 (toll-free domestic) or 862-298-0840 (international) or via webcast at https://www.investornetwork.com/event/presentation/53728.

    About EB-101
    EB-101 is an investigational, autologous, gene-corrected cell therapy poised to enter late-stage development for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into a patient's own skin cells (keratinocytes) and transplanting them back to the patient to enable normal Type VII collagen expression and facilitate wound healing. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Breakthrough Therapy, and Rare Pediatric designations for EB-101 and Orphan Drug designation in both the U.S. and EU.

    About Recessive Dystrophic Epidermolysis Bullosa
    Recessive dystrophic epidermolysis bullosa, or RDEB, is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning Type VII collagen which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Its preclinical assets include ABO-401, which uses the novel AIM™ AAV vector platform to address all mutations of cystic fibrosis. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates and is the only company with Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). For more information, visit www.abeonatherapeutics.com.

    Forward Looking Statement
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.  These statements include statements about the timing for CMC clearance for the VIITAL™ trial and the Company's beliefs relating thereto, the Company's ability to provide additional transport stability data points in response to the FDA clinical hold letter and the timing thereof, the Company's belief that completion of its CMC work and the durable safety and efficacy data will ultimately be critical to support a future Biologics License Application, the ability of its management team to lead the Company and deliver on key strategies, the market opportunities for the Company's products and product candidates, and the Company's goals and objectives.  We have attempted to identify forward looking statements by such terminology as "may," "will," "anticipate," "believe," "estimate," "expect," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the risk of whether or when the FDA will lift the clinical hold respecting the Company's planned Phase 3 clinical trial for EB-101, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Sofia Warner
    Senior Director, Investor Relations
    Abeona Therapeutics
    +1 (646) 813-4710

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

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  40. NEW YORK and CLEVELAND, Sept. 03, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that it has retained Jefferies LLC as its financial advisor to assist with the review of strategic options focused on advancing the Company's mission and maximizing stakeholder value. In response to interest, Abeona has initiated a process to explore a broad range of strategic alternatives, including, but not limited to the partnering of its various clinical and pre-clinical programs, or a sale or merger of the Company, in an effort to unlock the potential of those assets.  

    "With four clinical stage programs, a broad pre-clinical pipeline, GMP manufacturing facilities, and a…

    NEW YORK and CLEVELAND, Sept. 03, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that it has retained Jefferies LLC as its financial advisor to assist with the review of strategic options focused on advancing the Company's mission and maximizing stakeholder value. In response to interest, Abeona has initiated a process to explore a broad range of strategic alternatives, including, but not limited to the partnering of its various clinical and pre-clinical programs, or a sale or merger of the Company, in an effort to unlock the potential of those assets.  

    "With four clinical stage programs, a broad pre-clinical pipeline, GMP manufacturing facilities, and a talented team, Abeona remains committed to advancing the promise of genetic medicine," said João Siffert, M.D., Chief Executive Officer. "Given our expertise in gene and cell therapy, we believe we are well positioned to explore a variety of opportunities, pursue strategic partnerships and alliances, improve our capital structure, and accelerate development of candidates towards commercialization." 

    There can be no assurance this strategic review will result in the completion of any particular course of action. There is no defined timeline for completion of the review process and the Company does not intend to comment further unless a specific initiative is approved by the Board of Directors, the review process is concluded, or it is otherwise determined that other disclosure is appropriate.

    Steven H. Rouhandeh, Chairman of the Board commented, "Our management team and employees have developed multiple programs that are innovative and transformative for patients.  We look to translate our assets and capabilities into value for stakeholders and are confident that the strategic review process will facilitate our efforts to achieve that goal."

    About Abeona Therapeutics
    Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Its preclinical assets include ABO-401, which uses a novel vector from Abeona's AIM™ AAV capsid platform to address all mutations of cystic fibrosis. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). For more information, visit www.abeonatherapeutics.com.

    Forward Looking Statement
    This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.  These statements include statements about the ability of its management team to lead the Company and deliver on key strategies, the market opportunities for the Company's products and product candidates, and the Company's goals and objectives.  We have attempted to identify forward looking statements by such terminology as "may," "will," "anticipate," "believe," "estimate," "expect," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other reports filed by the Company with the Securities and Exchange Commission.  The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

    Investor Contact:
    Sofia Warner
    Senior Director, Investor Relations
    Abeona Therapeutics
    +1 (646) 813-4710

    Media Contact:
    Scott Santiamo
    Director, Corporate Communications
    Abeona Therapeutics
    +1 (718) 344-5843

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