ABEO Abeona Therapeutics Inc.

1.21
-0.02  -2%
Previous Close 1.23
Open 1.24
52 Week Low 0.9895
52 Week High 3.7899
Market Cap $122,484,687
Shares 101,227,014
Float 87,155,163
Enterprise Value $54,137,957
Volume 519,698
Av. Daily Volume 1,505,227
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Upcoming Catalysts

Drug Stage Catalyst Date
ABO-101 (Transpher B)
Sanfilippo syndrome type B (MPS IIIB)
Phase 1/2
Phase 1/2
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EB-101 - VITAL
Recessive dystrophic epidermolysis bullosa (RDEB)
Phase 3
Phase 3
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Drug Pipeline

Drug Stage Notes
ABO-102
Sanfilippo syndrome type A (MPS IIIA)
Phase 1/2
Phase 1/2
Phase 1/2 updated safety data released February 12, 2021. Well tolerated.
ABO-202
CLN1 disease (Infantile Batten disease)
Phase 1/2
Phase 1/2
Phase 1/2 trial planned.

Latest News

  1. Second clinical trial site activated in EB-101 pivotal Phase 3 VIITAL™ study

    Successful Type B meeting with U.S. FDA; Transpher A study will serve as the pivotal study for ABO-102 in MPS IIIA; Alignment with FDA on primary study endpoint

    Focusing resources on completing EB-101 and ABO-102 pivotal, registration-enabling studies

    Conference call scheduled for Thursday, July 29, 2021 at 8:30 a.m. ET

    NEW YORK and CLEVELAND, July 28, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced financial results for the second quarter 2021 and recent business progress.

    "The second quarter was a highly effective and productive quarter for Abeona, and we remain committed to…

    Second clinical trial site activated in EB-101 pivotal Phase 3 VIITAL™ study

    Successful Type B meeting with U.S. FDA; Transpher A study will serve as the pivotal study for ABO-102 in MPS IIIA; Alignment with FDA on primary study endpoint

    Focusing resources on completing EB-101 and ABO-102 pivotal, registration-enabling studies

    Conference call scheduled for Thursday, July 29, 2021 at 8:30 a.m. ET

    NEW YORK and CLEVELAND, July 28, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced financial results for the second quarter 2021 and recent business progress.

    "The second quarter was a highly effective and productive quarter for Abeona, and we remain committed to delivering operational excellence and bringing our gene therapies to patients with no approved treatments," said Michael Amoroso, Chief Executive Officer of Abeona. "As we continue to advance our clinical programs, we are delivering on meaningful milestones. We are focusing our resources on completing registration-enabling studies for patients with RDEB and MPS IIIA, and are preparing for the potential of two BLA filings."

    Second Quarter and Recent Highlights

    Corporate Updates

    • Appointed Vishwas Seshadri, Ph.D., M.B.A., as Senior Vice President, Head of Research & Clinical Development. Dr. Seshadri joins Abeona from Celgene Corporation, now a subsidiary of Bristol-Myers Squibb Company, and brings substantial experience in the life sciences industry overseeing product development, regulatory submissions, and commercialization for novel therapies including personalized, autologous cell therapies.

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

    • Activated UMass Memorial Medical Center in Worcester, MA as the second clinical trial site in the pivotal Phase 3 VIITAL™ study of its investigational EB-101 treatment for recessive dystrophic epidermolysis bullosa (RDEB).
    • Presented updated Phase 1/2a clinical trial results at the Society for Pediatric Dermatology (SPD) 46th Annual Meeting, with EB-101 treatment of large, chronic RDEB wounds continuing to show a considerable reduction in both wound burden and associated long-term pain for up to six years.

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

    • Abeona completed a successful Type B meeting with the U.S. Food and Drug Administration (FDA) regarding the pivotal trial to support filing and approval for ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). Based on the Type B meeting, the ongoing Transpher A study will serve as the pivotal study for ABO-102 and could potentially support a Biologics License Application (BLA) submission depending on the data set. In addition, Abeona also aligned with the FDA on the definition of the primary endpoint for the study, neurocognitive assessment using the raw score from the Bayley Scales of Infant and Toddler Development (BSITD) and the Kauffman Assessment Battery for Children (KABC-2), which are already part of the assessment plan in the Transpher A protocol.
    • Presented new brain MRI data during an oral presentation at the 16th International Symposium on MPS and Related Diseases, held during July 23-25, 2021. The MRI data indicated that ABO-102 increased grey matter, corpus callosum, and amygdala volumes in the brain in the three young patients with MPS IIIA at 24 months as compared to afflicted patients without treatment. The MRI data is consistent with previously reported results of preservation of neurocognitive development in these three young patients in the Transpher A study.
    • The Company is in the process of closing enrollment for the Transpher B trial. To date, four patients have been treated in the higher dose cohort of Transpher B. An additional three patients have been treated and a fourth patient will be treated with the higher dose through the Named Patient Program (NPP) in Germany, a compassionate use program that allows for patients with high unmet need to be treated at the request of the treating physician. The patients treated in the NPP are followed for safety and efficacy with the same rigor and frequency as patients in Transpher B. The Company intends to pool all patients' data from Transpher B and the NPP to assess therapeutic effect going forward. The Company expects 2-year neurocognitive data for the first patients treated in the high dose cohort of Transpher B beginning in the first half of 2022.

    Preclinical Pipeline

    • Presented new data at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting supporting the potential of Cre-mediated dual AAV vector technology to enable delivery of large genes targeted for treatment of Stargardt disease.
    • Completed non-human primate (NHP) studies comparing several of the company's AAV capsids with AAV8, the industry standard for intraocular administration. The results showed that AAV204, part of Abeona's in-licensed AIM™ capsid library, was superior to AAV8 using a recently developed route of ocular administration. In a separate NHP experiment, the company's AAV214 and AAV214D5 capsids demonstrated nearly identical levels of transduction compared with AAV8 of photoreceptor and retinal pigmented epithelium cells, which are the cell types most frequently affected in inherited retinal diseases. The NHP results support Abeona's strategy to advance multiple preclinical eye programs onward toward the clinic.

    Second Quarter Financial Results

    Cash, cash equivalents and short-term investments totaled $77.6 million as of June 30, 2021, compared to $86.8 million as of March 31, 2021. Net cash used in operating activities was $11.5 million for the second quarter of 2021.

    "While pursuing our key strategic priorities, we have thoughtfully and carefully managed our spending decisions. Across the organization, there is a balanced approach to not only focusing on moving towards our key milestones, but also utilizing our cash resources prudently and on time to deliver results," said Edward Carr, Chief Accounting Officer of Abeona.

    Total research and development (R&D) spending was $7.4 million for the second quarter of 2021, which is consistent with the $7.2 million spent in the first quarter of 2021. R&D expenses include the cost of clinical development of the EB-101 and MPS programs, manufacturing of the drug products for EB-101 and ABO-102, and preclinical ophthalmic research activities. Total general and administrative (G&A) spending was $5.5 million in the second quarter of 2021, down from the $6.6 million spent in the first quarter of 2021. The decrease in G&A in the second quarter of 2021 is primarily due to lower professional fees. Net loss was $15.2 million for the second quarter of 2021.

    Conference Call Details

    Abeona Therapeutics will host a conference call and webcast on Thursday, July 29, 2021 at 8:30 a.m. ET, to discuss its second quarter 2021 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: 7184522 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 investigational autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel investigational 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 development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona's novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. Abeona's fully integrated gene and cell therapy cGMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and planned 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
    Condensed Consolidated Statements of Operations and Comprehensive Loss
    (unaudited)
     
     For the three months ended June 30, For the six months ended June 30,
      2021   2020   2021   2020 
            
    Revenues$-  $-  $-  $- 
            
    Expenses:       
    Research and development 7,434,000   6,109,000   14,646,000   12,927,000 
    General and administrative 5,457,000   5,538,000   12,025,000   11,950,000 
    Depreciation and amortization 824,000   834,000   1,641,000   2,899,000 
    Licensed technology impairment charge -   -   -   32,916,000 
    Total expenses 13,715,000   12,481,000   28,312,000   60,692,000 
            
    Loss from operations (13,715,000)  (12,481,000)  (28,312,000)  (60,692,000)
            
    Interest and miscellaneous income 8,000   271,000   23,000   923,000 
    Interest expense (1,500,000)  (800,000)  (2,920,000)  (1,400,000)
    Net loss$(15,207,000) $(13,010,000) $(31,209,000) $(61,169,000)
            
    Basic and diluted loss per common share$(0.16) $(0.14) $(0.33) $(0.66)
            
    Weighted average number of common       
    shares outstanding – basic and diluted 96,509,783   92,704,203   95,378,503   92,533,354 
            
    Other comprehensive (loss)/income:       
    Change in unrealized (losses)/gains related to available-for-sale debt securities (4,000)  (253,000)  9,000   133,000 
    Comprehensive loss$(15,211,000) $(13,263,000) $(31,200,000) $(61,036,000)





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

    2021
     December 31,

    2020
    Current assets:   
    Cash and cash equivalents$27,179,000  $12,596,000 
    Short-term investments 50,380,000   82,438,000 
    Prepaid expenses and other current assets 1,321,000   2,708,000 
    Total current assets 78,880,000   97,742,000 
        
    Property and equipment, net 10,240,000   11,322,000 
    Right-of-use lease assets 6,489,000   7,032,000 
    Licensed technology, net 1,442,000   1,500,000 
    Goodwill 32,466,000   32,466,000 
    Other assets and restricted cash 1,158,000   1,136,000 
    Total assets$130,675,000  $151,198,000 
        
    LIABILITIES AND STOCKHOLDERS' EQUITY   
    Current liabilities:   
    Accounts payable$1,469,000  $4,695,000 
    Accrued expenses 2,190,000   3,410,000 
    Current portion of lease liability 1,720,000   1,713,000 
    Current portion of loan payable 1,758,000   330,000 
    Payable to licensor 34,434,000   31,515,000 
    Contract liability 296,000   296,000 
    Total current liabilities 41,867,000   41,959,000 
        
    Loan payable -   1,428,000 
    Long-term lease liabilities 4,722,000   5,260,000 
    Total liabilities 46,589,000   48,647,000 
        
    Commitments and contingencies -   - 
    Stockholders' equity:   
    Common stock - $0.01 par value; authorized 200,000,000 shares;   
    issued and outstanding 101,251,023 at June 30, 2021;   
    issued and outstanding 96,131,678 at December 31, 2020 1,013,000   961,000 
    Additional paid-in capital 684,987,000   672,304,000 
    Accumulated deficit (601,913,000)  (570,704,000)
    Accumulated other comprehensive loss (1,000)  (10,000)
    Total stockholders' equity 84,086,000   102,551,000 
    Total liabilities and stockholders' equity$130,675,000  $151,198,000 



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

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  2. Transpher A; pivotal study for intended registration of ABO-102 for MPS IIIA disease

    Alignment with FDA on primary study endpoint

    NEW YORK and CLEVELAND, July 28, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that the Company completed a successful Type B meeting with the U.S. Food and Drug Administration (FDA) regarding the pivotal trial to support filing and approval for its AAV-based gene therapy ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). ABO-102 is currently being evaluated in the single-arm Transpher A study in patients with MPS IIIA.

    Based on the Type B meeting with the FDA, the ongoing Transpher A study will…

    Transpher A; pivotal study for intended registration of ABO-102 for MPS IIIA disease

    Alignment with FDA on primary study endpoint

    NEW YORK and CLEVELAND, July 28, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that the Company completed a successful Type B meeting with the U.S. Food and Drug Administration (FDA) regarding the pivotal trial to support filing and approval for its AAV-based gene therapy ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). ABO-102 is currently being evaluated in the single-arm Transpher A study in patients with MPS IIIA.

    Based on the Type B meeting with the FDA, the ongoing Transpher A study will serve as the pivotal study for ABO-102 and could potentially support a Biologics License Application (BLA) submission depending on the data set. In addition, Abeona also aligned with the FDA on the definition of the primary endpoint for the study, neurocognitive assessment using the raw score from the Bayley Scales of Infant and Toddler Development (BSITD) and the Kauffman Assessment Battery for Children (KABC-2), which are already part of the assessment plan in the Transpher A protocol. Abeona intends to work closely with the FDA through the regenerative medicine advanced therapy (RMAT) mechanism to assemble the most robust pivotal data package possible for the registration of ABO-102.

    "We are grateful to the FDA for their guidance and collaborative exchange regarding the pivotal trial to support bringing ABO-102 to MPS IIIA patients who currently have no approved treatment," said Michael Amoroso, Chief Executive Officer of Abeona. "From 2016 to-date we have treated 21 patients in the Transpher A trial. We are excited about the safety and magnitude of benefit seen with our investigational ABO-102 therapy in the younger children from the higher dose cohort reported earlier this year. We remain hopeful that if the more recently dosed children in cohort 3 display a similar treatment effect, we could have an evaluable data set in 2022. The patients we serve have tremendous unmet need and we remain fully focused on operational excellence with the intent of now delivering potentially two pivotal data packages—one for ABO-102 and one for EB-101—in 2022."

    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 investigational autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel investigational 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 development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona's novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. Abeona's fully integrated gene and cell therapy cGMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and planned 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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  3. NEW YORK and CLEVELAND, July 26, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene therapy, today announced magnetic resonance imaging (MRI) data from the Phase 1/2 Transpher A clinical study indicating that ABO-102 increased grey matter, corpus callosum and amygdala volumes in the brain in three young patients with Sanfilippo Syndrome Type A (MPS IIIA) at 24 months as compared to afflicted patients without treatment. The new data was presented during an oral presentation at the 16th International Symposium on MPS and Related Diseases.

    "Brain volume loss is characteristic in children with MPS IIIA and is associated with long-term cognitive and physical disability. Specifically, grey matter…

    NEW YORK and CLEVELAND, July 26, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene therapy, today announced magnetic resonance imaging (MRI) data from the Phase 1/2 Transpher A clinical study indicating that ABO-102 increased grey matter, corpus callosum and amygdala volumes in the brain in three young patients with Sanfilippo Syndrome Type A (MPS IIIA) at 24 months as compared to afflicted patients without treatment. The new data was presented during an oral presentation at the 16th International Symposium on MPS and Related Diseases.

    "Brain volume loss is characteristic in children with MPS IIIA and is associated with long-term cognitive and physical disability. Specifically, grey matter is important for cognitive development, corpus callosum for motor function, and amygdala for fear learning as well as social/emotional development," said Vishwas Seshadri, Ph.D., M.B.A., Head of Research & Clinical Development of Abeona. "The new MRI data shows the potential of ABO-102 to increase brain grey matter, corpus callosum and amygdala volumes and is consistent with previously reported results of preservation of neurocognitive development in these three young patients in the Transpher A study."

    The Transpher A study primary endpoints are neurodevelopment and safety. Secondary endpoints include brain volume, behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and liver volume.

    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 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 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. Abeona's clinical programs include EB-101, its investigational autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel investigational 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 development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona's novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. Abeona's fully integrated gene and cell therapy cGMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and planned 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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  4. NEW YORK and CLEVELAND, July 23, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced the activation of a second clinical trial site in its pivotal Phase 3 VIITAL™ study of its investigational EB-101 treatment for recessive dystrophic epidermolysis bullosa (RDEB) at UMass Memorial Medical Center in Worcester, MA.

    The EB-101 pivotal VIITAL™ study is currently ongoing at Stanford University Medical Center in Palo Alto, CA. Target enrollment is 10 to 15 RDEB patients with approximately 35 large, chronic wound sites to be treated in total. Treatment with EB-101 uses gene transfer to deliver a functional COL7A1 gene into a patient's own skin cells (keratinocytes and…

    NEW YORK and CLEVELAND, July 23, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced the activation of a second clinical trial site in its pivotal Phase 3 VIITAL™ study of its investigational EB-101 treatment for recessive dystrophic epidermolysis bullosa (RDEB) at UMass Memorial Medical Center in Worcester, MA.

    The EB-101 pivotal VIITAL™ study is currently ongoing at Stanford University Medical Center in Palo Alto, CA. Target enrollment is 10 to 15 RDEB patients with approximately 35 large, chronic wound sites to be treated in total. Treatment with EB-101 uses gene transfer to deliver a functional COL7A1 gene into a patient's own skin cells (keratinocytes and its progenitors) and transplanting those cells back to the patient. EB-101 is believed to facilitate wound healing by supplementing Type VII collagen expression in RDEB patients who lack a fully functional COL7A1 gene. 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).

    "We are very pleased to have UMass Memorial Medical Center as one of our VIITAL™ study sites and look forward to collaborating with Dr. Karen Wiss, Director of Pediatric Dermatology at UMass Memorial to screen and enroll subjects as soon as possible," said Vishwas Seshadri, Ph.D., M.B.A., Head of Research & Clinical Development of Abeona. "With UMass and Stanford, we are able to provide convenient treatment locations on the East Coast and West Coast to make travel and logistics easier for patients and families, while also expanding physician experience with EB-101 as we plan for potential commercial launch."

    "We are excited to participate in the EB-101 VIITAL™ study and to offer this potentially promising investigational therapy to RDEB patients who currently have no adequate treatment options. We look forward to participating in the Phase 3 trial to evaluate the efficacy and safety of this investigational treatment," said Karen Wiss, M.D., FAAD, Professor of Dermatology and Pediatrics at UMass Medical School and Principal Investigator of the study at UMass Memorial Medical Center.

    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 Abeona's 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 35 large, chronic wound sites treated in total. Treatment with EB-101 involves using gene transfer to deliver the COL7A1 gene into a patient's own skin cells (keratinocytes and its progenitors) and transplanting those cells back to the patient. EB-101 is believed to enable normal Type VII collagen expression and facilitate wound healing. Abeona produces EB-101 for the VIITAL™ study at its fully integrated gene and cell therapy manufacturing facility in Cleveland, OH.

    In a Phase 1/2a clinical trial of EB-101, participants with RDEB were ≥18 years old, had two COL7A1 genetic mutations and chronic open wounds ≥ 20 cm2, for ≥ 12 weeks. Autologous keratinocytes were cultured from biopsies of intact skin and transduced with a retrovirus containing full-length COL7A1 to form gene-corrected epidermal sheets (EB-101) measuring 35 cm2. EB-101 was transplanted onto 38 chronic wound sites in 7 participants from 2013 to 2017. Investigator assessment of wound healing and pain assessment from last available visit was recorded, followed by a survey 3 to 6 years after treatment asking participants to rate change in pain compared with their pre-treatment state using a seven-point scale, ranging from 1 (very much improved) to 7 (very much worse). EB-101 is an investigational product not yet approved by the FDA.

    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 investigational autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel investigational 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 development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona's novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. Abeona's fully integrated gene and cell therapy cGMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and planned 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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  5. NEW YORK and CLEVELAND, July 07, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced updated Phase 1/2a clinical trial results up to six years following treatment with Abeona's investigational EB-101 for recessive dystrophic epidermolysis bullosa (RDEB). The data showed that investigator assessment of wound healing of 50% or greater from baseline was present in 69% of treated wounds (n=18/26) at 3 years, 93% (n=14/15) at 4 years, 80% (n=12/15) at 5 years, and 80% (n=4/5) at 6 years. The results were reported in a poster, titled "Long-Term Healing, Pain Reduction, and Patient-Reported Outcomes in Recessive Dystrophic Epidermolysis Bullosa Following EB-101 Treatment…

    NEW YORK and CLEVELAND, July 07, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced updated Phase 1/2a clinical trial results up to six years following treatment with Abeona's investigational EB-101 for recessive dystrophic epidermolysis bullosa (RDEB). The data showed that investigator assessment of wound healing of 50% or greater from baseline was present in 69% of treated wounds (n=18/26) at 3 years, 93% (n=14/15) at 4 years, 80% (n=12/15) at 5 years, and 80% (n=4/5) at 6 years. The results were reported in a poster, titled "Long-Term Healing, Pain Reduction, and Patient-Reported Outcomes in Recessive Dystrophic Epidermolysis Bullosa Following EB-101 Treatment of Large, Chronic Wounds," at the Society for Pediatric Dermatology (SPD) 46th Annual Meeting.

    The updated results also included investigator assessment of the presence of pain in treated wounds up to six years following treatment with EB-101. The data showed that pain was absent in all treated wounds that were evaluated at 3 years (n=26), 4 years (n=15), 5 years (n=15) and 6 years (n=5) of follow up, compared with presence of pain at baseline in 53% (n=20/38) of treated wounds. A separate survey of patient-reported pain at 3 years to 6 years following treatment with EB-101 was conducted and asked participants to rate change in pain compared with their pre-treatment state using a seven-point scale, ranging from 1 (very much improved) to 7 (very much worse). The survey data showed that 76% of the treated wounds with healing of 50% or greater were associated with improved pain scores, with 53% associated with "much/very much improved" pain scores.

    "RDEB is a debilitating and life-threatening rare genetic disorder with high rates of morbidity and mortality, without an approved treatment option," said Vishwas Seshadri, Ph.D., M.B.A., Head of Research & Clinical Development of Abeona. "It is important that potential new treatments can durably address large, chronic wounds, which are the most severe wounds that cause substantial pain in patients with RDEB. The updated Phase 1/2a results showed safety and durable efficacy follow up, with EB-101 treated wounds continuing to show a considerable reduction in both wound burden and associated long-term pain for up to six years. We are excited about the data and look forward to further investigating EB-101's potential to provide durable benefit in our ongoing pivotal Phase 3 VIITAL™ study."

    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 Abeona's 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 35 large, chronic wound sites treated in total. Treatment with EB-101 involves using gene transfer to deliver the COL7A1 gene into a patient's own skin cells (keratinocytes and its progenitors) and transplanting those cells back to the patient. EB-101 is believed to enable normal Type VII collagen expression and facilitate wound healing. Abeona produces EB-101 for the VIITAL™ study at its fully integrated gene and cell therapy manufacturing facility in Cleveland, OH.

    In a Phase 1/2a clinical trial of EB-101, participants with RDEB were ≥18 years old, had two COL7A1 genetic mutations and chronic open wounds ≥ 20 cm2, for ≥ 12 weeks. Autologous keratinocytes were cultured from biopsies of intact skin and transduced with a retrovirus containing full-length COL7A1 to form gene-corrected epidermal sheets (EB-101) measuring 35 cm2. EB-101 was transplanted onto 38 chronic wound sites in 7 participants from 2013 to 2017. Investigator assessment of wound healing and pain assessment from last available visit was recorded, followed by a survey 3 to 6 years after treatment asking participants to rate change in pain compared with their pre-treatment state using a seven-point scale, ranging from 1 (very much improved) to 7 (very much worse). EB-101 is an investigational product not yet approved by the FDA.

    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 investigational autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel investigational 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 development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona's novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. Abeona's fully integrated gene and cell therapy cGMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and planned 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
    

    Primary Logo

    View Full Article Hide Full Article
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