ABEO Abeona Therapeutics Inc.

2.08
+0.12  (+6%)
Previous Close 1.96
Open 2.2
52 Week Low 1.35
52 Week High 5.19
Market Cap $176,337,701
Shares 84,777,741
Float 70,388,044
Enterprise Value $77,690,701
Volume 3,292,381
Av. Daily Volume 814,785
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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 enrollment to be completed by end of 2020.
EB-101
Recessive dystrophic epidermolysis bullosa (RDEB)
Phase 3
Phase 3
Phase 3 enrollment to be completed early-2021.
ABO-202
CLN1 disease (Infantile Batten disease)
Phase 1/2
Phase 1/2
Phase 1/2 trial planned.
ABO-101
Sanfilippo syndrome type B (MPS IIIB)
Phase 1/2
Phase 1/2
Phase 1/2 presentation at WORLDSymposium February 12, 2020 noted trial has been well-tolerated to date, with no treatment-related severe adverse events.

Latest News

  1. 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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  2. 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

    Primary Logo

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  3. 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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  4. 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

    View Full Article Hide Full Article
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