ABEO Abeona Therapeutics Inc.

1.36
-0.02  -1%
Previous Close 1.38
Open 1.39
52 Week Low 0.9895
52 Week High 3.7899
Market Cap $134,692,949
Shares 99,038,933
Float 84,967,082
Enterprise Value $50,370,727
Volume 875,136
Av. Daily Volume 1,766,248
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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-101
Sanfilippo syndrome type B (MPS IIIB)
Phase 1/2
Phase 1/2
Phase 1/2 updated data announced February 12, 2021.
EB-101 - VITAL
Recessive dystrophic epidermolysis bullosa (RDEB)
Phase 3
Phase 3
Phase 3 enrollment to be completed in 2021.
ABO-202
CLN1 disease (Infantile Batten disease)
Phase 1/2
Phase 1/2
Phase 1/2 trial planned.

Latest News

  1. Patient enrollment ongoing for EB-101 pivotal Phase 3 VIITAL™ study

    Positive interim MPS III data presented at 17th Annual WORLDSymposium; additional neurocognitive assessments of patients treated in high dose cohort 3 in MPS IIIA Transpher A study and additional clinical data from MPS IIIB Transpher B study expected in 2021

    Strengthened management and board to support the company's focus on driving future growth, enhancing corporate governance, and creating additional shareholder value

    Conference call scheduled for Tuesday, May 25, 2021 at 8:30 a.m. ET

    NEW YORK and CLEVELAND, May 18, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced financial results for…

    Patient enrollment ongoing for EB-101 pivotal Phase 3 VIITAL™ study

    Positive interim MPS III data presented at 17th Annual WORLDSymposium; additional neurocognitive assessments of patients treated in high dose cohort 3 in MPS IIIA Transpher A study and additional clinical data from MPS IIIB Transpher B study expected in 2021

    Strengthened management and board to support the company's focus on driving future growth, enhancing corporate governance, and creating additional shareholder value

    Conference call scheduled for Tuesday, May 25, 2021 at 8:30 a.m. ET

    NEW YORK and CLEVELAND, May 18, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced financial results for the first quarter 2021 and recent business progress.

    "We are off to a fast start in 2021, reflecting our intense focus on execution," said Michael Amoroso, Chief Executive Officer of Abeona. "We are focused on completing enrollment in the EB-101 Phase 3 pivotal VIITAL™ study, gaining clarity on a regulatory path for ABO-102 in MPS IIIA, producing the first lot of Abeona-produced clinical grade product for ABO-102, and reporting additional neurocognitive and biomarker data from both the ABO-102 Transpher A and the ABO-101 Transpher B studies. We also have a robust preclinical pipeline, and we are conducting research assessing AAV capsids with the aim of IND-enabling studies in two to three eye indications. Importantly, our focus on building the right talent and experience on our leadership team positions us well to continue to advance our clinical programs toward delivering meaningful milestones later this year."

    First Quarter and Recent Highlights

    Corporate Updates

    • Appointed Michael Amoroso as President, Chief Executive Officer (CEO) and a member of the company's Board of Directors.
    • Abeona strengthened its Board of Directors with the appointment of four new independent members who bring relevant operational leadership experience with life sciences companies, including in the areas of clinical development, manufacturing of cell therapy and gene therapy products, and corporate and financial compliance, to support the company's focus on driving future growth and creating additional shareholder value.

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

    • Patient enrollment is ongoing for the EB-101 pivotal Phase 3 VIITAL™ study for RDEB. The company continues to expect to complete enrollment in the VIITAL™ study in 2021, depending upon the impact from the COVID-19 pandemic, including travel restrictions and safety concerns.
    • To support ongoing enrollment and commercial preparation, Abeona continues to work toward adding a second clinical site in the VIITAL™ study by the third quarter of 2021.
    • Presented data on long-term patient-reported outcomes following EB-101 treatment of RDEB wounds at the Society for Investigative Dermatology (SID) Virtual Meeting 2021, held from May 3-8, 2021. The results showed durable wound healing and reduction in pain through 6 years after treatment.

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

    • Presented new positive data from two ongoing Phase 1/2 clinical trials of ABO-102 in MPS IIIA and ABO-101 in MPS IIIB in late-breaking platform oral presentations at the 17th Annual WORLDSymposium™ in February 2021.
    • The FDA granted Abeona's request and scheduled a Type B meeting in June 2021 to discuss the data-to-date from the ABO-102 Transpher A study and the potential path to a Biologics License Application (BLA) submission for ABO-102 in MPS IIIA.

    Preclinical Pipeline

    • Presented new data supporting the potential of Cre-mediated dual AAV vector technology to enable delivery of large genes targeted for treatment of Stargardt disease during an oral presentation at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting, held virtually from May 1-7, 2021.
    • Abeona recently completed non-human primate (NHP) studies comparing several capsids with AAV8, the industry standard for intraocular administration, in order to further understand and characterize the company's AAV capsids. 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 AAVV214D5 capsids were tested versus AAV8 administered subretinally. Both capsids demonstrated nearly identical levels of transduction of photoreceptor and retinal pigmented epithelium (RPE) cells, which are the cell types most frequently affected in inherited retinal diseases, when compared with AAV8.
    • The results from the recently completed NHP studies support Abeona's strategy to advance multiple preclinical eye programs into the clinic.

    First Quarter Financial Results

    Cash, cash equivalents and short-term investments totaled $86.8 million as of March 31, 2021, compared to $95.0 million as of December 31, 2020. Net cash used in operating activities was $13.6 million for the first quarter of 2021.

    Research and development (R&D) expenses were $7.2 million for the first quarter of 2021, compared to $6.8 million in the comparable period in 2020. The increase in R&D expenses was primarily due to increased clinical and development work for the company's gene and cell therapy product candidates, and increased salary and related costs. General and administrative (G&A) expenses were $6.6 million for the first quarter of 2021, compared to $6.4 million in the same period in 2020. The increase in G&A expenses was primarily due to increased professional fees, partially offset by decreased salary and related costs, and decreases in net other G&A expenses.

    Net loss was $16.0 million for the first quarter of 2021, compared to net loss of $48.2 million for the comparable period in 2020. The decrease in net loss was primarily due to the non-cash impairment charge of $32.9 million related to the termination of the license agreement with REGENXBIO in the first quarter of 2020.

    Conference Call Details

    Abeona Therapeutics will host a conference call and webcast on Tuesday, May 25, 2021 at 8:30 a.m. ET, to discuss its first 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: 552097 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona's website at www.abeonatherapeutics.com. The archived webcast replay will be available for 30 days following the call.

    About Abeona Therapeutics

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

    Forward-Looking Statements

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



    Abeona Therapeutics Inc. and Subsidiaries

    Condensed Consolidated Statements of Operations and Comprehensive Loss

    (unaudited)
     
      For the three months ended March 31,
       2021   2020 
         
    Revenues $-  $- 
         
    Expenses:    
    Research and development  7,212,000   6,818,000 
    General and administrative  6,568,000   6,412,000 
    Depreciation and amortization  817,000   2,065,000 
    Licensed technology impairment charge  -   32,916,000 
    Total expenses  14,597,000   48,211,000 
         
    Loss from operations  (14,597,000)  (48,211,000)
         
    Interest and miscellaneous income  15,000   652,000 
    Interest expense  (1,420,000)  (600,000)
    Net loss $(16,002,000) $(48,159,000)
         
    Basic and diluted loss per common share $(0.17) $(0.52)
         
    Weighted average number of common    
    shares outstanding – basic and diluted  94,234,653   92,362,505 
         
    Other comprehensive income:    
    Change in unrealized gains related to available-for-sale debt securities  13,000   386,000 
    Comprehensive loss $(15,989,000) $(47,773,000)
         



    Abeona Therapeutics Inc. and Subsidiaries

    Condensed Consolidated Balance Sheets

    (unaudited)
     
    ASSETSMarch 31,

    2021
     December 31,

    2020
    Current assets:   
    Cash and cash equivalents$14,265,000  $12,596,000 
    Short-term investments 72,506,000   82,438,000 
    Prepaid expenses and other current assets 1,826,000   2,708,000 
    Total current assets 88,597,000   97,742,000 
        
    Property and equipment, net 10,978,000   11,322,000 
    Right-of-use lease assets 6,764,000   7,032,000 
    Licensed technology, net 1,471,000   1,500,000 
    Goodwill 32,466,000   32,466,000 
    Other assets and restricted cash 1,156,000   1,136,000 
    Total assets$141,432,000  $151,198,000 
        
    LIABILITIES AND STOCKHOLDERS' EQUITY   
    Current liabilities:   
    Accounts payable$2,476,000  $4,695,000 
    Accrued expenses 2,868,000   3,410,000 
    Current portion of lease liability 1,716,000   1,713,000 
    Current portion of loan payable 549,000   330,000 
    Payable to licensor 32,934,000   31,515,000 
    Deferred revenue 296,000   296,000 
    Total current liabilities 40,839,000   41,959,000 
        
    Loan payable 1,209,000   1,428,000 
    Long-term lease liabilities 4,994,000   5,260,000 
    Total liabilities 47,042,000   48,647,000 
        
    Commitments and contingencies -   - 
    Stockholders' equity:   
    Common stock - $0.01 par value; authorized 200,000,000 shares;   
    issued and outstanding 99,038,933 at March 31, 2021;   
    issued and outstanding 96,131,678 at December 31, 2020 990,000   961,000 
    Additional paid-in capital 680,103,000   672,304,000 
    Accumulated deficit (586,706,000)  (570,704,000)
    Accumulated other comprehensive income/(loss) 3,000   (10,000)
    Total stockholders' equity 94,390,000   102,551,000 
    Total liabilities and stockholders' equity$141,432,000  $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. NEW YORK and CLEVELAND, April 20, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that new data from its preclinical research will be highlighted during an oral presentation at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting, being held virtually from May 1-7, 2021.

    "Autosomal recessive Stargardt disease is caused by mutations in the ABCA4 gene, preventing removal of toxic substances from photoreceptor cells that result in photoreceptor death and progressive vision loss," said Linas Padegimas, Ph.D., Senior Director, Product Development at Abeona. "The preclinical data being presented at ARVO's Annual Meeting provides compelling…

    NEW YORK and CLEVELAND, April 20, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced that new data from its preclinical research will be highlighted during an oral presentation at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting, being held virtually from May 1-7, 2021.

    "Autosomal recessive Stargardt disease is caused by mutations in the ABCA4 gene, preventing removal of toxic substances from photoreceptor cells that result in photoreceptor death and progressive vision loss," said Linas Padegimas, Ph.D., Senior Director, Product Development at Abeona. "The preclinical data being presented at ARVO's Annual Meeting provides compelling evidence that two independent AAV vectors utilizing Cre recombinase can efficiently reconstitute the ABCA4 gene, leading to full-size ABCA4 protein expression. Future studies that explore Cre-mediated ABCA4 gene reconstitution and lipofuscin clearance in vivo could provide additional findings to inform clinical development plans for a dual AAV vector approach as a potential treatment for Stargardt disease. In addition, our Cre-mediated dual AAV vector technology holds potential benefit for other indications that require delivery of larger genes that exceed the encapsidation capacity of AAV vectors."

    Details of the oral presentation are as follows:

    Title: Dual AAV Vector Strategy for Expression of Large Genes Targeted for Stargardt Disease Gene Therapy Development

    Presenter: Dr. Brianna Barrett, Abeona Therapeutics Inc.

    Session Title: Gene therapy in ocular diseases

    Session Date and Time: May 4, 2021 from 2:15 PM to 3:45 PM EDT

    About Abeona Therapeutics

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

    Forward-Looking Statements

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



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

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  3. NEW YORK and CLEVELAND, April 19, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced the appointment of Dr. Leila Alland and Mr. Donald Wuchterl as new independent members to its Board of Directors.

    "Leila and Donald bring a collective wealth of diverse biologics experience to Abeona that spans clinical development and manufacturing of cell therapy and gene therapy products, as we continue to focus on the mission of bringing our novel gene and cell therapies to patients who currently have no approved treatment options," said Steven H. Rouhandeh, Chairman of Abeona's Board of Directors. "Their relevant expertise and backgrounds complement those of our current…

    NEW YORK and CLEVELAND, April 19, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today announced the appointment of Dr. Leila Alland and Mr. Donald Wuchterl as new independent members to its Board of Directors.

    "Leila and Donald bring a collective wealth of diverse biologics experience to Abeona that spans clinical development and manufacturing of cell therapy and gene therapy products, as we continue to focus on the mission of bringing our novel gene and cell therapies to patients who currently have no approved treatment options," said Steven H. Rouhandeh, Chairman of Abeona's Board of Directors. "Their relevant expertise and backgrounds complement those of our current directors, and I look forward to relying on the fresh perspectives of our strengthened and expanded board to help guide Abeona's corporate strategy and operational execution going forward."

    Dr. Alland, a pediatric hematologist-oncologist and accomplished physician-scientist, has been working in the biopharmaceutical industry since 2001 to bring novel therapies to patients. Dr. Alland is currently Chief Medical Officer of PMV Pharmaceuticals, Inc., a precision oncology company pioneering the discovery and development of small molecule, tumor-agnostic therapies targeting p53 mutants. Previously, Dr. Alland served as Chief Medical Officer at Affimed, and prior to that she held the same position at Tarveda Therapeutics. Dr. Alland also held leadership positions at AstraZeneca, Bristol-Myers Squibb, Novartis, and Schering-Plough, where she worked on a broad range of oncology products from early to late-stage development and contributed to multiple successful drug approvals.

    Dr. Alland obtained her medical degree from New York University School of Medicine, and her B.A. in Biology from the University of Pennsylvania. She completed her residency in Pediatrics at The Children's Hospital of Philadelphia, and her fellowship in Pediatric Hematology/Oncology at The New York Hospital and Memorial Sloan-Kettering Cancer Center. Earlier in her career, Dr. Alland served as Assistant Professor of Pediatrics at Albert Einstein College of Medicine, where she was awarded the James S. McDonnell Foundation Scholar Award and pursued basic cancer research while also caring for children with cancer and blood disorders. Since 2020, Dr. Alland has served on the Board of Directors of Cytovia Therapeutics, Inc., an immuno-oncology company developing engineered cellular and antibody therapies to treat cancer. Dr. Alland is a member of the Scientific Advisory Council of Columbia University's Center for Radiological Research and serves as a scientific reviewer for the Cancer Prevention and Research Institute of Texas.

    Mr. Wuchterl brings over 29 years of experience in the life sciences industry, with senior roles in operations and Chemistry, Manufacturing, and Controls (CMC). He has significant experience building and leading current Good Manufacturing Practices (cGMP) manufacturing organizations and facilities. Mr. Wuchterl currently serves as Senior Vice President and Chief Manufacturing Officer at T-knife Therapeutics, a next-generation T-cell receptor company developing innovative therapeutics for the benefit of solid tumor patients and their families. Previously, he served as Senior Vice President, Technical Operations & Quality at Audentes Therapeutics (an Astellas Company), a gene therapy company focused on developing and commercializing innovative products for patients living with serious, life-threatening rare neuromuscular diseases. Prior to Audentes, Mr. Wuchterl served as Senior Vice President and Chief Operating Officer at Cytovance Biologics, and held positions of increasing responsibility with Dendreon, Shire HGT, Amgen, Biogen Idec and Roche. Mr. Wuchterl has a B.S. in Business Administration from Colorado Technical University and an M.B.A. from Fitchburg State University.

    About Abeona Therapeutics

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

    Forward-Looking Statements

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



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

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

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

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

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

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

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

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

    Mark Alvino, President at Hudson Square Capital LLC

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

    About Abeona Therapeutics

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

    Forward-Looking Statements

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



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

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

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

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

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

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

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

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

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

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

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

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

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

    Recent Highlights

    Corporate Developments

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

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

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

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

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

    Preclinical Pipeline

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

    Fourth Quarter and Full Year 2020 Financial Results

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

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

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

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

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

    Conference Call Details

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

    About Abeona Therapeutics

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

    Forward-Looking Statements

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

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



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

    2020
     December 31,

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

    200,000,000 shares; issued and outstanding

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

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



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

    Primary Logo

    View Full Article Hide Full Article
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