chemicalbilly/E+ via Getty Images Abeona Therapeutics Inc. (NASDAQ:ABEO) dropped by more than half on Tuesday after the company received a Complete Response Letter from the FDA in response to the Company’s Biologics License Application for pz-cel. In one day, ABEO erased nearly a year's worth of gains that had been building on positive speculation that pz-cel would gain approval at next month's PDUFA. Those hopes were dashed as the FDA identified manufacturing issues that the company must take the next ...

Receives FDA Complete Response Letter (CRL) based on need for additional CMC information CRL did not identify deficiencies related to clinical efficacy or clinical safety data in BLA, and no new clinical studies requested by FDA to support approval Anticipates completing and submitting requested CMC information in 3Q 2024 Conference call and webcast on Tuesday, April 23, 2024 at 8:30 a.m. ET to provide details on the requested CMC information CLEVELAND, April 22, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutic ...

Exhibit 99.1 Abeona Therapeutics Reports Full Year 2023 Financial Results and Announces Completion of FDA Inspections - FDA completed Pre-License Inspection of Abeona’s Cleveland manufacturing facility, as well as inspection of clinical trial sites for pz-cel pivotal Phase 3 VIITAL™ study - CLEVELAND, March 18, 2024 – Abeona Therapeutics Inc. (Nasdaq: ABEO) today reported financial results for the full year of 2023 and provided an update on progress toward achieving key corporate objectives. Abeona also ann ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 Or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _______ to _______ Commission file number 001-15771 ABEONA THERAPEUTICS INC. (Exact name of registrant as specified in its charter) Delaware 83-0221517 (State or Oth ...

CLEVELAND, March 18, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO) today reported financial results for the full year of 2023 and provided an update on progress toward achieving key corporate objectives. Abeona also announced today that the U.S. Food and Drug Administration (FDA) has completed a Pre-License Inspection (PLI) of its Cleveland, Ohio manufacturing facility related to the Company's Biologics License Application (BLA) for pz-cel (prademagene zamikeracel) for recessive dystrophic ...

Group 1 - Abeona Therapeutics Inc. has granted equity awards to new non-executive employees, totaling up to 25,500 restricted shares of common stock, as a material inducement to employment [1] - The restricted stock awards will vest one-third yearly on each anniversary of the Grant Date, fully vesting on the third anniversary, contingent on continued employment [1] Group 2 - Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases [2] - The U.S. FDA has accepted and granted Priority Review for the Biologics License Application for pz-cel, with a target action date of May 25, 2024 [2] - The company has a fully integrated cGMP manufacturing facility capable of supporting commercial production of pz-cel upon FDA approval [2] - Abeona's development portfolio includes AAV-based gene therapies for ophthalmic diseases, utilizing next-generation AAV capsids to improve tropism profiles [2]

FDA Review Progress - The FDA has completed both the Bioresearch Monitoring (BIMO) inspection and the mid-cycle review meeting for Abeona Therapeutics' Biologics License Application (BLA) for pz-cel, targeting a PDUFA action date of May 25, 2024 [1][3] - The BIMO inspection, conducted from January 22 to January 24, 2024, at Abeona's headquarters, reviewed clinical study practices and did not result in any observations or FDA Form 483s [2] - The FDA confirmed no plans to convene an Advisory Committee for pz-cel and indicated that Risk Evaluation and Mitigation Strategies (REMS) are not anticipated at this time [3] Company Overview - Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, including recessive dystrophic epidermolysis bullosa (RDEB) [4] - The company's investigational therapy, pz-cel, is an autologous, COL7A1 gene-corrected epidermal sheet currently under FDA review with Priority Review status [4] - Abeona operates a fully integrated cell and gene therapy cGMP manufacturing facility, which supported the Phase 3 VIITAL™ trial and is capable of commercial production upon FDA approval [4] Development Pipeline - Abeona's development portfolio includes AAV-based gene therapies for ophthalmic diseases with high unmet medical needs [4] - The company is evaluating next-generation AAV capsids to improve tropism profiles for various devastating diseases [4]

CLEVELAND, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO) today announced that it has entered into a $50 million credit facility with the Avenue Venture Opportunities Fund, L.P. The credit agreement, which has a term of three and a half years, includes a first tranche of $20 million at closing, a second tranche of $10 million of committed capital, and an additional accordion option to upsize the credit facility by an additional $20 million upon satisfaction of certain terms and con ...

Financial Data and Key Metrics Changes - As of September 30, 2023, the company had cash, cash equivalents, restricted cash, and short-term investments totaling $54.1 million, an increase from $37.1 million as of June 30, 2023, primarily due to a $25 million gross proceeds from a registered direct offering in July 2023 [18] - Research and development expenses rose to $7.1 million for Q3 2023, compared to $5.5 million in Q3 2022, while general and administrative expenses increased to $4.2 million from $3.9 million in the same period [19] - The net loss attributable to common shareholders was $11.8 million for Q3 2023, or $0.48 loss per share, compared to a net loss of $6.4 million or $1 loss per share in Q3 2022 [19] Business Line Data and Key Metrics Changes - The company is focusing on the commercialization of pz-cel, an investigational therapy for recessive dystrophic epidermolysis bullosa (RDEB), with plans to supply up to 120 patient treatments in the first full year post-launch [10][12] - The company is onboarding 5 to 7 high-volume EB treatment centers to facilitate access to pz-cel upon approval [12] Market Data and Key Metrics Changes - The FDA's decision on the BLA review process for pz-cel is expected by late November 2023, with a potential approval in Q2 2024 if a priority review is granted [8][9] - The company anticipates a peak sales estimate of approximately $500 million over a five-year period, based on the assumption of treating around 500 patients annually [32] Company Strategy and Development Direction - The company aims to transition from a clinical-stage to a commercial-stage organization with the launch of pz-cel, leveraging the leadership team's previous experiences with autologous therapies [10][11] - The strategy includes engaging with payers to ensure broad access and favorable pricing for pz-cel, which is expected to provide significant value as a transformative therapy [15][16] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential impact of pz-cel on the RDEB community, highlighting strong clinical data and positive feedback from the medical community [20] - The company is preparing for a commercial launch while ensuring that its financial resources are sufficient to support operations into Q4 2024 [18] Other Important Information - The company has received rare pediatric disease designation from the FDA for pz-cel, which may allow it to obtain a priority review voucher valued at approximately $100 million upon approval [9] - The management team has been actively filling commercial roles to support the launch, including key account management and market access positions [17] Q&A Session Summary Question: Can you elaborate on patient identification efforts and involvement of prior trial participants? - Management indicated that they are identifying patients from previous studies and ongoing trials, which is encouraging for launch preparations [24][25] Question: How quickly can pz-cel be commercialized post-approval? - The estimated timeline for commercialization is about 1 to 2 months post-approval, focusing on establishing medical policies and treatment center readiness [29] Question: What assumptions underlie the peak sales estimate of $500 million? - The estimate is based on the ability to treat 500 patients annually over five years, with a pricing assumption in the seven-digit range [32] Question: Will the company reach out to community doctors for initial treatments? - The company plans to start with centers of excellence and may expand to community centers over time, ensuring education and awareness of pz-cel [35][36] Question: What is the potential for pz-cel to be used in younger patients? - Management is in discussions with the FDA regarding the potential to treat patients younger than six years old, based on ongoing studies [39][40] Question: Has the company started hiring for the sales team? - The company has successfully filled key commercial roles and does not anticipate a large sales team at launch due to existing interest from treatment centers [43][44] Question: What are the takeaways from Krystal Biotech's recent launch? - Management views the uptake of competing products positively, as it raises awareness and prepares the market for pz-cel [46][47]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 FORM 10-Q (Mark one) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-15771 ABEONA THERAPEUTICS INC. (Exact name of registrant as specified in its charter) Delaware 83-0221517 (State or other juri ...