Financial Data and Key Metrics Changes - As of June 30, 2023, the company had cash, cash equivalents, restricted cash, and short-term investments of $37.1 million, down from $40.7 million as of March 31, 2023 [9] - License and other revenues for Q2 2023 were $3.5 million, compared to $1 million in Q2 2022, driven by a clinical milestone achieved under a sub-agreement [10] - The net loss attributable to common shareholders was $16.7 million for Q2 2023, or $0.92 loss per share, compared to a net loss of $7.9 million, or $1.36 loss per share, in Q2 2022 [10] Business Line Data and Key Metrics Changes - Significant progress was made in the EB-101 program, with completed process performance qualification (PPQ) manufacturing runs for both retroviral vector and EB-101 drug product [5] - Additional efficacy and safety data from the EB-101 pivotal Phase III VIITAL study showed improved wound healing and pain reduction at six, 12, and 24 weeks compared to control wounds [6] Market Data and Key Metrics Changes - The company anticipates a priority review based on the expected timing of the BLA submission, with potential approval in Q2 2024 [5] - The company is engaging with top EB treatment centers in the U.S. and has received positive feedback regarding the high unmet need for EB-101 [6][19] Company Strategy and Development Direction - The company plans to submit the EB-101 BLA in Q3 2023, with preparations for commercialization already underway [5][6] - The company is focusing on building a sales force and engaging with payer groups to ensure broad market access post-approval [19] - The company is also preparing for potential expansion into international markets, with interest from pharmaceutical companies in Europe and Japan [25] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the progress made with the FDA regarding the BLA submission and the comparability of retroviral vectors [14][15] - The management highlighted the importance of patient advocacy groups in raising awareness and facilitating self-referrals as the launch approaches [22] - The company is optimistic about the market opportunity, citing the potential for increased diagnosis and awareness of RDEB patients [24] Other Important Information - The company raised $25 million in a registered direct offering in July 2023 to fund pre-commercialization activities [6] - The company has mapped out key commercial and medical roles to be filled in the second half of the year to support a successful launch [19] Q&A Session Summary Question: Feedback on the briefing package and pre-BLA meeting agenda - Management indicated that they have had collaborative interactions with the FDA and are confident in the data submitted for RVV comparability [14][15] Question: Commercial launch rollout and CMC scale-up - Management confirmed that they are preparing for the commercial launch and will be careful about capacity expansion until after approval [16][17] Question: Awareness in the RDEB community - Management expects increased awareness and self-referrals as the launch approaches, aided by patient advocacy groups [22] Question: Market size and patient access - Management noted that it is early to speculate on market size based on competitor data but remains encouraged by the initial feedback [28] Question: CMC activities prior to BLA filing - Management confirmed that they have completed the necessary CMC activities and are on track for a Q3 submission [31]

Financial Performance - License and other revenues for the three months ended June 30, 2023, were $3,500 thousand, compared to $1,346 thousand for the same period in 2022, indicating a year-over-year increase of approximately 160.5%[11] - Net loss for the three months ended June 30, 2023, was $16,654 thousand, compared to a net loss of $26,141 thousand for the same period in 2022, showing an improvement of approximately 36.2%[11] - Basic and diluted loss per common share for the three months ended June 30, 2023, was $(0.92), compared to $(5.16) for the same period in 2022, indicating a significant reduction in loss per share[11] - For the six months ended June 30, 2023, the net loss was $25.76 million, compared to a net loss of $26.14 million for the same period in 2022, representing a decrease of 1.5%[18] - The net loss for the six months ended June 30, 2022, was $26,141,000 after adjustments, compared to a net loss of $29,086,000 as reported[35] Assets and Liabilities - Total current assets decreased from $53,099 thousand as of December 31, 2022, to $44,038 thousand as of June 30, 2023, representing a decline of approximately 17.1%[9] - Total liabilities increased from $37,453 thousand as of December 31, 2022, to $44,205 thousand as of June 30, 2023, reflecting an increase of approximately 18.1%[9] - Cash and cash equivalents decreased from $14,217 thousand as of December 31, 2022, to $6,225 thousand as of June 30, 2023, a decline of approximately 56.1%[9] - Total stockholders' equity decreased from $26,761 thousand as of December 31, 2022, to $9,345 thousand as of June 30, 2023, representing a decline of approximately 65.1%[9] - As of June 30, 2023, total stockholders' equity was $9,345,000, a decrease from $26,761,000 as of December 31, 2022[13] Cash Flow and Investments - Cash outflows from operating activities for the six months ended June 30, 2023, were $22.03 million, slightly improved from $22.70 million in the prior year[18] - The company reported net cash provided by investing activities of $7.42 million for the six months ended June 30, 2023, compared to a net cash used of $0.32 million in the same period of 2022[18] - As of June 30, 2023, the company had cash, cash equivalents, restricted cash, and short-term investments totaling $37.1 million[25] - The company recorded a net decrease in cash, cash equivalents, and restricted cash of $7.99 million for the six months ended June 30, 2023[18] Stock and Equity - The company issued 1,891,761 shares of common stock under an open market sale agreement (ATM) during the same period, raising approximately $6,367,000[13] - The balance of common shares increased to 21,478,157 as of June 30, 2023, from 17,719,720 as of December 31, 2022[13] - The company sold 1,990,321 shares under the ATM Agreement, resulting in net proceeds of $6.6 million during the six months ended June 30, 2023[73] - The company completed a private placement on November 3, 2022, raising $35.0 million gross, with net proceeds of $32.6 million after related costs[74] Research and Development - Research and development expenses for the six months ended June 30, 2023, totaled $16,564 thousand, a decrease of approximately 3.7% compared to $17,203 thousand for the same period in 2022[11] - The company is actively seeking a commercialization partner for its product EB-101, which is critical for future revenue generation[6] - The company’s lead clinical program is EB-101, currently in development for recessive dystrophic epidermolysis bullosa (RDEB)[21] - The company’s development portfolio includes AAV-based gene therapies for ophthalmic diseases using the AIM™ capsid platform[21] Compensation and Expenses - Stock-based compensation expense for the six months ended June 30, 2023, was $927,000, down from $1,697,000 for the same period in 2022[13] - Total stock-based compensation expense for the three months ended June 30, 2023, was $709,000, compared to $540,000 for the same period in 2022, representing a 31.3% increase[78] - For the six months ended June 30, 2023, total stock-based compensation expense was $1.697 million, up from $1.586 million in 2022, reflecting a 7.0% increase[78] Other Financial Metrics - The accumulated deficit as of June 30, 2023, was $(721,097,000), reflecting ongoing operational losses[13] - The company reported an interest income of $417 thousand for the three months ended June 30, 2023, compared to $38 thousand for the same period in 2022, indicating a substantial increase[11] - Other comprehensive loss for the three months ended June 30, 2023, was $(30,000)[13] - The company recorded a settlement liability of $30.0 million to REGENXBIO Inc., with $4.4 million due in November 2024[58][59]

Abeona Therapeutics Inc. (NASDAQ:ABEO) Q1 2023 Earnings Conference Call May 24, 2023 8:30 AM ET Company Participants Vishwas Seshadri - Chief Executive Officer Joseph Vazzano - Chief Financial Officer Dmitriy Grachev - Chief Medical Officer Brian Kevany - Chief Technical Officer, Head of Research Madhav Vasanthavada - Vice President, Business Development Greg Gin - Vice President, Investor Relations and Corporate Communications Conference Call Participants Maury Raycroft - Jefferies Kristen Kluska - Cantor ...

1Q’23 Portfolio Update Conference Call Note regarding forward-looking statements This presentation contains certain statements that may be 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, including statements relating to the product portfolio and pipeline and clinical programs of Abeona Therapeutics Inc. (the “Company”), the market opportunities for all of the Company’s products andproduct candida ...

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 March 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 other jurisdic ...

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, 2022 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 ...

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, 2022 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) (State or Other Jurisdiction of incorpor ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 Title of each class Trading Symbol(s) Name of each exchange on which registered Common Stock, $0.01 par value ABEO Nasdaq Capital Market 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 June 30, 2022 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 numbe ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 Title of each class Trading Symbol(s) Name of each exchange on which registered Common Stock, $0.01 par value ABEO Nasdaq Capital Markets 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 March 31, 2022 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 num ...

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, 2021 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) | --- | --- | --- | |------------ ...