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 ...

Financial Performance - Total current assets decreased from $56,292 thousand as of December 31, 2021, to $39,204 thousand as of March 31, 2022, representing a decline of approximately 30.4%[8] - Cash and cash equivalents decreased from $32,938 thousand to $20,326 thousand, a reduction of about 38.2%[8] - Total liabilities decreased from $28,211 thousand to $23,729 thousand, a decline of approximately 16.0%[8] - The net loss for Q1 2022 was $20,791 thousand, compared to a net loss of $16,002 thousand in Q1 2021, reflecting an increase in loss of about 29.0%[10] - Net loss for the three months ended March 31, 2022, was $20,791 thousand, compared to a net loss of $16,002 thousand for the same period in 2021, representing a 30% increase in net loss year-over-year[12] - Cash outflows from operating activities for the three months ended March 31, 2022, were $13,687 thousand, slightly higher than $13,585 thousand for the same period in 2021[12] Expenses - Research and development expenses increased from $8,317 thousand in Q1 2021 to $10,545 thousand in Q1 2022, an increase of about 27.0%[10] - General and administrative expenses decreased from $6,280 thousand in Q1 2021 to $4,224 thousand in Q1 2022, a reduction of approximately 32.7%[10] - The company incurred impairment charges totaling $6,168 thousand in Q1 2022, including $1,355 thousand for licensed technology and $3,252 thousand for construction-in-progress[10] - The company recorded an impairment charge of $3.3 million for construction-in-progress related to the ABO-101 and ABO-102 programs for the three months ended March 31, 2022[29] - The company recorded an impairment charge of $1.6 million related to leases for the ABO-101 and ABO-102 programs for the three months ended March 31, 2022[38] - Stock-based compensation expense for the three months ended March 31, 2022, was $862,000, a decrease from $1.950 million in the same period of 2021[42] Equity and Financing - Total stockholders' equity decreased from $51,375 thousand as of December 31, 2021, to $31,443 thousand as of March 31, 2022, a decline of approximately 38.8%[11] - The company has not generated significant product revenues and has not achieved profitable operations, indicating a need for additional financing to support ongoing development activities[16] - The company has sufficient resources to fund operations for at least the next 12 months but will need to secure additional funding beyond that period[19] - On April 29, 2022, the Company agreed to issue and sell 1,000,006 shares of Series A Preferred Stock and 250,005 shares of Series B Preferred Stock at an offering price of $19.00 per share, resulting in gross proceeds of approximately $25.0 million[49] - The Company plans to conduct a reverse stock split of the outstanding shares of Common Stock, with the ratio to be determined by the Board of Directors[50] Assets and Investments - As of March 31, 2022, the company had cash, cash equivalents, restricted cash, and short-term investments totaling $37.2 million[16] - As of March 31, 2022, the company had short-term investments in U.S. treasury securities valued at $10,989 thousand[25] - Total assets measured at fair value as of March 31, 2022, amounted to $42.060 million, with $27.683 million classified under recurring assets[35] Stock Options and Awards - The company reported a total of 53,750,137 potential dilutive securities that could impact future net loss per share calculations[24] - As of March 31, 2022, the total compensation cost related to non-vested option awards not yet recognized is approximately $5.0 million with a weighted average remaining vesting period of 2.6 years[46] - The outstanding restricted stock awards decreased from 2,431,515 to 1,948,334 from December 31, 2021, to March 31, 2022, with a total unrecognized compensation expense of approximately $2.8 million expected to be recognized over a weighted average vesting period of 2.7 years[48] - The number of exercisable options as of March 31, 2022, is 3,516,716, with a weighted average exercise price of $1.51[46] - The Company granted 252,000 restricted stock awards during the three months ended March 31, 2022[48] - The aggregate intrinsic value of options is calculated based on the difference between the exercise price and the fair value of the Company's common stock[46] Settlement and Liabilities - The company has a settlement liability of $30 million with REGENXBIO, with $20 million already paid and $10 million due in the next two years[31] - As of March 31, 2022, the current portion of the payable to REGENXBIO is $4.7 million, and the long-term portion is $3.9 million[32] - Operating lease liabilities as of March 31, 2022, have a present value of $9.095 million, with total undiscounted payments of $11.506 million[40] - The company reported accrued expenses of $4.206 million as of March 31, 2022, down from $5.585 million in December 2021[36]

Clinical Programs - Abeona's lead clinical program, EB-101, is currently in a pivotal Phase 3 VIITAL™ clinical trial for recessive dystrophic epidermolysis bullosa (RDEB) with target enrollment achieved in Q1 2022[20]. - EB-101 has received multiple designations from the FDA, including Regenerative Medicine Advanced Therapy (RMAT), Breakthrough Therapy, Rare Pediatric Disease, and Orphan Drug designations[28]. - The co-primary endpoints of the VIITAL™ trial include a) proportion of EB-101 treated wounds with >50% healing from baseline at 24 weeks and b) improvement in pain at 24 weeks[30]. - The current management of RDEB involves supportive care, with no approved treatments available in the U.S. or Europe[25]. - ABO-102 demonstrated over 100% increase in lifespan in MPS IIIA animal models compared to untreated controls[38]. - In the pivotal clinical trial for ABO-102, subjects showed continuous cognitive gains within the normal range for non-afflicted children, particularly those treated before 30 months of age[39]. - As of March 2022, 24 patients were treated in the ABO-102 study, with no deaths or serious drug-related adverse events reported[40]. - The FDA recommended following all participants in the ABO-102 study to at least 60 months, delaying neurocognitive outcomes data readout to late-2024/early-2025[41]. - ABO-101 showed clear signals of biological effect with reductions in disease-specific biomarkers and liver volumes in MPS IIIB patients[45]. - The ABO-401 program demonstrated the ability to deliver the CFTR transgene to the lungs, correcting chloride current deficits in human CF cells[53]. Manufacturing and Development - Abeona has established cGMP manufacturing capabilities in Cleveland, Ohio, to support the production of EB-101 for clinical trials and future commercialization[32]. - The company has established a cGMP manufacturing facility in Cleveland, Ohio, enhancing supply chain control and production efficiency for clinical trials[54]. - The first two phases of the manufacturing facility build-out have been completed, totaling 14,000 square feet of state-of-the-art production and laboratory space[55]. - The company has developed optimized manufacturing processes for EB-101, ensuring sufficient scale for commercial manufacturing requirements[59]. - The company is working towards filing a potential BLA for EB-101, aligning with FDA CMC requirements for characterization and validation plans[60]. Intellectual Property - The company aims to expand its intellectual property portfolio by aggressively seeking patent rights for various aspects of its programs[18]. - The company has secured an exclusive license for AAV-based treatments for Mucopolysaccharidosis IIIA and IIIB, with four pending patent applications in the U.S. expected to expire between late 2029 and mid-2032[65]. - An exclusive license for AAV gene therapy for CLN3 disease has been obtained, including U.S. Patent No. 10,876,134, which is expected to expire in December 2035[66]. - The company has licensed a patent family from Stanford University for the treatment of Recessive Dystrophic Epidermolysis Bullosa, with patents granted in Europe, Australia, and Hong Kong, expected to expire in early 2037[67]. - Exclusive rights to AIM™ capsids have been licensed, with national stage applications filed in multiple countries, and the first patent in this family is projected to expire on November 6, 2036[68]. - The company has licensed rights to a patent portfolio for optimized CLN1 genes for treating CLN1 disease, with expected expiration around 2037[69]. - Patent applications related to gene therapy for Rett Syndrome are pending in several countries, with expected expiration around 2039[70]. - The company is actively seeking U.S. and international patent protection for various technologies, including methods for transferring genetic material into cells and AAV-based biological products[63]. Regulatory Environment - The FDA regulates biologic products, including gene therapy, under the FDCA and PHSA, requiring extensive testing and approval processes before marketing[72]. - Human clinical trials for biologic products typically involve three phases, with Phase 3 requiring two adequate and well-controlled trials for FDA approval[84]. - Post-approval clinical trials may be required by the FDA to gain additional experience regarding long-term safety follow-up[86]. - The FDA requires sponsors to submit IND safety reports within 15 calendar days for serious and unexpected adverse events[88]. - The FDA may suspend clinical trials if research subjects are exposed to unacceptable health risks[89]. - Manufacturers of biologics must comply with cGMP regulations and register their establishments with the FDA[92]. - The FDA reviews a BLA within 60 days to determine if it is substantially complete before accepting it for filing[101]. - The FDA aims to review 90% of standard BLAs in 10 months and 90% of priority BLAs in 6 months after acceptance[106]. - Orphan drug designation can provide financial incentives such as grant funding and tax advantages for products intended to treat rare diseases[107]. - The FDA may expedite the review of BLAs through programs like Fast Track for products addressing serious conditions[109]. - The FDA may require post-marketing clinical trials to further assess a biologic product's safety and effectiveness[105]. - The approval process may include inspections of manufacturing facilities to ensure compliance with cGMP[103]. - The FDA may issue a complete response letter outlining deficiencies in a BLA submission, requiring additional testing or information[104]. - The FDA's breakthrough therapy designation provides intensive guidance and a collaborative review process for products intended to treat serious diseases, potentially expediting development[111]. - Priority review applications are aimed to be completed within six months, significantly faster than the standard ten-month review period for serious condition treatments[111]. - Accelerated approval allows products to be approved based on surrogate endpoints that predict clinical benefits, with post-marketing trials required to confirm efficacy[113]. - The 21st Century Cures Act enables accelerated review for regenerative advanced therapies, which may include cell or gene therapies, to address unmet medical needs[115]. - Post-approval, manufacturers must comply with cGMP regulations, including quality control and reporting of adverse events, to ensure product safety and efficacy[116]. - The FDA can suspend licenses if public health is at risk and requires official lot release for certain products before distribution[117]. - The Drug Quality and Security Act mandates tracking and tracing of biopharmaceutical products to prevent counterfeit and ensure safety[122]. - The FDA grants 12 years of exclusivity for reference biologics, with biosimilar applications not permitted until four years post-approval of the reference product[128]. - The Rare Pediatric Disease Voucher Program allows sponsors to receive priority review vouchers for products treating serious pediatric diseases, which can be sold for substantial sums[131]. - Non-compliance with regulatory requirements can lead to severe penalties, including product recalls and criminal prosecution[135]. Market and Competition - The company anticipates facing intense competition as new product candidates enter the market and advanced technologies become available[159]. - Competitors may have greater financial, technical, and human resources, potentially leading to faster FDA approvals and stronger market positions[156][160]. - The company may face reduced commercial opportunities if competitors develop superior products[160]. Financial and Operational Considerations - The estimated annual cost of wound dressings for an RDEB patient ranges from $245,000 to significantly higher in severe cases[26]. - The incidence of RDEB is estimated to be between 0.2 to 3.05 per million births, with a potential prevalence of up to 3,850 patients in the U.S. who may benefit from EB-101[23]. - The U.S. government and state legislatures are increasingly focused on healthcare cost containment, which may impact drug pricing and reimbursement rates[150]. - In the EU, pricing and reimbursement schemes vary significantly by country, with some requiring agreement on reimbursement prices before marketing[151]. - The Affordable Care Act has introduced measures such as increased Medicaid rebates and changes affecting manufacturer discounts, impacting drug profitability[153]. Compliance and Governance - The company is committed to transparency and provides access to its annual reports and corporate governance policies on its website[166]. - The company operates under the U.S. Foreign Corrupt Practices Act, which governs interactions with foreign officials[155]. - The company is subject to various environmental regulations that could impact operations and financial liabilities[154]. - Compliance with healthcare laws and regulations can result in significant costs and potential penalties for violations, impacting operational strategies[148]. - The federal Anti-Kickback Statute prohibits remuneration for referrals related to healthcare services, impacting pharmaceutical marketing practices[145]. - The federal False Claims Act allows whistleblowers to pursue claims against entities presenting false claims for payment, affecting compliance and financial risk[147]. Workforce and Culture - As of March 21, 2022, the company had 90 full-time employees and maintains good relations with its personnel[165]. - The company competes for qualified personnel with numerous pharmaceutical and biotechnology firms, emphasizing a collaborative culture and employee well-being[164]. - The company sources specialized materials from multiple suppliers globally, ensuring availability through alternate suppliers[163]. - The company has a history of mergers and acquisitions, including the acquisition of Abeona Therapeutics LLC in May 2015[162].

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 September 30, 2020 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ________ to _______ ...

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 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, 2020 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 ...

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) [X] QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2020 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 ...