Core Viewpoint - Abeona Therapeutics received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel), indicating the need for additional Chemistry Manufacturing and Controls (CMC) information, but did not identify any deficiencies related to clinical efficacy or safety data [1][2][3] Group 1: Regulatory Update - The FDA's CRL was issued following a Late Cycle Review Meeting in March 2024, where it was noted that additional CMC information is required before the application can be approved [1][2] - Abeona plans to complete and submit the requested CMC information by the third quarter of 2024 [1][3] - A conference call is scheduled for April 23, 2024, to provide details on the requested CMC information [4] Group 2: Product Information - Prademagene zamikeracel (pz-cel) is an investigational treatment for recessive dystrophic epidermolysis bullosa (RDEB), utilizing gene-corrected epidermal sheets to address a defect in the COL7A1 gene [5][6] - The pivotal Phase 3 VIITAL™ study demonstrated that a single application of pz-cel can lead to sustained wound healing and pain reduction [3][5] - Pz-cel has received multiple designations from the FDA, including Regenerative Medicine Advanced Therapy and Breakthrough Therapy [6] Group 3: Company Overview - Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases [7] - The company operates a fully integrated gene and cell therapy manufacturing facility in Cleveland, Ohio, capable of supporting commercial production upon FDA approval [7]

[Company Announcement & Highlights](index=1&type=section&id=Company%20Announcement%20%26%20Highlights) Abeona Therapeutics reported 2023 financial results and completed key FDA inspections for its pz-cel BLA, including manufacturing and clinical sites [Full Year 2023 Financial Results and FDA Inspections Completion](index=1&type=section&id=Full%20Year%202023%20Financial%20Results%20and%20FDA%20Inspections%20Completion) Abeona Therapeutics reported its full year 2023 financial results and announced the completion of key FDA inspections for its pz-cel Biologics License Application (BLA), including a Pre-License Inspection of its Cleveland manufacturing facility and clinical trial site inspections - Abeona Therapeutics reported **full year 2023 financial results**[1](index=1&type=chunk) - FDA completed a **Pre-License Inspection (PLI)** of Abeona's Cleveland manufacturing facility for pz-cel BLA[2](index=2&type=chunk) - FDA also completed clinical study site inspections for the pivotal Phase 3 VIITAL™ study, with **no Form 483 observations**[2](index=2&type=chunk) [Regulatory & Commercial Progress](index=1&type=section&id=Regulatory%20%26%20Commercial%20Progress) Abeona advanced pz-cel's FDA review with priority status, prepared for U.S. commercial launch, and secured a $50 million credit facility [Pz-cel for RDEB Regulatory Updates](index=1&type=section&id=Pz-cel%20for%20RDEB%20Regulatory%20Updates) The FDA has made significant progress in its review of Abeona's pz-cel BLA for recessive dystrophic epidermolysis bullosa (RDEB), including granting Priority Review, completing various inspections, and reaffirming a target PDUFA date of May 25, 2024, without plans for an Advisory Committee - FDA completed a Pre-License Inspection (PLI) of the Cleveland manufacturing facility, issuing a **Form 483 with observations** related to process controls, to which Abeona submitted a response[2](index=2&type=chunk) - FDA accepted and granted **Priority Review** for the pz-cel BLA in November 2023, with a target PDUFA date of **May 25, 2024**[3](index=3&type=chunk) - FDA completed a Bioresearch Monitoring (BIMO) inspection and Mid-Cycle Meeting in January 2024, with **no Form 483s issued** from the BIMO inspection and **no Risk Evaluation and Mitigation Strategies (REMS) anticipated** for the BLA[4](index=4&type=chunk) [U.S. Commercial Launch Preparations for pz-cel](index=2&type=section&id=U.S.%20Commercial%20Launch%20Preparations%20for%20pz-cel) Abeona is actively preparing for the potential U.S. commercial launch of pz-cel, engaging with treatment sites, payers, and conducting market research to support reimbursement coverage - Abeona is **advancing key commercial activities** for pz-cel launch, including onboarding discussions with EB treatment sites, payer engagement, and hiring key commercial roles[5](index=5&type=chunk) - Payer market research supports **reimbursement coverage** of pz-cel at a price commensurate with the value of approved gene therapies[5](index=5&type=chunk) [Corporate Financing Highlights](index=2&type=section&id=Corporate%20Financing%20Highlights) Abeona secured a $50 million credit facility in January 2024, with an initial $20 million tranche funded, to support ongoing launch and commercialization preparations for pz-cel - Abeona entered a **$50 million credit facility** with Avenue Venture Opportunities Fund, L.P. in January 2024[5](index=5&type=chunk) - The credit agreement includes a **first tranche of $20 million**, funded in January 2024, a second tranche of $10 million, and an option for an additional $20 million[5](index=5&type=chunk) - Proceeds from the facility are intended to **support pz-cel launch and commercialization** preparations[5](index=5&type=chunk) [Financial Performance](index=2&type=section&id=Financial%20Performance) Abeona's 2023 financials show stable cash, increased licensing revenue, higher operating expenses, and an increased net loss [Full Year 2023 Financial Results and Cash Runway Guidance](index=2&type=section&id=Full%20Year%202023%20Financial%20Results%20and%20Cash%20Runway%20Guidance) Abeona maintained a stable cash position at the end of 2023 and significantly reduced net cash used in operating activities. With recent financing, the company projects its cash runway to extend into the first quarter of 2025 Cash and Investments (in millions) | Metric | Dec 31, 2023 | Dec 31, 2022 | | :----------------------------------------- | :----------- | :----------- | | Cash, cash equivalents, restricted cash and short-term investments | $52.6 | $52.5 | Net Cash Used in Operating Activities (in millions) | Period | 2023 | 2022 | | :----------- | :---- | :---- | | Net cash used in operating activities | $37.0 | $43.5 | - Current cash and credit facility are **sufficient to fund operations into Q1 2025**, excluding potential pz-cel revenue or Priority Review Voucher proceeds[6](index=6&type=chunk) [Revenue Analysis](index=2&type=section&id=Revenue%20Analysis) License and other revenues increased significantly in 2023, primarily driven by clinical milestone payments from a licensing agreement for an AAV-based gene therapy License and Other Revenues (in millions) | Metric | 2023 | 2022 | YoY Change | | :------------------------- | :---- | :---- | :--------- | | License and other revenues | $3.5 | $1.4 | +150% | - Revenues primarily represent **clinical milestone payments** under a licensing agreement with Taysha Gene Therapies for investigational AAV-based gene therapy for Rett syndrome[7](index=7&type=chunk) [Operating Expenses](index=2&type=section&id=Operating%20Expenses) Both research and development (R&D) and general and administrative (G&A) expenses increased in 2023, primarily due to increased headcount supporting BLA activities and pz-cel launch preparations Operating Expenses (in millions) | Metric | 2023 | 2022 | YoY Change | | :------------------------------- | :---- | :---- | :--------- | | Research and development expenses | $31.1 | $29.0 | +7.2% | | General and administrative expenses | $19.0 | $17.3 | +9.8% | - Increase in R&D expenses was primarily due to **increased headcount related to BLA activities**[8](index=8&type=chunk) - Increase in G&A expenses was primarily due to **increased headcount for the potential launch of pz-cel**[8](index=8&type=chunk) [Net Loss and EPS](index=2&type=section&id=Net%20Loss%20and%20EPS) Abeona reported an increased net loss attributable to common shareholders in 2023, with a corresponding decrease in loss per common share due to a higher weighted average number of shares outstanding Net Loss and EPS (in millions, except per share amounts) | Metric | 2023 | 2022 | YoY Change | | :----------------------------------------- | :---------- | :---------- | :--------- | | Net loss attributable to common shareholders | $(54.2) million | $(43.5) million | +24.6% | | Loss per common share | $(2.53) | $(5.53) | -54.3% | [Company Information](index=3&type=section&id=Company%20Information) Abeona Therapeutics is a clinical-stage biopharmaceutical company developing cell and gene therapies, with pz-cel for RDEB nearing FDA approval [About Abeona Therapeutics](index=3&type=section&id=About%20Abeona%20Therapeutics) Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, with pz-cel for RDEB as its lead investigational product nearing potential FDA approval, supported by its integrated cGMP manufacturing facility - Abeona Therapeutics is a **clinical-stage biopharmaceutical company** developing cell and gene therapies for serious diseases[10](index=10&type=chunk) - The U.S. FDA has granted **Priority Review** for the BLA for pz-cel (prademagene zamikeracel) for recessive dystrophic epidermolysis bullosa (RDEB), with a PDUFA target action date of **May 25, 2024**[10](index=10&type=chunk) - The company's development portfolio also includes **AAV-based gene therapies** for ophthalmic diseases and **novel, next-generation AAV capsids**[10](index=10&type=chunk) [Financial Statements](index=4&type=section&id=Financial%20Statements) This section details Abeona Therapeutics' consolidated statements of operations, comprehensive loss, and balance sheets for 2023 and 2022 [Consolidated Statements of Operations and Comprehensive Loss](index=4&type=section&id=Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) The consolidated statements of operations and comprehensive loss provide a detailed breakdown of Abeona Therapeutics' financial performance for the years ended December 31, 2023, and 2022, including revenues, expenses, and net loss Consolidated Statements of Operations and Comprehensive Loss (in thousands, except share and per share amounts) | Metric | 2023 | 2022 | | :--------------------------------------------------------------------------------------------------------- | :----------- | :----------- | | Revenues: | | | | License and other revenues | $3,500 | $1,414 | | Expenses: | | | | Royalties | $1,605 | $450 | | Research and development | $31,091 | $28,965 | | General and administrative | $19,004 | $17,256 | | Impairment of licensed technology | — | $1,355 | | Loss/(gain) on operating lease right-of-use assets | $(1,065) | $2,511 | | Impairment of construction-in-progress | — | $1,792 | | Total expenses | $50,635 | $52,329 | | Loss from operations | $(47,135) | $(50,915) | | Interest income | $2,117 | $431 | | Interest expense | $(418) | $(736) | | Change in fair value of warrant liabilities | $(11,695) | $11,383 | | Other income | $2,943 | $141 | | Net loss | $(54,188) | $(39,696) | | Deemed dividends related to Series A and Series B Convertible Redeemable Preferred Stock | — | $(3,782) | | Net loss attributable to Common Shareholders | $(54,188) | $(43,478) | | Basic and diluted loss per common share | $(2.53) | $(5.53) | | Weighted average number of common shares outstanding – basic and diluted | 21,380,476 | 7,861,515 | | Other comprehensive income (loss): | | | | Change in unrealized gains (losses) related to available-for-sale debt securities | $34 | $(99) | | Foreign currency translation adjustments | $29 | $(3) | | Comprehensive loss | $(54,125) | $(43,580) | [Consolidated Balance Sheets](index=5&type=section&id=Consolidated%20Balance%20Sheets) The consolidated balance sheets present Abeona Therapeutics' financial position as of December 31, 2023, and 2022, detailing assets, liabilities, and stockholders' equity Consolidated Balance Sheets (in thousands, except share and per share amounts) | Metric | December 31, 2023 | December 31, 2022 | | :--------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------- | :------------------ | :------------------ | | ASSETS | | | | Current assets: | | | | Cash and cash equivalents | $14,473 | $14,217 | | Short-term investments | $37,753 | $37,932 | | Restricted cash | $338 | $338 | | Other receivables | $2,444 | $188 | | Prepaid expenses and other current assets | $729 | $424 | | Total current assets | $55,737 | $53,099 | | Property and equipment, net | $3,533 | $5,741 | | Operating lease right-of-use assets | $4,455 | $5,331 | | Other assets | $277 | $43 | | Total assets | $64,002 | $64,214 | | LIABILITIES AND STOCKHOLDERS' EQUITY | | | | Current liabilities: | | | | Accounts payable | $1,858 | $1,811 | | Accrued expenses | $5,985 | $3,991 | | Current portion of operating lease liability | $998 | $1,773 | | Current portion of payable to licensor | $4,580 | — | | Other current liabilities | $1 | $204 | | Total current liabilities | $13,422 | $7,779 | | Payable to licensor | — | $4,163 | | Long-term operating lease liabilities | $4,402 | $5,854 | | Warrant liabilities | $31,352 | $19,657 | | Total liabilities | $49,176 | $37,453 | | Commitments and contingencies | | | | Stockholders' equity: | | | | Preferred stock - $0.01 par value; authorized 2,000,000 shares; No shares issued and outstanding as of December 31, 2023 and December 31, 2022, respectively | — | — | | Common stock - $0.01 par value; authorized 200,000,000 shares; 26,523,878 and 17,719,720 shares issued and outstanding as of December 31, 2023 and December 31, 2022, respectively | 265 | 177 | | Additional paid-in capital | $764,151 | $722,049 | | Accumulated deficit | $(749,524) | $(695,336) | | Accumulated other comprehensive loss | $(66) | $(129) | | Total stockholders' equity | $14,826 | $26,761 | | Total liabilities and stockholders' equity | $64,002 | $64,214 | [Disclosures & Contacts](index=2&type=section&id=Disclosures%20%26%20Contacts) This section provides conference call details, forward-looking statement disclaimers, and investor contact information [Conference Call Details](index=2&type=section&id=Conference%20Call%20Details) Abeona Therapeutics hosted a conference call and webcast on March 18, 2024, to discuss the financial results and corporate updates, with replay access available online - Abeona Therapeutics hosted a conference call and webcast on **March 18, 2024, at 8:30 a.m. ET**[9](index=9&type=chunk) - Access details: Dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) with Entry Code: 428606[9](index=9&type=chunk) - A live webcast and archived replay are available at https://investors.abeonatherapeutics.com/events for **30 days**[9](index=9&type=chunk) [Forward-Looking Statements](index=3&type=section&id=Forward-Looking%20Statements) This section contains forward-looking statements subject to various risks and uncertainties, including those related to FDA approvals, clinical trials, and financial market conditions, and the company disclaims any obligation to update these statements - The press release contains **forward-looking statements** as defined by Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934[11](index=11&type=chunk) - Actual results may **differ materially due to various factors**, including the timing and outcome of the pz-cel BLA submission, FDA inspections, regulatory approvals, and global economic conditions[11](index=11&type=chunk) - The Company undertakes **no obligation to revise or update** forward-looking statements, except as required by federal securities laws[11](index=11&type=chunk) [Investor and Media Contact](index=3&type=section&id=Investor%20and%20Media%20Contact) Contact information for investor relations and corporate communications is provided for inquiries - Investor and Media Contact: **Greg Gin, VP, Investor Relations and Corporate Communications**, Abeona Therapeutics[12](index=12&type=chunk) - Email: **ir@abeonatherapeutics.com**[12](index=12&type=chunk)

Regulatory Submissions and Approvals - In September 2023, the company submitted a Biologics License Application (BLA) for pz-cel to the FDA, which was accepted for filing and granted priority review in November 2023, with a target action date set for May 25, 2024[10][27]. - Pz-cel has been granted multiple designations by the FDA, including Regenerative Medicine Advanced Therapy (RMAT) and Breakthrough Therapy, which may provide benefits such as market exclusivity and expedited review[28][27]. - The FDA requires completion of preclinical laboratory tests and in vivo studies in accordance with current Good Laboratory Practice (GLP) regulations before marketing a biologic product candidate[59]. - An Investigational New Drug (IND) application must be submitted to the FDA, which becomes effective 30 days after receipt unless the FDA places the trial on clinical hold[62]. - The FDA may approve a Biologics License Application (BLA) based on a single Phase 3 clinical study under certain circumstances[68]. - The FDA may require a Risk Evaluation and Mitigation Strategy (REMS) for products to ensure safe use, which must be submitted with the BLA if deemed necessary[89]. - The FDA's Fast Track program allows for rolling reviews of BLAs, enabling earlier interactions and review of sections before the application is complete[94]. - Breakthrough therapy designation provides intensive guidance and may lead to priority review for products showing substantial improvement over existing therapies[95]. - Accelerated approval can be granted based on surrogate endpoints, but requires post-marketing studies to confirm clinical benefits[97]. - The FDA conducts inspections of manufacturing facilities and clinical sites to ensure compliance with regulatory standards before approving a BLA[87]. Clinical Development and Trials - The pivotal phase 3 VIITAL™ study demonstrated statistically significant improvements in wound healing and pain reduction for pz-cel in RDEB patients, with no serious treatment-related adverse events reported[25][24]. - Human clinical trials are conducted in three phases, with Phase 3 requiring two adequate and well-controlled trials for FDA approval[67][68]. - Post-approval clinical trials (Phase IV) may be required by the FDA to gain additional experience regarding long-term safety[70]. Manufacturing and Facilities - The company has established current Good Manufacturing Practice (cGMP) clinical-scale manufacturing capabilities in Cleveland, Ohio, which provide advantages in flexibility, scale, reliability, and reduced development risk[14]. - The company has established a cGMP manufacturing facility in Cleveland, Ohio, enhancing supply chain control and increasing supply capacity for clinical trials and commercial demand[38]. - The manufacturing facility has completed a 16,000+ square foot build-out, including a 6,000 square foot cGMP production facility and an additional 8,000 square feet of laboratory space[40]. - The company has produced three cGMP process validation lots for the retroviral vector used in pz-cel, supporting licensure and commercial manufacturing[41]. - The company has filed a Biologics License Application (BLA) to support commercial manufacturing of pz-cel from its Cleveland facility[44]. Market Potential and Financial Aspects - 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 COL7A1-mediated treatments like pz-cel[18]. - The annual cost of wound care for dystrophic epidermolysis bullosa (DEB) patients is identified to be 465% greater than the average annual healthcare cost, with wound care supplies potentially costing up to $996,000 per year[21]. - The company anticipates that healthcare reform measures may significantly affect the sales of its products, particularly regarding pricing and reimbursement policies[138]. - The company is exposed to risks related to coverage and reimbursement from third-party payors, which are increasingly reducing reimbursements for medical products[135][136]. Intellectual Property and Licensing - The company has licensed a patent family from Stanford University covering pz-cel, with patents expected to expire in early 2037, and additional applications pending in the U.S.[48]. - The AIM™ capsids licensed from UNC have a projected expiration date of November 6, 2036, with strong potential for delivering therapeutic transgenes[49]. - The company is actively seeking U.S. and international patent protection for various technologies, including AAV capsids and methods for treating genetic diseases[47]. Strategic Partnerships and Development - The company plans to leverage strategic partnerships and in-licensing to establish additional cell and gene therapy franchises and expand its intellectual property portfolio[15][16]. - The company aims to commercialize its assets either independently or through strategic partnerships, contingent upon FDA approval[12]. Competition and Market Environment - The company faces intense competition in the biotechnology sector, with competitors potentially having greater resources and experience[141][144]. - The company is focused on developing cell and gene therapies for life-threatening diseases, competing for skilled personnel in the pharmaceutical and biotechnology sectors[147]. Compliance and Regulatory Environment - The company is subject to significant regulatory scrutiny, including compliance with the federal False Claims Act and the Physician Payments Sunshine Act[132][134]. - Compliance with foreign regulatory requirements is critical, as failure can lead to fines, product recalls, and other legal consequences[120]. - The federal Anti-Kickback Statute prohibits remuneration for referrals or recommendations of pharmaceutical products, impacting marketing and distribution strategies[129]. - Manufacturers must comply with current Good Manufacturing Practice (cGMP) regulations and register their establishments with the FDA[75]. Workforce and Employment - The company had 84 full-time employees as of December 31, 2023, and maintains good relations with its personnel[148]. - The company has never experienced employment-related work stoppages, indicating a stable workforce[148]. - The company has contracts with scientific consultants and research organizations to support various aspects of drug development[148].

Core Insights - Abeona Therapeutics Inc. reported its financial results for the full year of 2023 and provided updates on its corporate objectives, particularly regarding the Biologics License Application (BLA) for pz-cel [1][10] Regulatory Updates - The FDA completed a Pre-License Inspection (PLI) of Abeona's Cleveland manufacturing facility for pz-cel, issuing a Form 483 with observations related to process controls [2] - The FDA's review of the pz-cel BLA is ongoing, with a target Prescription Drug User Fee Act (PDUFA) date set for May 25, 2024, and no Advisory Committee meeting planned [2][4] Financial Performance - For the year ended December 31, 2023, Abeona reported license and other revenues of $3.5 million, an increase from $1.4 million in 2022 [7] - Research and development expenses rose to $31.1 million in 2023 from $29.0 million in 2022, primarily due to increased headcount for BLA activities [8] - The net loss attributable to common shareholders for 2023 was $54.2 million, or $2.53 loss per share, compared to a net loss of $43.5 million, or $5.53 loss per share, in 2022 [8] Cash Position and Funding - As of December 31, 2023, the company had cash, cash equivalents, restricted cash, and short-term investments totaling $52.6 million, slightly up from $52.5 million in 2022 [6] - Abeona entered a $50 million credit facility with Avenue Venture Opportunities Fund, L.P., with the first tranche of $20 million funded in January 2024 [5] Commercial Launch Preparations - The company is advancing commercial activities in preparation for a potential U.S. launch of pz-cel, including payer engagement and market research [5]

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 ...

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]