Financial Performance - Abeona Therapeutics Inc. closed the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $155 million on June 27, 2025, contributing to a total cash resource of approximately $225 million as of June 30, 2025 [1][2] - The proceeds from the PRV, combined with existing cash, provide the company with over two years of operating capital, ensuring financial flexibility without the need for further capital infusion [2] Product Development - ZEVASKYN™ (prademagene zamikeracel) is the first and only U.S. approved autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [2][3] - The company anticipates the first patient treatment with ZEVASKYN in Q3 2025, with profitability projected for early 2026 [2] Company Overview - Abeona Therapeutics is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases [3] - The company operates a fully integrated cGMP manufacturing facility in Cleveland, Ohio, which serves as the production site for ZEVASKYN [3]

Core Insights - Abeona Therapeutics has announced that Beacon Therapeutics has exercised its option to license Abeona's patented AAV204 capsid for potential gene therapies targeting various retinal diseases, which could lead to significant financial benefits for Abeona through license payments and royalties [1][3]. Group 1: Licensing Agreement - The licensing agreement with Beacon Therapeutics is worldwide and non-exclusive, allowing Beacon to develop gene therapies for up to five ophthalmic disease targets using AAV204 [1][3]. - Abeona will receive an undisclosed upfront license payment, along with additional payments tied to development, regulatory, and sales milestones, plus tiered royalties on worldwide net sales of products incorporating AAV204 [3]. Group 2: AAV204 Capsid Details - AAV204 is a novel AAV capsid from the AIM™ capsid library, which has demonstrated high transduction levels in the macula and optic nerve after administration in preclinical studies [2][4]. - The AIM™ capsid library includes various AAV serotypes designed to improve delivery to tissues affected by genetic diseases, potentially evading immune responses [4]. Group 3: Company Overview - Abeona Therapeutics is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, including its autologous cell-based gene therapy ZEVASKYN™ for treating recessive dystrophic epidermolysis bullosa [5]. - The company operates a fully integrated cGMP manufacturing facility in Cleveland, Ohio, which supports the commercial production of ZEVASKYN [5].

Core Insights - Abeona Therapeutics Inc. has been added to the U.S. small-cap Russell 2000® Index and the broad-market Russell 3000® Index, effective June 30, 2025, marking a significant milestone for the company [1][2] - The inclusion in these indexes is expected to enhance the company's visibility and attract a wider range of investors, particularly following the FDA approval of ZEVASKYN™ [2] - The Russell 3000® Index includes the 3,000 largest U.S.-traded stocks, and membership is updated annually, providing a benchmark for investment strategies [3] Company Overview - Abeona Therapeutics is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases [4] - ZEVASKYN™ is the first autologous cell-based gene therapy approved for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [4] - The company operates a fully integrated cGMP manufacturing facility in Cleveland, Ohio, which is responsible for ZEVASKYN's commercial production [4]

Core Insights - ZEVASKYN™ (prademagene zamikeracel) is the first FDA-approved autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [1][2][9] - The pivotal Phase 3 VIITAL study demonstrated significant wound healing and pain reduction after a single treatment, with 81% of treated wounds showing at least 50% healing compared to 16% in control wounds [5][13] - The publication of the VIITAL study results in The Lancet marks a significant milestone for Abeona Therapeutics as it prepares for the U.S. launch of ZEVASKYN [3][4] Company Overview - Abeona Therapeutics Inc. is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, with ZEVASKYN being a key product in its portfolio [15] - The company operates a fully integrated cGMP cell and gene therapy manufacturing facility in Cleveland, Ohio, which is responsible for the commercial production of ZEVASKYN [15] Study Details - The VIITAL study was an intra-patient randomized, open-label, controlled Phase 3 trial involving 11 patients with RDEB, assessing 43 pairs of large chronic wounds [3][4] - The study's co-primary endpoints of wound healing and pain reduction were successfully met, with significant statistical differences observed [5][13] Clinical Results - At week 24, 65% of treated wounds achieved 75% or more healing compared to 7% of control wounds, indicating a substantial improvement in treatment efficacy [13] - Mean change in wound pain was -3.1 in treated wounds versus -0.9 in control wounds, demonstrating a significant reduction in pain levels [13] Safety Profile - No serious ZEVASKYN-related adverse events were reported, consistent with previous clinical experiences, and no cases of squamous cell carcinoma were observed in treated wounds [13][14]

Core Insights - Abeona Therapeutics has received FDA approval for its product pz-cel, which is a significant milestone for the company [1] - Prior to this approval, the company was assigned a Hold rating due to uncertainties surrounding its prospects [1] - The focus is on small and microcap biopharmaceutical companies, where mispricing is often most intense and institutional attention is limited [1] Company Analysis - Abeona Therapeutics operates in the biopharmaceutical sector, specifically targeting clinical stage products [1] - The company is part of a market segment that is often overlooked by institutional investors, leading to potential mispricing opportunities [1] - The analyst team emphasizes the importance of clinical information and consists mainly of high-ranking Life Sciences students from top UK universities [1] Market Context - The investment landscape for small biopharmaceutical companies is characterized by a lack of accurate analyst coverage, often influenced by investment banks with vested interests [1] - Retail investors make up a significant portion of public ownership in these companies, which can lead to price movements based on immaterial information [1]

Summary of Abeona Therapeutics (ABEO) Conference Call Company Overview - Abeona Therapeutics is a commercial stage cell and gene therapy company that recently received approval for ZivaSkin, a therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB) [5][70] - The company has a market cap of approximately $300 million and anticipates becoming profitable in the early half of 2026 [6][71] Key Developments - ZivaSkin was approved just over a month ago, and the company has entered into a sale agreement for a Priority Review Voucher (PRV) for $155 million [5][70] - Abeona is funded for eight quarters of operations without accounting for revenue generation [6][71] Launch Metrics and Treatment Centers - Lurie Children's Hospital has been activated as the first qualified treatment center for ZivaSkin [7][72] - The company is tracking patient throughput and referral volume from other centers of excellence [7][72] - The timeline to fully activate all five treatment centers is by the end of 2025 [9][74] Patient Demand and Treatment Capacity - There are currently an upper double-digit number of patients eligible for treatment across the five centers [11][73] - The company expects to treat 10 to 14 patients this year, with the capacity to ramp up to 10 patients per month by the first half of 2026 [18][37] - The treatment window runs from August to November, with minimal disruption expected due to a planned December manufacturing shutdown [20][21] Pricing and Payer Engagement - ZivaSkin is priced at $3.1 million, which is considered reasonable compared to the standard of care costs [28][30] - The company has not faced pushback from payers and has entered agreements covering over 100 million commercially insured lives [31][36] Financial Outlook - Abeona expects to achieve cash flow positivity in 2026 with a target of treating 36 patients [37][39] - The company is confident in exceeding this target, potentially treating up to 50 patients [38][39] Future Plans and Market Expansion - Abeona is exploring regulatory submissions outside the U.S. and assessing reimbursement scenarios in major markets like Western Europe and Japan [46][47] - The company has identified approximately 750 patients in the U.S. with RDEB, with a significant opportunity for repeat treatments [49][51] Competitive Landscape - ZivaSkin differentiates itself by providing long-lasting wound coverage with a single application, addressing both wound healing and quality of life improvements [53][54] Partner Programs - Abeona is eligible for royalties and milestones from partnered programs with Ultragenyx and Atacea, which are not factored into current projections [55][58] Key Milestones Ahead - The company aims to achieve its treatment goals for ZivaSkin and activate additional qualified treatment centers, with several PDUFA action dates for partnered programs expected in 2025 [66][67]

Company Overview - Abeona Therapeutics Inc. is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases [3] - The company's flagship product, ZEVASKYN™ (prademagene zamikeracel), is the first and only autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [3] - Abeona operates a fully integrated cGMP manufacturing facility in Cleveland, Ohio, which is dedicated to the commercial production of ZEVASKYN [3] Upcoming Events - The CEO, Vish Seshadri, and the Chief Commercial Officer, Madhav Vasanthavada, will participate in a fireside chat at the Jefferies Global Healthcare Conference on June 4, 2025, at 11:40 a.m. Eastern Time [1] - A live webcast of the event will be available on the Investors section of the Abeona website and will be archived for 30 days [2] Product Development - Abeona's development portfolio includes adeno-associated virus (AAV)-based gene therapies targeting ophthalmic diseases with significant unmet medical needs [3] - The company is evaluating next-generation AAV capsids to enhance tropism profiles for various severe diseases [3]

Financial Data and Key Metrics Changes - As of March 31, 2025, the company had cash, cash equivalents, short-term investments, and restricted cash of $84.5 million, down from $98.1 million as of December 31, 2024 [19] - Research and development expenses increased to $9.9 million for Q1 2025 from $7.2 million in Q1 2024, primarily due to increased headcount for manufacturing scale-up [20] - General and administrative expenses rose to $9.8 million for Q1 2025 from $7.1 million in Q1 2024, also due to increased headcount related to the ZivaSkin launch [20] - The net loss for Q1 2025 was $12 million, or $0.24 loss per share, compared to a net loss of $31.6 million, or $1.16 loss per share, in Q1 2024 [21] Business Line Data and Key Metrics Changes - The company launched ZivaSkin, the first autologous cell-based gene therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB), following FDA approval [5][6] - The first Qualified Treatment Center (QTC) was activated ahead of schedule, with Lurie Children's Hospital of Chicago being the first to offer ZivaSkin [8] - The company anticipates treating the first patient at Lurie in Q3 2025, with a goal of treating 10 to 14 patients by the end of 2025 [10][16] Market Data and Key Metrics Changes - The company has executed outcomes-based agreements with two payer contracting organizations to ensure access to ZivaSkin for patients [18] - The inbound interest from patients and families has been significant, with approximately 30 inquiries received in the first two weeks post-approval [12][13] Company Strategy and Development Direction - The company aims to achieve profitability driven by ZivaSkin revenues by early 2026, supported by the sale of a Priority Review Voucher (PRV) for $155 million [11][19] - The company plans to activate additional QTCs by the end of 2025, focusing on well-recognized institutions with experience in cell and gene therapy [14][15] - The company is also preparing to advance its ophthalmology pipeline, with plans to bring an asset into clinical trials in the second half of 2026 [45][46] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the initial launch of ZivaSkin and the positive response from patients and physicians [12][16] - The management highlighted the importance of payer agreements to ensure timely access to ZivaSkin for patients [18] - The company is confident in its financial position, stating that the proceeds from the PRV sale will fund operations for over two years [19] Other Important Information - The company presented two late-breaking abstracts at the Society for Investigative Dermatology Annual Meeting, reinforcing ZivaSkin's efficacy and safety profile [10] - The company is actively engaging with commercial payers and state Medicaid programs to expand access to ZivaSkin [19] Q&A Session Summary Question: What does site activation mean? - Site activation includes training staff and surgeons, allowing them to identify patients for ZivaSkin and begin the payer authorization process [25][27] Question: How many RDEB patients are eligible at Lurie Children's Hospital? - There are at least a couple of dozen RDEB patients treated at Lurie, with potential for more referrals from the local community [28][29] Question: What factors affect patient access to treatment? - Commercially insured patients are expected to have faster access compared to Medicaid patients, which may take longer [31][32] Question: What is the expected patient throughput at QTCs? - Initial throughput is expected to be around two patients per month per site, with potential to increase as centers gain experience [41][42] Question: What are the anticipated net proceeds from the PRV sale? - The net proceeds from the PRV sale are expected to be approximately $152 million [58] Question: How will billing and reimbursement work? - Revenue recognition will occur after patient treatment, with discussions already in place with insurance companies to ensure reimbursement [78][80]

Financial Data and Key Metrics Changes - As of March 31, 2025, the company had cash, cash equivalents, short-term investments, and restricted cash of $84.5 million, down from $98.1 million as of December 31, 2024, before accounting for the proceeds from the Priority Review Voucher (PRV) sale [17] - Research and development expenses increased to $9.9 million for Q1 2025 from $7.2 million in Q1 2024, primarily due to increased headcount for manufacturing scale-up [18] - General and administrative expenses rose to $9.8 million for Q1 2025 from $7.1 million in Q1 2024, also due to increased headcount related to the ZivaSkin launch [18] - The net loss for Q1 2025 was $12 million, or $0.24 loss per share, compared to a net loss of $31.6 million, or $1.16 loss per share, in Q1 2024 [19] Business Line Data and Key Metrics Changes - The company launched ZivaSkin, the first autologous cell-based gene therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB), following FDA approval [4][5] - The first Qualified Treatment Center (QTC) activated was Lurie Children's Hospital of Chicago, which is expected to treat the first patient in Q3 2025 [6][8] Market Data and Key Metrics Changes - The company has received inquiries from approximately 30 patients and families across the country since ZivaSkin's approval, indicating strong demand [10] - The company is actively engaging with commercial payers and state Medicaid programs to expand access to ZivaSkin [16] Company Strategy and Development Direction - The company aims to treat 10 to 14 patients with ZivaSkin in 2025 and anticipates a robust start to 2026 [13] - The company has entered into an agreement to sell the PRV for $155 million, which will strengthen its balance sheet and fund operations for over two years [9][17] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the initial launch of ZivaSkin and the positive response from patients and physicians [10][13] - The company is focused on ensuring that the value of ZivaSkin is recognized by payers and that patients have timely access to the therapy [16] Other Important Information - The company expects to achieve profitability driven by ZivaSkin revenues in early 2026 [17] - The company is on track to activate additional QTCs by the end of 2025, with ongoing discussions with recognized institutions [12] Q&A Session Summary Question: What does site activation mean? - Site activation means that staff and surgeons are trained and ready to identify patients for ZivaSkin treatment [22][25] Question: How many RDEB patients are eligible at Lurie Children's Hospital? - There are at least a couple of dozen patients treated at Lurie, with potential for more referrals from the local community [26] Question: What factors affect patient access to treatment? - Commercially insured patients are expected to have faster access compared to Medicaid patients, which may take longer [29] Question: What is the expected patient throughput at QTCs? - Initial throughput is expected to be two patients per month per site, with potential to ramp up to four patients per month at some centers [39] Question: What are the anticipated net proceeds from the PRV sale? - The net proceeds from the PRV sale are expected to be about $152 million [54] Question: How will billing and reimbursement work? - Revenue recognition will occur after patient treatment, with discussions already in place with insurance companies to ensure reimbursement [72][75]

Financial Performance - Net loss for the three months ended March 31, 2025, was $12,029 thousand, compared to a net loss of $31,578 thousand for the same period in 2024, representing a 61.9% improvement[15] - The company experienced a net cash used in operating activities of $18,402 thousand for the three months ended March 31, 2025, compared to $14,538 thousand for the same period in 2024, indicating a 26.5% increase in cash outflow[21] - Abeona Therapeutics reported net losses of $12.0 million and $31.6 million for the three months ended March 31, 2025, and 2024, respectively, with an accumulated deficit of approximately $825.3 million as of March 31, 2025[27] - Total expenses for the three months ended March 31, 2025, were $19,727 thousand, compared to $14,330 thousand for the same period in 2024, reflecting a 37.5% increase[15] Assets and Liabilities - Total current assets decreased from $100,853 thousand as of December 31, 2024, to $88,121 thousand as of March 31, 2025, a decline of approximately 12.7%[13] - Total liabilities decreased from $64,900 thousand as of December 31, 2024, to $57,968 thousand as of March 31, 2025, a reduction of about 10.7%[13] - The company’s total stockholders' equity decreased from $44,031 thousand as of December 31, 2024, to $41,396 thousand as of March 31, 2025, a decrease of about 6.0%[13] - Cash and cash equivalents decreased from $23,357 thousand as of December 31, 2024, to $15,936 thousand as of March 31, 2025, a decline of approximately 31.7%[13] Research and Development - Research and development expenses increased to $9,941 thousand for the three months ended March 31, 2025, up from $7,207 thousand in the same period of 2024, an increase of 38.1%[15] - For the three months ended March 31, 2025, total research and development costs amounted to $9.941 million, an increase of 38.1% from $7.207 million in the same period of 2024[106] Financing and Capital Structure - The Company raised $75 million gross from the sale of 12,285,056 shares of common stock and pre-funded warrants on May 7, 2024, with net proceeds of $70.2 million[75] - Under the ATM Agreement, the Company sold 1,312,283 shares of common stock during the three months ended March 31, 2025, resulting in net proceeds of $6.8 million[77] - The Company has a financial covenant requiring it to maintain at least $5 million in unrestricted cash at all times[67] - The Company has a total of 16,028,297 potential dilutive securities that could affect future net loss per share calculations[37] Debt and Obligations - The Company has a total long-term debt of $19,286,000 as of March 31, 2025, with current maturities of $8,148,000[63] - The Company has a total principal payment obligation of $20 million, with $5.926 million due in 2025, $8.889 million in 2026, and $5.185 million in 2027[71] - The effective interest rate on the Company's loans as of March 31, 2025, was 22.09%[65] Revenue and Milestones - The Company has not recognized any significant revenues to date and may continue to incur operating losses until ZEVASKYN™ can provide sufficient revenue[27] - The Company has not recognized any revenue during the three months ended March 31, 2025, from event-based milestone payments related to the sublicense agreements[95] - The Company is subject to remaining milestone payments totaling approximately $0.2 million due upon FDA approval of ZEVASKYN™ on April 28, 2025[89] Stock and Equity - The weighted average number of common shares outstanding increased from 27,315,537 for the three months ended March 31, 2024, to 49,778,801 for the same period in 2025, an increase of 82.3%[15] - Total stock-based compensation expense for the three months ended March 31, 2025, was $2,701,000, a 74.5% increase from $1,546,000 in the same period of 2024[85] - As of March 31, 2025, there were 5,250,307 restricted stock awards outstanding, with a grant date fair value per unit of $4.79[88] Regulatory and Approvals - On April 28, 2025, the FDA approved ZEVASKYN™ as the first and only autologous cell-based gene therapy for treating wounds in patients with RDEB[108] - The Company entered into an exclusive license agreement with Ultragenyx for AAV gene therapy ABO-102, with potential royalties ranging from mid-single-digit percentages up to 10% on net sales and up to $30 million in commercial milestone payments[100]