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]

FDA Approval and Product Launch - Abeona Therapeutics received FDA approval for ZEVASKYN™, the first autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [1] - The U.S. launch of ZEVASKYN is underway with the activation of the first Qualified Treatment Center (QTC) at Lurie Children's Hospital, expecting to treat the first patient in Q3 2025 [4] Financial Performance - Cash, cash equivalents, restricted cash, and short-term investments totaled $84.5 million as of March 31, 2025, down from $98.1 million as of December 31, 2024 [7] - The net loss for Q1 2025 was $12.0 million, or $0.24 loss per share, significantly improved from a net loss of $31.6 million, or $1.16 loss per share, in Q1 2024 [9] - Abeona's total assets decreased to $99.4 million as of March 31, 2025, from $108.9 million as of December 31, 2024 [18] Research and Development Expenses - Research and development expenses increased to $9.9 million for Q1 2025, compared to $7.2 million for the same period in 2024, primarily due to increased headcount for ZEVASKYN's commercial launch [8] - General and administrative expenses rose to $9.8 million in Q1 2025 from $7.1 million in Q1 2024, reflecting increased staffing for the ZEVASKYN launch [8] Sales and Market Access - Abeona entered into a sales agreement for a priority review voucher (PRV) for gross proceeds of $155 million, which will fund operations for over two years [6] - Abeona has executed value-based agreements with commercial payer groups covering approximately 100 million commercially-insured lives to ensure broad access to ZEVASKYN [5] - The company is actively discussing further access expansion for ZEVASKYN with multiple commercial and government payers [5]

Core Insights - Abeona Therapeutics has received FDA approval for ZEVASKYN™, the first autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [1][5] - The company is transitioning to a commercial-stage entity with the launch of ZEVASKYN and has activated its first treatment center [2][4] Recent Developments - ZEVASKYN's FDA approval marks a significant milestone for RDEB patients, with the first treatment expected in Q3 2025 [5] - Abeona has secured a $155 million non-dilutive capital through the sale of its Rare Pediatric Disease Priority Review Voucher (PRV), which will fund operations for over two years [3] - The company has entered into value-based agreements with commercial payer groups, ensuring access to ZEVASKYN for approximately 100 million commercially-insured lives [5] Financial Results - As of March 31, 2025, the company reported cash and cash equivalents totaling $84.5 million, down from $98.1 million at the end of 2024 [6] - Research and development expenses increased to $9.9 million in Q1 2025 from $7.2 million in Q1 2024, primarily due to scaling up manufacturing for ZEVASKYN [7] - The net loss for Q1 2025 was $12.0 million, a significant reduction from a net loss of $31.6 million in Q1 2024 [8][14]

Core Points - ZEVASKYN is the first and only cell-based gene therapy approved for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [2][8] - Ann & Robert H. Lurie Children's Hospital of Chicago is the first Qualified Treatment Center (QTC) for ZEVASKYN, with treatments expected to begin in Q3 2025 [1][3] - Abeona Therapeutics has launched the Abeona Assist™ program to provide personalized support for patients and families throughout the treatment journey [1][5] 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 cGMP manufacturing facility in Cleveland, Ohio, which is responsible for the commercial production of ZEVASKYN [15] Treatment Details - ZEVASKYN incorporates the COL7A1 gene into a patient's skin cells to produce functional type VII collagen, which is essential for skin integrity [8] - The therapy has shown clinically meaningful results in wound healing and pain reduction with a single application [8] Hospital and Research Center - Lurie Children's Hospital has been a center of excellence for genetic skin diseases for over 30 years and is recognized for its expertise in treating epidermolysis bullosa [3][12] - The hospital has been providing FDA-approved gene therapies since 2019 and is expanding its offerings, with ZEVASKYN being the tenth gene therapy available [4]

Core Insights - The FDA has approved ZEVASKYN as the first and only autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [1][16] - ZEVASKYN demonstrated significant wound healing and pain reduction in a pivotal Phase 3 study, with 81% of treated wounds showing 50% or more healing after six months [3][4] - The therapy will be available through Qualified Treatment Centers starting in the third quarter of 2025 [1][12] Company Overview - Abeona Therapeutics 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 [21] - The company operates a fully integrated cGMP manufacturing facility in Cleveland, Ohio, which will support the commercial production of ZEVASKYN [21] Clinical Evidence - The pivotal Phase 3 VIITAL™ study met its co-primary efficacy endpoints, showing statistically significant healing and pain reduction in large chronic RDEB wounds [3][4] - In a Phase 1/2a study, long-term improvements in wound healing and pain reduction were observed, with a median follow-up of 6.9 years [5] Patient Support and Accessibility - Abeona Assist™ is a patient services program designed to provide personalized support, including insurance benefits and financial assistance [13] - ZEVASKYN will be accessible through specialized treatment centers recognized for their expertise in epidermolysis bullosa [12] Market Impact - The approval of ZEVASKYN is seen as a significant advancement for the RDEB community, addressing a critical unmet need for effective wound treatment [2][8] - The company received a Rare Pediatric Disease Priority Review Voucher from the FDA, which it plans to monetize [14]

Group 1 - Recessive Dystrophic Epidermolysis Bullosa (RDEB) is characterized by chronic wounds covering a significant portion of the body, with an average of 30% affected and some patients experiencing up to 80% coverage [1] - The disease is described as extremely severe, indicating a high level of suffering and medical need for affected individuals [1] Group 2 - No relevant company or industry-specific information is provided in the documents [2] - The documents primarily focus on disclosures and disclaimers rather than specific investment opportunities or risks related to companies or industries [2]

Core Insights - 2025 is a critical year for Abeona Therapeutics as its leading pipeline candidate, prademagene zamikeracel (pz-cel), is under regulatory review by the U.S. Food and Drug Administration [1] Company Overview - Abeona Therapeutics is focused on developing innovative therapies, particularly in the healthcare sector [1] Regulatory Context - The U.S. Food and Drug Administration is currently reviewing prademagene zamikeracel (pz-cel), which is a significant milestone for the company [1]

Pz-cel (Prademagene zamikeracel) for RDEB - Pz-cel is anticipated to receive potential FDA approval for RDEB with a PDUFA date of April 29, 2025 [4, 74] - Phase 3 VIITAL study showed 81.4% of pz-cel treated wounds achieved >50% healing compared to control wounds at Week 24 [12] - Phase 3 VIITAL study showed 65.1% of pz-cel treated wounds achieved >75% healing compared to control wounds at Week 24 [12] - Phase 3 VIITAL study showed 16.3% of pz-cel treated wounds achieved complete healing compared to control wounds at Week 24 [12] - Pz-cel demonstrated a mean pain reduction from baseline at Week 24 [12] - The company estimates a peak annual US revenue of over $500 million for pz-cel [41] - The company anticipates gross margins of approximately 85-90% after the initial ramp-up [42] Pipeline and Partnerships - Ultragenyx's BLA for UX111 (ABO-102) in MPS IIIA has a PDUFA date of August 18, 2025 [4, 74] - The company is developing ABO-503 for X-linked retinoschisis (XLRS), with first-in-human opportunity in 1H 2026 [62, 65] Financial Resources - The company had $110 million in cash resources as of September 30, 2024, providing a runway through potential launch into 2026 [71, 72]

Financial Data and Key Metrics Changes - As of December 31, 2024, the company had cash, cash equivalents, short-term investments, and restricted cash of $98.1 million, compared to $52.6 million as of December 31, 2023, indicating a significant increase in financial resources [37] - Research and development expenses for the full year ended December 31, 2024, were $34.4 million, up from $31.1 million in 2023 [39] - General and administrative expenses rose to $29.9 million for the full year ended December 31, 2024, compared to $19 million in 2023, primarily due to commercial launch preparation costs [39] - The net loss for the full year ended December 31, 2024, was $63.7 million, or $1.55 loss per common share, compared to a net loss of $54.2 million, or $2.53 loss per common share in 2023 [40] Business Line Data and Key Metrics Changes - The company is preparing for the potential launch of pz-cel for recessive dystrophic epidermolysis bullosa (RDEB), with expectations to activate treatment centers and begin patient biopsies in the third quarter of 2025, pending FDA approval [10][12] - The estimated market opportunity in the U.S. for pz-cel includes approximately 1,500 treatment opportunities, with a cumulative revenue potential of over $2 billion [16] Market Data and Key Metrics Changes - The company estimates that approximately 750 RDEB patients in the U.S. with moderate to severe wounds are potential candidates for pz-cel treatment [16] - The payer mix for RDEB treatment shows that 60% to 65% of lives are covered by commercial plans, 30% to 35% by Medicaid, and less than 10% by Medicare [27] Company Strategy and Development Direction - The company aims to launch pz-cel in the U.S. and is focused on ensuring favorable access policies and reimbursement for treatment centers [26] - Plans include ramping up manufacturing capacity to support 200-plus annual pz-cel treatments by the second half of 2027 [35] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming PDUFA date for pz-cel and the potential to transform the treatment paradigm for RDEB patients [12] - The company anticipates a gradual ramp-up in treatment center activity post-launch, with initial treatment of one to two patients as centers become accustomed to the pz-cel treatment process [24] Other Important Information - The company has received a priority review designation from the FDA for pz-cel, with a PDUFA action date of April 29, 2025 [7][14] - The company is also in discussions with Ultragenyx regarding a partnered program for Sanfilippo syndrome type A, with a BLA submitted to the FDA and a PDUFA date of August 18, 2025 [13] Q&A Session Summary Question: FDA satisfaction with CMC work - Management believes they have addressed all FDA requests from the previous CRL and feel confident about the current review process [44][45] Question: Patient eligibility at treatment centers - Management indicated that approximately 30% of the 750 RDEB patients are located in seven centers of excellence, with discussions ongoing to identify eligible patients [51] Question: Key reasons for seeking pz-cel therapy - The primary driver for patients is the need for durable wound closure, which can minimize infections and improve quality of life [55] Question: Draft label alignment with expectations - Management confirmed that the draft label received from the FDA aligns with their expectations, with no major surprises anticipated [63] Question: Anticipation of patient backlog post-approval - Management expects a patient backlog initially as manufacturing capacity ramps up, with a queue of patients anticipated [64] Question: Physician education needs - There is a need for physician education, particularly for community physicians who have not been engaged until approval is secured [71] Question: PRV expectations and recent trends - Management does not anticipate any factors that would preclude the granting of a PRV and aims to optimize pricing for any potential sale [82][85] Question: Market deployment outside the U.S. - Interest exists from European and Asian markets, but discussions will be more meaningful post-U.S. approval [89] Question: Total number of treatment centers planned - The company does not anticipate exceeding 10 treatment centers, focusing on building experience at each center [99]