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]

Corporate Presentation March 2025 Note regarding forward-looking statements This presentation of Abeona Therapeutics Inc. (the "Company") contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. Such forward looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 199 ...

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]

Financial Performance - Abeona Therapeutics reported a net loss of $63.7 million for the full year 2024, compared to a net loss of $54.2 million in 2023, representing a 17.5% increase in losses[7]. - Research and development expenses increased to $34.4 million in 2024 from $31.1 million in 2023, primarily due to increased headcount for manufacturing capacity expansion[7]. - General and administrative expenses rose to $29.9 million in 2024 from $19.0 million in 2023, reflecting increased costs related to commercial launch preparations[7]. Cash and Assets - Cash, cash equivalents, short-term investments, and restricted cash totaled $98.1 million as of December 31, 2024, up from $52.6 million as of December 31, 2023, indicating a 86.5% increase[5]. - The total assets of Abeona increased to $108.9 million as of December 31, 2024, compared to $64.0 million as of December 31, 2023[15]. - The company estimates its current resources are sufficient to fund operations into 2026, excluding potential revenue from pz-cel sales[6]. Product Development and Regulatory Updates - The FDA has set a PDUFA target action date of April 29, 2025, for the review of the pz-cel Biologics License Application (BLA)[3]. - Abeona anticipates treating the first patient with pz-cel in the third quarter of 2025, pending FDA approval[3]. - The company has engaged five treatment centers in the U.S. to become qualified treatment centers for pz-cel[3]. - Abeona has obtained two additional patents for pz-cel, extending patent protection for its use in treating RDEB to June 2037 and for its packaging system to July 2040[3].

FDA Approvals and Designations - The FDA accepted and granted priority review for the Biologics License Application (BLA) for pz-cel, with a target action date set for May 25, 2024[15]. - The FDA has granted pz-cel multiple designations, including Regenerative Medicine Advanced Therapy (RMAT) and Breakthrough Therapy, which may provide market exclusivity and expedited review benefits[36]. - The FDA reviews a Biologics License Application (BLA) within 60 days to determine if it is substantially complete before acceptance for filing[96]. - The FDA aims to review 90% of standard BLAs in 10 months and 90% of priority BLAs in 6 months after acceptance for filing[101]. - The FDA may expedite the review of BLAs through programs like Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval[104][105]. - Fast Track designation allows for rolling review of BLAs, enabling sections to be reviewed before the application is complete[104]. Clinical Development and Studies - The VIITAL™ study demonstrated statistically significant improvements in wound healing and pain reduction for pz-cel, with a mean duration of chronic wounds prior to treatment being 6.2 years[33]. - ABO-503 demonstrated robust RS1 expression in the retina, improved cone photoreceptor density, and overall photoreceptor cell survival in preclinical studies[39]. - A pre-IND meeting for ABO-503 with the FDA provided comprehensive feedback to support a future IND submission, with IND-enabling studies expected to complete by the end of 2025[39]. - ABO-504 aims to efficiently reconstitute the full-length ABCA4 gene using a dual AAV vector strategy, with preclinical data showing expression levels similar to endogenous ABCA4 in wild-type animals[42]. - A pre-IND meeting for ABO-504 with the FDA was conducted in June 2023, providing feedback to support a future IND submission[42]. - ABO-505 is designed to express a functional copy of human Opa1 in the retina, with initial efficacy results suggesting improved visual acuity in treated mutant mice[44]. Manufacturing and Facilities - The company has established cGMP manufacturing capabilities in Cleveland, Ohio, to support the planned commercial launch of pz-cel, if approved[37]. - The company has established a cGMP manufacturing facility in Cleveland, Ohio, enhancing supply chain control and manufacturing efficiency for clinical trials[49]. - The facility spans over 16,000 square feet, with the first phase completed in 2018 and the second phase in 2019, supporting quality control and process development[50]. Intellectual Property and Licensing - The company aims to expand its intellectual property portfolio by aggressively seeking patent rights for various aspects of its programs[22]. - The company has licensed technologies for the treatment of various diseases, including RDEB, with patents expected to expire in early 2037 and mid-2040[59]. - The company has licensed two patent families for treating CLN1 disease, with one U.S. patent (No. 11,504,435) issued on November 22, 2022, projected to expire on January 12, 2039[64]. - The company has licensed rights to a patent family from UNC related to gene therapy for Rett Syndrome, with patents expected to expire no earlier than 2039[65]. - The company owns a patent family for multipartite delivery of large transgenes using AAV vectors, with patents not expected to expire before 2041[66]. - The company has a patent family for novel AAV capsid proteins and treating ophthalmic diseases, with patents not expected to expire before 2042[67]. - A pending PCT application for treating dominant optic atrophy and X-linked retinoschisis is expected to have patents that do not expire before 2043[68]. Regulatory Compliance and Challenges - The FDA regulates biologic products, including gene therapy, under the FDCA and PHSA, requiring extensive testing and approval processes[69]. - The FDA's approval process for biologic products includes preclinical tests, IND submission, and clinical trials, with a focus on safety and efficacy[71]. - Manufacturers of biologics must comply with cGMP regulations and register with the FDA, ensuring consistent production quality[87]. - The approval process may involve inspections of manufacturing facilities to ensure compliance with cGMP requirements[98]. - The FDA has the authority to suspend licenses immediately if there is a danger to public health, and manufacturers must comply with strict manufacturing controls[109]. - Approved products are subject to annual user fee requirements, excluding orphan products, and manufacturers face periodic inspections for compliance with cGMP[110]. - Failure to comply with regulatory requirements can lead to severe consequences, including product recalls, fines, and exclusion from federal healthcare programs[115]. Market Access and Competition - The company is engaging with healthcare stakeholders to understand market access and potential pricing for pz-cel[37]. - The company faces significant uncertainty regarding coverage and reimbursement for its products, as third-party payors are increasingly reducing reimbursements for medical products and services[138]. - In the EU, pricing and reimbursement schemes vary widely, with some countries requiring a reimbursement price agreement before marketing products, leading to high barriers for new product entry[139]. - The company anticipates intense competition as new product candidates enter the market, with competitors potentially having greater financial and technical resources[144]. - Mergers and acquisitions in the biotechnology and pharmaceutical industries may concentrate resources among fewer competitors, increasing competitive pressure[145]. - The company may face challenges in obtaining regulatory approval for its product candidates, as competitors could achieve approval more rapidly, establishing a strong market position[148]. Employment and Workforce - As of December 31, 2024, the company had 136 full-time employees[153]. - The company has never experienced employment-related work stoppages and maintains good relations with its personnel[153]. - The company competes for skilled personnel with numerous pharmaceutical and biotechnology firms[152]. - The company has contracts with scientific consultants and research organizations to support drug development[153]. Legal and Regulatory Environment - The company must comply with various healthcare laws and regulations, including the federal Anti-Kickback Statute and the Physician Payments Sunshine Act, which may constrain its business arrangements[134]. - The General Data Protection Regulation (GDPR) imposes strict rules on data privacy and security, affecting how the company processes personal data[140]. - The company is subject to environmental protection laws that govern the use and disposal of hazardous substances, which could result in liability if operations lead to contamination[142].

Core Viewpoint - Abeona Therapeutics is making significant progress with its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) aimed at treating recessive dystrophic epidermolysis bullosa (RDEB), with a target action date set for April 29, 2025 [1][6]. Financial Results - For the full year 2024, Abeona reported a net loss of $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 [7][11]. - Research and development expenses increased to $34.4 million in 2024 from $31.1 million in 2023, primarily due to increased headcount for manufacturing capacity expansion [7][11]. - General and administrative expenses rose to $29.9 million in 2024 from $19.0 million in 2023, attributed to costs associated with commercial launch preparations [7][11]. Cash Position and Runway - As of December 31, 2024, the company had cash, cash equivalents, short-term investments, and restricted cash totaling $98.1 million, up from $52.6 million a year earlier [4][5]. - Abeona estimates that its current financial resources are sufficient to fund operations into 2026, not accounting for potential revenue from pz-cel sales or proceeds from a Priority Review Voucher (PRV) [5]. Regulatory and Development Updates - The FDA has accepted the BLA resubmission for pz-cel and discussions are ongoing regarding post-marketing requirements and the draft label [2][6]. - Abeona plans to treat its first patient with pz-cel in the third quarter of 2025, pending FDA approval [1][6]. - The company is actively preparing for commercialization, including onboarding treatment centers and engaging payers to ensure patient access [6]. Intellectual Property - Abeona has obtained two additional patents for pz-cel, extending patent protection for its use in treating RDEB to June 2037 and for its packaging and transport system to July 2040 [6]. Conference Call - A conference call to discuss financial results and company updates is scheduled for March 20, 2025, at 8:30 a.m. ET [8].

Core Insights - Abeona Therapeutics Inc. will have its CEO, Vish Seshadri, present at the Leerink Partners Global Healthcare Conference on March 10, 2025 [1] - A live webcast of the presentation will be available on the company's website and archived for 30 days [2] Company Overview - Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases [3] - The company is working on Prademagene zamikeracel (pz-cel), an investigational autologous, COL7A1 gene-corrected epidermal sheet for recessive dystrophic epidermolysis bullosa [3] - Abeona has a fully integrated cGMP manufacturing facility that supports the production of pz-cel for its Phase 3 VIITAL™ trial and future commercial production upon FDA approval [3] - The company also has AAV-based gene therapies targeting ophthalmic diseases with high unmet medical needs, utilizing next-generation AAV capsids to enhance tropism profiles [3]

Group 1 - Abeona Therapeutics Inc. (NASDAQ: ABEO) has a lead program called pz-cel (prademagene zamikeracel), which is an autologous, engineered cell therapy currently in development for recessive dystrophic epidermolysis bullosa [1] - The company has not been reviewed in detail for approximately six years, indicating a potential gap in investor awareness or interest [1] Group 2 - The Total Pharma Tracker offers tools for DIY investors, including a software that provides extensive curated research material for any ticker [1] - In-house experts are available to assist investors by identifying the best investable stocks along with buy/sell strategies and alerts [2]

Financial Performance - The net loss for the three months ended September 30, 2024, was $30.269 million, compared to a loss of $11.836 million for the same period in 2023, reflecting a 156% increase in losses[12]. - The net loss for the nine months ended September 30, 2024 and 2023, respectively, was $54.4 million and $37.6 million, with an accumulated deficit of approximately $804.0 million as of September 30, 2024[26]. - The basic and diluted loss per common share for the three months ended September 30, 2024, was $(0.63), compared to $(0.48) for the same period in 2023[12]. - The company has incurred significant operating losses and negative cash flows from operations since inception, which are expected to continue for the foreseeable future[25]. - The company has incurred recurring losses since inception and expects to continue generating operating losses for the foreseeable future[26]. Assets and Liabilities - As of September 30, 2024, total current assets increased to $112.657 million from $55.737 million as of December 31, 2023, representing a 102% increase[9]. - Total liabilities rose to $74.819 million from $49.176 million, marking a 52% increase[10]. - The total stockholders' equity increased significantly to $45.773 million from $14.826 million, representing a 208% increase[11]. - Total assets measured at fair value as of September 30, 2024, were $109,168 million, an increase from $72,305 million as of December 31, 2023[51]. - Total liabilities measured at fair value as of September 30, 2024, were $38,789 million, up from $31,352 million as of December 31, 2023[55]. Cash Flow and Investments - Cash and cash equivalents increased to $15.726 million as of September 30, 2024, from $14.473 million as of December 31, 2023, a rise of 9%[9]. - As of September 30, 2024, the Company had cash, cash equivalents, restricted cash, and short-term investments totaling $110.0 million, expected to fund operations for at least the next 12 months[26]. - The total cash, cash equivalents, and restricted cash at the end of the period is $16,064,000, compared to $5,050,000 at the end of the prior year[20]. - Total available-for-sale short-term investments amounted to $93.975 million as of September 30, 2024, with unrealized losses primarily due to changes in interest rates[42]. Research and Development - Research and development expenses for the nine months ended September 30, 2024, totaled $25.366 million, up from $23.712 million in the same period of 2023, indicating a 7% increase[12]. - The company is developing pz-cel, an autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB)[21]. - The reimbursement of development and transition costs from Ultragenyx is recognized as a reduction of research and development costs[109]. Stock and Equity - The company issued 12,285,056 shares of common stock in connection with an underwritten offering, net of offering costs, during the reporting period[16]. - The Company sold 1,902,376 shares under the ATM Agreement during the nine months ended September 30, 2024, resulting in net proceeds of $10.0 million[84]. - The Company sold 3,284,407 shares and pre-funded warrants for an aggregate purchase price of $25.0 million gross on July 6, 2023[89]. - The Company sold 12,285,056 shares and pre-funded warrants for an aggregate purchase price of $75.0 million gross on May 7, 2024[90]. Revenue and Licensing - The company reported no license and other revenues for the three months ended September 30, 2024, compared to $3.5 million for the same period in 2023[12]. - The Company recognized no revenue from the sublicense agreements for the three and nine months ended September 30, 2024 and 2023[103]. - The transaction price for the sublicense agreement related to CLN1 disease includes $7.0 million of fixed consideration and up to $56.0 million of variable consideration[102]. - Under the Ultragenyx License Agreement, the Company is eligible to receive tiered royalties from mid-single-digit up to 10% on net sales and up to $30.0 million in commercial milestone payments[108]. Risks and Future Outlook - The company has highlighted potential risks including the timing and outcome of the FDA's review of its Biologics License Application for pz-cel, which could materially affect performance[7]. - The Company resubmitted its Biologics License Application (BLA) for pz-cel on October 28, 2024, with a target action date of April 29, 2025, set by the FDA[110]. - The BLA resubmission follows a Type A meeting with the FDA in August 2024, aligning on additional information required for Chemistry Manufacturing and Controls (CMC)[111]. Operating Costs and Expenses - Total operating lease costs for the nine months ended September 30, 2024, were $1,308 million, a decrease from $1,379 million in the same period of 2023[68]. - The Company incurred stock-based compensation expense of $4,673,000 for the nine months ended September 30, 2023, compared to $3,254,000 for the same period in 2022[20]. - Total stock-based compensation expense for the three months ended September 30, 2024 was $1.804 million, compared to $1.557 million for the same period in 2023[95].

Financial Performance - Abeona Therapeutics reported a net loss of $30.3 million for Q3 2024, compared to a net loss of $11.8 million in Q3 2023, reflecting an increase in losses due to higher expenses[8]. - Abeona's total expenses for Q3 2024 were $15.3 million, compared to $11.3 million in Q3 2023, indicating a significant increase in operational costs[12]. - Research and development expenses for Q3 2024 were $8.9 million, up from $7.1 million in the same period of 2023, while general and administrative expenses rose to $6.4 million from $4.2 million[8]. - Total stockholders' equity increased to $45,773 million as of September 30, 2024, compared to $14,826 million as of December 31, 2023[15]. - Accumulated deficit rose to $(803,965) million from $(749,524) million[15]. - Total liabilities and stockholders' equity reached $120,592 million, up from $64,002 million[15]. - Additional paid-in capital increased to $849,388 million from $764,151 million[15]. - Accumulated other comprehensive loss increased to $(84) million from $(66) million[15]. - Total shares issued and outstanding remained at 26,523,878 as of September 30, 2024, unchanged from December 31, 2023[15]. Cash Position - As of September 30, 2024, the company had cash, cash equivalents, short-term investments, and restricted cash totaling $110.0 million, down from $123.0 million as of June 30, 2024[6]. - Abeona's current cash resources are estimated to fund operations into 2026, excluding potential revenue from pz-cel sales[7]. Product Development and Regulatory Progress - The FDA accepted the resubmission of Abeona's Biologics License Application (BLA) for pz-cel, with a target action date set for April 29, 2025[1][4]. - Abeona has made significant progress in commercial readiness for pz-cel, including discussions with payors and onboarding treatment centers[2][4]. - The Centers for Medicare and Medicaid Services (CMS) assigned a product-specific procedure code for pz-cel, facilitating higher reimbursement levels for the therapy[3]. - The company has received new patents related to pz-cel, which are expected to enhance its intellectual property position in the market[3]. Operational Expansion - The company entered a lease agreement for additional manufacturing space in Cleveland, Ohio, to expand its production capacity[3].