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].

Abeona Therapeutics (ABEO) Q4 2024 Earnings Call March 20, 2025 07:30 AM ET Company Participants Greg Gin - Vice President, Investor Relations and Corporate CommunicationsVishwas Seshadri - Chief Executive OfficerMadhav Vasanthavada - Senior VP, Chief Commercial Officer & Head of Business DevelopmentBrian Kevany - Senior VP, CTO & CSOJoseph Vazzano - Chief Financial OfficerRaghuram Selvaraju - Managing Director, Healthcare Equity Research Conference Call Participants Kristen Kluska - Equity Research Analyst ...

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].

Core Viewpoint - The FDA has accepted the Biologics License Application (BLA) resubmission for pz-cel, targeting a PDUFA action date of April 29, 2025, indicating significant progress towards potential commercialization of the therapy for recessive dystrophic epidermolysis bullosa (RDEB) [1][2][3]. Company Progress - Abeona Therapeutics has made strides in commercial readiness for pz-cel, including discussions with payors and onboarding treatment centers [2][3]. - The company completed a Type A meeting with the FDA in August 2024, aligning on the BLA resubmission content and addressing Chemistry Manufacturing and Controls (CMC) issues noted in a previous Complete Response Letter [3]. - The Centers for Medicare and Medicaid Services (CMS) granted a product-specific procedure code for pz-cel and assigned it to a high reimbursement category, facilitating hospital billing and patient access [3]. - Abeona has expanded its manufacturing capacity with a new facility lease in Cleveland, Ohio, and received new patents related to pz-cel, with the '504 Patent expiring on January 3, 2037 [3][4]. Financial Results - As of September 30, 2024, the company reported cash and cash equivalents totaling $110.0 million, down from $123.0 million as of June 30, 2024 [4]. - Research and development expenses for Q3 2024 were $8.9 million, an increase from $7.1 million in Q3 2023, while general and administrative expenses rose to $6.4 million from $4.2 million in the same period [6]. - The net loss for Q3 2024 was $30.3 million, compared to a net loss of $11.8 million in Q3 2023 [6][10]. Pipeline and Partnerships - In July 2024, Abeona entered a non-exclusive agreement with Beacon Therapeutics to evaluate its AAV204 capsid for potential use in ophthalmology gene therapies [3]. - Ultragenyx participated in a pre-BLA meeting with the FDA regarding its partnered program UX111 AAV gene therapy for Sanfilippo syndrome type A, expected to be filed by the end of 2024 [3].

Core Viewpoint - Abeona Therapeutics Inc. has announced the acceptance of its Biologics License Application (BLA) resubmission for prademagene zamikeracel (pz-cel) by the FDA, targeting a decision date of April 29, 2025, for its investigational gene therapy for recessive dystrophic epidermolysis bullosa (RDEB) [1][2] Company Overview - Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, with pz-cel being a key investigational product for RDEB [5] - The company operates a fully integrated cGMP manufacturing facility capable of supporting commercial production of pz-cel upon FDA approval [5] Product Details - Prademagene zamikeracel (pz-cel) is an autologous, COL7A1 gene therapy designed to treat RDEB, a rare genetic skin disease caused by mutations in the COL7A1 gene, leading to a lack of functional collagen VII [4] - Pz-cel is derived from patients' own skin cells, which are genetically corrected to express collagen VII, and expanded into keratinocyte sheets for wound coverage in a single surgical application [4] - The BLA resubmission is backed by clinical efficacy and safety data from the pivotal Phase 3 VIITAL™ study and a Phase 1/2a study, with follow-up data extending up to 8 years [2] Regulatory Status - The FDA had previously accepted the BLA for pz-cel for Priority Review, and the company may be eligible for a Priority Review Voucher if pz-cel is approved [3]