Abeona Therapeutics (ABEO) Q1 2025 Earnings Call May 15, 2025 07:00 AM ET Speaker0 Good day, and welcome to the Abeona Therapeutics First Quarter twenty twenty five Conference Call. At this time, all participants are in a listen only mode and a question and answer session will follow the formal presentation. Please note this conference is being recorded. I will now turn the conference over to your host, Mr. Greg Chin. Sir, you may begin. Speaker1 Thank you, Ali, and good morning, and thanks everyone for joi ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 FORM 10-Q (Mark one) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-15771 ABEONA THERAPEUTICS INC. (Exact name of registrant as specified in its charter) (State or Other Jurisdiction of incorporatio ...

Exhibit 99.1 Abeona Therapeutics Reports First Quarter 2025 Financial Results and Corporate Updates - Received approval by U.S. Food and Drug Administration (FDA) for ZEVASKYN™ (prademagene zamikeracel), the first and only autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB) - - U.S. launch of ZEVASKYN underway with activation of first qualified treatment center (QTC) - - Entered into sales agreement for priorit ...

CLEVELAND, May 15, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today reported financial results and business highlights for the first quarter of 2025 and shared recent operational progress. "ZEVASKYN's approval just a few weeks ago is a landmark achievement for recessive dystrophic epidermolysis bullosa patients and signifies Abeona's transition to a commercial-stage cell and gene therapy company," said Vish Seshadri, Chief Executive Officer of Abeona. "We are rapidly advancing the laun ...

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]

- Abeona Assist™ patient services program offers personalized support for eligible patients and families throughout their treatment journey with ZEVASKYN - - ZEVASKYN fills a critical need for people living with RDEB, a debilitating dermatological condition with no cure - - Approval based on the pivotal Phase 3 VIITAL™ study, showing ZEVASKYN resulted in significant wound healing and pain reduction after a single treatment with a favorable safety profile - - ZEVASKYN to be available through Qualified Treatm ...

Recessive Dystrophic Epidermolysis Bullosa (RDEB) is an extremely horrible disease. On average, 30% of the body is covered in chronic wounds (that don’t heal even after years). Some patients have as much as 80% of their body covered in wounds. ForAnalyst’s Disclosure: I/we have a beneficial long position in the shares of RCEL either through stock ownership, options, or other derivatives. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Se ...

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]