Abeona Therapeutics® Reports Full Year 2024 Financial Results, Provides Pz-cel Regulatory Update and Commercial Launch Plans
Abeona Therapeutics reported its full year 2024 financial results and provided updates on pz-cel regulatory progress. The FDA's priority review of pz-cel BLA is advancing with a PDUFA date of April 29, 2025, with the company targeting first patient treatment in Q3 2025 if approved.
Financial highlights include cash position of $98.1 million as of December 31, 2024, up from $52.6 million year-over-year. The company reported a net loss of $63.7 million ($1.55 per share) compared to $54.2 million ($2.53 per share) in 2023. R&D expenses increased to $34.4 million from $31.1 million, while G&A expenses rose to $29.9 million from $19.0 million.
The company has secured additional facility space in Cleveland for manufacturing expansion and obtained two patents extending protection for pz-cel treatment to 2037 and transport system to 2040. Current cash runway is expected to fund operations into 2026.
Abeona Therapeutics ha riportato i risultati finanziari per l'intero anno 2024 e ha fornito aggiornamenti sul progresso normativo di pz-cel. La revisione prioritaria da parte della FDA del BLA di pz-cel sta avanzando con una data PDUFA fissata per il 29 aprile 2025, con l'azienda che punta a iniziare il trattamento del primo paziente nel terzo trimestre del 2025, se approvato.
I punti salienti finanziari includono una posizione di cassa di 98,1 milioni di dollari al 31 dicembre 2024, in aumento rispetto ai 52,6 milioni di dollari dell'anno precedente. L'azienda ha riportato una perdita netta di 63,7 milioni di dollari (1,55 dollari per azione) rispetto ai 54,2 milioni di dollari (2,53 dollari per azione) nel 2023. Le spese per R&S sono aumentate a 34,4 milioni di dollari rispetto ai 31,1 milioni di dollari, mentre le spese generali e amministrative sono salite a 29,9 milioni di dollari rispetto ai 19,0 milioni di dollari.
L'azienda ha assicurato ulteriore spazio per le strutture a Cleveland per l'espansione della produzione e ha ottenuto due brevetti che estendono la protezione per il trattamento pz-cel fino al 2037 e per il sistema di trasporto fino al 2040. Si prevede che l'attuale liquidità finanziaria possa sostenere le operazioni fino al 2026.
Abeona Therapeutics informó sobre sus resultados financieros del año completo 2024 y proporcionó actualizaciones sobre el progreso regulatorio de pz-cel. La revisión prioritaria de la FDA del BLA de pz-cel está avanzando con una fecha PDUFA del 29 de abril de 2025, con la empresa apuntando a iniciar el tratamiento del primer paciente en el tercer trimestre de 2025, si se aprueba.
Los aspectos financieros destacados incluyen una posición de efectivo de 98,1 millones de dólares al 31 de diciembre de 2024, un aumento con respecto a los 52,6 millones de dólares del año anterior. La empresa reportó una pérdida neta de 63,7 millones de dólares (1,55 dólares por acción) en comparación con los 54,2 millones de dólares (2,53 dólares por acción) en 2023. Los gastos de I+D aumentaron a 34,4 millones de dólares desde 31,1 millones de dólares, mientras que los gastos generales y administrativos aumentaron a 29,9 millones de dólares desde 19,0 millones de dólares.
La empresa ha asegurado espacio adicional para instalaciones en Cleveland para la expansión de la fabricación y ha obtenido dos patentes que extienden la protección para el tratamiento de pz-cel hasta 2037 y para el sistema de transporte hasta 2040. Se espera que la actual liquidez financie las operaciones hasta 2026.
Abeona Therapeutics는 2024년 전체 연도 재무 결과를 보고하고 pz-cel 규제 진행 상황에 대한 업데이트를 제공했습니다. pz-cel BLA에 대한 FDA의 우선 심사가 진행 중이며 PDUFA 날짜는 2025년 4월 29일로 설정되어 있으며, 회사는 승인이 이루어질 경우 2025년 3분기에 첫 환자 치료를 목표로 하고 있습니다.
재무 하이라이트에는 2024년 12월 31일 기준 현금 보유액이 9,810만 달러로, 전년 대비 5,260만 달러에서 증가했습니다. 회사는 2023년 5,420만 달러(주당 2.53달러)에 비해 6,370만 달러의 순손실(주당 1.55달러)을 보고했습니다. 연구개발(R&D) 비용은 3,440만 달러로 증가했으며, 일반 및 관리(G&A) 비용은 2,990만 달러로 증가했습니다.
회사는 제조 확장을 위해 클리블랜드에 추가 시설 공간을 확보하고 pz-cel 치료에 대한 보호를 2037년까지, 운송 시스템에 대한 보호를 2040년까지 연장하는 두 개의 특허를 취득했습니다. 현재 현금 유동성은 2026년까지 운영 자금을 지원할 것으로 예상됩니다.
Abeona Therapeutics a publié ses résultats financiers pour l'année complète 2024 et a fourni des mises à jour sur les progrès réglementaires de pz-cel. L'examen prioritaire de la FDA concernant le BLA de pz-cel avance, avec une date PDUFA fixée au 29 avril 2025, l'entreprise visant à traiter le premier patient au troisième trimestre 2025 si approuvé.
Les points forts financiers incluent une position de trésorerie de 98,1 millions de dollars au 31 décembre 2024, en hausse par rapport à 52,6 millions de dollars l'année précédente. L'entreprise a enregistré une perte nette de 63,7 millions de dollars (1,55 dollar par action) par rapport à 54,2 millions de dollars (2,53 dollars par action) en 2023. Les dépenses de R&D ont augmenté à 34,4 millions de dollars contre 31,1 millions de dollars, tandis que les dépenses générales et administratives ont augmenté à 29,9 millions de dollars contre 19,0 millions de dollars.
L'entreprise a sécurisé un espace supplémentaire à Cleveland pour l'expansion de la fabrication et a obtenu deux brevets prolongeant la protection du traitement pz-cel jusqu'en 2037 et du système de transport jusqu'en 2040. La liquidité actuelle devrait financer les opérations jusqu'en 2026.
Abeona Therapeutics hat seine Finanzzahlen für das gesamte Jahr 2024 veröffentlicht und Updates zum regulatorischen Fortschritt von pz-cel bereitgestellt. Die priorisierte Überprüfung des pz-cel BLA durch die FDA schreitet voran, mit einem PDUFA-Datum vom 29. April 2025, wobei das Unternehmen plant, die Behandlung des ersten Patienten im dritten Quartal 2025 zu beginnen, sofern genehmigt.
Zu den finanziellen Höhepunkten gehört eine Liquiditätsposition von 98,1 Millionen Dollar zum 31. Dezember 2024, ein Anstieg von 52,6 Millionen Dollar im Jahresvergleich. Das Unternehmen berichtete von einem Nettoverlust von 63,7 Millionen Dollar (1,55 Dollar pro Aktie) im Vergleich zu 54,2 Millionen Dollar (2,53 Dollar pro Aktie) im Jahr 2023. Die F&E-Ausgaben stiegen auf 34,4 Millionen Dollar von 31,1 Millionen Dollar, während die allgemeinen und administrativen Ausgaben auf 29,9 Millionen Dollar von 19,0 Millionen Dollar anstiegen.
Das Unternehmen hat zusätzlichen Platz in Cleveland für die Herstellungserweiterung gesichert und zwei Patente erhalten, die den Schutz für die pz-cel-Behandlung bis 2037 und für das Transportsystem bis 2040 verlängern. Es wird erwartet, dass die aktuelle Liquidität die Betriebe bis 2026 finanziert.
- Cash position increased to $98.1M from $52.6M year-over-year
- Two new patents secured extending protection until 2037 and 2040
- FDA priority review progress with PDUFA date set
- Sufficient cash runway into 2026
- Net loss increased to $63.7M from $54.2M year-over-year
- R&D expenses increased 10.6% to $34.4M
- G&A expenses rose 57.4% to $29.9M
Insights
Abeona Therapeutics' financial results and regulatory update signal a pivotal moment for the company as it approaches the potential commercialization of pz-cel for RDEB. The FDA review is progressing on schedule with a PDUFA date of April 29, 2025, and the receipt of draft labeling suggests the review is in advanced stages.
The company reported
Net losses widened to
Beyond financial metrics, the company has secured patent protection for pz-cel until 2037/2040, enhancing long-term value prospects. Additionally, Abeona may receive a Priority Review Voucher upon approval, which could be monetized for
Abeona's regulatory progress for pz-cel represents significant advancement toward addressing the substantial unmet need in recessive dystrophic epidermolysis bullosa (RDEB) - a rare, devastating genetic condition with treatment options. The ongoing FDA discussions regarding post-marketing requirements and draft labeling indicate the review is in its final stages, with potential approval by the April 29th PDUFA date.
The company's commercial preparation strategy appears comprehensive, with five geographically dispersed qualified treatment centers being onboarded. This approach aligns with the rare disease commercial model where specialized centers of excellence are critical for patient access and treatment delivery. The expansion of manufacturing capacity in Cleveland demonstrates confidence in potential commercial demand while addressing supply chain resilience.
Beyond pz-cel, Ultragenyx's progress with UX111 (formerly Abeona's ABO-102) for Sanfilippo syndrome type A adds a second potential value driver. The FDA's acceptance and Priority Review designation (PDUFA: August 18, 2025) for this asset validates Abeona's original development approach and suggests potential milestone payments if approved.
The concurrent advancement of both therapies under Priority Review designation demonstrates the FDA's recognition of the significant unmet needs in these rare genetic conditions. While pz-cel is positioned as a transformative potential therapy that could provide long-lasting healing for chronic RDEB wounds, the company's strategy to establish treatment infrastructure before approval increases the likelihood of successful commercialization if approved.
FDA priority review of pz-cel Biologics License Application (BLA) progressing with Prescription Drug User Fee Act (PDUFA) target action date of April 29, 2025
Launch preparations on track toward goal to treat first patient in 3Q 2025
CLEVELAND, March 20, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today reported financial results for the full year of 2024, and announced progress in the ongoing review with the U.S. Food and Drug Administration (FDA) regarding prademagene zamikeracel (pz-cel) and plans for the U.S. commercial launch of pz-cel for recessive dystrophic epidermolysis bullosa (RDEB), if approved. The Company will host a conference call and webcast today at 8:30 a.m. ET.
“The RDEB community continues to highlight the unmet medical need for new therapies that could provide long-lasting healing and pain reduction, even in tough large, chronic RDEB wounds,” said Vish Seshadri, Chief Executive Officer of Abeona. “With a PDUFA date of April 29, 2025, the BLA review by the FDA is on track as discussions are underway regarding post-marketing requirements and commitments as well as the draft label.”
Fourth Quarter and Recent Progress
Pz-cel for RDEB
- Abeona continues to work with the FDA to finalize the review of its Biologics License Application (BLA) for pz-cel as a potential new treatment for patients with RDEB. In November 2024, the FDA accepted the BLA resubmission for review and set a PDUFA target action date of April 29, 2025. On March 14, 2025, Abeona received draft United States Prescribing Information (USPI) from the FDA to initiate discussion on the label for pz-cel. Discussions are also ongoing with the FDA on post-approval marketing requirements and commitments for pz-cel. If approved, the Company anticipates the first patient treatment with pz-cel in the third quarter of 2025. Abeona may be eligible for a Priority Review Voucher (PRV) should pz-cel be approved.
- In anticipation of potential FDA approval of pz-cel, Abeona continues its commercialization efforts, including progress toward onboarding five geographically dispersed, well-recognized epidermolysis bullosa treatment centers in the U.S. as pz-cel qualified treatment centers (QTCs), engaging payers to ensure patient access, and educating key stakeholders.
- In preparation for potential pz-cel launch, Abeona has hired and trained personnel to support commercialization, manufacturing, supply chain and quality.
- Abeona entered into a lease agreement for additional facility space in Cleveland, Ohio to enable expansion of manufacturing capacity for pz-cel beyond the current planned manufacturing footprint.
- Obtained two additional patents from the United States Patent and Trademark Office for pz-cel, extending patent protection on the use of pz-cel for the treatment of RDEB to June 2037 and patent protection on the packaging and transport system for pz-cel to July 2040.
Pipeline and partnered programs
- In December 2024, Ultragenyx submitted a BLA to the FDA seeking accelerated approval for UX111 (formerly ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). In February 2025, Ultragenyx announced FDA acceptance and Priority Review of the UX111 BLA with a PDUFA date of August 18, 2025.
Full Year 2024 Financial Results and Cash Runway Guidance
Cash, cash equivalents, short-term investments and restricted cash totaled
Abeona estimates that its current cash and cash equivalents, short-term investments and restricted cash, as well as its credit facility, are sufficient resources to fund operations into 2026, before accounting for any potential revenue from commercial sales of pz-cel, if approved, or proceeds from the sale of a PRV, if awarded by the FDA.
Research and development expenses for the full year ended December 31, 2024 were
Conference Call Details
The Company will host a conference call and webcast on Thursday, March 20, 2025, at 8:30 a.m. ET, to discuss the financial results and company updates. To access the call, dial 877-545-0523 (U.S. toll-free) or 973-528-0016 (international) and Entry Code: 712069 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona’s website at https://investors.abeonatherapeutics.com/events. The archived webcast replay will be available for 30 days following the call.
About Abeona Therapeutics
Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Prademagene zamikeracel (pz-cel) is Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets currently in development for recessive dystrophic epidermolysis bullosa. The Company’s fully integrated cell and gene therapy cGMP manufacturing facility served as the manufacturing site for pz-cel used in its Phase 3 VIITAL™ trial, and is capable of supporting commercial production of pz-cel upon FDA approval. The Company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona’s novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. For more information, visit www.abeonatherapeutics.com.
Forward-Looking Statements
This press release 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. We have attempted to identify forward-looking statements by such terminology as “may,” “will,” “believe,” “anticipate,” “expect,” “intend,” “potential,” and similar words and expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to, the timing and outcome of the FDA’s review of our BLA resubmission for pz-cel; our plans to continue development of AAV-based gene therapies designed to treat ophthalmic diseases; the FDA’s grant of a Priority Review Voucher (“PRV”) in connection with the pz-cel BLA; the achievement of or expected timing, progress and results of clinical development, clinical trials and potential regulatory approvals; our pipeline of product candidates; our belief that pz-cel could potentially benefit patients with RDEB; our belief in the adequacy of the clinical trial data from our VIITAL™ clinical trial, together with the data generated in the pz-cel program to date, to support pz-cel’s regulatory approval; our dependence upon our third-party customers and vendors and their compliance with regulatory bodies; our estimates regarding expenses, future revenues, capital requirements, and needs for additional financing; our intellectual property position and our ability to obtain, maintain and enforce intellectual property protection and exclusivity for our proprietary assets; our estimates regarding the size of the potential markets for our product candidates, the strength of our commercialization strategies and our ability to serve and supply those markets; and future economic conditions or performance; risks associated with data analysis and reporting; and other risks disclosed in the Company’s most recent Annual Report on Form 10-K and subsequent periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.
| ABEONA THERAPEUTICS INC. AND SUBSIDIARIES Condensed Consolidated Statements of Operations and Comprehensive Loss ($ in thousands, except share and per share amounts) | ||||||||
| For the years ended December 31, | ||||||||
| 2024 | 2023 | |||||||
| Revenues: | ||||||||
| License and other revenues | $ | — | $ | 3,500 | ||||
| Expenses: | ||||||||
| Royalties | — | 1,605 | ||||||
| Research and development | 34,360 | 31,091 | ||||||
| General and administrative | 29,851 | 19,004 | ||||||
| Gain on operating lease right-of-use assets | — | (1,065 | ) | |||||
| Total expenses | 64,211 | 50,635 | ||||||
| Loss from operations | (64,211 | ) | (47,135 | ) | ||||
| Interest income | 4,246 | 2,117 | ||||||
| Interest expense | (4,208 | ) | (418 | ) | ||||
| Change in fair value of warrant and derivative liabilities | (755 | ) | (11,695 | ) | ||||
| Other income | 1,194 | 2,943 | ||||||
| Net loss | $ | (63,734 | ) | $ | (54,188 | ) | ||
| Basic and diluted loss per common share | $ | (1.55 | ) | $ | (2.53 | ) | ||
| Weighted average number of common shares outstanding - basic and diluted | 41,048,206 | 21,380,476 | ||||||
| Other comprehensive income (loss): | ||||||||
| Change in unrealized gains related to available-for-sale debt securities | 74 | 34 | ||||||
| Foreign currency translation adjustments | — | 29 | ||||||
| Comprehensive loss | $ | (63,660 | ) | $ | (54,125 | ) | ||
| ABEONA THERAPEUTICS INC. AND SUBSIDIARIES Condensed Consolidated Balance Sheets ($ in thousands, except share and per share amounts) | ||||||||
| December 31, 2024 | December 31, 2023 | |||||||
| ASSETS | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 23,357 | $ | 14,473 | ||||
| Short-term investments | 74,363 | 37,753 | ||||||
| Restricted cash | 338 | 338 | ||||||
| Other receivables | 1,652 | 2,444 | ||||||
| Prepaid expenses and other current assets | 1,143 | 729 | ||||||
| Total current assets | 100,853 | 55,737 | ||||||
| Property and equipment, net | 4,430 | 3,533 | ||||||
| Operating lease right-of-use assets | 3,552 | 4,455 | ||||||
| Other assets | 96 | 277 | ||||||
| Total assets | $ | 108,931 | $ | 64,002 | ||||
| LIABILITIES AND STOCKHOLDERS’ EQUITY | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 3,441 | $ | 1,858 | ||||
| Accrued expenses | 6,333 | 5,985 | ||||||
| Current portion of long-term debt | 5,926 | — | ||||||
| Current portion of operating lease liability | 823 | 998 | ||||||
| Current portion payable to licensor | — | 4,580 | ||||||
| Other current liabilities | 64 | 1 | ||||||
| Total current liabilities | 16,587 | 13,422 | ||||||
| Long-term operating lease liabilities | 3,262 | 4,402 | ||||||
| Long-term debt | 13,037 | — | ||||||
| Warrant liabilities | 32,014 | 31,352 | ||||||
| Total liabilities | 64,900 | 49,176 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders’ equity: | ||||||||
| Preferred stock - | — | — | ||||||
| Common stock - | 457 | 265 | ||||||
| Additional paid-in capital | 856,824 | 764,151 | ||||||
| Accumulated deficit | (813,258 | ) | (749,524 | ) | ||||
| Accumulated other comprehensive loss | 8 | (66 | ) | |||||
| Total stockholders’ equity | 44,031 | 14,826 | ||||||
| Total liabilities and stockholders’ equity | $ | 108,931 | $ | 64,002 | ||||
Investor and Media Contact: Greg Gin VP, Investor Relations and Corporate Communications Abeona Therapeutics ir@abeonatherapeutics.com
FAQ
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