Abeona Therapeutics Reports Full Year 2023 Financial Results and Announces Completion of FDA Inspections
Abeona Therapeutics Inc. reported financial results for 2023, with the FDA completing a Pre-License Inspection of its Cleveland manufacturing facility for pz-cel BLA. The FDA also inspected clinical trial sites for the pivotal Phase 3 VIITAL™ study, with no Form 483 observations noted. The FDA's review of the pz-cel BLA is ongoing, targeting a PDUFA date of May 25, 2024. Abeona also secured a $50 million credit facility, with cash runway guidance into Q1 2025. Full-year 2023 financial results showed $52.6 million in cash and investments, with $3.5 million in license and other revenues. Research and development expenses were $31.1 million, leading to a net loss of $54.2 million for 2023.
Positive
FDA completed Pre-License Inspection of Abeona's Cleveland manufacturing facility for pz-cel BLA.
FDA inspected clinical trial sites for the pivotal Phase 3 VIITAL™ study with no Form 483 observations.
FDA's ongoing review of the pz-cel BLA targets a PDUFA date of May 25, 2024.
Abeona secured a $50 million credit facility and has cash runway guidance into Q1 2025.
Full-year 2023 financial results showed $52.6 million in cash and investments, with $3.5 million in revenues.
Research and development expenses were $31.1 million, leading to a net loss of $54.2 million for 2023.
The completion of the FDA's Pre-License Inspection (PLI) of Abeona Therapeutics' manufacturing facility and the clinical trial sites for the pivotal Phase 3 VIITAL™ study is a significant milestone for the company. The PLI is a critical step in the FDA's Biologics License Application (BLA) review process for new therapies. The FDA's issuance of a Form 483 listing observations related to process controls at the Cleveland facility indicates areas that require remediation. However, the company's prompt response to the FDA, detailing implemented and ongoing corrective actions, suggests a proactive approach to compliance.
The lack of Form 483 observations at the clinical study sites is a positive indicator of adherence to Good Clinical Practice (GCP) standards and may bolster the BLA's prospects. The FDA's current stance of not planning an Advisory Committee meeting and not anticipating Risk Evaluation and Mitigation Strategies (REMS) at this time could streamline the review process for pz-cel, potentially leading to a faster market entry, subject to approval.
Investors should note that the company's commercial preparations, including payer engagement and market research, indicate confidence in the potential approval and market viability of pz-cel. The financial arrangement with Avenue Venture Opportunities Fund, L.P., providing a $50 million credit facility, demonstrates external confidence in Abeona's strategy and offers financial stability through the anticipated PDUFA date and beyond.
Abeona's financial results for the full year of 2023, with a total of $52.6 million in cash, cash equivalents, restricted cash and short-term investments, reflect a stable financial position. The net cash used in operating activities has decreased from the previous year, indicating improved cash management. The revenue from license and other sources, although modest, shows an upward trend year-over-year, which is a positive sign for the company's revenue diversification efforts.
The R&D and G&A expenses have increased, which is expected given the company's investment in BLA activities and preparation for the potential launch of pz-cel. The net loss attributable to common shareholders has also increased, but this is offset by a lower loss per common share due to changes in the weighted average number of shares outstanding.
The company's cash runway extension into the first quarter of 2025, before potential revenue from pz-cel or Priority Review Voucher sales, provides a cushion for Abeona to navigate the regulatory process and initial commercialization phase without immediate financial pressures. This timeline also allows investors to gauge the market response to pz-cel post-launch, should it receive FDA approval.
The potential approval and commercial launch of pz-cel for recessive dystrophic epidermolysis bullosa (RDEB) represent a significant opportunity for Abeona Therapeutics in a niche market with high unmet medical needs. RDEB is a rare genetic condition and current treatment options are limited, meaning pz-cel could effectively become a standard of care if proven effective and safe.
Market research supporting reimbursement coverage at a price in line with approved gene therapies indicates a viable pricing strategy that could facilitate payer acceptance and patient access. The one-time application nature of gene therapies like pz-cel often commands a higher price point due to the potential for long-term benefits, which could translate into substantial long-term revenue for Abeona if commercialization is successful.
However, the market adoption of pz-cel will depend on various factors, including clinical efficacy, safety profile, competition from other therapies and the ability of healthcare systems to provide access to such advanced treatments. Ongoing payer engagement and market research are important for understanding the market dynamics and ensuring successful product launch and adoption.
03/18/2024 - 07:30 AM
- FDA completed Pre-License Inspection of Abeona’s Cleveland manufacturing facility, as well as inspection of clinical trial sites for pz-cel pivotal Phase 3 VIITAL™ study -
CLEVELAND, March 18, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today reported financial results for the full year of 2023 and provided an update on progress toward achieving key corporate objectives.
Abeona also announced today that the U.S. Food and Drug Administration (FDA) has completed a Pre-License Inspection (PLI) of its Cleveland, Ohio manufacturing facility related to the Company’s Biologics License Application (BLA) for pz-cel (prademagene zamikeracel) for recessive dystrophic epidermolysis bullosa (RDEB). During the inspection, the FDA reviewed the facilities, systems, and processes at the Cleveland site. The FDA also observed the manufacturing process for pz-cel, as well as performance of in-process and release assays. The two-week PLI, which was conducted by five FDA inspectors, concluded on March 1, 2024. Upon completion of the inspection, a Form 483 was issued with observations related to process controls. On March 15, 2024, the Company submitted a response to the FDA, outlining already implemented and ongoing steps toward resolution that follow FDA guidance provided during the audit. In addition, the FDA has completed the clinical study site inspections of Stanford University School of Medicine in Palo Alto, CA and University of Massachusets Medical School in Worcester, MA, both of which enrolled subjects in the pivotal Phase 3 VIITAL™ study supporting the pz-cel BLA, with no Form 483 observations noted. The FDA’s review of Abeona’s pz-cel BLA is ongoing, with a target Prescription Drug User Fee Act (PDUFA) date of May 25, 2024, and the FDA does not currently plan to conduct an Advisory Committee.
“We appreciate the FDA’s collaborative conduct of the PLI, and look forward to working with them through the remainder of the BLA review.” said Vish Seshadri, Chief Executive Officer of Abeona.
Fourth Quarter and Recent Progress
Pz-cel for RDEB
In November 2023, the FDA accepted and granted Priority Review for the Company’s BLA for pz-cel for RDEB with a target PDUFA date of May 25, 2024. The FDA also advised that it does not plan to convene an Advisory Committee meeting to discuss the pz-cel application. In January 2024, the FDA completed both a Bioresearch Monitoring (BIMO) inspection of Abeona and the Mid-Cycle Meeting regarding the pz-cel BLA. Following completion of the BIMO inspection, the FDA inspector did not issue any observations or FDA Form 483s. At the BLA Mid-Cycle Review meeting, the FDA reaffirmed that it does not currently plan to convene an Advisory Committee for pz-cel. In addition, the FDA advised that Risk Evaluation and Mitigation Strategies (REMS) are not anticipated for the pz-cel BLA at this time, though review is ongoing, and reconfirmed the target PDUFA date of May 25, 2024. In March 2024, Abeona received a written Establishment Inspection Report (EIR), the formal report from the FDA regarding the BIMO inspection. The results of the inspection determined there were no deficiencies with the monitoring or management of study sites, IP accountability, retention of study site documents, or safety oversight. U.S. commercial launch preparations for pz-cel
Abeona continues to advance key commercial activities in preparation for a potential U.S. launch for pz-cel, including onboarding discussions with EB treatment sites, payer engagement, hiring key commercial roles, and conducting market research. The Company recently completed payer market research that supports reimbursement coverage of pz-cel at a price commensurate with the value of approved gene therapies that offer years of durable benefit following a one-time application. Corporate highlights
In January 2024, Abeona entered a $50 million credit facility with the Avenue Venture Opportunities Fund, L.P. The credit agreement includes a first tranche of $20 million , which was funded in January 2024, a second tranche of $10 million of committed capital, and an option for an additional $20 million upon satisfaction of certain terms and conditions. Proceeds from the facility are intended to support the Company’s ongoing preparations for launch and commercialization in anticipation of a potential FDA approval of pz-cel. Full Year 2023 Financial Results and Cash Runway Guidance
Cash, cash equivalents, restricted cash and short-term investments totaled $52.6 million as of December 31, 2023, compared to $52.5 million as of December 31, 2022. Net cash used in operating activities was $37.0 million for the full year of 2023, compared to $43.5 million in the full year of 2022.
In January 2024, Abeona received a first tranche of $20 million as part of the credit facility with Avenue Venture Opportunities Fund, L.P. Abeona estimates that its current cash and cash equivalents, restricted cash and short-term investments, as well as the credit facility with Avenue Venture Opportunities Fund, L.P, are sufficient resources to fund operations into the first quarter of 2025 before accounting for any revenue from commercial sales of pz-cel, if approved, or proceeds from the sale of a Priority Review Voucher, if awarded by the FDA.
License and other revenues for the year ended December 31, 2023 were $3.5 million , as compared to $1.4 million for the same period of 2022. The revenues in both years primarily represent clinical milestone payments under a licensing agreement with Taysha Gene Therapies for investigational AAV-based gene therapy for Rett syndrome.
Research and development expenses for the full year ended December 31, 2023 were $31.1 million , compared to $29.0 million for the full year ended December 31, 2022, primarily a result of increased headcount related to BLA activities. General and administrative expenses were $19.0 million for the full year ended December 31, 2023, compared to $17.3 million for the year ended December 31, 2022, primarily a result of increased headcount for the potential launch of pz-cel. Net loss attributable to common shareholders for the full year ended December 31, 2023 was $54.2 million , or $2.53 loss per common share as compared to $43.5 million , or $5.53 loss per common share, for the full year of 2022.
Conference Call Details
Abeona Therapeutics will host a conference call and webcast today, March 18, 2024, at 8:30 a.m. ET. To access the call, dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) and Entry Code: 428606 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed at https://investors.abeonatherapeutics.com/events . The archived webcast replay will be available for 30 days following the call.
About Abeona Therapeutics 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 our Biologics License Application submission to the FDA for pz-cel; the FDA’s grant of a Priority Review Voucher; continued interest in our rare disease portfolio; our ability to enroll patients in clinical trials; the outcome of future meetings with and inspections from the FDA or other regulatory agencies, including those relating to preclinical programs; the ability to achieve or obtain necessary regulatory approvals; the impact of any changes in the financial markets and global economic conditions; 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 For the years ended December 31, 2023 2022 Revenues: License and other revenues $ 3,500 $ 1,414 Expenses: Royalties 1,605 450 Research and development 31,091 28,965 General and administrative 19,004 17,256 Impairment of licensed technology — 1,355 Loss/(gain) on operating lease right-of-use assets (1,065 ) 2,511 Impairment of construction-in-progress — 1,792 Total expenses 50,635 52,329 Loss from operations (47,135 ) (50,915 ) Interest income 2,117 431 Interest expense (418 ) (736 ) Change in fair value of warrant liabilities (11,695 ) 11,383 Other income 2,943 141 Net loss $ (54,188 ) $ (39,696 ) Deemed dividends related to Series A and Series B Convertible Redeemable Preferred Stock — (3,782 ) Net loss attributable to Common Shareholders $ (54,188 ) $ (43,478 ) Basic and diluted loss per common share $ (2.53 ) $ (5.53 ) Weighted average number of common shares outstanding – basic and diluted 21,380,476 7,861,515 Other comprehensive income (loss): Change in unrealized gains (losses) related to available-for-sale debt securities 34 (99 ) Foreign currency translation adjustments 29 (3 ) Comprehensive loss $ (54,125 ) $ (43,580 )
ABEONA THERAPEUTICS INC. AND SUBSIDIARIES December 31, 2023 December 31, 2022 ASSETS Current assets: Cash and cash equivalents $ 14,473 $ 14,217 Short-term investments 37,753 37,932 Restricted cash 338 338 Other receivables 2,444 188 Prepaid expenses and other current assets 729 424 Total current assets 55,737 53,099 Property and equipment, net 3,533 5,741 Operating lease right-of-use assets 4,455 5,331 Other assets 277 43 Total assets $ 64,002 $ 64,214 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities: Accounts payable $ 1,858 $ 1,811 Accrued expenses 5,985 3,991 Current portion of operating lease liability 998 1,773 Current portion of payable to licensor 4,580 — Other current liabilities 1 204 Total current liabilities 13,422 7,779 Payable to licensor — 4,163 Long-term operating lease liabilities 4,402 5,854 Warrant liabilities 31,352 19,657 Total liabilities 49,176 37,453 Commitments and contingencies Stockholders' equity: Preferred stock - $0.01 par value; authorized 2,000,000 shares; No shares issued and outstanding as of December 31, 2023 and December 31, 2022, respectively — — Common stock - $0.01 par value; authorized 200,000,000 shares; 26,523,878 and 17,719,720 shares issued and outstanding as of December 31, 2023 and December 31, 2022, respectively 265 177 Additional paid-in capital 764,151 722,049 Accumulated deficit (749,524 ) (695,336 ) Accumulated other comprehensive loss (66 ) (129 ) Total stockholders' equity 14,826 26,761 Total liabilities and stockholders' equity $ 64,002 $ 64,214
What did the FDA complete for Abeona's Cleveland manufacturing facility?
The FDA completed a Pre-License Inspection of Abeona's Cleveland manufacturing facility for the pz-cel BLA.
What did the FDA inspect related to the pivotal Phase 3 VIITAL™ study?
The FDA inspected clinical trial sites for the pivotal Phase 3 VIITAL™ study with no Form 483 observations.
What is the target PDUFA date for the FDA's review of the pz-cel BLA?
The target Prescription Drug User Fee Act (PDUFA) date for the FDA's review of the pz-cel BLA is May 25, 2024.
What financial deal did Abeona secure recently?
Abeona secured a $50 million credit facility with the Avenue Venture Opportunities Fund.
What were the full-year 2023 financial results for Abeona?
Full-year 2023 financial results showed $52.6 million in cash and investments, with $3.5 million in license and other revenues.
What were the research and development expenses for Abeona in 2023?
Research and development expenses for Abeona in 2023 were $31.1 million.
