Abeona Therapeutics(ABEO) - 2023 Q4 - Annual Results

Company Announcement & Highlights Abeona Therapeutics reported 2023 financial results and completed key FDA inspections for its pz-cel BLA, including manufacturing and clinical sites Full Year 2023 Financial Results and FDA Inspections Completion Abeona Therapeutics reported its full year 2023 financial results and announced the completion of key FDA inspections for its pz-cel Biologics License Application (BLA), including a Pre-License Inspection of its Cleveland manufacturing facility and clinical trial site inspections - Abeona Therapeutics reported full year 2023 financial results[1] - FDA completed a Pre-License Inspection (PLI) of Abeona's Cleveland manufacturing facility for pz-cel BLA[2] - FDA also completed clinical study site inspections for the pivotal Phase 3 VIITAL™ study, with no Form 483 observations[2] Regulatory & Commercial Progress Abeona advanced pz-cel's FDA review with priority status, prepared for U.S. commercial launch, and secured a $50 million credit facility Pz-cel for RDEB Regulatory Updates The FDA has made significant progress in its review of Abeona's pz-cel BLA for recessive dystrophic epidermolysis bullosa (RDEB), including granting Priority Review, completing various inspections, and reaffirming a target PDUFA date of May 25, 2024, without plans for an Advisory Committee - FDA completed a Pre-License Inspection (PLI) of the Cleveland manufacturing facility, issuing a Form 483 with observations related to process controls, to which Abeona submitted a response[2] - FDA accepted and granted Priority Review for the pz-cel BLA in November 2023, with a target PDUFA date of May 25, 2024[3] - FDA completed a Bioresearch Monitoring (BIMO) inspection and Mid-Cycle Meeting in January 2024, with no Form 483s issued from the BIMO inspection and no Risk Evaluation and Mitigation Strategies (REMS) anticipated for the BLA[4] U.S. Commercial Launch Preparations for pz-cel Abeona is actively preparing for the potential U.S. commercial launch of pz-cel, engaging with treatment sites, payers, and conducting market research to support reimbursement coverage - Abeona is advancing key commercial activities for pz-cel launch, including onboarding discussions with EB treatment sites, payer engagement, and hiring key commercial roles[5] - Payer market research supports reimbursement coverage of pz-cel at a price commensurate with the value of approved gene therapies[5] Corporate Financing Highlights Abeona secured a $50 million credit facility in January 2024, with an initial $20 million tranche funded, to support ongoing launch and commercialization preparations for pz-cel - Abeona entered a $50 million credit facility with Avenue Venture Opportunities Fund, L.P. in January 2024[5] - The credit agreement includes a first tranche of $20 million, funded in January 2024, a second tranche of $10 million, and an option for an additional $20 million[5] - Proceeds from the facility are intended to support pz-cel launch and commercialization preparations[5] Financial Performance Abeona's 2023 financials show stable cash, increased licensing revenue, higher operating expenses, and an increased net loss Full Year 2023 Financial Results and Cash Runway Guidance Abeona maintained a stable cash position at the end of 2023 and significantly reduced net cash used in operating activities. With recent financing, the company projects its cash runway to extend into the first quarter of 2025 Cash and Investments (in millions) | Metric | Dec 31, 2023 | Dec 31, 2022 | | :----------------------------------------- | :----------- | :----------- | | Cash, cash equivalents, restricted cash and short-term investments | $52.6 | $52.5 | Net Cash Used in Operating Activities (in millions) | Period | 2023 | 2022 | | :----------- | :---- | :---- | | Net cash used in operating activities | $37.0 | $43.5 | - Current cash and credit facility are sufficient to fund operations into Q1 2025, excluding potential pz-cel revenue or Priority Review Voucher proceeds[6] Revenue Analysis License and other revenues increased significantly in 2023, primarily driven by clinical milestone payments from a licensing agreement for an AAV-based gene therapy License and Other Revenues (in millions) | Metric | 2023 | 2022 | YoY Change | | :------------------------- | :---- | :---- | :--------- | | License and other revenues | $3.5 | $1.4 | +150% | - Revenues primarily represent clinical milestone payments under a licensing agreement with Taysha Gene Therapies for investigational AAV-based gene therapy for Rett syndrome[7] Operating Expenses Both research and development (R&D) and general and administrative (G&A) expenses increased in 2023, primarily due to increased headcount supporting BLA activities and pz-cel launch preparations Operating Expenses (in millions) | Metric | 2023 | 2022 | YoY Change | | :------------------------------- | :---- | :---- | :--------- | | Research and development expenses | $31.1 | $29.0 | +7.2% | | General and administrative expenses | $19.0 | $17.3 | +9.8% | - Increase in R&D expenses was primarily due to increased headcount related to BLA activities[8] - Increase in G&A expenses was primarily due to increased headcount for the potential launch of pz-cel[8] Net Loss and EPS Abeona reported an increased net loss attributable to common shareholders in 2023, with a corresponding decrease in loss per common share due to a higher weighted average number of shares outstanding Net Loss and EPS (in millions, except per share amounts) | Metric | 2023 | 2022 | YoY Change | | :----------------------------------------- | :---------- | :---------- | :--------- | | Net loss attributable to common shareholders | $(54.2) million | $(43.5) million | +24.6% | | Loss per common share | $(2.53) | $(5.53) | -54.3% | Company Information Abeona Therapeutics is a clinical-stage biopharmaceutical company developing cell and gene therapies, with pz-cel for RDEB nearing FDA approval About Abeona Therapeutics Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, with pz-cel for RDEB as its lead investigational product nearing potential FDA approval, supported by its integrated cGMP manufacturing facility - Abeona Therapeutics is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases[10] - The U.S. FDA has granted Priority Review for the BLA for pz-cel (prademagene zamikeracel) for recessive dystrophic epidermolysis bullosa (RDEB), with a PDUFA target action date of May 25, 2024[10] - The company's development portfolio also includes AAV-based gene therapies for ophthalmic diseases and novel, next-generation AAV capsids[10] Financial Statements This section details Abeona Therapeutics' consolidated statements of operations, comprehensive loss, and balance sheets for 2023 and 2022 Consolidated Statements of Operations and Comprehensive Loss The consolidated statements of operations and comprehensive loss provide a detailed breakdown of Abeona Therapeutics' financial performance for the years ended December 31, 2023, and 2022, including revenues, expenses, and net loss Consolidated Statements of Operations and Comprehensive Loss (in thousands, except share and per share amounts) | Metric | 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) | Consolidated Balance Sheets The consolidated balance sheets present Abeona Therapeutics' financial position as of December 31, 2023, and 2022, detailing assets, liabilities, and stockholders' equity Consolidated Balance Sheets (in thousands, except share and per share amounts) | Metric | 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 | Disclosures & Contacts This section provides conference call details, forward-looking statement disclaimers, and investor contact information Conference Call Details Abeona Therapeutics hosted a conference call and webcast on March 18, 2024, to discuss the financial results and corporate updates, with replay access available online - Abeona Therapeutics hosted a conference call and webcast on March 18, 2024, at 8:30 a.m. ET[9] - Access details: Dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) with Entry Code: 428606[9] - A live webcast and archived replay are available at https://investors.abeonatherapeutics.com/events for 30 days[9] Forward-Looking Statements This section contains forward-looking statements subject to various risks and uncertainties, including those related to FDA approvals, clinical trials, and financial market conditions, and the company disclaims any obligation to update these statements - The press release contains forward-looking statements as defined by Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934[11] - Actual results may differ materially due to various factors, including the timing and outcome of the pz-cel BLA submission, FDA inspections, regulatory approvals, and global economic conditions[11] - The Company undertakes no obligation to revise or update forward-looking statements, except as required by federal securities laws[11] Investor and Media Contact Contact information for investor relations and corporate communications is provided for inquiries - Investor and Media Contact: Greg Gin, VP, Investor Relations and Corporate Communications, Abeona Therapeutics[12] - Email: ir@abeonatherapeutics.com[12]