8-K
ABEONA THERAPEUTICS INC. (ABEO)
UNITED
STATES
SECURITIES
AND EXCHANGE COMMISSION
Washington,
DC 20549
FORM
8-K
CURRENT
REPORT
PURSUANT
TO SECTION 13 OR 15(d) OF THE
SECURITIES
EXCHANGE ACT OF 1934
Date of report (Date of earliest event reported): March 20, 2025
ABEONA
THERAPEUTICS INC.
(Exact name of registrant as specified in its charter)
| Delaware | 001-15771 | 83-0221517 |
|---|---|---|
| (State<br> or other jurisdiction<br><br> <br>of<br> incorporation) | (Commission<br><br> <br>File<br> Number) | (I.R.S.<br> Employer<br><br> <br>Identification<br> No.) |
6555Carnegie Ave, 4th Floor
Cleveland,OH 44103
(Address of principal executive offices) (Zip Code)
(646)813-4701(Registrant’s telephone number, including area code)
N/A
(Former name or former address, if changed since last report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):
| ☐ | Written<br> communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
|---|---|
| ☐ | Soliciting<br> material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
| ☐ | Pre-commencement<br> communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
| ☐ | Pre-commencement<br> communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
Securities registered pursuant to Section 12(b) of the Act:
| Title<br> of Each Class | Trading<br> Symbol | Name<br> of each exchange on which registered |
|---|---|---|
| Common Stock, $0.01 par value | ABEO | Nasdaq Capital Markets |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company ☐
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Item2.02. Results of Operations and Financial Condition.
On March 20, 2025, Abeona Therapeutics Inc. issued a press release regarding its financial results for the year ended December 31, 2024. The full text of the press release is filed as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein.
The information in Item 2.02 of this Current Report on Form 8-K and Exhibit 99.1 attached hereto shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing.
Item9.01 Financial Statements and Exhibits.
(d) Exhibits.
| Exhibit No. | Description |
|---|---|
| 99.1 | Press release dated March 20, 2025 |
| 104 | Cover<br> Page Interactive Data File (embedded within the Inline XBRL document) |
SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| Abeona<br> Therapeutics Inc. | |
|---|---|
| (Registrant) | |
| By: | /s/ Joseph Vazzano |
| Name: | Joseph<br> Vazzano |
| Title: | Chief<br> Financial Officer |
Date: March 20, 2025
Exhibit99.1
AbeonaTherapeutics^®^ Reports Full Year 2024 Financial Results, Provides Pz-cel Regulatory Update and Commercial Launch Plans
FDApriority review of pz-cel Biologics License Application (BLA) progressing with Prescription Drug User Fee Act (PDUFA) target action dateof April 29, 2025
Launchpreparations on track toward goal to treat first patient in 3Q 2025
CLEVELAND, March 20, 2025 – 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.”
FourthQuarter and Recent Progress
Pz-celfor RDEB
| ● | Abeona<br> continues to work with the FDA to finalize the review of its Biologics<br> License Application (BLA) for pz-cel as a potential new treatment for patients with RDEB.<br> In November 2024, the FDA accepted the BLA resubmission for review and set a PDUFA target action date of April 29, 2025. On<br> March 14, 2025, Abeona received draft United States Prescribing Information (USPI) from the FDA to initiate discussion on the label<br> for pz-cel. Discussions are also ongoing with the FDA on post-approval marketing requirements and commitments for pz-cel. If approved,<br> the Company anticipates the first patient treatment with pz-cel in the third quarter of 2025. Abeona may be eligible for a Priority<br> Review Voucher (PRV) should pz-cel be approved. |
|---|---|
| ● | In<br> anticipation of potential FDA approval of pz-cel, Abeona continues its commercialization<br> efforts, including progress toward onboarding five geographically dispersed, well-recognized epidermolysis bullosa treatment centers<br> in the U.S. as pz-cel qualified treatment centers (QTCs), engaging payers to ensure patient access, and educating key stakeholders. |
| ● | In<br> preparation for potential pz-cel launch, Abeona has hired and trained personnel to support commercialization, manufacturing, supply<br> chain and quality. |
| ● | Abeona<br> entered into a lease agreement for additional facility space in Cleveland, Ohio to enable expansion of manufacturing capacity for<br> pz-cel beyond the current planned manufacturing footprint. |
| ● | Obtained<br> two additional patents from the United States Patent and Trademark Office for pz-cel, extending<br> patent protection on the use of pz-cel for the treatment of RDEB to June 2037 and patent protection on the packaging and transport<br> system for pz-cel to July 2040. |
Pipelineand partnered programs
| ● | In<br> December 2024, Ultragenyx submitted a BLA to the FDA seeking accelerated approval for UX111 (formerly ABO-102) AAV gene therapy as<br> a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). In February 2025, Ultragenyx announced FDA acceptance and Priority<br> Review of the UX111 BLA with a PDUFA date of August 18, 2025. |
|---|
FullYear 2024 Financial Results and Cash Runway Guidance
Cash, cash equivalents, short-term investments and restricted cash totaled $98.1 million as of December 31, 2024, compared to $52.6 million as of December 31, 2023.
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 $34.4 million, compared to $31.1 million for the full year ended December 31, 2023, primarily a result of increased headcount related to manufacturing capacity expansion in preparation for the potential launch of pz-cel, partially offset by reduced spending on clinical and development work. General and administrative (G&A) expenses were $29.9 million for the full year ended December 31, 2024, compared to $19.0 million for the year ended December 31, 2023. The increase in G&A expenses is primarily due to commercial launch preparation costs. Net loss for the full year ended December 31, 2024 was $63.7 million, or $1.55 loss per common share as compared to $54.2 million, or $2.53 loss per common share, for the full year of 2023.
ConferenceCall 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.
AboutAbeona 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-LookingStatements
*Thispress release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, asamended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. We have attemptedto 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 expressionsreferencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actualresults may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerousrisks 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-basedgene 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 clinicaldevelopment, clinical trials and potential regulatory approvals; our pipeline of product candidates; our belief that pz-cel could potentiallybenefit patients with RDEB; our belief in the adequacy of the clinical trial data from our VIITAL™ clinical trial, together withthe data generated in the pz-cel program to date, to support pz-cel’s regulatory approval; our dependence upon our third-partycustomers 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 propertyprotection 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 orperformance; risks associated with data analysis and reporting; and other risks disclosed in the Company’smost recent Annual Report on Form 10-K and subsequent periodic reports filed with the Securities and Exchange Commission. The Companyundertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring afterthe date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federalsecurities laws.
Investorand Media Contact:
Greg Gin
VP, Investor Relations and Corporate Communications
Abeona Therapeutics
ir@abeonatherapeutics.com
ABEONATHERAPEUTICS INC. AND SUBSIDIARIESCondensed 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 | ) |
ABEONATHERAPEUTICS INC. AND SUBSIDIARIES
Condensed Consolidated Balance Sheets
($ in thousands, except share and per share amounts)
| 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 - 0.01 par value; authorized 2,000,000 shares; No shares issued and outstanding as of December 31, 2024 and 2023, respectively | — | — | |||
| Common stock - 0.01 par value; authorized 200,000,000 shares; 45,644,091 and 26,523,878 shares issued and outstanding as of December 31, 2024 and 2023, respectively | 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 |
All values are in US Dollars.