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 31, 2022
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 incorporation) | (Commission<br><br> <br>File Number) | (I.R.S. Employer<br><br> <br>Identification No.) |
1330Avenue of the Americas, 33rd Floor,
NewYork, NY 10019
(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. ☐
Item 2.02. Results of Operations and Financial Condition.
On March 31, 2022, Abeona Therapeutics Inc. issued a press release regarding its results of operations and financial condition for the fourth quarter and full year 2021. 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 31, 2022, entitled “Abeona Therapeutics Announces Strategy Update and 2021 Financial Results” |
| 104 | Cover 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/ Brendan M. O’Malley |
| Name: | Brendan<br> M. O’Malley |
| Title: | Senior<br> Vice President, General Counsel |
Date: March 31, 2022
Exhibit99.1
AbeonaTherapeutics Announces Strategy Update and 2021 Financial Results
EB-101pivotal phase 3 VIITAL™ study in RDEB achieves target enrollment; top-line data expected in third quarter of 2022; gained FDA alignmenton CMC requirements for EB-101 including characterization and validation plans
Companyto focus R&D resources primarily on VIITAL™ data readout while actively pursuing potential commercialization partner for EB-101;maintain focus on preclinical eye gene therapy programs
Pursuestrategic partner to take over development activities for ABO-102 for MPS IIIA, while discontinuing development of ABO-101 for MPS IIIB
Reduceoperating expenses and extend cash runway to mid-2023
$50.9million in cash resources as of December 31, 2021
Conferencecall scheduled for today at 8:30 a.m. ET
NEW YORK and CLEVELAND, March 31, 2022 – Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in cell and gene therapy, today provided a business and strategy update and reported 2021 financial results.
“We are committed to developing novel cell and gene therapies for patients with rare diseases with no approved treatment options,” said Vish Seshadri, Ph.D., Chief Executive Officer of Abeona. “We are focused on EB-101 and, having recently achieved target enrollment in our pivotal Phase 3 VIITAL™ study, have increased confidence that we will share topline results in the third quarter of 2022. We also expect animal proof-of-concept data from our preclinical eye programs beginning in the second half of 2022 that could support pre-IND meetings with the FDA. We believe the strategic steps announced today reflect the operating discipline needed to extend our cash runway beyond these near-term catalysts.”
Strategyand Business Update
| ● | Following<br> a comprehensive portfolio review, Abeona announced today that it is focusing research and development (R&D) resources on the<br> topline data readout for its EB-101 pivotal Phase 3 VIITAL™ study while the Company actively pursues a potential commercialization<br> partner for EB-101. |
|---|---|
| ● | Target<br> enrollment was achieved in Abeona’s EB-101 pivotal Phase 3 VIITAL™ study for recessive dystrophic epidermolysis bullosa<br> (RDEB). The Company anticipates topline results for the co-primary endpoints related to wound healing and pain reduction measured<br> at 24 weeks post treatment in the third quarter of 2022. Abeona received positive feedback from the U.S. Food and Drug Administration<br> (FDA) related to a Type B meeting on the proposed Chemistry, Manufacturing and Controls (CMC) requirements of the EB-101 development<br> program, gaining alignment on the characterization and validation plans that could support a potential Biologics License Application<br> (BLA) for EB-101 in RDEB. |
| ● | In<br> connection with its shift in priorities, the Company has intensified its pursuit of a strategic partnership to take over development<br> activities for ABO-102, and has ceased build-out of additional AAV manufacturing space. As part of the FDA’s feedback on the<br> Transpher A Statistical Analysis Plan (SAP) in January 2022, the agency recommended that all participants be followed to an age of<br> at least 60 months, which would shift timing of the neurocognitive outcomes data readout to late-2024/early-2025, as compared to<br> the Company’s prior projection of the second quarter of 2023. |
| --- | --- |
| ● | As<br> part of the Company’s portfolio prioritization, Abeona will discontinue development of ABO-101 for MPS IIIB. |
| ● | Abeona<br> plans to continue development of AAV-based gene therapies designed to treat ophthalmic and other diseases and next-generation AAV-based<br> gene therapies using the novel AIM™ capsid platform and internal AAV vector research programs. Abeona will present results<br> from testing of novel AAV capsids in non-human primates at the Association for Research in Vision and Ophthalmology (ARVO) 2022 Annual<br> Meeting being held on May 1-4, 2022 in Denver, CO. The preclinical data could support pre-IND meetings with the FDA for Abeona’s<br> undisclosed eye gene therapy indications. The Company previously reported preclinical data showing the potential for AIM™ AAV<br> vectors to efficiently target the photoreceptor and retinal epithelium cell layers after intravitreal injection, creating the potential<br> for new pipeline candidates that can address multiple ophthalmic disorders. |
| ● | The<br> strategic changes will reduce the Company’s operating expenses and extend the estimated runway of current cash resources to<br> mid-2023. |
| ● | In<br> December 2021, Abeona raised approximately $17.5 million in aggregate gross proceeds, before underwriting discounts and commissions<br> and other offering expenses, from an underwritten public offering of common stock. |
| ● | Joseph<br> Vazzano was appointed as Chief Financial Officer (CFO) at Abeona, and will serve as the Company’s principal financial officer<br> and principal accounting officer effective March 31, 2022. Mr. Vazzano previously served as CFO of Avenue Therapeutics, Inc. (Nasdaq:<br> ATXI), where he secured multiple equity financings for Avenue and served in a leadership role for signing a complex, two-stage acquisition<br> of the company with future contingent value rights. |
FullYear 2021 Financial Results
Cash, cash equivalents, restricted cash and short-term investments totaled $50.9 million as of December 31, 2021, compared to $96.0 million as of December 31, 2020. Net cash used in operating activities was $65.7 million for the full year of 2021, including a $20 million payment in November 2021 in accordance with a settlement agreement with REGENXBIO. Net cash used in operating activities was $35.0 million for the full year of 2020.
License and other revenues for the full year of 2021 were $3.0 million, compared to $10.0 million in 2020. The revenue in 2021 resulted from a clinical milestone achieved in December 2021 under a sublicense agreement with Taysha Gene Therapies for ABO-202 for CLN1 disease.
R&D expenses were $34.3 million for the full year of 2021, compared to $30.1 million in 2020. General and administrative (G&A) expenses were $22.8 million for the full year of 2021, compared to $23.8 million in 2020.
Net loss was $84.9 million for the full year of 2021, or a $0.86 basic and diluted loss per common share as compared to a net loss of $84.2 million, or a $0.91 basic and diluted loss per common share, in 2020. The net loss in 2021 included a non-cash goodwill impairment charge of $32.5 million. The impairment charge has no impact on the Company’s cash position, cash flow from operating activities, and does not have any impact on future operations.
ConferenceCall Details
Abeona Therapeutics will host a conference call and webcast today, Thursday, March 31, 2022 at 8:30 a.m. ET, to discuss its full year 2021 financial results and business update. To access the call, dial 877-545-0320 (U.S. toll-free) or 973-528-0002 (international) and Entry Code: 851784 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 www.abeonatherapeutics.com. 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. Abeona’s lead clinical program is EB-101, its investigational autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development. 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. Abeona’s fully integrated gene and cell therapy cGMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and potential commercial production of AAV-based gene therapies. 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,” “estimate,”“expect,” and similar 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 bysuch forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limitedto the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition; continued interest in our raredisease portfolio; our ability to commercialize our EB-101 product candidate; obtaining a strategic partnership to take over developmentactivities for ABO-102; our ability to enroll patients in clinical trials; the outcome of any future meetings with the U.S. Food andDrug Administration or other regulatory agencies; the impact of competition; the ability to secure licenses for any technology that maybe necessary to commercialize our product candidates; the ability to achieve or obtain necessary regulatory approvals; the impact ofchanges in the financial markets and global economic conditions; risks associated with data analysis and reporting; reducing our operatingexpenses and extending our cash runway; our ability to execute our operating plan and achieve important anticipated milestones; and otherrisks disclosed in the Company’s most recent Annual Report on Form 10-K and other periodic reports filed with the Securities andExchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect eventsor 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.
Investorand Media Contact:
Greg Gin
VP, Investor Relations and Corporate Communications
Abeona Therapeutics
+1 (646) 813-4709
ggin@abeonatherapeutics.com
AbeonaTherapeutics Inc. and Subsidiaries
CondensedConsolidated Statements of Operations and Comprehensive Loss
(unaudited)
| For the three months ended December 31, | For the years ended<br> <br>December 31, | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 2021 | 2020 | 2021 | 2020 | |||||||||
| Revenues | $ | 3,000,000 | $ | 3,000,000 | $ | 3,000,000 | $ | 10,000,000 | ||||
| Expenses: | ||||||||||||
| Research and development | 11,701,000 | 9,243,000 | 34,325,000 | 30,139,000 | ||||||||
| General and administrative | 4,678,000 | 7,397,000 | 22,795,000 | 23,779,000 | ||||||||
| Depreciation and amortization | 807,000 | 840,000 | 3,250,000 | 4,586,000 | ||||||||
| Goodwill impairment charge | 32,466,000 | - | 32,466,000 | - | ||||||||
| Licensed technology impairment charge | - | - | - | 32,916,000 | ||||||||
| Total expenses | 49,652,000 | 17,480,000 | 92,836,000 | 91,420,000 | ||||||||
| Loss from operations | (46,652,000 | ) | (14,480,000 | ) | (89,836,000 | ) | (81,420,000 | ) | ||||
| Gain on settlement with licensor | - | - | 6,743,000 | - | ||||||||
| PPP loan payable forgiveness income | - | - | 1,758,000 | - | ||||||||
| Interest and miscellaneous income | 36,000 | 40,000 | 69,000 | 1,301,000 | ||||||||
| Interest and other expense | (67,000 | ) | (1,388,000 | ) | (3,670,000 | ) | (4,115,000 | ) | ||||
| Net loss | $ | (46,683,000 | ) | $ | (15,828,000 | ) | $ | (84,936,000 | ) | $ | (84,234,000 | ) |
| Basic and diluted loss per common share | $ | (0.45 | ) | $ | (0.17 | ) | $ | (0.86 | ) | $ | (0.91 | ) |
| Weighted average number of common | ||||||||||||
| shares outstanding – basic and diluted | 104,699,988 | 92,869,775 | 98,441,911 | 92,663,574 | ||||||||
| Other comprehensive income/(loss): | ||||||||||||
| Change in unrealized gains/(losses) related to available-for-sale debt securities | (1,000 | ) | (27,000 | ) | 9,000 | (10,000 | ) | |||||
| Foreign currency translation adjustments | (17,000 | ) | - | (26,000 | ) | - | ||||||
| Comprehensive loss | $ | (46,701,000 | ) | $ | (15,855,000 | ) | $ | (84,953,000 | ) | $ | (84,244,000 | ) |
AbeonaTherapeutics Inc. and Subsidiaries
CondensedConsolidated Balance Sheets
(unaudited)
| December 31, 2020 | |||||
|---|---|---|---|---|---|
| ASSETS | |||||
| Current assets: | |||||
| Cash and cash equivalents | 32,938,000 | $ | 12,596,000 | ||
| Short-term investments | 12,086,000 | 82,438,000 | |||
| Accounts receivable | 3,000,000 | - | |||
| Prepaid expenses, other current assets and restricted cash | 7,377,000 | 2,708,000 | |||
| Total current assets | 55,401,000 | 97,742,000 | |||
| Property and equipment, net | 12,339,000 | 11,322,000 | |||
| Right-of-use lease assets | 9,403,000 | 7,032,000 | |||
| Licensed technology, net | 1,384,000 | 1,500,000 | |||
| Goodwill | - | 32,466,000 | |||
| Other assets and restricted cash | 1,059,000 | 1,136,000 | |||
| Total assets | 79,586,000 | $ | 151,198,000 | ||
| LIABILITIES AND STOCKHOLDERS’ EQUITY | |||||
| Current liabilities: | |||||
| Accounts payable | 4,325,000 | $ | 4,695,000 | ||
| Accrued expenses | 5,585,000 | 3,410,000 | |||
| Current portion of lease liability | 1,818,000 | 1,713,000 | |||
| Current portion of PPP loan payable | - | 330,000 | |||
| Current portion of payable to licensor | 4,599,000 | 31,515,000 | |||
| Deferred revenue | 296,000 | 296,000 | |||
| Total current liabilities | 16,623,000 | 41,959,000 | |||
| PPP loan payable | - | 1,428,000 | |||
| Payable to licensor | 3,828,000 | - | |||
| Other long-term liabilities | 200,000 | - | |||
| Long-term lease liabilities | 7,560,000 | 5,260,000 | |||
| Total liabilities | 28,211,000 | 48,647,000 | |||
| Commitments and contingencies | - | - | |||
| Stockholders’ equity: | |||||
| Preferred stock - 0.01 par value; authorized 2,000,000 shares; no issued and outstanding shares at December 31, 2021 and<br> December 31, 2020 | - | - | |||
| Common stock - 0.01 par value; authorized 200,000,000 shares; issued and outstanding 147,205,422 at<br> December 31, 2021; issued and outstanding 96,131,678 at December 31, 2020 | 1,472,000 | 961,000 | |||
| Additional paid-in capital | 705,570,000 | 672,304,000 | |||
| Accumulated deficit | (655,640,000 | ) | (570,704,000 | ) | |
| Accumulated other comprehensive loss | (27,000 | ) | (10,000 | ) | |
| Total stockholders’ equity | 51,375,000 | 102,551,000 | |||
| Total liabilities and stockholders’ equity | 79,586,000 | $ | 151,198,000 |
All values are in US Dollars.