Abeona Therapeutics Announces Strategy Update and 2021 Financial Results
March 31 2022 - 7:30AM
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.”
Strategy and Business
Update
-
Following a comprehensive portfolio review, Abeona announced today
that it is focusing research and development (R&D) resources on
the topline data readout for its EB-101 pivotal Phase 3 VIITAL™
study while the Company actively pursues a potential
commercialization partner for EB-101.
-
Target enrollment was achieved in Abeona’s EB-101 pivotal Phase 3
VIITAL™ study for recessive dystrophic epidermolysis bullosa
(RDEB). The Company anticipates topline results for the co-primary
endpoints related to wound healing and pain reduction measured at
24 weeks post treatment in the third quarter of 2022. Abeona
received positive feedback from the U.S. Food and Drug
Administration (FDA) related to a Type B meeting on the proposed
Chemistry, Manufacturing and Controls (CMC) requirements of the
EB-101 development program, gaining alignment on the
characterization and validation plans that could support a
potential Biologics License Application (BLA) for EB-101 in
RDEB.
-
In connection with its shift in priorities, the Company has
intensified its pursuit of a strategic partnership to take over
development activities for ABO-102, and has ceased build-out of
additional AAV manufacturing space. As part of the FDA’s feedback
on the Transpher A Statistical Analysis Plan (SAP) in January 2022,
the agency recommended that all participants be followed to an age
of at least 60 months, which would shift timing of the
neurocognitive outcomes data readout to late-2024/early-2025, as
compared to the Company’s prior projection of the second quarter of
2023.
-
As part of the Company’s portfolio prioritization, Abeona will
discontinue development of ABO-101 for MPS IIIB.
-
Abeona plans to continue development of AAV-based gene therapies
designed to treat ophthalmic and other diseases and next-generation
AAV-based gene therapies using the novel AIM™ capsid platform and
internal AAV vector research programs. Abeona will present results
from testing of novel AAV capsids in non-human primates at the
Association for Research in Vision and Ophthalmology (ARVO) 2022
Annual 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 undisclosed eye gene therapy indications. The Company
previously reported preclinical data showing the potential for AIM™
AAV vectors to efficiently target the photoreceptor and retinal
epithelium cell layers after intravitreal injection, creating the
potential for new pipeline candidates that can address multiple
ophthalmic disorders.
-
The strategic changes will reduce the Company’s operating expenses
and extend the estimated runway of current cash resources to
mid-2023.
-
In December 2021, Abeona raised approximately $17.5 million in
aggregate gross proceeds, before underwriting discounts and
commissions and other offering expenses, from an underwritten
public offering of common stock.
-
Joseph Vazzano was appointed as Chief Financial Officer (CFO) at
Abeona, and will serve as the Company’s principal financial officer
and principal accounting officer effective March 31, 2022. Mr.
Vazzano previously served as CFO of Avenue Therapeutics, Inc.
(Nasdaq: ATXI), where he secured multiple equity financings for
Avenue and served in a leadership role for signing a complex,
two-stage acquisition of the company with future contingent value
rights.
Full Year 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.
Conference Call 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.
About Abeona 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-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,” “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 by such
forward-looking statements as a result of various important
factors, numerous risks and uncertainties, including but not
limited to the potential impacts of the COVID-19 pandemic on our
business, operations, and financial condition; continued interest
in our rare disease portfolio; our ability to commercialize our
EB-101 product candidate; obtaining a strategic partnership to take
over development activities for ABO-102; our ability to enroll
patients in clinical trials; the outcome of any future meetings
with the U.S. Food and Drug Administration or other regulatory
agencies; the impact of competition; the ability to secure licenses
for any technology that may be necessary to commercialize our
product candidates; the ability to achieve or obtain necessary
regulatory approvals; the impact of changes in the financial
markets and global economic conditions; risks associated with data
analysis and reporting; reducing our operating expenses and
extending our cash runway; our ability to execute our operating
plan and achieve important anticipated milestones; and other risks
disclosed in the Company’s most recent Annual Report on Form 10-K
and other 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 |
(unaudited) |
|
|
For the three months endedDecember 31, |
|
For the years endedDecember
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 |
) |
|
|
|
|
|
|
|
|
|
Abeona Therapeutics Inc. and Subsidiaries |
Condensed Consolidated Balance Sheets |
(unaudited) |
ASSETS |
December 31, 2021 |
|
December 31, 2020 |
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 December 31, 2020 |
|
- |
|
|
|
- |
|
Common stock - $0.01 par value; authorized 200,000,000 shares; |
|
|
|
issued and outstanding 147,205,422 at 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 |
|
|
|
|
|
Investor and Media Contact:
Greg Gin
VP, Investor Relations and Corporate Communications
Abeona Therapeutics
+1 (646) 813-4709
ggin@abeonatherapeutics.com
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