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
Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical
company developing cell and gene therapies for serious diseases.
The U.S. FDA has accepted and granted Priority Review with a PDUFA
target action date of May 25, 2024 for the Biologics License
Application for pz-cel (prademagene zamikeracel), 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-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
SUBSIDIARIESConsolidated Statements of Operations and
Comprehensive Loss(In thousands, except share and per share
amounts) |
|
|
|
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
SUBSIDIARIESConsolidated Balance Sheets(In thousands,
except share and per share amounts) |
|
|
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 |
|
|
|
|
|
Investor and Media Contact:
Greg Gin
VP, Investor Relations and Corporate Communications
Abeona Therapeutics
ir@abeonatherapeutics.com
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