Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader
in gene and cell therapy, today announced first quarter 2019
financial results and business highlights, which will be discussed
on a conference call scheduled for Tuesday, May 14 at 10:00 a.m.
ET. Interested parties are invited to participate in the call by
dialing 844-369-8770 (toll-free domestic) or 862-298-0840
(International) or via webcast at
https://www.investornetwork.com/event/presentation/48814.
“We’ve had a great start to 2019, with continued
progress across our pipeline, including the completion of CMC work
in advance of initiating our Phase 3 VITAL™ clinical trial
evaluating EB-101, our gene-corrected cell therapy for patients
with recessive dystrophic epidermolysis bullosa,” said João
Siffert, M.D., Chief Executive Officer. “The study, which will
utilize clinical material produced at our Cleveland manufacturing
facility, is on track to start mid-2019 following completion of
ongoing FDA review.”
“We have continued to advance our lysosomal
storage disease programs. Our increased efforts around patient
recruitment are starting to bear fruit as we observe an uptick in
patient screening for both MPS programs. In addition, we have
submitted the IND for our CLN1 program.” added Dr. Siffert.
First Quarter Financial Results:
Cash, cash equivalents and marketable securities
as of March 31, 2019 were $68.3 million, compared to $85.0 million
as of December 31, 2018. The decrease in cash of $16.7 million was
driven primarily by the net cash used for operating activities of
$15.1 million.
Research and development expenses for the first
quarter ended March 31, 2019 were $11.7 million, compared to $8.2
million for first quarter 2018. The increase in research and
development expense was primarily attributable to increased R&D
headcount, related facility costs and internal manufacturing
costs.
General and administrative expenses for the
first quarter ended March 31, 2019 were $5.7 million, compared to
$2.9 million for the first quarter 2018. The increase in general
and administrative expenses was primarily attributable to increased
headcount and related facility costs.
Net loss was $0.39 per share for the first
quarter of 2019, compared to $0.22 per share in the same period of
2018.
First Quarter and Recent Highlights:
- May 8, 2019: Abeona Therapeutics Announces Presentation of
EB-101 Data at Society for Investigative Dermatology Annual
Meeting-- Follow up data showed that three years after
treatment with investigational product EB-101, a majority of RDEB
patients had durable wound healing and improvements in pain and
itching. There were no serious treatment-related adverse events
three years post-treatment and no replication competent virus
present at any time point.
- May 1, 2019: Reported Preclinical Data Demonstrating Broad
Therapeutic Potential of AIM™ Gene Therapy in Retinal Diseases at
Association for Research in Vision and Ophthalmology Annual
Meeting-- Intravitreal administration of the Company’s novel
AIM™ AAV204 capsid in non-human primates led to robust transgene
expression in the inner and outer retina. These preclinical data
support the potential use of intravitreal administration to deliver
gene therapy in an out-patient setting for a wide range of
inherited and acquired retinal diseases.
- April 30, 2019: Reported New Preclinical Data Demonstrating
Therapeutic Potential of ABO-401 for Treatment of Cystic Fibrosis
at American Society of Gene and Cell Therapy Annual Meeting--
ABO-401, the Company’s novel gene therapy for cystic fibrosis (CF)
efficiently delivered a highly-expressed, functional copy of human
mini-CFTR (hCFTR) to the lung of CF mice and restored CFTR function
in human CF patient nasal and bronchial epithelial cells.
- April 4, 2019: Received FDA Fast Track Designation for ABO-101
for Treatment of Sanfilippo Syndrome Type B (MPS IIIB)
- February 11, 2019: Appointed João Siffert, M.D. Chief Executive
Officer
- January 31, 2019: Presented New Supportive Data for Novel Gene
Therapies at WORLDSymposium™-- Platform and poster
presentations highlighted study results on biodistribution and
tissue tropism of the next-generation AIM™ AAV vector platform in
Pompe and Fabry diseases, as well as data from programs in MPS IIIA
and CLN3 disease.
- January 8, 2019: Strengthened Financial Leadership with New
Executive Appointments: Christine Silverstein as Chief Financial
Officer and Edward Carr as Chief Accounting Officer
“We expect 2019 to be a transformative year for
Abeona, as we prepare to execute on a number of milestones in the
clinic, in our mission to develop gene and cell therapies that
could transform the treatment of serious diseases,” said Steven H.
Rouhandeh, Chairman of the Board and Executive Chairman. “We are
encouraged to continue advancing the proprietary technology of our
AIM™ vector platform, especially following recent promising data
presented across multiple indications, for which we continue to
evaluate avenues of development internally, and with potential
partners.”
About Abeona Therapeutics
Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical
company developing gene and cell therapies for serious diseases.
The Company’s clinical programs include EB-101, its autologous,
gene-corrected cell therapy for recessive dystrophic epidermolysis
bullosa, as well as ABO-102 and ABO-101, novel NAV® AAV9-based
gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and
MPS IIIB), respectively. The Company’s portfolio of AAV9-based gene
therapies also features ABO-202 and ABO-201 for CLN1 disease and
CLN3 disease, respectively. Its preclinical assets include ABO-401,
which uses the novel AIM™ AAV vector platform to address all
mutations of cystic fibrosis. Abeona has received numerous
regulatory designations from the FDA and EMA for its pipeline
candidates and is the only company with Regenerative Medicine
Advanced Therapy designation for two candidates (EB-101 and
ABO-102). For more information, visit
www.abeonatherapeutics.com.
Forward Looking StatementThis
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. These
statements include but are not limited to statements related to our
estimates regarding expenses, future revenues, capital
requirements, and needs for additional financing; our ability to
raise capital; our ability to fund our operating expenses and
capital expenditure requirements for at least the next
12 months with our existing cash and cash equivalents; our
expectation that we will continue to incur losses; our belief that
we will expend substantial funds to conduct research and
development programs; our future ability to achieve profitability
at all or on a sustained basis; our cash burn rate; the dilutive
effect that raising additional funds by selling additional equity
securities would have on the relative equity ownership of our
existing investors; our belief that we have a rich pipeline of
products and product candidates; our ability to continue to develop
our novel adeno-associated virus (“AAV”)-based gene therapy
platform technology to treat neurologic disorders, cystic fibrosis
and eye disorders in human subjects; our belief that EB-101 could
potentially benefit patients with recessive dystrophic
epidermolysis bullosa (“RDEB”); positive feedback from regulators
on our manufacturing of clinical trial product for EB-101; our
ability to initiate a Phase III clinical trial for patients with
RDEB; our ability to complete enrollment of patients into clinical
trials to secure sufficient data to assess efficacy and safety; our
belief that AAV treatment could potentially benefit patients with
Sanfilippo syndrome type A (“MPS IIIA”) and Sanfilippo syndrome
type B (“MPS IIIB”); our ability to add clinical sites and identify
additional patients for our Phase I/II clinical trial for patients
with MPS IIIA and MPS IIIB; our ability to continue to secure and
maintain regulatory designations for our product candidates; our
ability to develop manufacturing capability compliant with current
good manufacturing practices for our product candidates; our
ability to manufacture gene therapy products and produce an
adequate product supply to support clinical trials and potentially
future commercialization; our ability to secure timely regulatory
review related to our clinical program; our belief in the adequacy
of the data from clinical trials in EB-101 and expansion cohort of
our Phase I/II clinical trial in ABO-102 (AAV-SGSH) for MPS IIIA,
together with the data generated in the program to date, to support
regulatory approvals; our intellectual property position and our
ability to obtain, maintain and enforce intellectual property
protection and exclusivity for our proprietary assets; the rate and
degree of market acceptance of our product candidates for any
indication once approved; 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; our ability to meet our obligations contained in
license agreements to which we are party; and the terms of future
licensing arrangements or collaborations. We have attempted
to identify forward looking statements by such terminology as
“may,” “will,” “anticipate,” “believe,” “estimate,” “expect,”
“intend,” 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: continued interest in our rare disease portfolio,
our ability to submit protocols and protocol amendments to
regulatory agencies, our ability to initiate and enroll
patients in clinical trials, the adequacy of manufacturing
capabilities, the impact of competition, the ability to secure
licenses or establish intellectual property rights for any
technology that may be necessary to continue to develop and
commercialize our products, 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, and other risks as may be
detailed from time to time in the Company’s annual reports on Form
10-K and quarterly reports on Form 10-Q and other reports filed by
the Company with the Securities and Exchange Commission. The
Company undertakes no obligation to revise the forward-looking
statements or update them to reflect events or circumstances
occurring after the date of this presentation, whether as a result
of new information, future developments or otherwise, except as
required by the federal securities laws.
Investor Contact: Sofia Warner Senior Director,
Investor Relations Abeona Therapeutics +1 (646)
813-4710swarner@abeonatherapeutics.com
Media Contact: Scott Santiamo Director,
Corporate Communications Abeona Therapeutics +1 (718) 344-5843
ssantiamo@abeonatherapeutics.com
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