Full Press Release Details
Abeona Therapeutics Reports Second Quarter
2019 Financial Results and Business Updates
patients with MPS IIIB treated in the Transpher B Study of ABO-102 gene therapy
interim MPS IIIA data showed preservation of neurocognitive development in youngest patients with robust and sustained improvement
in clinically relevant biomarkers
initiation of Phase 3 VIITALTM study evaluating EB-101 in recessive dystrophic epidermolysis bullosa in Q4
to host investor conference call Monday, August 12 at 10:00 a.m. ET
NEW YORK and CLEVELAND, August 9, 2019 - Abeona Therapeutics
Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced second quarter 2019 financial results
and business updates, which will be discussed on a conference call scheduled for Monday, August 12 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/51859.
"The second quarter was
highlighted by progress made in both of our MPS III programs," said Jo o Siffert, M.D., Chief Executive Officer. "Data
from our Transpher A study showed that children with MPS IIIA who were treated early with ABO-102 preserved neurocognitive development
within the normative range 12-18 months post treatment. Our MPS IIIB program has also progressed, with enrollment of additional
patients in cohort 2 of the Transpher B study. Our team remains highly focused on our lead programs, including the start of our
VIITAL Phase 3 clinical trial in recessive dystrophic epidermolysis bullosa, continued enrollment in the MPS III programs, and
preparations to start the clinical trial in CLN1 disease."
Second Quarter Financial
Cash, cash equivalents and
marketable securities as of June 30, 2019, were $62.5 million compared to $68.3 million as of March 31, 2019. The decrease in cash
was driven primarily by the net cash used in operating activities of $15.2 million.
Research and development expenses
for the second quarter ended June 30, 2019 were $16.3 million compared to $7.9 million in the same period of 2018. The increase
in R&D expense was primarily attributable to increased in-house manufacturing activities and related headcount costs.
General and administrative expenses for the second quarter ended June 30, 2019 were $5.6 million compared to $4.6 million in the same period of
2018. The increase in G&A expenses was primarily due to increased headcount and related facility costs.
Net loss was $0.49 per share
for the second quarter of 2019 compared to $0.26 per share in the same period of 2018.
Second Quarter and Recent
Steven H. Rouhandeh, Abeona's
Executive Chairman, said, "Abeona has continued the development of its breakthrough gene and cell therapies for rare genetic
diseases through 2019 with important regulatory and clinical achievements secured. We look forward to progressing our MPS programs,
and to starting of our Phase 3 VIITAL trial in EB before year end."
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 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 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. These statements include statements
about the Company's clinical trials and its products and product candidates, future regulatory interactions with regulatory
authorities, as well as the Company's goals and objectives. 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 continued interest
in our rare disease portfolio, 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 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, litigation
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 periodic reports filed by the Company 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 presentation, whether as a result of new information, future developments or otherwise, except as required by the federal
Senior Director, Investor Relations
Director, Corporate Communications