Full Press Release Details
Abeona Therapeutics Reports Q4 2016 Financial
Results and Business Highlights
to Host Investor Conference Call on Monday, April 10th at 10:00 am ET to
Provide Progress Report on Corporate Developments
NEW YORK, NY and CLEVELAND, OH -
March 31, 2017 -- Abeona Therapeutics Inc. (NASDAQ: ABEO), a leading clinical-stage biopharmaceutical company focused on developing
novel gene therapies for life-threatening rare genetic diseases, today announced financial results for the fourth quarter. The
Company will host a call to update investors on recent clinical developments and year-end financial results on Monday, April 10th
at 10:00 am (Eastern). Interested parties are invited to participate in the call by dialing 877-269-7756 (toll free domestic) or
201-689-7817 (International).
"The past year has been characterized
by advancement across all clinical programs within Abeona, having achieved many important regulatory and clinical milestones in
2016. Notably, we initiated dosing of our high-dose cohort in ABO-102 this January after demonstrating promising safety and biopotency
in the completed low-dose cohort last year. The strength of our clinical programs was validated not only through critically reviewed
clinical data, but also through the achievement of multiple EMA and FDA designations for our gene therapy programs in 2016. Another
important highlight was the addition of our AIMTM vector platform, which provides
Abeona the ability to leverage a proprietary AAV capsid portfolio for next generation gene therapies across multiple existing and
potential new indications," stated Timothy J. Miller, Ph.D, President and CEO.
Abeona Recent Highlights:
"The fourth quarter of 2016 brought
significant progress in our goal of building a strong leadership position in the development of novel therapies for rare diseases,"
stated Steven H. Rouhandeh, Executive Chairman. "With the positive biopotency data seen in our ABO-102 Phase 1/2 clinical
trial in Sanfilippo syndrome Type A (MPS IIIA) and the continued enrollment of the high dose cohort, along with data seen in our
ongoing EB-101 Phase 2 study in epidermolysis bullosa, and the addition of our own proprietary vector platform, we remain focused
on furthering our regulatory and clinical progress as we continue our work for 2017."
4th Quarter and Year-end Summary Financial
About Abeona: Abeona Therapeutics
Inc. is a leading clinical-stage biopharmaceutical company developing gene therapies for life-threatening rare genetic diseases.
Abeona's lead programs include ABO-102 (AAV-SGSH) and ABO-101 (AAV-NAGLU), adeno-associated virus (AAV) based gene therapies for
Sanfilippo syndrome (MPS IIIA and IIIB, respectively). Abeona is also developing EB-101 (gene-corrected skin grafts) for recessive
dystrophic epidermolysis bullosa (RDEB), EB-201 for epidermolysis bullosa (EB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten
disease (JNCL), ABO-202 (AAV-CLN1) gene therapy for treatment of infantile Batten disease (INCL), and ABO-301 (AAV-FANCC) for
Fanconi anemia (FA) disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood
diseases. In addition, Abeona has a plasma-based protein therapy pipeline, including SDF Alpha (alpha-1 protease inhibitor)
for inherited COPD, using its proprietary SDF (Salt Diafiltration) ethanol-free process. For more information, visit www.abeonatherapeutics.com.
Christine Silverstein
Vice President, Investor Relations
Abeona Therapeutics Inc.
Vice President, Communications & Operations
Abeona Therapeutics Inc.
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 that involve
risks and uncertainties. These statements are subject to 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 impact of competition; the ability
to develop our products and technologies; the ability to achieve or obtain necessary regulatory approvals; the impact of changes
in the financial markets and global economic conditions; our belief that initial signals of biopotency and clinical activity, which
suggest that ABO-102 successfully reached target tissues throughout the body, including the central nervous system; our statement
that global ABO-102 enrollments in Europe and Australia are expected to commence later this year; our belief that the data demonstrate
an early and robust systemic delivery of ABO-102, and the increased reductions in CNS GAG support our approach for intravenous
delivery for subjects with Sanfilippo syndromes, and other risks as may be detailed from time to time in the Company's Annual Reports
on Form 10-K and other reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligations
to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances
occurring after the date of this release, whether as a result of new information, future developments or otherwise.