Full Press Release Details
Abeona Therapeutics Reports First Quarter
2018 Financial Results and Business Highlights
Investor Conference Call on Monday, May 14th
NEW YORK and CLEVELAND - May 11,
2018: Abeona Therapeutics Inc. (NASDAQ: ABEO), a leading clinical- stage biopharmaceutical company focused on developing novel
cell and gene therapies for life-threatening rare genetic diseases, today announced financial results for the first quarter of
2018. The Company will host a call to update investors on recent clinical developments and quarter financial results on Monday,
May 14th at 10:00 am (Eastern). Interested parties are invited to participate in the call by dialing 877-407-9210 (toll free
domestic) or 201-689-8049 (International) or via webcast http://www.investorcalendar.com/event/29196.
"Abeona has made significant
progress across multiple fronts, including various regulatory achievements, additional enrollments in our MPS IIIA trial, and the
reporting of encouraging initial results for our MPS IIIB trial," stated Carsten Thiel, Ph.D., Abeona's CEO. "Notably,
both our lead clinical programs, EB-101 and ABO-102, were recently granted RMAT designation by the FDA, supporting the strong safety
and biopotency evidence from these programs, and their potential to address the unmet medical need for RDEB and MPS IIIA patients."
1st Quarter Summary Financial
Abeona Recent Highlights:
"2018 continues to be a year of execution
for Abeona. With the expanded leadership, we look to a number of milestones in the year including the commencement of our pivotal
Phase 3 trial in EB, the completion of enrollment in our Phase 1/2 trial in MPS IIIA, and significant progress on our in-house
GMP manufacturing facility," stated Steven H. Rouhandeh, Abeona's Executive Chairman. "With the team focusing
their efforts and leading the charge on clinical development, we look forward to our continued dialogue with the regulatory bodies
and advancing our gene therapies to rare disease patients."
About Abeona: Abeona
Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for life-threatening rare genetic
diseases. Abeona's lead programs include EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB),
ABO-102 (AAV-SGSH), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and ABO-101 (AAV-NAGLU),
an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type B (MPS IIIB). Abeona is also developing ABO-201
(AAV-CLN3) gene therapy for CLN3 disease, ABO-202 (AAV-CLN1) for treatment of CLN1 disease, EB-201 for epidermolysis bullosa (EB),
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 is developing a proprietary vector platform, AIM , for next generation
product candidates. For more information, visit www.abeonatherapeutics.com.
Christine Silverstein
SVP, Investor Relations & Finance
Abeona Therapeutics Inc.
Berry & Company Public Relations
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, without
limitation, statements about our ability to develop our products and technologies; our plans for continued development and internationalization
of our clinical programs; our expectation to commence our pivotal Phase3 trial in EB; that patients will continue to be identified,
enrolled, treated and monitored in the EB-101 clinical trial, and that studies will continue to indicate that EB-101 is well-tolerated
and may offer significant improvements in wound healing; the completion of enrollment in our Phase 1/2 trial in MPS IIIA; we have
recently initiated enrollment in our MPS IIIB program; our expectation that we will continue to advance our gene therapy for MPS
IIIA patients; and our goal of achieving significant progress on our in-house GMP manufacturing facility. Such 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 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; 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 and the increased
reductions in CNS GAG support our approach for intravenous delivery for subjects with Sanfilippo syndromes, 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 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