NEW YORK and CLEVELAND,
March 16, 2018 (GLOBE NEWSWIRE) -- 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 fourth quarter. The Company will host a call to
update investors on recent clinical developments and year-end
financial results on Tuesday, March 27th 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).
"The past year was marked by
several defining events in the company's history, having advanced
our two lead clinical programs, EB-101 in Epidermolysis Bullosa and
ABO-102 in MPS IIIA, and initiated our third clinical program,
ABO-101 in MPS IIIB. The strong safety and biopotency data
observed in our three active clinical trials and the strategic
initiative of building an in-house commercial GMP manufacturing
facility further strengthens our position in developing gene and
cell therapies to treat these devastating and rare pediatric
diseases," stated Timothy J. Miller, Ph.D., President and CEO.
4th Quarter
and Year-end Summary Financial Results:
- Cash position: Cash, cash equivalents and
marketable securities as of December 31,
2017 were $137.8 million, compared to $56.5
million as of September 30, 2017. Net cash used in
operating activities in the twelve months ended December 31,
2017 was $22.9 million as compared to $13.0
million in the same period in 2016, an increase of $9.9
million.
- Offering: During the fourth quarter, on
October 19, 2017, Abeona closed an underwritten public
offering of 5,750,000 shares of common stock, at a public offering
price of $16.00 per share. The gross proceeds to the
Company were $92 million, before deducting the underwriting
discounts and commissions and estimated offering expenses payable
by the Company.
- Revenues: Revenues were $215
thousand for the fourth quarter of 2017, compared to $256
thousand in the fourth quarter of 2016. Revenues for twelve
months ended December 31, 2017 were $837 thousand,
compared to $889 thousand in the same period in
2016. Revenues consisted of a combination of royalties from
marketed products, primarily MuGard®, and recognition of deferred
revenues related to upfront payments from early license
agreements.
- Loss per share: Loss per share
was $0.19 for the fourth quarter of 2017, compared to a
loss per share of $0.18 in the comparable period in 2016.
Loss per share was $0.66 for the twelve months ended
December 31, 2017, compared to a loss per share
of $0.64 in the same period in 2016.
Abeona
Recent Highlights:
- March 15, 2018: Abeona received FDA Rare
Pediatric Disease Designation for ABO-202 gene therapy program in
CLN1 disease
- February 12, 2018: Received FDA Orphan Drug
Designation for ABO-202 program in CLN1 disease
- February 8, 2018: Reported top-line data from
Phase 1/2 gene therapy trial in MPS IIIA
- ABO-102 results presented at WORLDSymposium for Lysosomal
Diseases showed significant time- and dose-dependent reduction of
underlying disease pathology, including decreased CSF and urine
GAGs (HS fragments) and diminished liver volumes
- Evidence of cognitive benefit at six months post
treatment in Cohort 2 and at one year in Cohort
1
- Company receives FDA allowance to lower
enrollment age to six months
- February 7, 2018: Reported on initial safety and
biopotency signals in MPS IIIB gene therapy clinical
trial
- ABO-101 is well tolerated and demonstrates early
biopotency signals with significant disease-specific heparan
sulfate (HS) reductions in cerebral spinal fluid, urine, and plasma
and greater than 300-fold increase in NAGLU enzyme activity
observed in first subject at 30 days post
injection
- January 29, 2018: Received FDA Regenerative
Medicine Advanced Therapy designation for EB-101 in Epidermolysis
Bullosa
- December 20, 2017: Enrolled first patient in
ABO-101 Phase 1/2 clinical trial for MPS IIIB
- November 9, 2017: Enrolled first subject in Spain
in ongoing Phase 1/2 clinical trial in MPS IIIA
- October 16, 2017: Announced $13.85M grant from
leading Sanfilippo syndrome foundations for clinical development of
MPS III gene therapies
- October 11, 2017: Enrolled first two patients in
the Cohort 3 expansion of the Phase 1/2 clinical trial in MPS
IIIA
- October 6, 2017: Announced top-line one year data
from ABO-102 MPS IIIA Trial at ARM's Cell & Gene Meeting on the
Mesa
- October 4, 2017: Abeona broke ground on GMP
commercial manufacturing facility for cell and gene therapies
"2017 was transformative for
Abeona, with significant progress made towards our goal of building
a strong leadership position in rare disease gene therapy
development, and manufacturing technology and capability," stated
Steven H. Rouhandeh, Executive Chairman. "The momentum will
continue in 2018, with the continued enrollment in the high-dose
cohort in our ABO-102 Phase 1/2 clinical trial in Sanfilippo
syndrome Type A (MPS IIIA), alongside the clinical and regulatory
progress in moving our EB program to a pivotal Phase 3 study and
the additional work on our own proprietary AIM(TM) vector
platform."
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(TM), for next
generation product candidates. For more information, visit
www.abeonatherapeutics.com.
Investor
Contact:
Christine Silverstein
SVP, Investor Relations & Finance
Abeona Therapeutics Inc.
+1 (646) 813-4707
csilverstein@abeonatherapeutics.com
Media
Contact:
Lynn Granito
Berry & Company Public Relations
+1 (212) 253-8881
lgranito@berrypr.com
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
include, without limitation, statements about our ability to
develop our products and technologies; our plans for continued
development and internationalization of our clinical programs; 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 and we plan to initiate a
pivotal Phase III trial early next year; 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.
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 or otherwise.