Abeona Therapeutics Reports Fourth Quarter 2017 Financial Results and Business Highlights
March 16 2018 - 5:10PM
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™ 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™,
for next generation product candidates. For more information,
visit www.abeonatherapeutics.com.
Investor Contact:Christine
SilversteinSVP, Investor Relations & FinanceAbeona Therapeutics
Inc.+1 (646) 813-4707csilverstein@abeonatherapeutics.com
Media Contact:Lynn GranitoBerry
& Company Public Relations+1 (212)
253-8881lgranito@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.
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