Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage
biopharmaceutical company focused on developing novel gene
therapies for life-threatening rare diseases, announced financial
results for the first quarter and recent business highlights.
“We are very pleased with our progress during
the first quarter and recent weeks, including the
encouraging results reported from our lead programs, ABO-102
for MPS and EB-101 for RDEB IIIA patients. Along with the
critically reviewed clinical data, the strength of our clinical
programs was also underscored by the achievement of additional EMA
and FDA designations for our gene therapy programs within the
quarter,” stated Timothy J. Miller, Ph.D., President and
CEO.
Abeona Recent Highlights:
May 12, 2017, Abeona Therapeutics announced top-line data for
ABO-102 Phase 1/2 MPS IIIA gene therapy trial at ASGCT --
Positive dose response in central nervous system with 60.7% +/-
8.8% reduction of disease-causing heparan sulfate GAG observed in
Cohort 2 -- Reduction of disease manifestation observed in
decreased liver volume of 14.81% (+/- 1.2%) -- ABO-102
well-tolerated in six subjects through more than 1,100 days
cumulative follow-up with no Serious Adverse Events -- Cohort
1 demonstrated stabilized or improved Leiter Nonverbal IQ scores at
six months
May 9, 2017, Abeona Therapeutics received regulatory approval to
initiate clinical trial in Australia with ABO-102 Gene Therapy For
Patients with MPS IIIA
May 2, 2017, Abeona Therapeutics provided update on EB-101 Phase
1/2a gene therapy for severe form of Epidermolysis Bullosa from the
Society for Investigative Dermatology Conference-- EB-101
demonstrated significant wound healing (defined as greater than 50%
healed) in 100% of treated wounds (36/36) at 3 months; 89% (32/36)
at 6 months, 83% (20/24) at 12 months, 88% (21/24) at 24 months and
100% (6/6) at 36 months post-administration-- Clinical endpoints
supported by data from Natural History Study observations from
1,436 wounds in 128 patients with Recessive Dystrophic
Epidermolysis Bullosa (RDEB)
On March 8, 2017, Abeona announced the European Medicines Agency
(EMA) Committee for Orphan Medicinal Products had granted Orphan
Drug Designation for EB-101 in Epidermolysis Bullosa.
On February 17, 2017, Abeona reported positive data from the
leading clinical gene therapy program for Sanfilippo syndrome type
A patients, ABO-102, demonstrating central nervous system (CNS) and
peripheral organ disease biopotency.
On February 1, 2017, Abeona enrolled the first high-dose subject
in ABO-102 ongoing Phase 1/2 trial in MPS III. ABO-102
received Fast Track Designation and has been granted Orphan Product
Designation in the USA and Europe, and has also received the Rare
Pediatric Disease Designation in the United States. Global
ABO-102 enrollments in Europe and Australia are expected to
commence later this year.
On January 19, 2017, the EMA Committee for Orphan Medicinal
Products granted Orphan Drug Designation (EMA/OD/226/16) for
Abeona’s gene therapy program ABO-101 for children impacted by
Sanfilippo syndrome type B (MPS IIIB). ABO-101 has previously
been granted Orphan Product Designation in the United States and
received the Rare Pediatric Disease Designation as a pre-requisite
part of the FDA’s Priority Review Voucher (PRV) process. The
FDA has allowed the Investigational New Drug (IND) for a Phase 1/2
clinical trial, and enrollments are anticipated to begin later this
year.
On January 3, 2017, the EMA Committee for Orphan Medicinal
Products granted Orphan Drug Designation for Abeona's ABO-201
program (AAV-CLN3), the AAV-based single intravenous gene therapy
program for juvenile Batten disease, a fatal lysosomal storage
disease of the nervous system caused by autosomal-recessive
mutations in the CLN3 gene.
"We started 2017 making meaningful 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) recently and the initiation of our 2nd
international clinical site, along with two year follow-up data
seen in our ongoing EB-101 Phase 2 study in epidermolysis bullosa,
we look forward to continuing our work for 2017.”
1st Quarter Summary Financial
Results:
- Cash position: Cash, cash equivalents and marketable
securities as of March 31, 2017 were $63.2 million, compared to
$69.1 million as of December 31, 2016. Net cash used in operating
activities in the three months ended March 31, 2017 was $5.9
million as compared to $2.5 million in the same period in 2016.-
Revenues: Revenues were $186 thousand for the first quarter of
2017, compared to $235 thousand in the first quarter of 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 $0.13 for the first quarter of 2017,
compared to a loss per share of $0.17 in the comparable period in
2016.
About Abeona: Abeona
Therapeutics Inc. is a clinical-stage biopharmaceutical company
developing gene therapies for life-threatening rare genetic
diseases. Abeona's lead programs include ABO-102 (AAV-SGSH), an
adeno-associated virus (AAV) based gene therapy for Sanfilippo
syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts)
for recessive dystrophic epidermolysis bullosa (RDEB). Abeona is
also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B
(MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten
disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile
Batten disease (INCL), 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 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.
Investor Contact:Christine Silverstein Vice
President, Investor Relations Abeona Therapeutics Inc. +1
(212)-786-6212 csilverstein@abeonatherapeutics.com
Media Contact:Andre’a Lucca Vice President,
Communications & Operations Abeona Therapeutics Inc. +1
(212)-786-6208alucca@abeonatherapeutics.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 the
statement that the ability to develop our products and
technologies; 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 addition of two additional global clinical sites will
accelerate our ability to enroll and evaluate ABO-102 as a
potential treatment for patients with Sanfilippo syndrome type A,
or MPS IIIA. 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, 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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