Abeona Announces Top-Line One Year Data from ABO-102 MPS IIIA Trial at ARM’s Cell & Gene Meeting on the Mesa

--Demonstrated durable and significant reduction of underlying disease pathology across multiple clinical measures in Cohort 1 (n=3) compared to controls (n=8-12)

--Systemic Biopotency demonstrated time- and dose-dependent reductions of disease causing Heparan Sulfate in the Cerebrospinal fluid (CSF) and liver volumes

--Preservation of deep brain architecture observed after intravenous administration

--Stabilization of neurocognitive assessment scores at one year post-injection

NEW YORK and CLEVELAND, Oct. 06, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene and cell therapies for life-threatening rare diseases, announced one year data from Cohort 1 of the ongoing ABO-102 Phase 1/2 trial for Sanfilippo syndrome Type A (MPS IIIA). The results demonstrated robust and durable clinical effects achieved one year post-administration, with significant reductions in biopotency and biophysical measures, preservation of deep brain architecture, and stabilization across multiple neurocognitive assessments reported in comparison to untreated control subjects.  The ongoing gene therapy trial for ABO-102 (AAV-SGSH) is utilizing a single intravenous injection for treatment of subjects with Sanfilippo syndrome Type A (MPS IIIA), a rare autosomal-recessive lysosomal storage disease. Results were announced during the Alliance for Regenerative Medicine’s Cell & Gene Meeting on the Mesa being held this week in La Jolla, CA.

“Abeona continues to advance its world-leading gene therapy for MPS IIIA patients and we are excited about the updated results seen in Cohort 1 one year post-dosing. The observation of  dose- and time- dependent responses in the CSF at 12 months provides strong additional support for our clinical approach, and comparison of data from six treated and fifteen control subjects supports the intravenous delivery approach for patients with a lysosomal storage disease. We are encouraged by the continued reductions in liver volumes, signs of brain architecture preservation and preliminary evidence of stabilized neurocognitive decline,” stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. “We look forward to accelerating enrollment with our active global sites in Spain and Australia and reporting additional clinical data from the ABO-102 global MPS IIIA trial later this year.”

Per the design of the clinical trial, subjects received a single, intravenous injection of ABO-102 to deliver the AAV viral vector systemically throughout the body to introduce a corrective copy of the gene that underlies the cause of the MPS IIIA disease. Subjects were evaluated at multiple time points post-injection for safety assessments and initial signals of biopotency and clinical activity. Results indicate that ABO-102 successfully reached target tissues throughout the body, including in the central nervous system. Highlighted data on three patients treated in Cohort 1 of  the gene therapy trial, compared to 8-15 control subjects, included:

Biopotency Assessments: ABO-102 demonstrated dose- and time-dependent durable reduction in the CNS of heparan sulfate (HS), the sugar molecule that is the hallmark of the disease.

--At one year post-injection, two patients in Cohort 1 demonstrated reduction of 69.3% +/- 5.7% (P<0.001) in cerebral spinal fluid (CSF) heparan sulfate (HS).  One patient in the Cohort was unable to be accessed due to an adverse event unrelated to the therapy. 

Biophysical Assessments: durable biophysical reductions of disease burden with reductions observed in liver and preservation of key deep brain areas observed.

--Hepatomegaly - Cohort 1 subjects demonstrated normalization of liver volumes of 80% (+/- 16.2%) points at one year (P<0.005) post-injection. The natural history study in 25 subjects with MPS III demonstrated that subjects had increased liver volumes averaging 220% at baseline that was maintained over a year of follow-up.
--Brain MRI Data - initial analysis of Cohort 1 patient MRI data showed evidence of stabilization of area of deep brain architecture in the thalamus and putamen (P<0.05) at one year post-administration.

Cognitive Assessments: evidence of cognitive clinical benefit and stabilization at one year in Cohort 1.

--Cognitive assessments at the 12-month time point for Cohort 1 showed evidence of stabilization in the Leiter-R non-verbal IQ (n=2) and Vineland (adaptive behavior) (n=3, P=0.05) scales.

Safety: well-tolerated in all subjects through approximately 2,000 days cumulative post-injection.

--No drug related serious adverse events related to the drug were reported in subjects in the cohort receiving ABO-102 (Cohort 1: 5E12 vg/kg) through over 2,000 cumulative follow-up days.

“We remain encouraged by signs of tolerability and biological effects that we have observed in Cohort 1 one year post-injection,” stated  Juan Ruiz, MD, Ph.D., MBA, Chief Medical Officer, Abeona Therapeutics Inc.  “Importantly, the stabilization of deep brain architecture at 1 year highlights the potential of intravenous route of delivery in accessing the basal ganglia to promote neural stabilization. We are pleased to see continued decreases in CSF HS in the Cohort one year post-administration, along with positive signs of neurocognitive and physical benefits at the low dose.”

The ongoing Phase 1/2 clinical trial, which has received FastTrack designation, Orphan Product Designation, and Rare Pediatric Disease designation by the U.S. Food and Drug Administration, is designed to evaluate safety and efficacy of ABO-102 in patients with MPS IIIA.  The global clinical study is supported by a 25-subject MPS III Natural History Study, (Truxal et. al., 2016, Mol. Genet. Metab.), which included potential efficacy assessments consisting of neurocognitive evaluations, biochemical assays and MRI data generated over one year of follow-up assessments.

About Abeona

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell 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 proprietary vector platform, AIM™, for next generation product candidates.  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:
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, our expectation that we will continue to advance our gene therapy for MPS IIIA patients, our expectation of accelerating enrollment with our active global sites in Spain and Australia, and that we remain encouraged by signs of tolerability and biological effects observed in Cohort 1 post injection.  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.

Released October 6, 2017