Abeona Therapeutics, Inc. Cleveland, OH

Abeona Announces $13.85M Grant from Leading Sanfilippo Syndrome Foundations for Clinical Development of MPS III Gene Therapies
  • Nine global foundations collaborate to grant $13.85 million for the continued advancement of lead Sanfilippo gene therapy programs
  • Company receives infusion of an additional $5 million from exercise of outstanding warrants, totaling $18.85 million in proceeds

NEW YORK and CLEVELAND, Oct. 16, 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 today a collaborative agreement between nine Sanfilippo foundations to provide approximately $13.85 million of grants to Abeona in installments for the advancement of the Company’s clinical stage gene therapies for Sanfilippo Syndrome Type A (MPS IIIA) and Sanfilippo Syndrome Type B (MPS IIIB).

“Abeona is pleased to continue our global collaboration with the Sanfilippo foundations to help further advance our gene therapy programs for MPS III disease,” said Timothy J. Miller, Ph.D., president and chief executive officer of Abeona Therapeutics.  “The effort and expertise that we continue to commit to the ABO-102 and ABO-101 programs puts us in a strong position to further extend the important progress reported to date.  We are grateful to the foundations for their ongoing commitment to identifying and facilitating the development of clinical innovation to treat patients with MPS III disease.”

“Stop Sanfilippo considers that, based on the very good clinical data recently published by Abeona on the Phase I/II trial, this is a great opportunity to support a further step on this program making it possible to treat more patients and allowing a broader clinical indication for this potential gene therapy potential treatment,” said Emilio Lopez Alvarez, President of Stop Sanfilippo in Spain.

Team Sanfilippo Foundation, Stop Sanfilippo Fundación, Fundación Sanfilippo B, Sanfilippo Children’s Foundation, the National MPS Society, the Red Sanfilippo Foundation, the Children’s Medical Research Foundation, Abby Grace Foundation, and Fondation Sanfilippo Suisse collectively collaborated on the grant to Abeona.

“The importance of reducing the heparan sulfate as a cause of disease burden cannot be understated, and the clinical data demonstrated by Abeona enabled us to provide additional support in the pursuit of finding new paradigms to treat all children with Sanfilippo syndrome,” stated Carl Kapes, Board Member of Team Sanfilippo.

Additionally, Abeona received $5.0 million through the cash exercise of 625,000 common stock purchase warrants.  Each warrant was exercised to purchase one share of common stock for $8.00 per share. The warrants were issued as part of a $10.0 million financing completed in May 2015 and would have otherwise been exercisable until November 2017.

Abeona Therapeutics Enrolls First Two Patients in Pivotal Expansion of Phase 1/2 Clinical Trial in MPS IIIA
  • ABO-102 Patients Enrolled in Cohort 3 at 3E13 vg/kg
  • Accelerated enrollments begin with first Patient Enrolled at clinical site in Australia; Patient Screening at Spain site underway
  • Day 30 results demonstrate 67% Heparan sulfate reduction in the CNS and 92% reduction in urinary heparan sulfate and normalization of 76% points in liver volume

NEW YORK and CLEVELAND, Oct. 11, 2017 (GLOBE NEWSWIRE) — Abeona Therapeutics Inc. (Nasdaq:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced today that two patients were enrolled in the Company’s ABO-102 Phase 1/2 clinical trial, at sites in Australia and the US. Both patients have been treated with the Company’s Cohort 3 dose of ABO-102 (3 x 1013vg/kg).

“The recently announced one-year data on our Cohort 1 patients showed durable, time-dependent responses as measured in reductions of heparan sulfate storage pathology in the CSF and urine, reduction in liver volume, stabilization of deep brain architecture and signals of stabilization of neurocognitive decline one-year post-administration. After seeing dose-dependent improvements in Cohort 2, with ABO-102 being well tolerated to date, Abeona, together with our principal investigators, dose-escalated to potentially enhance clinical benefits and prolong durability; a decision supported by the regulatory agencies across the three countries supporting our trial,” stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. He continued, “ABO-102 continues to be well-tolerated at all doses at all follow up timeframes, and has enabled an accelerated enrollment schedule over the coming months. We look forward to reporting additional clinical data in the ABO-102 global trial later this year.”

Per the design of the pivotal expansion, subjects in the ABO-102 trial receive a single, intravenous injection of ABO-102 to systemically deliver the AAV viral vector throughout the body and CNS, introducing a corrective copy of the SGSH gene that underlies the cause of the MPS IIIA disease. Subjects are evaluated at multiple time points post-injection for safety assessments and initial signals of biopotency and clinical activity, which indicate that ABO-102 successfully reached target tissues throughout the body, including the central nervous system.

Biopotency Assessments: ABO-102 demonstrated a significant reduction of the sugar molecule that is the hallmark of the diseases in the CNS, heparan sulfate (HS) in the first Cohort 3 subject that has reached 30 days post-injection:

            –66.7% reduction in CSF heparan sulfate
–92.3% reduction in urinary heparan sulfate

Biophysical Assessments: Hepatomegaly – the first Cohort 3 subject through 30 days post-injection demonstrated a 76% reduction in liver volume.

“From numerous pre-clinical, clinical and natural history studies of many rare neurodegenerative conditions, including MPS IIIA and other lysosomal storage diseases, it is strongly suggested that treatment outcomes will be improved by earlier intervention, at the highest possible tolerated doses,” stated Juan Ruiz, M.D., Ph.D., Abeona’s Chief Medical Officer. “We remain encouraged by the early outcome data of all enrolled patients that shows a clear dose-dependent and time-dependent response to ABO-102, and a good safety profile to date,” he continued.

Abeona enrolled the first patient at Adelaide Women’s and Children’s Hospital in Adelaide, Australia and the additional patient at the US site, Nationwide Children’s Hospital.  The Company also commenced patient screening at the Spain clinical site, Clinico Universitario de Santiago de Compostela.

“We are pleased to initiate our enrollment in the ABO-102 trial,” stated Nick Smith, M.D., Ph.D, Principal Investigator and Department Head of Neurology at the Adelaide Women’s & Children’s Hospital in Australia. “MPS IIIA is a profoundly disabling and progressive neurodegenerative disease with no approved treatments available. The encouraging clinical data from all cohorts to date, continues to support a whole-body treatment approach using an intravenously delivered AAV to deliver and remove disease pathology in multiple organs of the body, particularly the brain. We are grateful to the many patient foundations and parents who have supported the research needed to advance a potential treatment for this devastating unmet medical need.”

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.

Abeona Therapeutics Announces Dedication of Commercial Gene Therapy Manufacturing Facility in Cleveland, Ohio
  •  Facility to support development of advanced gene and cell therapies for treatment of life-threatening rare diseases
  • Leaders from local government, life sciences and medical technology to join on October 4 for dedication of first anticipated gene and cell therapy production center in Ohio

CLEVELAND, Oct. 04, 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 today the ground-breaking of the first anticipated commercial gene therapy manufacturing facility in Ohio. The Cleveland-based facility, named The Elisa Linton Center for Rare Disease Therapies, will have the capacity to produce advanced gene and cell therapies to treat serious and debilitating rare diseases. The dedication and ground-breaking ceremony is being held today, October 4, 2017.

“We are very excited to announce the creation of The Elisa Linton Center for Rare Disease Therapies, which will be a global resource for production of gene therapies with the potential to bring new treatments to rare disease patients around the world,” said Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. “It is especially fitting that this center is named for Elisa Linton, who was born with Sanfilippo syndrome, a rare terminal disease. The memory of Elisa and courage of her family continue to be a great inspiration to all members of the rare disease community.”

The Elisa Linton Center for Rare Disease Therapies will initially be used to produce ABO-101 and ABO-102, investigational gene therapies currently in development at Abeona for the treatment of patients with Sanfilippo syndrome, and EB-101, an investigational autologous cell therapy for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare and devastating skin disorder. The Center will also house Abeona’s expanded viral-vector lab, which will develop and produce unique and proprietary vectors used for the delivery of gene therapies. The 6,000 square foot Center will be built-out and validated over the next 12 months.

“The Epidermolysis Bullosa Medical Research Foundation, along with the EB Research Partnership, is a longtime supporter of research that may help patients living with EB including the development of EB-101. The development of EB-101 is a great example of the progress we are making in EB research today,” said Paul Joseph, Chief Financial Officer of the Epidermolysis Bullosa Medical Research Foundation. “We are very pleased to support Abeona taking steps to establish a world-class gene therapy production facility that will bring new hope to people living with EB and other serious diseases and conditions.”

Several leaders from local government and life sciences are scheduled to attend the dedication ceremony of the Center, including representatives from United States Senator Sherrod Brown’s office, Case Western Reserve University, BioEnterprise, MidTown Cleveland, Inc. and JumpStart Inc.

“Today’s groundbreaking celebration represents another successful investment in Ohio innovation and manufacturing,” said Senator Brown. “It’s exciting to see investments in gene therapy treatments for rare diseases happening right here in Cleveland, helping to cement Ohio’s leadership in the healthcare and technology industries. Today’s ceremony is another step forward for the health of rare disease patients and for the strength of the region’s economy.”

“We applaud Abeona’s decision to open a state-of-the-art gene therapy facility in Cleveland’s Health-Tech Corridor,” said Aram Nerpouni, President and CEO of BioEnterprise. “The Cleveland bioscience industry has grown remarkably in the past decade and Abeona’s investment is a potent example of our region’s momentum. We look forward to our ongoing relationship with Abeona and other emerging bioscience companies that continue to grow and strengthen our local economy.”

About EB Medical Research Foundation: As the leader in research funding, the Epidermolysis Bullosa Medical Research Foundation is an all-volunteer, non-profit 501(c) Foundation dedicated to funding research for Epidermolysis Bullosa to determine its causes, develop successful treatments, and ultimately find a cure. EB is a rare, debilitating and often fatal genetic disorder which causes painful scarring and blistering of the skin and internal organs. Our continued goal is to raise awareness through special events, the media and fundraising programs. www.ebmrf.org

About BioEnterprise: BioEnterprise is a business formation, recruitment, and acceleration effort designed to support the growth of bioscience companies. Located in Cleveland, BioEnterprise provides management counsel and support services to health IT, medical device, and biopharmaceutical companies. BioEnterprise founders are Case Western Reserve University, Cleveland Clinic, and University Hospitals. Additional technology partners include the NASA Glenn Research Center, Cleveland State University, and BioOhio. The initiative comprises the collective activities of BioEnterprise and its partners’ commercialization offices: The Case Office of Technology Transfer, Cleveland Clinic Innovations, and University Hospitals Cleveland – Center for Clinical Research. The combined efforts of these groups has created, recruited, and accelerated more than 300 companies in 15 years. For more information: www.BioEnterprise.com.

Abeona Therapeutics Announces Data Safety Monitoring Board Approves ABO-102 Dose Escalation for Second Cohort in Phase 1/2 Clinical Trial for Sanfilippo Syndrome Type
  • ABO-102 (AAV-SGSH) delivers first-in-man AAV-based gene therapy administered by single intravenous injection to treat CNS and peripheral manifestations of Sanfilippo syndrome type A
  • Data Safety Monitoring Board (DSMB) approves ABO-102 dose escalation for the high-dose cohort

NEW YORK, NY and CLEVELAND, OH, 10/05/16 — Abeona Therapeutics Inc. (NASDAQ: ABEO), a clinical-stage biopharmaceutical company focused on developing gene therapies for life-threatening rare diseases, announced today that the Data Safety Monitoring Board (DSMB), an independent group of medical experts closely monitoring the clinical trial, has reviewed the initial safety data from the low dose cohort (n=3) in the Phase 1/2 clinical trial of ABO-102 (AAV-SGSH) enrolling at Nationwide Children’s Hospital in Columbus, Ohio. Following review of the safety data, the DSMB authorized that the clinical trial proceed with enrollment and dose escalation for the second cohort. The high-dose cohort will enroll up to six additional patients dosed at 1.0 X 1013 vg/kg, which is twice the amount of ABO-102 received by patients in the low-dose cohort.

“These early results support Abeona’s unique approach to treating patients with Sanfilippo syndrome, where there are both profound CNS and whole body manifestations of the disease,” stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. “We look forward to reporting on future progress and potential for ABO-102 as we begin to enroll patients at the high dose and open additional clinical sites internationally.”

Abeona’s ABO-102 program has been granted both Orphan Product Designation and Rare Pediatric Disease Designation in the USA and plans to open two additional clinical sites, one in Spain and one in Australia, to test ABO-102. A Phase 1/2 clinical study of ABO-102 in Spain was recently approved by the Agencia Espanola de Medicamentos y Productos Sanitarios, and the Company is preparing to conduct this clinical study at Cruces University Hospital in Bilbao, Spain.

Sanfilippo Syndrome Type A, or MPS IIIA, is a rare lysosomal storage disease caused by genetic mutations that result in a deficiency of SGSH enzyme activity, leading to abnormal accumulation of certain sugars (specifically, the glycosaminoglycan (GAG) heparan sulfate) in the central nervous system (CNS) and systemic tissues and organs. The accumulation of heparan sulfate results in neurocognitive decline, speech loss, loss of mobility, and premature death.

About ABO-102 (AAV-SGSH): ABO-102 is an adeno-associated viral (AAV)-based gene therapy for patients with MPS IIIA (Sanfilippo syndrome), that is delivered as a one-time intravenous injection. ABO-102 delivers a functioning, corrective copy of the SGSH gene to cells of the central nervous system (CNS) and other organs with the goal of correcting the underlying deficits caused by the inborn genetic errors that are the cause the disease. ABO-102 has been well tolerated through 30 day post-injection in subjects injected with the low-dose (n=3). Encouraging signs of early biopotency have been observed in urinary and CSF GAG (glycosaminoglycan, specifically, heparan sulfate) measurements, as well as potential disease-modifying effects in the liver and spleen of the initial subjects enrolled and treated in the trial. The clinical study is supported by neurocognitive evaluations, biochemical assessments and MRI data generated in a 25-subject MPS III Natural History Study, also conducted at Nationwide Children’s Hospital, where patients continued through one-year of follow up assessments.

About Sanfilippo syndromes (or mucopolysaccharidosis (MPS) type III): a group of four inherited genetic diseases each caused by a single gene defect, described as type A, B, C or D, which cause enzyme deficiencies that result in the abnormal accumulation of glycosaminoglycans (GAGs, or sugars) in body tissues. MPS III is a lysosomal storage disease, a group of rare inborn errors of metabolism resulting from deficiency in normal lysosomal function. The incidence of MPS III (all four types combined) is estimated to be 1 in 70,000 births. Mucopolysaccharides are long chains of sugar molecule used in the building of connective tissues in the body. There is a continuous process in the body of replacing used materials and breaking them down for disposal. Children with MPS III are missing an enzyme, which is essential in breaking down the used mucopolysaccharides called heparan sulfate. The partially broken down mucopolysaccharides remain stored in cells in the body causing progressive damage. In MPS III, the predominant symptoms occur due to accumulation within the central nervous system (CNS), including the brain and spinal cord, resulting in cognitive decline, motor dysfunction, and eventual death. Importantly, there is no cure for MPS III and treatments are largely supportive care.

About Abeona: Abeona Therapeutics Inc. is a clinical stage company developing gene and plasma-based therapies for life-threatening rare genetic diseases. Abeona’s lead programs are ABO-102 (AAV-SGSH) and ABO-101 (AAV-NAGLU), adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome (MPS IIIA and IIIB), respectively. Abeona is also developing EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL); ABO-202 (AAV-CLN1) gene therapy for treatment of infantile Batten disease (INCL), and ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder 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 our proprietary SDF™ (Salt Diafiltration) ethanol-free process. For more information, visit www.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, our plans for continued development and internationalization of our clinical programs in Spain and Australia; that early results support our unique approach to treating patients with Sanfilippo syndrome; that encouraging signs of early biopotency have been observed as well as potential disease-modifying effects in the liver and spleen of the initial subjects enrolled and treated in the trial; and that ABO-102 is well-tolerated through 30 day post-injection in subjects injected with the low dose; These 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 ability to successfully continue our clinical trials; the impact of competition; the ability to develop our products and technologies; the ability to achieve or obtain necessary regulatory approvals; the impact of changes in the financial markets and global economic conditions; and other risks as may be detailed from time to time in the Company’s Annual Reports on Form 10-K 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.

Cleveland, OH, May 24, 2016  Abeona Therapeutics Inc. (NASDAQ: ABEO), a clinical-stage biopharmaceutical company focused on delivering gene therapy and plasma-based products for severe and life-threatening rare diseases, today announced the FDA has allowed an Investigational New Drug (IND) Application for its Phase 1/2 Clinical Study with gene therapy candidate ABO-101 (AAV-NAGLU) for patients with Sanfilippo syndrome type B (MPS IIIB) to be conducted at Nationwide Children’s Hospital (Columbus, OH). This is the second FDA allowance for a gene therapy trial from Abeona this year, following allowance of an IND in February for ABO-102, for patients with MPS IIIA which commenced with dosing of the first cohort of patients this month.

“We’re very excited to bring decades of research into clinical trials for this unmet clinical need,” noted Kevin M. Flanigan, M.D., principal investigator with the Center for Gene Therapy at Nationwide Children’s and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine. “As seen in other gene therapy trials, using AAV9 delivered by intravenous injection has strong potential to treat patients with Sanfilippo type B, a disease with profound central nervous system manifestations.”

“Abeona is committed to building a leadership position in the development of innovative treatments for orphan diseases, and this second FDA IND allowance represents an important milestone in advancing our rare disease product pipeline to the clinic,” stated Steven H. Rouhandeh, Executive Chairman of Abeona. “We remain active from a corporate development perspective in identifying complementary orphan drug programs as well as potential co-development partners, towards our goal of achieving value for our patients, their families, our academic partners and other stakeholders.”

A Natural History Study for Sanfilippo, conducted by Nationwide Children’s Hospital, has been completed in 25 patients to characterize how the rare disease progresses in its natural state. Information about a rare disease’s natural history aids in clinical trial design, identifying study end points and developing and validating clinical outcome measures, and biomarkers.

“ABO-101 is Abeona’s second AAV-based gene therapy program to advance to clinical trials. This first-in-man Phase 1/2 clinical trial is delivered by a single intravenous injection to treat the brain and peripheral manifestations of Sanfilippo. In pre-clinical models, an injection of ABO-101 restored the NAGLU enzyme activity in the cerebral spinal fluid and serum, and also corrected the lysosomal storage pathology throughout the CNS and in widespread somatic organs,” noted Timothy J. Miller, Ph.D., President & CEO. “A single injection also led to the correction of astrocytosis and neurodegeneration, hallmarks of secondary damage in the central nervous system in MPS IIIB. Importantly, intravenous gene delivery improved both cognitive and motor functions, as well as extended survival in preclinical models. We are very encouraged by the results seen in other clinical trials and look forward to further building on the positive results seen in previous studies. Given the high level of need for therapies for Sanfilippo syndromes, we remain committed to advancing both our AAV-based gene therapy programs, ABO-101 and ABO-102, targeting two types of the inherited genetic disease, MPS IIIB and MPS IIIA.”

“The collective efforts of multiple foundations have led us to this great achievement, and we are grateful for the groundbreaking research of Drs. McCarty and Fu. We thank Nationwide and Abeona for helping advance potential MPS IIIB gene therapies for our kids,” said Susan Wilson, Director of The Children’s Medical Research Foundation.

“This milestone is the culmination of more than a decade of research,” said Haiyan Fu, Ph.D., the developer of the gene therapy with Doug McCarty, Ph.D. both principal investigators at Nationwide Children’s. “It was achieved through the support of a translational research grant from the National Institutes of Neurologic Disease and Stroke. In addition to the NINDS, our entire team would like to thank many patient and family foundations for their longstanding support and financial commitment to advancing research and developing treatments for this heartbreaking disease.”

About Sanfilippo syndromes: Sanfilippo syndromes (or mucopolysaccharidosis (MPS) type III) are a group of four inherited genetic diseases each caused by a single gene defect, described as type A, B, C or D, which cause enzyme deficiencies that result in the abnormal accumulation of glycosaminoglycans (sugars) in body tissues. MPS III is a lysosomal storage disease, a group of rare inborn errors of metabolism resulting from deficiency in normal lysosomal function. The incidence of MPS III (all four types combined) is estimated to be 1 in 70,000 births. Mucopolysaccharides are long chains of sugar molecule used in the building of connective tissues in the body. There is a continuous process in the body of replacing used materials and breaking them down for disposal. Children with MPS III are missing an enzyme which is essential in breaking down the used mucopolysaccharides called heparan sulfate. The partially broken down mucopolysaccharides remain stored in cells in the body causing progressive damage. Babies may show little sign of the disease, but as more and more cells become damaged, symptoms start to appear. In MPS III, the predominant symptoms occur due to accumulation within the central nervous system (CNS), including the brain and spinal cord, resulting in cognitive decline, motor dysfunction, and eventual death. Importantly, there is no cure for MPS III and treatments are largely supportive care.

About ABO-101 (AAV-NAGLU): ABO-101 is next generation adeno-associated viral (AAV)-based gene therapy for MPS III (Sanfilippo syndrome), which involves a one-time delivery of a normal copy of the defective gene to cells of the central nervous system with the aim of reversing the effects of the genetic errors that cause the disease. After a single dose in Sanfilippo preclinical models, ABO-101 induced cells in the CNS and peripheral organs to produce the missing enzymes and help repair damage caused to the cells. Preclinical in-vivo efficacy studies in Sanfilippo syndrome have demonstrated functional benefits that remain for months after treatment. A single dose of ABO-101 significantly restored normal cell and organ function, corrected cognitive defects that remained months after drug administration, increased neuromuscular control and increased the lifespan of animals with MPS III over 100% one year after treatment compared to untreated control animals. These results are consistent with studies from several laboratories suggesting AAV treatment could potentially benefit patients with for Sanfilippo syndrome Type A and B, respectively. In addition, safety studies conducted in animal models of Sanfilippo syndromes have demonstrated that delivery of AB0-101 are well tolerated with minimal side effects.

About Abeona: Abeona Therapeutics Inc. delivers gene therapy and plasma-based products for severe and life-threatening rare diseases. Abeona’s lead programs are ABO-101 (AAV-NAGLU) and ABO-102 (AAV-SGSH), adeno-associated virus (AAV)-based gene therapies for Sanfilippo syndrome (MPS IIIB and IIIA). The company is also developing ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JBD); and ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder using a novel CRISPR/Cas9-based gene editing approach to gene therapy program for rare blood diseases. In addition, Abeona is developing plasma protein therapies 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

About The Research Institute at Nationwide Children’s Hospital
Ranked 9th of only 12 children’s hospitals on U.S. News & World Report’s 2015-16 “America’s Best Children’s Hospitals Honor Roll,” Nationwide Children’s Hospital is one of the nation’s largest not-for-profit freestanding pediatric healthcare networks providing care for infants, children and adolescents as well as adult patients with congenital disease. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s faculty train the next generation of pediatricians, scientists and pediatric specialists. The Research Institute at Nationwide Children’s Hospital is one of the Top 10 National Institutes of Health-funded free-standing pediatric research facilities in the U.S., supporting basic, clinical, translational and health services research at Nationwide Children’s. The Research Institute encompasses three research facilities totaling 525,000 square feet dedicated to research. More information is available at NationwideChildrens.org/Research.

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 plans for continued development and internationalization of our clinical programs, management plans for the Company, and general business outlook. These 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 develop our products and technologies; the ability to achieve or obtain necessary regulatory approvals; the impact of changes in the financial markets and global economic conditions; and other risks as may be detailed from time to time in the Company’s Annual Reports on Form 10-K 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.

Cleveland, OH, March 24, 2016 – Abeona Therapeutics announced that the Phase I/II clinical trial for MPS IIIA at Nationwide Children’s Hospital in Columbus, OH has now gone live on the clinicaltrials.gov website.  Please click here for eligibility criteria and contact information.