Late Breaking News

Late Breaking News
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 A
  • 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.

 

Cleveland, OH, November 15, 2015 – Abeona Therapeutics is focused on bringing forth gene therapy and plasma based products for severe and life threatening rare diseases. The company was created in 2013 through a close collaboration with a dozen Sanfilippo foundations to progress the work of Drs. Douglas McCarty and Haiyan Fu from Nationwide Children’s Hospital in Columbus, Ohio. The collective efforts raised nearly $5 million dollars by the end of 2014 to start drug manufacture and steps to clinical trial.

In May of this year, the company was acquired by PlasmaTech Biopharmaceuticals, a publicly traded organization that had also been focused on treating rare diseases by utilizing a proprietary purification technology for selecting therapeutic proteins from human blood plasma. The newly formed entity chose to return to the Abeona Therapeutics name as it better reflected the commitment to rare or underserved diseases.

Abeona will soon begin the two gene therapy clinical trials for MPS IIIA and MPS IIIB, expected to first enroll in the US at Nationwide Children’s Hospital late this year or early in 2016.  We are very encouraged by the results we have witnessed pre-clinically with regard to the distribution to different areas of the brain, ability to address the systemic manifestations, and reduction of GAG’s. This is to be a one-time dose, delivered by IV and has the ability to cross the blood brain barrier; a lower risk for complication compared to intrathecal or intracranial delivery. Additionally, we have recently added pre-clinical programs to focus to Fanconi’s Anemia and different forms of Batten disease.

Cleveland, OH, December 1, 2014 – Biotechnology startup Abeona Therapeutics announced today it closed $3.6 million to complete preclinical development of therapies for children with Sanfilippo syndrome (mucopolysaccharidosis type III, MPS III), a rare terminal genetic disorder that results in death in children before  they reach their mid-teens. Investors include U.S.-based Cure Sanfilippo Foundation, Sanfilippo Research Foundation (SRF), Team Sanfilippo, the Abby Grace Foundation, and the National MPS Society. Abeona also generated strong support from the international Sanfilippo community, with investments from Spain-based Stop Sanfilippo and Sanfilippo B Foundation, Geneva, Switzerland-based Fondation Sanfilippo and Mexico-based Red Sanfilippo Foundation. Support for the program has also been provided by The Children’s Medical Research Foundation, Inc. (CMRF) and Canadian led Sanfilippo Children’s Research Foundation (SCRF).

“For over a decade, our Sanfilippo research has been supported by Ben’s Dream – SRF, and Cure Kirby – CMRF, and jointly by A Life for Elisa – SCRF, Team Sanfilippo and LivLife Foundation since 2010. We are very thankful that our joint efforts have led to our planned clinical trials in 2015,” said Drs. Douglas McCarty and Haiyan Fu, Abeona’s scientific founders and investigators at Nationwide Children’s Hospital.”

Sanfilippo syndrome is a group of 4 deadly genetic diseases resulting from the body’s inability to properly break down certain sugars. Symptoms often appear in  the first year of life, and the disease causes progressive muscular and cognitive decline in children after the age of two. There is no cure and currently no approved treatments for Sanfilippo syndrome. As such, children afflicted with Sanfilippo syndrome experience progressive loss of speech, the ability to eat and walk, and rarely live past their second decade of life. Abeona Therapeutics is focusing initially on gene therapies for Sanfilippo syndrome types A and B.

“This funding helps support our global development of gene therapies for treating patients with Sanfilippo syndrome. We have formed a very special, international, collaborative effort where Abeona has become the voice of over a dozen patient advocacy groups desiring accelerated access to potential therapies,” explained Tim Miller, Ph.D, Abeona’s President/CEO. “We are pleased to collaborate with ationwide Children’s Hospital in the development of these therapies as we approach clinical trials.”

“We are excited to be working with Abeona and the impressive world-wide collaboration of Sanfilippo foundations they have organized. Their emphasis on developing minimally-invasive treatments is reassuring as a parent. The dedication and approachability of the team has built confidence in the program and allows foundations to feel connected to the pursuit of a cure,” said Kathleen Buckley, President of the Team Sanfilippo Foundation.

Time is a critical factor for children with Sanfilippo syndrome: 70% of the children with the disease will not reach their 18th birthday. “We are poised to initiate clinical trials in early-to-mid 2015 for these much needed therapies,” said Kevin Flanigan, MD, principal investigator, Center for Gene Therapy, and neurologist at Nationwide Children’s Hospital and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine. He is also principal investigator for the ongoing Sanfilippo Natural History Study and upcoming gene transfer trials at Nationwide Children’s.

The collaborative efforts of Abeona, the supporting Sanfilippo foundations, and Nationwide Children’s Hospital were recognized by receipt of the Global Genes “RARE Champion of Hope” Collaborations in Advocacy award in late 2013 and by recent support from the National MPS Society. “The mission of the National MPS Society is to support promising therapies for these devastating, rare diseases, and we are pleased to support Abeona’s progress towards clinical trials, ” said Barbara Wedehase, Executive Director.

Cleveland-based Abeona Therapeutics is a start-up company focused on developing gene therapy based treatments for patients with lysosomal storage diseases, with lead products focusing on MPS IIIA and IIIB. Abeona Therapeutics received Orphan Product Designations for MPS IIIA and IIIB in 2014 and clinical trials are anticipated to begin in early-to-mid 2015.
About Abeona Therapeutics
Abeona Therapeutics – named after the Roman Goddess who is the protector of children – was formed in early 2013 to help focus the search for a cure for Sanfilippo Syndrome and provide a unifying voice between patient advocate groups, researchers, clinicians and investors. Abeona Therapeutics is the result of collaborative efforts between Nationwide Children’s Hospital and multiple international patient advocate groups for developing Sanfilippo therapies. The collaboration has helped focus parents and caregivers on a leading therapy with broad potential to provide long-term benefits to children with Sanfilippo Syndrome. Clinical trials for Sanfilippo types A and B are anticipated to begin in early-to-mid 2015.

 

About Nationwide Children’s Hospital
Ranked 7th of only 10 children’s hospitals on U.S. News & World Report’s 2014-15 “America’s Best Children’s Hospitals Honor Roll” and among the Top 10 on Parents Magazine’s 2013 “Best Children’s Hospitals” list, 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.

 

 

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