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Msg  55 of 58  at  7/27/2021 8:12:27 PM  by

jerrykrause


Abeona Therapeutics Announces New MRI Data Showing Increased Brain Volume in Young Patients with Sanfilippo Syndrome Type A (MPS IIIA) After Treatment with ABO-102 Gene Therapy

 Pharma & Healthcare Monitor Worldwide
 
 

Abeona Therapeutics Announces New MRI Data Showing Increased Brain Volume in Young Patients with Sanfilippo Syndrome Type A (MPS IIIA) After Treatment with ABO-102 Gene Therapy

 
 

Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene therapy, today announced magnetic resonance imaging (MRI) data from the Phase 1/2 Transpher A clinical study indicating that ABO-102 increased grey matter, corpus callosum and amygdala volumes in the brain in three young patients with Sanfilippo Syndrome Type A (MPS IIIA) at 24 months as compared to afflicted patients without treatment. The new data was presented during an oral presentation at the 16th International Symposium on MPS and Related Diseases.

Brain volume loss is characteristic in children with MPS IIIA and is associated with long-term cognitive and physical disability. Specifically, grey matter is important for cognitive development, corpus callosum for motor function, and amygdala for fear learning as well as social/emotional development, said Vishwas Seshadri, Ph.D., M.B.A., Head of Research & Clinical Development of Abeona. The new MRI data shows the potential of ABO-102 to increase brain grey matter, corpus callosum and amygdala volumes and is consistent with previously reported results of preservation of neurocognitive development in these three young patients in the Transpher A study.

The Transpher A study primary endpoints are neurodevelopment and safety. Secondary endpoints include brain volume, behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and liver volume.

About the Transpher A Study

The Transpher A Study (NCT02716246) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). The study, also known as ABT-001, is intended for patients from birth to 2 years of age, or patients older than 2 years with a cognitive developmental quotient of 60% or above. ABO-102 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

About ABO-102

ABO-102 is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). ABO-102 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations for the ABO-102 clinical program. In the EU, the Company holds PRIME and Orphan medicinal product designations.

About Sanfilippo Syndrome Type A (MPS IIIA)

Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the CNS and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIA present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive facial features with thick eyebrows or a unibrow, full lips and excessive body hair for ones age, and liver/spleen enlargement are also present in early childhood. MPS IIIA is caused by genetic mutations that lead to a deficiency in the SGSH enzyme responsible for breaking down glycosaminoglycans, which accumulate in cells throughout the body resulting in rapid health decline associated with the disorder.

About Abeona Therapeutics

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeonas clinical programs include EB-101, its investigational autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel investigational AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Companys development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeonas novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. Abeonas fully integrated gene and cell therapy cGMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL study and is capable of clinical and planned commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com .

 


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