PlasmaTech Biopharmaceuticals Announces Completion of Sanfilippo Syndrome (MPS IIIB) Toxicology Studies at American Society of Gene and Cell Therapy Annual Meeting
NEW ORLEANS, LA--(Marketwired - May 14, 2015) -
Preclinical Studies Support Strong Safety Profile and Investigational New Drug (Ind) Application Filing for Treating Sanfilippo Syndrome (Mucopolysaccharidosis Iiib), a Rare, Fatal Autosomal Recessive Neurodegenerative Disorder
Clinical Studies Planned to Commence in 2015
PlasmaTech Biopharmaceuticals, Inc. (PTBI), a biopharmaceutical company focused on gene therapy and cell therapy products for severe and life-threatening rare diseases announced today that IND enabling safety study data utilizing their adeno-associated virus (AAV9) gene therapy product was presented at the annual meeting of the American Society for Gene and Cell Therapy. The company recently announced its acquisition of Abeona Therapeutics, which has been developing AAV gene therapies for the treatment of Sanfilippo syndromes (MPS IIIA and MPS IIIB).
Sanfilippo syndrome is an inherited genetic disorder that affects children. Children born with Sanfilippo syndrome experience progressive intellectual and motor disability, speech difficulties, seizures, and premature death. As yet, there are no treatments available that can halt or reverse the symptoms of Sanfilippo syndrome.
"These preclinical studies demonstrate increased enzyme levels in target tissues, a strong safety profile, and support our planned clinical trials," stated Tim Miller, Ph.D., President & CEO of Abeona, and prospective CEO of the combined companies upon closing of the transaction.
About Sanfilippo syndrome (Mucopolysaccharidosis IIIB, MPS IIIB):
Sanfilippo syndrome is a group of 4 deadly genetic diseases resulting from the body's inability to properly break down certain sugars. In the class of lysosomal storage diseases, 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. 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. There is no cure and currently no approved treatments for Sanfilippo syndrome.
MPS IIIB is a progressive neuromuscular disease with profound CNS involvement. In this condition, a build-up of a substance called glycosaminoglycans (GAGs) occurs in the lysosomes of cells, particularly in the central nervous system. This is directly related to the malfunction of the a-N-acetylglucosaminidase (NaGlu) enzyme, resulting in dramatic loss of intellectual ability, and a high premature death.
No FDA-approved treatment is currently available for this devastating disorder. Pre-clinical studies using a single intravenous injection of ABX-B (also known as rAAV9.CMV.hNAGLU), have demonstrated restoration of NaGlu activity and corrected the lysosomal storage pathology (histopathology and GAG content) throughout the CNS and in widespread somatic organs. Importantly, intravenous gene delivery of ABX-B improved cognitive and motor functions and normalized the survival of IIIB mice.
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