LEXINGTON, MA, USA I December 15, 2014 I Synageva BioPharma Corp. (GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, announced today that the investigational new drug (IND) application to the U.S. Food & Drug Administration (FDA) to evaluate SBC-103 as a treatment for mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome) is active. The company plans to start enrolling patients with MPS IIIB in a Phase 1/2 study investigating intravenous administration of SBC-103 shortly and to report preliminary data from this study in 2015.
SBC-103 is a recombinant form of the natural human alpha-N-acetyl-glucosaminidase (NAGLU) enzyme that has favorable properties for enabling cellular uptake and has shown the ability to overcome the challenges previously encountered in producing recombinant human NAGLU.
The advancement of SBC-103 towards the clinic is supported by preclinical studies demonstrating that intravenously administered SBC-103 is able to cross the blood-brain barrier and reduce heparan sulfate (HS) storage in the brain and in the liver in an MPS IIIB animal model. In addition, SBC-103 demonstrated transport across an in vitro model of the blood-brain barrier and distributed into the brain in non-human primate studies.
The company recently completed a study assessing the integrity of the blood-brain barrier and other disease abnormalities in patients with MPS IIIB and plans to report data during the first quarter of 2015.
The company is also conducting natural history studies in MPS IIIB. These include a retrospective natural history study of deceased MPS IIIB patients that began in July 2013 and a prospective, longitudinal natural history study in living MPS IIIB patients that began in September 2014. Natural history studies can help build an understanding of the etiology, manifestations and progression of disease and provide insights into biomarkers and other measures of clinical outcome.
SBC-103 was granted orphan designation by the U.S. Food and Drug Administration (FDA) in April 2013 and the European Medicines Agency (EMA) in June 2013.
The mucopolysaccharidoses consist of a group of rare LSDs caused by a deficiency of enzymes needed to break down complex sugars called glycosaminoglycans. The MPS III syndromes (also known as Sanfilippo syndromes) share complications with other MPS diseases but represent a clinically distinct subset with marked central nervous system degeneration. MPS IIIB is caused by a decrease in NAGLU enzyme activity which leads to the buildup of abnormal amounts of HS in the brain and other organs. The accumulation of abnormal HS, particularly in the central nervous system, leads to severe cognitive decline, behavioral problems, speech loss, increasing loss of mobility, and premature death.
Synageva routinely posts information that may be important to investors in the “Investor Relations” section of the company’s website at www.synageva.com. Synageva encourages investors and potential investors to consult this website regularly for important information about the company.
SOURCE: Synageva
Post Views: 251
LEXINGTON, MA, USA I December 15, 2014 I Synageva BioPharma Corp. (GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, announced today that the investigational new drug (IND) application to the U.S. Food & Drug Administration (FDA) to evaluate SBC-103 as a treatment for mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome) is active. The company plans to start enrolling patients with MPS IIIB in a Phase 1/2 study investigating intravenous administration of SBC-103 shortly and to report preliminary data from this study in 2015.
SBC-103 is a recombinant form of the natural human alpha-N-acetyl-glucosaminidase (NAGLU) enzyme that has favorable properties for enabling cellular uptake and has shown the ability to overcome the challenges previously encountered in producing recombinant human NAGLU.
The advancement of SBC-103 towards the clinic is supported by preclinical studies demonstrating that intravenously administered SBC-103 is able to cross the blood-brain barrier and reduce heparan sulfate (HS) storage in the brain and in the liver in an MPS IIIB animal model. In addition, SBC-103 demonstrated transport across an in vitro model of the blood-brain barrier and distributed into the brain in non-human primate studies.
The company recently completed a study assessing the integrity of the blood-brain barrier and other disease abnormalities in patients with MPS IIIB and plans to report data during the first quarter of 2015.
The company is also conducting natural history studies in MPS IIIB. These include a retrospective natural history study of deceased MPS IIIB patients that began in July 2013 and a prospective, longitudinal natural history study in living MPS IIIB patients that began in September 2014. Natural history studies can help build an understanding of the etiology, manifestations and progression of disease and provide insights into biomarkers and other measures of clinical outcome.
SBC-103 was granted orphan designation by the U.S. Food and Drug Administration (FDA) in April 2013 and the European Medicines Agency (EMA) in June 2013.
The mucopolysaccharidoses consist of a group of rare LSDs caused by a deficiency of enzymes needed to break down complex sugars called glycosaminoglycans. The MPS III syndromes (also known as Sanfilippo syndromes) share complications with other MPS diseases but represent a clinically distinct subset with marked central nervous system degeneration. MPS IIIB is caused by a decrease in NAGLU enzyme activity which leads to the buildup of abnormal amounts of HS in the brain and other organs. The accumulation of abnormal HS, particularly in the central nervous system, leads to severe cognitive decline, behavioral problems, speech loss, increasing loss of mobility, and premature death.
Synageva routinely posts information that may be important to investors in the “Investor Relations” section of the company’s website at www.synageva.com. Synageva encourages investors and potential investors to consult this website regularly for important information about the company.
SOURCE: Synageva
Post Views: 251
