Synageva BioPharma Announces Active Investigational New Drug Application For SBC-103 For The Treatment Of Mucopolysaccharidosis IIIB
LEXINGTON, Mass., Dec. 15, 2014 /PRNewswire/ -- Synageva BioPharma Corp. (NASDAQ: 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.
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Forward-Looking Statements
This news release contains "forward-looking statements". Such statements generally can be identified by the use of words such as "anticipate," "expect," "plan," "could," "intend," "believe," "may," "will," "estimate," "forecast," "project," or words of similar meaning. These forward-looking statements address, among other matters, the timing to enroll patients and report preliminary data for the SBC-103 Phase 1/2 study, the timing to report data on the company's study to assess the integrity of the blood-brain barrier and other disease abnormalities in patients with MPS IIIB, and the potential impact of natural history studies. Many factors may cause actual results to differ materially from forward-looking statements, including inaccurate assumptions and a broad variety of risks and uncertainties some of which are known, such as, the risk of delays in initiating or completing our clinical trials, unanticipated costs or delays in our research and development programs, unanticipated delays in our submission timelines, risk that the outcomes of our clinical trials may not support registration or further development of our product candidates due to safety, efficacy or other reasons, the content and timing of decisions by the FDA and other regulatory authorities, and the risks identified under the heading "Risk Factors" in Synageva's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on October 30, 2014 and other filings Synageva periodically makes with the SEC, and others of which are not known. Preclinical and clinical trial data are subject to differing interpretations, and regulatory agencies, as well as medical and scientific experts, may not share Synageva's views regarding these data or its implications. Synageva may encounter problems or delays in preclinical and clinical development and the regulatory process. No forward-looking statement is a guarantee of future results or events, and investors should avoid placing undue reliance on such statements. Synageva undertakes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.
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