Synageva BioPharma Completes Targeted Enrollment in Phase 1/2 Trial with SBC-103 for Mucopolysaccharidosis IIIB

LEXINGTON, Mass., June 4, 2015 /PRNewswire/ -- Synageva BioPharma Corp. GEVA, a biopharmaceutical company developing therapeutic products for rare disorders, today announced the completion of enrollment in a Phase 1/2 trial with SBC-103 in patients with mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome). 

About the trial

The trial reached its targeted enrollment of nine patients, who are two years of age or greater but less than 12 years of age, with a definitive diagnosis of MPS IIIB and developmental delay.  Patients are being treated in one of three different dosing cohorts (0.3 mg/kg, 1.0 mg/kg or 3.0 mg/kg) with every other week intravenous administrations of SBC-103 for 24 weeks.  Patients who meet qualifying criteria may continue dosing with SBC-103 for an extended period. 

The primary endpoint of the trial is safety and tolerability of intravenous administration of SBC-103 in patients with MPS IIIB.  The study will also evaluate the effects of dosing with SBC-103 on the change from baseline levels of total heparan sulfate (HS) in urine, serum, and cerebral spinal fluid (CSF), as well as measure the effects on neurocognitive and developmental function and change in brain structures as assessed by magnetic resonance imaging.  

About SBC-103

MPS IIIB is a rare, devastating and life threatening disease which typically presents in children during the first few years of life.  Genetic mutations result in decreased activity of the alpha-N-acetyl-glucosaminidase (NAGLU) enzyme, which leads to a buildup of abnormal amounts of HS in the brain and throughout the body.  Over time, this unrelenting systemic accumulation of HS causes progressive and severe cognitive decline, behavioral problems, speech loss, loss of mobility, and premature death. 

MPS IIIB patients have significant unmet medical need, as current treatments are palliative for the behavioral problems, sleep disturbances, seizures, and other complications, and do not address the root cause of MPS IIIB and stop disease progression.

SBC-103 is the recombinant form of natural human NAGLU designed to replace the missing (or deficient) NAGLU enzyme.  SBC-103 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 was supported by preclinical studies demonstrating that intravenously administered SBC-103 was able to cross the blood-brain barrier and reduce HS storage in the brain 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 CSF in non-human primate studies.

SBC-103 was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) in April 2013 and the European Medicines Agency (EMA) in June 2013 and received Fast Track designation by the FDA in January 2015.

About Synageva

Synageva is a biopharmaceutical company focused on the discovery, development, and commercialization of therapeutic products for patients with rare diseases. The company's pipeline consists of protein therapeutic programs for rare diseases with unmet medical need which are currently at various stages of development.  The company is planning for a global launch of Kanuma™ (sebelipase alfa) for the treatment of LAL Deficiency and is dosing patients in a Phase 1/2 trial with its second, first-mover program, SBC-103 for MPS IIIB.  The company's third, first-mover program, SBC-105, is an enzyme replacement therapy in preclinical development for disorders of calcification.  In addition to these first-mover programs, the pipeline also consists of opportunities that leverage the company's manufacturing platform and other capabilities to create potentially bio-superior treatments for patient populations where there is still unmet medical need.  The company has recently produced enzymes targeting Hunter syndrome, Fabry disease and Pompe disease with expression levels and activity that support further preclinical development.

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, potential approval and plans for the potential launch of Kanuma, the ability for the company's manufacturing platform to create potentially bio-superior treatments, and plans to further support preclinical development for a potentially bio-superior program. 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, unanticipated costs or delays in the company's research and development programs, risk of delays in completing the company's preclinical and clinical trials, risk that the outcomes of the preclinical or clinical trials may not support registration or further development of the company's product candidates due to safety, efficacy or other reasons, the timing for initiating and completing potential preclinical and clinical studies, the timing for reporting data from potential preclinical and clinical studies, the content and timing of decisions by the FDA, EMA and other regulatory authorities, our ability to prepare for potential commercial launch of Kanuma, the ability to continue to produce favorable product attributes and characteristics expressed using the company's proprietary platform, and the risks identified under the heading "Risk Factors" in the company's quarterly report on Form 10-Q for the fiscal quarter ending March 31, 2015 filed with the Securities and Exchange Commission (SEC) 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.

"Synageva BioPharma™" and "Kanuma™" are trademarks, and "Dedicated to Rare Diseases®" is a registered trademark, of Synageva BioPharma Corp.

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SOURCE Synageva BioPharma Corp.