Synageva BioPharma Corp. (Synageva) (NASDAQ:GEVA), a biopharmaceutical company developing therapeutic products for rare diseases, today announced data presentations at the upcoming LDN WORLD Symposium being held February 11-13 in San Diego, California, including three oral presentations and three posters describing sebelipase alfa, lysosomal acid lipase deficiency (LAL Deficiency), and SBC-103 for MPS IIIB.
Oral Presentations at this year’s LDN WORLD Symposium include the following:
Long Term Clinical Effect and Safety of Sebelipase Alfa in Adults with Lysosomal Acid Lipase Deficiency–Thursday, February 13 at 2:45 p.m., presented by Chester B. Whitley, PhD, MD, Professor, Director of the Gene Therapy Center, Advanced Therapies and PKU Clinic, Department of Pediatrics and Experimental and Clinical Pharmacology at the University of Minnesota, Minneapolis, MN.
Clinical Effect of Sebelipase Alfa on Survival and Growth in Infants with Lysosomal Acid Lipase Deficiency (Wolman Disease)–Thursday, February 13 at 2:30 p.m., presented by Vassili Valayannopoulos, MD, PhD, Clinical Lead of the Lysosomal Storage Disease Clinic at Necker-Enfants Malades Hospital, Paris, France.Intravenous SBC-103, A Recombinant Human Alpha-N-Acetylglucosaminidase Reduces CNS Heparan Sulfate Content in a Mucopolysaccharidosis IIIB Mouse Model–Wednesday, February 12 at 2:00 p.m., presented by Sandra Rojas-Caro, MD, Synageva, Lexington, MA. Poster presentations at this year’s LDN WORLD Symposium include the following: Sustained Elevations in LDL Cholesterol and Serum Transaminases from Early Childhood are Common in Lysosomal Acid Lipase Deficiency, Quinn et al. Severe and Rapid Disease Course in the Natural History of Infants with Lysosomal Acid Lipase Deficiency, Jones et al. SBC-103, A Recombinant Human Alpha-N-Acetylglucosaminidase, Demonstrates Mannose-6-Phosphate Receptor Dependent Transport in an In Vitro Blood Brain Barrier Model, Rossomando et al. Sebelipase alfa for LAL Deficiency LAL Deficiency is a rare autosomal recessive lysosomal storage disease (LSD) caused by a marked decrease in LAL enzyme activity. LAL Deficiency presenting in children and adults, historically called Cholesteryl Ester Storage Disease (CESD), is an underappreciated cause of cirrhosis and accelerated atherosclerosis. These complications are due to the buildup of fatty material in the liver, blood vessel walls and other tissues as a result of the decreased LAL enzyme activity. Infants presenting with LAL Deficiency, historically called Wolman disease, show very rapid progression with death, usually in the first six months of life. Affected infants develop severe malabsorption, growth failure and liver complications. Sebelipase alfa (SBC-102) is a recombinant form of the human LAL enzyme being developed by Synageva as an enzyme replacement therapy for LAL Deficiency. Synageva is evaluating sebelipase alfa in global Phase 3 clinical trials in infants, children and adults with LAL Deficiency. Sebelipase alfa has been granted orphan designation by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Japanese Ministry of Health, Labour and Welfare. Additionally, sebelipase alfa received fast track designation by the FDA, and Breakthrough Therapy designation by the FDA for LAL Deficiency presenting in infants. SBC-103 for MPS IIIB The mucopolysaccharidoses (MPS) 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. Mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome) is caused by a decrease in alpha-N-acetyl-glucosaminidase (NAGLU) enzyme activity which leads to the buildup of abnormal amounts of heparan sulfates (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. SBC-103 is a recombinant form of the human NAGLU enzyme being developed by Synageva as an enzyme replacement therapy for MPS IIIB. Using various dosing approaches, SBC-103 reduced HS substrate storage in the brain, liver and kidney in an MPS IIIB animal model. SBC-103 has been granted orphan designation by the FDA and the EMA. Synageva’s additional pipeline programs and manufacturing platform Synageva’s additional pipeline programs include other proteins targeting rare diseases at various stages of preclinical development. These diseases are characterized by significant morbidity and mortality and these programs are selected based on scientific rationale, high unmet medical need, potential to impact disease course and strategic alignment with the company’s corporate focus. In addition to these novel pipeline programs, Synageva is leveraging its manufacturing platform to develop improved biologic therapies for diseases with high unmet medical need.