Synageva BioPharma Corp. (Synageva) (NASDAQ:GEVA), a clinical stage biopharmaceutical company developing therapeutic products for rare diseases, today announced presentations at the upcoming LDN WORLD Symposium being held February 13-15 in Orlando, Florida. During an oral presentation on Friday, February 15, at 1:00 PM EST, Dr. Manisha Balwani will present 38-week data from the Phase I/II extension study of sebelipase alfa in adults with late onset lysosomal acid lipase deficiency (LAL Deficiency). An additional poster at the LDN WORLD Symposium will highlight the ability of SBC-103 to reduce the accumulation of substrate in the brain of a Mucopolysaccharidosis IIIB (also known as MPS IIIB, or Sanfilippo B) animal model.
Data Presentations and Synageva Satellite Symposium at the LDN WORLD Symposium
On Friday, February 15, at 1:00 PM EST, Manisha Balwani, MD, MS, Assistant Professor of Genetics and Genomic Sciences and Assistant Professor of Medicine, Mount Sinai School of Medicine, New York, NY, will present 38-week data from the Phase I/II extension study of sebelipase alfa. Nine adults with LAL Deficiency were enrolled in the Phase I/II trial. After completing the initial portion of the Phase I/II trial, patients were allowed to continue treatment with sebelipase alfa as part of a long-term, open-label extension study. Eight of nine patients have enrolled in the extension study and seven of these eight have now completed the first 38 weeks of the study.
Sebelipase alfa effects observed in this study at 24 weeks were sustained through 38 weeks. Sebelipase alfa continues to reduce liver damage with sustained reductions in both ALT and AST, frequently into the normal range. In addition, maintenance of the improvements in the dyslipidemia associated with LAL Deficiency was also observed. Sebelipase alfa produced mean percent decreases for ALT and AST from the initial baseline to week 38 of the extension study of 54% and 40%, respectively (p=0.016 for both comparisons). In addition, sebelipase alfa resulted in mean percent decreases from the initial baseline to week 38 of the extension study for LDL-C of 47% (p=0.016), total cholesterol of 33% (p=0.016), triglycerides of 29% (p=0.047), as well as a mean increase in HDL of 15% (p=0.313).