Synageva BioPharma reports study results of sebelipase alfa in adults with late onset LAL Deficiency

May 9 2013

Synageva BioPharma Corp. (Synageva) (NASDAQ:GEVA), a biopharmaceutical company developing therapeutic products for rare diseases, today reported 12-month results from an ongoing extension study with sebelipase alfa in adults with late onset Lysosomal Acid Lipase (LAL) Deficiency. The data will be presented today by Reena Sharma, M.D., Consultant, Adult Inherited Metabolic Diseases, Salford Royal Hospital, U.K., during an oral presentation at the European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) annual meeting being held in London, U.K., May 8-11, 2013.

“LAL Deficiency: Why Missing the Diagnosis Matters”

Details from the extension study with sebelipase alfa in adults with late onset LAL Deficiency

Nine adults with LAL Deficiency enrolled in the Phase 1/2 trial. After completing the initial four-week portion of the trial, patients were allowed to continue treatment with sebelipase alfa as part of a long-term, open-label extension study. Eight of nine patients enrolled in the extension study and six of these eight have now completed the first 12 months of the study.

At one year, the effects of sebelipase alfa in this study were sustained as compared to the effects at nine months. Sebelipase alfa continues to provide evidence of a reduction in liver damage with sustained reductions in both ALT and AST, frequently into the normal range. Sebelipase alfa produced mean percent decreases for ALT and AST from the initial baseline to month 12 of the extension study of 56% and 40%, respectively>

Sebelipase alfa was generally well tolerated throughout the initial 12 months of the extension study. The majority of adverse events were mild and unrelated to sebelipase alfa. Infusion-related reactions were uncommon and the majority were gastrointestinal (diarrhea, abdominal cramping) events of mild severity. One patient with a moderate (Grade 2) allergic type infusion-related reaction, who paused treatment with sebelipase alfa at nine months of the extension study, remains off treatment with further tests pending. No anti-drug antibodies have been detected in any subjects tested to date in either the initial portion or extension portion of the Phase 1/2 study. A single patient during the extension study developed acute cholecystitis and cholelithiasis (two serious adverse events) which were later treated with elective cholecystectomy. These two serious adverse events were considered unlikely related to sebelipase alfa. This patient continues treatment with sebelipase alfa without a change in dosing and administration.

Synageva-sponsored satellite symposium

During the ESPGHAN meeting, Synageva will sponsor a satellite symposium entitled, "LAL Deficiency: Why Missing the Diagnosis Matters" chaired by Patrick McKiernan, M.D., Consultant Paediatrician, The Liver Unit, Birmingham Children's Hospital, Birmingham, U.K. The symposium is being held May 9 at 5:45-7:15 p.m., GMT.