Synageva BioPharma Corp. Announces Active Investigational New Drug Application for SBC-103 for the Treatment of Mucopolysaccharidosis IIIB
Dec 15 14
Synageva BioPharma Corp. announced that the investigational new drug 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.
Synageva BioPharma Corp. Presents New Data from Phase 3 Study of Sebelipase Alfa in Children and Adults with LAL Deficiency at the AASLD Meeting
Nov 10 14
Synageva BioPharma Corp. announced that data from its Phase 3, global, double-blind, placebo-controlled trial evaluating sebelipase alfa in children and adults with lysosomal acid lipase deficiency (LAL Deficiency) were presented for the first time at a medical conference during the late-breaking session of The Liver Meeting(R), the 65(th) Annual Meeting of the American Association for the Study of Liver Diseases (AASLD). Positive top-line results from this trial were announced on June 30, 2014 and were a key focus of today's presentation. The new data presented at the AASLD meeting by a principal investigator described the magnitude of the impact of sebelipase alfa on a broad range of important disease-related abnormalities, and provided new insights into the severity of patients' liver disease and dyslipidemia at baseline. The investigator also presented data demonstrating sustained reductions in alanine aminotransferase (ALT, the study's primary endpoint and a marker of liver injury) and further improvements in LDL cholesterol at week 36 from the ongoing open-label extension period of the study. Trial design and baseline characteristics. The Phase 3 trial enrolled 66 children and adults with LAL Deficiency. Patients enrolled in the trial were randomized on a one-to-one basis to every other week infusions of sebelipase alfa (1 mg/kg) or placebo for the double-blind treatment period of 20 weeks. LAL Deficiency patients enrolled in the trial presented with multiple clinically important abnormalities at baseline. The median age of patients enrolled in the trial was 13 years of age (range 4-58) and fibrosis and/or cirrhosis was documented in 100% (32/32) of patients who had baseline biopsies. Data from the oral presentation further revealed that of the patients who had biopsies at baseline, 47% (15/32) had bridging fibrosis (defined as Ishak score 3 or 4) and an additional 31% (10/32) of patients had cirrhosis. Dyslipidemia was also common at baseline, with median LDL cholesterol levels of 204 mg/dL, which is in the very high category (greater than 190 mg/dL), and abnormally low median HDL cholesterol levels of 32.5 mg/dL, despite 39% (26/66) of patients already receiving lipid-lowering medications. Data from today's presentation also showed that 58% (38/66) of patients had LDL cholesterol levels at baseline in the very high category (greater than 190 mg/dL) despite 24% (9/38) of these patients receiving lipid-lowering medications. Impact on disease-related abnormalities. The trial met the primary endpoint with 31% (11/36) of sebelipase alfa patients reaching normalization of ALT (defined as 34-43 U/L, depending on age and gender) compared with 7% (2/30) of placebo patients (p=0.027). All sebelipase alfa patients showed a decrease in ALT with mean changes in ALT of -57.9 U/L and -6.7 U/L in the sebelipase alfa and placebo arms, respectively. In addition, treatment with sebelipase alfa showed significant improvements in multiple disease-related parameters of dyslipidemia and liver injury.
Synageva BioPharma Corp. Reports Consolidated Earnings Results for the Third Quarter and Nine Months Ended September 30, 2014; Reiterates Earnings Guidance for the Year 2014
Oct 30 14
Synageva BioPharma Corp. reported consolidated earnings results for the third quarter and nine months ended September 30, 2014. For the quarter ended September 30, 2014, the company reported net loss of $48.3 million compared to a net loss of $28.1 million for the corresponding quarter of the prior year. Revenue was $1.326 million for the quarter ended September 30, 2014 consisted of Fuzeon royalties from Hoffman-La Roche Inc., compared to $1.369 million a year ago. Loss from operations was $48,038,000 compared to $28,196,000 a year ago. Loss before provision for income taxes was $48,303,000 compared to $28,123,000 a year ago. Net loss was $1.46 per diluted share, compared to $1.02 a year ago.
For the nine months, the company's total revenue was $5,255,000 compared to $9,900,000 a year ago. Loss from operations was $134,827,000 compared to $64,959,000 a year ago. Loss before provision for income taxes was $134,933,000 compared to $64,714,000 a year ago. Net loss was $134,933,000 or $4.15 per diluted share, compared to $64,714,000 or $2.38 per diluted share a year ago.
The company reiterates its previous net loss guidance of between $190 million and $205 million for 2014. The net loss is primarily due to investments supporting the global clinical development program for sebelipase alfa, development of SBC-103, expansion of the global clinical, medical affairs and commercial infrastructure, expansion of manufacturing capabilities, as well as advancement of other preclinical pipeline programs.