Synageva BioPharma Corp. announced that sebelipase alfa met the primary endpoint in a randomized, double-blind, placebo-controlled phase III study (Acid Lipase Replacement Investigating Safety and Efficacy − ARISE). Sebelipase alfa showed statistically significant normalization of alanine aminotransferase (ALT) (a measure of liver injury) in patients suffering from lysosomal acid lipase deficiency (LAL deficiency).
In this study, 66 patients suffering from LAL deficiency were randomized to sebelipase alfa (1 mg/kg) or placebo. This was above Synageva’s original target of 50 patients. The patients were diagnosed with multiple clinically important abnormalities at baseline.
Following 20 weeks of treatment, sebelipase alfa significantly reduced LDL cholesterol, non-HDL cholesterol and triglycerides, while increasing HDL cholesterol compared to placebo. This led to improvement in dyslipidemia in patients.
Additionally, 63% of the patients (whose paired liver biopsies were available both at baseline and at 20 weeks) treated with sebelipase alfa achieved improvement in hepatic steatosis compared to 40% of the patients treated with placebo.
Sebelipase alfa is Synageva’s lead pipeline candidate. It enjoys orphan drug status in the U.S., EU and Japan. Moreover, it has fast track designation in the U.S. as well as Breakthrough Therapy designation (LAL deficiency in infants).
Sebelipase alfa being the lead candidate at Synageva, its successful development and approval would be a major boost for the company. We expect investor focus to remain on pipeline updates from the company. The company intends to file marketing applications in the U.S. and Europe for sebelipase alfa by the end of the first quarter of 2015.
Synageva carries a Zacks Rank #2 (Buy). Some better-ranked stocks in the biotech sector include Biogen Idec Inc. (BIIB - Free Report) , The Medicines Company (MDCO - Free Report) and Regeneron Pharmaceuticals, Inc. (REGN - Free Report) . All these stocks carry a Zacks Rank #1 (Strong Buy).