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Synageva offering LSD patients renewed hope

Authored by James Sheppard

As part of Liftstream’s commitment to rare diseases, in the run up to Rare Disease Day 2013 we will be profiling 10 companies that are leading the rare disease research effort. In the seventh of the series we look at the US biopharmaceutical company Synageva.

Synageva is a clinical stage biopharma company dedicated solely to rare disease research. Synageva currently has 5 investigational compounds. The programmes Synageva is currently investigating primarily focus around Lysosomal Storage Disorders (LSDs) using enzyme replacement therapy (ERT). Synageva’s lead asset is Sebelipase Alfa (SBC-102) which is currently being investigated for Lysosomal Acid Lipase (LAL) Deficiency and is in phase II/III clinical trials. Sebelipase alpha received orphan drug status in both the US and EU in 2010 for Sebelipase Alpha. The clinical program for Sebelipase alpha is the first compound to be tested in LAL Deficiency and early clinical data looks very promising. In February 2013 Synageva initiated a phase III trial for Sebelipase alpha in children & adults with late onset LAL Deficiency. The ARISE trial will recruit 50 participants and the primary endpoint will be at 20 weeks.

Synageva are currently partnered with a range of companies from global pharma to innovative biotechs. In 2012 Synageva expanded their collaboration with Mitsubishi Pharma to develop drugs for an undisclosed orphan drug. Synageva are also partnered with the Belgian biotech to-BBB for drug delivery for Rare Brain Disorders. Synageva have actively pursued a partnering strategy when developing novel compounds and seeks to out-license advanced programs.

Synageva merged with Trimeris Inc in November 2011 and the newly formed company was immediately listed on the NASDAQ stock exchange. In January 2012, Synageva announced a proposed $60m offering of common stock. This was followed by a 2nd round of $80m and a 3rd – $80m round in January 2013. In 2010, Synageva relocated their head office to Lexington, Massachusetts. This move was in an effort to increase laboratory space and expand Synageva’s R&D capacity within rare diseases. Synageva also now has a presence in Europe.

Liftstream is supporting Rare Disease Day 2013. In profiling this company we hope to bring greater awareness to rare diseases and the companies working in this area. Liftstream is sponsoring the 2013 Orphan Drug Congress and we look forward to having contact with you at these events or by contacting us via Linkedin or Twitter

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