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Oxyrane seeking to advance Pompe Disease programme in 2013

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 showcasing 10 companies that are leading rare disease research. In the fourth of the series we look at UK based company Oxyrane. Oxyrane are based in the Manchester Health Corridor in Northern England, UK.

Oxyrane, founded in 2006, develops enzyme replacement therapies (ERT) for the treatment of lysosomal storage disorders (LSDs). Oxyrane’s lead product is OXY2098-95 which is currently in pre-clinical testing for Pompe Disease. Pompe disease is a rare disorder where the progressive accumulation of glycogen results in muscle weakness – particularly in the heart and skeletal muscle. In November 2012 Oxyrane announced positive results from a study undertaken in collaboration with a Belgian University showed that LSDs can be treated with modified enzymes such as OXY2098-95. The increased level of targeting shown in the trial resulted in a 20 fold increase in cellular uptake of the enzyme, thus having the potential to significantly improve treatment for patients with LSDs. This trial has laid the foundations for Oxyrane to move forward into clinical development.  Oxyrane now hopes to move forward into clinical development in the coming months with a Phase I trial being planned. Oxyrane also has 4 other programmes they are hoping to progress in various LSD indications.

In order to aid development and possibly commercialisation, Oxyrane raised $26.5m in Series D funding in November 2011.  Funding was led by the Dutch Venture Capital company Forbion Capital Partners and supported by existing investor New Science Venture. This funding round also brought on board a new investor in the Morningside Group. The funding will allow for Oxyrane to complete the preclinical development and initiate the clinical phases for OXY2098-95.

Oxyrane recently announced that they would be continuing their expansion in the US, where in January 2013 they opened a new office. Alongside this new office, Oxyrane have made a number of key senior appointments including appointing industry veteran Philip Astley-Sparke as Chairman of the Board and Dr. Charles Richard as Chief Medical Officer. Both Mr Astley-Sparke and Dr. Richard have extensive experience in orphan and biologics drugs. Dr. Richard was previously Head of Translational Medicine at Shire HGT. Further to this Oxyrane made two more senior appointments in the US with James Weston becoming VP of Regulatory Affairs and Susan Flint as Head of Clinical Operations. Following the appointments and recent funding rounds Oxyrane are well placed to make significant in-roads into the clinical development of their lead asset OXY2098-95.

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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