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Ultragenyx focused on ultra-rare disease future

Authored by Paul Foster

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 5th of the series we look at the US biopharmaceutical company Ultragenyx.

Ultragenyx is a privately owned developmental stage biotechnology company established in April 2010. The Ultragenyx management team headed by Emil D. Kakkis, M.D. are extremely experienced in rare diseases most of whom have worked with Dr Kakkis at Biomarin. They currently have 27 employees based at their Novato, CA headquarters and have plans to increase headcount by another 8-14 people in 2013.

As with all companies in this series Ultragenyx focus on rare diseases but have also taken the step to develop products for ultra-rare diseases. Ultragenyx currently has 4 products in development two of which have entered into clinical development. The lead product Sialic Acid is currently being developed for the treatment of Heriditary Inclusion Body Myopathy (HIBM) an autosomal recessive neuromuscular disorder, which presents in early adulthood as a weakness in distal muscles of the lower extremities which ultimately leads to loss of strength and confines the patient to a wheelchair. A phase II dose ranging study is currently underway. It is estimated that there are approximately 2000 patients living with this disorder in the developed world.

The other product Ultragenyx have in clinical development is triheptanoin, which it recently in-licenced from the Baylor Research Institute for the treatment of Fatty Acid Oxidisation Disorders (FAOD), which is thought to be one of the causes of Sudden Infant Death Syndrome. In Investigator led trials triheptanoin has been shown to reduce muscle rupture, hypoglycaemia and cardiomyopathy in a variety of FAODs and there are currently 22 patients that have been receiving triheptanoin for up to 10 years through a compassionate use programme. Ultragenyx hold the rights for North America and an exclusive option for the Rest of the World. A pre-IND meeting is scheduled for 2nd Quarter 2013.

Ultragenyx are close to moving a third product into the clinical phase with UX003 for the treatment of Sly Syndrome scheduled for Phase I/II clinical trials later this year.

Ultragenyx have some ambitious plans and have surrounded themselves with a strong syndicate of investors. In the last 12 months Ultragenyx have raised $120 million. Its recent Series B funding ($75 million)was over-subscribed and attracted specialist rare disease companies such as Shire and Sanofi—Genzyme into their fold.  It has also garnered support from institutional investors that do not traditionally invest in private companies. Ultragenyx’s aim is to become a commercial stage corporation by 2016 funded by a potential IPO in 2014 and with the interest already expressed by the investment community and the experienced management team in place they are in an ideal situation to buck the current IPO trend.

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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