Longtime biotech exec Philip Astley-Sparke will take the U.S. lead for uniQure, the Amsterdam-based developer, which recently won the first European approval for a new gene therapy. Named as its new U.S. president, Astley-Sparke--who ran BioVex up until Amgen ($AMGN) bought it out in a $1 billion deal and recently was named a Forbion venture partner--tells FierceBiotech that his first task will be to secure a new manufacturing site for the pioneering uniQure, which is planning to file for its first U.S. approval later this year.
"The whole platform has been de-risked," says Astley-Sparke, citing the EU approval of uniQure's Glybera, a gene therapy that treats rare cases of lipoprotein lipase deficiency, an enzyme deficiency that prevents patients from metabolizing fatty blood particles, triggering inflammation that can lead to a long list of serious ailments. Only one or two people in a million suffer from the condition, and Glybera is expected to test payers' appetite for rare disease drugs once it hits the market with a price expected to approach $1 million, according to a recent report from Reuters.
Amsterdam Molecular Therapeutics had failed on several occasions to win an approval for Glybera. Early in 2012, Forbion financed a transfer of the AMT assets into uniQure, which is now one of several upstart biotechs which believe that the decades-long roller coaster ride for gene therapies is ending as a new generation of treatments starts to win regulatory approval. Cambridge, MA-based bluebird bio is also committed to pushing new gene therapies to regulators. And Astley-Sparke says his task will be to build the infrastructure of uniQure as it plots more product launches over the next 5 years.
"Hemophilia B is the next priority," says Astley-Sparke, with treatments for other conditions like porphyria to follow. His responsibilities as U.S. president also include scouting for fresh capital, which may involve a possible IPO at some point as uniQure builds up its commercial arm.
- here's the press release