After $1 Million Drug Flop, UniQure Revives in Gene Therapy - (Bloomberg via NewsPoints Desk)

  • UniQure, which pulled its gene therapy Glybera a year ago due to "extremely limited" use of the $1-million treatment for a rare blood-fat condition, is shifting its technology to the market for hemophilia, reported Bloomberg.

  • Chief Scientific Officer Sander van Deventer said UniQure expects to begin a late-stage study of its hemophilia therapy AMT-061 early in the third quarter, aiming to be first to market with such a treatment as early as 2020.

  • Drugmakers including Pfizer and Spark Therapeutics are developing ways to use harmless viruses to insert disease-healing copies of abnormal or missing DNA into cells, although the approach is "off to a slow start," the news source said. Meanwhile, GlaxoSmithKline's Strimvelis, a $730 000 gene therapy for a rare immune deficiency, has also seen few takers.

  • Analyst Gbola Amusa of Chardan Capital Markets has suggested Glybera and Strimvelis are poor indicators of how the rest of the field will fare because of their small markets, but Bloomberg Intelligence analyst Elizabeth Krutoholow said UniQure is now targeting a market worth $1.5 billion annually, making hemophilia "a good target for gene therapies."

  • Van Deventer said he was confident that cost would not be an obstacle to sales, saying "in the developed world, there is no resistance to relatively high-priced drugs, as long as they work very well."

  • UniQure is also developing experimental gene therapies for Huntington's disease and collaborating with Bristol-Myers Squibb on treatments for cardiovascular illnesses. Van Deventer indicated that UniQure would consider new partners, although he said the company is not looking to sell itself and has the funding to carry out its plans.

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