After $1 million drug flop, UniQure revives in gene therapy

Bloomberg

UniQure NV is getting a second chance.
The Dutch biotech company is angling for a comeback in gene therapy after Glybera—its groundbreaking, $1 million treatment for a rare condition—fizzled in 2017. Now UniQure is shifting the technology to the more-promising market for hemophilia, its once-battered shares have rallied back, and the company may be a takeover target.
Heightened attention from potential partners makes the company feel “a little bit like a beehive,” Chief Scientific Officer Sander van Deventer said in an interview. “There’s not a single week we do not have discussions like that.”
Drugmakers including Pfizer Inc. and Spark Therapeutics Inc. are devising ways to use harmless viruses to insert disease-healing copies of abnormal or missing DNA into cells. While the approach has potential to cure disorders that are difficult or impossible to treat, it’s off to a slow start. UniQure withdrew Glybera five years after approval because of “extremely limited” use of the pricey treatment for a dangerous blood-fat condition.
GlaxoSmithKline Plc’s Strimvelis, a $730,000 gene therapy for a rare immune deficiency, has also seen few takers.
Glybera and Strimvelis aren’t good indicators of how the rest of the field will fare because of their small markets, Gbola Amusa, an analyst at Chardan Capital Markets, wrote in a December note.
Hemophilia looks like another story. In hemophilia B, a form of the clotting disorder, UniQure is looking at a market that’s worth $1.5 billion annually, according to Elizabeth Krutoholow, a Bloomberg Intelligence analyst.
UniQure, maker of the first gene therapy approved in the Western world, is betting that the approach’s one-time cost will prove itself in hemophilia B with long-term savings and convenience. While conventional clotting-factor replacements allow most patients to control bleeding, the regimen can be demanding, isn’t always effective, and can cost more than $200,000 per person each year—in some cases, as much as $1 million.
Those numbers make hemophilia “a good target for gene therapies,” Krutoholow said.
Companies are still wrestling with how much to charge for DNA treatments, which are designed as permanent fixes to be given just once.