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 last year. 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.

After $1 Million Drug Flop, UniQure Revives in Gene Therapy

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.

After $1 Million Drug Flop, UniQure Revives in Gene Therapy

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. Spark earlier this year set a price of $850,000 -- or $425,000 per eye -- on its therapy for a rare, inherited form of blindness. The company is also offering discounts tied to effectiveness.

Some hemophilia gene therapies could sell for more than $1 million per patient, according to Chardan’s Amusa. Van Deventer said he was confident that cost would not be an obstacle to sales.

“In the developed world, there is no resistance to relatively high-priced drugs, as long as they work very well,” he said.

Amsterdam-based UniQure expects to begin a late-stage study of its hemophilia therapy, called AMT-061, early in the third quarter, aiming to be first to market with such a treatment as early as 2020, Van Deventer said. The drugmaker is racing against Spark and Pfizer, partners on another treatment for the same disease.

New Partners

UniQure is also developing experimental gene therapies for Huntington’s disease and collaborating with Bristol-Myers Squibb Co., which owns about 7.5 percent of the shares, on treatments for cardiovascular illnesses. The company would consider new partners, depending on what they’re prepared to pay, said Van Deventer, who co-founded UniQure’s predecessor and is a partner at venture capital firm Forbion Capital Partners.

Success with hemophilia would complete UniQure’s turnaround from just 12 months ago, when its shares languished near an all-time low. They’ve surged fourfold in the past year and rose 0.7 percent to $22.72 at 10:30 a.m. in New York, giving the drugmaker a market value of about $719 million.

The company isn’t looking to sell itself and has the funding to carry out its plans, Van Deventer said. Still, it wouldn’t be realistic to rule out an approach, he said. BioMarin Pharmaceutical Inc. may attempt to purchase or partner with UniQure this year, Chardan’s Amusa said. BioMarin didn’t respond to a request for comment.

“I’m not excluding at some point in time there will be an acquisition of this company, as there has been for many other companies in the area,” Van Deventer said.

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