UniQure's Gene Therapy Boosts Clotting in Two Hemophiliacs

  • Factor IX levels rose to 4.5% and 5.5% of what is normal
  • Gene therapy would compete with Pfizer, Biogen products

UniQure NV, the Dutch biotechnology company that developed the first gene therapy approved in Europe, said its experimental treatment for hemophilia B increased blood clotting ability in two patients. The shares rose as much as 21 percent in trading before U.S. markets opened.

The therapy, known as AMT-060, was designed to fix a genetic flaw caused by missing or defective factor IX, a protein that clots blood. The two adult patients received a low dose of the treatment. Factor IX rose to 5.5 percent and 4.5 percent of the normal level from less than 2 percent in the patients, the company said in a statement Thursday. Three other people who received a low dose of the therapy in the trial haven’t yet reached the full 12 weeks of follow-up, UniQure said.

UniQure rose as high as $18.10 in pre-market trading, up from its Wednesday closing price of $14.91.

Factor IX levels of more than 5 percent are considered a mild form of the disease, which results in bleeding happening only rarely, according to Gbola Amusa, an analyst at Chardan Capital Markets LLC in New York who recommends buying UniQure shares. The company plans to start enrolling patients who will receive a higher dose of the therapy in the trial this quarter.

Mixed Results

Gene therapies have shown mixed results in recent months. Spark Therapeutics Inc. said in October its treatment for rare form of blindness improved patients’ sight, while Bluebird Bio Inc. shares plunged the most ever in December after a company trial of a sickle cell disease therapy disappointed investors.

Current treatments for hemophilia B involve regular self-injections of the missing clotting factor IX to prevent prolonged internal and external bleeding episodes in case of injury. The market leader is Pfizer Inc.’s BeneFix, followed by Biogen Inc.’s Alprolix. Novo Nordisk A/S, which also makes a competing product, said Thursday it filed for approval of a long-acting factor IX product to European regulators.

Available treatments cost $300,000 to $440,000 a year in the U.S. and can be as high as $1 million a year in some patients, according to Amusa. Hemophilia B can cost the health system more than $20 million per patient when medicines, doctors, nurses, and hospital storage are included in the bill, he said.

Therapy Costs

The cost of the gene therapy should be compared with other orphan drugs that target rare diseases, which on average cost about $250,000 a year, according to Joern Aldag, who stepped down as UniQure’s chief executive officer last month. Given the expense, UniQure and other developers have considered alternative forms of reimbursement. For example, payments may be spread out over several years and could cease if a patient dies, Aldag said last year.

UniQure also developed Glybera, which treats a potentially fatal genetic disease that results in acute swelling of the pancreas. Glybera in 2012 became the first gene therapy approved for use in Europe. UniQure is also partnered with Bristol-Myers Squibb Co. on a therapy for congestive heart failure.

UniQure in December said Dan Soland, former chief operating officer of ViroPharma Inc., would succeed Aldag.

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