- Treatment with uniQure's experimental gene therapy for hemophilia B led to sustained increases in Factor IX activity levels in three patients for up to 16 weeks after a single dose, updated results from an ongoing Phase 2b study showed.
- No patients reported any bleeding events or required an infusion of Factor IX replacement therapy, uniQure said Friday in an announcement that has spurred a 13% jump in the company's share price through Monday morning.
- UniQure also announced the the first patient in the company's larger Phase 3 study has been treated, setting the biotech on pace to fully enroll the key trial by year's end.
UniQure has some history in gene therapy, having tried its hand at marketing Glybera (alipogene tiparvovec), the first gene therapy for an inherited disease approved in Europe. No market materialized, however, leading uniQure to pull the drug in 2017.
Now, the biotech's work in hemophilia is getting more attention, particularly as its candidate for hemophilia B — called AMT-061 — advances through the clinic.
This latest dataset on the therapy follows a readout last November that lifted the biotech's market value by nearly half a billion dollars. Updated results show two of the three patients reached Factor IX activity levels close to or just above the "normal" threshold of between 50% and 150%.
Factor IX activity levels in the third patient reached 25% of normal at 14 weeks post-administration. No patient experienced any material loss in activity following treatment. One of the three experienced a "slight" elevation in liver enzymes that resolved without additional treatment, uniQure said.
Meanwhile, uniQure has dosed the first patient in its HOPE-B pivotal study of AMT-061, putting enrollment of that trial on track to conclude by the end of 2019.
With Phase 3 testing underway, uniQure maintains its position at the forefront of hemophilia B gene therapy development alongside Spark Therapeutics and Pfizer, which are developing fidanacogene elaparvovec.
Hemophilia gene therapies more broadly are seeing substantial advances. BioMarin Pharmaceutical's valoctocogene roxaparvovec, for instance, was shown to have eliminated spontaneous bleeding and restored blood clotting protein levels to normal or near-normal levels in patients with hemophilia A through two years of treatment.