- UniQure released preliminary topline results for its AMT-060 therapy for treatment of hemophilia B. The Dutch firm reported a positive response in two treated patients who had passed a 12-week follow-up threshold. Three other patients had been dosed but had not passed the 12-week mark yet.
- UniQure's shares jumped nearly 16% from Wednesday (prior to the data) to Thursday's closing price.
- Success will be measured by the ability of these patients to produce Factor IX phenotypic features at 5% to 10% of normal levels—what's needed to turn a case of severe hemophilia B into a case of mild-to-moderate hemophilia B.
While it's too early to say that AMT-600 is the gene-therapy answer for patients with hemophilia B, the early results are tentatively encouraging. The two patients reaching, respectively, 12 and 20 weeks post-treatment are now producing 4.5% to 5.5% of normal Factor IX levels. Prior to treatment, both patients were producing only 1% to 2% of normal.
Additionally, the therapy seems to be well tolerated, although one patient has had spikes in liver enzymes around 10 weeks post-treatment.
Enthusiasm is tempered, however, by the short time horizon of the results. Patients can have widely varying responses over time, as Bluebird Bio's researchers found out when testing their therapy on patients with beta-thalassemia. If results continue to show positive responses over time, UniQure's therapy seems promising—especially considering its success with Glybera in the E.U.