UniQure Making Progress Toward US and European Approval of AMT-060 for Hemophilia B

UniQure has taken major steps this year toward U.S. and European regulatory approval of AMT-060, its gene-therapy treatment for hemophilia B.

In January, the U.S. Food and Drug Administration (FDA) gave AMT-060 breakthrough therapy designation for patients with severe hemophilia B. In April the European Medicines Agency (EMA) gave it PRIME designation. The designations are designed to accelerate the development and regulatory review of drugs that appear to be better than current treatments.

The designations came after regulators reviewed the results of a Phase 1/2 open-label, dose-escalating study of AMT-060 (NCT02396342) in 10 hemophilia B patients.

Five of the 10 patients received a lower dose of AMT-060 without an immunosuppressant therapy, and the other five a higher dose. Patients received the doses intravenously.

UniQure presented the results of the trial in December 2016 at the 58^th American Society of Hematology annual meeting in San Diego.

The presentation looked at how patients who received the lower dose of AMT-060 were doing at 52 weeks, and how patients who received the higher dose were doing at 31 weeks.

All patients in the lower-dose group maintained the level of the factor IX clotting factor they needed to minimize bleeding episodes, uniQure said. The result was no bleeding episodes at all in the final 14 weeks of the 52-observation period. AMT-060 also led to an improvement in the state of the disease in the five patients in the higher-dose group, the company said.

In terms of safety, AMT-060 was well-tolerated, with no patients experiencing adverse events, uniQure said.

In a meeting with company officials after the trial, the FDA said it recognized AMT-060’s benefits and had no safety concerns with it. UniQure will have a similar meeting with European regulators in coming months. Such meetings are held to determine whether it’s safe to proceed to a Phase 3 trial, and to let regulators evaluate the Phase 3 trial design that a company is proposing.

“In early 2017, we continued to make progress across our strategic priorities, including our gene therapy programs in hemophilia B, Huntington’s disease and congestive heart failure,” Matthew Kapusta, chief executive officer of uniQure said in a press release. “Over the past few months, AMT-060 received Breakthrough Therapy and PRIME designation based on the promising data from our ongoing Phase I/II study, and we look forward to presenting updated data from the study in July at ISTH.  We also initiated discussions with the FDA regarding planning for a pivotal study, and expect additional interactions with regulators in 2017 as we prepare for late-stage development.”

ISTH is the International Society on Thrombosis and Haemostasis, whose annual meeting will be in Berlin from July 8-13.

In addition to AMT-060 for hemophilia B, uniQure’s gene-therapy portfolio includes a treatment for Huntington’s disease, AMT-130.