Hemophilia B Gene Therapy AMT-060 Moves to Large-scale Production, Says UniQure

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by Alice Melão |

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Pharmaceutical firm uniQure N.V. has developed an optimized large-scale process to manufacture its lead gene, therapydate, to treat hemophilia B. This new platform will allow the Dutch company to boost production of its AMT-060 gene therapy at its Lexington, Massachusetts, facility, in accordance with Good Manufacturing Practices guidelines.

This should enable uniQure to meet the requirements of both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) by early fall, the company’s CEO, Matt Kapusta, said in a press release.

“We have made significant progress over the past several months to now be in a position where we have developed a commercial-scale process and are evaluating our completed batches to assess comparability,” Kapusta said. “We look forward to finalizing this work in anticipation of meetings with regulators to further discuss plans to advance our hemophilia B program into a pivotal study next year.”

AMT-060 gene therapy is based on a viral vector to deliver a therapeutic form of human factor IX gene, or FIX. This potential therapy is currently being evaluated in a Phase 1/2 trial (NCT02396342) in patients with severe hemophilia B and advanced joint disease.

The trial’s most recent long-term results show that AMT-060 is safe and well tolerated, while reducing annual spontaneous bleeding rates by 84 percent to an average of 0.5 annual bleeds after gene transfer.

These results were subject of an oral presentation, “Updated Results from a Dose-escalation Study in Adults with Severe or Moderate-severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy: up to 1.5 Years Follow-up,” given during the 26th Biennial Congress of the International Society on Thrombosis and Haemostasis in Berlin. A summary appears in the meeting’s book of abstracts.

Based on the trial’s results, the FDA granted AMT-060 its breakthrough therapy designation in January 2017. The EMA awarded the gene therapy its PRIME designation in April 2017. In addition, uniQure says it will extend its new manufacturing platform to production of AMT-130 to treat Huntington’s disease.