UniQure Rallies On Positive Late-Stage Data For Hemophilia B Gene Therapy

UniQure NV (NASDAQ: QURE) shares were advancing to four-month highs Thursday in reaction to a positive clinical data readout.

What Happened: The Lexington, Massachusetts-based company announced positive top-line data from a pivotal Phase 3 study, dubbed HOPE-B, that evaluated its investigational adeno-associated virus five-based gene-therapy candidate etranacogene dezaparvovec for the treatment of patients with severe and moderately severe hemophilia B.

Top-line pivotal data show that a single administration of the investigational gene therapy led to sustained increases of Factor IX, or FIX, to functionally curative levels capable of eliminating the need for regular infusions to control and prevent bleeding episodes, the company said.

FIX is a protein produced naturally by the body and helps in the clotting of blood.

The HOPE-B study is a multinational trial in adult males with moderately severe hemophilia, and it did not exclude patients based on pre-existing neutralizing antibodies to AAV5.

The findings from the study were as follows:

All 54 patients dosed with a single intravenous infusion gene therapy candidate at 2x1013 gc/kg showed a rapid increase in FIX activity from 2% or less to a mean of 37.2% at 26 weeks, meeting the first primary endpoint.

No correlation was observed between pre-existing NAbs and FIX activity in patients with NAb titers up to 678.2.

The gene-therapy candidate was generally well-tolerated, with no treatment-related serious adverse events.

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Why It's Important: The data set is the first from any Phase 3 gene-therapy study in hemophilia B, and the study boasts one of the largest sets of patients receiving a single gene therapy investigational product to be reported to date, UniQure said.

"The ability to dose a gene therapy in patients with pre-existing neutralizing antibodies has not been demonstrated for any other gene therapy and illustrates the potentially unique ability of our AAV5 platform to address the needs of a broad set of patients living with hemophilia B and other disorders," Ricardo Dolmetsch, president of R&D at UniQure, said in a statement. 

What's Next: The data is scheduled for an oral presentation at the 62nd Annual Meeting of the American Society of Hematology on Dec. 8.

Based on interactions with the FDA and EMA, the company said it plans to incorporate FIX activity and bleeding rates at 52 weeks as additional co-primary endpoints in the study.

The company is planning to hold its pre-BLA meeting with the FDA and will be completing the last patient's 52-week follow-up visit in the first quarter of 2021.

QURE Price Action: At last check, UniQure shares were up 7.16% to $45.82. 

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