William Blair initiated research coverage of uniQure N.V. (QURE $29.36). Based in the Netherlands, uniQure N.V. is focused on developing and commercializing adeno-associated virus (AAV) gene therapies for the treatment of severe genetic disorders of the liver and CNS.

Analyst Raju Prasad said that uniQure currently has two clinical assets, the most advanced of which has the potential to be a commercial product by the end of 2022. “UniQure also has a deep preclinical pipeline that has the potential to expand the utility of AAV and overcome some of the current limitations of the technology,” he said.

Prasad said, “UniQure’s most advanced gene therapy program, EtranaDez for the treatment of hemophilia B, is being evaluated in a Phase III trial. EtranaDez has induced increases in factor IX (FIX) activity that approach non-hemophiliac levels and are durable out to 52 weeks post-treatment. Increases in FIX activity have also corresponded with meaningful hemostatic outcomes, including a reduction in bleeds and FIX usage. Top-line, 78-week data from the Phase III trial is expected in fourth quarter 2021, followed by a BLA submission by the company’s commercial partner, CSL Behring, in the beginning of 2022. We view the top-line data readout and regulatory filing as significantly de-risked based on the Phase III 52-week data, Phase IIb two-year data, and the FDA’s final cellular and gene therapy guidance documents. Lastly, we highlight uniQure has the potential to receive $300 million in near-term regulatory and commercial milestone payments from the licensing agreement.”

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