uniQure NV · Q2 2022 earnings

Briefing

Reported financial results for the second quarter of 2022 and highlighted recent progress across its business.

uniQure announced encouraging data from the lower-dose cohort in its Phase I/II study of AMT-130 and the advancement of the U.S. and European regulatory reviews for the marketing applications for etranacogene dezaparvovec in hemophilia B. The company held cash and cash equivalents of $500.5 million as of June 30, 2022.

• Announced 12-month data on the lower-dose cohort of AMT-130 in Huntington’s disease showed the investigative gene therapy was generally well tolerated at this dose with a mean reduction of 53.8% of mutant Huntingtin protein (mHTT) observed in cerebral spinal fluid (CSF).
• Announced postponement of AMT-130 higher-dose procedures due to recent suspected unexpected severe adverse reactions at this dose; Lower-dose procedures are not affected and no impact is expected on anticipated data readouts in 2023.
• Advancing regulatory reviews of the U.S. and European marketing applications for etranacogene dezaparvovec in hemophilia B.
• Received notification that GMP certification will be provided to our Lexington manufacturing facility by the European authorities.

Headline financials

Revenue & EPS history

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