uniQure Q1 2022 Earnings Report
Key Takeaways
uniQure reported a strong start to 2022, marked by advancements in key programs, progress in research activities, and the strengthening of its executive leadership team. Partner CSL Behring submitted a Marketing Authorization Application in Europe for etranacogene dezaparvovec. The company received $55 million in milestone payments, and made significant advancements enrolling patients in Phase I/II studies for AMT-130 in Huntington’s disease, completing 32 patient procedures to date.
Partner CSL Behring submitted Marketing Authorization Application (MAA) in Europe for etranacogene dezaparvovec in hemophilia B; MAA validated by the European Medicines Agency (EMA) and granted accelerated assessment.
Received $55 million in milestone payments associated with global submissions of marketing and license applications.
Completed patient enrollment of higher dose cohort of U.S. Phase I/II study of AMT-130 and lower dose cohort of European Phase Ib/II study; total of 32 patient procedures completed to date.
One year safety and biomarker data from all 10 patients in lower dose cohort of U.S. Phase I/II study of AMT-130 in Huntington’s disease, including mHTT and NfL biomarkers, expected in second quarter 2022.
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uniQure Revenue by Segment
Forward Guidance
The company provided forward-looking statements regarding enrollment in clinical trials, disclosure of data, and initiation of toxicity studies.