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uniQure Announces 2024 Financial Results and Highlights Recent Company Progress
QUREuniQure(QURE) GlobeNewswire News Room·2025-02-27 12:05

Core Insights - uniQure has made significant clinical and operational progress in 2024, particularly with AMT-130 for Huntington's disease, including alignment with the FDA on the Accelerated Approval pathway [2][5] - The company has completed patient enrollment in various Phase I/II studies, including AMT-130, AMT-260, AMT-191, and AMT-162, and has received favorable recommendations from Independent Data Monitoring Committees [1][2] - Financially, uniQure reported a cash position of approximately 367.5millionasofDecember31,2024,withnetproceedsfromarecentfinancingexpectedtofundoperationsintothesecondhalfof2027[1][7]ClinicalDevelopments−TheFDAgrantedRMATdesignationforAMT−130,indicatingitspotentialtoaddressunmetmedicalneedsinHuntington′sdisease[2]−ThecompanyhasinitiatedpreparationsforaBiologicsLicenseApplication(BLA)submissionforAMT−130,withtheFDAagreeingthatongoingPhaseI/IIstudydatamayserveastheprimarybasisforthissubmission[2][5]−EnrollmentinthePhaseI/IIstudiesofAMT−191forFabrydiseaseandAMT−162forSOD1−ALSisprogressing,withthesecondcohortsexpectedtobegindosingsoon[1][2]FinancialPerformance−RevenuefortheyearendedDecember31,2024,was367.5 million as of December 31, 2024, with net proceeds from a recent financing expected to fund operations into the second half of 2027 [1][7] Clinical Developments - The FDA granted RMAT designation for AMT-130, indicating its potential to address unmet medical needs in Huntington's disease [2] - The company has initiated preparations for a Biologics License Application (BLA) submission for AMT-130, with the FDA agreeing that ongoing Phase I/II study data may serve as the primary basis for this submission [2][5] - Enrollment in the Phase I/II studies of AMT-191 for Fabry disease and AMT-162 for SOD1-ALS is progressing, with the second cohorts expected to begin dosing soon [1][2] Financial Performance - Revenue for the year ended December 31, 2024, was 27.1 million, an increase from 15.8millionin2023,drivenbyhigherlicenseandcollaborationrevenues[8][20]−Researchanddevelopmentexpensesdecreasedto15.8 million in 2023, driven by higher license and collaboration revenues [8][20] - Research and development expenses decreased to 143.8 million in 2024 from 214.9millionin2023,reflectingcost−cuttingmeasures[10]−Selling,general,andadministrativeexpensesalsodecreasedto214.9 million in 2023, reflecting cost-cutting measures [10] - Selling, general, and administrative expenses also decreased to 52.7 million in 2024 from 74.6millionin2023,indicatingimprovedoperationalefficiency[11]StrategicActions−ThecompanycompletedthesaleofitsLexingtonmanufacturingfacilityandunderwentarestructuringthatreduceditsworkforcebyapproximately6574.6 million in 2023, indicating improved operational efficiency [11] Strategic Actions - The company completed the sale of its Lexington manufacturing facility and underwent a restructuring that reduced its workforce by approximately 65%, significantly lowering cash burn by about 70 million per year [2][7] - A public offering of 5.1 million ordinary shares at $17.00 per share was completed, providing additional funding for operations [7][8] - Upcoming investor events are scheduled, including participation in healthcare conferences in March and April 2025 [4][6]