uniQure Announces Third Quarter 2024 Financial Results and Highlights Recent Company Progress

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uniQure Inc.
uniQure Inc.

~ Type B meeting scheduled with the FDA in the fourth quarter of 2024 to initiate discussions regarding a potential expedited clinical development pathway for AMT-130 in Huntington’s disease ~

~ Presented positive interim data from Phase I/II trial of AMT-130 demonstrating slowing of Huntington’s disease progression and reductions in key biomarker of neurodegeneration ~

~ Initiated patient dosing in new Phase I/II studies of AMT-162 in SOD1-ALS and AMT-191 in Fabry disease; First patient enrolled in observational stage of Phase I/II study of AMT-260 in mTLE ~

~ Completed sale of Lexington manufacturing facility and announced organizational restructuring expected to significantly reduce operating expenses and annual cash burn ~

~ Strong cash position of approximately $435 million as of September 30, 2024, following retirement of $50 million of debt, with cash runway through the end of 2027 ~

LEXINGTON, Mass. and AMSTERDAM, Nov. 05, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the third quarter of 2024 and highlighted recent progress across its business.

“uniQure has made significant strides during the third quarter both clinically and operationally. We advanced our pipeline of clinical gene therapy programs, including the presentation of positive long-term follow-up data on AMT-130 supporting significant, dose-dependent slowing of Huntington’s disease progression,” stated Matt Kapusta, chief executive officer of uniQure. “We have scheduled a Type B meeting with the FDA for late November and welcome the opportunity, as part of the Regenerative Medicine Advanced Therapy (RMAT) designation, to discuss the potential for an accelerated development pathway for AMT-130. We believe the recently announced compelling clinical data, combined with AMT-130’s manageable safety profile and the lack of therapeutic options for patients in need, present a strong case for accelerated development. In addition, dosing has begun across two new Phase I/II studies in SOD1-ALS and Fabry disease, and we are making substantial progress towards the initiation of a third clinical trial in mesial temporal lobe epilepsy, with the first patient recently enrolling into the observational phase of the study.”

“We also delivered on one of our key corporate goals, which was to take meaningful actions to streamline operations and preserve capital. Following the sale of our Lexington manufacturing facility, we announced a strategic reorganization expected to further reduce our cash burn and operating expenses,” he continued. “These decisions, which are delivering an immediate favorable impact, have extended our cash runway through the end of 2027 and multiple clinical and regulatory milestones with the potential to generate shareholder value.”

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