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uniQure Update on AMT-130

December 19, 2023

Huntington's Disease Youth Organization

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This morning, uniQure issued a press release announcing interim data from 39 patients enrolled in the ongoing Phase I/II clinical trials of AMT-130, [HD Gene TRX-1 and HD Gene TRX-2], for the treatment of Huntington’s disease being conducted in the United States and Europe. The analysis includes up to 30 months of follow-up. The key takeaways are as follows:

• AMT-130 continues to be generally well-tolerated across both dose cohorts • Patients administered AMT-130 continue to show evidence of preserved neurological function relative to the natural history of the disease • Neurofilament Light Chain (NfL) in the cerebrospinal fluid (CSF) continues to show a favorable trend with low dose patients below baseline 30 months
• NfL continues to show a favorable trend with high dose patients near baseline at 18 months • Data support uniQure’s continuing clinical development of AMT-130 and pursuing regulatory interactions for ongoing development

In the US, the Phase I/II clinical trial of AMT-130 is exploring the safety, tolerability, and efficacy signals in 26 patients. In Europe, the Phase IB/II clinical trial of AMT-130 is exploring the safety, tolerability, and efficacy signals in an open label clinical trial with 13 patients. Additional details are available on http://www.clinicaltrials.gov for HD Gene TRX-1 (NCT04120493) and for HD Gene TRX-2 (NCT05243017).

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