uniQure Resumes Enrollment in Huntington Disease Gene Therapy Trial


The DSMB has recommended the trial to continue with additional risk mitigation procedures.

uniQure has resumed enrollment in the higher dose cohort of its phase 1/2 study (NCT05243017) of AMT-130 in European adults with Huntington disease (HD) on the recommendation of a Data Safety Monitoring Board (DSMB).1

“We are pleased that following a comprehensive review of all available safety, biomarker and imaging data, the DSMB has recommended that we resume patient enrollment at the higher dose in the ongoing Phase 1b/2 study evaluating AMT-130 in HD,” Matt Kapusta, chief executive officer, uniQure, said in a statement.1

uniQure had previously paused enrollment in the study after the occurrence of suspected unexpected severe adverse reactions (SUSARs) in the high-dose cohort while the low-dose cohort remained unaffected.2 Three patients treated with a high dose were affected with localized inflammatory responses and other related symptoms; 2 from a single clinical site in the European cohort and 1 from the US cohort. The US patient’s symptoms were originally deemed unrelated to the therapy in March 2022 but were reclassified as a SUSAR following the European reports.

The DSMB made the recommendation to resume enrollment after reviewing the available safety, biomarker and imaging data from the ongoing trials of AMT-130.1 All 3 patients affected by the SUSARs have since fully recovered. The DSMB’s recommendation also included the implementation of additional risk mitigation procedures during the first 2 weeks after administering AMT-130. These measures include a 7-day, in-person, post-surgical visit. The DSMB reaffirmed the treatment protocols in the trial, including the use of immunosuppression at the discretion of the treating physician.

READ MORE: Huntington Disease Gene Therapy Promotes Reduction in Mutant Huntington Protein, Neurofilament Light

“Patient safety will always be our utmost priority, and we are grateful for the close collaboration between our clinical team, study investigators and most importantly, the patients, all of whom were instrumental in completing our comprehensive investigations. We believe that AMT-130 has the potential to provide a positive impact for patients with this devastating disease for which there is no currently approved treatment. We look forward to finishing patient enrollment in the higher-dose cohort of the European study in the first half of 2023 and remain on track to announce data from the U.S. Phase 1/2 study in the second quarter of 2023,” Kapusta added.1

uniQure will resume patient dosing as soon as possible. Altogether, 10 patients have been treated in the European study, 6 in the low-dose cohort and 4 of 9 in the high-dose cohort. The randomized, double-blinded phase 1/2 US study (NCT04120493) is fully enrolled, with 10 patients in the low-dose cohort and 16 in the high-dose cohort. Further 1-to-2 year follow-up data from the US trial will be announced in the second quarter of 2023.

1. uniQure announces third quarter 2022 financial results and highlights recent company progress. News release. UniQure. November 2, 2022. https://www.globenewswire.com/news-release/2022/11/02/2546342/0/en/uniQure-Announces-Third-Quarter-2022-Financial-Results-and-Highlights-Recent-Company-Progress.html
2. uniQure Announces Second Quarter 2022 Financial Results and Highlights Recent Company Progress. News release. UniQure. August 8, 2022. https://www.globenewswire.com/news-release/2022/08/08/2493736/0/en/uniQure-Announces-Second-Quarter-2022-Financial-Results-and-Highlights-Recent-Company-Progress.html
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