Tucked in its Q2 earnings release, UniQure N.V. (NASDAQ: QURE) said that in July, it had observed suspected, unexpected severe adverse reactions (SUSARs) in two patients after they were treated with the "higher dose" of AMT-130 in a European Phase 1b/2 trial.
AMT-130 is a gene therapy candidate for Huntington's disease.
A third patient treated back in March in the U.S., had their side effect deemed unrelated to the candidate, but it was then reclassified as a severe adverse reaction after review.
As a result, it's halted dosing in the high-dose cohort. A safety review is ongoing and expected to complete in Q4 of 2022
The DSMB does not view these findings as dose-limiting toxicity, and no clear root cause has yet been identified.
The company added that they are considering potential risk mitigation plans over the next 2-3 months.
The patients were hospitalized, with the U.S. patient suffering a severe headache.
The company held cash and cash equivalents of $500.5 million, sufficient to fund operations into 1H of 2025.
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