uniQure N.V. Stock News

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UniQure says it still sees a path to regulatory approval for the drug candidate, AMT-130, even as the experimental gene therapy for Huntington's disease falls hort in a recent study.

Uniqure stock plummeted Tuesday on a disappointing four-year update for its experimental gene therapy in patients with Huntington's disease.

UniQure , said ​on Tuesday ‌its Huntington's treatment showed ​44% ​slowing of disease ⁠progression, but did ​not reach ​statistical significance in an ongoing ​early-to-mid-stage ​trial.

~ In 12 high-dose patients at 48 months, both cUHDRS and TFC continued to demonstrate meaningful slowing of disease progression and clear dose-dependent response; the primary endpoint of cUHDRS showed 44% slowing of disease progression (non-significant p=0.144) and TFC showed a 61% slowing of disease progression (nominal p=0.008) ~

Uniqure stock wavered Wednesday after the Food and Drug Administration accepted its application for approval of a Huntington's gene therapy.

Dutch gene therapy developer uniQure said on Wednesday it had applied for U.S. approval of its experimental treatment for Huntington's disease after the FDA reversed ​its earlier position on the drug, sending the company's shares ‌up 5% in premarket trading.

~ Company also announces the submission of a Marketing Authorisation Application for ifezuntirgene inilparvovec in the U.K. ~ ~ Both submissions are supported by the three-year data analysis from the Phase I/II study, in which ifezuntirgene inilparvovec demonstrated a slowing of disease progression ~ LEXINGTON, Mass. and AMSTERDAM, Sept.