Why uniQure’s stock price jumped today

What happened

After gene therapy specialist uniQure (QURE 1.00%) announced positive interim results from a Phase 1/2 clinical trial early Thursday, its stock surged, gaining 18.5% as of 10:54 a.m. ET. This gene therapy, AMT-130, is intended to treat Huntington’s disease and according to the latest update, it appears to have an acceptable side effect profile when used at low doses. Even 12 months after treatment, there were no serious adverse events related to it among the six patients receiving treatment in the study’s low-dose cohort.

The company also reported preliminary evidence suggesting the drug may be effective in reducing levels of certain disease-related biomarkers. The report did not say whether patients in the trials actually had fewer symptoms at this time.

So what

AMT-130 is uniQure’s most mature wholly-owned pipeline program, although it has another treatment in Phase 3 trials that it is pursuing with the help of a research and development collaborator. Thursday’s update therefore means that biotechnology is on the verge of generating revenue from sales and becoming profitable without outside help.

But it’s important to remember that the road to commercialization of AMT-130 is still quite a long one and a lot could go wrong along the way.

Now what

During the second half of this year, uniQure will seek to finalize the recruitment of an open label trial for AMT-130 in the European Union. Then, in the second quarter of 2023, it will analyze two full years of safety and efficacy follow-up data for the low-dose cohort, and review the first year of data for the high-dose cohort.

All eyes will be on the low-dose wing efficacy data in any future data updates, as they will illuminate the promise of AMT-130 as a functional treatment for Huntington’s disease.

Alex Carchidi has no position in the stocks mentioned. The Motley Fool has no position in the stocks mentioned. The Motley Fool has a disclosure policy.

Karen J. Nelson