After gene-therapy specialist uniQure (NASDAQ: QURE) reported 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. That gene therapy, AMT-130, is intended to treat Huntington’s disease, and per the latest update, it appears to have an acceptable side effect profile when used at a low dose. Even 12 months after treatment, there weren’t any serious adverse events linked to it among the six patients receiving the treatment in the low-dose cohort of the study.
The company also reported some early evidence suggesting that the medicine could be effective at reducing the levels of certain disease-linked biomarkers. The report did not address whether patients in the trials actually experienced fewer symptoms as of yet.
AMT-130 is uniQure’s most mature wholly-owned pipeline program, though it does have another treatment in phase 3 trials that it’s pursuing with the help of a research and development collaborator. So Thursday’s update means that the biotech is one step closer to generating revenue from sales and becoming profitable without outside help.
But it’s important to remember that the road to commercializing AMT-130 is still quite long, and a lot could go wrong along the way.
In the second half of this year, uniQure will seek to finalize enrollment of an open-label trial for AMT-130 in the European Union. Then, in Q2 2023, it’ll analyze a full two years of follow-up safety and efficacy data for the low-dose cohort, and take a look at the first year of data for the high-dose cohort.
All eyes will be on the efficacy data from the low dose wing in any future data updates, as it will shed light on AMT-130’s promise as a functional cure for Huntington’s disease.