What happened
Shares of gene therapy specialist uniQure (QURE -0.22%) fell by 42% during the first three and a half days of trading this week, according to data provided by S&P Global Market Intelligence. The stock's big weekly loss stems from a mixed interim analysis of a phase 1/2 study for the experimental gene therapy AMT-130 for Huntington's disease.
Huntington's disease is an inherited neurodegenerative disorder that causes the breakdown of cells inside the brain. At present, there are no approved therapies to treat the underlying cause of this often fatal disease. uniQure is attempting to change this situation with its experimental gene therapy AMT-130, which works by lowering the production of the mutant huntingtin protein (mHTT).
So what
What spooked investors about this interim analysis? While uniQure painted an overall positive picture in its press release, the interim data from the trial were less than straightforward, to say the least. Specifically, patients in the high-dose cohort exhibited an increase in the level of mHTT, while patients in the low-dose cohort experienced a decrease in the disease-causing protein from baseline over the study period.
If AMT-130 works as advertised, however, both dosing cohorts should have seen a marked decrease in mHTT from baseline. In a conference call with analysts, uniQure's management blamed the assay involved in measuring mHTT levels in cerebrospinal fluid for the counterintuitive result. So, in a nutshell, it's not entirely clear whether these findings are biologically accurate or a byproduct of a faulty assay.
Now what
Despite these mixed results, uniQure plans on moving forward with AMT-130's clinical development. Nonetheless, investors may want to take a cautious approach with this gene therapy stock for the time being. The long and short of it is that AMT-130 is unlikely to be approved on an accelerated basis.
