So just as a way of introduction, the AMT-130 study, remember, is a single-dose gene therapy trial. All of the patients that are presented, they had their surgery to implant the drug into their striatum over three years ago, and the presentation is on the patients that have reached the three-year follow-up data. And the first thing is it’s a safety study. So the most important thing from a safety standpoint is there are no new safety events...
So just as a way of introduction, the AMT-130 study, remember, is a single-dose gene therapy trial. All of the patients that are presented, they had their surgery to implant the drug into their striatum over three years ago, and the presentation is on the patients that have reached the three-year follow-up data. And the first thing is it’s a safety study. So the most important thing from a safety standpoint is there are no new safety events. There are no new neurologic problems or things coming up at the three-year mark. So that’s important to note as gene therapy is invasive and, you know, continued safety or no new safety things is a good sign. And then in particular, the results that have made a lot of buzz are the clinical results for these patients. So all of the patients were unblinded at a year. So in the three-year analysis, we’re comparing to an external matched control group. And compared to that external control group, the treated patients are relatively stable, whereas the external control group are following the natural progression of the disease. And so at the three-year mark, this continued further separation from the control group. And so the treated patients are significantly less progressed than the external control group. And when you compare the differences in how much they progress, it’s about 75% less progressed. So that’s great. And it’s on a composite Unified Huntington’s Disease Rating Scale measure. A composite measure combines motor, cognitive, and functional measures in there. And then the other detailed part of it is that in a composite measure, you can sometimes hide something negative with something positive. So it’s important to note that the individual subscales of the composite measure are also all positive, favoring the treated patients. So that’s, again, a positive result. And then the last major result that’s being presented is the CSF neurofilament light chain data, which neurofilament light is a known biomarker for progression in a lot of neurodegenerative diseases, such as MS, ALS and is increasingly accepted in Huntington’s disease as well, normally increases over the lifetime of a patient with neurodegenerative disorders, and in this trial, the patients had about this flat or slightly decreased NFL levels at the three-year mark compared to what we would think should be an increase over time. So those are the summary of the findings of the AMT-130 trial that are being presented here today.
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