The interim Phase I trial data was only from 35 people, but results look good so far.
An experimental gene-editing therapy that aims to lower bad cholesterol for the long-term after a single infusion is off to a positive start in an early clinical trial.
Researchers running a Phase I safety trial for the drug, dubbed VERVE-102, published interim results from just 35 patients this week in the New England Journal of Medicine. Though the numbers are small and the analysis is preliminary, VERVE-102 appeared safe, with no serious adverse events reported from the treatment, even at the largest doses. The most significant finding was a temporary, mild increase in a liver enzyme that suggested minor injury in the liver, where the drug works.
The small amount of data also hints that the drug is effective. The subgroup of participants who received the largest dose have seen their bad cholesterol—that is, their low-density lipoprotein or LDL—drop 62 percent, to a mean of 78 mg per deciliter. For people with high cholesterol—like the participants in the trial—a reduction of this magnitude could cut the risk of cardiovascular disease from plaque buildup in arteries by an estimated 50 percent if it’s sustained for over 20 years. The trial only has up to 18 months of follow-up data so far, but from that, the positive effects of VERVE-102 seem to be holding up. The LDL reductions have been sustained in all the subgroups.
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