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to silence a specific stretch of RNA and, by doing so, enable the body to produce dystrophin. The drug recently earned notice in The Lancet when it showed an ability to restore some production of dystrophin proteins in an previous early-stage clinical trial—although it didn’t fully restore the dystrophin. That earlier trial also was very limited in that it involved a direct injection into a foot muscle, and the AVI drug wasn’t yet formulated into a more practical intravenous infusion that circulates throughout the body, as was used in the study reported today.
If AVI can show in subsequent trials that its drug is safe, and can be delivered throughout the body, it would be a big step forward even if it’s modestly effective, University of Washington researcher Jeff Chamberlain told me back in September. About one in every 3,500 boys born worldwide have Duchenne Muscular Dystrophy.
AVI said its treatment appeared to be well-tolerated in the current study, and boys in the study reported the usual headache, muscle ache, and upper respiratory infections often seen in pediatric studies. One patient dropped out of the trial with heart damage (cardiomyopathy), although researchers didn’t consider that drug-related. Two more serious adverse events were reported among patients during the 14-week follow-up period after they quit taking the drug, and those also weren’t considered drug-related, AVI said.
Much more data is still to come from this trial in the U.K. Another eight patients are receiving treatment at higher doses of 10 and 20 milligrams per kilogram for 12 weeks, and if the trend continues from what researchers saw in the mid-range doses, then those boys could start producing even more dystrophin. AVI says it has completed the required studies in mice and monkeys that it needs to open a clinical trial in the U.S.
Once all of the follow-up data is in from this trial in the U.K., AVI is hopeful that it will be able to draw a connection between increased production of dystrophin and an improvement in patients’ muscular dystrophy symptoms. The full data will be published in a scientific journal, says CEO Les Hudson.
Shares of AVI climbed 6 percent at the opening bell today to $1.59.