An experimental RNA interference drug from Arrowhead Pharmaceuticals lowered levels of tau, a protein linked to Alzheimer's disease, in an early trial in healthy volunteers, BioPharma Dive and Clinical Trials Arena reported.

At the highest dose, given as two shots two weeks apart, the drug, called ARO-MAPT, cut tau levels by an average of 73% after 105 days, BioPharma Dive reported. Tau in spinal fluid fell by as much as 85% in that group, Clinical Trials Arena reported.

At lower doses, tau fell by 40% and 69%, both outlets reported. The effect was still seen 90 days after the last dose, which Arrowhead said supports dosing once every three months or less often, Clinical Trials Arena reported.

The drug is injected under the skin and still reached the brain, BioPharma Dive reported. That is a hurdle for many drugs because of the blood-brain barrier. Arrowhead said the results "provide clinical validation" of its delivery platform.

Side effects were mild, BioPharma Dive reported. They included reactions at the injection site, bruising, respiratory infections and headaches.

"These early clinical data are exciting because they demonstrate that ARO-MAPT can achieve deep and durable reductions of tau expression in the brain following a simple subcutaneous administration," said Arrowhead CEO Christopher Anzalone, Clinical Trials Arena reported.

Outside experts urged caution. Stifel analyst James Condulis wrote that the results "look compelling," but he said the drug is at a very early stage and tau has not been clearly proven as a good Alzheimer's target, BioPharma Dive reported. Other tau-targeting drugs have struggled, the outlet said.

The results come from healthy volunteers, not Alzheimer's patients, Clinical Trials Arena noted. The next part of the trial will include people with mild Alzheimer's disease, BioPharma Dive reported.

What we know: ARO-MAPT cut spinal-fluid tau by an average of up to 73% in healthy volunteers, and side effects were mild.
What we don't: Whether lowering tau will slow memory loss in people with Alzheimer's.