Arrowhead Pharmaceuticals is getting the green light to move ahead with studies on two of its drug compounds ... in New Zealand.
Arrowhead is based in Pasadena, California but its research and development team is in Madison. As of about a year ago, that included about 65 people.
The New Zealand Medicines and Medical Devices Safety Authority has given Arrowhead permission to hold the first human studies of ARO-HBV, a possible treatment for patients with the chronic hepatitis B virus, and ARO-AAT, being developed to treat a rare, genetic liver disease associated with the alpha-1 antitrypsin deficiency.
The company said it expects to be able to start administering doses of the drugs in late March and it will monitor the safety, tolerability and any effect on the study participants.
The chronic hepatitis B study will involve up to five groups of six patients each; the drug for the genetic disease will track up to five groups with eight subjects each.
Arrowhead is developing drugs to treat diseases by silencing the genes that cause them, using RNA interference.
Arrowhead also has received orphan drug designation from the U.S. Food and Drug Administration for ARO-AAT, the drug to treat the rare liver disease. The company had filed a clinical trial application in December 2017, asking for regulatory permission to start its first studies on humans with ARO-AAT.
Arrowhead has several other potential drugs in the pipeline, some also aimed at liver diseases and others directed at different targets, CEO Christopher Anzalone told a conference call with analysts earlier in February.
One drug, ARO-HIF2, is being developed to treat clear cell renal cell carcinoma, and ARO-LUNG1 is designed to fight an undisclosed lung disease, he said.
"We have generated some very promising data showing that after inhaled administration we can achieve substantial knockdown of lung expressed targets with long duration of effect. We can foresee a host of new diseases we can attack and multiple targets for each of those diseases," Anzalone said.
The publicly traded company sold more shares of stock in January, providing net proceeds of $56.8 million, chief financial officer Ken Myszkowski told analysts. He said that will help Arrowhead "continue to advance our pipeline through the clinic for many quarters."