Arrowhead Pharmaceuticals has initiated a Phase 2 clinical trial for its drug candidate aimed at treating rare liver diseases associated with alpha-1 antitrypsin (AAT) deficiency, the company said on Tuesday.
AAT deficiency is a genetic disorder that involves mutation of the gene that encodes the AAT molecule. With this mutation, AAT gets trapped inside the liver cells that produce it, rendering the molecule unable to perform functions such as protecting the lungs from inflammation. That can increase the odds of developing lung disease, while the buildup of AAT proteins in the liver can also lead to disease in that organ.
The recently launched study of ARC-AAT, as Arrowhead’s drug candidate is known, will involve eight to 12 patients with the disorder, the company said in a news release. They will be divided into two cohorts, with one receiving a larger dose of the drug. Both groups will receive seven doses of ARC-AAT, with four-week intervals in between treatments. Likewise, all patients will undergo pre- and post-dose liver biopsies, with the latter completed at day 183, the company said. The trial will take place at facilities in Canada, Ireland, and Sweden, according to the release.
“There remains no medical treatment for the liver disease associated with AAT [deficiency], which is increasingly being recognized by patients and physicians as a serious problem,” Bruce Given, Arrowhead’s chief operating officer, said in a prepared statement. “Our Phase 2…study should give us, and the AAT [deficiency] community in general, the first insights into whether ARC-AAT can stop the progression of liver disease and possibly even allow the liver to recover and heal existing damage.”
Other companies with AAT deficiency drugs in development include Israel-based Kamada (NASDAQ: KMDA) and Cambridge, MA-based Intellia Therapeutics (NASDAQ: NTLA), which is working to create treatments with CRISPR-Cas9 gene editing technology and went public in May.
Arrowhead (NASDAQ: ARWR) is headquartered in Pasadena, CA, but its research and development operations are based in Madison, WI.
ARC-AAT has been designated as an orphan drug in the U.S. and Europe, a first for Arrowhead. President and CEO Chris Anzalone has said previously that the number of people in the U.S. with AAT deficiency is estimated to be about 100,000, and the total is thought be similar in Europe.
Shares in Arrowhead closed at $7.16 Tuesday, up slightly from Friday’s close. The company’s stock price has increased more than 125 percent from February, when it dipped to $3.18 per share. Shares briefly climbed above the $8 mark last month, before giving back some of those gains.