Arrowhead CEO Christopher Anzalone Addresses This 'Really Important Event' In Lung Disease

Anzalone says that the company is "cautiously optimistic" that ARO-RAGE will help improve lung function in patients with idiopathic pulmonary fibrosis.

Arrowhead CEO Christopher Anzalone Addresses This 'Really Important Event' In Lung Disease

Arrowhead Pharmaceuticals, Inc. (ARWR), announced Thursday that a single dosage of its experimental drug nearly eliminated a protein linked to asthma. In response, ARWR stock jumped.

Arrowhead has released the first results from its study on a high dose of ARO-RAGE, an inhaled treatment. RAGE stands for receptor for advanced-glycation-end products, which is a protein found on lung cells. It's also associated with asthma and other lung diseases.

The treatment reduced the amount of protein in volunteers by up to 95%. Arrowhead, according to Investor's Business Daily, said that the 184-milligram dosage achieved similar reductions among asthma patients as did a 44 milligram dose for healthy volunteers.

Christopher Anzalone, Arrowhead's Chief Executive Officer, says that the results provide new evidence that the company’s platform -- which involves silence genes -- can work in an increasing number of organs. RNA-interfering or RNAi drugs have traditionally been used to treat liver diseases.

He said, "I'm not going to overstate this, but it's an important event," in May just before the release of data. "We have always believed that RNAi was a very powerful modality, but only if you can apply it to diseases where it can be used. This is a new area that is vital for the field.

ARWR's stock rose 2.4% in premarket trading today, near 35.20.

ARWR Stock - A Breakthrough in Lung Disease?

Anzalone believes RNAi is in a heyday. ARO-RAGE's results are also a breakthrough in pulmonary diseases and could boost ARWR stock.

Inhalable drugs are tricky to make. Arrowhead discovered this with an early study on another drug for cystic fibrosis. It went back to drawing boards. Researchers consider that the RAGE is an unreachable protein. He said there's "cemetery of failures" in this space.

It remains "a target-rich environment."

He said that "it's a variety of targets which have been unreachable." "So, now we could blow the door wide open in these areas."

Arrowhead Pipeline Expanded

Arrowhead also studies ARO-RAGE for chronic obstructive lung disease (COPD) and pulmonary fibroids. ARWR's stock soared by almost 12% when Arrowhead announced interim results of ARO RAGE at lower doses on April 25.

Arrowhead is attempting to boost its pipeline. By 2025, the company hopes to market or have in clinical trials 20 products using its new system called TRiM.

Arrowhead shares are down in recent weeks after reaching a high of 42.48 early May. indicates that ARWR remains above both its 50-day and 200-day moving averages.