Arrowhead Pharmaceuticals said Thursday a single dose of its experimental drug almost completely eliminated a troublesome protein tied to asthma. In response, ARWR stock yo-yoed and ended the regular session with a fractional gain.
The results are the first from Arrowhead's study of a high-dose of its inhaled treatment, known as ARO-RAGE. RAGE is shorthand for receptor for advanced glycation end products, a protein that shows up on lung cells. Further, it's associated with numerous lung diseases, including asthma.
On average, the treatment knocked down 90% of the protein and up to 95% in healthy volunteers. The 184-milligram dose achieved similar reductions in asthma patients as a 44-milligram dose in healthy volunteers, Arrowhead told Investor's Business Daily.
Arrowhead Chief Executive Christopher Anzalone says the results offer fresh evidence the company's platform — which involves silencing genes — can be used in a growing number of organs. Traditionally, RNA-interfering drugs, or RNAi, work in diseases of the liver.
"I don't want to overstate it, but I think it's a really important event," he said in May before the data release. "We always thought RNAi is a very important modality but, if and only if, you can bring it to the diseases that can be addressed by it. So, this is one more full space that is important for the field."
On today's stock market, ARWR stock rose 0.8% to close at 34.67.
ARWR Stock: A Breakthrough In Lung Disease?
Anzalone says it's a heyday for RNAi drugs. But the results of ARO-RAGE are also a breakthrough for pulmonary diseases, and could stoke ARWR stock.
First, making inhaled drugs is tricky. Arrowhead found that out with an early study of another drug in cystic fibrosis. So, it went back to the drawing board. Second, researchers consider the RAGE protein to be undruggable. There's a "cemetery" of failures in that space, he said.
But it remains a "target-rich environment."
"It's many different targets that have been undruggable," he said. "So now, potentially, we are blowing the door open in these areas."
Expanding Arrowhead's Pipeline
The study results released Thursday focus on asthma, but Arrowhead is also studying ARO-RAGE in chronic obstructive pulmonary disease, or COPD, and pulmonary fibrosis. ARWR stock surged almost 12% on April 25, the day Arrowhead unveiled interim results for lower doses of ARO-RAGE.
It's all part of an effort to really bolster Arrowhead's pipeline. The company hopes to have 20 clinical stage or marketed products that use its new system, dubbed TRiM, in the year 2025.
Arrowhead shares have pulled back as of late after hitting a recent high at 42.48 in early May. Still, ARWR stock remains above its 50-day moving average and 200-day line, MarketSmith.com shows.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
