In September, Arrowhead Pharmaceuticals (NASDAQ: ARWR) released clinical trials data showing its hepatitis B virus (HBV) candidate had significantly lower levels of antigens. Last week, Chris Anzalone, president and CEO of Arrowhead—which is based in Pasadena, CA, but houses its R&D operations in Madison, WI—presented at Cantor Fitzgerald’s annual healthcare conference in New York. Xconomy Wisconsin spoke with Anzalone about current clinical trials involving Arrowhead’s drug candidates, the challenge of fundraising, and other topics. Here is a condensed and lightly edited transcript of our conversation.
Xconomy: Why did you and your team decide to change the name of the company from Arrowhead Research to Arrowhead Pharmaceuticals in April?
Chris Anzalone: It really just reflects where we are as a company. Given where we are with our multiple clinical programs, we feel more like a pharmaceutical company than a research organization.
But we will always be a research-based company, and the research isn’t going anywhere. If anything, we are doubling down on R&D. We are expanding our R&D capabilities in Wisconsin as we speak because we feel so strongly that our base technology is so powerful.
X: One of Arrowhead’s candidates that’s currently in human clinical trials, ARC-520, is for patients afflicted with HBV. What does the big picture look like when it comes to that virus?
CA: Chronic hepatitis B is the most common serious liver infection. It affects between 350 and 400 million people [worldwide]. It is a substantial unmet medical need because there’s no cure right now.
X: Can you describe the progress Arrowhead has made from the time you decided to pursue a HBV treatment to the present?
CA: Several years ago, when we decided to go after hepatitis B, the world thought we were crazy. [Investors on Wall Street] didn’t really appreciate the clinical problems associated with hepatitis B, and the size of the opportunity. Now, a vast majority of big pharmas and big biotechs have programs against hepatitis B. So we feel validated, and like we are the clear intellectual leaders of the space.
We’ve learned an awful lot about the virus. We had data come out, starting in the fall of 2015. We’ve literally changed textbooks on hepatitis B. I think that we are at a really interesting point in the life history of the company, as well as the life history of the study of hepatitis B, because I think we’re going to know a lot more about this disease, and about how well we can combat it, [by] the end of 2016.
X: Has the fact that, in your words, Arrowhead’s initial vision has been “validated,” made it easier to fund clinical trials and some of the other steps necessary in bringing a drug to market?
CA: When we started talking about [developing a drug for HBV patients], there were very smart biotech investors who got it. We acquired Roche’s RNAi business back in 2011. At that time, it was difficult for us to convince [investors] of the quality of this opportunity. But we just kept at it, and then we sort of turned the corner in the middle of 2012. Funding a company is never easy. We have found ample access to capital to run these studies. It’s been quite doable for us.
X: Can you talk about Arrowhead’s other candidate that’s currently in human clinical trials, for alpha-1 antitrypsin deficiency?
CA: Sure. Alpha-1 antitrypsin (AAT) deficiency is a genetic disease. It involves mutation of the gene encoding AAT. And this enzyme is important. Among other things, it protects the lungs from inflammation. People make boatloads of this stuff—maybe two grams a day. With this genetic mutation, you have these cells that are making loads and loads of protein, but they can’t get out of those cells. And so it’s really pro-inflammatory, and that can lead to liver disease.
X: ARC-AAT, Arrowhead’s candidate for AAT deficiency, has been designated as an orphan drug. Had you done that with previous candidates?
CA: This is the first time for us, and it’s important. This is a clear orphan population; it’s thought there’s about 100,000 potential patients in the U.S., and a similar portion in Europe. Orphan designation is going to help us not only with speed to approval, hopefully, but also will help us with communication to the FDA.
We are really in unchartered territory with the FDA on this. There has not been a drug that has gone after the liver disease associated with AAT deficiency before. So we need to work closely with the FDA to figure out what appropriate endpoints are, et cetera. The orphan designation will help us do that in a more efficient manner.