Pharmalot: So what went wrong with Krystexxa? Ferrari: I came on board right the time of the launch (in early 2011) and I think there were a couple of things. From my point of view, the launch of a product like this into a white space, if you will, or a market not well defined previously, without any competitors before us, took a lot of time to get the rheumatology specialists up to speed on this product. Only now can we say there’s an understanding of the product out there now. We were changing the treatment paradigm for a condition with nothing like it previously. Allopurinol (a pill used for chronic gout) was the primary treatment for 40 years, but there were not a lot of options, whether it worked or not.
So Krystexxa was not only a new option, but a new treatment – an intravenous that is administered every two weeks over a period of time. There were some expectations it would be a blockbuster. The fact is that it’s an orphan drug in an orphan space. It took a lot of work to educate the rheumatology community. It’s taken us a good year or year and a half to do that. But none of that was really done prior to the launch.
Pharmalot: Tell us about Krystexxa. Why should it be successful? Ferrari: Well, the infusion is for two hours. And a patient also has to be pre-medicated and has to remain after infusion, for an observation period. So it’s a three-hour process, on average and it happens every two weeks. But we’ve shown in our clinical trials that we’re treating a chronically bad situation and doing it in a more acute manner. What we see happening with current use is that most patients remain on the drug for approximately six months and are usually watched or maintained on an oral product afterwards, although there are patients who remain on the drug for an extended period of time – some do so over three years, but that’s the exception. Remember, these are refractory chronic gout patients – they’ve failed on other treatments.
Pharmalot: This is expensive, though, yes? Ferrari: The cost per vial is $2,900 approximately. And patients are getting somewhere, on average, of 10 vials in treatment over five or six months. But when you look where we are in the orphan space, you’re not on a drug for the duration of your life. With so many others, you are, whether it’s a good or bad outcome, and that can be very expensive.
Pharmalot: Where do you stand with the reorganization? Ferrari: We’ve reduced the entire workforce. A lot of people think we just reduced the sales force, but the entire workforce was cut by 35 percent. The sale force went from 62 to 38 people in the field, and so the savings over a 12-month period will probably give us somewhere in the neighborhood of $55 million. What’s we’ve done is refocused on specialties – rheumatology and nephrology. And even though we reduced the workforce, our sales went up. In the last quarter, we were able to say we saw a 30 percent increase in sales, quarter over quarter. It’s a great thing when you consider what happened to us in the second quarter, the lawsuit and the reorganization. But we’re done with the reorganization. I don’t see anything in the future right now that says we have to reduce more. We’re well-positioned with the product. And all of our studies are moving along very well over last three to six months.
Pharmalot: Tell us about the studies. How are these supposed to broaden the Krystexxa market? Ferrari: The focus is on a couple of areas going forward. One is dialysis. Patients with renal failure may also have RCG, but the problem is that physicians have limited options. Oral drugs have to be given at a low dose that’s not effective or not given at all. A question a nephrologist will ask is the drug taken out of system when we do dialysis? So we’re doing some kinetic work on the product to see if it is not removed during dialysis. This would open up a patient population for us. Nephrologists might put patients on the product if they don’t have to be re-dosed after every session of dialysis.
Pharmalot: What kind of population are we talking about? Ferrari: The dialysis population is about 450,000 right now in the US. And many are diagnosed with gout? Maybe 8 or 9 percent of that population has gout, so that’s 36,000. Now, how many of them have RCG? There are another few thousand patients with RCG. But for us, the issue is they’re probably not being treated with Krystexxa.
Pharmalot: Just the same, you’re still a one-trick pony. Ferrari: Well, we do get a little bit of revenue from a product that was around in the beginning of Savient, although it’s since gone generic. But we still do get some name brand sales, maybe a couple of hundred thousand in the last quarter. But with Krystexxa, there are other populations that we’re looking at. For instance, transplant patients are limited in terms of which oral drugs they can take. Renal transplant patients should not be given Allopurinol (a commonly used pill for chronic gout). So we’re running an induction and maintenance trial. This would involve someone with high serum uric acid levels and we give them Krystexxa for 4 to 5 months and later put them on oral maintenance. The difference here is you don’t have to have a certain serum uric acid level to get in the study. The label right now says the level should not be below 6. Right now, the now only patients we can accept on Krystexxa are patients who have not normalized their serum uric acid level. So the trial would be for patients at any level.
Right now, our market is approximately 120,000 people in the US. The induction and maintenance trial and dialysis trial and organ transplant trial will probably open our market to 400,000 to 500,000 patients.
Pharmalot: But you’re not going to go it alone, right? Ferrari: Yes, the other part we’re looking into is to see whether there are potential collaborators or products to bring into this company, sticking with the rheumatology and nephrology spaces. That’s the other piece as we move forward. But things are moving in the right direction. The stock was pretty low and briefly hit $3, we fought off a pretty vicious lawsuit. I think we’ve come back from the dead. So we can start to explore these opportunities. A lot of companies didn’t want much to do with us, thinking we wouldn’t survive. So we’d like a co-promote deal and we’d like to diversify the portfolio.
Pharmalot: And how is the partnership hunt going? Ferrari: We’re looking for a partner in Europe and the rest of the world. There are a number of companies that have expressed interest and we’re in active discussions. We don’t want to go it alone in Europe. It would have cost us $30 million or more to go forward in Europe at a time when we were battling on a lot of fronts. But right now, we have two years’ worth of cash. And we’re not under any pressure to raise any more. But we think any partnership ex-US will bring us some good revenues, including what we believe will bring upfront payments and royalties. Right now I think we’re in a pretty good spot.
Pharmalot: Any other milestones coming up? Ferrari: We’re expecting to hear back from the CHMP in Europe over the next couple of weeks on an opinion. That will be a milestone. We haven’t seen anything, in theory, to suggest we won’t, so at this point, we believe we’re headed on the right track. Again, our goal is that we’ll have a partnership hopefully announced in the near future, but I don’t have a firm contract with anybody at this point, but we’re evaluating offers and expect a good solid partner for Europe in the near future. We’ll also have a lot of activity at the ACR (the annual American College of Rheumatology meeting, which takes place in Washington, DC, next month). There will be eight abstracts published, some oral presentations. And the induction and maintenance will take a period of time, but will take place in US and Europe, which should speed that up. The other two trials – dialysis and transplant – should not take that long…
Right now, the percentage of patients who respond to Krystexxa is between 40 and 50 percent. We want to know, from an immunogenicity point of view, why some people do not respond… It’s a foreign enzyme to the body. So what we’re looking at is to find out why this is happening. We’ll institute this in 2013. We’re looking to better understand response rate in order to keep more patients on the drug than are on it right now.