Big Pharma giants are gathering in Chicago this week to wine and dine doctors at the American Heart Association's Scientific Sessions, hoping docs will keep prescribing their multi-billion-dollar heart drugs. Not many little biotechs can compete in this league, but Hayward, CA-based Anthera Pharmaceuticals
Anthera is a small fish in this pond, having raised about $37 million in its initial public offering and burned through just about $93 million of investor cash in its five-year history. Anthera certainly fell off a number of radar screens when its stock dipped from its initial price of $7 to a low of $2.82 earlier this year. But the company has since bounced back to $6.12 at yesterday's close, and it is clearly eager to show some early stage clinical trial of its lead drug at the American Heart Association meeting on Wednesday.
The idea at Anthera is to help prevent relapses in seriously ill heart patients by tamping down excess inflammation. The idea that inflammation causes plaque buildups in blood vessels to rupture, leading to heart attacks and strokes, hasn't been solidly established in the scientific community, but evidence has been mounting for years to support the idea. Anthera, founded in 2004, is now in the midst of working to prove that a once-daily anti-inflammatory pill can significantly reduce the risk of heart attack, stroke, and death when taken in combination with the whole kitchen sink of other drugs like cholesterol-lowering agents, blood pressure meds, and anti-clotting agents. If Anthera can gather that kind of proof, it may have a drug that could be taken by as many as 1.5 million patients in the U.S. who get rushed to the hospital each year with a heart condition and are hoping to avoid a relapse that could be debilitating or fatal. The drug could be worth "multi-billions," Anthera says.
"This is a true paradigm shift in the treatment of cardiovascular disease," CEO Paul Truex says.
Anthera has at least a couple years of work ahead before it will know if it can live up to that billing. But the company has clearly staked out an interesting strategy in pursuit of the next big market for cardiovascular disease, in a way that a little cash-burning biotech company might actually be able to handle. The company, founded in 2004, got its early backing from Vantage Point Venture Partners, Sofinnova Ventures, Caxton Advantage Life Sciences Fund, HBM BioCapital, and others.
The lead drug Anthera's investors are betting on here is varespladib methyl (A002), which was originally designed by Eli Lilly and Japan-based Shionogi to inhibit multiple forms of an inflammatory enzyme called sPLA2. There is no FDA approved drug that works to block this target, although competitors like GlaxoSmithKline, Via Pharmaceuticals, and Eisai Pharmaceuticals are all pursuing some variation on the theme.
By shutting down the enzyme, Anthera hopes it will prevent plaque from forming in blood vessels, and make it more stable there, Truex says.
Anthera, which got a license to the compound from Lilly and Shionogi, went on to run mid-stage clinical trials. It tested its drug in more than 1,000 patients, which gave it enough evidence to entice IPO investors. The extra cash was immediately put to work in the next couple months as Anthera started enrolling patients in the pivotal trial called Vista-16, which it hopes will be good enough to convince the FDA to clear the drug for sale.
The study will randomly assign as many as 6,500 patients with a heart attack (acute coronary syndrome) to get either the Anthera drug or a placebo. These are very sick patients, taking lots of heart medications already, and about one out of every 12 (8.5 percent) is expected to have another serious coronary event, Truex says. About 85 percent of the coronary events are thought to occur in the first 16 weeks, when inflammation is running rampant, so Anthera has designed its study around this short-term (and therefore, stock market-friendly) time frame.
Anthera designed the study, so the moment there are 385 major coronary events in the study (heart attacks, strokes, deaths), then its statisticians will rip off the study blind and see whether the patients on the Anthera drug suffered those outcomes at a lower rate. To declare victory, Anthera needs to see an 18 percent improvement in relative risk compared with the placebo. That might sound great, but in absolute terms, it's pretty modest. If Anthera's drug is that good, then about 6.9 percent or 7 percent of patients on it (about one out of 18 instead of one out of 12) will still suffer a major coronary event.
One of Anthera's main tasks is to generate enthusiasm among doctors and patients for its trial. Because it needs to enroll an estimated 5,000 to 6,500 patients to get the required number of coronary events, it will take until about the end of 2011 to complete enrollment, Truex says. Results should be ready by the first half of 2012, and if all goes well, the company will be able to seek approval from the FDA by the end of 2012.
Since this would be coming online in the era of health reform, Anthera had better have a compelling reason for how prescribing the new drug can save the system money. Chief financial officer Chris Lowe was certainly ready for that question, although I'm sure that serious health economic studies will have to be done to make the argument really compelling. Just off the top, he noted that initial heart attacks cost the health care system between $20,000 and $40,000, but second ones tend to cost about three times as much because they are more damaging. If the Anthera drug can reach its primary goal, he says, "then you have a drug that will provide serious benefit to the health care system."
There's an interesting financial strategy at work here, too. Anthera is going it alone, with a team of just 25 employees, managing this pivotal trial and hoping to ship off to the FDA. Rather than hand over the majority control of the drug to a Big Pharma partner in exchange for cash, the board chose to raise more money through the IPO and retain 100 percent of the drug's rights in North America and Europe. It was feasible to do this for a relatively small pivotal study for cardiology, which usually requires 10,000 or more patients followed over a fairly long period of time -- a proposition that requires too much time and money for most biotechs. So if Anthera can hold on all the way through Phase III, and deliver the reduced risk of heart attack and stroke it foresees, then it could have a pretty valuable property to sell to one of those Big Pharma companies looking to fill holes in their pipelines left by the billion-dollar heart drugs of yesteryear. Anthera says it believes it has patent protection on its new drug out to 2019.
That would certainly provide Big Pharma companies many more opportunities to wine and dine doctors like the ones in Chicago this week, regaling them with stories about how this drug helps patients and reduces costs.
"We think this aligns the interest of payers, patients, and shareholders, ultimately," Lowe says.
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Luke Timmerman is the National Biotech Editor of Xconomy, and the Editor of Xconomy Seattle. You can email him at email@example.com, or follow him at twitter.com/ldtimmerman.