San Diego-based Arena Pharmaceuticals has spent a dozen years, and raised almost $1 billion from investors and partners, to create a new drug that helps millions of people lose weight. This month, the company will find out whether the time and money was well spent.
Arena (NASDAQ: ARNA) plans to unveil results of a 4,008-patient clinical trial in September that will answer vital questions about its experimental pill, lorcaserin. The study, called Blossom, is designed to see whether a high or low dose of the treatment can help patients lose weight over a full year compared to a placebo. The study will also look at whether the Arena drug helps with many ailments that stem from obesity—high blood pressure, cholesterol, high blood sugar, and inflammation, to name a few.
If this clinical trial confirms the findings of another major trial from earlier this year, Arena will bundle the data together and ship it to the FDA by the end of this year for approval to start selling lorcaserin in the U.S. Arena will have a very busy fall, as the results are scheduled for a full presentation at The Obesity Society’s scientific meeting in October. If the results withstand all that public scrutiny, the company hopes to entice a Big Pharma company with the muscle to put together a mass marketing campaign aimed at the roughly two-thirds of people in the U.S. who are considered overweight or obese.
“It’s an exciting time,” says Arena CEO Jack Lief. “It’s been a long time. I can’t wait.”
Any drug for obesity has to have a squeaky clean safety profile because it would potentially be taken by millions of people without an imminently life-threatening condition like cancer.
While the bar is high, obesity represents a potentially gigantic market. The leading treatment on the market is a four-decade old generic amphetamine, called phentermine, that most people avoid because it causes insomnia and high blood pressure.
Arena really has not taken any interim peeks at the clinical trial results while the study is ongoing, so it will have a motherlode of raw data to sift through for the first time. When I spoke with Lief and Arena’s chief scientific officer, Dominic Behan, last week, they were optimistic as usual that this study would confirm the safety and effectiveness they saw earlier this year in the 3,182-patient clinical trial known as Bloom. “We don’t expect any bad stuff to come out, because Bloom was so robust,” Lief says.
Based on those results, and assuming that Blossom provides the added confirmation the FDA wants to see, Arena has clearly been sharpening up its marketing strategy and competitive position against rival treatments from San Diego-based Orexigen Therapeutics (NASDAQ: OREX) and Mountain View, CA-based Vivus (NASDAQ: VVUS).
Both Arena and Orexigen underwhelmed investors when they released their first clinical trial results. Orexigen, based on my recent interview with CEO Mike Narachi, says it doesn’t plan to market its treatment to the masses who are a little bit overweight, but instead wants to pitch it to people who are more seriously obese, and need to lose weight to protect themselves from all the “co-morbidities” of obesity, like diabetes and high cholesterol.
Arena, by contrast, says its safety profile was so clean in the earlier study that it has confidence to market its product to the broader population of people who want to lose weight, Lief says. The drug showed an ability to be used in a broad patient population, help people lose weight fast, keep it off for a year, and improve important secondary measurements of cardiac and metabolic health, Behan says.
Importantly, the earlier study followed patients for two full years to see whether the Arena drug caused any damage to the heart, and didn’t show any serious concerns. This type of aggressive monitoring was required by the FDA, because Arena’s drug is designed to work in a similar way as Wyeth’s fen-phen combination did in the 1990s, before that drug was pulled off the market because it damaged heart valves. The Arena drug was designed to be more specific—to interact with an enzyme in the brain that controls feelings of fullness—without hitting a similar enzyme on the heart that led to the undoing of fen-phen.
Arena has heard the criticism from investors who say the safety looks fine, but the effectiveness isn’t that compelling. Lief took that one head-on when pressed. “We’re not talking about a wimpy drug from an efficacy perspective,” he says.
To buttress his case, he pointed to results from the earlier study that showed two-thirds of patients lost at least 5 percent of their body weight on lorcaserin, more than double the number who did that well in the placebo group. About one-third of patients on lorcaserin lost 10 percent of their body weight, which was triple the rate who achieved that threshold on a placebo, Lief says. Those readings of effectiveness meet one of the important criteria the FDA has said it wants to see from any new obesity drug.
What that shows is that individual patients can respond quite differently to the drug, and since obesity is related to so many genes, the company can’t say for sure which patients are likely to respond or why. When the data is taken all together, the average patient lost 5.8 percent of their body weight on lorcaserin, compared with 2.2 percent weight loss among those on placebo. The FDA likes to see at least 5 percentage points of improvement from an obesity drug, so Arena fell short on that score.
Lief dismissed that as unimportant, saying the FDA only needs to see a drug hit one of the two main effectiveness goals, not both. “No single drug has ever done that reliably,” he says.
Arena is clearly going to hang its hat on safety. The way I heard it, the message boils down to this: it won’t hurt, and it might help. “We’re positioning our drug to be the standard for broad usage,” Lief says. “Most people can benefit from losing some weight.”
And of course, physicians, the FDA, and insurers will look closely at all the secondary measurements of benefit to see if the drug is really worth it. This demanding set of measurements is part of why the drug development process takes a long time and costs so much. Arena obviously hopes these results will be bulletproof, because it’s hard to imagine a do-over for something with such high stakes.
“We’re going to have robust data from more than 7,000 patients. There were no shortcuts,” Lief says.
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