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Pearl is betting that it has found a way to differentiate itself partly by using simple, stable metered inhalers like the kind used by over-the-counter bronchodilators like Primatene Mist. Other companies have attempted to use dry powder inhalers, which are harder for patients to use, Rosen says.
It’s always a tricky business to write about clinical trial data when you can’t get an up-close look at the full results, so it should be taken with some grain of salt what Pearl is reporting today. I pressed Rosen for details, and here’s what I picked up.
The trial was designed to see if the Pearl combo drug could surpass the market leader, Spiriva, its components given alone, and a placebo. The main goal was to see if patients on the Pearl combination had an improvement in FEV1 scores, a common measurement of lung capacity. The Pearl drug, taken once in the morning and once in the evening before bed, was superior to Spiriva and its other component, and the difference was highly statistically significant—meaning it is unlikely to be a fluke—Pearl says. The new drug combo also worked faster than the components alone, although Pearl didn’t say by how much.
The drugs were administered for one week. In terms of adverse events, Rosen says researchers saw the same things that have been observed in the individual components. “We didn’t see anything unusual, or out of the ordinary,” Rosen says.
Much more testing will be required to prove this drug is the real deal, and that’s partly why Pearl had to raise so much more money. Because it’s a combination therapy, another Phase II clinical trial of about 500 patients will have to be done between now and the end of 2012 to confirm the right dose, Rosen says. More money, or potentially support from a partner, will be required to take this drug all the way to FDA approval with a study of “probably over 1,000 patients,” Rosen says.
While there’s no way to say with a straight face that Pearl has the proof it needs to make this a real drug, it’s also an interesting sign of the times that it designed a more rigorous than usual clinical trial at this relatively early stage of the game. That was a risky decision, and one based on where Pearl sees the healthcare market going in the future, in a world that’s not just focused on effectiveness, but cost-effectiveness.
“It’s important to show early on you are better than the standard of care,” Rosen says. “That’s why we included Spiriva, and Foradil in our trials. People in the past were focused on showing the product was real and effective versus placebo. But we felt it was important to show early on that what we were working on was really worthwhile. It was a risk-it increased the complexity of the trial. The risk of small trial is that you might not show you’re as good as or better than what’s out there. We felt it was important to demonstrate.”
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