Pearl Therapeutics, Fresh Off Big VC Round, Tops $3B Lung Drug in Head-to-Head Trial

12/1/10Follow @xconomy

Pearl Therapeutics bagged a whopping $69 million venture round last month, and now it’s pretty clear why.

The Redwood City, CA-based biotech company is reporting clinical trial results today that show its inhalable drug for chronic obstructive pulmonary disease worked better than standard Tiotropium (Spiriva), a $3 billion annual seller for Pfizer and Boehringer Ingelheim. The Pearl Therapeutics product, which uses a combination of two treatments in a standard inhaler, was about 50 percent more effective than other therapies it was compared against in a study of 118 patients with moderate to very severe chronic obstructive pulmonary disease, says Pearl CEO Howard Rosen. The difference was highly statistically significant, but Pearl is saving most of the important detailed results for a scientific meeting and peer-reviewed publication in 2011.

“Being able to increase lung function 10-15 percent can be difference between sitting around all day versus going to work and doing normal activities,” Rosen says. “This is as good as, or better than, what we had hoped for.”

Pearl is racing to be one of the new leaders in field of developing drugs against chronic obstructive pulmonary disease, an umbrella term for emphysema and chronic bronchitis, which is usually brought on by smoking. The disease is now the fourth-leading cause of death in the U.S., killing 100,000 people a year, according to the National Emphysema Foundation. Emphysema alone is estimated to cost the U.S. health system an estimated $5 billion to $9 billion a year. Treatments on the market today, like Spiriva, are only designed to treat symptoms and aren’t really able to alter the course of the disease.

Pearl made a big splash last month when it scored the large venture round from Vatera Healthcare Partners, Clarus Ventures, New Leaf Ventures, and 5AM Ventures. Those groups rallied behind its concept of creating what’s known as a LAMA/LABA combination, which essentially creates a simple inhalable formulation of two drugs that have two different ways of working to clear up constricted airways. Specifically, the company’s lead candidate combines glycopyrrolate with formoterol in a product that’s dubbed PT-003.

Anytime a $3 billion drug like Spiriva comes along in such a broad potential market for a life-threatening condition like chronic obstructive pulmonary disease (COPD), you know every Big Pharma company has its eye on competing for a piece of the market. It’s true in this case, where GlaxoSmithKline has had a longstanding collaboration to create a LAMA/LABA combo drug with South San Francisco-based Theravance (NASDAQ: THRX), as does Forest Laboratories (NYSE: FRX) and Swiss drug giant Novartis.

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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