Chelsea, MA-based Civitas Therapeutics announced today that its inhaled form of L-dopa—the drug that many Parkinson’s disease patients take to relieve shaking and other debilitating symptoms—performed well in a Phase 1 clinical trial. The trial was designed to test whether the drug, called CVT-301, is safe, and whether delivering it to the lungs gets it into the bloodstream at levels that are likely to produce a therapeutic outcome. So far, so good, the company says. Civitas will present comprehensive data from the study at a future scientific gathering.
Civitas seems to have passed a small, but important, first test for CVT-301. In 2011, Civitas was spun out from Waltham, MA-based Alkermes (NASDAQ: ALKS), which had recently abandoned plans to develop an inhaled insulin product for diabetes. Civitas started up with $20 million in financing from Canaan Partners and Longitude Capital, and a state-of-the-art, 90,000 square foot manufacturing and R&D facility that it inherited from Alkermes. “Right when we walked in, we were in brand spanking new labs,” says Glenn Batchelder, co-founder and CEO of Civitas.
The idea behind Civitas was, in the most basic terms, to find a better use for the technology that Alkermes and its development partner Eli Lilly (NYSE: LLY) spent millions to develop. Inhaled insulin didn’t gain traction in the diabetes market, Batchelder says, because the devices that were initially developed were large and clunky, and therefore not very convenient for patients to carry around. More importantly, he says, inhaled insulin “didn’t have a clinical benefit” over injected insulin. “That was a learning.”
Civitas pursued L-Dopa because of the potential for an inhaled form of the drug to surpass the pill form in both convenience and performance, Batchelder told Xconomy during a December tour of the company’s headquarters. The pill form of L-dopa works, but some patients have to take it several times a day to see any impact, and it can be difficult to control how much of the drug gets into the bloodstream. “Food affects the variability,” Batchelder says. “They never know how much of the drug is getting to the blood. There’s also variability in how long it takes for the drug to produce an effect. Some people stop living their lives because the pills don’t solve the problem.”
CVT-301 is a fine powder that’s delivered via a small, plastic inhaler similar to an asthma inhaler. Civitas only had to make a few small adjustments to the diabetes inhaler to ensure the device would be easy enough for Parkinson’s patients to handle, Batchelder says. “You just breathe in, and the powder goes into the lungs,” he says. “No matter how hard you breathe, you get the same exact dose.” The device, unlike some of the early insulin inhalers that were developed, is small and discreet. “It’s five plastic parts, two springs, and a staple,” Batchelder says. According to the company’s statement, CVT-301 delivered consistently precise doses during the Phase 1 trial, and the drug was immediately absorbed into the body.
Civitas is developing CVT-301 with the help of New York-based Michael J. Fox Foundation for Parkinson’s Research, which announced on November 29 that it had awarded a grant of undisclosed size to the company. And Civitas inherited most of the manufacturing equipment it needs—not to mention tons of supplies—from Alkermes. During the recent tour, Batchelder and Civitas’ chief scientific officer Rick Batycky pointed out a cavernous room on the ground floor, which is serving as a holding pen for fillers, solvents, and other lab supplies, until it’s needed for commercial manufacturing. “The labs are outfitted with everything they need,” Batycky says. “When they run out, they just come here. It’s like going to Wal-Mart. It’s very valuable. We haven’t needed to buy a lot.”
If all goes well in future trials, Civitas hopes to file for approval in 2015, Batchelder says. With so much of the manufacturing infrastructure already in place, it won’t be hard to get up and running fast, he says. “That’s the beauty of having this facility,” he says. “It gives us the ability to scale this up and commercialize the drug. We wouldn’t have been able to create that as a small company.”