For decades, people with Parkinson’s disease have been taking L-dopa pills to keep symptoms of the neurodegenerative disease under control. But now the Michael J. Fox Foundation is betting that a new form of the drug can be inhaled into the lungs to provide fast relief when the old standby isn’t getting the job done.
The New York-based Michael J. Fox Foundation for Parkinson’s Research is announcing today it is providing an undisclosed grant award to help Chelsea, MA-based Civitas Therapeutics develop an inhaled form of levodopa (L-dopa) for Parkinson’s disease. Civitas plans to use the Fox Foundation support, along with its $20 million Series A investment from earlier this year, to run a pair of clinical trials over the next year that will seek to prove that an inhalable L-dopa can be a viable alternative to the pill form. The first clinical trial of the drug, CVT-301, is set to begin before year’s end, says Civitas CEO Glenn Batchelder.
Patients with Parkinson’s tend to take L-dopa pill about three times a day, to try to keep a steady amount of the drug in the bloodstream. Take too much, and it won’t do much good, and it can cause side effects. Too little means that the telltale symptoms like tremors and stiffness start to appear. Various companies have tried other ways to deliver steady doses of the drug, through skin patches or infusion-based medicines, with little to show for it. If Civitas, a spinoff from Waltham, MA-based Alkermes (NASDAQ: ALKS), can create an inhalable form, it could end up providing a quick and steady dose for those instances when the pills don’t work. The demand for such a treatment could be big, since about 1 million people in the U.S. suffer from Parkinson’s.
“People have been trying to deliver L-dopa, the gold standard treatment, for 40 years,” says Batchelder. “We believe this is the technology that will really make a difference.” Todd Sherer, the CEO of the Michael J. Fox Foundation, added in a statement that L-dopa delivery challenges “represent a critical unmet need in Parkinson’s disease.”
Civitas has a lot more resources than the typical venture-backed startup might have to pursue this kind of challenge. The company is housed in a Chelsea, MA, facility that represents more than $100 million of investment, which Alkermes built up years ago to make inhalable insulin for diabetics through a partnership with Eli Lilly (NYSE: LLY). Lilly scrapped that program in 2008, which got people thinking about what other inhalable drugs could be made there. By January, the assets were spun out into a new company, Civitas Therapeutics, which secured $20 million in a financing co-led by Canaan Partners and Longitude Capital.
The key challenge that Civitas is facing is with what you could call drug transportation. The existing L-dopa pills are swallowed and make their way into the intestines, where they get absorbed into the bloodstream. Sometimes the amount of drug concentration that actually gets into the blood can be erratic, however, because L-dopa can get blocked in the digestive tract when there are large protein molecules from food that get in the way, Batchelder says.
“Orally, it goes in, and it may get into your blood in 30 or 60 or 90 minutes, or maybe not much will get in at all,” Batchelder says. “Through the lungs, you can get precise dose.”
Civitas will have to prove in clinical trials that it can deliver a consistent amount of the drug into the bloodstream, and that nothing really unexpected happens. Batchelder contends that because L-dopa has been well-studied for many years, it won’t have to go through a lot of extra hoops in clinical trials like novel molecules that are just being tested in people for the first time. The first results from what he calls a “proof of concept” trial will be available before the end of 2012, Batchelder says.
If Civitas can prove that its drug is essentially equal to the existing L-dopa, and that it can be delivered in the kind of blood concentrations that are known to control symptoms, then it will be interesting to see how the product might be positioned in the market. The drug is designed to be made into a powder, delivered via a plastic inhaler, which is “about the size of a Bic lighter,” Batchelder says. That means patients could carry it around with them, and give themselves a dose when they feel like symptoms are about to strike, much like how migraine patients feel an “aura” coming on right before a headache hits.
The inhalable drug, at least in the beginning, isn’t being developed simply as a more-convenient replacement for the oral L-dopa. That would be a tough sell, given that the old drug is cheap and generic, and the new drug would probably have a hard time showing it provides superior symptom relief. Animal studies thus far have suggested that the Civitas drug lasts a similar amount of time in the bloodstream as the oral pill, but that the new formulation gets absorbed into the blood more quickly.
If the Civitas drug can navigate trials and eventually reach the market, it could end up providing a bit of a security blanket for many Parkinson’s patients, Batchelder says. Some patients are afraid of what will happen to them at work, or out in social situations, if their drug suddenly wears off and they can’t control their symptoms. The anxiety that comes from that uncertainty actually makes people more likely to suffer symptoms when they are “off” the drug, Batchelder says. So having the inhalable backup in their purse or briefcase could provide valuable peace of mind, as well as a fast-acting drug when it’s needed, he says.
“We believe we could have a big impact on patients’ lives,” Batchelder says.
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