Startups Progress with Novel Drugs for Parkinson’s Disease

In the world of Parkinson’s disease drug development, one promising candidate after another has succumbed to what scientists like to call the “Valley of Death”—the period between the discovery of a promising compound and success in human trials. Nevertheless, more than 30 drugs for the disease are in development and a number of startups are beginning to show progress, in some cases by improving on older drugs—and in one early stage program, by employing coffee extract.

At last week’s 6th Annual Parkinson’s Disease Therapeutics Conference in New York, sponsored jointly by The Michael J. Fox Foundation for Parkinson’s Research and The New York Academy of Sciences, those in the packed meeting room heard about treatments in the pipeline that show promise in relieving the debilitating symptoms of Parkinson’s from Civitas Therapeutics of Chelsea, MA, and PsychoGenics of Tarrytown, NY, among others. Also discussed were some very early stage results for drugs that might actually modify the course of the disease. That’s the brass ring in Parkinson’s research, and one that no company, large or small, has yet come close to reaching.

That ring will still likely be out of reach for the foreseeable future, according to Civitas CEO Glenn Batchelder. “Everyone would love to see a disease-modifying drug,” he told me at the meeting, “but that’s still decades away.”

About one million Americans are currently living with Parkinson’s, and some 60,000 people are diagnosed with the disease each year, according to the Michael J. Fox Foundation. Parkinson’s is a degenerative neurological disease resulting from the loss of dopamine-producing brain cells; symptoms include severe tremors, difficulty walking, and sensory and memory dysfunctions. Currently the most effective drug for relief of symptoms is levodopa, first discovered some 50 years ago. But levodopa can cause tremors and jerky movements of its own known as dyskinesia (Michael J. Fox has suffered from these jerky movements for years), along with gastrointestinal side effects. It is also slow to be absorbed into the blood stream.

Civitas, spun out of Waltham, MA-based Alkermes (NASDAQ: ALKS) in 2011, is developing an inhaled version of levodopa called CVT-301, delivered via a nasal spray, that is meant to be faster acting than the oral form of the drug and safer because it doesn’t have to pass through the gut. At the meeting, Civitas Chief Medical Officer Martin Freed announced that the first human trial of CVT-301, completed earlier this year, showed that the drug reached therapeutic levels in the blood within five minutes of inhalation, and was safe and well-tolerated when compared with levodopa. Civitas started a Phase 2 study of the drug this summer, funded in part by the Fox Foundation.

Other companies are also trying to figure out ways to make levodopa safer and more effective. PsychoGenics first developed eltoprazine for adult attention deficit hyperactivity disorder, and continues to test it for that indication, but also believes the drug can relieve levodopa-induced dyskinesia. CEO Emer Leahy told the meeting that in an early-stage clinical trial eltoprazine suppressed levodopa-induced dyskinesia for up to 45 days, was well tolerated, and merits further study.

Drugs that can actually modify the course of Parkinson’s are much further behind, with none in human trials at this point. One pre-clinical candidate garnered a lot of interest at the meeting in part because of its origin. Signum Biosciences  of Monmouth Junction, NJ, is developing a molecule discovered in coffee extract called eicosenoyl-5-hydroxytrypamide, or EHT, that can protect the memory circuits in the brain. Signum chairman and Princeton University chemistry professor Jeffry Stock said the company has created a nutritional supplement enriched with EHT, and that Signum is now trying to discover potential drugs based on the molecule , with the help of a $295,000 grant from the Fox Foundation. EHT’s origin is far, far different than anything that has been tried against Parkinson’s so far, but as speaker after speaker noted at the meeting, scientists must be open to all kinds of novel paths if they are to navigate through the Valley of Death.

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