There has never been much innovation to brag about in the treatment of the neurodegenerative disease that killed Yankees legend Lou Gehrig. But South San Francisco-based Cytokinetics (NASDAQ: [[ticker:CYTK]]) is reporting today on clinical trial results that offer a hint it could be onto something for treating amyotrophic lateral sclerosis (ALS).
An experimental drug from Cytokinetics met its main goal of demonstrating safety in 49 patients, while showing signs after two weeks of beating a placebo on a standard test of disease progression, according to results presented today at the American Academy of Neurology meeting in New Orleans. The result was from a small study which wasn’t designed to show a clinical benefit, and the findings were not statistically significant, meaning it could have been a fluke. But the finding is provocative enough that Cytokinetics has been emboldened to take the next step, and invest significant time and money to advance to the final phase of clinical trials normally required for FDA approval of a new drug.
“The idea you can see a change after two weeks on a functional rating scale is encouraging,” says Jeremy Shefner, the chair of the Department of Neurology at the Upstate Medical University at the State University of New York, and the study’s principal investigator. “It’s something we’ve never seen in ALS before.”
Cytokinetics has been on a long and hard journey since its founding in 1997, attempting to show there is a clinical benefit to its scientific understanding of muscle cell contraction. There are lots of potential applications of drugs that can improve muscle function, but Cytokinetics has good reason to focus its energy on ALS. This neurodegenerative disease, which robs people of their ability to walk and speak, eventually kills people when motor neurons break down so much that people can’t breathe. The only approved drug for this condition, Sanofi’s riluzole (Rilutek), only delays the time it takes for people to end up on a ventilator. About 30,000 people in the U.S. are thought to have the disease, and life expectancy is about two to five years, according to the ALS Association.
The study which Cytokinetics sponsored was designed to test the safety of its twice-daily oral pill, CK-357. The basic hope is that the Cytokinetics drug will activate a complex in muscle cells that makes them more sensitive to calcium, which should help the skeletal muscle function with more force and stamina. While the drug doesn’t attempt to solve the underlying cause of disease—the death of motor neurons—it is believed that it could help patients by improving the way their functioning muscles work, Shefner says.
Cytokinetics presented some preliminary results last November from 24 patients who got its drug in a mid-stage clinical trial. In the first part of the study, patients were randomly assigned to get either a placebo or a low, medium, or high dose of the Cytokinetics compound for two weeks. The second stage of the study was the same, except patients got a placebo or the combination of the Cytokinetics drug and the standard treatment from Sanofi.
Dizziness was the most common adverse event reported in patients, and it was mostly mild and went away shortly after treatment started, researchers said. No patients dropped out of the study early because of adverse events.
What’s more interesting, and what provides the basis for further testing, is what researchers saw
