Cytokinetics Inc.’s experimental treatment for Lou Gehrig’s disease didn’t cause serious side effects in a trial, a positive step for testing the drug’s benefits, the company said.
The therapy, CK-2017357, caused mild dizziness in some of the 24 patients in the second of three trials typically required for U.S. regulatory approval, the San Francisco-based company said today in a statement. Three patients with the disease, amyotrophic lateral sclerosis, saw their symptoms decrease, said Robert Blum, Cytokinetics chief executive officer.
“Even after two weeks, in this study we saw trends toward improvement,” Blum said in an interview.
ALS, a fatal disease of the central nervous system also known as Lou Gehrig’s disease after the New York Yankees baseball player who died from it in 1941, has no cure. Patients suffer a progressive loss of muscle control and typically die within three to five years of respiratory failure. About 5 people in 100,000 worldwide will suffer from the disease, according to the National Institutes of Health.
Cytokinetics’ medicine seeks to increase muscle responsiveness, force and endurance by activating a protein that makes it more sensitive to nerve signals. The company is testing it in patients who aren’t taking riluzole, a generic drug approved by U.S. regulators that has shown the ability to slow the disease’s progression and prolong life in some patients.
The company said its next trial will test the safety of its drug in 24 patients who are on the generic medicine, also known as Rilutek.
Cytokinetics is in late-stage negotiations with potential partners to help develop the treatment further, Blum said. The company would exchange sales rights outside the U.S. for a royalty payment or profit sharing, which would help pay for further studies required by the Food and Drug Administration.
“The partnership is really important to make all this happen, and we’re pretty confident we’re moving toward closing a deal,” Blum said.
Cytokinetics rose 6 percent to $1.02 at the close of trading in New York, its largest single day increase since Oct. 12. The shares have declined 51 percent this year. The results of the trial were released at the International Symposium on ALS/MND in Sydney.