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New Lou Gehrig’s Disease Hope

Wednesday, November 30, 2011 1:27 PM

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Cytokinetics' experimental treatment for amyotrophic lateral sclerosis didn’t cause serious side effects in a trial, a positive step for testing the drug’s benefits, the company said.

The ALS 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 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.

Cytokinetics is in late-stage negotiations with potential partners to help develop the drug further, Blum said. The company would exchange sales rights outside the United States 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.

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.

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