Cytokinetics' upbeat analysis on PhIIa ALS data fails to impress investors

Shares of South San Francisco-based Cytokinetics ($CYTK) slid south this morning--after the developer touted the results for its experimental therapy designed to improve the lives of people suffering from ALS, or Lou Gehrig's disease. In a small but significant Phase IIa study, patients in both dosage groups fared better than the placebo arm of the trial. But the positive data failed to impress investors. The biotech's shares dropped 20 percent on the news.

At the higher, 500 mg dose, 29 of 65 patients rated their muscle strength and fatigue as "better" just six hours after treatment, compared to 18 of 63 patients in the placebo arm. And 15 of 63 patients said they felt better after the 250 mg dose, compared to eight patients taking a placebo. The drug--CK-2017357-is designed to spur more forceful muscle contractions, boosting patients' sense of strength. And another batch of mid-stage data is expected in the next few months.

"This is a disease for which there is absolutely no effective therapy," lead investigator Jeremy Shefner tells Reuters. "To show that you had nearly 45 percent of patients feeling better on a drug after a single dose is quite an amazing finding."

"We are now planning to proceed into a multiple-dose clinical trial to understand how longer-term dosing of CK-2017357 may impact the functional status of these patients," said Andrew A. Wolff, MD, FACC, Cytokinetics' senior VP of clinical research and development.

- check out the Cytokinetics release
- here's the Reuters report