Phase 2 Trial of ALS Drug Yields Encouraging Results

by Amy Madsen on Tue, 2011-12-13 12:15

Trial participants receiving the highest dose of CK-2017357 showed symptom improvement in the first stage of an ongoing phase 2 trial of this experimental drug

Article Highlights:
  • Results from the first stage of a phase 2 trial to test the experimental drug CK-2017357 in ALS show that the drug was safe and well-tolerated at multiple dose levels, when given on a daily basis.
  • Some trial participants who were treated with the highest dose showed measurable improvement in motor function and muscle strength.
  • Cytokinetics already has moved on to Part B of the phase 2 trial, in which CK-2017357 is being tested in people with ALS who also are taking riluzole. (Participants in Part A did not take riluzole.)

In the first stage of an ongoing phase 2 clinical trial to test the experimental therapy CK-2017357 in amyotrophic lateral sclerosis (ALS), the drug was found to be safe and well-tolerated.

Participants who received the highest dose showed improved scores on tests that measure motor and breathing function, muscle strength and fatigue.

CK-2017357 is designed to work by increasing muscle sensitivity to calcium, which in turn is expected to increase skeletal muscle force and improve muscle function.

The drug is the lead therapeutic candidate for ALS from South San Francisco biotechnology company Cytokinetics, and was granted “orphan drug” status by the U.S. Food and Drug Administration (FDA) in March 2010. (Orphan drug status provides financial incentives for the development of drugs for rare diseases.)

The phase 2 trial results were reported Nov. 30, 2011, at the 22nd International Symposium on ALS/MND in Sydney, Australia, by Jeremy Shefner, professor and chair of the department of neurology at Upstate Medical University, State University of New York in Syracuse. (Shefner directs the MDA/ALS Center at that institution, but MDA is not involved with this study.)

About the new findings

Shefner reported on Part A of the phase 2 trial, in which 24 people with ALS who were not taking riluzole were randomly assigned to one of four groups in which they received daily oral doses of either a placebo or CK-2017357 at a dose of 125, 250 or 375 milligrams.

Clinical assessments took place at scheduled intervals during the two-week treatment phase, and again one week after the final dose.

Investigators found dose-dependent levels of the drug in the blood. Four out of five people who were treated with the highest dose of the drug reported overall improvement. Three out of the five improved by at least one point on the ALS Functional Rating Scale-Revised (ALSFRS-R), a validated ratings scale used by physicians to assess symptom  progression in individuals with ALS.

Although dizziness was a reported side effect in those taking CK-2017357, it tended to be mild, and lessened or stopped with continued treatment over the two-week dosing period. The drug was well-tolerated at all dose levels, and blood levels of the drug increased with the higher doses.

For more, read Cytokinetics’ press release: Cytokinetics Announces Positive Results from Phase 2 Clinical Trial Evaluating CK-2017357 in Patients with Amyotrophic Lateral Sclerosis.

Analysis from the trial still is ongoing, and Cytokinetics expects to report additional results at a later date.

CK-2017357 testing continues

Cytokinetics already has moved on to Part B of the phase 2 trial, in which CK-2017357 is being tested in people with ALS who are taking riluzole (brand name Rilutek). Riluzole is the only drug approved by the FDA for treatment of ALS.

For more see CK-2017357 now being tested in combination with Riluzole. Or, read Cytokinetics' Nov. 1, 2011, press release.

For details on participating in the trial, and for contact information, see A Study to Evaluate the Effects of Multiple Doses of CK-2017357 in Patients With ALS; or enter NCT01378676 into the search box at

Your rating: None Average: 4.9 (7 votes)
MDA cannot respond to questions asked in the comments field. For help with questions, contact your local MDA office or clinic or email See comment policy