Key SMA Patient Trial of Skeletal Muscle Activator Getting Underway

Cytokinetics has announced additional details about a planned Phase 2 clinical trial of CK-2127107 in patients with spinal muscular atrophy (SMA). Investigators believe CK-2127107, a skeletal muscle activator, has the potential to improve muscle function, either alone or in combination with other drugs.

The company, in collaboration with its partner Astellas, recently held an investigator meeting to finalize the details of the trial. The first trial will enroll 75 patients — both ambulatory and non-ambulatory teens and adults with SMA type II, III or IV — across 10 to 12 centers in the U.S. The trial will measure respiratory and muscle function in the population to test the drug’s safety and efficacy.

Both Cytokinetics and Astellas work with Cure SMA — an organization dedicated to supporting SMA research — to pursue its four therapeutic approaches: two addressing the underlying survival motor neuron (SMN) protein deficiency that occurs in SMA, and two addressing the muscle and nerve weakness caused by the SMN deficiency. Researchers are hopeful that CK-2127107 will show positive results to preserve muscle strength in human clinical trials, and possibly lend itself to combination with SMA therapies currently in development.

Cure SMA contributed early seed funding to the CK-2127107 trial, which speaks to two of its primary goals: pursuing a breadth of treatment options, and ensuring treatments for all types, all ages, and all stages of SMA. “We are excited to see the continued progress of this drug into the next phase of clinical trials, and particularly excited to see this trial focused on teens and adults,” Cure SMA said in a press release.

Added Robert Blum, CEO of Cytokinetics: “Based on our discussions with our lead investigator professor John Day of Stanford University and others in the SMA community, we know there is great anticipation for this trial, particularly because it will be focused on patients 12 years and older, a population that is typically not eligible for other ongoing trials which are more focused on an infant population.” The company plans to initiate the trial later this quarter, he said.

In 2014, Cytokinetics released what Cure SMA called “encouraging data” from preclinical CK-2127107 studies. The data showed the approach had positive effects in preserving muscle strength and reducing muscle fatigue, setting the groundwork for the clinical trials. You can read more about it here.