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

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. This latest trial announcement speaks to two of Cure SMA’s primary goals: pursuing a breadth of treatment options, and ensuring we have treatments for all types, all ages, and all stages of SMA.

An investigator meeting was held last weekend to finalize details of the trial. The first trial will enroll 75 patients across 10-12 centers in the US. Participants will be teens and adults with SMA type II, III, or IV—both ambulatory and non-ambulatory.

The trial will measure respiratory and muscle function in order to see if the drug is safe and effective in individuals with SMA. More specific details on the trial will be announced in the next month.

The clinical trials for CK-2127107 came about because of early seed funding from Cure SMA, supporting research focused on the potential application of these types of skeletal muscle activators to SMA. In 2014, Cytokinetics released encouraging data from preclinical studies conducted with our funding. The data showed this approach had positive effects in preserving muscle strength and reducing muscle fatigue, setting the groundwork for the ongoing clinical trials.

Cure SMA has been working with partners, including Cytokinetics and Astellas, to pursue four different therapeutic approaches: two of these approaches would address the underlying survival motor neuron (SMN) protein deficiency that occurs in SMA, and two would address the muscle and nerve weakness that is caused by the SMN deficiency. The goal here is that CK-2127107 will show positive results in preserving muscle strength in human clinical trials, and may lend itself to combination with other SMA therapies currently in development.

“We have finalized the clinical trial protocol with our partner Astellas and we look forward of initiating this trial later this quarter,” said 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.”