Cytokinetics, in partnership with Astellas, has officially announced that recruitment will begin in the second half of 2015 for a Phase 2 clinical trial testing CK-2127107, a muscle activator. This randomized, double-blind study is expected to enroll 75 patients with SMA type II, III, or IV. Patients will be dosed for four weeks, and will be tested for muscle strength and mobility.

Cytokinetics and Astellas had first announced their plans for a Phase 2 clinical trial in January.

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 initial Phase 2 announcement in January, and for this week’s release of further details.

“Under the supervision of Dr. Fady Malik, we performed preclinical research relating to skeletal muscle activators. That was funded by Cure SMA, who provided grant funding around which we did some very impressive preclinical research published in recent years. That also provided a catalyst for our interest to pursue this in a Phase 2 trial,” said Robert Blum, CEO of Cytokinetics.

During this week’s announcement, Dr. John Day from Stanford University also provided a general update on the current state of SMA research, including the potential of combination therapies for SMA.

Cure SMA has been working with pharmaceutical 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.

Cure SMA’s hope is that these different treatments may be used together, to combat SMA from all sides. Dr. Day likewise expressed hope that, not only will CK-2127107 continue to show positive results in preserving muscle strength, but it will lend itself to combination with other SMA therapies currently in development.

Our thanks to Cytokinetics and Astellas for their hard work in moving this program forward. When the trial recruitment sites and criteria are posted, we will provide a further update.