At last week’s Annual SMA Conference, Cytokinetics released additional data from their Phase 1 study of CK-2127107, a skeletal muscle activator. Cytokinetics, in partnership with Astellas, has already announced that a Phase 2 study will begin later this year.

Our partnership with Cytokinetics highlights two strengths of Cure SMA’s research model: providing seed funding to show the early promise of a program, and then leveraging partnerships with larger pharmaceutical companies as the programs move into clinical trials.

Providing a Platform for Data Sharing

Cytokinetics is one of several companies to release new data from their program at this year’s SMA Researcher Meeting.

The SMA Researcher Meeting is the largest in the world focused on SMA, and this year alone a record number of more than 300 researchers attended. This combination—a broad reach yet a very particular focus on SMA—makes the SMA Researcher Meeting the ideal setting for pharmaceutical companies to release information on SMA drug programs. Using our conference as vehicle to highlight the progress of programs fulfills several of the primary goals of our conference, including the sharing of timely data and the building of new partnerships and collaborations.

New Phase 1 Data Released

Data from Cytokinetics’ Phase 1 studies also showed significant dose-, concentration-, and frequency-dependent increases in the force of muscle contraction elicited by nerve stimulation in healthy volunteers. The increases in force were most evident in the mid-range of nerve stimulation frequency that reflect those used in many activitities of daily living, consistent with preclinical studies. By directly increasing skeletal muscle function, CK-2127107 may enhance physical performance in patients with neuromuscular diseases including SMA by allowing the nerves that remain to bigger influence on muscle activity. These data support further evaluation of CK-2127107 and its potential to effect measures of muscle performance and function in SMA patients.

Cure SMA’s Long-Term Approach

In addition to providing a platform for Cytokinetics and other companies to continue moving their research through clinical trials, Cure SMA was also involved in their early stages of this program.

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 current stage of progress.