Cytokinetics and Astellas Announce New Phase 2 Clinical Trial

Cytokinetics and Astellas jointly announced plans to begin a Phase 2 clinical trial in 2015 in patients with spinal muscular atrophy. The trial will test CK-2127107, a muscle activator. CK-2127107 will be the seventh SMA drug program to advance to clinical trials.

Cure SMA provided funding to Cytokinetics for research focused on the potential application of these types of skeletal muscle activators to SMA. Earlier this year, 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.

“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.

“I recently met with members of the senior management team at Cytokinetics. We discussed the preclinical research relating to skeletal muscle activators which had previously been funded by Cure SMA and the potential for this research to inform the development of new medicines for patients with SMA,” said Jill Jarecki, PhD, Cure SMA Research Director. “I was especially impressed by Cytokinetics’ passion and commitment to pursue improved treatment options for patients with SMA. We are very pleased that Cytokinetics and Astellas have now announced that they will initiate a Phase 2 clinical trial of CK-2127107 in patients with SMA in 2015 and look forward to results from that trial which we hope may extend the translational findings of the initial work funded by our organization.”

In non-clinical models of spinal muscular atrophy, a fast skeletal muscle troponin activator has demonstrated increases in submaximal skeletal muscle force in response to neuronal input and delays in the onset and reductions in the degree of muscle fatigue. CK-2127107 has been the subject of five completed Phase 1 clinical trials in healthy volunteers, which evaluated safety, tolerability, bioavailability, pharmacokinetics and pharmacodynamics.

Today’s announcement highlights several strengths of our research funding model:

  • We provided early seed funding to provide supportive evidence that this approach could potentially be useful in SMA. Now, two major pharmaceutical companies—Cytokinetics and Astellas—are planning to embark on a Phase 2 clinical trial in SMA.
  • We invested in a variety of different approaches, attacking SMA from all sides. Other programs currently in clinical trials, such as gene therapy and ISIS-SMNRx, address the genetics of SMA. CK-2127107 may address the muscle weakness and fatigue that are caused by SMA. Ultimately, these complementary approaches could be used together.

Please visit our research section for more information on our research model.

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