Cytokinetics Presents Positive Clinical Data For SMA Drug Candidate

Cytokinetics Presents Positive Clinical Data For SMA Drug Candidate
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During the SMA Annual Conference Cytokinetics presented results from their Phase 1 study of CK-2127107, a novel small molecule activator of the fast skeletal muscle troponin complex to improve skeletal muscle function in diseases and medical conditions associated with neuromuscular dysfunction, muscular weakness, and/or muscle fatigue.

The company, in collaboration with Astellas, recently announced they will begin a Phase 2 study. According with Cure SMA, this collaboration focuses on two particular strengths of Cure SMA’s research model: providing seed funding to show the early promise of a program and collaborating with a pharmaceutical company as the program moves into clinical studies.

Cytokinetics was one of the companies presenting data during this year’s SMA Researcher Meeting. This is the largest meeting in the world with a focus on SMA, with more than 300 researchers from different international centers attending this year. The meeting is the perfect setting for companies to present novel material on their SMA drug programs.

The results from Cytokinetics’ Phase 1 clinical trial showed significant dose-, concentration-, and frequency-dependent increases in the contraction of muscle force, promoted by nerve stimulus in healthy volunteers. The company’s CK-2127107 investigational drug was developed using pre-clinical studies to increase skeletal muscle function and physical performance in SMA patients, by allowing the nerves that remain to bigger influence on muscle activity.

Cure SMA provides a platform for Cytokinetics and other companies to continue the development of their investigations through clinical studies, maintaining its involvement in the early stages of this program and providing early seed funding for the CK-2127107 clinical trials, which has already resulted in promising data from preclinical studies. The data showed this treatment had positive effects in preserving muscle strength and reducing muscle fatigue, setting the foundations for the current studies under development.

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Isis Pharmaceuticals, Inc.  recently revealed its latest data on the Phase 2, open-label and multiple-dose study of ISIS-SMNRx in children diagnosed with spinal muscular atrophy (SMA) who are still receiving treatment in an open-label extension (OLE) study.

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.
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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.
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