Cytokinetics is developing CK-2127107 (2-aminoalkyl-5-N-heteroarylpyrimidine) in collaboration with Astellas. CK-2127107 is a skeletal muscle troponin activator that may improve muscle function and physical performance in people with SMA.
How CK-2127107 Works
The therapy’s developers want CK-2127107 to be the next-generation skeletal muscle compound to slow the rate of calcium release from the regulatory troponin (protein involved in muscle contraction) complex of fast skeletal muscle fibers, sensitizing the sarcomere to calcium, leading to an increase in skeletal muscle contractibility. In early phase trials, CK-2127107 revealed activity that reduces the degree of muscle fatigue.
Clinical Trials For CK-2127107
Five Phase 1 clinical trials in healthy volunteers have been completed, testing safety, tolerability, bioavailability, pharmacokinetics (how the drug moves in the body), and pharmacodynamics (effects and mechanism of the drug). CK-2127107 was well-tolerated at single doses up to 4,000 mg, and its pharmacokinetics profile was linear and dose-proportional across the range of doses studied. Other data also showed significant dose-concentration and frequency-dependent increases in the force of muscle contraction elicited by nerve stimulation in the volunteers.
The positive results led to the ongoing Phase 2 trial for Type 2, 3, or 4 people with SMA. Its primary objective is to demonstrate a pharmacodynamics effect on measures of skeletal muscle function or fatiguability. This trial (NCT02644668) will enroll 72 patients in the U.S. in two sequential, ascending dose cohorts (150 and 450 mg). Each cohort will then be separated into groups, by ability to walk versus inability to walk, to receive CK-2127107 twice a day for eight weeks. The outcome measures include respiratory and motor screenings, and safety and pharmacokinetics profiling. It is estimated that this study will be completed by December 2016.
Next steps for CK-2127107
Since CK-2127107 is still in early trials, there are no plans to file regulatory submissions.
Read the latest news about CK-2127107 at SMA News Today.
Follow the latest developments for all experimental Spinal Muscular Atrophy treatments on the SMA Therapy Tracker.
SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.