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C-101, also called Myodur, is developed for the treatment of Duchenne’s muscular dystrophy (DMD) which is a morbid genetic disease that can lead to death in late adolescence due to... C-101 includes a carnitine carrier molecule and a leupeptin analogue, a known calpain inhibitor.
- Mechanism
- Curator reviewed
C-101 targets muscle cells by using a carnitine carrier molecule and inhibits calpain by attaching a leupeptin analogue onto the carrier. Leupeptin has been studied extensively in a variety of animal models and was shown to be an effective means of slowing or delaying muscle wasting. Carnitine is a compound present in skeletal muscle involved in the transfer of fatty acids across mitochondrial membranes. In C-101, the carnitine carrier molecule directs the leupeptin analogue into targeted cells where it inhibits the up-regulation of calpain. Therefore, by targeting a calpain inhibitor to muscle cells, it is believed that C-101 will prevent the degradation and promote the preservation of functional muscle.
- Primary indication
- Investigated for use/treatment in muscular dystrophy.Curator reviewed
- Formula / weight
- C27H53N7O7 · 587.763 g/mol (avg)
- Code names
- C-101
Resolves to
JOITUBWXBFATOR-HKBOAZHASA-NSMILESCCOC(OCC)[C@H](CCCNC(N)=N)NC(=O)[C@H](CC(C)C)NC(=O)CCC(=O)N[C@H](CC([O-])=O)C[N+](C)(C)CWhat you can answer from here — as of March 06, 2025
Which drugs share a target with L-aminocarnityl-succinyl-leucyl-argininal-diethylacetal, and which of those have an active Phase 3 trial?