This drug entry is a stub and has not been fully annotated. It is scheduled to be annotated soon.

Identification

Generic Name
L-aminocarnityl-succinyl-leucyl-argininal-diethylacetal
DrugBank Accession Number
DB06124
Background

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 accelerated skeletal muscle breakdown. C-101 includes a carnitine carrier molecule and a leupeptin analogue, a known calpain inhibitor. Calpain is the primary protease that degrades skeletal muscle.

Type
Small Molecule
Groups
Investigational
Structure
Weight
Average: 587.763
Monoisotopic: 587.400647075
Chemical Formula
C27H53N7O7
Synonyms
Not Available
External IDs
  • C 101
  • C-101
  • C101

Pharmacology

Indication

Investigated for use/treatment in muscular dystrophy.

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Contraindications & Blackbox Warnings
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Pharmacodynamics

Not Available

Mechanism of action

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.

TargetActionsOrganism
UCalpain-3Not AvailableHumans
Absorption

Not Available

Volume of distribution

Not Available

Protein binding

Not Available

Metabolism
Not Available
Route of elimination

Not Available

Half-life

Not Available

Clearance

Not Available

Adverse Effects
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Toxicity

Not Available

Pathways
Not Available
Pharmacogenomic Effects/ADRs
Not Available

Interactions

Drug Interactions
This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
Not Available
Food Interactions
Not Available

Products

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International/Other Brands
Myodur

Categories

Drug Categories
Not Available
Classification
Not classified
Affected organisms
Not Available

Chemical Identifiers

UNII
BK727F83NO
CAS number
1006685-44-4
InChI Key
JOITUBWXBFATOR-HKBOAZHASA-N
InChI
InChI=1S/C27H53N7O7/c1-8-40-26(41-9-2)20(11-10-14-30-27(28)29)33-25(39)21(15-18(3)4)32-23(36)13-12-22(35)31-19(16-24(37)38)17-34(5,6)7/h18-21,26H,8-17H2,1-7H3,(H7-,28,29,30,31,32,33,35,36,37,38,39)/t19-,20+,21+/m1/s1
IUPAC Name
(3R)-3-(3-{[(1S)-1-{[(2S)-5-carbamimidamido-1,1-diethoxypentan-2-yl]carbamoyl}-3-methylbutyl]carbamoyl}propanamido)-4-(trimethylazaniumyl)butanoate
SMILES
CCOC(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)C

References

General References
Not Available
ChemSpider
32699207

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount

Pharmacoeconomics

Manufacturers
Not Available
Packagers
Not Available
Dosage Forms
Not Available
Prices
Not Available
Patents
Not Available

Properties

State
Solid
Experimental Properties
Not Available
Predicted Properties
PropertyValueSource
Water Solubility0.0124 mg/mLALOGPS
logP-0.97ALOGPS
logP-6.4Chemaxon
logS-4.7ALOGPS
pKa (Strongest Acidic)3.71Chemaxon
pKa (Strongest Basic)11.85Chemaxon
Physiological Charge1Chemaxon
Hydrogen Acceptor Count10Chemaxon
Hydrogen Donor Count6Chemaxon
Polar Surface Area207.79 Å2Chemaxon
Rotatable Bond Count22Chemaxon
Refractivity187.34 m3·mol-1Chemaxon
Polarizability65.49 Å3Chemaxon
Number of Rings0Chemaxon
Bioavailability0Chemaxon
Rule of FiveNoChemaxon
Ghose FilterNoChemaxon
Veber's RuleNoChemaxon
MDDR-like RuleNoChemaxon
Predicted ADMET Features
Not Available

Spectra

Mass Spec (NIST)
Not Available
Spectra
Not Available

Targets

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Kind
Protein
Organism
Humans
Pharmacological action
Unknown
General Function
Titin binding
Specific Function
Calcium-regulated non-lysosomal thiol-protease.
Gene Name
CAPN3
Uniprot ID
P20807
Uniprot Name
Calpain-3
Molecular Weight
94252.95 Da

Drug created at November 18, 2007 18:30 / Updated at June 12, 2020 16:52