Repositioning Candidate Details
| Candidate ID: | R0828 |
| Source ID: | DB05528 |
| Source Type: | approved; investigational |
| Compound Type: | small molecule |
| Compound Name: | Mipomersen |
| Synonyms: | Mipomersen |
| Molecular Formula: | C230H324N67O122P19S19 |
| SMILES: | COCCO[C@H]1[C@@H](O)[C@H](COP(O)(=O)S[C@H]2[C@H](COP(O)(=O)S[C@H]3[C@H](COP(O)(=O)S[C@H]4[C@H](COP(O)(=O)S[C@@H]5[C@@H](COP(O)(=O)S[C@H]6C[C@@H](O[C@@H]6COP(O)(=O)S[C@H]6C[C@@H](O[C@@H]6COP(O)(=O)S[C@H]6C[C@@H](O[C@@H]6COP(O)(=O)S[C@H]6C[C@@H](O[C@@H]6COP(O)(=O)S[C@H]6C[C@@H](O[C@@H]6COP(O)(=O)S[C@H]6C[C@@H](O[C@@H]6COP(O)(=O)S[C@H]6C[C@@H](O[C@@H]6COP(O)(=O)S[C@H]6C[C@@H](O[C@@H]6COP(O)(=O)S[C@H]6C[C@@H](O[C@@H]6COP(O)(=O)S[C@H]6C[C@@H](O[C@@H]6COP(O)(=O)S[C@@H]6[C@@H](COP(O)(=O)S[C@@H]7[C@@H](COP(O)(=O)S[C@@H]8[C@@H](COP(O)(=O)S[C@@H]9[C@@H](COP(O)(=O)S[C@@H]%10[C@@H](CO)O[C@H]([C@@H]%10OCCOC)N%10C=NC%11=C%10N=C(N)NC%11=O)O[C@H]([C@@H]9OCCOC)N9C=C(C)C(N)=NC9=O)O[C@H]([C@@H]8OCCOC)N8C=C(C)C(N)=NC8=O)O[C@H]([C@@H]7OCCOC)N7C=C(C)C(=O)NC7=O)O[C@H]([C@@H]6OCCOC)N6C=C(C)C(N)=NC6=O)N6C=NC7=C6N=CN=C7N)N6C=NC7=C6N=C(N)NC7=O)N6C=C(C)C(=O)NC6=O)N6C=C(C)C(N)=NC6=O)N6C=C(C)C(=O)NC6=O)N6C=NC7=C6N=C(N)NC7=O)N6C=C(C)C(N)=NC6=O)N6C=C(C)C(=O)NC6=O)N6C=C(C)C(=O)NC6=O)N6C=C(C)C(N)=NC6=O)O[C@H]([C@@H]5OCCOC)N5C=NC6=C5N=C(N)NC6=O)O[C@@H]([C@H]4OCCOC)N4C=C(C)C(N)=NC4=O)O[C@@H]([C@H]3OCCOC)N3C=NC4=C3N=CN=C4N)O[C@@H]([C@H]2OCCOC)N2C=C(C)C(N)=NC2=O)O[C@@H]1N1C=C(C)C(N)=NC1=O |
| Structure: |
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| DrugBank Description: | Mipomersen sodium, which was known as the investigational drug, isis-301012, is the salt form of a synthetic phosphorothioate oligonucleotide. Mipomersen sodium prevents the formation of apo B-100, resulting in a decrease in the levels of apolipoprotein B (apo B), low density lipoprotein (LDL), and total cholesterol. Mipomersen is indicated in patients with homozygous familial hypercholesterolemia as an adjunct to diet and other lipid-lowering medications. It is marketed under the brand name Kynamro in the United States, and the FDA label includes a black box warning of hepatoxicity. Specifically, elevations in the liver enzymes, i.e. transaminases, and in liver fat (hepatic steatosis) have been reported. Due to this serious risk of liver toxicity, mipomersen sodium is only available to patients under the restricted program called Kynamro Risk Evaluation and Mitigation Strategy program. |
| CAS Number: | 1000120-98-8 |
| Molecular Weight: | 7177.11 |
| DrugBank Indication: | Used in patients with homozygous familial hypercholesterolemia as an adjunct to diet and other lipid-lowering medications. |
| DrugBank Pharmacology: | Mipomersen sodium decreases the levels of apolipoprotein B (apo B), low density lipoprotein (LDL) non-high density lipoprotein-cholesterol, and total cholesterol. |
| DrugBank MoA: | Mipomersen binds to the mRNA that codes for apoB-100. This binding leads to double-stranded RNA, which is degraded by RNase H and prevents translation of the mRNA to form the apo B-100 protein. |
| Targets: | mRNA of ApoB-100 binder |
| Inclusion Criteria: | Indication associated |
| Strategy ID | Strategy | Synonyms | Related Targets | Related Drugs |
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| Target ID | Target Name | GENE | Action | Class | UniProtKB ID | Entry Name |
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