Mipomersen

Summary

Mipomersen (CHEMBL2219536) is an approved oligonucleotide (ATC C10AX11); indicated across 1 condition including cardiovascular disorder.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Oligonucleotide
• ATC class: C10AX11
• Indications: 1 condition
• Clinical trials: 9
• Chemistry: 7177 Da · C230H324N67O122P19S19

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2219536
Name	Mipomersen
Type	Oligonucleotide
Max phase	4
FDA approved	no
PubChem CID	44564108
ATC	C10AX11
Molecular formula	C230H324N67O122P19S19
Molecular weight	7177
InChIKey	TZRFSLHOCZEXCC-HIVFKXHNSA-N

SMILES: CC1=CN(C(=O)NC1=O)[C@H]2C[C@@H]([C@H](O2)COP(=O)(O)S[C@H]3C[C@@H](O[C@@H]3COP(=O)(O)S[C@H]4C[C@@H](O[C@@H]4COP(=O)(O)S[C@H]5C[C@@H](O[C@@H]5COP(=O)(O)S[C@H]6C[C@@H](O[C@@H]6COP(=O)(O)S[C@H]7C[C@@H](O[C@@H]7COP(=O)(O)S[C@H]8C[C@@H](O[C@@H]8COP(=O)(O)S[C@H]9C[C@@H](O[C@@H]9COP(=O)(O)S[C@@H]1[C@H](O[C@H]([C@@H]1OCCOC)N1C=C(C(=NC1=O)N)C)COP(=O)(O)S[C@@H]1[C@H](O[C@H]([C@@H]1OCCOC)N1C=C(C(=O)NC1=O)C)COP(=O)(O)S[C@@H]1[C@H](O[C@H]([C@@H]1OCCOC)N1C=C(C(=NC1=O)N)C)COP(=O)(O)S[C@@H]1[C@H](O[C@H]([C@@H]1OCCOC)N1C=C(C(=NC1=O)N)C)COP(=O)(O)S[C@@H]1[C@H](O[C@H]([C@@H]1OCCOC)N1C=NC2=C1N=C(NC2=O)N)CO)N1C=NC2=C(N=CN=C21)N)N1C=NC2=C1N=C(NC2=O)N)N1C=C(C(=O)NC1=O)C)N1C=C(C(=NC1=O)N)C)N1C=C(C(=O)NC1=O)C)N1C=NC2=C1N=C(NC2=O)N)N1C=C(C(=NC1=O)N)C)SP(=O)(O)OC[C@@H]1[C@H](C[C@@H](O1)N1C=C(C(=O)NC1=O)C)SP(=O)(O)OC[C@@H]1[C@H](C[C@@H](O1)N1C=C(C(=NC1=O)N)C)SP(=O)(O)OC[C@@H]1[C@H]([C@H]([C@@H](O1)N1C=NC2=C1N=C(NC2=O)N)OCCOC)SP(=O)(O)OC[C@H]1[C@@H]([C@@H]([C@H](O1)N1C=C(C(=NC1=O)N)C)OCCOC)SP(=O)(O)OC[C@H]1[C@@H]([C@@H]([C@H](O1)N1C=NC2=C(N=CN=C21)N)OCCOC)SP(=O)(O)OC[C@H]1[C@@H]([C@@H]([C@H](O1)N1C=C(C(=NC1=O)N)C)OCCOC)SP(=O)(O)OC[C@H]1[C@@H]([C@@H]([C@H](O1)N1C=C(C(=NC1=O)N)C)OCCOC)O

IUPAC name: [(2R,3R,4S,5R)-5-(4-amino-5-methyl-2-oxopyrimidin-1-yl)-3-[[(2R,3R,4S,5R)-5-(4-amino-5-methyl-2-oxopyrimidin-1-yl)-3-[[(2R,3R,4S,5R)-3-[[(2R,3R,4S,5R)-5-(4-amino-5-methyl-2-oxopyrimidin-1-yl)-3-[[(2R,3S,5R)-3-[[(2R,3S,5R)-3-[[(2R,3S,5R)-3-[[(2R,3S,5R)-5-(4-amino-5-methyl-2-oxopyrimidin-1-yl)-3-[[(2R,3S,5R)-3-[[(2R,3S,5R)-3-[[(2R,3S,5R)-5-(4-amino-5-methyl-2-oxopyrimidin-1-yl)-3-[[(2R,3S,5R)-3-[[(2R,3S,5R)-3-[[(2R,3S,5R)-5-(4-amino-5-methyl-2-oxopyrimidin-1-yl)-3-[[(2R,3R,4S,5R)-3-[[(2S,3S,4R,5S)-5-(4-amino-5-methyl-2-oxopyrimidin-1-yl)-3-[[(2S,3S,4R,5S)-3-[[(2S,3S,4R,5S)-5-(4-amino-5-methyl-2-oxopyrimidin-1-yl)-3-[[(2S,3S,4S,5S)-5-(4-amino-5-methyl-2-oxopyrimidin-1-yl)-3-hydroxy-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyl-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyl-5-(6-aminopurin-9-yl)-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyl-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyl-5-(2-amino-6-oxo-1H-purin-9-yl)-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyloxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyl-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyl-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyloxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyl-5-(2-amino-6-oxo-1H-purin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyl-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyloxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyl-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyl-5-(2-amino-6-oxo-1H-purin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyl-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyl-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyl-4-(2-methoxyethoxy)-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyl-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphoryl]sulfanyl-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-[(2R,3R,4S,5R)-5-(2-amino-6-oxo-1H-purin-9-yl)-2-(hydroxymethyl)-4-(2-methoxyethoxy)oxolan-3-yl]sulfanylphosphinic acid

Also known as: Mipomersen, MIPOMERSEN

Parent form; salt/anhydrous children: CHEMBL502097

Patent coverage: 451 distinct patent families (1,142 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

1 indication (1 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
cardiovascular disorder	4	MONDO:0004995	EFO:0000319

Clinical trials

Total trials: 9.

Phase distribution

Phase	Trials
PHASE3	4
PHASE1	3
PHASE2	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00607373	PHASE3	COMPLETED	Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
NCT00794664	PHASE3	COMPLETED	Safety and Efficacy of Mipomersen in Patients With Severe Hypercholesterolemia on a Maximally Tolerated Lipid-Lowering Regimen and Who Are Not on Apheresis
NCT01475825	PHASE3	COMPLETED	A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol
NCT01598948	PHASE3	COMPLETED	Effect of Mipomersen on LDL-Cholesterol Levels in Patients Treated by Regular Apheresis
NCT00362180	PHASE2	COMPLETED	Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration
NCT00707746	PHASE2	COMPLETED	Safety and Efficacy Study of ISIS 301012 (Mipomersen) Administration in High Risk Statin Intolerant Subjects
NCT01061814	PHASE1	COMPLETED	A Study to Evaluate 3 Different Dosing Regimens of Mipomersen Administered Via Subcutaneous Injections to Healthy Volunteers
NCT01299298	PHASE1	COMPLETED	A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Single Doses of Mipomersen in Japanese Healthy Volunteers
NCT01414881	PHASE1	COMPLETED	Study to Assess the Effects of Mipomersen on Lipid and Lipoprotein Metabolism in Healthy Subjects

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).