Details of the Drug

General Information of Drug (ID: DMMQ7BY)

Drug Name
Fitusiran
Synonyms ALN-AT3SC
Indication
Disease Entry ICD 11 Status REF
Hemophilia 3B10.0 Phase 3 [1]
Drug Type
Small interfering RNA
Sequence
>Subunit 1
GUUAACACCAUUUACUUCAAX
>Subunit 2
UUGAAGUAAAUGGUGUUAACCAG
Structure
3D MOL is unavailable 2D MOL
#Ro5 Violations (Lipinski): 5 Molecular Weight (mw) 1711
Logarithm of the Partition Coefficient (xlogp) -5.9
Rotatable Bond Count (rotbonds) 63
Hydrogen Bond Donor Count (hbonddonor) 20
Hydrogen Bond Acceptor Count (hbondacc) 30
Chemical Identifiers
Formula
C78H139N11O30
IUPAC Name
N-[1,3-bis[3-[3-[5-[(2R,3R,4R,5R,6R)-3-acetamido-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxypentanoylamino]propylamino]-3-oxopropoxy]-2-[[3-[3-[5-[(2R,3R,4R,5R,6R)-3-acetamido-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxypentanoylamino]propylamino]-3-oxopropoxy]methyl]propan-2-yl]-12-[(2R,4R)-4-hydroxy-2-methylpyrrolidin-1-yl]-12-oxododecanamide
Canonical SMILES
C[C@@H]1C[C@H](CN1C(=O)CCCCCCCCCCC(=O)NC(COCCC(=O)NCCCNC(=O)CCCCO[C@H]2[C@@H]([C@H]([C@H]([C@H](O2)CO)O)O)NC(=O)C)(COCCC(=O)NCCCNC(=O)CCCCO[C@H]3[C@@H]([C@H]([C@H]([C@H](O3)CO)O)O)NC(=O)C)COCCC(=O)NCCCNC(=O)CCCCO[C@H]4[C@@H]([C@H]([C@H]([C@H](O4)CO)O)O)NC(=O)C)O
InChI
InChI=1S/C78H139N11O30/c1-50-42-54(96)43-89(50)65(104)26-12-10-8-6-5-7-9-11-25-64(103)88-78(47-111-39-27-61(100)82-33-19-30-79-58(97)22-13-16-36-114-75-66(85-51(2)93)72(108)69(105)55(44-90)117-75,48-112-40-28-62(101)83-34-20-31-80-59(98)23-14-17-37-115-76-67(86-52(3)94)73(109)70(106)56(45-91)118-76)49-113-41-29-63(102)84-35-21-32-81-60(99)24-15-18-38-116-77-68(87-53(4)95)74(110)71(107)57(46-92)119-77/h50,54-57,66-77,90-92,96,105-110H,5-49H2,1-4H3,(H,79,97)(H,80,98)(H,81,99)(H,82,100)(H,83,101)(H,84,102)(H,85,93)(H,86,94)(H,87,95)(H,88,103)/t50-,54-,55-,56-,57-,66-,67-,68-,69+,70+,71+,72-,73-,74-,75-,76-,77-/m1/s1
InChIKey
RUPXJRIDSUCQAN-PQNNUJSWSA-N
Cross-matching ID
PubChem CID
119058042
UNII
SV9W47ZLE1
DrugBank ID
DB15002
TTD ID
D46RKC

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
SERPINC1 messenger RNA (SERPINC1 mRNA) TTZ4MD5 ANT3_HUMAN Inhibitor [2]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

References

1 ClinicalTrials.gov (NCT03549871) A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis (ATLAS-PPX). U.S. National Institutes of Health.
2 Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy. N Engl J Med. 2017 Aug 31;377(9):819-828.