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
• #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
  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]

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.