Details of the Drug

General Information of Drug (ID: DMOYS8A)

• Drug Name: PTG-300
• Synonyms: Rusfertide; Rusfertide [USAN]; UNII-XM71MYX0IQ; XM71MYX0IQ; PTG-300FB; WHO 11456; 1628323-80-7; Isovaleryl-Asp-Thr-His-Phe-Pro-cyclo(Cys-Ile-Lys(palm-gamma-Glu)- Phe-Glu-Pro-Arg-Ser-Lys-Gly-Cys)-Lys-NH2

Indication

Disease Entry	ICD 11	Status	REF
Hemochromatosis	5C64.1Y	Phase 2	[1]
Polycythemia vera	2A20.4	Phase 2	[2]

• Drug Type: Peptide
• #Ro5 Violations (Lipinski): 4:
  Molecular Weight (mw); 2442
  Logarithm of the Partition Coefficient (xlogp); -1.5
  Rotatable Bond Count (rotbonds); 67
  Hydrogen Bond Donor Count (hbonddonor); 29
  Hydrogen Bond Acceptor Count (hbondacc); 34
• Chemical Identifiers:
  Formula: C114H181N27O28S2
  IUPAC Name: (2S)-5-[4-[(3S,6S,9S,12S,15R,20R,26S,29S,32S,35S)-26-(4-aminobutyl)-6-benzyl-12-[(2S)-butan-2-yl]-32-(3-carbamimidamidopropyl)-3-(2-carboxyethyl)-15-[[(2S)-1-[(2S)-2-[[(2S)-2-[[(2S,3R)-2-[[(2S)-3-carboxy-2-(3-methylbutanoylamino)propanoyl]amino]-3-hydroxybutanoyl]amino]-3-(1H-imidazol-4-yl)propanoyl]amino]-3-phenylpropanoyl]pyrrolidine-2-carbonyl]amino]-20-[[(2S)-1,6-diamino-1-oxohexan-2-yl]carbamoyl]-29-(hydroxymethyl)-2,5,8,11,14,22,25,28,31,34-decaoxo-17,18-dithia-1,4,7,10,13,21,24,27,30,33-decazabicyclo[33.3.0]octatriacontan-9-yl]butylamino]-2-(hexadecanoylamino)-5-oxopentanoic acid
  Canonical SMILES: CCCCCCCCCCCCCCCC(=O)N[C@@H](CCC(=O)NCCCC[C@H]1C(=O)N[C@H](C(=O)N[C@H](C(=O)N2CCC[C@H]2C(=O)N[C@H](C(=O)N[C@H](C(=O)N[C@H](C(=O)NCC(=O)N[C@@H](CSSC[C@@H](C(=O)N[C@H](C(=O)N1)[C@@H](C)CC)NC(=O)[C@@H]3CCCN3C(=O)[C@H](CC4=CC=CC=C4)NC(=O)[C@H](CC5=CNC=N5)NC(=O)[C@H]([C@@H](C)O)NC(=O)[C@H](CC(=O)O)NC(=O)CC(C)C)C(=O)N[C@@H](CCCCN)C(=O)N)CCCCN)CO)CCCNC(=N)N)CCC(=O)O)CC6=CC=CC=C6)C(=O)O
  InChI: InChI=1S/C114H181N27O28S2/c1-7-9-10-11-12-13-14-15-16-17-18-19-26-46-90(145)125-79(113(168)169)47-49-89(144)121-53-32-29-42-76-99(154)133-80(58-71-36-22-20-23-37-71)101(156)132-78(48-50-93(148)149)111(166)140-55-34-44-87(140)107(162)130-77(43-33-54-122-114(118)119)100(155)136-84(64-142)104(159)129-75(41-28-31-52-116)98(153)123-63-92(147)127-85(105(160)128-74(97(117)152)40-27-30-51-115)65-170-171-66-86(106(161)138-95(69(5)8-2)109(164)131-76)137-108(163)88-45-35-56-141(88)112(167)83(59-72-38-24-21-25-39-72)135-102(157)81(60-73-62-120-67-124-73)134-110(165)96(70(6)143)139-103(158)82(61-94(150)151)126-91(146)57-68(3)4/h20-25,36-39,62,67-70,74-88,95-96,142-143H,7-19,26-35,40-61,63-66,115-116H2,1-6H3,(H2,117,152)(H,120,124)(H,121,144)(H,123,153)(H,125,145)(H,126,146)(H,127,147)(H,128,160)(H,129,159)(H,130,162)(H,131,164)(H,132,156)(H,133,154)(H,134,165)(H,135,157)(H,136,155)(H,137,163)(H,138,161)(H,139,158)(H,148,149)(H,150,151)(H,168,169)(H4,118,119,122)/t69-,70+,74-,75-,76-,77-,78-,79-,80-,81-,82-,83-,84-,85-,86-,87-,88-,95-,96-/m0/s1
  InChIKey: JRVOBXXOZFTSRF-GVIPULMVSA-N
• Cross-matching ID:
  PubChem CID: 155884410
  CAS Number: 1628323-80-7
  TTD ID: DQ3AM0
• Repurposed Drugs (RPD): Click to Jump to the Detailed RPD Information of This Drug

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Hepcidin (HAMP)	TTPCG5T	HEPC_HUMAN	Replacement	[3]

Molecular Expression Atlas of This Drug

• The Studied Disease: Hemochromatosis
• ICD Disease Classification: 5C64.1Y

Molecule Name	Molecule Type	Gene Name	p-value	Fold-Change	Z-score
Hepcidin (HAMP)	DTT	HAMP	5.37E-01	0.18	0.47

References

• [1] ClinicalTrials.gov (NCT04202965) PTG-300 in Subjects With Hereditary Hemochromatosis. U.S. National Institutes of Health.
• [2] ClinicalTrials.gov (NCT04767802) PTG-300 in Patients With Polycythemia Vera and Elevated Hematocrit. U.S. National Institutes of Health.
• [3] Clinical pipeline report, company report or official report of Protagonist Therapeutics.