drisapersen   Click here for help

GtoPdb Ligand ID: 13535

Synonyms: GSK2402968 | Kyndrisa® | PRO051
Compound class: Nucleic acid
Comment: Drisapersen (PRO051; GSK2402968) is a 2'-O-methyl phosphorothioate antisense oligonucleotide. It was designed an an exon 51-skipping oligonucleotides to treat Duchenne muscular dystrophy by restoring the production of partially functional dystrophin protein [2].
2D Structure
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2D Structure

An image of the ligand's 2D structure. For small molecules with SMILES these are drawn using the NCI/CADD Chemical Identifier Resolver. Click on the image to access the chemical structure search tool with the ligand pre-loaded in the structure editor. For other types of ligands, e.g. longer nucleotides and peptides, a manually drawn representation of the molecule may be provided.

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SMILES / InChI / InChIKey
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SMILES / InChI / InChIKey

SMILES (Simplified Molecular Input Line Entry Specification) A specification for unambiguously describing the structure of chemical molecules using short ASCII strings. Canonical SMILES specify a unique representation of the 2D structure without chiral or isotopic specifications. Isomeric SMILES include chiral specification and isotopes.

Standard InChI (IUPAC International Chemical Identifier) and InChIKey InChI is a non-proprietary, standard, textual identifier for chemical substances designed to facilitate linking of information and database searching. An InChIKey is a simplified version of a full InChI, designed for easier web searching.

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)

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Canonical SMILES CO[C@@H]1[C@@H]([C@@H](COP(=S)(O)O[C@@H]2[C@@H](COP(=S)(O)O[C@@H]3[C@@H](COP(=S)(O)O[C@@H]4[C@@H](COP(=S)(O)O[C@@H]5[C@@H](COP(=S)(O)O[C@@H]6[C@@H](COP(=S)(O)O[C@@H]7[C@@H](COP(=S)(O)O[C@@H]8[C@@H](COP(=S)(O)O[C@@H]9[C@@H](COP(=S)(O)O[C@@H]%10[C@@H](COP(=S)(O)O[C@@H]%11[C@@H](COP(=S)(O)O[C@@H]%12[C@@H](COP(=S)(O)O[C@@H]%13[C@@H](COP(=S)(O)O[C@@H]%14[C@@H](COP(=S)(O)O[C@@H]%15[C@@H](COP(=S)(O)O[C@@H]%16[C@@H](COP(=S)(O)O[C@@H]%17[C@@H](COP(=S)(O)O[C@@H]%18[C@@H](COP(=S)(O)O[C@@H]%19[C@@H](COP(=S)(O)O[C@@H]%20[C@@H](CO)O[C@H]([C@@H]%20OC)N%21C=CC(=O)NC%21=O)O[C@H]([C@@H]%19OC)N%22C=CC(=NC%22=O)N)O[C@H]([C@@H]%18OC)N%23C=NC%24=C%23N=CN=C%24N)O[C@H]([C@@H]%17OC)N%25C=NC%26=C%25N=CN=C%26N)O[C@H]([C@@H]%16OC)N%27C=NC%28=C%27N=C(N)NC%28=O)O[C@H]([C@@H]%15OC)N%29C=NC%30=C%29N=C(N)NC%30=O)O[C@H]([C@@H]%14OC)N%31C=NC%32=C%31N=CN=C%32N)O[C@H]([C@@H]%13OC)N%33C=NC%34=C%33N=CN=C%34N)O[C@H]([C@@H]%12OC)N%35C=NC%36=C%35N=C(N)NC%36=O)O[C@H]([C@@H]%11OC)N%37C=NC%38=C%37N=CN=C%38N)O[C@H]([C@@H]%10OC)N%39C=CC(=O)NC%39=O)O[C@H]([C@@H]9OC)N%40C=NC%41=C%40N=C(N)NC%41=O)O[C@H]([C@@H]8OC)N%42C=NC%43=C%42N=C(N)NC%43=O)O[C@H]([C@@H]7OC)N%44C=CC(=NC%44=O)N)O[C@H]([C@@H]6OC)N%45C=NC%46=C%45N=CN=C%46N)O[C@H]([C@@H]5OC)N%47C=CC(=O)NC%47=O)O[C@H]([C@@H]4OC)N%48C=CC(=O)NC%48=O)O[C@H]([C@@H]3OC)N%49C=CC(=O)NC%49=O)O[C@H]([C@@H]2OC)N%50C=CC(=NC%50=O)N)O[C@H]1N%51C=CC(=O)NC%51=O)O
Isomeric SMILES CO[C@@H]1[C@@H]([C@H](O[C@H]1N2C=CC(=O)NC2=O)COP(=S)(O)O[C@@H]3[C@H](O[C@H]([C@@H]3OC)N4C=CC(=NC4=O)N)COP(=S)(O)O[C@@H]5[C@H](O[C@H]([C@@H]5OC)N6C=CC(=O)NC6=O)COP(=S)(O)O[C@@H]7[C@H](O[C@H]([C@@H]7OC)N8C=CC(=O)NC8=O)COP(=S)(O)O[C@@H]9[C@H](O[C@H]([C@@H]9OC)N%10C=CC(=O)NC%10=O)COP(=S)(O)O[C@@H]%11[C@H](O[C@H]([C@@H]%11OC)N%12C=NC%13=C(N=CN=C%13%12)N)COP(=S)(O)O[C@@H]%14[C@H](O[C@H]([C@@H]%14OC)N%15C=CC(=NC%15=O)N)COP(=S)(O)O[C@@H]%16[C@H](O[C@H]([C@@H]%16OC)N%17C=NC%18=C%17N=C(NC%18=O)N)COP(=S)(O)O[C@@H]%19[C@H](O[C@H]([C@@H]%19OC)N%20C=NC%21=C%20N=C(NC%21=O)N)COP(=S)(O)O[C@@H]%22[C@H](O[C@H]([C@@H]%22OC)N%23C=CC(=O)NC%23=O)COP(=S)(O)O[C@@H]%24[C@H](O[C@H]([C@@H]%24OC)N%25C=NC%26=C(N=CN=C%26%25)N)COP(=S)(O)O[C@@H]%27[C@H](O[C@H]([C@@H]%27OC)N%28C=NC%29=C%28N=C(NC%29=O)N)COP(=S)(O)O[C@@H]%30[C@H](O[C@H]([C@@H]%30OC)N%31C=NC%32=C(N=CN=C%32%31)N)COP(=S)(O)O[C@@H]%33[C@H](O[C@H]([C@@H]%33OC)N%34C=NC%35=C(N=CN=C%35%34)N)COP(=S)(O)O[C@@H]%36[C@H](O[C@H]([C@@H]%36OC)N%37C=NC%38=C%37N=C(NC%38=O)N)COP(=S)(O)O[C@@H]%39[C@H](O[C@H]([C@@H]%39OC)N%40C=NC%41=C%40N=C(NC%41=O)N)COP(=S)(O)O[C@@H]%42[C@H](O[C@H]([C@@H]%42OC)N%43C=NC%44=C(N=CN=C%44%43)N)COP(=S)(O)O[C@@H]%45[C@H](O[C@H]([C@@H]%45OC)N%46C=NC%47=C(N=CN=C%47%46)N)COP(=S)(O)O[C@@H]%48[C@H](O[C@H]([C@@H]%48OC)N%49C=CC(=NC%49=O)N)COP(=S)(O)O[C@@H]%50[C@H](O[C@H]([C@@H]%50OC)N%51C=CC(=O)NC%51=O)CO)O
InChI InChI=1S/C211H275N76O119P19S19/c1-329-136-116(295)77(369-178(136)271-33-24-99(289)252-206(271)304)40-349-407(310,426)389-118-79(371-180(138(118)331-3)268-30-21-96(212)249-203(268)301)42-351-409(312,428)392-121-81(373-183(141(121)334-6)273-35-26-101(291)254-208(273)306)44-352-410(313,429)393-122-82(374-184(142(122)335-7)274-36-27-102(292)255-209(274)307)45-353-411(314,430)394-123-83(375-185(143(123)336-8)275-37-28-103(293)256-210(275)308)46-355-415(318,434)396-125-85(377-187(145(125)338-10)277-65-238-105-156(215)226-59-232-162(105)277)48-358-413(316,432)391-120-80(372-182(140(120)333-5)270-32-23-98(214)251-205(270)303)43-354-417(320,436)402-131-94(386-193(151(131)344-16)283-71-244-111-168(283)258-198(221)263-173(111)296)57-366-424(327,443)405-132-91(383-194(152(132)345-17)284-72-245-112-169(284)259-199(222)264-174(112)297)54-363-414(317,433)395-124-84(376-186(144(124)337-9)276-38-29-104(294)257-211(276)309)47-356-416(319,435)397-126-89(381-188(146(126)339-11)278-66-239-106-157(216)227-60-233-163(106)278)52-361-422(325,441)403-133-92(384-195(153(133)346-18)285-73-246-113-170(285)260-200(223)265-175(113)298)55-364-420(323,439)400-130-88(380-192(150(130)343-15)282-70-243-110-161(220)231-64-237-167(110)282)51-360-419(322,438)399-128-90(382-190(148(128)341-13)280-68-241-108-159(218)229-62-235-165(108)280)53-362-423(326,442)404-134-95(387-196(154(134)347-19)286-74-247-114-171(286)261-201(224)266-176(114)299)58-367-425(328,444)406-135-93(385-197(155(135)348-20)287-75-248-115-172(287)262-202(225)267-177(115)300)56-365-421(324,440)401-129-87(379-191(149(129)342-14)281-69-242-109-160(219)230-63-236-166(109)281)50-359-418(321,437)398-127-86(378-189(147(127)340-12)279-67-240-107-158(217)228-61-234-164(107)279)49-357-412(315,431)390-119-78(370-181(139(119)332-4)269-31-22-97(213)250-204(269)302)41-350-408(311,427)388-117-76(39-288)368-179(137(117)330-2)272-34-25-100(290)253-207(272)305/h21-38,59-95,116-155,178-197,288,295H,39-58H2,1-20H3,(H,310,426)(H,311,427)(H,312,428)(H,313,429)(H,314,430)(H,315,431)(H,316,432)(H,317,433)(H,318,434)(H,319,435)(H,320,436)(H,321,437)(H,322,438)(H,323,439)(H,324,440)(H,325,441)(H,326,442)(H,327,443)(H,328,444)(H2,212,249,301)(H2,213,250,302)(H2,214,251,303)(H2,215,226,232)(H2,216,227,233)(H2,217,228,234)(H2,218,229,235)(H2,219,230,236)(H2,220,231,237)(H,252,289,304)(H,253,290,305)(H,254,291,306)(H,255,292,307)(H,256,293,308)(H,257,294,309)(H3,221,258,263,296)(H3,222,259,264,297)(H3,223,260,265,298)(H3,224,261,266,299)(H3,225,262,267,300)/t76-,77-,78-,79-,80-,81-,82-,83-,84-,85-,86-,87-,88-,89-,90-,91-,92-,93-,94-,95-,116-,117-,118-,119-,120-,121-,122-,123-,124-,125-,126-,127-,128-,129-,130-,131-,132-,133-,134-,135-,136-,137-,138-,139-,140-,141-,142-,143-,144-,145-,146-,147-,148-,149-,150-,151-,152-,153-,154-,155-,178-,179-,180-,181-,182-,183-,184-,185-,186-,187-,188-,189-,190-,191-,192-,193-,194-,195-,196-,197-,407?,408?,409?,410?,411?,412?,413?,414?,415?,416?,417?,418?,419?,420?,421?,422?,423?,424?,425?/m1/s1
InChI Key HUGILZFVSVLCAO-XVKRXUDYSA-N

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)
No information available.
Summary of Clinical Use Click here for help
BioMarin Pharmaceutica/GlaxoSmithKline progressed drisapersen for DMD to phase 3 trial, but it failed to meet its primary endpoint, and evidence of toxicity (renal effects and thrombocytopenia [1]) was detected. This program of exon skipping oligonucleotides for DMD was subsequently terminated.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT01480245 Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy Phase 3 Interventional GlaxoSmithKline
NCT01254019 A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy Phase 3 Interventional GlaxoSmithKline
NCT01910649 A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration Phase 2 Interventional BioMarin Pharmaceutical 1