Rentosertib

Chemical compound From Wikipedia, the free encyclopedia

Rentosertib (also known as ISM001‑055[1] and INS018_055) is an investigational new drug that is being evaluated for the treatment of idiopathic pulmonary fibrosis (IPF). It targets TNIK (TRAF2 and NCK-interacting protein kinase).[1] It is asserted to be the first drug generated entirely by generative artificial intelligence to reach mid-stage human clinical trials, and the first to target a novel AI-discovered biological pathway.[2][4][5]

Other namesISM001-055;[1] INS018_055[2][3]
Routes of
administrationOral
CAS Number
PubChem CID
Quick facts Clinical data, Other names ...
Rentosertib
INN: Rentosertib
Clinical data
Other namesISM001-055;[1] INS018_055[2][3]
Routes of
administration		Oral
Identifiers
  • [2,5′-Bi-1H-imidazole]-5-carboxamide, 4′-(4-fluorophenyl)-1′-(1-methylethyl)-N-[4-(4-methyl-1-piperazinyl)phenyl]
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
Chemical and physical data
FormulaC27H30FN7O
Molar mass487.583 g·mol−1
3D model (JSmol)
  • CC(C)N1C=NC(=C1C2=NC=C(N2)C(=O)NC3=CC=C(C=C3)N4CCN(CC4)C)C5=CC=C(C=C5)F
  • InChI=1S/C27H30FN7O/c1-18(2)35-17-30-24(19-4-6-20(28)7-5-19)25(35)26-29-16-23(32-26)27(36)31-21-8-10-22(11-9-21)34-14-12-33(3)13-15-34/h4-11,16-18H,12-15H2,1-3H3,(H,29,32)(H,31,36)
  • Key:ZVDNXHUSIKGTSF-UHFFFAOYSA-N
Close

Development

Rentosertib was developed using a generative AI platform designed to identify novel disease-associated targets and rapidly design optimized small-molecule inhibitors.[1][6][7] TNIK was identified as a central regulator of fibrosis and inflammation in IPF. Rentosertib selectively inhibits TNIK, and progressed from target discovery through phase 0 and 1 clinical trials in under 30 months.[1]

Clinical trials

A multicenter, double-blind, placebo-controlled, randomized phase 2a trial was conducted in China, testing the drug in 71 IPF patients from July 2023 to June 2024, encompassing an administration period of 12 weeks. Participants were randomly assigned to receive 30 mg once daily (QD), 30 mg twice daily (BID), 60 mg QD, or placebo.[1]

The trial's primary endpoint was the incidence of treatment-emergent adverse events (TEAEs).[1]

Nomenclature

In March 2025, the United States Adopted Names (USAN) Council approved "Rentosertib" as the official nonproprietary name for ISM001-055.[6]

References

Related Articles

Wikiwand AI