Rogocekib

Rogocekib
Clinical data
Other names	CTX-712
Legal status
Legal status	Investigational;
Identifiers
	IUPAC name 2-[(1R)-1-Fluoroethyl]-5-[[6-(4-methoxypyrrolo[2,1-f][1,2,4]triazin-5-yl)-2-methylimidazo[4,5-b]pyridin-1-yl]methyl]-1,3,4-oxadiazole;
CAS Number	2144751-78-8;
PubChem CID	138522180;
DrugBank	DB21739;
UNII	XE88VQP94E;
Chemical and physical data
Formula	C19H17FN8O2
Molar mass	408.397 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES CC1=NC2=C(N1CC3=NN=C(O3)[C@@H](C)F)C=C(C=N2)C4=C5C(=NC=NN5C=C4)OC;
	InChI InChI=1S/C19H17FN8O2/c1-10(20)18-26-25-15(30-18)8-27-11(2)24-17-14(27)6-12(7-21-17)13-4-5-28-16(13)19(29-3)22-9-23-28/h4-7,9-10H,8H2,1-3H3/t10-/m1/s1; Key:OENNTZBJPRRGFL-SNVBAGLBSA-N;

Rogocekib (development code CTX-712) is an investigational oral small-molecule inhibitor of CDC2-like kinases (CLKs), primarily targeting CLK2 with high potency (IC₅₀ of 1.4 nM).^[1]^[2]^[3] It is being developed as a potential treatment for certain cancers, particularly hematologic malignancies.

Research

Rogocekib is under investigation for the treatment of relapsed or refractory acute myeloid leukemia (AML) and higher-risk myelodysplastic syndromes (HR-MDS). It has received Orphan Drug Designation from the U.S. Food and Drug Administration for relapsed/refractory AML.^[4] Rogocekib was discovered through structure-based drug design and lead optimization.^[1]

Mechanism of action

Rogocekib acts as a selective inhibitor of CLKs, enzymes involved in the phosphorylation of serine/arginine-rich proteins that regulate RNA splicing. By inhibiting CLKs, rogocekib disrupts aberrant RNA splicing patterns commonly observed in cancer cells, leading to accumulation of abnormal RNAs and selective induction of apoptosis in malignant cells while sparing normal cells.

Clinical development

Chordia Therapeutics Inc., a Japanese clinical-stage biotechnology company, is developing rogocekib.^[5] Clinical trials include:

A first-in-human Phase I clinical trial in Japan demonstrated safety and pharmacodynamic effects.^[5]
An ongoing multicenter Phase I/II trial in the United States is evaluating rogocekib in patients with relapsed/refractory AML and HR-MDS.^[6]

References

1 2 Kawakita Y, Kojima T, Nii N, Ito Y, Sakauchi N, Banno H, et al. (October 2025). "Discovery of Rogocekib (CTX-712): A Potent and Selective CLK Inhibitor for Cancer Treatment". ACS Medicinal Chemistry Letters. 16 (10): 1870–1875. doi:10.1021/acsmedchemlett.5c00412. PMC 12516396. PMID 41089490.
↑ "Ligand page for rogocekib". IUPHAR/BPS Guide to Pharmacology.
↑ "Rogocekib (CTX-712)". MedChemExpress.
↑ "Chordia Therapeutics Secures Orphan Drug Designation for Rogocekib in the U.S." Markets Insider. January 9, 2025.
1 2 "Chordia Therapeutics Publishes Phase 1 Results of CLK Inhibitor Rogocekib for R/R AML/HR-MDS in Blood Advances" (PDF).
↑ "A Study of CTX-712 in Relapsed/Refractory Acute Myeloid Leukemia and Higher Risk Myelodysplastic Syndromes". clinicaltrials.gov. 17 September 2025.

[Kawakita_2025-1] 1 2 Kawakita Y, Kojima T, Nii N, Ito Y, Sakauchi N, Banno H, et al. (October 2025). "Discovery of Rogocekib (CTX-712): A Potent and Selective CLK Inhibitor for Cancer Treatment". ACS Medicinal Chemistry Letters. 16 (10): 1870–1875. doi:10.1021/acsmedchemlett.5c00412. PMC 12516396. PMID 41089490.

[2] "Ligand page for rogocekib". IUPHAR/BPS Guide to Pharmacology.

[3] "Rogocekib (CTX-712)". MedChemExpress.

[4] "Chordia Therapeutics Secures Orphan Drug Designation for Rogocekib in the U.S." Markets Insider. January 9, 2025.

[Chordia-5] 1 2 "Chordia Therapeutics Publishes Phase 1 Results of CLK Inhibitor Rogocekib for R/R AML/HR-MDS in Blood Advances" (PDF).

[6] "A Study of CTX-712 in Relapsed/Refractory Acute Myeloid Leukemia and Higher Risk Myelodysplastic Syndromes". clinicaltrials.gov. 17 September 2025.

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