Chemistry:Rogocekib
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
- ↑ 1.0 1.1 "Discovery of Rogocekib (CTX-712): A Potent and Selective CLK Inhibitor for Cancer Treatment". ACS Medicinal Chemistry Letters 16 (10): 1870–1875. October 2025. doi:10.1021/acsmedchemlett.5c00412. PMID 41089490.
- ↑ "Ligand page for rogocekib". IUPHAR/BPS Guide to Pharmacology. https://www.guidetopharmacology.org/GRAC/LigandDisplayForward?ligandId=13217.
- ↑ "Rogocekib (CTX-712)". MedChemExpress. https://www.medchemexpress.com/ctx-712.html.
- ↑ "Chordia Therapeutics Secures Orphan Drug Designation for Rogocekib in the U.S.". Markets Insider. January 9, 2025. https://trial.medpath.com/news/cf293b76089cecdb/chordia-therapeutics-secures-orphan-drug-designation-for-rogocekib-in-the-u-s.
- ↑ 5.0 5.1 "Chordia Therapeutics Publishes Phase 1 Results of CLK Inhibitor Rogocekib for R/R AML/HR-MDS in Blood Advances". https://www.chordiatherapeutics.com/en/ir/news/auto_20250925562212/pdfFile.pdf.
- ↑ A Study of CTX-712 in Relapsed/Refractory Acute Myeloid Leukemia and Higher Risk Myelodysplastic Syndromes. 17 September 2025. https://clinicaltrials.gov/study/NCT05732103.
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