P3, N=453, Active, not recruiting, Deciphera Pharmaceuticals, LLC | Trial completion date: Dec 2025 --> Dec 2026

P1/2, N=25, Not yet recruiting, Thomas Jefferson University

P2, N=75, Completed, Institute of Hematology, Chinese Academy of Medical Sciences / West China Hospital of Sichuan University; Chengdu Zenitar Biomedical Technology Co., L

P2, N=60, Not yet recruiting, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences (IHCAMS);West China Hospital of Sichuan University;Shengjin

P1, N=24, Completed, Chengdu Xinhua Hospital; Chengdu Zenitar Biomedical Technology Co.Ltd.

Furthermore, there is an absence of severe toxicities that have arisen with other agents in the TGCT treatment space, specifically liver failure. Vimseltinib is a favorable option for patients with TGCTs and further efforts to determine its place in the sequence of overall management are needed.

The results of in vitro-in vivo extrapolation (IVIVE) indicated that coadministration of pexidartinib at a clinically approved dose (400 mg twice daily) with the drugs primarily cleared by UGT1A1, UGT1A6, UGT1A7, UGT1A9, and UGT2B15 would result in a higher risk of DDI. In summary, our results provide useful information for the mechanism underlying pexidartinib-induced hepatotoxicity and clinical safe medication of pexidartinib.

P1, N=6, Completed, Sunshine Lake Pharma Co., Ltd. | Not yet recruiting --> Completed

Our investigation reveals a transcriptionally defined malignant population under IRF9 control that orchestrates immunosuppressive microenvironmental reprogramming via EFNA1-mediated signaling networks. The EFNA1-Linifanib combination may represent a potential therapeutic approach to mitigate anti-angiogenic resistance and restrain metastatic progression in colorectal carcinoma.

The representative compound 6s demonstrates superior dual-targeting properties, exhibiting 150-fold higher FLT3 inhibition (half-maximal inhibitory concentration (IC50) = 14 nM) compared with the reference drug tandutinib (IC50 = 2098 nM) and 2.9-fold higher HDAC1 inhibition (IC50 = 27 nM) relative to vorinostat (SAHA; IC50 = 79 nM). Importantly, in the Jeko-1 xenograft model, 6s achieves 53.34 % tumor growth inhibition at a dose of 30 mg/kg with no observable toxicity. Collectively, these results indicate that 6s is a potent dual FLT3/HDAC inhibitor with promising therapeutic potential for hematologic malignancies.

Overall, dapolsertib demonstrated modest single-agent activity in patients with AML. This trial was registered at www.clinicaltrials.gov as #NCT03008187.

P1, N=80, Not yet recruiting, Sunshine Lake Pharma Co., Ltd.