Oh Yeju, An Da Eun, Park Jaebeom, Koh Byumseok, Cho Kyung-Jin, Jeon Hongjun
Therapeutics & Biotechnology Division, Korea Research Institute of Chemical Technology, Daejeon 34114 Republic of Korea; Medicinal Chemistry & Pharmacology, University of Science & Technology, Daejeon 34113 Republic of Korea.
Therapeutics & Biotechnology Division, Korea Research Institute of Chemical Technology, Daejeon 34114 Republic of Korea.
Bioorg Med Chem Lett. 2025 May 1;120:130114. doi: 10.1016/j.bmcl.2025.130114. Epub 2025 Jan 27.
KX-01 (tirbanibulin, Klisyri®) is a recently FDA-approved drug for treating actinic keratosis, with a distinct dual mechanism of action combining microtubule disruption and non-ATP-competitive Src inhibition. This unique mechanism and novel chemotype highlight KX-01's potential as a payload for antibody-drug conjugates. In this study, we synthesized and evaluated KX-01 derivatives to enhance anticancer potency and explore functional groups suitable for antibody conjugation. Notably, replacing the morpholine group with an N-benzoylpiperazine scaffold resulted in an analog with significantly improved in vitro antiproliferative activity, attributed to enhanced microtubule disruption and Src inhibition. Furthermore, introducing a phenol or aniline functionality as a common linker attachment point preserved substantial cytotoxicity. These results suggest the potential of KX-01 derivatives for future use as ADC payloads.
KX-01(替拉替布,Klisyri®)是一种最近获美国食品药品监督管理局(FDA)批准用于治疗光化性角化病的药物,具有独特的双重作用机制,将微管破坏与非ATP竞争性Src抑制相结合。这种独特的机制和新型化学类型凸显了KX-01作为抗体药物偶联物有效载荷的潜力。在本研究中,我们合成并评估了KX-01衍生物,以增强抗癌效力并探索适合抗体偶联的官能团。值得注意的是,用N-苯甲酰基哌嗪支架取代吗啉基团产生了一种体外抗增殖活性显著提高的类似物,这归因于微管破坏和Src抑制作用的增强。此外,引入酚或苯胺官能团作为常见的连接点保留了相当大的细胞毒性。这些结果表明KX-01衍生物未来用作ADC有效载荷的潜力。