LegoChem Bioscience and ABL Bio said Monday that their global partner CStone Pharmaceuticals has received investigational new drug (IND) approval for CS5001, antibody-drug conjugate (ADC) anticancer drug candidate jointly developed by the two Korean companies.
LegoChem Bio licensed out the development and worldwide sales right (except for Korea) of its ADC anticancer drug candidate developed jointly with ABL Bio to CStone Pharmaceuticals in October 2020.
After receiving the development rights, CStone Pharmaceuticals started developing CS5001, a potential global best-in-class ADC targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1) with multiple differentiated features, including proprietary site-specific conjugation, tumor-selective cleavable linker, and pro-drug technology.
According to CStone Pharmaceuticals, ROR1 is an oncofetal protein with low or no expression in adult tissues but the high expression in various cancers, including various forms of leukemia and non-Hodgkin lymphoma, breast, lung, and ovarian cancers, making it an ideal ADC target.
Results from preclinical studies showed that CS5001 exhibited potent and selective cytotoxicity to a variety of ROR1-expressing cancer cell lines and demonstrated remarkable in vivo antitumor activity in both hematological and solid tumor xenograft models.
“We are glad that the IND application of CS5001 received the SMP letter from the U.S. FDA in 2021,” CStone Pharmaceuticals CSO Archie Tse said. “The preclinical pharmacology data were encouraging and demonstrated CS5001’s therapeutic potential in multiple hematological and solid malignancies.”
Tse stressed that there are only three ROR1 ADCs, including CS5001, in clinical development.
“The upcoming first-in-human phase I study aims to evaluate the safety, tolerability, pharmacokinetics, and preliminary antitumor activity of CS5001 in advanced B cell lymphomas and solid tumors,” Tse said. “We will make every effort to advance this clinical trial of CS5001, and have also submitted the clinical trial notification (CTN) application in Australia and plan to submit the IND application in China soon.”