Kringle Pharma, Inc. announced the completion of patient enrollment in its ongoing Phase III clinical study of KP-100IT, the intrathecal formulation of recombinant human HGF, in patients with acute spinal cord injury. The study enrolled 25 patients at 5 clinical sites. Following the six-month observation period of the last patient, KRINGLE will conduct the efficacy and safety analysis with all subjects and then report top-line results from the study.

HGF was originally discovered as an endogenous mitogen for mature hepatocytes. Subsequent studies demonstrated that HGF exerts multiple biological functions based on its mitogenic, motogenic, anti-apoptotic, morphogenic, anti-fibrotic, and angiogenic activities, and facilitates regeneration and protection of a wide variety of organs. HGF exerts neurotrophic effects and enhances neurite outgrowth, and the therapeutic effect of HGF on spinal cord injury has been demonstrated in animal models by Professors Hideyuki Okano and Masaya Nakamura at Keio University School of Medicine.

Expectations for HGF as a novel therapeutic agent are increasing for the treatment of spinal cord injury. In parallel with the ongoing Phase III study, KRINGLE launched a collaborative research program with Professors Hideyuki Okano and Masaya Nakamura at Keio University School of Medicine in 2021, aiming to create next-generation therapies for spinal cord injury. In this research, transplantation of human iPS cell- derived neural stem/progenitor cells owned by Keio University, combined with scaffold-mediated deliveryof HGF developed by KRINGLE demonstrated the restoration of locomotor and urinary functions for the first time in the world in the rodent model of chronic complete spinal cord transection.