Suzhou, May 8, 2023 - Kintor Pharmaceutical Limited (“Kintor Pharma”, HKEX: 9939), a clinical-stage biotechnology company developing innovative small molecules and biological therapeutics, announced that the Phase I clinical trial of in-house developed GT1708F (Hedgehog/SMO Inhibitor) for treatment of hematologic malignancies in China has been successfully completed. The result showed that GT1708F has demonstrated a good safety and tolerability profile, and all patients experienced no dose-limiting toxicity (the “DLT”) or drug-related serious adverse events (the “SAE”). Preliminary efficacy was observed starting from 180mg dose level in dose escalation stage for patients with acute myeloid leukemia (the “AML”) who failed multi-line therapies, and the myeloid blasts decreased by up to 62% compared to the baseline in AML patients.

The Phase I clinical trial is a study to evaluate the safety, tolerability, pharmacokinetic and preliminary efficacy of GT1708F for treatment of patients with hematological malignancies, and the main purpose is to evaluate the safety and tolerability of different doses of GT1708F in patients with hematological malignances. Professor Jianxiang Wang（王建祥） and Professor Junyuan Qi（齐军元）of the Institute of Hematology, Chinese Academy of Medical Sciences are the leading principal investigators (leading PI) of this trial.

A total of 18 patients were enrolled in the trial, including 15 patients with AML and 3 patients with myelodysplastic syndrome (“MDS”). The doses and enrollment were 20mg once daily (“QD”) (1 case), 40mg QD (1 case), 80mg QD (4 cases), 120mg QD (3 cases), 180mg QD (3 cases), 240mg QD (3 cases), and 320mg QD (3 cases), respectively. The results showed that all patients experienced no DLT or drug-related SAE. The overall safety of each dose group of GT1708F was good, most of the treatment related adverse events (“TEAE”) were mild, and no TEAE resulting in death occurred.

GT1708F is a SMO protein inhibitor with high activity and specificity, which is in the leading position among drugs with the same target in terms of inhibitory activity. In vitro studies showed that its combination use with the AML drug BCL-2 inhibitor has a synergistic effect, and can significantly increase the apoptosis of malignant hematoma cells induced by the latter.

Dr. Youzhi Tong, founder, Chairman and Chief Executive Officer of Kintor Pharma, commented, “In addition to targeting blood cancer and solid tumors, the latest research and clinical results show that the Hedgehog signaling pathway plays a vital role in idiopathic pulmonary fibrosis (“IPF”) diseases. At present, there is a huge unmet clinical need in the field of IPF, and there is no effective therapy that can be used to terminate the fibrotic process or cure the disease. Currently, the company is actively preparing for relevant clinical trial applications for IPF and tumor indications.”