Nanjing-based Neurodawn Pharmaceutical Co., Ltd has announced that its Category 1 drug, injectable Y-1, has obtained clinical trial approval from the National Medical Products Administration (NMPA) for the treatment of primary intracranial malignant tumors.
TRPML1 Inhibitor’s Non-Clinical Study Results
Non-clinical studies have demonstrated that the TRPML1 inhibitor, Y-1, can effectively inhibit the proliferation of both central nervous system (CNS) and non-CNS tumor cells. It achieves this by blocking the cell cycle at the G0/G1 phase and inducing novel forms of tumor cell death, characterized by the induction of vacuolization phenotype in tumor cells. These findings suggest that Y-1 may offer a new approach to treating various types of cancer.
Pre-Clinical Penetration and Efficacy of Y-1
Pre-clinical studies have shown that Y-1 can efficiently penetrate the blood-brain barrier (BBB), a critical factor for drugs targeting brain tumors, to reach and inhibit the growth of brain solid tumors. The drug has been effective against both temozolomide (TMZ)-sensitive and TMZ-insensitive brain gliomas, prolonging the survival period of mice with brain carcinoma in situ. Additionally, when used in combination with TMZ, Y-1 has been shown to enhance the efficacy of chemotherapy, indicating its potential as a valuable adjunct in cancer treatment regimens.-Fineline Info & Tech
