Myosin Therapeutics Inc, a Florida-based clinical-stage biotechnology company developing therapies targeting molecular motors, announced on Monday that the US Food and Drug Administration (FDA) has accepted the company's Investigational New Drug (IND) application for MT-125.

The active IND allows the company to initiate a Phase 1 study to evaluate MT-125 in combination with standard of care radiation in patients with newly diagnosed IDH wild type, MGMT unmethylated glioblastoma. This subset of patients faces a particularly poor prognosis due to limited responsiveness to current chemotherapy.

MT-125 is a selective inhibitor of non-muscle myosin II (NMII), a molecular motor protein that drives tumour proliferation and invasion, resistance to therapy and oxidative stress, and immune evasion. By specifically targeting NMII, MT-125 is designed to disrupt these critical mechanisms of glioblastoma progression. In preclinical models, MT-125 demonstrated potent anti-tumour activity and enhanced the efficacy of radiotherapy.

MT-125 has been granted Orphan Drug Designation by the FDA for the treatment of malignant gliomas, including glioblastoma, recognising the potential of MT-125 in this rare and devastating disease and providing additional regulatory and financial support for the drug's development.

"We appreciate the FDA's rapid and comprehensive review of our IND submission," said Valerie Ahmuty, Myosin Therapeutics head of Regulatory Affairs. "The population of patients we hope to support with MT-125 is in dire need of treatment options."