Jupiter, FL – Myosin Therapeutics, Inc., a clinical-stage biotechnology company developing first-in-class therapies targeting molecular motors, today announced that the U.S. 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 tumor 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-tumor 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, recognizing the potential of MT-125 in this rare and devastating disease and providing additional regulatory and financial support for the drug’s development.
“Glioblastoma remains one of the most aggressive and treatment-resistant cancers, with limited advances over the past two decades,” said Dr. Courtney Miller, Founder, and Chief Executive Officer of Myosin Therapeutics. “In collaboration with the Mayo Clinic, we have rapidly advanced our MT-125 program in glioblastoma, and we’re driven and encouraged by the FDA’s clearance to proceed with our first-in-human trial. Preclinical studies suggest targeting non-muscle myosin II represents a uniquely holistic approach to tackling this complex and devastating disease.”
“We appreciate the FDA’s rapid and comprehensive review of our IND submission,” said Valerie Ahmuty, head of Regulatory Affairs at Myosin Therapeutics. “The population of patients we hope to support with MT-125 is in dire need of treatment options.”
About Myosin Therapeutics
Myosin Therapeutics is a biotechnology company based in Jupiter, FL, advancing therapies that target molecular motor proteins involved in cancer and neurological disease. The company’s lead program, MT-125, is being developed for glioblastoma and other treatment-resistant solid tumors.
For more information, visit www.myosintherapeutics.com.
