Fast Track is an FDA program designed to facilitate development and expedite review of drugs that treat serious conditions and address unmet medical needs. This designation benefits patients and drug sponsors by enabling more frequent interactions with the FDA and providing eligibility for rolling review of a marketing application.

Read more about Myosin Therapeutics Receives FDA Fast Track Designation for MT-125 in Glioblastoma.

The funding will support Myosin Therapeutic’s Phase I STAR-GBM dose escalation and expansion trial of MT-125, a first-in-class novel small molecule therapeutic being evaluated in patients with newly diagnosed, MGMT unmethylated glioblastoma. Glioblastoma remains among the most lethal cancers, with median survival measured in months. MT-125 targets non-muscle myosin II, a critical driver of tumor cell invasion, proliferation and treatment resistance, representing a novel therapeutic approach that is distinct from existing standards of care.

Myosin Therapeutics Awarded $4.5 Million NCI Bridge Grant to Advance Phase I Trial of MT-125 in Glioblastoma.

The experimental medication, called MT-125, has received approval from the FDA to move to clinical trials as a possible first-line treatment for the most aggressive form of the brain cancer.

Each year, 14,000 people in the United States receive the devastating news that they have glioblastoma. It is a cancer with an average survival of just 14 to 16 months. Standard treatments include surgery, radiation and chemotherapy. But half of glioblastoma patients have a subtype that doesn’t respond to any approved cancer drugs, said Courtney Miller, Ph.D., a professor and academic affairs director at The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology.

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.

Myosin Therapeutics, Inc. is advancing its lead compound, MT-125, to treat glioblastoma, the most aggressive brain cancer with an average survival time of only 8 months. MT-125 targets a molecular nanomotor, blocking both tumor growth and invasion. The FDA granted orphan drug designation to MT-125 for malignant gliomas, underscoring its potential to improve outcomes for patients with this devastating disease. 

The company is also progressing MT-110 for the treatment of methamphetamine use disorder (MUD), Millions worldwide suffer from MUD, yet no FDA-approved treatments currently exist. The latest funding, combined with NIH support, will allow MT-110 to enter Phase 1 clinical trials.