MT-125

A brain-penetrant, dual NMIIA/IIB inhibitor currently being evaluated in the Phase 1/2 STAR-GBM study (NCT07185880). MT-125 simultaneously prevents tumor invasion and proliferation, while activating tumor cell’s own stress responses, leading to their elimination. In preclinical studies, MT-125 also increased the effectiveness of radiation and FDA-approved oncogenic signaling inhibitors (Kenchappa et al, Cell, 2025). MT-125 is being administered with standard of care radiotherapy in the STAR-GBM trial.

Orphan Drug & Fast Track

Orphan-first expansion, with a clear path to broader oncology. We pursue an orphan-first strategy to move faster in well-defined populations while maximizing regulatory incentives and value protection. As we establish human safety and PK in GBM, we are expanding the MT-125 opportunity into additional high-mechanical-demand cancers where preclinical sensitivity tracks with reliance on cancer cell mechanics – not tissue of origin or driver mutations.

Pipeline Expansion

As one example, AML is a high-mechanical-demand hematologic setting where NMII dependence is pronounced and negatively correlated with survival; in multiple AML models, including PDX, MT-125 drives tumor cell death and extends survival.

MT-228, an NMIIA/IIB small molecule inhibitor with unique characteristics is being pursued for non-orphan oncology indications.