M4K2009

M4K Pharma is developing a series of brain-penetrant small molecule inhibitors targeting ALK2 (activin receptor-like kinase 2), also known as ACVR1, for the treatment of pediatric brain cancers. The program specifically targets Diffuse Intrinsic Pontine Glioma (DIPG) and H3 K27M mutant Diffuse Midline Gliomas (DMG), where ALK2 is frequently dysregulated due to gain-of-function mutations in the ACVR1 gene (found in 25-33% of cases). These compounds, including leads like M4K2009 and M4K2149, are designed to cross the blood-brain barrier and selectively inhibit the kinase activity of ALK2 to slow tumor progression. M4K Pharma operates under an open science model, sharing all research data publicly to accelerate the development of affordable pediatric therapies.

Other names

ALK2 inhibitorALK-2 inhibitorALK 2 inhibitorACVR1 inhibitorACVR-1 inhibitorACVR 1 inhibitor