Jupiter, FL – Myosin Therapeutics, a clinical-stage biotechnology company advancing first-in-class therapies targeting molecular motor proteins, announced the publication of two major papers in the journal Cell that establish non-muscle myosin II (NMII) as a druggable target with broad therapeutic implications.
The first article in Cell, “Development of Clinically Viable Non-Muscle Myosin II Small Molecule Inhibitors,” details the optimization and development of a new pharmacological class of small molecule inhibitors of the enzyme NMII, an actin-binding motor protein that regulates cellular functions such as division, synaptic remodeling, and immune evasion. The study describes how an iterative structure-based approach overcame historical challenges to yield brain-penetrant small molecules with high specificity and excellent tolerability.
The work originated in the lab of neuroscientist Courtney Miller, Ph.D., who teamed up with medicinal chemist, Theodore Kamenecka, Ph.D., and structural biologist, Patrick Griffin, Ph.D. With support from the National Institute of Neurological Disorders and Stroke (NINDS/NIH) Blueprint Neurotherapeutics Program and National Institute for Drug Abuse (NIDA), the three led a program to develop and bring NMII inhibitors to the clinic. The team identified MT-110 as a clinical candidate for the treatment of stimulant use disorder. MT-110 and MT-125, another NMII inhibitor being pursued in oncology, serves as the cornerstone of the company they co-founded, Myosin Therapeutics.
The potential of MT-125 in glioblastoma (GBM) is detailed in the second Cell article , “MT-125 Inhibits Non-Muscle Myosin IIA and IIB and Prolongs Survival in Glioblastoma.” GBM is an extremely aggressive, treatment-resistant brain cancer. The team collaborated with Mayo Clinic scientist and neuro-oncologist Steven Rosenfeld, M.D., Ph.D. The research was supported by NINDS and the National Cancer Institute (NCI).
In preclinical GBM models, MT-125 demonstrated robust monotherapy activity, as well as synergy with radiation and FDA-approved oncology therapeutics at suppressing tumor growth and extending survival. The publication provides detailed mechanistic insight into these therapeutic actions and informs MT-125’s clinical development strategy.
“As a clinician treating glioblastoma my entire career, collaboration with Myosin Therapeutics is meaningful” said Dr. Rosenfeld. “There’s great promise that our research will translate into improved outcomes for patients who urgently need them.”
Miller said “The work detailed in these articles establishes NMII inhibitors as a new therapeutic class and we’re passionate about using them to tackle big health challenges, starting with glioblastoma and addiction.”
