Myosin wins $2M grant to advance brain cancer treatment MT-125
Florida funding to support Phase 1/2 clinical trial in glioblastoma
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Myosin Therapeutics has been awarded $2 million in funding to support a Phase 1/2 clinical trial testing its lead experimental cancer therapy, MT-125, in glioblastoma, an aggressive form of glioma.
The Florida Department of Health is providing the funds through the Casey DeSantis Florida Cancer Innovation Fund (FCIF), a statewide initiative designed to support the development of innovative cancer therapies and strengthen Florida’s life sciences ecosystem.
Myosin said it was selected for the award based on the scientific innovation and clinical potential of MT-125.
“This support from the Florida Department of Health will help accelerate the clinical development of MT-125 and strengthen Florida’s role as a growing hub for biotechnology innovation,” Patrick Griffin, PhD, co-founder and chief scientific officer of Myosin, said in a company press release. “Programs like FCIF play an important role in enabling emerging biotechs to translate promising scientific discoveries into clinical programs that can ultimately benefit patients.”
Dubbed STAR-GBM (NCT07185880), the study will enroll up to 36 adults aged 18 and older with newly diagnosed glioblastoma. The Phase 1 portion of the study will assess the therapy’s safety, tolerability, and pharmacokinetics (how it moves through and is processed in the body) when given at multiple ascending doses. It will also generate early clinical data to guide dosing in the trial’s Phase 2 portion, which will evaluate the most promising doses in a larger patient population.
Mayo Clinic trial tests therapy’s safety, tolerability
The trial is being conducted in collaboration with Mayo Clinic, with recruitment planned across its clinical research units in Florida, Arizona, and Minnesota. The award will support clinical trial activities being conducted at the Mayo Clinic site in Florida.
“We are honored to receive support from the Casey DeSantis Florida Cancer Innovation Fund as we bring MT-125 to patients,” said Courtney Miller, PhD, co-founder and CEO of Myosin. “This funding helps support the continued progress of our Phase 1/2 STAR-GBM trial and reflects the growing strength of Florida’s life sciences community in advancing innovative cancer therapies.”
Glioblastoma is a highly aggressive and fast-growing type of brain cancer that arises from the uncontrolled growth of astrocytes, cells that normally support and protect nerve cells in the brain. Treatment options usually rely on surgery followed by radiation and chemotherapy or other treatments. However, responses are often short-lived, with high rates of recurrence and poor outcomes.
MT-125 is a small molecule being developed for glioblastoma and other hard-to-treat cancers, including acute myeloid leukemia, an aggressive blood cancer, and pancreatic cancer. It is designed to block two proteins, non-muscle myosin IIA and IIB, which regulate processes that cells rely on to divide and grow, move, and invade surrounding tissue. Such proteins, especially myosin IIA, are often present at higher levels in several types of cancer, including glioblastoma.
By targeting these proteins, the therapy aims to limit tumor growth and spread while also triggering stress responses within cancer cells that may lead to their elimination, the company said.
In preclinical studies using mouse models of glioblastoma, MT-125 showed a strong ability to reach the brain, block cancer cell spread, and prolong survival. The treatment also increased stress responses in cancer cells that led to DNA damage.
MT-125 showed greater effects when combined with radiation or other cancer drugs, improving survival compared with either treatment alone.
In the Phase 1 portion of STAR-GBM, groups of up to six participants will receive increasing doses of MT-125 (25-100 mg) given five days per week, followed by two days off, over six weeks. The therapy will be given in combination with standard radiotherapy.
While the main goal is to assess the treatment’s safety and tolerability, researchers will also evaluate how the drug moves through the body and determine the recommended dose for the study’s Phase 2 portion. Once an appropriate dose is identified, the therapy will be further evaluated at one or two selected dose levels in combination with radiotherapy in Phase 2.
Researchers will also explore early signs of effectiveness, including tumor response, time without disease worsening (progression-free survival), and overall survival.
MT-125 has received U.S. Food and Drug Administration (FDA) orphan drug designation for malignant gliomas and fast track designation for glioblastoma. Orphan designation is granted to therapies targeting rare diseases and can provide incentives such as market exclusivity and regulatory support. Fast track designation is intended to speed the development and review of therapies for serious conditions with unmet medical needs.
