FDA Fast Tracks MT-125 for Glioblastoma

The U.S. Food and Drug Administration (FDA) has granted fast track designation to MT-125 for the treatment of patients with glioblastoma.

The development was announced in a news release issued by Myosin Therapeutics, a clinical-stage biotechnology company behind the treatment.

Fast track, as the FDA explains on its website, is a process that is designed to facilitate the development and expedite the review of drugs that treat serious conditions and fill an unmet medical need, with the aim of getting new drugs to patients earlier.

“Receiving fast track designation validates our conviction that MT-125 has the potential to offer an entirely novel treatment approach to patients with even the most aggressive forms of glioblastoma” said Dr. Courtney Miller, chief executive officer of Myosin Therapeutics, in a statement included in the news release. “We are energized by the open communication with the FDA that the Fast Track offers because it will ensure we advance MT-125 as quickly as possible with our patient-centered approach.”

In June, the company announced that the FDA had accepted an investigational new drug application for MT-125, allowing for the initiation of a phase 1 study to evaluate the experimental treatment in combination with standard of care radiation among patients with newly diagnosed, IDH wild-type, MGMT-unmethylated glioblastoma.

How Does MT-125 Work to Treat Glioblastoma?

MT-125 is, according to the company, 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, the company explained, MT-125 is designed to disrupt these critical mechanisms of glioblastoma progression. It was also previously granted orphan drug designation by the FDA for the treatment of malignant gliomas, including glioblastoma.

“Glioblastoma remains one of the most aggressive and treatment-resistant cancers, with limited advances over the past two decades,” said Miller said in a news release issued in June. “In collaboration with the Mayo Clinic, we have rapidly advanced our MT-125 program in glioblastoma, and we’re driven and encouraged by the FDA’s clearance to proceed with our first-in-human trial. Preclinical studies suggest targeting non-muscle myosin II represents a uniquely holistic approach to tackling this complex and devastating disease.”

“We appreciate the FDA’s rapid and comprehensive review of our IND submission,” said Valerie Ahmuty, head of regulatory affairs at Myosin Therapeutics, also stated in the news release. “The population of patients we hope to support with MT-125 is in dire need of treatment options.”

More About Glioblastoma

Glial cells, as defined by the National Cancer Institute, are any of the cells that hold nerve cells in place and help them work the way they should.

Glioma, while not a specific type of brain tumor, is a general term for tumors that start in the glial cells such as astrocytomas, which include glioblastomas, according to the American Cancer Society, which noted that approximately three out of 10 brain tumors are gliomas. High-grade astrocytomas tend to grow quickly and spread to surrounding brain shalf of all gliomas and are the most common malignant brain tumors in adults.

Glioblastomas, according to the American Brain Tumor Association, make up approximately 14% of all primary brain tumors, and on average more than 12,000 cases of glioblastoma are diagnosed each year in the United States.

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References:

1. “Myosin Therapeutics Receives FDA Fast Track Designation for MT-125 in Glioblastoma,” news release; https://myosintherapeutics.com/myosin-therapeutics-receives-fda-fast-track-designation-for-mt-125-in-glioblastoma/
2. “Fast Track,” FDA; https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track
3. “Myosin Therapeutics Receives FDA Clearance to Initiate First-in-Human Trial of MT-125 in Glioblastoma,” news release; https://myosintherapeutics.com/myosin-therapeutics-receives-fda-clearance-to-initiate-first-in-human-trial-of-mt-125-in-glioblastoma/
4. “Glial cell,” National Cancer Institute; https://www.cancer.gov/publications/dictionaries/cancer-terms/def/glial-cell
5. “Types of Brain and Spinal Cord Tumors in Adults,” American Cancer Society; https://www.cancer.org/cancer/types/brain-spinal-cord-tumors-adults/about/types-of-brain-tumors.html
6. “Glioblastoma (GBM),” American Brain Tumor Association; https://www.abta.org/tumor_types/glioblastoma-gbm/