Jupiter, FL – [February 21, 2024] – Today, Myosin Therapeutics is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for MT-125 for the treatment of malignant gliomas, including glioblastoma (GBM), the most common and lethal glioma. Despite aggressive surgery, chemotherapy, and radiotherapy, median survival for this critically unmet need is only 15 months, with fewer than 5% of patients surviving beyond 5 years.
The granting of ODD status for MT-125 marks a critical milestone in Myosin’s efforts to develop innovative therapeutics for patients suffering from malignant gliomas. ODD provides several significant benefits designed to support and expedite MT-125’s development, including seven years of market exclusivity in the U.S. upon FDA approval. Additionally, Myosin will receive tax credits covering up to 25% of the costs associated with research and development, as well as potential grant funding from the FDA to aid clinical trials. The ODD also waives certain FDA fees, offering financial relief throughout the development process, and provides protocol assistance, helping streamline the design of clinical studies. Moreover, the designation grants a faster regulatory review process and enhances marketability, facilitating MT-125’s path to patients who urgently need more effective treatment options for GBM.
“Our receipt of the Orphan Drug Designation for MT-125 in the treatment of malignant gliomas is a critical milestone for Myosin Therapeutics, as it validates both the need for more effective therapeutics for the treatment of glioblastoma and our novel, comprehensive therapeutic approach,” said Dr. Courtney Miller, CEO and co-founder of Myosin Therapeutics. “This designation provides regulatory and financial support to advance our clinical program. In addition, because the orphan drug designation process involves an in-depth review of our efficacy data package, it underscores MT-125’s high potential to improve outcomes for GBM patients, a population that has long faced very limited treatment options.”
MT-125 is a first-in-class, small molecule selective dual inhibitor of non-muscle myosin (NM) IIA and IIB. It represents a mechanistically novel approach in GBM treatment, aiming to improve outcomes in a field that has seen little advancement in effective therapies. Myosin Therapeutics is now preparing to initiate a Phase 0/1b clinical trial with MT-125, in collaboration with Mayo Clinic, to gather critical data on its safety, pharmacokinetic profile, and efficacy.
About Myosin Therapeutics
Myosin Therapeutics is a Jupiter, FL-based biotechnology company spun out of the Wertheim UF Scripps Institute (formerly Scripps Research, Florida). The company is focused on developing therapies for oncology and central nervous system indications with a platform for targeting molecular nanomotor proteins. Myosin’s lead programs for glioblastoma and stimulant use disorder target non-muscle myosin II motor proteins.
For more information, email contact@myosintherapeutics.com
