FDA Grants Malignant Mesothelioma Treatment Orphan Drug Designation

Biologic therapy targets genetic defects found in various cancers.

A new treatment option for a rare form of cancer may soon be available to patients.

The FDA granted Orphan Drug Designation to MTG-201 for the treatment malignant mesothelioma. The advanced biologic drug targets the Dickkopf-3 genetic defect found in various cancers. The Dickkopf-3 gene produces the Reduced Expression in Immortalized Cells protein (REIC) that is vital in apoptosis cancer cell death.

Mesothelioma is a rare, aggressive cancer affecting pleural cavity lining. Current treatments for the disease are limited, especially in patients with advanced disease. There remains a dire need for treatments that directly attack mesothelioma cancer cells to induce the immune system against the disease.

"There is a significant need for new treatment options for patients with mesothelioma given this is one of the most aggressive and poorly treated cancers. MTG-201 represents a very novel approach to treating this cancer by selectively inducing apoptosis and inducing an immunologic response against the cancer," said Richard Lowenthal, CEO and president of MTG Biotherapeutics. "MTG has already demonstrated that ability of MTG-201 to induce selective apoptosis in phase 1 studies for prostate cancer, another cancer immortalized by hypermethylation of the Dickkopf-3 gene. Mesothelioma has been found to be the most sensitive cancer to this mechanism of action and as such we are moving MTG-201 into phase 1 development for this indication."

MTG-201 is the first of a series of immuno-oncology drugs in the pipeline targeting REIC protein expression with hypermethylation of the Dickkopf-3 gene. Through the expression of the REIC protein from inside cancer cells, MTG-201 generates a selective apoptosis caused by stress, which directly kills cancer and reduces the cancer burden.

Due to the selective apoptotic mechanism, MTG-201 stimulates production of activated T-cell lymphocytes that target and eliminate residual cancer cells. Through this mechanism, MTG-201 could work in tandem with checkpoint inhibitors currently in development.

The investigational drug is also currently in clinical development to treat prostate cancer, with preclinical programs ongoing for liver and bladder cancer treatment.