New Mesothelioma Treatment Receives FDA Orphan Drug Designation

Illustration of mesothelioma research

An Orphan Drug Designation has been granted to a new mesothelioma treatment by the US Food and Drug Administration (FDA). MTG Biotherapeutics (MTG) is the immune-oncology company that specializes in new immune system-based cancer treatments and developed the drug, which has been named MTG-201. Discoveries at Okayama Medical Hospital in Japan led to its creation.

Immuno-oncology is an evolving field of treatment that utilizes a patient’s immune system to fight off cancer. Cancer cells are extremely difficult to fight off because they don’t die off like natural cells when the time is right. Rather, they continue to live and multiply.

Being a part of the US FDA Orphan Drug Designation program gives status as “intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the USA.” This way the company will receive the support needed to perfect the treatment.

If approved, this designation can provide many benefits to MTG Biotherapeutics, including seven years of US market exclusivity, tax credits and fee exemption from FDA applications.

“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 immunological response against cancer,” said CEO and President of MTG, Rob Lowenthal.

MTG-201 works by targeting Dickkopf-3 gene defects. Dickkopf-3 produces a protein called Reduced Expression in Immortalized Cells (REIC). It’s a vital protein in the downstream mechanism of apoptosis (cell death) when non-present cancer cells can’t die.

The result is an increase in active cancer cells dying by convincing the cells to kill themselves like normal, healthy cells would at the end of their life.

“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.”

Not only does MTG-201 rely on REIC protein expression to induce apoptosis and directly kill the cancer, but it also stimulates the production of activated T-cell lymphocytes to kill residual cancer cells. Thus, making it an excellent candidate to work with checkpoint inhibitors such as anti-PD-1, anti-PD-L1, and anti-CTLA-4 antibodies.

The only US FDA-approved treated for mesothelioma patients is pemetrexed combined with platinum-based therapy, although there are many other orphan drugs, in addition to this promising MTG-201, in the development stage.