Orphan Drug Designation May Lead to New Immunotherapy Treatment for Mesothelioma

Another pharmaceutical company is dedicating resources to help find an effective treatment for mesothelioma. Its efforts have led to an Orphan Drug Designation from the U.S. Food and Drug Administration, and comes on the heels of CRS-207 and tremelimumab, two other drugs developed to make a difference for mesothelioma patients, receiving the same designation from the FDA.

According to an Aug. 21 press release from MTG Biotherapeutics, a U.S. venture of Momotaro-Gene, a Japanese biotechnology company, the FDA has granted Orphan Drug Designation for the company’s immuno-oncology drug, MTG-201, for the treatment of malignant mesothelioma.

Mesothelioma is a rare, aggressive cancer that attacks the linings of the lungs, heart or abdomen. Although recent breakthroughs in treatment have improved the life expectancy for some patients, the prognosis for patients is poor with survival often less than 18 months. The only proven cause of the cancer is past exposure to asbestos.

“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,” said Richard Lowenthal, CEO and President of MTG, in the press release. “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 targets the Dickkopf-3 gene defects. According to MTG, the Dickkopf-3 gene produces the REIC (Reduced Expression in Immortalized Cells) protein, which helps drive cell apoptosis, or death. However, when REIC is absent, cancer cells stay alive and continue to grow and divide.

MTG-201 is an immunotherapy that helps re-activate a mesothelioma patient’s weakened immune system. MTG-201 is also being evaluated for efficacy in combination with anti-PD-1, anti-PD-L1 and anti-CTLA-4 antibodies. Keytruda is an anti-PD-1 drug that the mesothelioma community is keeping a close eye on. Two long-term mesothelioma survivors, Lou Williams of Australia, and Mavis Nye of England, have had significant tumor shrinkage and improved quality of life while taking the drug.

“MTG-201 represents a very novel approach to treating this cancer by selectively inducing apoptosis and inducing an immunologic response against the cancer,” said Lowenthal. “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.”

The FDA notes that the Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders. The designation came about from the Congressional Orphan Drug Act as an incentive for pharmaceutical companies to focus development efforts on rare diseases.

The Act was initiated because Congress recognized that pharmaceutical companies were not willing, or able, to expend the resources to develop drugs for a small number of people. A disease or condition receives an orphan disease designation in the U.S. because it affects fewer than 200,000 Americans at any given time. Mesothelioma is diagnosed in close to 3,000 Americans each year.

Orphan designation qualifies the sponsor of the drug for various development incentives including tax credits for qualified clinical testing. The granting of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval.

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