Toniribate, a Novel Topoisomerase II Inhibitor, Receives FDA Orphan Drug Designation for Relapsed/Refractory Biliary Tract Cancer

VCBeat (WeChat ID: vcbeat) has learned that Toniribate, a novel orphan drug co-developed by two biopharmaceutical companies, Mundipharma EDO and Imbrium Therapeutics, has recently received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of recurrent, refractory biliary tract cancer.

Toniribate, a novel topoisomerase II inhibitor, demonstrates favorable therapeutic efficacy in recurrent and refractory biliary tract cancer. Phase 2 trials have yielded positive results for toniribate. The drug undergoes carboxylesterase-mediated hydrolysis at the tumor site to release active etoposide, thereby inhibiting topoisomerase II and inducing tumor DNA fragmentation.

Biliary tract cancer is a rare malignancy and the second most common primary hepatobiliary cancer after hepatocellular carcinoma. Approximately 8,000 patients are diagnosed with biliary tract cancer annually in the United States. Radical resection is the only curative treatment option for patients with biliary tract cancer; however, those with recurrent or refractory disease are not candidates for radical surgery and typically require second-line therapy. Currently, there is no standard of care for patients with recurrent or refractory biliary tract cancer, and only a limited number of drugs are under development.

Thomas Mehrling, CEO of Mundipharma EDO, stated, “We are pleased that the FDA has recognized the value of etoposide Toniribate in the treatment of recurrent and refractory biliary tract cancer. Moving forward, we will work with Imbrium Therapeutics to accelerate the development of etoposide Toniribate, aiming to initiate Phase 3 clinical trials in the European Union, the United States, Australia, and other countries by 2020.”

It is understood that orphan drugs, also known as rare disease drugs, are so named because few pharmaceutical companies focus on their development due to the small patient population, limited market demand, and high R&D costs. To incentivize pharmaceutical companies to develop treatments for rare diseases, companies that receive orphan drug designation from the U.S. Food and Drug Administration (FDA) are granted seven years of market exclusivity and tax credits for clinical trials.

Mundipharma EDO is a biopharmaceutical company dedicated to oncology research, developing novel therapeutic regimens for patients with recurrent and rare cancers.

Imbrium Therapeutics is a subsidiary of the pharmaceutical company Purdue Pharma. As a clinical-stage biopharmaceutical company, it is dedicated to developing novel therapeutics to advance medical science. Currently, the company is developing new drugs for the treatment of cancer and central nervous system disorders.

(Compiled by Jiao Yanli)