The investigational new drug (IND) application submitted by UBI Pharma (6562) for the newly developed targeted anticancer drug UB-941 has been approved by the Taiwan Food and Drug Administration (TFDA), Ministry of Health and Welfare for the implementation of the phase I clinical trial. The trial is a multinational, multicenter, two-stage clinical trial. It is designed to investigate the safety and efficacy of the new drug in patients with solid tumors with mutations in the BRAF gene.

The phase I clinical trial of UB-941 was approved by the U.S. FDA in August 2016, while the application for change of dosage form submitted after completion of dosage form optimization was approved in November 2018. The bioavailability of the new oral tablet is approximately 4 times higher than that of the original powder dosage form. Furthermore, tablets are more convenient for the patient, which facilitates the implementation of the trial.

UB-941 is an inhibitor designed specifically for BRAF gene mutations. The BRAF gene is part of a family called proto-oncogenes. Mutations to proto-oncogenes can induce uncontrolled cell growth and may turn normal cells into cancer cells. The BRAF V600E mutation is commonly found in melanoma, colon, rectal, ovarian and thyroid cancers. Preclinical studies have shown that the biological activity and safety of UB-941 are better than commercially available drugs, and low doses of UB-941 have an inhibitory effect on the cancer cells with the BRAF V600E mutation. Animal studies also have demonstrated that UB-941 can effectively inhibit tumor growth with fewer side effects, and the side effects are easier to recover from when compared with commercially available drugs. In summary, UB-941 combines all major advantages, including high specificity, good anticancer activity, and better medication safety; in addition, in the future it may provide patients with a safer and more effective option for medication.

Cancer is the second leading cause of death worldwide. The global anticancer drug market was approximately US$133 billion in 2017, and the market demand is continuing to grow year by year. It is estimated that the market size will reach US$178.8 billion by 2023. The estimated total amount of sales of the two commercially available BRAF inhibitors is US$1.3 billion for 2019, indicating great market potential.

UBI Pharma stated that gene testing for diagnosis will be incorporated after the successful development of the targeted anticancer drug UB-941. Future development of UB-941 will focus on precision medicine to ensure the drug is a highly safe, low-dose, effective, and affordable anticancer drug for targeted therapy.