Nov 10, 2021

Pharos iBio’s AML drug candidate wins FDA’s orphan drug status 바로가기 기자명 Shim Hyun-tai 닫기 Pharos iBio said its PHI-501, a next-generation anticancer drug for acute myeloid leukemia (AML), has won orphan drug designation from the U.S. Food and Drug Administration (FDA). Pharos iBio’s PHI-501, a next-generation anticancer drug for acute myeloid leukemia (AML), has won orphan drug status from the U.S. Food and Drug Administration. It was the U.S. regulator’s second orphan drug designation of the Korean company’s product. FDA granted similar status to Pharos iBio’s main pipeline, PHI-101, a next-generation FMS-like tyrosine kinase 3 (FLT3) inhibitor for AML, in 2019. PHI-501 is the first targeted anticancer therapeutics against neuroblastoma-RAS (NRAS) mutation using a more effective method for inhibition using synthetic lethality. NRAS is a protein that plays a significant role in cell differentiation, proliferation, and survival in acute myeloid leukemia. However, NRAS mutants continuously send signals for cell growth which also leads to cancer growth. According to Decision Resources Group, a global health industry research firm, AML is a rare disease that shows increased occurrence in the older populations above 65. It is particularly prevalent in the U.S. and Europe. The group expected the global market for AML treatments to reach $2.5 billion by 2025 with a 15 percent annual growth. Pharos iBio holds eight new drug pipelines, including PHI-101, and originally built big data and AI-based platform technology. Through open innovations, the company plans to promote joint research and development and technology transfer with global pharmaceutical companies and overseas organizations. “We will benefit from various incentives, including shortened review period for clinical approval and commercialization, reduced application fee with tax exemption, and a seven-year marketing exclusivity,” said Pharos iBio CDO Han Hye-jung, former principal scientist at Roche Sequencing Solutions. “We will speed up the clinical trials of PHI-501 to enter the global market.” The company has recently built a synthesis and bio laboratory at its headquarters in Anyang, Gyeonggi Province, establishing an infrastructure to discover new drug candidates independently. Pharos iBio received approval for the first phase 1 clinical trial of PHI-101 in Korea and continued to strengthen its ability to develop new drugs for rare and incurable diseases.