A rare disease drug developed using artificial intelligence has been used in the clinical site to treat patients. Market expectations are high as it confirms the possibility of developing new drugs using AI.
Pharos iBio said Thursday that it received approval for the therapeutic use of PHI-101, a treatment for relapsed or refractory acute myeloid leukemia (AML), from the Ministry of Food and Drug Safety Wednesday.
The approval of PHI-101 for therapeutic use was made on an “individual basis,” as the ministry gave the go-ahead to a healthcare provider to treat the individual patient. The applicant was the Catholic University of Korea-Seoul St. Mary’s Hospital.
Therapeutic use approval is a system that allows investigational drugs for patient treatment. It is designed to provide treatment opportunities for patients with new infectious diseases and emergency patients without alternative treatment. However, it differs from a conditional license in that it approves a single treatment.
PHI-101, for which Pharos iBio is conducting a multinational phase 1b clinical trial, is a targeted anticancer drug for patients with AML who have relapsed or are refractory to existing drugs. It targets the FLT3 (FMS-like tyrosine kinase 3) gene mutation, which is present in about 30-35 percent of ALM patients.
Patients with FLT3 mutations are known to have about twice a lower survival rate and a higher risk of relapse than those without. According to the company, PHI-101 has shown in clinical trials that it could reduce malignant myeloid cells in patients with drug-resistant mutations from existing FLT3 inhibitors.
Pharos iBio discovered PHI-101 through its AI drug discovery platform, Chemiverse. Pharos iBio explained that it discovered PHI-101 with low cardiotoxicity by utilizing Chemiverse's lead discovery and optimal drug filter module ADMET.
In addition, Pharos iBio has expanded the indication of PHI-101 to recurrent ovarian cancer and is conducting phase I clinical trials in Korea.
Besides, it is jointly researching and developing KRAS anticancer drug, PHI-201, with Yuhan Corp.
Last month, it signed a contract manufacturing organization (CDMO) agreement with Daewoong Bio to develop raw materials for the solid cancer drug PHI-501 clinical trials.
"We are pleased that PHI-101 can provide treatment opportunities for patients in critical situations," CEO Yun Jeong-hyuk said. "We will continue contributing to public health by developing rare and incurable drugs with high unmet medical needs."
The news sent shares of Pharos IBio, listed on the Kosdaq market on March 27, to its daily upper limit. As of 11 a.m. Thursday, its share price was 18,920 won ($14.5), up 29.95 percent from the previous day's closing price of 14,560 won.