Pharos iBio, an AI-backed new drug developer based in Korea, said it would conduct a translational study with Johns Hopkins University School of Medicine to develop a combination therapy using its pipeline PHI-101 to treat relapsed or refractory acute myeloid leukemia (AML).  

A translational study involves the application of findings from basic scientific research to clinical or real-world settings in order to improve medical practice and patient outcomes.

The Korean company and Donald Small, MD, Ph.D, a professor of pediatric oncology at Johns Hopkins who is a world-renowned expert in AML, will conduct the study to assess the efficacy of PHI-101 in combination with azacitidine (brand name: Vidasa) or venetoclax (brand names: Venclexta and Venclyxto), which are used as the first-line treatment of AML. 

Professor Small has over 30 years of research experience with FLT3, the target protein of PHI-101, and has led clinical studies of several FLT3 inhibitors in development, Pharos iBio said. He is also advising on the design and the U.S. clinical hospital network for the PHI-101 global phase 2 clinical trial in the U.S. 

The ongoing PHI-101 clinical trial is in a multinational phase 1b study in AML patients who have not responded to other existing drugs or whose disease has relapsed after treatment.

On Aug. 2, Pharos iBio received approval from the Ministry of Food and Drug Safety

“for the therapeutic use” of PHI-101 to treat relapsed or refractory AML.

The therapeutic use approval allowed the Catholic University of Korea Seoul St. Mary’s Hospital, the applicant, to treat  patients with relapsed or refractory AML on an individual basis.

Pharos iBio has expanded the indications for PHI-101 by utilizing its AI-powered drug discovery platform, “Chemiverse.” Chemiverse utilizes the latest AI algorithms such as transformer generation models and big data of approximately 6.2 billion protein 3D structures and compounds. It is used in Pharos iBio's in-house drug discovery process to predict new targets, identify candidates, and expand indications.

The Korean company is using Chemiverse to work on PHI-101 targeting AML, recurrent ovarian cancer, triple-negative breast cancer, and as a radiosensitizer. 

"PHI-101 for AML is our flagship pipeline, and we are preparing for a global phase 2 clinical trial, including the U.S., Australia, and Korea, targeted for the first half of 2024,” said Yoon Jeong-Hyuk Yoon, CEO of Pharos iBio. "The combination therapy translational study with Johns Hopkins University School of Medicine is also part of our 'One Source Multi-Use' strategy."

Shares of Pharos iBio, listed on Kosdaq,  were down 2.8 percent to trade at 15,270 won  ($11.54) as of 11:28 a.m., Monday.