Pharos iBio announced positive results from its Phase 1 clinical trial of PHI-101, a novel, first-in-class treatment for acute myeloid leukemia (AML). The study demonstrated a 50% overall complete response rate (OCR) among evaluable patients. These findings, to be presented at the upcoming American Society of Hematology (ASH) Annual Meeting, suggest PHI-101's potential as a much-needed therapeutic option for AML patients, especially those with FLT3 mutations or who have relapsed after prior FLT3 inhibitor therapy.
Targeting FLT3 Mutations in AML
PHI-101-AML, derived from Pharos iBio's Chemiverse AI drug discovery platform, is designed to inhibit the growth of cancer cells by targeting the FLT3 gene mutation. This mutation is present in approximately 30-35% of AML patients and is associated with poorer survival rates and a higher risk of relapse compared to those without the mutation. The drug's mechanism of action offers a targeted approach to address this specific genetic driver of AML.
Phase 1 Trial Results
The Phase 1b study of PHI-101-AML, conducted at a recommended expanded dose of 160 mg monotherapy, included patients with relapsed or refractory AML. Notably, 75% of patients in the Phase 1b study had relapsed or failed after prior FLT3 inhibitor therapy. Despite this challenging patient population, 50% of evaluable patients achieved a complete response, which includes complete remission (CR), CR with incomplete hematologic recovery, and morphologic leukemia-free status (MLFS).
The trial was a collaborative effort involving several leading Korean university hospitals, including Seoul National University Hospital, Asan Medical Center, Samsung Medical Center, Pusan National University Hospital, and the Catholic University of Korea Seoul St. Mary's Hospital, as well as the ICON Cancer Center in Australia.
Development and Future Plans
Pharos iBio aims to complete the Phase 1 clinical trial of PHI-101-AML this year and pursue early commercialization through conditional marketing authorization based on Phase 2 clinical trial results. Han Hye-jung, chief innovation officer of Pharos iBio, stated, "As we continue to advance PHI-101 in phase 1, we continue to see excellent potential as a next-generation FLT3 inhibitor. We plan to complete the phase 1 of PHI-101-AML and simultaneously file an investigational new drug (IND) application for a global phase 2 clinical trial, accelerating our R&D efforts to bring new treatment options to patients with rare diseases as soon as possible."
