Ascentage Pharma announced that it has received clearance from the US Food and Drug Administration (FDA) to initiate a global registrational Phase III trial of olverembatinib (HQP1351) in previously treated adult patients with chronic-phase chronic myeloid leukemia (CML-CP). Under protocol HQP1351CG301, titled "A Global Multicenter, Open-Label, Randomized, Phase III Registrational Study of Olverembatinib in Patients with Chronic-Phase Chronic Myeloid Leukemia (POLARIS-2"), the study is designed to assess the efficacy and safety of olverembatinib in the treatment of patients with CML-CP with or without the T315I mutation. The US FDA's clearance of the trial marks the first global registrational Phase III trial for olverembatinib in patients with CP-CML to be conducted.

The trial is scheduled to commence during the first half of 2024. Olverembatinib is a global best-in-class novel drug developed by Ascentage Pharma. In November 2023, olverembatinib was approved for a new indication in adult patients with CML-CP resistant and/or intolerant of first-and second-generation TKIs.

And in early 2024, the drug was included in the latest guidelines from the National Comprehensive Cancer Network (NCCN) for the management of CML. The company has built a pipeline of 9 clinical drug candidates, including novel, highly potent Bcl-2, and dual Bcl-2/Bcl-xL inhibitors, as well as candidates aimed at IAP and MDM2-p53 pathways, and next-generation tyrosine kinase inhibitors (TKIs). Ascentage Pharma is also the only company in the world with active clinical programs targeting all three known classes of key apoptosis regulators.

The company is conducting more than 40 Phase I/II clinical trials in the US, Australia, Europe, and China. Ascentage Pharma has been designated for multiple Major National R&D Projects, including five Major New Drug Projects, one New Drug Incubator status, four Innovative Drug Programs, and one Major Project for the Prevention and Treatment of Infectious Diseases. To date, the drug had been included into the China 2022 National Reimbursement Drug List (NRDL).

Furthermore, olverembatinib has been granted an Orphan Drug Designation (ODD) and a Fast Track Designation (FTD) by the US FDA, and an Orphan Designation by the EMA of the EU. To date, Ascentage Pharma has obtained a total of 16 ODDs, 2 FTDs, and 2 Rare Pediatric Disease (RPD) Designations from the US FDA and 1 Orphan Designation from the EMA of the EU for 4 of the company's investigational drug candidates. Leveraging its robust R&D capabilities, Ascentage Pharma has built a portfolio of global intellectual property rights and entered into global partnerships with numerous renowned biotechnology and pharmaceutical companies and research institutes such as UNITY Biotechnology, MD Anderson Cancer Center, Mayo Clinic, Dana-Farber Cancer Institute, MSD, and AstraZeneca.

Any of these intentions may alter in light of future registrational trials.