Global Registrational Phase III Study of Olverembatinib (HQP1351) Cleared by FDA

ROCKVILLE, MD, USA, and SUZHOU, China, February 13, 2024—Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in developing novel therapies for cancer, age-related diseases, and chronic hepatitis B (CHB), announced today 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 (HQP1351) 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. As the first and only China-approved third-generation BCR-ABL inhibitor, olverembatinib can effectively target BCR-ABL and a spectrum of BCR-ABL mutants, including the T315I mutation. In November 2021, olverembatinib was approved in China for the treatment of adult patients with TKI-resistant CML-CP or accelerated-phase CML (CML-AP) harboring the T315I mutation. 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.

 Dr. Dajun Yang, Chairman and CEO of Ascentage Pharma, remarked that, “FDA’s clearance of POLARIS-2 marks another major milestone for olverembatinib and our company at large, as we conduct a range of global registrational trials of our major assets to combat challenging hematologic malignancies. We are most encouraged that our cutting-edge, patient-centric innovation, which has culminated in a safe and effective treatment in China even in patients with relapsed, refractory, and/or difficult-to-treat cases of CML, will bring meaningful improvement to patients’ lives. Recent inclusion of olverembatinib in the prestigious NCCN CML management guidelines also marks another major milestone for olverembatinib, signalling its widespread acceptance by the oncology community. Fulfilling our mission of addressing unmet clinical needs in China and around the world, we will continue to investigate olverembatinib and accelerate our clinical development programs to benefit more patients.”

 *Olverembatinib is an investigational drug that has not been approved for any indication outside the Chinese mainland.

About Ascentage Pharma

Ascentage Pharma (6855.HK) is a globally focused biopharmaceutical company engaged in developing novel therapies for cancers, chronic hepatitis B, and age-related diseases. On October 28, 2019, Ascentage Pharma was listed on the Main Board of the Stock Exchange of Hong Kong Limited with the stock code 6855.HK.

Ascentage Pharma focuses on developing therapeutics that inhibit protein-protein interactions to restore apoptosis, or programmed cell death. 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.

Olverembatinib, the company’s core drug candidate developed for the treatment of drug-resistant chronic myeloid leukemia (CML) and the company’s first approved product, has been granted Priority Review Designations and Breakthrough Therapy Designations by the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA). 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. The company has built a talented team with global experience in the discovery and development of innovative drugs and is setting up its world-class commercial manufacturing and Sales & Marketing teams. One pivotal aim of Ascentage Pharma is to continuously strengthen its R&D capabilities and accelerate its clinical development programs, in order to fulfil its mission of addressing unmet clinical needs in China and around the world for the benefit of more patients.

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