FDA Approves Olverembatinib Phase III Trial for CML-CP

Ascentage Pharma, a biopharmaceutical firm dedicated to pioneering treatments for cancer, age-related disorders, and chronic hepatitis B, has been granted approval by the US FDA to launch a pivotal Phase III clinical trial for olverembatinib (HQP1351). This global study will involve adult patients who have been previously treated for chronic-phase chronic myeloid leukemia (CML-CP), and it will evaluate the drug's efficacy and safety in managing the condition, irrespective of the presence of the T315I mutation.

The trial, known as POLARIS-2, is set to begin in the first half of 2024 and represents a significant step forward for olverembatinib, which is already recognized as a leading third-generation BCR-ABL inhibitor in China. This drug has demonstrated its ability to target BCR-ABL and its various mutations, including the challenging T315I mutation. The drug received its initial approval in China for treating TKI-resistant CML-CP or CML-AP with the T315I mutation in November 2021. Later, in November 2023, it was approved for a broader patient group with CML-CP who are resistant to or intolerant of earlier-generation TKIs. By early 2024, olverembatinib had also been incorporated into the National Comprehensive Cancer Network's (NCCN) guidelines for CML management.

Dr. Dajun Yang, Ascentage Pharma's Chairman and CEO, highlighted the FDA's approval as a significant milestone for the company and its mission to address unmet medical needs globally. He emphasized the innovative and patient-focused approach that led to the development of a safe and effective treatment in China for CML patients with complex cases. The inclusion of olverembatinib in the NCCN guidelines is a testament to its acceptance and potential impact on the oncology field.

Ascentage Pharma, listed on the Hong Kong Stock Exchange since 2019, is at the forefront of developing novel therapeutics that target protein-protein interactions to restore apoptosis. The company's pipeline includes nine clinical drug candidates, covering a range of innovative therapies and next-generation TKIs. With a strong commitment to clinical research, Ascentage Pharma is conducting over 40 Phase I/II trials across the US, Australia, Europe, and China. The company's dedication to innovation has been recognized through multiple national R&D projects and designations.

Olverembatinib, Ascentage Pharma's flagship drug candidate for drug-resistant CML, has received Priority Review and Breakthrough Therapy Designations from China's NMPA. It has also been granted Orphan Drug and Fast Track Designations by the US FDA and an Orphan Designation by the EU's EMA. Ascentage Pharma has secured a total of 16 ODDs, 2 FTDs, and 2 RPD Designations from the US FDA, and 1 Orphan Designation from the EMA for its drug candidates.

The company's robust R&D capabilities have facilitated the creation of a global intellectual property portfolio and strategic partnerships with leading biotech and pharmaceutical entities. Ascentage Pharma is building a world-class team for commercial manufacturing and sales and marketing, with a focus on enhancing R&D and accelerating clinical development to meet the needs of patients worldwide.