SUZHOU, China, and ROCKVILLE, MD, January 16, 2024—Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in developing novel therapies for cancer, chronic hepatitis B (CHB), and age-related diseases, announced today that olverembatinib (R&D Code: HQP1351) has been included in the latest guidelines from the National Comprehensive Cancer Network (NCCN) for the management of Chronic Myeloid Leukemia (CML),1 marking a major development in global recognition for its best-in-class drug candidate, which has been approved in China for the management of tyrosine kinase inhibitor (TKI)-resistant patients with chronic- and accelerated-phase CML (CML-CP and CML-AP).
NCCN is a not-for-profit organization allying 33 chiefly NCI-designated comprehensive cancer centers across the United States. Cancer management recommendations promulgated in the NCCN guidelines are continuously updated and downloaded more than 13 million times per year by practitioners and other key stakeholders. Serving as a standard for clinical practices in oncology in the US, the NCCN Guidelines is also one of the most widely adopted clinical guidelines for the global oncology community.
The NCCN guidelines report efficacy responses to olverembatinib in Chinese Phase I and II trials,2 including major cytogenetic response (MCyR) rates of 79% and 47%, complete cytogenetic response (CCyR) rates of 69% and 47%, and major molecular response (MMR) rates of 56% and 45%, in patients with CML-CP and CML-AP, respectively.
Clinical trials also suggest that olverembatinib has demonstrated effects regarding safety of the compound, with its common adverse events such as thrombocytopenia, hypertriglyceridemia, and chiefly asymptomatic elevation of enzymes that include creatine phosphokinase and liver transaminase, being typically grade 1-2 and manageable.
Findings reported at the 2023 American Society of Hematology (ASH) Annual Meeting continued to demonstrate that olverembatinib is well tolerated and effective in managing heavily TKI-pretreated CML, including in patients with challenging clinical profiles such as those with the T315I mutation who would otherwise have limited therapeutic options. These studies showed that olverembatinib significantly prolonged the rigorous efficacy benchmark of event-free survival (EFS) and reduced clinical events by 60% compared to physicians’ choices of best available therapies (BATs) in China (including imatinib, dasatinib, and nilotinib)3. Moreover, olverembatinib has shown encouraging clinical benefit in patients who have failed prior treatment with other targeted drugs, including the third-generation TKI ponatinib and the allosteric STAMP inhibitor asciminib.4
Olverembatinib is a novel, small-molecule, orally administered, third-generation BCR-ABL1 TKI initially approved in China in November 2021 for the treatment of adult patients with TKI-resistant CML-CP or CML-AP harbouring the T315I mutation; and then in November 2023 for the treatment of adult patients with CML-CP resistant to and/or intolerant of first- and second-generation TKIs. Olverembatinib is being jointly commercialized in China by Ascentage Pharma and Innovent Biologics.
Dr. Dajun Yang, Chairman and CEO of Ascentage Pharma, remarked that, “Inclusion in the prestigious NCCN guidelines marks another major milestone for olverembatinib. This event signals important recognition of our drug candidate by the oncology community. We are most encouraged that our cutting-edge, patient-centric innovation, which has culminated in a safe and effective treatment even in relapsed, refractory, and/or difficult-to-treat cases of CML, will bring meaningful improvement to patients’ lives. 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.”
Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma, said, “This recognition of olverembatinib as a potential CML treatment by the NCCN largely validates the impressive results on this drug candidate reported to date, including selection for Oral Presentation at the 2023 ASH Annual Meeting for the sixth consecutive year. Moving forward, we will expedite our clinical development programs to bring more safe and effective therapies to patients in need. These initiatives include our ongoing pivotal registrational Phase III trials of olverembatinib, including a study in patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia [NCT06051409].”
* Olverembatinib is an investigational drug that has not been approved for any indication outside the Chinese mainland
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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 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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