Ascentage Pharma’s Core Drug Candidate HQP1351 Granted Fast Track Designation by the US FDA, Marking Another Milestone in Its Development

SUZHOU, China, and ROCKVILLE, Md.May 7, 2020 /PRNewswire/ — Ascentage Pharma (6855.HK), a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, today announced that the US Food and Drug Administration (FDA) has granted HQP1351, the Company’s core drug candidate, a Fast Track Designation (FTD) for the treatment of patients with chronic myeloid leukemia (CML) with certain genetic mutations who have failed to respond to treatments with existing tyrosine kinase inhibitors (TKIs). This is the first FTD obtained by Ascentage Pharma, and it marks another milestone for HQP1351 following its recent Orphan Drug Designation by FDA.

FTD is designed by FDA to expedite the development and review of drug candidates to treat serious disease/conditions that present urgent unmet clinical needs. This FTD for HQP1351 will lead to a series of benefits that could accelerate the clinical development and review for this drug candidate, including more frequent communications and meetings with FDA during the clinical development; and being allowed to enter Rolling Review, a process that allows the company to submit New Drug Applications (NDAs) by sections, rather than waiting until all required materials become available. This FTD also paves the way for HQP1351 to be qualified for Accelerated Approval and Priority Review designations in the future.

A key factor in determining FTD for a drug candidate is its ability to address an unmet medical need with additional clinical benefits to patients. To qualify for FTD, a drug candidate needs to demonstrate its potential to treat a condition with no existing therapies, offer significant clinical advantages over current treatment options, or bring clinical benefits to patients intolerant to existing therapies or with poor responses to them. This FTD represents FDA’s recognition of HQP1351’s potential in addressing some of these unmet medical needs.

CML is a rare hematologic malignancy with an annual incidence rate of approximately 1.9 cases/100,000. BCR-ABL tyrosine kinase inhibitors (TKIs) have significantly improved clinical management of CML. However, despite clinical benefits offered by the first-generation BCR-ABL inhibitor imatinib (Gleevec®), and several second-generation TKIs, many patients develop drug resistance. Such acquired resistance to TKIs is a major challenge in the treatment of CML. BCR-ABL tyrosine kinase mutations represent a key mechanism of acquired drug resistance; T315I, which is the most common drug-resistant mutation, occurs in about 25% of patients with drug-resistant CML. Patients with the T315I mutation are resistant to both first- and second-generation BCR-ABL inhibitors, hence presenting an urgent unmet medical need for next-generation BCR-ABL inhibitors to more effectively target the T315I mutation. Although a third-generation TKI has already been approved in the United States, there are remaining concerns about its safety As a result, patients who failed to respond to existing TKI therapies continue to present an urgent unmet clinical need for safer and more effective therapies.

HQP1351 is a novel, orally active, potent third-generation BCR-ABL inhibitor designed to effectively target BCR-ABL mutants, including T315I, and it is being developed for the treatment of patients with CML resistant to first- and second-generation TKIs. HQP1351 is the first China-developed third-generation BCR-ABL inhibitor targeting drug-resistant CML. The drug candidate is currently being evaluated in pivotal Phase II studies in China, and Ascentage Pharma plans to submit an NDA for HQP1351 this year. In July 2019, HQP1351 was cleared by FDA to enter a Phase Ib study. Data from the Phase I clinical study of HQP1351 were selected for oral presentations at the American Society of Hematology (ASH) Annual Meetings two years in a row and was nominated as “Best of ASH” research in 2019. Early data from studies of HQP1351 have demonstrated promising efficacy as well as favorable safety and tolerability profiles.

“Drug resistance to earlier-generation TKI represents an urgent unmet clinical need globally. HQP1351 is a novel, orally active, and potent third-generation BCR-ABL inhibitor being developed by an innovative biopharmaceutical company in China. In our Phase I study with a large sample size of more than 100 patients, HQP1351 demonstrated promising efficacy and a favorable safety profile, with clinical responses in many patients with relapsed or refractory CML who had no effective treatment option,” Professor Xiaojun Huang, Director of the Institute of Hematology, Peking University, and the principal investigator of HQP1351 in China. “HQP1351 has the potential of becoming a new option in the clinical management of drug-resistant CML, symbolizing the great advances in biopharmaceutical R&D in China. This FTD by FDA signifies global recognition of the clinical data from China studies, and will hopefully soon benefit patients with CML worldwide. We look forward to further progress in the clinical development of HQP1351.”

“HQP1351 is a China-developed third-generation BCR-ABL inhibitor. With this Fast Track Designation, received right after the recent Orphan Drug Designation by FDA, Ascentage Pharma has reached another major milestone in the global development of HQP1351,” said Dr. Dajun Yang, Chairman & CEO of Ascentage Pharma. “These two designations for HQP1351 indicate the urgency for addressing the unmet clinical need in the treatment of CML, and a recognition of HQP1351’s promising efficacy and safety profile by an ex-China health authority as supported by existing data. This FTD will help strengthen our communications and collaboration with FDA in future clinical development and expedite the development and review of HQP1351 in the US. Staying committed to the mission of addressing unmet clinical needs in China and around the world, we will further accelerate the clinical development of HQP1351 to hopefully soon provide a safer and more effective treatment option to patients with CML.”

About Ascentage Pharma

Ascentage Pharma (6855.HK) is a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, CHB, and age-related diseases. The Company focuses on developing therapeutics that inhibit protein-protein interactions to restore apoptosis, or programmed cell death. On October 28, 2019, Ascentage Pharma was listed on the Main Board of the Stock Exchange of Hong Kong Limited.

Ascentage Pharma has built a pipeline of eight clinical drug candidates, including a novel, highly potent Bcl-2/Bcl-xL inhibitor, as well as candidates aimed at IAP and MDM2-p53 pathways, and next-generation tyrosine kinase inhibitors. The Company is conducting more than 30 Phase I/II clinical trials to evaluate the eight drug candidates in the US, Australia, and China.

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