Ascentage Pharma's Core Drug Candidate HQP1351 Granted Orphan Drug Designation by the US FDA for the Treatment of Patients with Chronic Myeloid Leukemia

SUZHOU, China, and ROCKVILLE, Md., May 4, 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 (US FDA) has granted HQP1351, the Company's core drug candidate, an Orphan Drug Designation (ODD) for the treatment of chronic myeloid leukemia (CML). This is the first ODD obtained by Ascentage Pharma.

The term "orphan drugs" refers to pharmaceutical products developed for the prevention, diagnosis, and treatment of rare diseases or conditions. In the United States, an orphan disease is defined as a disease or condition with a prevalence of less than 200,000 patients in the country. Since the Orphan Drug Act was passed in 1983, the US government has provided incentives and policy support to encourage development of orphan drugs. This ODD from FDA qualifies HQP1351 for various development incentives, including a tax credit of 50% of expenditures incurred in clinical studies, a waiver of the New Drug Application (NDA) fee, research grant awarded by FDA, and, most importantly, 7 years of US market exclusivity upon approval.

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 TKI 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 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.

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. In July 2019, HQP1351 was cleared by FDA to enter a Phase Ib study. HQP1351 is the first China-developed third-generation BCR-ABL inhibitor targeting drug-resistant CML. The drug candidate is currently being evaluated in a pivotal Phase II study in China, and Ascentage Pharma plans to submit an NDA for HQP1351 this year. 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 a finalist of "Best of ASH" research in 2019.

"There is significant unmet clinical need in the treatment of CML globally. This ODD from FDA marks a major milestone for HQP1351 which will bring about the incentives and support that will enable us to further accelerate the global development and commercialization of this drug candidate," said Dr. Dajun Yang, Chairman & CEO of Ascentage Pharma. "Given the favorable safety and efficacy data obtained thus far, we will expedite the development and are hopeful that HQP1351 will soon benefit patients worldwide."

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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