Antengene Corporation Limited, a leading innovative, commercial-stage global biopharmaceutical company dedicated to discovering, developing and commercializing first-in-class and/or best-in-class medicines for cancer, announced the China National Medical Products Administration (NMPA) has approved a Phase II open-label study designed to evaluate the safety, tolerability and efficacy of the next-generation selective inhibitor of nuclear export (SINE) compound ATG-016 in patients with high-risk myelodysplastic syndromes (MDS).
ATG-016 is being developed for treating patients with MDS or solid tumors. ATG-016 and other SINE compounds inhibit the nuclear export protein called Exportin 1 (XPO1) that helps cancers grow by removing tumor suppressor proteins from the nucleus. ATG-016 is an orally-active, highly-specific next-generation XPO1 inhibitor with an improved pharmacological profile versus the first novel SINE compound, ATG-010/selinexor/XPOVIO®. These attributes can potentially enable more frequent dosing and a better tolerated dosing regimen.
“Antengene is dedicated to bringing first-in-class medicines to people in China and beyond, with a special focus on resistant and advanced cancers. We are the first company in the Asia Pacific Region with a SINE program. Our eltanexor program is comprised of three studies in mainland China, to evaluate ATG-016 in patients with advanced MDS and solid tumors.” said Dr. Jay Mei, Antengene’s Founder, Chairman and CEO. “We are encouraged by activity seen with this drug in the previously reported eltanexor data from a Phase I study in MDS patients and eager to advance this study, with the goal of benefiting patients in China with high-risk MDS.”
The KCP-8602-801 trial is an on-going open-label Phase I/II study initiated by Karyopharm Therapeutics Inc. (“Karyopharm”) to evaluate the safety, tolerability, and efficacy of ATG-016 in patients of 6 “Sub-parts” of relapsed/refractory cancer indications. Antengene will participate in the Part F Phase II of this study in which high-risk MDS patients will be enrolled to evaluate ATG-016 monotherapy. MDS is a malignancy originated in the bone marrow hemopoietic stem-cells of which the incidence increases significantly with age. The median overall survival (OS) of patients with intermediate, high, and very high-risk MDS are 3 years, 1.6 years, and 0.8 years, respectively, and patients at these risk levels have a high probability of progressing to acute myeloid leukemia (AML). Previously, Karyopharm reported data from the Part F Phase I of the study [ATG-016 monotherapy in patients with hypomethylating agent (HMA)-refractory MDS] at the American Society of Hematology (ASH) Annual Meeting, demonstrating an overall response rate (ORR) of 53% and a median OS of 9.86 months in efficacy-evaluable patients. This compares favorably to the historical survival of four to six months for patients with HMA-refractory MDS.