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Bone marrow proliplation abnormal syndrome is a group of heterogeneic myeloid cloning diseases originating from bone marrow hematopoietic stem cells, clinically manifested as ineffective hematopoietic, reduced exostular blood cells, high-risk transformation to acute myeloid leukemia.
patients with bone marrow-growth abnormal syndrome, where de-methylation drugs are difficult to treat, the medium total survival period (OS) is only 4-6 months, and follow-up treatment options are limited.
preclinical studies have shown that selective nuclear output inhibitor (SINE) compounds block a variety of tumor suppressor proteins (e.g. p53), The nuclear output of IkB, p21) causes it to accumulate and invigorate in the nucleus to play an anti-tumor role, and SINE compounds can also reduce the nucleation and transformation of a variety of cancer-causing genes combined with elF4E mRNA (c-Myc, Bcl-2, Bcl-6, cyclin D), so that tumor cells selective apoptosis.
ATG-016 belongs to a new generation of SINE compounds, compared to the first generation of nuclear output inhibitors, ATG-016 shows very small blood-brain barrier permeability and a wider treatment window, which shows initial anti-tumor activity in patients with higher-risk bone marrow growth abnormal syndrome.
"ATG-016 clinical trial approval reflects the efficient execution of Deqi Pharmaceuticals' research and development team and is the first clinical approval de niece obtained by Deqi Pharmaceuticals at Chinese mainland since its launch. "The first generation of selective nuclear output inhibitors, Celinexor, has shown extensive activity in blood malignancies and solid tumors and has been approved by the FDA for recurring multiple myeloma and diffuse large B-cell lymphomas," said Dr. Jianming Mei, founder, chairman and CEO of
Deki Pharmaceuticals.
ATG-016 as a second-generation oral SINE compound, can reduce the penetration of the blood-brain barrier, with a wider treatment window, can potentially reduce adverse events, improve drug tolerance.
" About ATG-016 ATG-016 (eltanexor) is a second generation selective nuclear output inhibitor compound.
compared to the first generation of SINE compounds, ATG-016 has lower blood barrier penetration and a wider treatment window, which can be better tolerable for higher frequency administration and longer drug exposure at high concentrations.
In this way, ATG-016 may be suitable for more adaptive disorders, and we plan to conduct phase I/II clinical studies on MDS in China, and we plan to further develop ATG-016 for high-risk tumors (such as KRAS mutated solid tumors) and viral infections associated with malignancies (e.g. nasopharyngeal cancer) in the Asia Pacific region.
note: There are deletions in the original text