Second-generation SINE compounds! Deqi Pharmaceuticals Class 1 new drugs were declared clinically accepted in China.

Screenshot Source: CDE official website ATG-016 is a second-generation selective nuclear output inhibitor (selective resectionor of nuclear export, SINE) that initially demonstrated a larger therapeutic window in the pipeline, promising higher frequency administration and long-term drug exposure at high concentrations, or will be suitable for more indications.
SINE compound inhibits the nuclear output of the substrate by co-priced at the binding point of the nuclear output protein 1, and promotes cancer cell death through apoptosis.
studies have shown that ATG-016 blocks the nucleus output of tumor suppressor proteins and other regulatory proteins, causing them to accumulate in the nucleus and develop their anti-cancer activity through apoptosis.
in preclinical studies, ATG-016 had only weak brain penetration in rodents and monkeys.
this reduction in brain penetration may be the reason why ATG-016 has a therapeutic window improvement.
, animals treated with ATG-016 ornithost showed lower weight loss and improved appetite.
this will allow ATG-016 to be given more frequently and with higher doses and more persistent drug exposure.
according to Deki Pharmaceuticals prospectus, SINE compounds have a competitive advantage in treating MDS compared to other treatment options, including initial significant efficacy and safety, oral dosing and less side effects.
, ATG-016 had previously observed positive results in a phase 1/2 clinical study of older patients with HR-MDS.
of the 20 patients with assessable efficacy, 7 received complete bone marrow remission (mCR) with an ORR of 35%.
, ATG-016 is currently in Phase 1/2 trials of blood and solid malignancies conducted by Karyopharm and has demonstrated exciting efficacy and controlled safety.
cancers include high-risk MDS, metastatic colorectal cancer (mCRC), metastatic desopathic prostate cancer (mCRPC), and other types of advanced cancer.
Deqi Pharmaceuticals plans to conduct an open-label, one-arm Phase 1/2 clinical trial (HATCH trial) for high-risk MDS in China, studying the effectiveness, safety and pharmacodynamics of ATG-016 monopharmaceuticals in about 60 patients in China who have failed treatments with demethylated drugs.
dose increment period (phase 1) endpoint includes RP2D, dose expansion period (phase 2) endpoint includes efficacy tests such as total remission rate, remission duration, progression-free lifetime, total lifetime, and tumor progression time.
myeloid dysplomatic syndrome is a type of cancer caused by blood cells that form in the bone marrow that are dysfunctional or dysfunctional.
, the average survival time of low-risk MDS patients is 6 years, and the average survival time of high-risk MDS patients is about 18 months.
, in addition to de-methylation drugs, there is currently no effective treatment for MDS patients, there are still a large number of clinical medical needs have not been met.
the clinical development of ATG-016 and is expected to benefit this group.
addition, Deki Pharmaceuticals plans to further develop ATG-016 for more common adaptive disorders in the Asia Pacific region (e.g. solid tumors with KRAS mutations) and viral infections associated with malignant tumors (e.g. nasopharyngeal cancer).
source: Medical Mission Hills.