Chi-Med's HMPL-523 suav nrog hauv qeb kev kho mob zoo

Chi-Med Pharmaceuticals (HUTCHMED) tsis ntev los no tau tshaj tawm tias Lub Tsev Haujlwm Saib Xyuas Tshuaj Ntsuam Xyuas Tshuaj (CDE) ntawm National Medical Products Administration (NMPA) tau suav nrog HMPL-523 ua cov tshuaj kho mob txhawm rau kho cov kab mob ntev uas tau txais. tsawg kawg yog ib qho kev kho ua ntej. Cov neeg laus uas muaj lub cev tiv thaiv kab mob thrombocytopenia (ITP). HMPL-523 yog ib qho tshiab ntawm qhov ncauj spleen tyrosine kinase (Syk) inhibitor. ITP yog ib qho mob autoimmune los ntshav uas ua rau los ntshav hnyav thiab muaj kev cuam tshuam loj thiab muaj ntau yam cuam tshuam rau cov neeg mob kev noj qab haus huv thiab lub neej zoo.

Cov tshuaj kho mob ntawm Lub Xeev Food and Drug Administration suav nrog cov tshuaj tshiab uas siv rau kev tiv thaiv thiab kho lub neej loj- hem cov kab mob thiab tsis muaj kev tiv thaiv thiab kev kho mob zoo lossis muaj txiaj ntsig zoo hauv kev kho mob piv nrog cov kev kho mob uas twb muaj lawm . Rau ntau yam uas suav nrog hauv cov tshuaj kho mob, cov ntawv thov kev pom zoo raws li txoj cai thiab kev tshuaj xyuas ua ntej thiab daim ntawv thov kev pom zoo tuaj yeem xa tuaj thaum xa daim ntawv thov kev lag luam tshiab.

Kev suav nrog HMPL-523 raws li kev kho kom zoo yog raws li kev txhawb nqa ntawm Phase 1b cov ntaub ntawv kawm. Cov ntaub ntawv cuam tshuam tau raug nthuav tawm raws li kev nthuav qhia ntawm qhov ncauj ntawm 63rd American Society of Hematology (ASH) Lub Rooj Sib Tham Txhua Xyoo thaum Lub Kaum Ob Hlis 2021: Raws li cov ntaub ntawv txiav tawm ntawm lub Cuaj Hlis 30, 2021, tag nrho ntawm 34 tus neeg mob tau raug randomized rau HMPL{{24} }523 Hauv pab pawg kho mob, 11 tus neeg mob tau raug xa mus rau pawg placebo. Ntawm cov no, 11 (68.8 feem pua) ntawm 16 tus neeg mob randomized tau txais kev pom zoo koob tshuaj ntawm 300 mg ib hnub ntawm HMPL-523 nyob rau theem 2 kev sim tshuaj (RP2D) tau teb rau lub sijhawm ob-dig muag ntawm thawj 8 lub lis piam. kawm. Hauv qhov sib piv, tsuas yog 1 (9.1 feem pua) ntawm 11 tus neeg mob randomized tau txais cov placebo teb. Hauv 16-lub lim tiam tom ntej qhib-daim ntawv lo, lwm tus neeg mob tau pib randomized kom tau txais RP2D HMPL-523 teb.

Four patients randomly assigned to receive placebo crossed over to receive RP2D HMPL-523 after the first 8-week double-blind period, and all 4 patients responded. Across the 2 phases of the study, 16 of 20 patients (80 percent ) responded and 8 (40 percent ) achieved durable responses. Response was defined as at least one platelet count Greater than or equal to 50 10E9/L. Durable response was defined as platelet count Greater than or equal to 50 10E9/L on at least 4 of the last 6 visits. 】

ITP yog ib hom kab mob autoimmune uas tshwm sim los ntawm kev tiv thaiv kev puas tsuaj ntawm platelets thiab txo cov platelet ntau lawm. Cov neeg mob tuaj yeem tshwm sim nrog daim tawv nqaij petechiae, purpura, thiab plab hnyuv thiab / lossis urethral mucosal los ntshav. ITP kuj tseem cuam tshuam nrog kev qaug zog (qhia txog li 39 feem pua ​​​​ntawm cov neeg laus nrog ITP) thiab lub neej tsis zoo (xws li kev xav, kev ua haujlwm, thiab kev noj qab haus huv, thiab kev ua haujlwm lossis kev sib raug zoo). Qhov tshwm sim ntawm thawj ITP hauv cov neeg laus yog kwv yees li ntawm 3.3 rau 100,000 cov neeg laus hauv ib xyoos, thiab feem ntau yog 9.5 rau 100,000 cov neeg laus.

HMPL-523 yog ib qho tshiab, xaiv tau zoo, muaj zog me me molecule inhibitor, hais lus, tsom mus rau tus po tyrosine kinase (Syk). Raws li ib qho tseem ceeb ntawm cov protein nyob rau hauv B cell signaling pathway, Syk yog ib tug paub tab kho lub hom phiaj rau ntau yam subtypes B cell lymphomas thiab autoimmune kab mob. Chi-Med tam sim no tuav tag nrho cov cai rau HMPL-523 thoob ntiaj teb.

Txij li thaum platelet kev puas tsuaj ntawm ITP muaj feem xyuam rau Syk- tswj phagocytosis ntawm platelets khi rau Fc receptors, Syk inhibitors yuav yog ib qho kev cog lus ITP.