Bristol-Myers mob plawv myosin allosteric inhibitor mavacamten nkag hauv kev txheeb xyuas hauv Tebchaws Asmeskas!

Bristol-Myers Squibb (BMS) tsis ntev los no tshaj tawm tias Tsoomfwv Meskas Khoom Noj thiab Tshuaj (FDA) tau lees txais daim ntawv thov tshuaj tshiab (NDA) rau mavacamten, ib qho tshiab, lub qhov ncauj, tag nrho cov kev hloov kho ntawm lub plawv myosin rau kev kho mob Cov neeg mob uas muaj tus mob hypertrophic cardiomyopathy ( oHCM), uas yog ib qho kev mob plawv ntev ntev uas muaj qhov xwm txheej siab. Lub FDA tau teem lub hom phiaj hnub tim rau Daim Ntawv Qhia Tshuaj Siv Tshuaj (PDUFA) li Lub Ib Hlis 28, 2022.

mavacamten yog thawj qhov-hauv-chav, qhov ncauj, allosteric myosin inhibitor rau kev kho mob ntawm cov kab mob uas inherently tshwm sim los ntawm kev mob plawv ntau dhau thiab ua rau lub plawv tsis zoo. Mavacamten yog xav txo qhov kev cog lus myocardial los ntawm kev txwv tsis pub tsim ntau dhau ntawm myosin-actin hla kev txuas hniav. Qhov tsim ntawm ntau dhau ntawm myosin-actin cross choj tuaj yeem ua rau muaj kev cuam tshuam ntau myocardial, sab laug ventricular hypertrophy thiab txo kev ua raws. Hauv cov kev tshawb fawb hauv chav kho mob thiab preclinical, mavacamten tseem ua qhia txog biomarkers uas txo cov kev ntxhov siab hauv lub plawv, txo ntau cov myocardial contraction, thiab nce diastolic ua raws.

Thaum Lub Xya Hli 2020, Asmeskas FDA tau tso cai mavacamten kev tsim tshuaj yaj yeeb tshuaj rau oHCM. mavacamten tau tsim tawm los kho cov kev puas siab ntsws mob plawv (oHCM). Raws li cov txheej txheem ntawm kev nqis tes ua thiab cov pov thawj ntawm kev ua haujlwm kho mob, mavacamten tseem tab tom tshawb fawb txog kev kho mob rau cov kev mob tshwm sim tsis cuam tshuam hypertrophic cardiomyopathy (HCM) thiab lub plawv tsis ua haujlwm nrog rau kev khaws cia ua feem (HFpEF).

Kev xa tawm ntawm mavacamten NDA raws li kev soj ntsuam ntawm qhov tseem ceeb Phase 3 EXPLORER-HCM mus sib hais. Kev sib hais tau ua rau cov neeg mob oHCM thiab piv rau mavacamten nrog cov tshuaj placebo. Cov txiaj ntsig pom tau pom tias mavacamten ua rau pom qhov ua kom muaj zog, nrog kev kho mob tseem ceeb hauv cov tsos mob, ua haujlwm ntawm lub neej thiab muaj lub neej zoo, nrog rau nws lub peev xwm los daws cov sab laug ventricular outflow kev thaiv kev. Hauv txoj kev kawm EXPLORER-HCM, txhua txoj kev kawm theem pib thiab theem siab tau mus txog qhov tseem ceeb.

Roland Chen, MD, Tus Lwm Thawj Coj ntawm Tsoom Fwm ntawm Txoj Kev Txhim Kho hlab ntsha ntawm Bristol-Myers Squibb tau hais tias:" HCM yog cov mob caj ces tshaj plaws hauv lub siab. Nws yog hom mob ntev, tiv thaiv, thiab nce qib. Cov neeg mob yuav tuaj yeem ua pa thiab kiv taub hau. Cov tsos mob ua rau qaug zog thiab qaug zog, nrog rau mob hnyav, ua lub neej hloov pauv, nrog rau lub plawv tsis ua hauj lwm, tsis tuaj yeem, mob ntshav hlab plawv thiab tuag sai sai. Hnub no FDA tau lees txais mavacamten NDA pub peb muab oHCM cov neeg mob nrog txoj kev ua kom tau zoo. Kev kho mob ntawm HCM yog ib kauj ruam ze dua. Mavacamten yog thawj-hauv chav kawm myosin inhibitor uas tuaj yeem daws qhov teeb meem tsis haum molecular ntawm tus kabmob. Peb tau cog lus rau kev txhawb nqa kev kho mob ntawm HCM cov neeg mob thiab tos ntsoov yuav koom tes nrog FDA."

mavacamten tshuaj qauv

mavacamten (MYK-461) tau tsim los ntawm MyoKardia. Thaum Lub Kaum Hlis 5, 2020, Bristol-Myers Squibb tshaj tawm tias nws yuav kis tau MyoKardia rau USD 13,1 billion nyiaj ntsuab thiab tus nqi them 60%. Thaum Lub Kaum Ib Hlis 17, 2020, Bristol-Myers Squibb tshaj tawm tias kev yuav khoom ntawm MyoKardia tau ua tiav. Qhov kev kis tau no yog Bristol-Myers Squibb' s thib ob ua lag luam loj tshaj plaws tom qab nws tau $ 74 nphom qhov kev tshawb fawb ntawm Celgene hauv 2019.

Nws yog tsim nyog sau cia tias thaum Lub Yim Hli 11, 2020, LianBio, uas tau raug tsim los ntawm lub tuam txhab peev kev nthuav dav kev pab tswv yim, tau tsim los ntawm cov qauv. Tib hnub ntawd, nws tau tshaj tawm ob txoj kev sib koom tes tseem ceeb. Ib qho yog los qhia BridgeBio Pharma' s khoom xa hluav taws xob xa mus rau Tuam Tshoj, thiab lwm qhov Qhov project yog los qhia MyoKardia' s Mavecamten rau Tuam Tshoj.

Bristol-Myers Squibb muaj kev cia siab rau mavacamten. Thaum nws kis tau MyoKardia, lub tuam txhab tshaj tawm tias mavacamten yuav dhau los ua ib tug neeg tho kev tshuaj rau kev kho mob ntawm HCM. Qhov kev lag luam tseem muaj kev cia siab zoo txog kev ua lag luam kev cia siab ntawm mavacamten. Thaum Lub Kaum Ob Hlis 2020, Tshuaj Ntsuas Vantage, lub koom haum tshawb fawb kev lag luam tshuaj, tso" Top 10 Tshiab Tshuaj Nrog Kev Siv Lag Luam hauv 2021" Hauv cov npe no, mavacamten tau theem thib peb. Txheeb xyuas Vantage kwv yees hais tias mavacamten' s thoob ntiaj teb kev muag khoom hauv 2026 yuav muaj peev xwm mus txog 2 nphom Asmeskas nyiaj.