Acadia Trofinetide Phase III Clinical Top-Line Cov txiaj ntsig zoo

Nyob rau 2021-12-06, Tebchaws Asmeskas lub sijhawm, Acadia Pharmaceuticals (Nasdaq: ACAD) tau tshaj tawm cov txiaj ntsig zoo tshaj plaws ntawm nws Phase III kev soj ntsuam ntawm nws cov neeg siv tshuaj, Trofinetide.Kev sim theem III, hu ua Lavender, feem ntau yog siv los ntsuas kev nyab xeeb thiab kev ua tau zoo ntawm Trofinetide hauv kev kho mob ntawm Rett syndrome (RS).Tag nrho ntawm 189 qhov kev kawm tau cuv npe, txhua tus poj niam hnub nyoog 5-20 xyoo nrog RS.

Lavender yog ob qhov muag tsis pom kev, randomized, kev sim tshuaj placebo ntawm 12 lub lis piam nrog cov ntsiab lus tseem ceeb ntawm RS Cov Lus Nug Txog Tus Cwj Pwm (RSBQ) thiab Clinical Outcome Global Rating Scale (CGI-I), raws li kev soj ntsuam los ntawm cov neeg ua haujlwm saib xyuas neeg mob thiab kws kho mob, feem;Qhov tseem ceeb Qhov kawg thib ob yog Kev Sib Txuas Lus thiab Kev Coj Tus Cwj Pwm Kev Loj Hlob rau Cov Me Nyuam thiab Cov Me Nyuam Me (CSBS-DP-IT-Social), uas yog siv los ntsuas kev loj hlob ntawm kev sib raug zoo, kev hais lus, thiab kev coj cwj pwm hauv cov me nyuam mos thiab cov menyuam yaus hnub nyoog 6-24. Lub hnub nyoog ntawm lub hlis, thiab tuaj yeem siv rau autism Kev kuaj ntxov ntxov rau cov tsos mob ntawm kev loj hlob qeeb thiab lwm yam kev loj hlob qeeb, raws li kev soj ntsuam los ntawm cov neeg ua haujlwm saib xyuas neeg mob.

Cov txiaj ntsig tau pom tias Trofinetide muaj kev txhim kho tseem ceeb hauv ob qho tib si thawj qhov kawg piv rau cov placebo.Cov kev hloov pauv ntawm cov hauv paus hauv RSBQ rau cov placebo thiab Trofinetide ntawm Lub Limtiam 12 yog -1.7 vs -5.1 (p = 0.0175);CGI-I cov qhab nia yog 3.8 vs 3.5 (p = 0.0030).Lub caij no, qhov kev hloov pauv ntawm cov hauv paus hauv CSBS-DP-IT-Social yog -1.1 thiab -0.1 rau cov placebo thiab Trofinetide, feem.

Ob qho tib si thawj thiab thawj theem nrab kawg ntawm Lavender tau pom tias muaj peev xwm ntawm Trofinetide rau kev kho mob ntawm RS, txawm li cas los xij, nws yuav tsum tau muab sau tseg tias tus nqi ntawm kev tshawb fawb kev kho mob cuam tshuam nrog cov xwm txheej tsis zoo tshwm sim (TEAEs) tau siab dua hauv Trofinetide caj npab piv rau cov placebo. , Ob leeg yog 2.1% thiab 17.2%, feem.Ntawm lawv, cov xwm txheej tsis zoo tshaj plaws yog:

① Diarrhea - Trofinetide yog 80.6% (97.3% ntawm cov uas mob me mus rau nruab nrab) thiab cov placebo yog 19.1%;

② ntuav - Trofinetide yog 26.9% (96% ntawm cov uas mob me mus rau nruab nrab) thiab cov placebo yog 9.6%;

③ Cov xwm txheej tsis zoo tshwm sim hauv 3.2% ntawm cov ncauj lus hauv ob pawg.

Cov ntsiab lus hauv Lavender mus sib hais yuav tau txais Trofinetide tom qab ua tiav qhov kev sim lossis hauv cov ntawv qhib Lilac thiab Lilac-2 nthuav dav, thiab> 95% ntawm cov kev kawm uas ua tiav Lavender txoj kev kawm tau xaiv hloov mus rau Lilac qhib-label. Kev nthuav dav Kev Tshawb Fawb, kev tshawb pom yuav raug nthuav tawm rau lub rooj sib tham kho mob yav tom ntej.