I-Acadia Trofinetide iSigaba III III Iziphumo zeklinikhi ephezulu

On 2021-12-06, US time, Acadia Pharmaceuticals (Nasdaq: ACAD) announced the positive top-line results of its Phase III clinical trial of its drug candidate, Trofinetide. Ityala lesigaba sesithathu elibizwa ngokuba yiLavender, lisetyenziselwa ukuvavanya ukhuseleko kunye nokusebenza ngempumelelo kweTrofinetide kunyango lwe-ret syndrome (RS). Zizonke izifundo ezili-189 zabhaliswa, bonke abantu basetyhini baneminyaka emi-5 ukuya kwengama-20 ngeRs.

I-lavender yayiyinto engaboniyo, ingena nzulu, ilawulwa yi-placesbhore kwiiveki eziziintloko ze-RS zokuziphatha (i-RSBQ) kunye neziphumo zekliniki Eyona nto iphambili kwisiphelo senqanaba lesibini kukunxibelelana nokuziphatha kweentsana kunye nabantwana abancinci (i-CSBS-DP-I-IT-I-Autism), esetyenziselwa ukufunyanwa kwe-14 ukuya kwintlalo-ntle ye-Autis, kwaye isetyenziselwa ukulibazisa, njengoko kuvavanywa ngabasebenzi abaziziseko.

Iziphumo zibonise ukuba i-gooffinide yayinophuculo olubalulekileyo kuzo zombini iindlela zokuphelisa zithelekiswa ne-placebo. Utshintsho oluvela kwi-Spline kwi-psbq ye-placebo kunye neTrofinetide kwiveki ye-12 yayi -1.7 vs -5.1 (P = 0.0175); I-CGI-i-3.8 vs 3.5 (P = 0.0030). Okwangoku, utshintsho ovela kwi-SISLline kwi-CSBS-DP-IT-I-IT-I-IT-I-LE-IT-2000.1 ye-placebo kunye neTrofinetide, ngokulandelelana.

Zombini ezona ndawo ziphambili kunye neziphambili zeLavender zibonisa amandla etronetinetide onyango lwe-Rs, nangona kunjalo, kufuneka iqatshelwe ukuba i-trofrinetive engafaniyo ithelekiswa ne-prefinimede ye-Trofinetive enxulumene ne-preminetide, ezimbini i-2.1%, ngokwahlukeneyo. Phakathi kwazo, ezona ziganeko zibalulekileyo zixhaphakileyo:

I-Haarrrhea - iTrofinettide yayiyi-80.6% (97.3% yayo ilula ukumodareyitha) kunye ne-placebo yayiyi-19.1%;

I-gorating-trofunide yayiyi-26.9% (i-96% yayo inobulali ukumodareyitha) kunye ne-placebo yayiyi-9.6%;

③ Iziganeko ezimbi ezibi zenzeka kwi-3.2% yezifundo kuwo omabini amaqela.

Izifundo kwi-lavender ziya kuqhubeka nokufumana i-trofrinetide emva kokugqitywa kwetyala okanye kwi-ilokhwe evulekileyo ye-Lilac kunye ne-Ilac-2 yokwandisa i-LILAC inyule ukutshintshela kwintlanganiso evulekileyo yeLevender, iziphumo ziya kuziswa kwintlanganiso yezonyango evulekileyo.