Acadia Trofinetide Phase III Fa'afoma'i Fa'afoma'i Tu'u-Line I'uga Lelei

I le 2021-12-06, US taimi, Acadia Pharmaceuticals (Nasdaq: ACAD) na faʻasalalau faʻaiʻuga lelei pito i luga o lona Vaega III faʻataʻitaʻiga faʻataʻitaʻiga o lona sui tauva, Trofinetide.O le vaega III faʻataʻitaʻiga, e taʻua o Lavender, e masani ona faʻaaogaina e iloilo ai le saogalemu ma le aoga o Trofinetide i le togafitiga o Rett syndrome (RS).E 189 mataupu na lesitalaina, o tamaitai uma e 5-20 tausaga ma le RS.

Lavender o se faʻataʻitaʻiga lua-tauaso, faʻavasegaina, placebo-pulea faʻataʻitaʻiga o 12 vaiaso faʻatasi ai ma tuʻufaʻatasiga tuʻufaʻatasia autu o le RS Behavioral Questionnaire (RSBQ) ma le Clinical Outcome Global Rating Scale (CGI-I), e pei ona iloiloina e le aufaigaluega tausi maʻi ma fomaʻi, i le faasologa;key O le pito lona lua o le Fesoʻotaʻiga ma Faʻatusa Faʻataʻitaʻiga Fua Faʻatatau mo Pepe ma Tamaiti (CSBS-DP-IT-Social), lea e masani ona faʻaaogaina e iloilo ai le atinaʻeina o amioga faʻaagafesootai, tautala, ma faʻatusa i pepe ma tamaiti laiti 6-24 masina le matua, ma e mafai ona fa'aoga mo autism Su'esu'e vave mo fa'ailoga o le tuai o le atina'e ma isi fa'atuai o le atina'e, e pei ona iloiloina e le aufaigaluega tausi soifua.

O fa'ai'uga na fa'aalia ai o le Trofinetide na i ai ni fa'alelei tele i itu taua e lua fa'atusatusa ile placebo.Suiga mai le laina faavae ile RSBQ mo placebo ma Trofinetide ile Vaiaso 12 ole -1.7 vs -5.1 (p=0.0175);O togi CGI-I e 3.8 vs 3.5 (p=0.0030).I le taimi nei, o le suiga mai le laina faavae i le CSBS-DP-IT-Social o le -1.1 ma le -0.1 mo placebo ma Trofinetide, i le faasologa.

O pito muamua ma tulaga lua lua o Lavender na faʻaalia ai le gafatia o Trofinetide mo le togafitiga o le RS, peitaʻi, e tatau ona maitauina o le fua faatatau o suʻesuʻega togafitiga faʻagata e fesoʻotaʻi ma faʻalavelave faʻafuaseʻi (TEAEs) na maualuga atu i le Trofinetide lima pe a faʻatusatusa i le placebo. , O le lua e 2.1% ma le 17.2%, i le faasologa.Faatasi ai ma i latou, o mea leaga sili ona taatele o:

① Matātā – Trofinetide o le 80.6% (97.3% o ia mea e malu i le feololo) ma le placebo e 19.1%;

② Suʻi - Trofinetide o le 26.9% (96% o ia mea e laʻititi i le feololo) ma le placebo e 9.6%;

③ Fa'alavelave matuia na tutupu i le 3.2% o mataupu i vaega uma e lua.

O mataupu i le Lavender trial o le a faʻaauau pea ona maua Trofinetide pe a maeʻa le faʻataʻitaʻiga poʻo le faʻailoga tatala Lilac ma Lilac-2 suʻesuʻega faʻalauteleina, ma> 95% o mataupu na maeʻa suʻesuʻega Lavender na filifili e sui i le Lilac tatala-igoa. fa'alautele Su'esu'ega, o le a tu'uina atu su'esu'ega i se fono fa'afoma'i a sau.