I-Acadia Trofinetide Phase III Imiphumela Yomtholampilo Ephezulu Emihle

Ngo-2021-12-06, isikhathi sase-US, i-Acadia Pharmaceuticals (Nasdaq: ACAD) yamemezela imiphumela emihle ephezulu yokuhlolwa kwayo komtholampilo kweSigaba III sekhandidethi layo lezidakamizwa, i-Trofinetide.Uhlolo lwesigaba III, olubizwa nge-Lavender, lusetshenziswa kakhulu ukuhlola ukuphepha nokusebenza kwe-Trofinetide ekwelapheni i-Rett syndrome (RS).Kwabhaliswa izifundo ezingu-189, bonke abesifazane abaneminyaka engu-5-20 abane-RS.

I-Lavender yayiyisivivinyo esiphindwe kabili esiyimpumputhe, esingahleliwe, esilawulwa yi-placebo samasonto e-12 neziphetho eziyinhloko ezihlanganisiwe ze-RS Behavioral Questionnaire (RSBQ) kanye ne-Clinical Outcome Global Rating Scale (CGI-I), njengoba ihlolwe ngabasebenzi abahlengikazi nodokotela, ngokulandelana;ukhiye Isiphetho sesibili Isilinganiso Sokuthuthukiswa Kokuziphatha Okungokomfanekiso Kwezinsana Nezingane Ezisacathula (CSBS-DP-IT-Social), esisetshenziselwa kakhulu ukuhlola ukuthuthukiswa kokuziphatha komphakathi, ngamazwi, nezingokomfanekiso ezinsaneni nasezinganeni ezincane 6–24 izinyanga zeminyaka yobudala, futhi ingasetshenziselwa i-autism Ukuhlolwa kusenesikhathi kwezimpawu zokubambezeleka kokukhula nokunye ukubambezeleka kwentuthuko, njengoba kuhlolwa abasebenzi abahlengikazi.

Imiphumela ibonise ukuthi i-Trofinetide ibe nokuthuthuka okuphawulekayo kuzo zombili iziphetho eziyinhloko uma kuqhathaniswa ne-placebo.Izinguquko kusukela kwesisekelo ku-RSBQ ye-placebo ne-Trofinetide Evikini le-12 zazingu- -1.7 vs -5.1 (p=0.0175);Izikolo ze-CGI-I zazingu-3.8 vs 3.5 (p=0.0030).Ngaleso sikhathi, uguquko olusuka kusisekelo ku-CSBS-DP-IT-Social lwaluthi -1.1 kanye -0.1 lwe-placebo ne-Trofinetide, ngokulandelana.

Kokubili iziphetho eziyinhloko neziyisisekelo ze-Lavender zibonise amandla e-Trofinetide ekwelapheni i-RS, nokho-ke, kufanele kuqashelwe ukuthi izinga lokuyeka ukwelashwa ocwaningweni okuhlobene nezehlakalo ezimbi ezivelayo (TEAEs) laliphezulu engalweni ye-Trofinetide uma kuqhathaniswa ne-placebo. , Laba bobabili bebengu-2.1% no-17.2%, ngokulandelana.Phakathi kwazo, izenzakalo ezimbi ezivame kakhulu zaziyizi:

① I-Diarrhea - i-Trofinetide yayingu-80.6% (97.3% yakhona eyayithambile ukuya emaphakathi) futhi i-placebo yayingu-19.1%;

② Ukuphalaza - i-Trofinetide yayingu-26.9% (96% yakhona eyayithambile ukuya emaphakathi) kanye ne-placebo yayingu-9.6%;

③ Izehlakalo ezimbi kakhulu zenzeke ku-3.2% wezifundo kuwo womabili amaqembu.

Izifundo ocwaningweni lwe-Lavender zizoqhubeka nokuthola i-Trofinetide ngemva kokuphothula ukuhlola noma kuzifundo zokwandisa ilebula ye-Lilac ne-Lilac-2, futhi >95% wezifundo eziqede ucwaningo lwe-Lavender zikhethe ukushintshela kulebula evulekile ye-Lilac. ukwanda Ucwaningo, okutholakele kuzokwethulwa emhlanganweni wezokwelapha ozayo.