Acadia Trofinetide Phase III Clinical Top-Line Zotsatira Zabwino

Pa 2021-12-06, nthawi yaku US, Acadia Pharmaceuticals (Nasdaq: ACAD) adalengeza zotsatira zabwino zapamwamba za mayeso ake azachipatala a Phase III omwe adafuna kumwa mankhwala, Trofinetide.Mayesero a Gawo III, otchedwa Lavender, amagwiritsidwa ntchito makamaka poyesa chitetezo ndi mphamvu ya Trofinetide pochiza matenda a Rett (RS).Ophunzira 189 adalembetsa, azimayi onse azaka zapakati pa 5-20 ndi RS.

Lavender anali mayesero awiri akhungu, osasinthika, omwe amayendetsedwa ndi placebo kwa masabata a 12 ndi mapeto ophatikizana a RS Behavioral Questionnaire (RSBQ) ndi Clinical Outcome Global Rating Scale (CGI-I), monga momwe anayesedwa ndi anamwino ogwira ntchito ndi madokotala, motsatira;fungulo Mapeto achiwiri ndi Communication and Symbolic Behavior Development Scale for Infants and Toddlers (CSBS-DP-IT-Social), yomwe imagwiritsidwa ntchito makamaka poyesa kukula kwa chikhalidwe cha anthu, mawu, ndi machitidwe ophiphiritsa mwa makanda ndi ana aang'ono 6-24 miyezi yakubadwa, ndipo angagwiritsidwe ntchito kwa Autism Kuwunika koyambirira kwa zizindikiro za kuchedwa kwachitukuko ndi kuchedwa kwina kwachitukuko, monga momwe amawunikiridwa ndi ogwira ntchito ya unamwino.

Zotsatira zinawonetsa kuti Trofinetide inali ndi kusintha kwakukulu m'mapeto oyambilira onse kuyerekeza ndi placebo.Zosintha kuchokera pazoyambira mu RSBQ za placebo ndi Trofinetide pa Sabata 12 zinali -1.7 vs -5.1 (p=0.0175);Zotsatira za CGI-I zinali 3.8 vs 3.5 (p=0.0030).Panthawiyi, kusintha kuchokera ku chiyambi mu CSBS-DP-IT-Social kunali -1.1 ndi -0.1 kwa placebo ndi Trofinetide, motsatira.

Onse oyambirira ndi oyambirira sekondale mapeto a Lavender anasonyeza kuthekera kwa Trofinetide pa chithandizo cha RS, komabe, ziyenera kudziwidwa kuti mlingo wa kusiya mankhwala ophunzirira zokhudzana ndi zochitika zowonongeka (TEAEs) unali wapamwamba mu mkono wa Trofinetide poyerekeza ndi placebo. , Awiriwo anali 2.1% ndi 17.2%, motero.Zina mwa izo, zochitika zodziwika kwambiri zinali:

① Kutsekula m'mimba - Trofinetide inali 80.6% (97.3% yomwe inali yochepa mpaka yochepa) ndipo placebo inali 19.1%;

② Kusanza - Trofinetide inali 26.9% (96% yomwe inali yochepa mpaka yochepa) ndipo placebo inali 9.6%;

③ Zochitika zoyipa kwambiri zidachitika mu 3.2% ya maphunziro m'magulu onse awiri.

Anthu omwe ali mumyeso wa Lavender apitilizabe kulandira Trofinetide akamaliza kuyesako kapena m'maphunziro okulitsa a Lilac ndi Lilac-2, ndipo > 95% mwa anthu omwe adamaliza maphunziro a Lavender adasankha kusinthira ku Lilac open-label. kukulitsa Kafukufuku, zomwe zapezedwa zidzaperekedwa ku msonkhano wachipatala womwe ukubwera.