Isigaba se-Acadia Trofinetide Isigaba III imiphumela ye-top-line ephezulu

Ngo-2021-12-06, isikhathi sase-US, i-Acarmaceuticals (NASDAQ: acad) imemezele imiphumela emihle yomugqa ophezulu wesigaba se-III sesigaba i-III Chrinecty Chrimity of ukhetho lwezidakamizwa, i-trofofetide. Isivivinyo sesigaba III, esibizwa ngeLavender, sisetshenziselwa ikakhulukazi ukuhlola ukuphepha nokusebenza kahle kwe-trofonetide ekwelashweni kweRetT Syndrome (RS). Kwabhalise izifundo eziyi-189, bonke abesifazane abaneminyaka engu-5-20 bene-Rs.

ILavender yayiyimpumputhe emibili, engahleliwe, i-placebo-elawulwa amasonto ayi-12 ngama-Endpoition ahlanganisiwe we-RS Bearity Newnaire (RSBQ) kanye nomphumela wemitholampilo womhlaba (CGI-I), njengoba kuhlolwe abahlengikazi kanye nodokotela, ngokulandelana; Ukhiye Endpoint yesibili iyisilinganiso sokuthuthuka nokuziphatha okungokomfanekiso kwezingane kanye nezingane (CSBS-DP-IT-Social), futhi zingasetshenziselwa ukuhlolwa kusenesikhathi kwezimpawu zokubambezeleka kwezentuthuko kanye nokunye ukubambezeleka kwabahlengikazi, njengoba kuhlolwa abahlengikazi.

Imiphumela ikhombisile ukuthi i-trofinetide yayinokuthuthuka okuphambili kuzona zombili izindlela zokugcina ziqhathaniswa ne-placebo. Izinguquko ezivela kusisekelo ku-RSBQ ye-placebo ne-trofonetide ngeviki 12 zazingu -1.7 vs -5.1 (p = 0.0175); Izikolo ze-CGI-i zazingama-3.8 vs 3.5 (p = 0.0030). Okwamanje, ushintsho olusuka kwisisekelo ku-CSBS-DP-IT-Social lwalungu -1.1 no -0.1 lwe-placebo kanye ne-trofofetide, ngokulandelana.

Zombili izindawo zokugcina ezisezingeni eliphakeme neziyisisekelo se-lavender zikhombise amandla e-trofonetide yokwelashwa kwama-Rs, kepha kufanele kuphawulwe ukuthi izinga lokuncishiswa kokwelashwa okuvela ku-trofinetide averal ephathelene ne-profofetide ingalo ye-trofinetide, lawo amabili ayengu-17,2%, ngokulandelana. Phakathi kwazo, izehlakalo ezimbi kakhulu ezingezinhle yilezi:

① Isifo sohudo - i-trofinetide kwakungama-80.6% (97.3% abumnene ukuze alinganise) futhi i-placebo yayingu-19,1%;

② Ukuhlanza - I-Trofinetide kwakungu-26.9% (96% wawo ayemnene ukuze alinganise) futhi i-placebo yayingu-9.6%;

③ Imicimbi emibi kakhulu yenzeke ku-3.2% yezifundo kuwo womabili amaqembu.

Izihloko esivivinyweni se-lavender zizoqhubeka nokuthola i-trofonetide ngemuva kokuphothulwa kwecala noma izifundo ezivulekile zeLilac naseLilac-2 yezihloko eziphothule ucwaningo lwe-lavender oluvulekile ukuguqula ucwaningo lwezokwelashwa oluvulekile, okutholakele.