I-Acadia Trofinetide iSigaba sesi-III seziphumo zoNyango oluPhezulu oluPhakamileyo

Ngo-2021-12-06, ixesha lase-US, i-Acadia Pharmaceuticals (Nasdaq: ACAD) yabhengeza iziphumo ezilungileyo zodidi oluphezulu lweSigaba sesi-III solingo lwezonyango lomgqatswa wayo wechiza, iTrofinetide.Ulingo lwesigaba III, olubizwa ngokuba yiLavender, lusetyenziswa ikakhulu ukuhlola ukhuseleko nokusebenza kweTrofinetide kunyango lweRett syndrome (RS).Itotali yezifundo ze-189 zabhaliswa, bonke abafazi abaneminyaka eyi-5-20 iminyaka kunye neRS.

I-Lavender yayiyi-double-blind blind, i-randomized, i-placebo-controlled controlled trial of 12 weeks with dised primary endpoints of the RS Behavioral Questionnaire (RSBQ) kunye ne-Clinical Outcome Global Rating Scale (CGI-I), njengoko ihlolwe ngabasebenzi abahlengikazi kunye noogqirha, ngokulandelanayo;isitshixo Isiphelo sesibini siSikali soNxibelelwano kunye neSimbo soPhuhliso lweSimbo kwiintsana kunye nabantwana abancinci (CSBS-DP-IT-Social), esetyenziselwa kakhulu ukuvavanya ukuphuhliswa kwentlalontle, intetho, kunye nokuziphatha okungokomfanekiso kwiintsana kunye nabantwana abancinci 6-24 iinyanga ubudala, kwaye ingasetyenziselwa Autism Ukuhlolwa kwangethuba iimpawu zokulibaziseka kuphuhliso kunye nolunye ulibaziseko zophuhliso, njengoko kuhlolwe ngabasebenzi abongikazi.

Iziphumo zabonisa ukuba i-Trofinetide ibe nophuculo olubalulekileyo kuzo zombini iziphelo eziphambili xa kuthelekiswa ne-placebo.Utshintsho olusuka kwisiseko kwi-RSBQ ye-placebo kunye ne-Trofinetide kwiVeki ye-12 yaba -1.7 vs -5.1 (p=0.0175);Amanqaku eCGI-I ayeyi-3.8 vs 3.5 (p=0.0030).Okwangoku, utshintsho olusuka kwisiseko kwi-CSBS-DP-IT-Social yayingu-1.1 kunye -0.1 ye-placebo kunye ne-Trofinetide, ngokulandelanayo.

Zombini iziphelo eziphambili neziziiprayimari eziziisekondari zeLavender zibonise amandla eTrofinetide kunyango lwe-RS, nangona kunjalo, kufuneka kuqatshelwe ukuba izinga lokuyeka unyango lokufunda olunxulumene neziganeko ezimbi ezivelayo (TEAEs) zaziphezulu kwingalo yeTrofinetide xa kuthelekiswa ne-placebo. , Ezi zimbini zaziyi-2.1% kunye ne-17.2%, ngokulandelanayo.Phakathi kwazo, ezona ziganeko zibi zixhaphake kakhulu zezi:

① I-Diarrhea - i-Trofinetide yayingu-80.6% (i-97.3% yayo yayincinci ukuya kumodareyitha) kunye ne-placebo yi-19.1%;

② Ukuhlanza - i-Trofinetide yayiyi-26.9% (i-96% yayo yayincinci ukuya kumodareyitha) kunye ne-placebo yi-9.6%;

③ Iziganeko ezimbi kakhulu zenzeke kwi-3.2% yezifundo kuwo omabini amaqela.

Izifundo kulingo lweLavender ziyakuqhubeka nokufumana iTrofinetide emva kokugqitywa kolingo okanye kwileyibhile evulekileyo yezifundo zokwandiswa kweLilac kunye neLilac-2, kwaye > 95% yezifundo ezigqibe isifundo seLavender zikhethe ukutshintshela kwi-Lilac open-label. ukwanda Uphando, iziphumo ziya kuboniswa kwintlanganiso yezonyango ezayo.