Acadia Trofinetide Icyiciro cya III Clinical Top-Line Ibisubizo Byiza

Ku ya 2021-12-06, ku isaha yo muri Amerika, Acadia Pharmaceuticals (Nasdaq: ACAD) yatangaje ibisubizo byiza byo mu cyiciro cya gatatu cy’iburanisha ry’amavuriro y’umukandida w’ibiyobyabwenge, Trofinetide.Ikigeragezo cya III, cyitwa Lavender, gikoreshwa cyane mugusuzuma umutekano ningirakamaro bya Trofinetide mukuvura syndrome ya Rett (RS).Hiyandikishije amasomo 189, abagore bose bafite imyaka 5-20 hamwe na RS.

Lavender yari impumyi ebyiri, itabigenewe, igenzurwa na platbo yibyumweru 12 hamwe nibisobanuro byibanze byibibazo bya RS Imyitwarire (RSBQ) hamwe na Clinical Outcome Global Rating Scale (CGI-I), nkuko byasuzumwe n'abakozi b'abaforomo n'abaganga;urufunguzo Iherezo rya kabiri ni Itumanaho n’itumanaho ryerekana iterambere ry’impinja n’abana bato (CSBS-DP-IT-Imibereho), rikoreshwa cyane cyane mu gusuzuma iterambere ry’imyitwarire mbonezamubano, imvugo, n’ikigereranyo ku mpinja n’abana bato 6–24 amezi y'amavuko, kandi irashobora gukoreshwa muri autism Kwisuzumisha hakiri kare ibimenyetso byerekana gutinda kwiterambere nibindi bidindiza iterambere, nkuko byasuzumwe nabakozi baforomo.

Ibisubizo byerekanaga ko Trofinetide yagize iterambere ryinshi mubice byombi byibanze ugereranije na placebo.Impinduka kuva kuri baseline muri RSBQ kuri placebo na Trofinetide mucyumweru cya 12 byari -1.7 vs -5.1 (p = 0.0175);Amanota ya CGI-I yari 3.8 vs 3.5 (p = 0.0030).Hagati aho, impinduka ziva muri CSBS-DP-IT-Imibereho yari -1.1 na -0.1 kuri placebo na Trofinetide.

Byombi byibanze nibyibanze byanyuma bya Lavender byerekanaga ubushobozi bwa Trofinetide yo kuvura RS, ariko, twakagombye kumenya ko igipimo cyo guhagarika imiti yo kwiga kijyanye nibibazo bivuka (TEAEs) cyari kinini mumaboko ya Trofinetide ugereranije na placebo , Byombi byari 2,1% na 17.2%.Muri byo, ibintu byakunze kugaragara ni:

Indwara y'impiswi - Trofinetide yari 80,6% (97.3% muri yo yari yoroheje kandi igereranije) naho umwanya wa 19.1%;

Kuruka - Trofinetide yari 26.9% (96% muri yo yari yoroheje kandi igereranije) naho umwanya wa 9,6%;

Ibyabaye bibi byabaye muri 3.2% byamasomo mumatsinda yombi.

Ibintu biri mubigeragezo bya Lavender bizakomeza kwakira Trofinetide nyuma yo kurangiza urubanza cyangwa mumasomo afunguye Lilac na Lilac-2, kandi> 95% byamasomo yarangije kwiga Lavender bahisemo kwimukira kuri Lilac ifunguye-label kwagura Ubushakashatsi, ibisubizo bizashyikirizwa inama yubuvuzi iri hafi.