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Acadia Trofinetide Phase III Clinical Top-Line Results Ezi

Na 2021-12-06, oge US, Acadia Pharmaceuticals (Nasdaq: ACAD) kwupụtara nsonaazụ kacha mma nke nnwale ụlọ ọgwụ Phase III nke onye nyocha ọgwụ ya, Trofinetide.A na-ejikarị ule nke III, nke a na-akpọ Lavender, iji nyochaa nchekwa na ịdị irè nke Trofinetide na ọgwụgwọ Rett syndrome (RS).Ngụkọta nke isiokwu 189 ka edebara aha, ụmụ nwanyị niile dị afọ 5-20 nwere RS.

Lavender bụ onye kpuru ìsì okpukpu abụọ, nke a na-achịkwaghị achịkwa, nke na-achịkwa placebo nke izu 12 na-ejikọta isi njedebe nke RS Behavioral Questionnaire (RSBQ) na Clinical Outcome Global Rating Scale (CGI-I), dị ka ndị ọrụ nọọsụ na ndị dọkịta na-enyocha, n'otu n'otu;isi Ihe njedebe nke abụọ bụ Nkwukọrịta na Nkwukọrịta Omume Mmepe maka Ụmụaka na Ụmụaka (CSBS-DP-IT-Social), bụ nke a na-ejikarị enyocha mmepe nke mmekọrịta mmadụ na ibe, okwu ọnụ na ihe atụ na ụmụ ọhụrụ na ụmụaka 6-24. ọnwa nke afọ, na enwere ike iji ya mee ihe maka nyocha mbụ nke autism maka mgbaàmà nke igbu oge mmepe na oge mmepe ndị ọzọ, dị ka ndị ọrụ nọọsụ na-enyocha.

Nsonaazụ gosiri na Trofinetide nwere mmụba dị ukwuu na isi njedebe abụọ bụ isi ma e jiri ya tụnyere placebo.Mgbanwe site na ntọala na RSBQ maka placebo na Trofinetide na Week 12 bụ -1.7 vs -5.1 (p=0.0175);Akara CGI-I bụ 3.8 vs 3.5 (p=0.0030).Ka ọ dị ugbu a, mgbanwe sitere na ntọala na CSBS-DP-IT-Social bụ -1.1 na -0.1 maka placebo na Trofinetide, n'otu n'otu.

Ma isi na isi nke abụọ nke Lavender gosipụtara ikike nke Trofinetide maka ọgwụgwọ RS, Otú ọ dị, ekwesịrị iburu n'obi na ọnụ ọgụgụ nke nkwụsị ọgwụgwọ ọmụmụ metụtara ihe omume ọjọọ (TEAEs) dị elu na ogwe aka Trofinetide ma e jiri ya tụnyere placebo. , Ha abụọ bụ 2.1% na 17.2%, n'otu n'otu.N'ime ha, ihe ọjọọ ndị a na-ahụkarị bụ:

① Ọrịa afọ ọsịsa - Trofinetide bụ 80.6% (97.3% nke dị nro na agafeghị oke) na placebo bụ 19.1%;

② Vomiting - Trofinetide bụ 26.9% (96% n'ime ha dị nro na-agafeghị oke) na placebo bụ 9.6%;

③ Ihe ọjọọ dị njọ mere na 3.2% nke isiokwu dị na otu abụọ ahụ.

Ihe ndị dị na ule Lavender ga-aga n'ihu na-enweta Trofinetide mgbe emechara ikpe ahụ ma ọ bụ na-emeghe-labelụ Lilac na Lilac-2 ọmụmụ mgbasawanye, na> 95% nke isiokwu ndị dechara Lavender ọmụmụ họọrọ ịgbanwe gaa na Lilac open-label. Nnyocha mgbasawanye, a ga-ewepụta nchoputa na nzukọ ahụike na-abịa.

Trofinetide


Oge nzipu: Feb-17-2022