ʻO Acadia Trofinetide Phase III ʻO nā hualoaʻa maikaʻi

Ma 2021-12-06, US manawa, ua hoʻolaha ʻo Acadia Pharmaceuticals (Nasdaq: ACAD) i nā hopena hopena maikaʻi loa o kāna hoʻokolohua lapaʻau Phase III o kāna moho lāʻau, Trofinetide.Hoʻohana nui ʻia ka hoʻāʻo ʻana o ka pae III, i kapa ʻia ʻo Lavender, e loiloi i ka palekana a me ka pono o Trofinetide i ka mālama ʻana i ka Rett syndrome (RS).He 189 ka nui o na kumuhana i kakau inoa ia, o na wahine a pau mai 5-20 makahiki me RS.

ʻO Lavender kahi hoʻāʻo ʻelua makapō, randomized, placebo-controlled trial o 12 wiki me ka hui pū ʻana i nā helu hope o ka RS Behavioral Questionnaire (RSBQ) a me Clinical Outcome Global Rating Scale (CGI-I), e like me ka loiloi ʻia e nā limahana kahu hānai a me nā kauka, kēlā me kēia;kī ʻO ka helu hope ʻelua ʻo ka Communication and Symbolic Behavior Development Scale for Infants and Toddlers (CSBS-DP-IT-Social), i hoʻohana nui ʻia e loiloi i ka hoʻomohala ʻana o nā ʻano pilikanaka, waha, a me nā ʻano hōʻailona i nā pēpē a me nā keiki liʻiliʻi 6–24 mahina o ka makahiki, a hiki ke hoʻohana ʻia no ka maʻi autism ʻO ka nānā mua ʻana no nā hōʻailona o ka lohi o ka ulu ʻana a me nā lohi ulu ʻē aʻe, e like me ka loiloi ʻana e nā limahana kahu.

Ua hōʻike ʻia nā hopena he hoʻomaikaʻi koʻikoʻi ʻo Trofinetide ma nā ʻaoʻao hope ʻelua i hoʻohālikelike ʻia me ka placebo.ʻO nā hoʻololi mai ka papa kuhikuhi ma RSBQ no kahi placebo a me Trofinetide i ka Hepetoma 12 he -1.7 vs -5.1 (p=0.0175);ʻO nā helu CGI-I he 3.8 vs 3.5 (p=0.0030).I kēia manawa, ʻo ka hoʻololi ʻana mai ka baseline ma CSBS-DP-IT-Social he -1.1 a me -0.1 no kahi placebo a me Trofinetide.

Ua hōʻike ʻia nā ʻaoʻao mua a me ke kula kiʻekiʻe o Lavender i ka hiki o Trofinetide no ka mālama ʻana iā RS, akā naʻe, pono e hoʻomaopopo ʻia ʻoi aku ka kiʻekiʻe o ka nui o ka hoʻopau ʻana i ka hoʻopaʻa ʻana e pili ana i nā hanana ʻino (TEAEs) i ka lima Trofinetide i hoʻohālikelike ʻia me ka placebo. , He 2.1% a me 17.2% ka elua.I waena o lākou, ʻo nā mea ʻino loa maʻamau:

① ʻO ka maʻi maʻi maʻi - ʻo Trofinetide he 80.6% (97.3% o ia mau mea maʻalahi a haʻahaʻa) a he 19.1% ka placebo;

② ʻO ka luaʻi - ʻo Trofinetide he 26.9% (96% o ia mau mea maʻalahi a haʻahaʻa) a he 9.6% ka placebo;

③ Ua loaʻa nā hanana pōʻino koʻikoʻi ma 3.2% o nā kumuhana ma nā hui ʻelua.

E hoʻomau ka loaʻa ʻana o Trofinetide i nā kumuhana i ka hoʻokolokolo Lavender ma hope o ka pau ʻana o ka hoʻāʻo ʻana a i ʻole i nā haʻawina hoʻonui ʻia ʻo Lilac a me Lilac-2, a >95% o nā kumuhana i hoʻopau i ka haʻawina Lavender i koho e hoʻololi i ka Lilac open-label. hoʻonui ʻia ʻO ka noiʻi, e hōʻike ʻia nā ʻike i kahi hālāwai olakino e hiki mai ana.