Acadia Trofinetide Phase III Clinical Yepamusoro-Mutsara Migumisiro Yakanaka

Pana 2021-12-06, nguva yeUS, Acadia Pharmaceuticals (Nasdaq: ACAD) yakazivisa mhedzisiro yakanaka yepamusoro-yepamusoro-soro yemuyedzo wekiriniki wePhase III yemumiriri wezvinodhaka, Trofinetide.Muyedzo wechikamu chechitatu, unonzi Lavender, unonyanya kushandiswa kuongorora kuchengetedzeka uye kushanda kweTrofinetide mukurapa kweRett syndrome (RS).Zvidzidzo zve189 zvakanyoreswa, vakadzi vese vane makore 5-20 vane RS.

Lavender yakanga iri muedzo wakapetwa kaviri-mapofu, randomized, placebo-controlled controlled mavhiki e12 pamwe chete nemagumo ekupedzisira eRS Behavioral Questionnaire (RSBQ) uye Clinical Outcome Global Rating Scale (CGI-I), sezvakaongororwa nevakoti vashandi nevarapi, maererano;kiyi Chikamu chechipiri cheKukurukurirana uye Chiratidzo cheKuvandudza Mafambiro eVacheche neVacheche (CSBS-DP-IT-Social), iyo inonyanya kushandiswa kuongorora kukura kwemagariro, kutaura, uye maitiro ekufananidzira mucheche nevana vaduku 6-24. mwedzi yezera, uye inogona kushandiswa kune autism Kuongororwa kwekutanga kwezviratidzo zvekukura kunonoka uye kumwe kunonoka kwekukura, sekuongororwa nevashandi vehukoti.

Mhedzisiro yacho yakaratidza kuti Trofinetide yaive nekuvandudzwa kwakakura mune ese ekutanga ekupedzisira zvichienzaniswa ne placebo.Shanduko kubva kune yekutanga muRSBQ ye placebo neTrofinetide paSvondo 12 yaive -1.7 vs -5.1 (p=0.0175);CGI-I zvibodzwa zvaive 3.8 vs 3.5 (p=0.0030).Zvichakadaro, shanduko kubva kune yekutanga muCSBS-DP-IT-Social yaive -1.1 uye -0.1 ye placebo neTrofinetide, zvichiteerana.

Zvose zvekutanga uye zvekutanga zvekupedzisira zvekupedzisira zveLavender zvakaratidza kukwanisa kweTrofinetide pakurapa RS, zvisinei, zvinofanira kuonekwa kuti chiyero chekurega kurapwa kwekudzidza kune chokuita nezviitiko zvinowanzoitika zvakashata (TEAEs) yakanga yakakwirira muTrofinetide ruoko kana ichienzaniswa ne placebo. , Vaviri vaiva 2.1% uye 17.2%, maererano.Pakati pavo, zviitiko zvinowanzoitika zvakashata ndezvi:

① Diarrhea - Trofinetide yaiva 80.6% (97.3% yaive yakapfava kusvika pakati) uye placebo yaiva 19.1%;

② Kurutsa - Trofinetide yaiva 26.9% (96% iyo yakanga iri munyoro kusvika pakati) uye placebo yaiva 9.6%;

③ Zviitiko zvakakomba zvakashata zvakaitika mu3.2% yezvidzidzo mumapoka ese ari maviri.

Vanodzidza muyedzo yeLavender vacharamba vachiwana Trofinetide mushure mekupedza kutongwa kana muzvidzidzo zvekuwedzera Lilac neLilac-2, uye >95% yevadzidzi vakapedza chidzidzo cheLavender vakasarudza kuchinjira kuLilac open-label. kuwedzera Tsvagiridzo, zvakawanikwa zvichaunzwa kumusangano wekurapa unouya.