Acadia Pharmaceuticals Presents New DAYBUE Clinical Data At The 2024 American Academy Of Neurology Annual Meeting
Acadia Pharmaceuticals Presents New DAYBUE Clinical Data At The 2024 American Academy Of Neurology Annual Meeting
Acadia Pharmaceuticals Inc. (NASDAQ:ACAD) today announced that two oral presentations featuring DAYBUE (trofinetide) findings in Rett syndrome will be presented at the American Academy of Neurology (AAN) 2024 annual meeting, being held this week in Denver, CO.
阿卡迪亞製藥公司（納斯達克股票代碼：ACAD）今天宣佈，將在本週在科羅拉多州丹佛舉行的美國神經病學會（AAN）2024年年會上發表兩份口頭報告，介紹DAYBUE（曲非替德）在雷特綜合徵中的發現。
The presentations include outcomes from the DAFFODIL, a Phase 2/3 open-label study evaluating the safety, tolerability and exploratory efficacy of DAYBUE in girls aged two to four living with Rett syndrome, as well as findings from exit interviews with caregivers of individuals living with Rett syndrome who participated in the LAVENDER, LILAC, LILAC-2 and DAFFODIL studies.
這些演講包括DAFFODIL的結果，這是一項2/3期開放標籤研究，評估了DAYBUE對兩至四歲患有雷特綜合徵的女孩的安全性、耐受性和探索性療效，以及對參與薰衣草、丁香、LILAC-2 和水仙花研究的雷特綜合徵患者護理人員的退出訪談結果。
AAN Oral Presentations:
AAN 口頭演講：
- Oral Presentation 003/Abstract 3540: Trofinetide for the Treatment of Girls Aged Two to Four Years with Rett Syndrome: Final Results from the Open-label DAFFODIL Study, Wednesday, April 17 at 3:54pm MT
- Oral Presentation 008/Abstract 2877: Assessing Experiences with Trofinetide for Rett Syndrome: Interviews with Caregivers of Patients in Open-label Studies, Wednesday, April 17 at 4:54pm MT
- 口頭陳述 003/Abstract 3540：Trofinetide 用於治療患有雷特綜合症的兩到四歲女孩：開放標籤 DAFFODIL 研究的最終結果，美國東部時間 4 月 17 日星期三下午 3:54
- 口頭報告 008/Abstract 2877：評估Trofinetide治療雷特綜合徵的經驗：對開放標籤研究中患者護理人員的訪談，美國東部標準時間4月17日星期三下午 4:54
About Rett Syndrome
關於雷特綜合症
Rett syndrome is a rare, complex, neurodevelopmental disorder that may occur over four stages and affects approximately 6,000 to 9,000 patients in the U.S., with approximately 5,000 patients currently diagnosed according to an analysis of healthcare claims data.1-4 A child with Rett syndrome exhibits an early period of apparently normal development until six to 18 months, when their skills seem to slow down or stagnate. This is typically followed by a duration of regression when the child loses acquired communication skills and purposeful hand use. The child may then experience a plateau period in which they show mild recovery in cognitive interests, but body movements remain severely diminished. As they age, those living with Rett may continue to experience a stage of motor deterioration which can last the rest of the patient's life.5 Rett syndrome is typically caused by a genetic mutation on the MECP2 gene.5 In preclinical studies, deficiency in MeCP2 function has been shown to lead to impairment in synaptic communication, and the deficits in synaptic function may be associated with Rett manifestations.5-7
雷特綜合徵是一種罕見的複雜神經發育障礙，可能分四個階段發生，影響美國約6,000至9,000名患者，根據對醫療索賠數據的分析，目前約有5,000名患者被診斷出來。1-4 患有雷特綜合徵的兒童表現出明顯正常的早期發育期，直到六到十八個月，那時他們的技能似乎減緩或停滯不前。當孩子失去獲得的溝通技巧和有針對性的用手時，通常會有一段時間的回歸。然後，孩子可能會經歷一個停滯期，在此期間，他們的認知興趣略有恢復，但身體動作仍然嚴重減弱。隨着年齡的增長，患有Rett的患者可能會繼續經歷運動衰退階段，該階段可能會持續患者的餘生。5 雷特綜合徵通常由Rett的基因突變引起 MECP2 gene.5 在臨床前研究中，meCP2 功能的缺乏已被證明會導致突觸溝通受損，突觸功能的缺陷可能與 Rett 表現有關。5-7
Symptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities.8 Most Rett patients typically live into adulthood and require round-the-clock care.2,9
雷特綜合徵的症狀還可能包括形成手部刻板印象，例如擰手和拍手以及步態異常。8 大多數雷特患者通常活到成年，需要全天候護理。2,9
About DAYBUE (trofinetide)
關於 DAYBUE（trofinetide）
Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1. The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown. Trofinetide is thought to enhance neuronal synaptic function and morphology. This hypothesis is supported by findings from studies of trofinetide in a methyl-CpG-binding protein 2 gene (Mecp2) mouse model of Rett syndrome, in which increased branching of the dendrites that form synapses and synaptic plasticity signals were observed.10
Trofinetide 是胰島素樣生長因子 1 的 N 末端三肽的合成類似物。trofinetide對雷特綜合徵患者發揮治療作用的機制尚不清楚。Trofinetide被認爲可以增強神經元突觸功能和形態。對甲基-CPG結合蛋白2基因中的曲非內肽的研究結果支持了這一假設（Mecp2) 雷特綜合徵的小鼠模型，在該模型中觀察到形成突觸和突觸可塑性信號的樹突分支增多。10
譯文內容由第三人軟體翻譯。