Anavex是總部位于美國紐約的一家臨床階段的生物制藥公司，專注于開發(fā)差異化的療法用于神經(jīng)退行性疾病和神經(jīng)發(fā)育疾病的治療，包括阿爾茨海默?。ˋD）、Rett綜合征以及其他中樞神經(jīng)系統(tǒng)疾病。

近日，該公司宣布，將在8月份啟動一項IIb/III期臨床試驗，評估新型藥物Anavex 2-73治療早期AD的療效和安全性。該研究是一項雙盲、隨機、安慰劑對照、48周研究，計劃入組450例患者，這些患者將以1:1:1的比例隨機接受2種劑量Anavex2-73或安慰劑的治療。

與以往的單靶標療法不同，Anavex 2-73是一種“一石三鳥”的新型療法，旨在阻擊與AD病理學相關的3個靶標：淀粉樣蛋白、tau蛋白、炎癥。之前，以淀粉樣蛋白為靶標的數(shù)十個藥物在臨床研究中均已遭遇失敗。

Anavex 2-73是一種sigma-1受體（S1R）跨膜蛋白激活劑，S1R作為一種分子伴侶和功能調節(jié)劑，被認為參與調節(jié)細胞內的穩(wěn)態(tài)，通過減少諸如氧化應激、蛋白質錯誤折疊、線粒體功能障礙、炎癥，來保持細胞處于健康、穩(wěn)定的狀態(tài)，所有這些在AD患者大腦中均存在。S1R激活已經(jīng)證明能夠減少AD的主要病理生理學標志：β淀粉樣蛋白、過度磷酸化tau蛋白和炎癥增加。

此前，在入組了32例澳大利亞早期AD患者的一項IIa期臨床研究中，Anavex 2-73展現(xiàn)出了改善注意力水平、工作記憶以及改善精神運動功能的初步療效。有些患者甚至恢復了已失去的機能，比如彈奏鋼琴的能力。

Anavex公司總裁兼CEO Christopher U Missling博士表示，非常高興能獲得監(jiān)管批準啟動這項IIb/III期臨床研究。該公司將利用IIa期研究中所發(fā)現(xiàn)的基因組生物標志物來篩選納入IIb/III期研究的患者。

Anavex 2-73成功的希望或比較渺茫。特別是考慮到之前已經(jīng)有數(shù)十種靶向淀粉樣蛋白的藥物、多種抗炎療法（包括布洛芬）遭遇失敗，而到目前為止，tau蛋白靶向療法的結果也差強人意。不過，Anavex公司卻認為，Anavex 2-73的廣泛作用結合基因組學精準醫(yī)學生物標志物驅動的臨床研究設計，將有望提高該藥的成功機會。

目前，Anavex公司也正在計劃啟動一項II期臨床，評估Anavex 2-73治療帕金森的潛力。除了Anavex 2-73之外，該公司臨床前資產中還有幾個治療AD的藥物，包括Anavex 3-71和Anavex 1-41。該公司神經(jīng)管線資產如上圖所示。

Source:

Anavex Life Sciences Receives Approval To Initiate Phase 2b/3 Clinical Trial of ANAVEX?2-73 for the Treatment of Early Alzheimer’s Disease

• ANAVEX?2-73 Being Studied as Potential First Precision Medicine Biomarker-guided, Targeted Therapeutic in Alzheimer’s Disease

• Primary and Secondary Endpoints measuring Safety, and Cognitive and Functional Efficacy (ADAS-Cog, ADCS-ADL and CDR-SB)

NEW YORK – July 3, 2018 – Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental diseases including Alzheimer’s disease, Rett syndrome and other central nervous system (CNS) diseases, today announced that the Company has received approval by the Australian Human Research Ethics Committee to initiate its Phase 2b/3 double-blind, randomized, placebo-controlled, 48-week safety and efficacy trial of ANAVEX?2-73 for the treatment of early Alzheimer’s disease. The Phase 2b/3 study is scheduled to initiate enrollment of approximately 450 patients, randomized 1:1:1 to two different ANAVEX?2-73 doses or placebo within the next month. As part of the planned international study, North American sites will be added.

“We are extremely pleased to gain approval to initiate the ANAVEX?2-73 Phase 2b/3 study for the treatment of early Alzheimer’s disease, an area of very high unmet need for the growing number of patients around the world,” said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex.

The ANAVEX?2-73 Phase 2b/3 study design includes genomic precision medicine biomarkers identified in the ANAVEX?2-73 Phase 2a study. Primary and secondary endpoints will assess safety and both cognitive and functional efficacy, measured through ADAS-Cog, ADCS-ADL and CDR-SB. ANAVEX?2-73 Phase 2a Alzheimer’s disease study previously demonstrated dose dependent improvement in exploratory endpoints of cognition (MMSE) and function (ADCS-ADL).

About ANAVEX?2-73

ANAVEX?2-73 activates the Sigma-1 receptor (S1R) protein, which serves as a molecular chaperone and functional modulator involved in restoring homeostasis. S1R activation has demonstrated ability to reduce key pathophysiological signs of Alzheimer’s disease: beta amyloid, hyperphosphorylated tau, and increased inflammation. In a Phase 2a Alzheimer’s disease study, ANAVEX?2-73 has shown dose dependent improvement in exploratory endpoints of cognition (MMSE) and function (ADCS-ADL). Full genomic analysis of ANAVEX?2-73 Phase 2a Alzheimer’s disease patients was performed. The ANAVEX?2-73 Phase 2b/3 study design includes genomic precision medicine biomarkers identified in the ANAVEX?2-73 Phase 2a study.

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