首個針對吉蘭巴雷綜合症的治療進展




Annexon宣布在2025年PNS會議上介紹Tanruprubart作為首個潛在的針對性治療Guillain-Barré綜合症(GBS)的臨床進展

2025年5月9日 17:00 ET | 來源:Annexon Biosciences

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國際Guillain-Barré綜合症結果研究(IGOS)首次口頭報告顯示,與現行標準護理相比,Tanruprubart(前稱ANX005)在匹配患者群體中改善了結果的真實世界證據(RWE)

第三期試驗的新分析突顯了Tanruprubart對GBS患者的早期和持久治療效果及生活質量的改善

加州布里斯班,2025年5月9日(GLOBE NEWSWIRE)——Annexon, Inc.(納斯達克:ANNX)是一家生物製藥公司,專注於為生活在嚴重經典補體介導的神經炎症疾病中的人們開發新型療法。今天宣布在2025年英國愛丁堡國際會議中心舉行的2025年周邊神經學會(PNS)年會上,將介紹有關Tanruprubart(前稱ANX005)改善結果的口頭和海報報告。

GBS是一種神經肌肉急症和罕見的自體免疫疾病,目前沒有FDA批准的療法,其特徵是迅速進展的嚴重虛弱,可能導致完全癱瘓,通常需要重症監護和機械通氣。Tanruprubart是一種首創的單克隆抗體,旨在阻止C1q,這是經典補體級聯反應的起始分子,通過一次輸注來阻止GBS急性階段的神經炎症和神經損傷,以改善和加快整體康復。

口頭報告

“IGOS中的比較有效性:ANX005與靜脈免疫球蛋白或血漿置換對Guillain-Barré綜合症的比較”

* IGOS演講者:Eveline Wiegers醫生,博士
* 日期/時間:5月19日星期一,英國夏令時間(BST)下午3:55至4:10

海報報告

“將早期補體抑制與GBS的長期結果聯繫起來:客觀測量支持Tanruprubart(ANX005)的療效”

* 演講者:Glenn Morrison博士
* 日期/時間:5月18日星期日,英國夏令時間(BST)下午2:30至3:20

“Tanruprubart(ANX005)改善Guillain-Barré綜合症患者的健康相關生活質量,相較於安慰劑”

* 演講者:Glenn Morrison博士
* 日期/時間:5月19日星期一,英國夏令時間(BST)下午2:10至3:10。該海報還被選為閃電口頭報告,時間為下午5:30至5:35。

“Tanruprubart(ANX005)在廣泛患者範圍內治療Guillain-Barré綜合症的療效”

* 演講者:Henk-André Kroon醫生
* 日期/時間:5月19日星期一,英國夏令時間(BST)下午2:10至3:10

關於Tanruprubart(前稱ANX005)
Annexon的主要研究療法Tanruprubart是一種首創的選擇性、針對性和快速作用的藥物,旨在通過停止C1q在周邊和中樞神經系統的活性來減少炎症和神經損傷。在GBS中,Tanruprubart旨在尋找C1q並防止其與周邊神經的目標結合。Tanruprubart是靜脈注射,並已觀察到幾乎立即阻止C1q功能的效果。GBS的有效治療目標是迅速停止對神經細胞的自體免疫損傷,使患者能夠更快地恢復肌肉力量,恢復獨立性並回到病前的活動。Tanruprubart已獲得美國食品藥品監督管理局的快速通道和孤兒藥物認證,以及歐洲藥品管理局的孤兒藥物認證,用於治療GBS。

關於Guillain-Barré綜合症(GBS)
GBS是一種罕見的神經肌肉急症,源於急性自體抗體和經典補體介導對周邊神經的攻擊,通常在健康人感染後發生。這是一種急性、迅速進展的疾病,治療干預的時間框架非常狹窄。GBS每年在美國和歐洲導致超過22,000人住院。周邊神經損傷迅速進展,導致急性神經肌肉癱瘓,可能造成重大疾病、殘疾和死亡。目前,美國對GBS沒有批准的治療。GBS的長期疾病負擔每年給美國醫療系統帶來數十億美元的經濟成本。

關於Annexon
Annexon Biosciences(納斯達克:ANNX)正在開發能夠阻止經典補體驅動的神經炎症的療法,作為針對數百萬人生活在嚴重神經炎症疾病中的首創治療。我們的新科學方法專注於C1q,這是經典補體強效炎症通路的起始分子,當其失去方向時會導致組織損傷和喪失。通過針對C1q,我們的免疫療法旨在在疾病發生之前停止這一神經炎症級聯反應。我們的產品線涵蓋三個不同的治療領域——自體免疫、神經退行性和眼科疾病,並包括針對超過800萬人未滿足需求的目標研究藥物。Annexon的使命是向患者提供改變遊戲規則的療法,使他們能夠過上更好的生活。

在這篇報導中,Annexon的Tanruprubart作為GBS的潛在治療方案,展示了生物製藥領域的創新。這不僅是對GBS患者的一次希望,更是對未來神經炎症治療的全新視角。隨著臨床數據的積累,Tanruprubart的療效和安全性將成為醫學界關注的焦點。值得注意的是,GBS的治療選擇非常有限,這使得Tanruprubart的成功研發具有重要的臨床意義。未來的研究將需要密切監測其長期效果,並探索其在其他神經炎症疾病中的潛在應用。

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