DJ Axovant Gene Therapies: FDA Lifts Clinical Hold on AXO-AAV-GM2
By Chris Wack
Axovant Gene Therapies Ltd. said Monday the U.S. Food and Drug Administration has lifted its clinical hold and cleared the investigational new drug application to initiate a registrational study of AXO-AAV-GM2 gene therapy to treat patients with Tay-Sachs disease and Sandhoff disease.
The company said it has received a letter from the FDA indicating that it has satisfactorily addressed all issues related to the clinical hold. In October, Axovant received the FSA's rare-pediatric-disease designation for AXO-AAV-GM2.
Axovant said it is aiming to advance the program through strategic partnerships with leading research organizations. The company recently announced a partnership with Viralgen to support AAV-based vector manufacturing of clinical trial material for the registrational study. Axovant expects to begin patient identification and site startup activities in preparation for dosing children in the planned clinical study.
AXO-AAV-GM2 is an investigational gene therapy for Tay-Sachs and Sandhoff diseases, which are rare, monogenic neurodegenerative lysosomal storage disorders caused by mutations in the genes.
In 2019, clinical evidence from two patients under an investigator-initiated study found that treatment with AXO-AAV-GM2 was generally well-tolerated and associated with improved bioactivity outcomes.
The study will enroll both infantile and juvenile subjects with GM2 gangliosidosis in the U.S. The two-part trial, sponsored by Axovant, will consist of a dose ranging cohort evaluating the safe and efficacious dose of the gene therapy, followed by an efficacy cohort, both of which form the basis of the registrational program.
Axovant Gene shares were up 8% to $2.06 in premarket trading.
Write to Chris Wack at chris.wack@wsj.com
(END) Dow Jones Newswires
November 09, 2020 09:05 ET (14:05 GMT)
Copyright (c) 2020 Dow Jones & Company, Inc.
DJ Axovant基因療法:FDA解除Axo-AAV-GM2的臨牀控制
克里斯·瓦克(Chris Wack)著
Axovant基因療法有限公司週一説,美國食品和藥物管理局(FDA)已經解除了臨牀控制,批准了正在研究的新藥申請,啟動了Axo-AAV-GM2基因療法的註冊研究,用於治療泰-薩克斯病(Tay-Sachs disease)和桑德霍夫病(Sandhoff Disease)患者。
該公司表示,它已經收到FDA的一封信,表明它已經令人滿意地解決了與臨牀擱置相關的所有問題。10月份,Axovant獲得了FSA對Axo-AAV-GM2的罕見兒科疾病稱號。
Axovant表示,它的目標是通過與領先的研究機構建立戰略合作伙伴關係來推進該計劃。該公司最近宣佈與Viralgen建立合作伙伴關係,以支持基於AAV的臨牀試驗材料的註冊研究載體製造。Axovant希望開始患者識別和網站啟動活動,為在計劃中的臨牀研究中給兒童服用藥物做準備。
Axo-AAV-GM2是一種針對Tay-Sachs和Sandhoff病的研究基因療法,這兩種疾病是一種罕見的單基因神經退行性溶酶體儲存疾病,由基因突變引起。
2019年,在一項由研究人員發起的研究中,來自兩名患者的臨牀證據發現,使用Axo-AAV-GM2治療通常耐受性良好,並與改善生物活性結果有關。
這項研究將在美國招募患有GM2神經節苷脂增多症的嬰兒和青少年受試者。這項由Axovant贊助的兩部分試驗將包括一個評估基因療法安全有效劑量的劑量範圍隊列,隨後是一個有效性隊列,兩者都構成註冊計劃的基礎。
Axovant Gene股價在盤前交易中上漲8%,至2.06美元。
寫信給克里斯·瓦克(chris.wack@wsj.com)
(完)道瓊通訊社
2020年11月9日東部時間09:05(格林尼治標準時間14:05)
版權所有(C)2020道瓊斯公司。