2023年6月20日

Alkeus製藥最近宣布係列B籌集了1.5億美元的融資支持注冊和發布gildeuretinol(堿性- 001),這是一個潛在的疾病修飾,精密醫學治療黃斑變性疾病(主要遺傳導致失明的兒童和年輕人)。這一輪融資是由貝恩資本(Bain Capital)生命科學與其他參與TCGX,威靈頓管理和Sofinnova投資。加上Alkeus還宣布,約書亞沼澤,博士,生物技術產業先鋒和頂點藥品的創始人,已加入Alkeus執行主席。黃斑變性疾病是導致失明的兒童和年輕人,影響超過30000人在美國和全世界超過150000。黃斑變性疾病患者通常天生視力正常,但ABCA4基因的突變導致加速二聚或凝結的維生素A的眼睛,造成視網膜損傷和隨後的進步視力喪失,早在五歲開始。Gildeuretinol是第一個也是唯一一個藥在臨床開發解決黃斑變性疾病的潛在機製,大幅降低維生素A二聚的眼睛,不影響正常的視力。2期臨床數據顯示統計學和臨床意義的減緩視網膜黃斑變性患者的損害。美國食品和藥物管理局已經批準了突破性治療名稱和孤兒藥物名稱gildeuretinol(堿性- 001)。Alkeus計劃提交批準的NDA gildeuretinol在2024年。關鍵引用:“這1.5億美元所得資金將允許Alkeus擴大我們的團隊,推動gildeuretinol NDA盡快提交和FDA的批準。 I am thrilled to work hand in hand with Joshua as Executive Chairman as we build a patient-focused company. Joshua’s deep experience leading the successful development and commercialization of transformative medicines will be of tremendous value to Alkeus during this period of organizational growth, in preparation for the launch of gildeuretinol.” – Leonide Saad, Ph.D., CEO, President, and Co-Founder of Alkeus “Stargardt is a progressive, debilitating disease that leads inevitably to devastating vision loss. The genetic cause is well characterized, but to date there is no effective treatment. Leonide and the Alkeus team have brought gildeuretinol from the lab all the way to positive and compelling clinical data across multiple stages of the disease, including a Phase 2 placebo-controlled trial in patients with advanced disease, where we have reported a statistically- and clinically-significant slowing of disease. Additional clinical data show halting of disease in patients, including children.” “We are now poised to make this transformative medicine a reality for all Stargardt patients.” – Joshua Boger, Ph.D., Executive Chairman of Alkeus Trending on Pulse 2.0