仁新醫藥斯特格病變新藥 獲准開展陸三期臨床試驗 2022-09-18經濟日報／ 記者 謝柏宏 仁新（6696）16日代子公司Belite Bio, Inc公告，已獲中國國家藥品監督管理局（NMPA）核准，可於中國大陸開展LBS-008藥物針對斯特格病變的三期臨床試驗，目前該三期臨床試驗已於美國、英國、德國、比利時、瑞士、香港、台灣、澳洲等地展開，並已收至多名病患，預計全球收案約60名患者。仁新醫藥指出，斯特格病變（簡稱STGD1）目前仍無治療藥物，全球存在巨大未被滿足的醫療需求，根據美國投資銀行的研究報告，STGD1全球市場規模預估在2030年可達到年12.7億美元（約台幣393億元）。這次公布的LBS-008藥物針對STGD1的三期臨床試驗，為首個核准於中國大陸開展的STGD1臨床試驗，也是目前全球唯一STGD1三期臨床試驗，加上已獲得FDA快速審查認定（Fast Track）的優勢，LBS-008有機會成為全球首個STGD1核准藥物，搶攻龐大市場商機。仁新董事長林雨新表示，STGD1的發病率為萬分之一，是最常見的遺傳性視網膜失養症，導致許多成人和兒童的中央視力模糊、喪失。LBS-008為一口服藥錠，預計可及早治療，以維持視網膜組織的健康並解決巨大未滿足的醫療需求。」仁新醫藥指出，子公司Belite目前正同步進行LBS-008針對STGD1為期兩年的臨床二期試驗、和為期兩年的臨床三期試驗。該臨床二期試驗於澳洲及台灣招募13名青少年受試者，儘管此階段之STGD1為惡化極為快速的階段，但用藥六個月之初步數據顯示，13名患者中有八名（或61.5%）至少有一隻眼睛的最佳矯正視力獲得改善，其中兩名患者的雙眼之最佳矯正視力獲得改善。此外，13名受試者中僅有一名受試者在用藥六個月後產生視網膜萎縮（萎縮範圍約為0.3平方毫米）。Belite公司預計於今年第4季取得該二期臨床試驗用藥一年之數據。LBS-008治療STGD1的三期臨床試驗，是一項多中心、隨機分配、雙盲與安慰劑對照的研究，持續二年，以評估LBS-008治療STGD1青少年患者的安全性及有效性。此外，由於乾性黃斑部病變（簡稱乾性AMD）之病理生理學與STGD1相似，同為有毒雙維甲酸（bisretinoids）於視網膜過度積累，使得感光細胞死亡而導致患者失明，LBS-008獲美國國家衛生研究院（NIH）藍圖計畫認可，並給予研發贊助，NIH更公開表示LBS-008為一款具潛力減緩或抑制乾性AMD惡化的市場首見口服藥物。Belite相信LBS-008可透過口服給藥的方式，及早治療STGD1及乾性AMD。Belite預計於今年第4季度啟動乾性AMD的臨床2/3期試驗，搶攻千億未被滿足的醫療需求市場。
About LBS-008 (aka Tinlarebant): Tinlarebant is a novel oral therapy that prevents the buildup of toxins in the eye that cause STGD1 and contribute to advanced dry AMD. These toxins are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), a carrier protein that transports retinol to the eye. By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of toxins that have been implicated in STGD1 and dry AMD. Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease designation in the U.S., and Orphan Drug Designation in the U.S. and Europe for the treatment of STGD1.
Stargardt Disease: STGD1 is the most common inherited retinal dystrophy (causing blurring or loss of central vision) in both adults and children. The disease is caused by a dysfunctional retina-specific gene (ABCA4) which results in massive accumulation of toxic vitamin A byproducts (known as "bisretinoids") in the retina leading to retinal cell death and progressive loss of central vision. The fluorescent properties of bisretinoids and the development of retinal imaging have helped ophthalmologists identify and monitor disease progression. Importantly, STGD1 and dry AMD share a similar pathophysiology which is characterized by the excessive accumulation of cytotoxic bisretinoids, retinal cell death, and loss of vision. Vision loss occurs slowly, despite peripheral expansion of "dead retina", until the disease reaches the center of the eye (the macula).
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