Ocugen Begins Phase 3 Trial for Gene Therapy to Treat Retinitis Pigmentosa
Ocugen Begins Phase 3 Trial for Gene Therapy to Treat Retinitis Pigmentosa
Ocugen, Inc. ("Ocugen" or the "Company") (NASDAQ:OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that the first patient has been dosed in its Phase 3 liMeliGhT clinical trial for OCU400—a modifier gene therapy product candidate being developed for retinitis pigmentosa (RP).
生物技術公司ocugen（OCGN納斯達克）專注於發現、開發和商業化新的基因和細胞治療和疫苗，日前宣佈其OCU400項目——一種修改劑基因治療候選藥品已經在視網膜色素變性（RP）的3期liMeliGhT臨床試驗中成功給首例患者進行了劑量。
"Each clinical milestone achieved by OCU400 brings us closer to providing a one-time treatment for life to patients living with RP," said Dr. Shankar Musunuri, Chairman, CEO and Co-founder of Ocugen. "Dosing the first patient is especially significant and makes our dedication to serving RP patients—300,000 in the U.S. and Europe and 1.6 million worldwide—more tangible."
“每個OCU400取得的臨床里程碑都讓我們更接近爲RP患者提供一次性終身治療，”ocugen董事長、首席執行官和聯合創始人Shankar Musunuri博士表示，“對第一位患者進行治療尤其重要，讓我們更加有決心服務於RP患者——在美國和歐洲有300,000人，在全球有1.6百萬人。”
The Phase 3 liMeliGhT clinical trial was informed by positive Phase 1/2 OCU400 data that suggests positive trends in Best-Corrected Visual Acuity (BCVA) and Multi-Luminance Mobility Testing (MLMT), and Low-Luminance Visual Acuity (LLVA) among treated eyes. 89% (16/18) of RP subjects demonstrated preservation or improvement in the treated eye either on BCVA or LLVA or MLMT scores from baseline. 80% (8/10) of RHO mutation subjects experienced either preservation or improvement in MLMT scores from baseline. 78% (14/18) of subjects demonstrated preservation or improvement in treated eyes in MLMT scores from baseline.
這個3期liMeliGhT臨床試驗得到了1/2期OCU400數據的支持，表明治療組視力最佳矯正視力（BCVA）、多光度移動測試（MLMT）和低光視力（LLVA）呈現積極趨勢，治療後18個RP受試者中，89%（16/18）的患者在BCVA、LLVA或MLMT得分方面保持或改善了治療眼的視力。80%（8/10）的RHO突變患者的MLMT得分比基線得分有所保持或提高。78%（14/18）的受試者在治療後的MLMT得分方面保持或改善了治療眼的視力。RHO突變患者
The Phase 3 study—with the duration of one year—will have a sample size of 150 participants—one arm of 75 participants with RHO gene mutations and the other arm with 75 participants that are gene agnostic. In each arm, participants will be randomized 2:1 to the treatment group (2.5 x 1010 vg/eye of OCU400) and untreated control group, respectively. Patients eight years of age and older, with early through late-stage advancement of RP, are being recruited to participate in the liMeliGhT study.
這個持續一年的3期臨床研究將有150名參與者，其中一組有75名患有RHO基因突變的患者，另一組有75名基因混合型患者。在每組患者中，被隨機分配爲治療組（每隻眼睛使用2.5 x 1010個OCU400病毒載體）和未接受治療的對照組，比例爲2:1。參與liMeliGhT研究的患者必須爲8歲或以上，早期到晚期的RP。RHO各類基因
Luminance Dependent Navigation Assessment (LDNA)—a more sensitive and specific measurement of function than MLMT used in previous Phase 3 clinical trials—is the primary endpoint for the study. The Phase 3 liMeliGhT study will focus on the proportion of responders, in treated and untreated groups, achieving an improvement of at least 2 Lux levels from baseline in the study eyes.
在該研究中，比亮度的導航評估（LDNA）是研究的主要終點，該評估比之前的3期臨床試驗中使用的MLMT測量方法更敏感、更具特異性。3期liMeliGhT研究將重點關注在治療組和對照組中，用治療眼至少在研究眼中達到比基線提高至少2勒克斯等級的反應者比例。