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).
生物技术公司ocugen（OCGN纳斯达克）专注于发现、开发和商业化新的基因和细胞治疗和疫苗，日前宣布其OCU400项目——一种修改剂基因治疗候选药品已经在视网膜色素变性（RP）的3期liMeliGhT临床试验中成功给首例患者进行了剂量。
"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."
“每个OCU400取得的临床里程碑都让我们更接近为RP患者提供一次性终身治疗，”ocugen董事长、首席执行官和联合创始人Shankar Musunuri博士表示，“对第一位患者进行治疗尤其重要，让我们更加有决心服务于RP患者——在美国和欧洲有300,000人，在全球有1.6百万人。”
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.
这个3期liMeliGhT临床试验得到了1/2期OCU400数据的支持，表明治疗组视力最佳矫正视力（BCVA）、多光度移动测试（MLMT）和低光视力（LLVA）呈现积极趋势，治疗后18个RP受试者中，89%（16/18）的患者在BCVA、LLVA或MLMT得分方面保持或改善了治疗眼的视力。80%（8/10）的RHO突变患者的MLMT得分比基线得分有所保持或提高。78%（14/18）的受试者在治疗后的MLMT得分方面保持或改善了治疗眼的视力。RHO突变患者
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.
这个持续一年的3期临床研究将有150名参与者，其中一组有75名患有RHO基因突变的患者，另一组有75名基因混合型患者。在每组患者中，被随机分配为治疗组（每只眼睛使用2.5 x 1010个OCU400病毒载体）和未接受治疗的对照组，比例为2:1。参与liMeliGhT研究的患者必须为8岁或以上，早期到晚期的RP。RHO各类基因
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.
在该研究中，比亮度的导航评估（LDNA）是研究的主要终点，该评估比之前的3期临床试验中使用的MLMT测量方法更敏感、更具特异性。3期liMeliGhT研究将重点关注在治疗组和对照组中，用治疗眼至少在研究眼中达到比基线提高至少2勒克斯等级的反应者比例。