MALVERN, Pa., April 10, 2024 (GLOBE NEWSWIRE) -- 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 Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) reviewed the study design, endpoints and planned statistical analysis of the pivotal OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP) and provided acceptability of the U.S.-based trial for submission of a Marketing Authorization Application (MAA)..

宾夕法尼亚州马尔文，2024年4月10日（环球通讯社）--Ocugen，Inc.（“Ocugen”或“公司”）（纳斯达克：OCGN），一家专注于发现、开发和商业化新基因和细胞疗法以及疫苗的生物技术公司，今天宣布，欧洲药品管理局（EMA）人类使用药品委员会（CHMP）审查了关键的OCU400色素性视网膜炎（RP）3期聚光灯临床试验的研究设计、终点和计划统计分析，并提供了美国试验提交上市许可申请（MAA）的可接受性。。

EMA provided this opinion based on safety and tolerability of OCU400 demonstrated in the Phase 1/2 study. The Phase 3 liMeliGhT study will have a sample size of 150 participants primarily in the U.S.—one arm of 75 participants with RHO gene mutations and the other arm with 75 participants that are gene agnostic (representing multiple gene mutations associated with RP).

EMA根据1/2期研究中证明的OCU400的安全性和耐受性提供了这一观点。第三阶段的聚光灯研究将有150名参与者的样本量，主要来自美国-一组75名RHO基因突变参与者，另一组75名基因不可知参与者（代表与RP相关的多个基因突变）。

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..

在每只手臂中，参与者将以2:1的比例随机分配到治疗组（2.5 x 1010 vg/眼OCU400）和未治疗对照组。。

The positive scientific advice from EMA is in alignment with U.S. FDA clearance of the IND amendment to initiate the Phase 3 liMeliGhT clinical trial of OCU400. OCU400 is the first gene therapy to enter Phase 3 with a broad RP indication. Previously, OCU400 received broad Orphan Drug Designation for RP and Leber congenital amaurosis in the EU..

EMA的积极科学建议与美国FDA批准IND修正案一致，以启动OCU400的3期聚光灯临床试验。OCU400是第一个进入第3阶段的基因疗法，具有广泛的RP适应症。此前，OCU400在欧盟获得了RP和Leber先天性黑蒙的广泛孤儿药物指定。。

“We are very grateful to EMA for their collaborative discussions and support in providing a gene-agnostic therapeutic option to RP patients with severe unmet medical need,” said Dr. Shankar Musunuri, Chairman, CEO and Co-founder of Ocugen. “This positive opinion is a critical step in providing our game-changing modifier gene therapies to patients globally.”.

Ocugen董事长、首席执行官兼联合创始人Shankar Musunuri博士表示：“我们非常感谢EMA的合作讨论和支持，为严重未满足医疗需求的RP患者提供了基因不可知的治疗选择。”。“这种积极的观点是为全球患者提供改变游戏规则的修饰基因疗法的关键一步。”。

The EMA opinion is an extremely favorable outcome, as it will potentially reduce the time and cost to gain marketing authorization in the EU. With this milestone, OCU400 remains on track for 2026 BLA and MAA approval targets.

EMA的意见是一个非常有利的结果，因为它可能会减少在欧盟获得营销授权的时间和成本。凭借这一里程碑，OCU400有望实现2026年BLA和MAA批准目标。

About OCU400

OCU400 is the Company’s gene-agnostic modifier gene therapy product based on NHR gene, NR2E3. NR2E3 regulates diverse physiological functions within the retina—such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks. Through its drive functionality, OCU400 resets altered/affected cellular gene-networks and establishes homeostasis—a state of balance, which has the potential to improve retinal health and function in patients with inherited retinal diseases..

OCU400是该公司基于NHR基因NR2E3的基因不可知修饰基因治疗产品。NR2E3调节视网膜内的多种生理功能，例如感光细胞的发育和维持，代谢，光转导，炎症和细胞存活网络。通过其驱动功能，OCU400重置了改变/受影响的细胞基因网络，并建立了稳态-一种平衡状态，有可能改善遗传性视网膜疾病患者的视网膜健康和功能。。

About RP

关于RP

RP is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina, leading to vision loss and blindness. Currently, RP is associated with mutations in more than 100 genes.

RP是一组罕见的遗传性疾病，涉及视网膜细胞的分解和丧失，导致视力丧失和失明。目前，RP与100多个基因的突变有关。

There are no approved treatment options that slow or stop the progression of multiple forms of RP. Proposed treatments for RP include gene-replacement therapy, retinal implant devices, retinal transplantation, stem cells, vitamin therapy, and other pharmacological treatments. Current gene-replacement therapies are promising but are limited to treating just a single mutation.

目前还没有批准的治疗方案可以减缓或阻止多种形式的RP的进展。拟议的RP治疗包括基因替代疗法，视网膜植入装置，视网膜移植，干细胞，维生素疗法和其他药物治疗。目前的基因替代疗法很有希望，但仅限于治疗单个突变。

In addition, while gene therapies may provide a new functional gene, they do not necessarily eliminate the underlying genetic defect, which may still cause stress and toxic effects leading to retina degeneration. Therefore, the development of gene-specific replacement therapy is highly challenging, especially when multiple and unknown genes are involved.

此外，虽然基因疗法可能提供一种新的功能基因，但它们不一定能消除潜在的遗传缺陷，这种缺陷仍可能导致压力和毒性作用，从而导致视网膜变性。因此，基因特异性替代疗法的发展极具挑战性，特别是当涉及多个未知基因时。

Thus, novel therapeutic approaches targeting broader RP disease in a gene agnostic manner offer greater hope for patients..

因此，以基因不可知的方式针对更广泛的RP疾病的新型治疗方法为患者提供了更大的希望。。

About Ocugen, Inc.

关于Ocugen，Inc。

Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital.

Ocugen，Inc.是一家生物技术公司，专注于发现、开发和商业化新型基因和细胞疗法、生物制剂和疫苗，以改善健康并为全球患者带来希望。我们正在通过勇敢的创新创造新的科学途径，利用我们独特的智力和人力资本，对患者的生活产生影响。

Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn..

我们突破性的修饰基因治疗平台有可能用单一产品治疗多种视网膜疾病，我们正在推进传染病研究，以支持公共卫生和骨科疾病，以解决未满足的医疗需求。在www.ocugen.com上了解更多信息，并在X和LinkedIn上关注我们。。

Forward-Looking Statements

前瞻性声明

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties.

本新闻稿包含1995年《私人证券诉讼改革法》所指的前瞻性声明，包括但不限于关于现有数据的定性评估、潜在益处、对正在进行的临床试验的期望、预期的监管备案和预期的开发时间表的声明，这些声明受到风险和不确定性的影响。

We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements.

在某些情况下，我们可能会使用诸如“预测”、“相信”、“潜在”、“提议”、“继续”、“估计”、“预期”、“期望”、“计划”、“打算”、“可能”、“可能”、“可能”、“将会”、“应该”或其他表达未来事件或结果不确定性的词语来识别这些前瞻性陈述。

Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities.

这些陈述受到许多重要因素、风险和不确定性的影响，这些因素、风险和不确定性可能导致实际事件或结果与我们目前的预期有重大差异，包括但不限于初步、中期和一线临床试验结果可能不代表最终临床数据，也可能与最终临床数据不同的风险；正在进行的临床试验或通过对现有临床试验数据的进一步分析，可能会出现不利的新临床试验数据；早期的非临床和临床数据以及测试可能无法预测后期临床试验的结果或成功；并且临床试验数据受到不同的解释和评估，包括监管机构的解释和评估。

These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in thi.

这些以及其他风险和不确定性在我们向美国证券交易委员会（SEC）提交的定期文件中有更全面的描述，包括我们向SEC提交的季度和年度报告中题为“风险因素”的部分中描述的风险因素。我们在该报告中所做的任何前瞻性声明。

Contact:

联系人：

Tiffany Hamilton

蒂芙尼汉密尔顿

Head of Communications

通信主管

Tiffany.Hamilton@ocugen.com

Tiffany.Hamilton@ocugen.com