Ocugen Announces Dosing Completion of Subjects With Geographic Atrophy in Cohort 2 of Phase 1/2 ArMaDa Clinical Trial of OCU410—A Modifier Gene Therapy
Ocugen Announces Dosing Completion of Subjects With Geographic Atrophy in Cohort 2 of Phase 1/2 ArMaDa Clinical Trial of OCU410—A Modifier Gene Therapy
MALVERN, Pa., April 19, 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 dosing is complete in the second cohort of its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA)—a modifier gene therapy candidate being developed for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD). GA affects approximately 1 million people in the United States alone.
賓夕法尼亞州馬爾文，2024年4月19日（GLOBE NEWSWIRE）——專注於發現、開發和商業化新型基因和細胞療法及疫苗的生物技術公司Ocugen, Inc.（“OCUGEN” 或 “公司”）（納斯達克股票代碼：OCGN）今天宣佈，其 OCU410（AAV-哈羅拉）——一種用於地理萎縮（GA）的改性基因療法候選藥物，地理萎縮是乾性年齡相關性黃斑變性（dAMD）的晚期。僅在美國，GA就影響了大約100萬人。
"We are very enthusiastic about the potential of OCU410 as a one-time, gene-agnostic option for the treatment of GA," said Dr. Huma Qamar, Chief Medical Officer of Ocugen. "OCU410 regulates multiple pathways involved with the disease, including lipid metabolism, inflammation, oxidative stress, and membrane attack complex (complement) with a single sub-retinal injection."
Ocugen首席醫學官胡瑪·卡馬爾博士說：“我們對 OCU410 作爲一次性、與基因無關的治療遺傳學選擇的潛力非常熱情。”“OCU410 通過單次視網膜下注射來調節與該疾病相關的多種途徑，包括脂質代謝、炎症、氧化應激和膜攻擊複合物（補體）。”
Dosing in the second cohort is complete and 3 subjects received 200 mL single subretinal administration of the medium dose (5x1010 vg/mL) of OCU410. Up to 13 leading retinal surgery centers across the United States are participating in the ArMaDa clinical trial.
第二組隊列的給藥已完成，3名受試者接受了中等劑量（5x10）的200 mL單次視網膜下給藥10 OCU410 的 vg/mL)。美國多達13個領先的視網膜手術中心正在參與Armada臨床試驗。
"Currently we have two FDA approved, anti-complement therapies for GA targeting a single pathway of the disease, which has multifactorial and complex etiology," said Syed M. Shah, MD, Vice Chair of Research and Digital Medicine and Director of Retina Service at Gundersen Health System, La Crosse, WI, and the lead investigator for the OCU410 Phase 1/2 trial. "The limited benefit comes with the burden of continued multiple intravitreal injections spanning over several years. This novel modifier gene therapy has the potential to transform the therapeutic landscape in GA treatment."
威斯康星州拉克羅斯市Gundersen Health System研究和數字醫學副主席兼視網膜服務主任、OCU410 1/2期試驗首席研究員賽義德·沙阿說：“目前，我們有兩種經美國食品藥品管理局批准的針對該疾病的單一途徑的抗補體療法，其病因具有多因素和複雜的病因。”“有限的好處是持續多次玻璃體內注射的負擔。這種新的改性基因療法有可能改變遺傳性糖尿病治療的治療格局。”
A Data and Safety Monitoring Board meeting will convene next month to review the 4-week safety data of the medium dose cohort before proceeding with high dose, which is the final dose in the Phase 1 dose-escalation study.
下個月將召開數據和安全監測委員會會議，審查中等劑量隊列的4周安全數據，然後繼續使用高劑量，這是1期劑量遞增研究的最後一劑量。
The ArMaDa Phase 1/2 clinical trial will assess the safety of unilateral subretinal administration of OCU410 in subjects with GA and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose-ranging study consisting of three dose levels [low dose (2.5×1010 vg/mL), medium dose (5×1010 vg/mL), and high dose (1.5 ×1011 vg/mL). Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study in which subjects will be randomized in a 1:1:1 ratio to either one of two OCU410 treatment groups or to an untreated control group.
Armada 1/2 期臨床試驗將評估 GA 受試者單側視網膜下給藥 OCU410 的安全性，並將分兩個階段進行。第一階段是一項多中心、開放標籤、劑量範圍的研究，包括三個劑量水平 [低劑量（2.5×10）]10 vg/mL），中等劑量（5×1010 vg/mL）和高劑量（1.5 ×10）11 vg/mL）。第 2 階段是一項隨機、結果受試者盲目劑量擴展研究，受試者將以 1:1:1 的比例隨機分配到兩個 OCU410 治療組中的一個或未經治療的對照組。
The Company will continue to provide clinical updates on an ongoing basis.
該公司將繼續持續提供臨床更新。
About dAMD and GA
dAMD affects approximately 10 million Americans and more than 266 million people worldwide. It is characterized by the thinning of the macula. The macula is the part of the retina responsible for clear vision in one's direct line of sight. dAMD involves the slow deterioration of the retina with submacular drusen (small white or yellow dots on the retina), atrophy, loss of macular function and central vision impairment. dAMD accounts for 85-90% of the total AMD population.
關於 damD 和 GA
dAMD 影響了大約一千萬美國人和全球超過 2.66 億人。它的特徵是黃斑變薄。黃斑是視網膜中負責在直接視線範圍內保持清晰視力的部分。dAMD 涉及視網膜緩慢退化，伴有黃斑下凹陷（視網膜上的小白點或黃點）、萎縮、黃斑功能喪失和中樞視力損傷。dAMD 佔 AMD 總人口的 85-90%。
About OCU410
OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (ROR Related Orphan Receptor A) gene. The RORA protein plays an important role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role as well as inhibiting the complement system in in-vitro and in-vivo (animal model) studies. These results demonstrate the ability for OCU410 to target multiple pathways linked with dAMD pathophysiology. Ocugen is developing AAV-RORA as a one-time gene therapy for the treatment of GA.
關於 OCU410
OCU410 利用 AAV 交付平台對視網膜進行視網膜分娩 RORA （ROR 相關孤兒受體 A）基因。RORA蛋白在脂質代謝、減少脂褐素沉積和氧化應激中起着重要作用，在體外和體內（動物模型）研究中表現出抗炎和抑制補體系統的作用。這些結果表明，OCU410 能夠靶向與 dAMD 病理生理學相關的多種途徑。Ocugen 正在開發 AAV-RORA 作爲治療GA的一次性基因療法。
About 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 patients' lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. 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.
關於 Ocugen, Inc.
Ocugen, Inc. 是一家生物技術公司，專注於發現、開發和商業化新型基因和細胞療法、生物製劑和疫苗，這些療法可以改善健康狀況，爲全球患者帶來希望。我們正在通過勇敢的創新對患者的生活產生影響——開闢新的科學道路，利用我們獨特的智力和人力資本。我們的突破性修飾基因療法平台有可能使用單一產品治療多種視網膜疾病，我們正在推進傳染病研究，以支持公共衛生和骨科疾病，以滿足未得到滿足的醫療需求。如需了解更多信息，請訪問 www.ocugen.com 然後關注我們 X 和 領英。
Forward-Looking Statements
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. 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. 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. 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 this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.
前瞻性陳述
本新聞稿包含1995年《私人證券訴訟改革法》所指的前瞻性陳述， 包括但不限於關於現有數據的定性評估、潛在益處、對正在進行的臨床試驗的預期、預期的監管申報和預期的開發時間表的聲明， 它們受到風險和不確定性的影響。在某些情況下，我們可能會使用 “預測”、“相信”、“潛在”、“提議”、“繼續”、“估計”、“預期”、“預期”、“計劃”、“打算”、“可能”、“可能”、“可能”、“將”、“應該” 等術語或其他表達未來事件或結果不確定性的詞語來識別這些前瞻性陳述。此類陳述受許多重要因素、風險和不確定性的影響，這些因素、風險和不確定性可能導致實際事件或結果與我們當前的預期存在重大差異，i包括但不限於以下風險：初步、中期和一線臨床試驗結果可能不代表最終臨床數據，也可能與最終臨床數據有所不同；正在進行的臨床試驗或通過對現有臨床試驗數據的進一步分析可能會出現不利的新臨床試驗數據；早期的非臨床和臨床數據及測試可能無法預測後期臨床試驗的結果或成功；以及臨床試驗數據受不同的解釋和評估，包括監管機構的評估。我們在向美國證券交易委員會（SEC）提交的定期文件中更全面地描述了這些風險和不確定性，包括我們向美國證券交易委員會（SEC）提交的季度和年度報告中題爲 “風險因素” 的部分中描述的風險因素。我們在本新聞稿中所作的任何前瞻性陳述僅代表截至本新聞稿發佈之日。除非法律要求，否則在本新聞稿發佈之日之後，無論是由於新信息、未來事件還是其他原因，我們都沒有義務更新本新聞稿中包含的前瞻性陳述。
Contact:
Tiffany Hamilton
Head of Communications
Tiffany.Hamilton@ocugen.com
聯繫人：
蒂芙尼漢密爾頓
傳播主管
Tiffany.Hamilton@ocugen.com
譯文內容由第三人軟體翻譯。