Ocugen, Inc. Announces Completion of Dosing in Subjects With Geographic Atrophy Secondary to DAMD in High-Dose Cohort of Phase 1/2 ArMaDa Clinical Trial of OCU410—A Novel Modifier Gene Therapy
Ocugen, Inc. Announces Completion of Dosing in Subjects With Geographic Atrophy Secondary to DAMD in High-Dose Cohort of Phase 1/2 ArMaDa Clinical Trial of OCU410—A Novel Modifier Gene Therapy
• Initiated Phase 2 clinical trial
• 開展第2期臨床試驗
MALVERN, Pa., July 25, 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 third cohort of its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA)—a novel 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.
賓夕法尼亞州馬爾文，2024年7月25日（環球新聞通訊社）-- 生物技術公司Ocugen，Inc.（“Ocugen”或“公司”）（NASDAQ：OCGN）專注於發現、開發和商業化新型基因和細胞治療和疫苗，今天宣佈，其用於地理萎縮（GA）的一種新型修飾基因治療候選藥物OCU410（AAV-hRORA）的第1/2期ArMaDa臨床試驗第三隊給藥工作已完成，該藥物是治療乾性年齡相關性黃斑變性（dAMD）晚期的一種新型修飾基因治療候選藥物。在美國，大約有100萬人患有GA。
"Currently, there are two FDA approved anti-complement therapies for GA, which targets only one pathway for a disease that has a multifactorial and complex etiology," said Majda Hadziahmetovic, MD, Associate Professor of Ophthalmology, Vitreoretinal Diseases and Surgery, Duke University Eye Center, and the lead investigator for the OCU410 Phase 1/2 trial. "Though these treatments are a significant milestone, they provide limited benefits and involve a continuous regimen of multiple intravitreal injections over several years. This modifier gene therapy could potentially transform the landscape of GA treatment."
“目前，針對GA，已有兩種FDA批准的抗補體療法只針對其中一種途徑，而這種疾病具有多因素和複雜的病因，”說Majda Hadziahmetovic，MD，眼科、玻璃體視網膜疾病和手術的副教授杜克大學眼科中心以及OCU410第1/2期試驗的主要研究者。“雖然這些治療方法是一個重要的里程碑，但它們提供的益處有限，需要在數年內進行多次玻璃體內注射的持續治療方案。該修飾基因治療藥物可能會改變GA治療的局面。”
Three subjects received a single subretinal administration of the highest dose (200 μL of 1.5x1011 vg/mL) being tested, which completed the dosing for the Phase 1 stage of the trial. The ArMaDa clinical trial for OCU410 is being performed at 14 leading retinal surgery centers across the United States.
三名受試者接受了最高劑量（200μL的1.5x1011vg/mL）的單次視網膜下給藥，這完成了該試驗的第一階段給藥。OCU410的ArMaDa臨床試驗在美國的14個主要視網膜外科中心進行。
"We are very encouraged about the potential of OCU410 as a one-time treatment option for GA," said Dr. Huma Qamar, Chief Medical Officer of Ocugen.
Ocugen的首席醫學官Dr. Huma Qamar表示：“我們對將OCU410作爲GA的一次性治療選擇的潛力感到非常鼓舞。”
We have initiated a Phase 2 clinical trial that will assess the safety and efficacy of OCU410 in a larger group of patients who will be randomized into either of two treatment groups (medium- or high-dose) or a control group.
我們啓動了一項第2期臨床試驗，將評估OCU410在更大的病人群中的安全性和有效性，這些病人將被隨機分爲兩個治療組（中劑量或高劑量）或對照組。
The Company will continue to provide clinical updates on a periodic basis.
公司將繼續定期提供臨床更新。
About the Phase 1/2 ArMaDa clinical trial
The ArMaDa Phase 1/2 clinical trial will assess the safety of unilateral subretinal administration of OCU410 in subjects with geographic atrophy and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose-escalation 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.
關於第1/2期ArMaDa臨床試驗
ArMaDa第1/2期臨床試驗將評估單側視網膜下給藥OCU410治療地理萎縮病人的安全性，並分爲兩個階段進行。第1階段是一個多中心、開放標籤、劑量遞增的研究，包括三個劑量水平[低劑量（2.5×1010vg/mL）、中劑量（5×1010 vg/mL）和高劑量（1.5×1011vg/mL）]。第2階段是一個隨機分組、結局盲評估、劑量擴大研究，病人將以1:1:1的比例隨機分配到兩個OCU410治療組之一或未治療的對照組。
About dry age-related macular degeneration (dAMD) and geographic atrophy (GA)
Dry age-related macular degeneration (dAMD) affects approximately 10 million Americans and more than 266 million people worldwide. It is characterized by the thinning of the macula, the portion 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 all AMD cases.
關於乾性年齡相關性黃斑變性（dAMD）和地理萎縮（GA）
乾性年齡相關性黃斑變性（dAMD）在美國約有1000萬人，全球有超過2.66億人受影響。它的特徵是黃斑區的稀疏，黃斑區是負責眼中直接視線的清晰視力的一部分。dAMD包括視網膜緩慢惡化、黃斑下降（視網膜上的小白點或黃色點）、萎縮、黃斑失去功能和中心視力損傷。dAMD佔所有AMD病例的85-90%。
About OCU410
OCU410 utilizes an adeno-associated virus (AAV) 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 both in vitro and in vivo (animal model) studies. These results demonstrate the ability of OCU410 to target multiple pathways linked with dAMD pathophysiology. Ocugen is developing AAV-RORA as a one-time gene therapy for the treatment of geographic atrophy.
OCU410利用AAV傳遞平台進行視網膜傳遞“RORA”（ROR相關孤兒受體A）基因。 RORA蛋白在脂質代謝中發揮重要作用，減少脂褐質沉積和氧化應激，並在體內和體外（動物模型）研究中顯示出抗炎作用以及抑制互補系統。這些結果證明OCU410具有針對與dAMD病理生理學相關的多個途徑的能力。 Ocugen正在開發AAV-RORA作爲治療GA的一次性基因療法。
OCU410利用腺相關病毒（AAV）平台對視網膜的RORA（ROR Related Orphan Receptor A）基因進行傳遞。RORA蛋白在脂質代謝、減少脂褐質沉積和氧化應激方面發揮重要作用，並在體外和體內（動物模型）研究中發揮抗炎作用並抑制補體系統。這些結果表明OCU410對與dAMD病理生理學有關的多個途徑產生作用。Ocugen正在開發AAV-RORA作爲治療地理萎縮的一次性基因治療藥物。
About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies 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 and follow us on X and LinkedIn.
Ocugen是一家生物技術公司，致力於發現，開發和商業化新型基因和細胞療法以及疫苗，以改善全球患者的健康併爲他們帶來希望。我們通過勇敢的創新影響着患者的生活，開拓了新的科學路徑，利用我們獨特的智力和人力資本。我們的突破性調節基因治療平台具有使用單一產品治療多種視網膜疾病的潛力，並且我們正在推進傳染病的研究，以支持公共衛生和改善骨科疾病，以滿足醫療領域的未滿足需求。訪問更多信息，請瀏覽網站，並在X和LinkedIn上關注我們。
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治療和疫苗，以改善全球患者的健康狀況和爲他們帶來希望。我們通過勇敢的創新對患者的生命產生影響，開創新的科學路徑，利用我們獨特的智力和人力資本。我們的突破性修飾基因治療平台有可能用單一產品治療多種視網膜疾病，我們正在推進感染性疾病的研究，以支持公共衛生和解決骨科疾病的未滿足醫療需求。請訪問了解更多信息。和我們一起X和頁面。LinkedIn.
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前瞻性聲明
本新聞發佈中包含根據1995年《私人證券訴訟改革法案》所作出的前瞻性聲明，包括但不限於，關於可用數據的定性評估、潛在效益、有關於正在進行的臨床試驗的預期、預計的監管申報和預計的開發時間表的聲明，這些聲明受到風險和不確定性的影響。在某些情況下，我們可能使用例如“預計”，“認爲”，“潛在”，“建議”，“繼續”，“估計”，“預計“，”計劃“，”打算“，”可能“，”可能“，”將，“應該”或其他表達不確定性的字詞，以確定這些前瞻性陳述。這些陳述受衆多重要因素、風險和不確定性的影響，這些因素、風險和不確定性可能使我們當前的預期與實際事件或結果發生實質性不符，包括但不限於，初步、中期和頂線臨床試驗結果可能不代表最終臨床數據，並且可能與最終臨床數據不同；不利的新的臨床試驗數據可能在正在進行的臨床試驗中或通過現有臨床試驗數據的進一步分析中出現；早期的非臨床和臨床數據和測試可能無法預測後續臨床試驗的結果或成功；臨床試驗數據可能會因不同的解釋和評估而有所不同，包括監管機構。這些和其他風險和不確定性更詳細地描述在我們與證券交易委員會（SEC）的定期文件中，包括在我們提交給SEC的季度和年度報告中的“風險因素”一節描述的風險因素中。我們在本新聞發佈中作出的任何前瞻性聲明僅在本新聞發佈之日作出。除法律要求外，我們假定沒有義務在本新聞發佈之後更新其中包含的前瞻性聲明，無論是因爲獲得新信息、未來事件還是其他原因。
Contact:
Tiffany Hamilton
Head of Communications
Tiffany.Hamilton@ocugen.com
聯繫方式：
蒂芙尼·漢密爾頓
通信主管
Tiffany.Hamilton@ocugen.com
譯文內容由第三人軟體翻譯。