Ocugen Announces Dosing Completion of Subjects With Stargardt Disease in Cohort 2 of Phase 1/2 GARDian Clinical Trial of OCU410ST—A Modifier Gene Therapy
Ocugen Announces Dosing Completion of Subjects With Stargardt Disease in Cohort 2 of Phase 1/2 GARDian Clinical Trial of OCU410ST—A Modifier Gene Therapy
"There remains a great unmet medical need for patients with Stargardt disease, which is the most common inherited retinal disease affecting the center of the vision and does not have any FDA-approved treatment options. OCU410ST is a novel modifier gene therapy that provides hope to these patients," said Benjamin Bakall, MD, PhD, Director of Clinical Research at Associated Retina Consultants and Clinical Assistant Professor at University of Arizona, College of Medicine – Phoenix. "I am excited that we completed dosing of the last patient in Cohort 2, who received medium dose of this novel therapeutic leveraging a gene-agnostic approach, at Associated Retina Consultants (ARC) in Phoenix, AZ with the surgical team led by Dr. Mark Kwong, Medical Director of ARC."MALVERN, Pa., May 15, 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 GARDian clinical trial for OCU410ST (AAV-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease as a one-time treatment for life.
賓夕法尼亞州馬爾文，2024年5月15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 OCU410ST（AAV-哈羅拉) — 一種用於Stargardt病的改性基因療法候選藥物正在開發中，可作爲一次性終身療法。
"The completion of dosing for Cohort 2 participants signifies an important clinical milestone for our pioneering modifier gene therapy," said Huma Qamar, MD, MPH, Chief Medical Officer of Ocugen. "We are encouraged by the ongoing positive safety and tolerability profile demonstrated by OCU410ST, enabling us to consider higher doses in patients as we progress with the dose-escalation study. We look forward to sharing preliminary safety and efficacy data from Phase 1 of the clinical trial."
Ocugen首席醫學官Huma Qamar醫學博士、公共衛生碩士表示：“隊列2參與者的給藥完成標誌着我們開創性的修飾基因療法的一個重要的臨床里程碑。”“OCU410ST 表現出的持續積極的安全性和耐受性使我們感到鼓舞，這使我們能夠在劑量遞增研究的進展中考慮爲患者提供更高的劑量。我們期待分享臨床試驗第一階段的初步安全性和有效性數據。”
Six patients with Stargardt disease have been dosed in the Phase 1/2 clinical trial to date. An additional three patients will be dosed with the high dose (Cohort 3) of OCU410ST in the dose-escalation phase.
迄今爲止，在1/2期臨床試驗中，已經給六名Stargardt病患者服藥。在劑量遞增階段，另外三名患者將接受高劑量（隊列 3）的 OCU410ST 劑量。
"There remains a great unmet medical need for patients with Stargardt disease, which is the most common inherited retinal disease affecting the center of the vision and does not have any FDA-approved treatment options. OCU410ST is a novel modifier gene therapy that provides hope to these patients," said Benjamin Bakall, MD, PhD, Director of Clinical Research at Associated Retina Consultants and Clinical Assistant Professor at University of Arizona, College of Medicine – Phoenix. "I am excited that we completed dosing of the last patient in Cohort 2, who received medium dose of this novel therapeutic leveraging a gene-agnostic approach, at Associated Retina Consultants (ARC) in Phoenix, AZ with the surgical team led by Dr. Mark Kwong, Medical Director of ARC."
“Stargardt病患者仍有大量未得到滿足的醫療需求，Stargardt病是影響視力中心的最常見的遺傳性視網膜疾病，沒有任何經美國食品藥品管理局批准的治療選擇。OCU410ST 是一種新的修飾基因療法，爲這些患者帶來希望。” 美國視網膜顧問協會臨床研究主任、亞利桑那大學鳳凰城醫學院臨床助理教授本傑明·巴卡爾說。“我很高興我們在亞利桑那州菲尼克斯的Associated Retina Consultants（ARC）與ARC醫學總監Mark Kwong博士領導的外科團隊一起完成了對2隊列中最後一位患者的給藥，該患者採用基因不可知的方法接受了中等劑量的這種新型療法。”
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 Cohort 3 (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，然後繼續進行隊列3（高劑量），這是1期劑量遞增研究的最後一劑量。
The GARDian clinical trial will assess the safety and efficacy of unilateral subretinal administration of OCU410ST in subjects with Stargardt disease and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose-ranging study consisting of three dose levels [low dose (3.75×1010 vg/mL), medium dose (7.5×1010 vg/mL), and high dose (2.25×1011 vg/mL)]. Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study in which adult and pediatric subjects will be randomized in a 1:1:1 ratio to either one of two OCU410ST dose groups or to an untreated group.
Gardian臨床試驗將評估Stargardt病受試者單側視網膜下給藥 OCU410ST 的安全性和有效性，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（3.75×10）]10 vg/mL），中等劑量（7.5×1010 vg/mL）和高劑量（2.25×10）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，在該研究中，成人和兒科受試者將以 1:1:1 的比例隨機分配到兩個 OCU410ST 劑量組中的一個或未經治療的組。
Ocugen is committed to finding solutions for people with inherited retinal disease for whom no effective treatment options exist. While an orphan disease, Stargardt affects approximately 100,000 people in the United States and Europe combined.
Ocugen致力於爲沒有有效治療選擇的遺傳性視網膜疾病患者尋找解決方案。雖然Stargardt是一種孤兒病，但總共影響了美國和歐洲約10萬人。
The Company expects to provide a clinical trial update for OCU410ST in the third quarter of 2024.
該公司預計將在2024年第三季度提供 OCU410ST 的臨床試驗最新情況。
About Stargardt Disease
Stargardt disease is a genetic eye disorder that causes retinal degeneration and vision loss. Stargardt disease is the most common form of inherited macular degeneration. The progressive vision loss associated with Stargardt disease is caused by the degeneration of photoreceptor cells in the central portion of the retina called the macula.
關於Stargardt病
Stargardt病是一種遺傳性眼部疾病，可導致視網膜變性和視力喪失。Stargardt病是遺傳性黃斑變性的最常見形式。與Stargardt病相關的漸進性視力喪失是由視網膜中央部分稱爲黃斑的感光細胞退化引起的。
Decreased central vision due to loss of photoreceptors in the macula is the hallmark of Stargardt disease. Some peripheral vision is usually preserved. Stargardt disease typically develops during childhood or adolescence, but the age of onset and rate of progression can vary. The retinal pigment epithelium (RPE), a layer of cells supporting photoreceptors, is also affected in people with Stargardt disease.
由於黃斑中感光器喪失而導致的中心視力下降是Stargardt病的標誌。通常會保留一些周邊視力。Stargardt病通常發生在兒童或青春期，但發病年齡和進展速度可能有所不同。Stargardt病患者的視網膜色素上皮（RPE）是支持光感受器的細胞層，也受到影響。
About OCU410ST
OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. It represents Ocugen's modifier gene therapy approach, which is based on Nuclear Hormone Receptor (NHR) RORA that regulates pathway links to Stargardt disease such as lipofuscin formation, oxidative stress, compliment formation, inflammation, and cell survival networks.
關於 OCU410ST
OCU410ST 利用 AAV 交付平台進行視網膜交付 RORA （RAR 相關孤兒受體 A）基因。它代表了Ocugen的修飾基因治療方法，該方法基於核激素受體（NHR） RORA 它調節與Stargardt病的通路，例如脂褐素的形成、氧化應激、補體形成、炎症和細胞存活網絡。
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 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及疫苗，這些療法和疫苗可改善健康狀況，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，並且我們正在推進傳染病研究，以支持公共衛生和骨科疾病，以滿足未滿足的醫療需求。在以下網址了解更多 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，包括但不限於有關可用數據的定性評估、潛在收益、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，這些陳述受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，包括但不限於初步、中期和一線臨床試驗結果可能無法表示且可能與最終臨床數據不同的風險；在正在進行的臨床試驗中或通過對現有臨床試驗數據的進一步分析，可能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 of 可能無法預測後期臨床試驗的結果或成功與否；以及臨床試驗數據將受到不同的解釋和評估，包括監管機構的解釋和評估。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。
