Ocugen, Inc. Announces First Patient Dosed in Phase 1 Clinical Trial of OCU200—a Novel Integrin-Targeting Biologic for Diabetic Macular Edema
Ocugen (NASDAQ: OCGN) has initiated the Phase 1 clinical trial of OCU200 for diabetic macular edema (DME) with the first patient dosed. The trial is a multicenter, open-label, dose-escalation study evaluating three dosage levels: 0.025 mg, 0.05 mg, and 0.1 mg, with patients receiving two intravitreal injections six weeks apart.
OCU200 is a novel recombinant fusion protein targeting integrin receptors, designed to treat DME, diabetic retinopathy (DR), and wet age-related macular degeneration (wet AMD). The treatment aims to address an unmet need, particularly for the 30-40% of patients who don't respond to current anti-VEGF therapies. These conditions affect approximately 12 million people in the US and 130 million worldwide.
The company plans to pursue OCU200's approval as a first-line therapy for all three conditions, with patient follow-up extending to three months post-treatment.
Ocugen (NASDAQ: OCGN) ha avviato lo studio clinico di Fase 1 di OCU200 per l'edema maculare diabetico (DME) con il primo paziente trattato. Lo studio è multicentrico, in aperto, e prevede un'escalation delle dosi valutando tre livelli di dosaggio: 0,025 mg, 0,05 mg e 0,1 mg, con i pazienti che ricevono due iniezioni intravitreali a distanza di sei settimane l'una dall'altra.
OCU200 è una nuova proteina di fusione ricombinante mirata ai recettori integrinici, progettata per trattare DME, retinopatia diabetica (DR) e degenerazione maculare legata all'età umida (wet AMD). Il trattamento si propone di soddisfare un bisogno non soddisfatto, in particolare per il 30-40% dei pazienti che non rispondono alle attuali terapie anti-VEGF. Queste condizioni colpiscono circa 12 milioni di persone negli Stati Uniti e 130 milioni a livello globale.
La società prevede di perseguire l'approvazione di OCU200 come terapia di prima linea per tutte e tre le condizioni, con un follow-up dei pazienti che si estende fino a tre mesi dopo il trattamento.
Ocugen (NASDAQ: OCGN) ha iniciado el ensayo clínico de Fase 1 de OCU200 para el edema macular diabético (DME) con el primer paciente dosificado. El ensayo es un estudio multicéntrico, abierto y de escalada de dosis que evalúa tres niveles de dosificación: 0.025 mg, 0.05 mg y 0.1 mg, con pacientes que reciben dos inyecciones intravítreas con un intervalo de seis semanas.
OCU200 es una proteína de fusión recombinante novedosa que apunta a los receptores de integrinas, diseñada para tratar DME, retinopatía diabética (DR) y degeneración macular relacionada con la edad húmeda (wet AMD). El tratamiento tiene como objetivo satisfacer una necesidad no cubierta, particularmente para el 30-40% de los pacientes que no responden a las terapias actuales anti-VEGF. Estas condiciones afectan aproximadamente a 12 millones de personas en los EE. UU. y 130 millones en todo el mundo.
La empresa planea buscar la aprobación de OCU200 como terapia de primera línea para las tres condiciones, con un seguimiento a los pacientes que se extiende hasta tres meses después del tratamiento.
Ocugen (NASDAQ: OCGN)은 당뇨병성 황반 부종(DME) 치료를 위한 OCU200의 1상 임상 시험을 시작하였으며, 첫 번째 환자가 투여되었습니다. 이 시험은 다기관, 공개, 용량 증량 연구로, 0.025 mg, 0.05 mg 및 0.1 mg의 세 가지 용량 수준을 평가하며, 환자는 6주 간격으로 두 번의 유리체 내 주사를 받습니다.
OCU200은 인테그린 수용체를 표적으로 하는 새로운 재조합 융합 단백질로, DME, 당뇨병성 망막병증(DR) 및 습성 노인성 황반 변성(wet AMD)을 치료하도록 설계되었습니다. 이 치료법은 현재의 항-VEGF 요법에 반응하지 않는 30-40%의 환자를 포함한 미충족 수요를 해결하는 것을 목표로 합니다. 이러한 질환은 미국에서 약 1200만 명, 전 세계적으로는 1억 3000만 명에 영향을 미칩니다.
회사는 OCU200을 세 가지 질환에 대한 1차 치료제로 승인받기 위해 노력할 계획이며, 환자 추적 관찰은 치료 후 3개월까지 연장될 것입니다.
Ocugen (NASDAQ: OCGN) a lancé l'essai clinique de Phase 1 d'OCU200 pour l'œdème maculaire diabétique (DME) avec le premier patient traité. L'essai est une étude multicentrique, en ouvert, d'escalade de doses évaluant trois niveaux de dosage : 0,025 mg, 0,05 mg et 0,1 mg, les patients recevant deux injections intravitréennes à six semaines d'intervalle.
OCU200 est une nouvelle protéine de fusion recombinante ciblant les récepteurs d'intégrines, conçue pour traiter le DME, la rétinopathie diabétique (DR) et la dégénérescence maculaire liée à l'âge humide (wet AMD). Le traitement vise à répondre à un besoin non satisfait, en particulier pour les 30-40 % des patients qui ne répondent pas aux traitements anti-VEGF actuels. Ces affections touchent environ 12 millions de personnes aux États-Unis et 130 millions dans le monde.
La société prévoit de demander l'approbation d'OCU200 en tant que thérapie de première ligne pour les trois conditions, avec un suivi des patients s'étendant jusqu'à trois mois après le traitement.
Ocugen (NASDAQ: OCGN) hat die klinische Phase-1-Studie zu OCU200 bei diabetischem Makulaödem (DME) begonnen, wobei der erste Patient behandelt wurde. Die Studie ist eine multizentrische, offene Dosiseskalationsstudie, die drei Dosierungsniveaus bewertet: 0,025 mg, 0,05 mg und 0,1 mg, wobei die Patienten zwei intravitrealen Injektionen mit einem Abstand von sechs Wochen erhalten.
OCU200 ist ein neuartiges rekombinantes Fusionsprotein, das auf Integrinrezeptoren abzielt und zur Behandlung von DME, diabetischer Retinopathie (DR) und nasser altersbedingter Makuladegeneration (nasses AMD) entwickelt wurde. Die Behandlung zielt darauf ab, einen unerfüllten Bedarf zu decken, insbesondere für die 30-40 % der Patienten, die nicht auf die derzeitigen anti-VEGF-Therapien ansprechen. Diese Erkrankungen betreffen etwa 12 Millionen Menschen in den USA und 130 Millionen weltweit.
Das Unternehmen plant, die Genehmigung von OCU200 als Erstlinientherapie für alle drei Erkrankungen anzustreben, wobei die Nachsorge der Patienten bis zu drei Monate nach der Behandlung reicht.
- Initiated Phase 1 clinical trial with first patient dosed
- Addresses large market opportunity (12M US patients, 130M worldwide)
- Potential to treat 30-40% of patients who don't respond to current therapies
- Early-stage clinical trial (Phase 1) with long development pathway ahead
- Safety and efficacy data not yet available
Insights
This Phase 1 trial initiation for OCU200 represents a critical milestone in developing a potentially groundbreaking treatment for retinal diseases. The trial's design, incorporating three dose cohorts (0.025 mg, 0.05 mg and 0.1 mg) with two intravitreal injections six weeks apart, is well-structured to establish the safety profile and optimal dosing regimen.
The dual-action mechanism of OCU200 is particularly innovative - combining tumstatin's anti-inflammatory and anti-VEGF properties with transferrin's targeted delivery system. This approach could potentially address the 30-40% non-response rate to current anti-VEGF therapies, targeting a significant market gap. The addressable market is substantial, with 12 million patients in the US and 130 million worldwide affected by DME, DR, or wet AMD.
For general understanding: Think of OCU200 as a "smart missile" - the transferrin part acts as the guidance system, ensuring the medicine reaches the right place in the eye, while tumstatin is the payload that fights the disease. Current treatments are more like "carpet bombing" - less precise and potentially requiring higher doses to be effective.
This clinical milestone could significantly impact OCGN's market position in the ophthalmology space. The current global anti-VEGF market is valued at approximately
The addressable market size of 130 million patients globally represents a substantial revenue opportunity. Even capturing a modest market share could translate to significant returns, given typical pricing for novel retinal therapeutics. For context: current anti-VEGF treatments can cost
Simple explanation: Imagine a restaurant introducing a new dish that not only tastes better but also costs less to make and serves more customers who couldn't eat the previous options. That's what OCU200 could potentially do in the eye treatment market - better treatment, potentially lower costs and helping more patients.
MALVERN, Pa., Jan. 16, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the first patient has been dosed in the OCU200 Phase 1 clinical trial for diabetic macular edema (DME).
“OCU200 has the potential to change the treatment landscape for DME, diabetic retinopathy (DR), and wet age-related macular degeneration (wet AMD) with its unique mechanism of action, binding the active component—tumstatin—to integrin receptors that play a crucial role in disease pathogenesis,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen. “OCU200 holds the promise to benefit all DME patients, including the 30
The OCU200 Phase 1 clinical trial is a multicenter, open-label, dose-escalation study to assess drug safety via intravitreal injection in three cohorts: low dose (0.025 mg), medium dose (0.05 mg), and high dose (0.1 mg). All subjects will receive a total of two intravitreal injections of OCU200 six weeks apart. Patient follow-up will take place up to three months after the last injection.
Approximately 12 million people in the United States and 130 million people worldwide are affected by DME, DR or wet AMD. Patients affected by these diseases share common symptoms, such as blurriness in vision and progressive vision loss as the disease progresses. The formation of fragile and leaky new blood vessels leads to fluid accumulation in and around the retina, causing damage to vision.
“I am seeing an increasing rate of vision-threatening diseases associated with diabetes at my clinic and am eager to provide a new therapeutic option to these patients,” said Dr. David Almedia, Vitreoretinal Surgeon and Clinician Scientist, President and CEO of Erie Retina Research, and Founder and President of Case X Global in Erie, Pennsylvania. “There remains a considerable unmet medical need for DME and DR patients with currently available anti-VEGF treatments.”
OCU200 is a recombinant fusion protein that consists of two parts connected by a linker: tumstatin, the active component, acts as an anti-inflammatory, anti-VEGF agent by binding to integrin receptors; and transferrin, which targets the drug to the choroid and retina by binding transferrin receptors on endothelial cells. These features will potentially enable OCU200 to reduce the vascular permeability, inflammation, and neovascularization that drive the pathophysiology of DME, DR, and wet AMD at a significantly lower dose compared to currently approved therapies.
“We are enthusiastic about getting patients started in the OCU200 Phase 1 clinical trial and sharing not only safety but preliminary efficacy data as the study progresses,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “OCU200 brings an innovative biologic candidate to Ocugen’s ophthalmology portfolio targeting blindness diseases.”
The Company intends to pursue approval to use OCU200 as a first-line therapy for DME, DR, and wet AMD.
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.
Cautionary Note on 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; the ability of OCU200 to perform in humans in a manner consistent with nonclinical or preclinical study 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.
Contact:
Tiffany Hamilton
Head of Communications
Tiffany.Hamilton@ocugen.com
FAQ
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