Data and Safety Monitoring Board Reviews Cohort 1 Safety Data and Approves Dosing Cohort 2 in the Clinical Trial of OCU200—a Novel Fusion Protein for Diabetic Macular Edema
Ocugen (NASDAQ: OCGN) announced positive safety data from the first cohort of its Phase 1 clinical trial for OCU200, a novel fusion protein designed to treat diabetic macular edema (DME). The Data and Safety Monitoring Board (DSMB) has approved proceeding with the second cohort after reviewing safety data from the initial group.
The trial is structured as a multicenter, open-label, dose-escalation study across three cohorts: low dose (0.025 mg), medium dose (0.05 mg), and high dose (0.1 mg). Patients receive two intravitreal injections six weeks apart with a 6-month follow-up period. No serious adverse events related to OCU200 have been reported.
OCU200, combining tumstatin and transferrin proteins, targets approximately 12 million people in the US and 130 million worldwide affected by DME, diabetic retinopathy (DR), and wet age-related macular degeneration. The company aims to complete the Phase 1 trial in H2 2025, particularly addressing the 30-40% of DME patients who don't respond to current anti-VEGF therapies.
Ocugen (NASDAQ: OCGN) ha annunciato dati positivi sulla sicurezza dal primo gruppo del suo studio clinico di fase 1 per OCU200, una nuova proteina di fusione progettata per trattare l'edema maculare diabetico (DME). Il Comitato per il Monitoraggio dei Dati e della Sicurezza (DSMB) ha approvato il proseguimento con il secondo gruppo dopo aver esaminato i dati di sicurezza del gruppo iniziale.
Lo studio è strutturato come uno studio multicentrico, open-label, di escalation della dose attraverso tre gruppi: bassa dose (0,025 mg), dose media (0,05 mg) e alta dose (0,1 mg). I pazienti ricevono due iniezioni intravitreali a sei settimane di distanza con un periodo di follow-up di 6 mesi. Non sono stati segnalati eventi avversi gravi correlati a OCU200.
OCU200, che combina le proteine tumstatina e transferrina, mira a circa 12 milioni di persone negli Stati Uniti e 130 milioni a livello mondiale colpite da DME, retinopatia diabetica (DR) e degenerazione maculare senile umida. L'azienda prevede di completare lo studio di fase 1 nel secondo semestre del 2025, affrontando in particolare il 30-40% dei pazienti con DME che non rispondono alle attuali terapie anti-VEGF.
Ocugen (NASDAQ: OCGN) anunció datos positivos de seguridad del primer grupo de su ensayo clínico de fase 1 para OCU200, una nueva proteína de fusión diseñada para tratar el edema macular diabético (DME). El Comité de Monitoreo de Datos y Seguridad (DSMB) ha aprobado continuar con el segundo grupo tras revisar los datos de seguridad del grupo inicial.
El ensayo está estructurado como un estudio multicéntrico, abierto y de escalado de dosis a través de tres grupos: dosis baja (0.025 mg), dosis media (0.05 mg) y dosis alta (0.1 mg). Los pacientes reciben dos inyecciones intravítreas con seis semanas de diferencia y un período de seguimiento de 6 meses. No se han reportado eventos adversos graves relacionados con OCU200.
OCU200, que combina proteínas de tumstatina y transferrina, tiene como objetivo a aproximadamente 12 millones de personas en EE. UU. y 130 millones en todo el mundo afectadas por DME, retinopatía diabética (DR) y degeneración macular húmeda relacionada con la edad. La empresa espera completar el ensayo de fase 1 en el segundo semestre de 2025, abordando especialmente el 30-40% de los pacientes con DME que no responden a las terapias anti-VEGF actuales.
Ocugen (NASDAQ: OCGN)은 당뇨병성 황반 부종(DME) 치료를 위해 설계된 새로운 융합 단백질 OCU200의 1상 임상 시험 첫 번째 집단에서 긍정적인 안전성 데이터를 발표했습니다. 데이터 및 안전성 모니터링 위원회(DSMB)는 초기 집단의 안전성 데이터를 검토한 후 두 번째 집단으로 진행하는 것을 승인했습니다.
이 시험은 저용량(0.025 mg), 중용량(0.05 mg), 고용량(0.1 mg)의 세 집단으로 구성된 다기관 개방형 용량 증량 연구로 구조화되어 있습니다. 환자들은 6주 간격으로 두 번의 유리체 내 주사를 받고 6개월의 추적 관찰 기간을 가집니다. OCU200과 관련된 심각한 부작용은 보고되지 않았습니다.
OCU200은 튐스타틴과 트랜스페린 단백질을 결합하여 DME, 당뇨병성 망막병증(DR), 노인성 습성 황반변성을 앓고 있는 미국 내 약 1,200만 명과 전 세계 1억 3천만 명을 대상으로 합니다. 이 회사는 2025년 하반기까지 1상 시험을 완료할 계획이며, 현재의 항-VEGF 치료에 반응하지 않는 DME 환자의 30-40%를 특히 다루고자 합니다.
Ocugen (NASDAQ: OCGN) a annoncé des données de sécurité positives de la première cohorte de son essai clinique de phase 1 pour OCU200, une nouvelle protéine de fusion conçue pour traiter l'œdème maculaire diabétique (DME). Le Comité de Surveillance des Données et de la Sécurité (DSMB) a approuvé la poursuite avec la deuxième cohorte après avoir examiné les données de sécurité du groupe initial.
L'essai est structuré comme une étude multicentrique, ouverte et d'escalade de dose à travers trois cohortes : faible dose (0,025 mg), dose moyenne (0,05 mg) et forte dose (0,1 mg). Les patients reçoivent deux injections intravitréennes à six semaines d'intervalle avec une période de suivi de 6 mois. Aucun événement indésirable grave lié à OCU200 n'a été signalé.
OCU200, combinant les protéines tumstatine et transferrine, cible environ 12 millions de personnes aux États-Unis et 130 millions dans le monde touchées par le DME, la rétinopathie diabétique (DR) et la dégénérescence maculaire liée à l'âge humide. L'entreprise vise à terminer l'essai de phase 1 au second semestre 2025, en s'attaquant particulièrement aux 30 à 40 % des patients atteints de DME qui ne répondent pas aux thérapies anti-VEGF actuelles.
Ocugen (NASDAQ: OCGN) hat positive Sicherheitsdaten aus der ersten Kohorte seiner Phase-1-Studie für OCU200 bekannt gegeben, einem neuartigen Fusionsprotein, das zur Behandlung von diabetischem Makulaödem (DME) entwickelt wurde. Das Data and Safety Monitoring Board (DSMB) hat die Fortsetzung mit der zweiten Kohorte genehmigt, nachdem es die Sicherheitsdaten der ursprünglichen Gruppe überprüft hat.
Die Studie ist als multizentrische, offene, dosissteigernde Untersuchung über drei Kohorten strukturiert: niedrige Dosis (0,025 mg), mittlere Dosis (0,05 mg) und hohe Dosis (0,1 mg). Die Patienten erhalten zwei intravitrealen Injektionen im Abstand von sechs Wochen mit einer Nachbeobachtungszeit von 6 Monaten. Es wurden keine schwerwiegenden unerwünschten Ereignisse im Zusammenhang mit OCU200 berichtet.
OCU200, das Tumstatin- und Transferrin-Proteine kombiniert, zielt auf etwa 12 Millionen Menschen in den USA und 130 Millionen weltweit ab, die von DME, diabetischer Retinopathie (DR) und feuchter altersbedingter Makuladegeneration betroffen sind. Das Unternehmen plant, die Phase-1-Studie im zweiten Halbjahr 2025 abzuschließen, insbesondere für die 30-40 % der DME-Patienten, die nicht auf die aktuellen Anti-VEGF-Therapien ansprechen.
- DSMB approved progression to second cohort after favorable safety review
- No serious adverse events reported in first cohort
- Addresses large market of 12M US patients and 130M globally
- Potential solution for 30-40% of DME patients unresponsive to current treatments
- Early-stage Phase 1 trial with no efficacy data yet
- Complete trial results not expected until H2 2025
Insights
Ocugen's announcement regarding the OCU200 clinical trial represents a positive early milestone in their development program. The Data and Safety Monitoring Board's approval to proceed to the second cohort indicates the drug demonstrated acceptable safety in the first cohort of this Phase 1 trial, which is important for novel biologics. While this is still very early-stage data, the clean safety profile with no serious adverse events is encouraging.
The OCU200 compound is scientifically interesting as it combines tumstatin and transferrin proteins, targeting integrin receptors on endothelial cells - a different mechanism than current standard-of-care anti-VEGF therapies. This potentially addresses the 30-40% of DME patients who don't respond to existing treatments.
Market opportunity is substantial with approximately 12 million people in the US and 130 million worldwide affected by the target conditions (DME, DR, wet AMD). However, investors should recognize several remaining hurdles: this is only a Phase 1 safety study with efficacy data still pending, the trial includes just three small cohorts, and completion isn't expected until H2 2025. The favorable safety profile is necessary but not sufficient for ultimate commercial success, as efficacy will be the determinant in later trials.
This Phase 1 clinical trial update for OCU200 demonstrates typical early-stage progression with safety as the primary endpoint. The DSMB approval to advance to cohort 2 following clean safety data from cohort 1 is procedurally significant, though expected in the absence of serious adverse events. The intravitreal delivery method is standard for retinal therapeutics, presenting minimal novelty in administration.
The trial design follows conventional dose-escalation methodology with three cohorts (0.025mg, 0.05mg, 0.1mg), two administrations at 6-week intervals, and a 6-month follow-up. This design is appropriate for generating preliminary safety data but in detecting efficacy signals.
OCU200's differentiated mechanism targeting integrin receptors rather than VEGF pathways potentially addresses an important treatment gap, as current anti-VEGF therapies fail in 30-40% of DME cases. However, while the company suggests potential applications across multiple retinal conditions (DME, DR, wet AMD), this Phase 1 trial appears focused solely on DME. The projected completion in H2 2025 puts any meaningful efficacy readouts at least 6-9 months away. While the safety milestone is positive, the true clinical and commercial potential remains unproven until efficacy data emerges in subsequent development phases.
- OCU200 has a very favorable safety and tolerability profile
- No serious adverse events related to the study drug have been reported
- Dosing of the second cohort has been approved
MALVERN, Pa., March 18, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the Data and Safety Monitoring Board (DSMB) for the OCU200 clinical trial recently convened and reviewed safety data following dosing of the first cohort in the dose-escalation portion of the Phase 1 study and approved continuation of dosing in the second cohort. OCU200 is a novel fusion protein consisting of two human proteins, tumstatin and transferrin, with the potential to treat diabetic macular edema (DME).
“OCU200 is given intravitreally,” said Peter Chang, MD, FACS, Co-President and Partner of the Massachusetts Eye Research and Surgery Institution (MERSI). “No serious adverse events related to OCU200 have been reported to date.”
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 two doses six weeks apart, and patients will be followed for up to 6 months.
“It is encouraging that we have successfully completed dosing in the low dose cohort for OCU200, a novel biologic that has a very favorable safety and tolerability profile,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “There remains a considerable unmet medical need for the
Approximately 12 million people in the United States and 130 million people worldwide are affected by DME, DR, or wet AMD. Patients affected by them share common symptoms, such as blurriness in vision and progressive vision loss, as the diseases progress. The formation of fragile and leaky new blood vessels leads to fluid accumulation in and around the retina, causing damage to vision.
OCU200 has the potential to change the treatment landscape for DME, DR, and wet AMD with its unique mechanism of action, binding the active component—tumstatin—to integrin receptors on active endothelial cells that play a crucial role in disease pathogenesis.
OCU200 brings an innovative biologic candidate to Ocugen’s ophthalmology portfolio targeting blindness diseases. The Company intends to complete the Phase 1 OCU200 clinical trial in the second half of 2025 and to provide preliminary safety and efficacy updates throughout the year.
About OCU200
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.
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
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com
FAQ
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