Opthea Announces Publication in Diabetic Macular Edema
Opthea (OPT) has published Phase 1b clinical trial results for sozinibercept combination therapy in diabetic macular edema (DME) in the Translational Vision Science & Technology journal. The study evaluated nine patients receiving sozinibercept (0.3, 1, or 2 mg) combined with aflibercept (2 mg) every four weeks for twelve weeks.
The trial demonstrated that sozinibercept was well-tolerated with no dose-limiting toxicities. Results showed a dose-response relationship with increased visual acuity gains at higher doses, with the 2 mg combination showing the best results. All doses achieved meaningful central subfield thickness reduction.
The company plans to advance its DME program following anticipated Phase 3 topline data readouts for wet AMD from the COAST study in early Q2 2025 and ShORe in mid-2025. DME affects approximately 19 million people globally, with projections reaching 29 million by 2045.
Opthea (OPT) ha pubblicato i risultati della fase 1b di uno studio clinico sulla terapia combinata con sozinibercept per il edema maculare diabetico (DME) nella rivista Translational Vision Science & Technology. Lo studio ha valutato nove pazienti che ricevevano sozinibercept (0,3, 1 o 2 mg) combinato con aflibercept (2 mg) ogni quattro settimane per dodici settimane.
La sperimentazione ha dimostrato che sozinibercept è stato ben tollerato, senza tossicità limitanti della dose. I risultati hanno mostrato una relazione dose-risposta con un aumento dell'acutezza visiva alle dosi più elevate, con la combinazione da 2 mg che ha mostrato i risultati migliori. Tutte le dosi hanno raggiunto una significativa riduzione dello spessore del sottocampo centrale.
L'azienda prevede di avanzare il suo programma DME in seguito ai dati preliminari attesi dalla fase 3 per l'AMD umido dallo studio COAST all'inizio del secondo trimestre del 2025 e dallo studio ShORe a metà del 2025. Il DME colpisce circa 19 milioni di persone a livello globale, con proiezioni che raggiungono 29 milioni entro il 2045.
Opthea (OPT) ha publicado los resultados del ensayo clínico de fase 1b para la terapia combinada con sozinibercept en edema macular diabético (DME) en la revista Translational Vision Science & Technology. El estudio evaluó a nueve pacientes que recibieron sozinibercept (0,3, 1 o 2 mg) combinado con aflibercept (2 mg) cada cuatro semanas durante doce semanas.
El ensayo demostró que sozinibercept fue bien tolerado, sin toxicidades limitantes de la dosis. Los resultados mostraron una relación dosis-respuesta, con aumentos en la agudeza visual a dosis más altas, siendo la combinación de 2 mg la que mostró los mejores resultados. Todas las dosis lograron una reducción significativa del grosor del subcampo central.
La empresa planea avanzar con su programa de DME tras la esperada lectura de los datos de fase 3 para la AMD húmeda del estudio COAST a principios del segundo trimestre de 2025 y de ShORe a mediados de 2025. El DME afecta aproximadamente a 19 millones de personas en todo el mundo, con proyecciones que alcanzan los 29 millones para 2045.
옵테아 (OPT)는 당뇨병성 황반 부종 (DME)에 대한 소지니버셉트 병합 요법의 1b상 임상 시험 결과를 Translational Vision Science & Technology 저널에 발표했습니다. 이 연구는 12주간 4주마다 0.3, 1 또는 2mg의 소지니버셉트와 2mg의 아플리버셉트를 병용 치료받은 9명의 환자를 평가했습니다.
시험 결과 소지니버셉트는 잘 견딜 수 있으며 용량 한계를 초과하는 독성이 없음을 보여주었습니다. 결과는 고용량에서 시각적 선명도가 증가하는 용량-반응 관계를 보여주었고, 2mg 조합이 가장 좋은 결과를 보였습니다. 모든 용량에서 중심 하위 영역 두께의 중요한 감소가 나타났습니다.
회사는 2025년 2분기 초에 있을 COAST 연구의 습성 AMD에 대한 3상 주요 데이터 공개와 2025년 중반의 ShORe 연구 발표를 따라 DME 프로그램을 진행할 계획입니다. DME는 전 세계적으로 약 1,900만 명에게 영향을 미치며, 2045년까지 2,900만 명에 이를 것으로 예상됩니다.
Opthea (OPT) a publié les résultats des essais cliniques de phase 1b concernant la thérapie combinée avec sozinibercept pour l'œdème maculaire diabétique (DME) dans la revue Translational Vision Science & Technology. L'étude a évalué neuf patients recevant du sozinibercept (0,3, 1 ou 2 mg) en combinaison avec de l'aflibercept (2 mg) toutes les quatre semaines pendant douze semaines.
L'essai a démontré que le sozinibercept était bien toléré, sans toxicités limitantes de dose. Les résultats ont montré une relation dose-réponse avec des gains d'acuité visuelle accrus à des doses plus élevées, la combinaison de 2 mg montrant les meilleurs résultats. Toutes les doses ont permis une réduction significative de l'épaisseur du sous-champ central.
L'entreprise prévoit d'avancer son programme DME suite aux résultats prévus de phase 3 pour l'AMD humide issus de l'étude COAST au début du deuxième trimestre 2025 et de l'étude ShORe à la mi-2025. Le DME touche environ 19 millions de personnes dans le monde, avec des projections atteignant 29 millions d'ici 2045.
Opthea (OPT) hat die Ergebnisse der Phase 1b klinischen Studie zur Kombinationstherapie mit Sozinibercept bei diabetischem Makulaödem (DME) in der Fachzeitschrift Translational Vision Science & Technology veröffentlicht. Die Studie untersuchte neun Patienten, die Sozinibercept (0,3, 1 oder 2 mg) in Kombination mit Aflibercept (2 mg) alle vier Wochen über einen Zeitraum von zwölf Wochen erhielten.
Die Studie zeigte, dass Sozinibercept gut vertragen wurde, ohne dass dosislimitierende Toxizitäten auftraten. Die Ergebnisse zeigten eine Dosis-Wirkungs-Beziehung mit erhöhten Sehschärfegewinnen bei höheren Dosen, wobei die Kombination von 2 mg die besten Ergebnisse zeigte. Alle Dosen führten zu einer signifikanten Reduzierung der zentralen Subfelddicke.
Das Unternehmen plant, sein DME-Programm voranzutreiben, nachdem die erwarteten Ergebnisse der Phase 3 für die feuchte AMD aus der COAST-Studie Anfang des zweiten Quartals 2025 und aus der ShORe-Studie Mitte 2025 vorliegen werden. DME betrifft weltweit etwa 19 Millionen Menschen, mit Prognosen, die bis 2045 29 Millionen erreichen werden.
- Phase 1b trial showed positive safety profile with no dose-limiting toxicities
- Demonstrated dose-dependent improvement in visual acuity
- All tested doses achieved meaningful reduction in central subfield thickness
- Addresses large market opportunity with 19 million DME patients globally
- Small trial size of only 9 patients limits statistical significance
- Commercial launch timeline extends beyond 2025
- Must await completion of wet AMD trials before advancing DME program
Insights
This Phase 1b clinical trial publication marks a important milestone for sozinibercept in DME treatment. The study's design targeting patients with persistent DME who previously received anti-VEGF-A therapy is particularly noteworthy. The dose-response relationship observed with increasing BCVA gains at higher sozinibercept doses (2mg showing best results) provides compelling evidence for the drug's biological activity.
The dual mechanism targeting both VEGF-C/D pathways, while combining with standard anti-VEGF-A therapy (aflibercept), represents an innovative approach. The demonstrated safety profile and meaningful CST reduction across all dose levels establish a solid foundation for future development. With 19 million DME patients globally and projections reaching 29 million by 2045, the market opportunity is substantial.
Critical near-term catalysts include the wet AMD Phase 3 COAST and ShORe trial readouts in Q2 2025 and mid-2025 respectively. These results will likely influence the DME program's progression and overall platform validation.
The strategic positioning of sozinibercept in the DME market shows significant commercial potential. Currently dominated by anti-VEGF-A therapies, the DME treatment landscape presents a clear opportunity for innovative combination approaches. The publication in TVST and inclusion in ARVO's special issue adds scientific credibility and visibility within the ophthalmology community.
The focus on patients with persistent DME is particularly strategic, addressing a key market gap where current treatments show suboptimal results. This positions sozinibercept as a potential solution for both treatment-naive patients and those with inadequate response to existing therapies. The rapidly growing diabetic population provides a robust growth trajectory for the DME market, making this an attractive commercial opportunity for Opthea's pipeline expansion beyond wet AMD.
Phase 1b clinical data underpins sozinibercept’s potential as a novel, first-in-class VEGF-C/D ‘trap’ to improve visual and anatomic outcomes in DME
Published in peer-reviewed journal Translational Vision Science & Technology and included in the Anti-VEGF Special Journal Issue of ARVO
DME program to be advanced after wet AMD topline data readout anticipated for COAST in early CY Q2 2025 and ShORe in mid-2025
MELBOURNE, Australia and PRINCETON, N.J., Jan. 07, 2025 (GLOBE NEWSWIRE) -- Opthea Limited (ASX/NASDAQ: OPT “Opthea”, the “Company”), a clinical-stage biopharmaceutical company developing novel therapies to treat highly prevalent and progressive retinal diseases, including wet age-related macular degeneration (wet AMD), today announced the publication of its Phase 1b trial of sozinibercept combination therapy in diabetic macular edema (DME) in the peer-reviewed journal Translational Vision Science & Technology (TVST), issued on December 19, 2024.
The publication, Phase 1b Dose Escalation Study of Sozinibercept Inhibition of Vascular Endothelial Growth Factors C and D With Aflibercept for Diabetic Macular Edema, evaluated the outcomes in previously anti-VEGF-A monotherapy treated patients with persistent DME, a difficult-to-treat patient population. The article was also included in the Anti-VEGF Special Journal Issue of TVST, the official Journal of The Association for Research in Vision and Ophthalmology (ARVO).
“The DME trial results underpin sozinibercept’s potential as a novel, first-in-class VEGF-C/D ‘trap’ to elevate the standard of care in retinal diseases including DME, by preventing blood vessel growth and vascular leakage in the retina and delivering improved visual and anatomic outcomes when combined with standard-of-care anti-VEGF-A therapies,” said Frederic Guerard, PharmD, Chief Executive Officer of Opthea. “Whilst our immediate focus is to prepare for the anticipated sozinibercept Phase 3 topline data readout in wet AMD of COAST in early CY Q2 2025 and ShORe in mid-2025, we also plan to advance our clinical development program of sozinibercept in DME.”
In this first-in-human, open-label, multicenter, dose escalating Phase 1b DME trial, nine patients received sozinibercept (0.3, 1, or 2 mg) in combination with aflibercept (2 mg) once every four weeks for twelve weeks. The primary endpoint was safety, and the secondary endpoints included mean change from baseline in best-corrected visual acuity (BCVA) and anatomical parameters, including central subfield thickness (CST). Sozinibercept combination therapy was well tolerated with no dose-limiting toxicities. The trial also demonstrated a dose-response relationship of increased gains in BCVA for ascending doses of sozinibercept, with the 2 mg sozinibercept combination arm demonstrating the highest BCVA gain. All sozinibercept doses demonstrated a meaningful CST reduction. These data, published in TVST, support the further investigation of sozinibercept as a treatment for DME, a disease with rapidly increasing prevalence and unmet medical need.
Diabetic macular edema is the leading cause of central vision loss in people living with diabetes. It is estimated to affect around 19 million people worldwide, and with the rise of diabetes, the prevalence is expected to increase to 29 million by 2045. DME occurs when blood vessels in the retina swell and leak, leading to fluid accumulation in the retina.
Anti-VEGF-A therapies are the current standard of care for DME patients. There is an unmet need for early intervention to prevent irreversible damage of the retina, as well as improve visual outcomes for patients treated with anti-VEGF-A therapies alone, which may lead to suboptimal vision outcomes.
About Opthea
Opthea (ASX/NASDAQ:OPT) is a biopharmaceutical company developing novel therapies to address the unmet needs in the treatment of highly prevalent and progressive retinal diseases, including wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME).
Opthea’s lead product candidate, sozinibercept, is being evaluated in two fully enrolled pivotal Phase 3 clinical trials (COAST, NCT04757636, and ShORe, NCT04757610) for use in combination with standard-of-care anti-VEGF-A therapies to improve the overall efficacy and deliver superior vision gains compared to standard-of-care anti-VEGF-A agents alone.
To learn more, visit our website at www.opthea.com and follow us on X and LinkedIn.
Forward-Looking Statements
This ASX announcement contains certain forward-looking statements, including within the meaning of the US Private Securities Litigation Reform Act of 1995. The words “expect”, “believe”, “should”, “could”, “may”, “will”, “plan” and other similar expressions are intended to identify forward-looking statements. Forward-looking statements in this ASX announcement include statements regarding the potential importance of VEGF-C and VEGF-D signaling pathways to the pathogenesis of retinal diseases, sozinibercept’s potential as a novel, first-in-class VEGF-C/D ‘trap’ to improve visual and anatomic outcomes in DME and prevent blood vessel growth and vascular leakage in the retina, sozinibercept’s potential to deliver superior visual outcomes in wet AMD when combined with standard-of-care anti-VEGF-A therapies, the potential for the registrational program for sozinibercept in wet AMD, and the anticipated timing for the results of the COAST and ShORe Phase 3 wet AMD trials and the potential for such results to support a broad label and, if successful, enable sozinibercept to be approved for use in combination with any anti-VEGF-A for the treatment of wet AMD patients and sozinibercept’s clinical development in and potential as a treatment for diabetic macular edema and the expected prevalence of DME. Forward-looking statements, opinions and estimates provided in this ASX announcement are based on assumptions and contingencies which are subject to change without notice, as are statements about market and industry trends, which are based on interpretations of current conditions. Forward-looking statements are provided as a general guide only and should not be relied upon as an indication or guarantee of future performance. They involve known and unknown risks and uncertainties and other factors, many of which are beyond the control of Opthea and its directors and management and may involve significant elements of subjective judgment and assumptions as to future events that may or may not be correct. These statements may be affected by a range of variables which could cause actual results or trends to differ materially, including but not limited to future capital requirements, the development, testing, production, marketing and sale of drug treatments, regulatory risk and potential loss of regulatory approvals, ongoing clinical studies to demonstrate sozinibercept’s safety, tolerability and therapeutic efficacy, clinical research organization and labor costs, intellectual property protections, and other factors that are of a general nature which may affect the future operating and financial performance of the Company including risk factors set forth in Opthea’s Annual Report on Form 20-F filed with the US Securities and Exchange Commission (the “SEC”) on August 30, 2024, and other future filings with the SEC. Actual results, performance or achievement may vary materially from any projections and forward-looking statements and the assumptions on which those statements are based. Subject to any continuing obligations under applicable law or any relevant ASX listing rules, Opthea disclaims any obligation or undertaking to provide any updates or revisions to any forward-looking statements in this ASX announcement to reflect any change in expectations in relation to any forward-looking statements or any change in events, conditions or circumstances on which any such statement is based, except as otherwise required by applicable law.
Authorized for release to ASX by Frederic Guerard, CEO
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Source: Opthea Limited
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