Opthea Announces Phase 2b Wet AMD Publication
Opthea (ASX/NASDAQ: OPT) has published Phase 2b trial data for sozinibercept in the peer-reviewed journal Ophthalmic Surgery, Lasers and Imaging Retina. The study analyzed predictors of response to sozinibercept combination therapy with ranibizumab in treatment-naïve wet AMD patients.
Key findings revealed that in patients with occult and minimally classic lesions excluding RAP (73% of study population), the combination therapy showed a statistically significant 5.7 letter improvement in best corrected visual acuity compared to ranibizumab alone. These patients also demonstrated higher rates of ≥15 letter gains and improved retinal anatomy with reduced CNV area at week 24.
The company's ongoing Phase 3 trials, COAST and ShORe, have been designed based on these findings, with topline data expected in early Q2 CY 2025 and mid-CY 2025, respectively. Wet AMD affects approximately 3.5 million people in the US and Europe, with many patients still not achieving optimal vision outcomes with current anti-VEGF-A therapies.
Opthea (ASX/NASDAQ: OPT) ha pubblicato i dati della sperimentazione di fase 2b per sozinibercept nella rivista peer-reviewed Ophthalmic Surgery, Lasers and Imaging Retina. Lo studio ha analizzato i predittori di risposta alla terapia combinata con sozinibercept e ranibizumab in pazienti con AMD umida naïve al trattamento.
I risultati chiave hanno rivelato che nei pazienti con lesioni occulte e minimamente classiche, escluse le RAP (73% della popolazione dello studio), la terapia combinata ha mostrato un miglioramento statisticamente significativo di 5,7 lettere nell'acuità visiva corretta migliore rispetto al solo ranibizumab. Questi pazienti hanno anche dimostrato tassi più elevati di guadagni ≥15 lettere e un miglioramento dell'anatomia retinica con riduzione dell'area CNV alla settimana 24.
Le sperimentazioni di fase 3 in corso dell'azienda, COAST e ShORe, sono state progettate sulla base di questi risultati, con dati preliminari attesi all'inizio del secondo trimestre del 2025 e a metà del 2025, rispettivamente. L'AMD umida colpisce circa 3,5 milioni di persone negli Stati Uniti e in Europa, con molti pazienti che non raggiungono ancora risultati visivi ottimali con le attuali terapie anti-VEGF-A.
Opthea (ASX/NASDAQ: OPT) ha publicado los datos del ensayo de fase 2b para sozinibercept en la revista revisada por pares Ophthalmic Surgery, Lasers and Imaging Retina. El estudio analizó los predictores de respuesta a la terapia combinada de sozinibercept con ranibizumab en pacientes con AMD húmeda que no habían recibido tratamiento.
Los hallazgos clave revelaron que en pacientes con lesiones ocultas y mínimamente clásicas, excluyendo las RAP (73% de la población del estudio), la terapia combinada mostró una mejora estadísticamente significativa de 5.7 letras en la agudeza visual corregida en comparación con ranibizumab solo. Estos pacientes también demostraron tasas más altas de ganancias de ≥15 letras y una anatomía retiniana mejorada con reducción del área CNV en la semana 24.
Los ensayos en fase 3 en curso de la compañía, COAST y ShORe, han sido diseñados en base a estos hallazgos, con datos preliminares esperados a principios del segundo trimestre de 2025 y a mediados de 2025, respectivamente. La AMD húmeda afecta aproximadamente a 3.5 millones de personas en EE. UU. y Europa, con muchos pacientes que aún no logran resultados visuales óptimos con las terapias anti-VEGF-A actuales.
옵테아 (ASX/NASDAQ: OPT)는 소지니버셉트에 대한 2b상 시험 데이터를 동료 심사 저널인 Ophthalmic Surgery, Lasers and Imaging Retina에 발표했습니다. 이 연구는 치료를 받지 않은 습식 AMD 환자에서 소지니버셉트와 라니비주맙의 병용 요법에 대한 반응 예측 인자를 분석했습니다.
주요 발견은 RAP를 제외한 잠재적이고 최소한의 고전적 병변을 가진 환자(연구 인구의 73%)에서 병용 요법이 라니비주맙 단독에 비해 통계적으로 유의미한 5.7자 개선을 보였다는 것입니다. 이 환자들은 또한 ≥15자 증가율이 더 높고 24주 차에 CNV 영역이 감소하면서 망막 해부학이 개선되었습니다.
회사의 진행 중인 3상 시험인 COAST와 ShORe는 이러한 발견을 기반으로 설계되었으며, 각각 2025년 2분기 초와 2025년 중반에 주요 데이터가 예상됩니다. 습식 AMD는 미국과 유럽에서 약 350만 명에게 영향을 미치며, 많은 환자들이 현재의 항-VEGF-A 치료로 최적의 시각 결과를 달성하지 못하고 있습니다.
Opthea (ASX/NASDAQ: OPT) a publié les données de l'essai de phase 2b pour sozinibercept dans la revue évaluée par des pairs Ophthalmic Surgery, Lasers and Imaging Retina. L'étude a analysé les prédicteurs de réponse à la thérapie combinée sozinibercept et ranibizumab chez des patients naïfs de traitement atteints de DMLA humide.
Les résultats clés ont révélé que chez les patients présentant des lésions occultes et minimales classiques, excluant les RAP (73 % de la population de l'étude), la thérapie combinée a montré une amélioration statistiquement significative de 5,7 lettres en acuité visuelle corrigée par rapport au ranibizumab seul. Ces patients ont également démontré des taux plus élevés de gains ≥15 lettres et une anatomie rétinienne améliorée avec une réduction de la zone CNV à la semaine 24.
Les essais de phase 3 en cours de l'entreprise, COAST et ShORe, ont été conçus sur la base de ces résultats, avec des données préliminaires attendues au début du deuxième trimestre 2025 et à la mi-2025, respectivement. La DMLA humide touche environ 3,5 millions de personnes aux États-Unis et en Europe, de nombreux patients n'atteignant toujours pas des résultats visuels optimaux avec les thérapies anti-VEGF-A actuelles.
Opthea (ASX/NASDAQ: OPT) hat die Phase-2b-Studienergebnisse für Sozinibercept in der von Experten begutachteten Zeitschrift Ophthalmic Surgery, Lasers and Imaging Retina veröffentlicht. Die Studie analysierte Prädiktoren für die Reaktion auf die Kombinationstherapie mit Sozinibercept und Ranibizumab bei behandlungsnaiven Patienten mit feuchter AMD.
Wichtige Ergebnisse zeigten, dass bei Patienten mit okkulten und minimal klassischen Läsionen, die RAP ausschlossen (73% der Studienpopulation), die Kombinationstherapie eine statistisch signifikante Verbesserung von 5,7 Buchstaben in der besten korrigierten Sehschärfe im Vergleich zu Ranibizumab allein aufwies. Diese Patienten zeigten auch höhere Raten von ≥15 Buchstaben Gewinnen und eine verbesserte Netzhautanatomie mit reduzierter CNV-Fläche in der Woche 24.
Die laufenden Phase-3-Studien des Unternehmens, COAST und ShORe, wurden auf der Grundlage dieser Ergebnisse entworfen, wobei die vorläufigen Daten für Anfang des zweiten Quartals 2025 und Mitte 2025 erwartet werden. Feuchte AMD betrifft etwa 3,5 Millionen Menschen in den USA und Europa, wobei viele Patienten mit den aktuellen Anti-VEGF-A-Therapien immer noch nicht optimale Sehergebnisse erzielen.
- Phase 2b trial showed significant 5.7 letter vision improvement vs standard therapy
- Higher proportion of patients gained ≥15 letters in vision improvement
- Phase 3 trials enrollment aligns with best-responding patient characteristics
- Large market opportunity with 3.5M patients in US and Europe
- Phase 3 trial results still pending - significant regulatory milestone ahead
- Competing in market with established anti-VEGF-A therapies
Insights
Opthea's publication of Phase 2b data for sozinibercept in a peer-reviewed journal represents a significant clinical milestone that strengthens the company's scientific positioning ahead of upcoming pivotal readouts. The data demonstrates that in a substantial subset of patients (73% of the trial population) with specific lesion characteristics, sozinibercept combination therapy provided a statistically significant 5.7 letter improvement in visual acuity compared to standard-of-care ranibizumab monotherapy.
This publication provides clinical validation for Opthea's targeted approach. By identifying specific angiographic predictors of treatment response, the company has optimized patient selection for their Phase 3 trials, potentially increasing the probability of success. The ability to predict which patients will respond best to therapy is increasingly valuable in the ophthalmology space, where precision medicine approaches can differentiate new entrants in a competitive market.
With topline data from the Phase 3 COAST trial expected in early Q2 2025 (within weeks) and ShORe results mid-year, Opthea is approaching critical inflection points. Wet AMD represents a substantial market opportunity affecting approximately 3.5 million people in the US and Europe alone, with significant unmet needs despite existing treatments. If Phase 3 results replicate these Phase 2b findings, sozinibercept could position itself as a meaningful advance in wet AMD treatment.
The publication of Opthea's sozinibercept Phase 2b data in OSLI Retina provides valuable insights into the drug's mechanism of action and target patient population. The 5.7 letter visual acuity gain over ranibizumab alone in patients with occult and minimally classic lesions (excluding RAP) is clinically meaningful in wet AMD treatment, where even small improvements in vision can significantly impact patient quality of life.
The subgroup analyses revealing predictive lesion characteristics align with real-world observations in retinal practice. By demonstrating superior outcomes in specific CNV lesion types, Opthea has potentially identified a precision medicine approach in retinal disease - an area where treatments have traditionally followed a one-size-fits-all methodology. The improved anatomical outcomes, including better drying of the retina and reduced CNV area, further support sozinibercept's differentiated mechanism targeting both VEGF-A and VEGF-C/D pathways.
The unmet need in wet AMD remains substantial despite current anti-VEGF-A therapies. Many patients experience suboptimal responses, treatment resistance, or require frequent injections. If sozinibercept's combination approach proves successful in the upcoming Phase 3 readouts, it could address key limitations of existing therapies. The company's strategic decision to enrich their Phase 3 trials with patients having these responsive lesion types demonstrates clinical development sophistication that may increase probability of technical success.
Baseline angiographic lesion characteristics predictive of clinical response
Published in peer-reviewed journal Ophthalmic Surgery, Lasers and Imaging Retina
MELBOURNE, Australia and PRINCETON, N.J., March 03, 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 sozinibercept Phase 2b data in the peer-reviewed journal Ophthalmic Surgery, Lasers and Imaging (OSLI) Retina.
The paper entitled “Sozinibercept Combination Therapy for Neovascular Age-Related Macular Degeneration: Phase 2b Study Subgroup Analysis by Lesion Type” reports pre-specified and post-hoc analyses of angiographic predictors of response to sozinibercept combination therapy with ranibizumab in treatment-naïve patients with wet AMD. The analyses are based on choroidal neovascularization (CNV) type lesions (occult, minimally classic, and predominantly classic), and the presence and absence of retinal angiomatous proliferation (RAP) on visual acuity and anatomical outcomes. The data were derived from the randomized, controlled Phase 2b trial in which sozinibercept 2 mg combination therapy led to superior visual gains compared to ranibizumab monotherapy at 24 weeks.
The Phase 2b pre-specified analyses of subgroups showed that in patients with occult and minimally classic lesions excluding RAP, which represented
“Angiographic lesion characteristics being predictive of patient response are consistent with data reported in real-world trials and have informed the design of Opthea’s sozinibercept Phase 3 clinical program,” said Frederic Guerard, PharmD, Chief Executive Officer of Opthea. “In fact, the patient demographics and baseline characteristics from COAST and ShORe include a high proportion of enrolled patients with these best responding lesion types. We are looking forward to the anticipated topline data readouts for COAST in early Q2 CY 2025 and for ShORe in mid-CY 2025.”
Wet AMD remains the leading cause of vision loss in the elderly, impacting about 3.5 million people in the US and Europe alone. The unmet medical need in wet AMD is significant, with many patients failing to achieve optimal vision outcomes despite treatment with anti-VEGF-A therapies.
About Opthea’s Clinical Development Program
The Company’s pivotal Phase 3 wet AMD program is comprised of two fully enrolled, concurrent, multicenter, double-masked, randomized clinical trials, COAST (Combination OPT-302 with Aflibercept Study) and ShORe (Study of OPT-302 in combination with Ranibizumab). The trials are designed to assess the safety and superior efficacy of sozinibercept combination therapy versus standard-of-care anti-VEGF-A in wet AMD. The Phase 3 program is designed to support a broad label and, if successful, enable sozinibercept to be approved for use in combination with any anti-VEGF-A therapy in wet AMD patients. Sozinibercept has received Fast Track Designation from the US FDA for the treatment of wet AMD. To learn more about Opthea’s Phase 3 clinical trial program, please visit ClinicalTrials.gov for COAST, NCT04757636, and ShORe, NCT04757610.
In Opthea’s prospective, randomized and controlled Phase 2b trial, including 366 treatment-naïve wet AMD patients, sozinibercept was administered in combination with standard-of-care ranibizumab for the treatment of wet AMD. Sozinibercept combination therapy met the pre-specified primary efficacy endpoint of a statistically superior gain in visual acuity at 24 weeks, compared to ranibizumab alone. In addition, secondary outcomes were positive with the combination therapy, including more patients gaining vision of 10 or more EDTRS letters, with improved anatomy, a reduction in swelling and vascular leakage, and a favorable safety profile. These statistically significant results were published in Ophthalmology in February 2023.
About Opthea
Opthea (ASX/NASDAQ:OPT) is a biopharmaceutical company developing novel therapies to treat vision-threatening eye diseases, including wet age‐related macular degeneration (wet AMD) and diabetic macular edema (DME), which remain leading causes of vision loss worldwide.
Opthea’s lead product candidate in Phase 3 development, sozinibercept, is a first-in-class VEGF-C/D ‘trap’ inhibitor being evaluated in combination with standard-of-care anti-VEGF-A therapies to deliver superior vision to wet AMD patients. Sozinibercept has the potential to become the first therapy in 20 years to enable patients with wet AMD to live fuller and healthier lives.
To learn more, visit our website at www.opthea.com and follow us on X and LinkedIn.
Inherent Risks of Investment in Biotechnology Companies
There are a number of inherent risks associated with the development of pharmaceutical products to a marketable stage. The lengthy clinical trial process is designed to assess the safety and efficacy of a drug prior to commercialization and a significant proportion of drugs fail one or both of these criteria. Other risks include uncertainty of patent protection and proprietary rights, whether patent applications and issued patents will offer adequate protection to enable product development, the obtaining of necessary drug regulatory authority approvals and difficulties caused by the rapid advancements in technology. Companies such as Opthea are dependent on the success of their research and development projects and on the ability to attract funding to support these activities. Investment in research and development projects cannot be assessed on the same fundamentals as trading and manufacturing enterprises. Therefore, investment in companies specializing in drug development must be regarded as highly speculative. Opthea strongly recommends that professional investment advice be sought prior to such investments.
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 Opthea’s ongoing COAST and ShORe clinical trials, including the timing for anticipated topline data readouts and their potential to support a broad label and, if successful, to enable sozinibercept to be approved for use in combination with any anti-VEGF-A therapy in wet AMD patients; the potential benefits of sozinibercept, including to deliver superior visual outcomes in wet AMD when combined with standard-of-care anti-VEGF-A therapies and to become the first therapy in 20 years to enable patients with wet AMD live fuller and healthier lives; and the estimated patient population of wet AMD. 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 achievements 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, PharmD, CEO
| Investor Inquiries | Media Inquiries |
| PJ Kelleher LifeSci Advisors, LLC Email: pkelleher@lifesciadvisors.com Phone: 617-430-7579 | Silvana Guerci-Lena NorthStream Global Partners Email: silvana@nsgpllc.com |
Join our email database to receive program updates:
Tel: +61 (0) 3 9826 0399, Email: info@opthea.com Web: www.opthea.com
Source: Opthea Limited
FAQ
What were the key results of Opthea's Phase 2b trial for sozinibercept in wet AMD?
When will Opthea (OPT) release Phase 3 COAST and ShORe trial results?
What patient population was studied in Opthea's Phase 2b sozinibercept trial?
How many patients are affected by wet AMD in Opthea's target markets?
