What Opthea does

Opthea Limited (ASX:OPT) is an Australian clinical-stage biopharmaceutical company headquartered in Melbourne with US operations in Princeton, New Jersey, focused on a single lead asset: OPT-302 (sozinibercept), a first-in-class recombinant 'trap' fusion protein comprising extracellular domains of VEGFR-3 fused to an antibody Fc fragment. The molecule binds and inhibits VEGF-C and VEGF-D, preventing activation of VEGFR-3, and is designed for combination use with anti-VEGF-A standard-of-care therapies. Opthea was historically advancing sozinibercept in wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME); following a Phase 3 wet AMD clinical trial failure in June 2026 that triggered an approximately 97% share price decline, the company relaunched as 'Opthea Reimagined' and refocused OPT-302 on lymphangioleiomyomatosis (LAM), an orphan lung disease primarily affecting women and characterised by progressive cystic destruction of the lung and abnormal lymphatic remodeling.

The business is asset-centric and pre-revenue: no products are approved or commercialised, and all operating capital has been sourced from repeated post-IPO equity placements (approximately A$90M in August 2023 and A$171.5M in June 2024), a US$90M two-tranche equity placement in August 2022, and a non-dilutive Development Funding Agreement of up to US$170M with Carlyle, Abingworth and Launch Therapeutics. The asset is supported by an extensive issued and pending patent portfolio and an existing large human safety dataset from prior wet AMD trials involving thousands of patients. Scientific credibility is anchored by a Scientific Advisory Board co-chaired by Professor Frank McCormack (founding LAM Foundation Scientific Director) and respiratory physician Deborah Yates.

Opthea's intended go-to-market model is a specialty/rare-disease commercialisation approach for LAM — concentrated engagement with KOLs, the 60+ site LAM Clinic Network, and patient-advocacy organisations such as the LAM Foundation — rather than a broad primary-care sales motion. Should sozinibercept reach approval, distribution would target hospital-based pulmonary specialists and integrated delivery networks via a focused specialty sales force. Opthea Limited is publicly listed on the ASX and files Annual Reports on Form 20-F with the U.S. SEC.

Name

Opthea

Legal name

Opthea Limited

Website

https://opthea.com

Company type

Public

Founded year

1984

Operating status

Operating

Headcount range

11–50 employees

Short description

Opthea Limited (ASX:OPT) is a pre-revenue Australian clinical-stage biopharmaceutical company developing OPT-302 (sozinibercept), a first-in-class VEGF-C/D 'trap' fusion protein, now repositioned for the orphan lung disease lymphangioleiomyomatosis (LAM) following a 2026 Phase 3 wet AMD trial failure.

Ownership category

akta.pro rank

Product category

Biopharmaceutical Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Ophthalmology Specialty Pharmaceuticals (HLAIACAK)

Keywords

• Clinical-stage biopharmaceutical
• VEGF-C/D therapeutics
• Orphan disease biologics
• Recombinant fusion proteins
• Lymphangioleiomyomatosis treatment

Headquarters

HQ city

South Yarra

HQ country

Australia

HQ region

Oceania

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Others

Revenue model

1. None — pre-revenue: Opthea is a clinical-stage biopharmaceutical company. As of the latest available material, the company has no marketed products and is not generating product revenue. Its lead asset OPT-302 (sozinibercept) is in Phase 3 clinical development (and being repositioned for the orphan disease LAM). All operating capital is sourced from equity financings and a non-dilutive Development Funding Agreement.

Go-to-market motion2 records

Marketing channels6 records

Core offering

Opthea is a clinical-stage biopharmaceutical company developing OPT-302 (sozinibercept), a first-in-class recombinant VEGF-C/D 'trap' fusion protein designed to inhibit VEGF-C and VEGF-D ligands before they activate VEGFR-3. Under its 'Opthea Reimagined' strategy, the company is repositioning OPT-302 as a treatment for lymphangioleiomyomatosis (LAM), an orphan lung disease, while leveraging prior human safety datasets from its earlier retinal disease program (wet AMD and DME).

Product overview

Opthea Limited (ASX:OPT) is an Australian clinical-stage biopharmaceutical company operating as a single-asset-focused developer centered on one core product, OPT-302 (also known as sozinibercept), a first-in-class VEGF-C/D 'trap' fusion protein. Under the company's 'Opthea Reimagined' strategy, OPT-302 is being repositioned as the lead asset for lymphangioleiomyomatosis (LAM), an orphan lung disease, leveraging prior human safety and activity data generated when the molecule was previously investigated as a combination therapy with anti-VEGF-A standard-of-care for retinal diseases such as wet AMD. Sozinibercept combination therapy is positioned as the second named offering, representing the molecule's prior combination-therapy clinical positioning in ophthalmology; the two named products are not separate commercial franchises but rather the same molecule (OPT-302/sozinibercept) presented under its current (LAM) and prior (retinal combination-therapy) clinical framings.

Differentiator

Problem solved

Functional benefit

Brands

• Sozinibercept: First-in-class VEGF-C/D 'trap' fusion protein; the lead product candidate (also known by development code OPT-302) being developed for wet AMD and, under the 'Opthea Reimagined' strategy, for lymphangioleiomyomatosis (LAM).
• OPT-302

Products and services

• OPT-302 (sozinibercept) OPT-302 is a first-in-class recombinant VEGF-C/D 'trap' fusion protein that binds VEGF-C and VEGF-D to prevent activation of VEGFR-3, interrupting signaling implicated in cystic lung destruction and disease progression in LAM. The molecule comprises extracellular domains of VEGFR-3 fused with an antibody Fc fragment to extend half-life, and is intended for use alongside standard-of-care therapies. It has been administered to thousands of patients in prior wet AMD clinical trials.

Quantifiable outcome

• OPT-302 has already been administered to thousands of patients in prior wet AMD clinical trials, providing extensive human safety and activity datasets.
• +1 more outcomes

Segments3 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature5 records

Scale indicators7 records

Recent moves6 records

Expansion highlights5 records

Broad incumbents

• Genentech (Roche): Developer of ranibizumab (Lucentis), another anti-VEGF-A standard of care for wet AMD and DME, and a major ophthalmology franchise (Susvimo, Vabysmo). Direct therapeutic-area overlap with OPT-302's prior retinal combination positioning.
• Regeneron Pharmaceuticals: Regeneron developed Eylea (aflibercept), the leading anti-VEGF-A therapy for wet AMD and DME and the standard-of-care that OPT-302 was designed to combine with. Highly relevant comparability around the VEGF therapeutic axis and ophthalmology commercial infrastructure.
• Alnylam Pharmaceuticals: Leader in RNAi-based therapeutics including Onpattro for hATTR amyloidosis, demonstrating a rare-disease commercial playbook with specialty distribution and orphan-drug pricing economics. Relevant benchmark for an orphan biologic targeting a small patient population.
• Vertex Pharmaceuticals: Established rare-disease biopharma (cystic fibrosis, sickle cell) with a model of taking a single mechanistic insight into a dominant orphan franchise. Useful comparable for evaluating Opthea's LAM-focused strategy at scale.

Direct peers

• Kodiak Sciences: Clinical-stage ophthalmology company developing next-generation therapies for retinal diseases including wet AMD (KSI-301 / tarcocimab). Closest direct peer in the retinal combination-therapy category Opthea was competing in pre-relaunch.
• Apellis Pharmaceuticals: Approved Syfovre for geographic atrophy and pegcetacoplan pipeline in retinal indications. Comparable late-stage retinal disease biotech profile and similar clinical development trajectory.
• Insmed: Commercial-stage rare-disease biotech with a focus on serious pulmonary diseases (e.g., ARIKAYCE for MAC lung disease, brensocatib for bronchiectasis). Comparable orphan/rare respiratory disease development model relevant to Opthea's LAM pivot.
• BridgeBio Pharma: Clinical-stage rare-disease focused biopharmaceutical company with a model of advancing single-mechanism assets in genetically defined diseases. Comparable single-asset orphan drug development structure and capital intensity profile.

Regional players

• Avecho Biotechnology: ASX-listed drug-delivery and pharmaceutical company on which Opthea NED Kathy Connell also serves as a Non-Executive Director. Regional Australian small-cap biotech peer for capital-markets and board-composition comparison rather than direct therapeutic overlap.

Others

• Scientia Capital: Specialised life-sciences investment fund founded by Opthea NED Lawrence Gozlan. Included as an ecosystem/peer relationship rather than a therapeutic competitor; useful context for understanding Opthea's investor base.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/Opthea1
• Facebookfacebook.com/opthea-413947051988144
• LinkedInlinkedin.com/company/opthea

Compliance1 record

Revenue estimate

Valuation estimate

Number of profiles

Profiles8 records

Funding overview

Funding rounds6 records

Investors2 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Opthea