Annexin Pharmaceuticals AB has received approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 2a proof-of-concept study evaluating its drug candidate ANXV in diabetic retinopathy (DR). The study marks a strategic expansion of the company's ophthalmology portfolio, building on promising Phase 2a results in retinal vein occlusion (RVO) reported in February 2025.
The adaptive study design will include patients with moderate to severe diabetic retinopathy alongside newly diagnosed RVO patients, enabling evaluation of shorter treatment cycles than previously tested. This approach addresses specific questions raised by potential licensing partners regarding treatment duration optimization.
Study Design and Timeline
The Phase 2a study will be conducted at The Retina Clinic in London, UK, under the leadership of Principal Investigator Professor Paulo-Eduardo Stanga. The adaptive design initially includes three patients with diabetic retinopathy and three patients with newly diagnosed RVO, all treated with ANXV for five days and followed with detailed assessments for 30 days.
"The adaptive study design, with evaluation of two indications in parallel at a single clinic, enables the study to be conducted in a time- and cost-effective manner," according to the company. Patient recruitment is expected to begin in the third quarter of 2025, with initial data anticipated approximately three months later and topline results expected before year-end.
Clinical Rationale and Market Opportunity
Diabetic retinopathy represents a significant unmet medical need, affecting over 100 million people globally according to company estimates. The disease occurs when high blood sugar levels damage small blood vessels in the retina, leading to leakage, oxygen deficiency, and formation of fragile new blood vessels.
"Diabetic retinopathy is basically a vascular disease caused by damage to small blood vessels in the retina. Our previous promising Phase 2a data in RVO strengthen our expectation that ANXV may also be effective in diabetic retinopathy – a serious condition where there is a high demand for new treatment options," said Anders Haegerstrand, CEO of Annexin Pharmaceuticals.
Current treatments including anti-VEGF injections, laser treatment, and surgery are often costly, require repeated interventions, and do not always provide sufficient efficacy. The prevalence is expected to rise sharply with increasing diabetes rates globally.
Building on RVO Success
The expansion into diabetic retinopathy builds on encouraging Phase 2a results in RVO, where ANXV demonstrated clinically relevant efficacy signals and a favorable safety profile without limiting treatment-related adverse events. Both conditions involve similar vascular pathology and retinal swelling, with comparable standard treatment approaches using repeated intraocular injections.
Professor Paulo-Eduardo Stanga commented: "I have reviewed the Phase 2a data in RVO and ANXV appears as a safe and promising drug candidate for the treatment of RVO and DR. I believe we will be able to further document safety and tolerability, and capture potential early and beneficial effects in both diseases."
Strategic Partnership Implications
The study addresses key questions arising in ongoing partnership discussions, particularly regarding treatment duration optimization. The company is evaluating whether positive effects can be achieved with a shorter treatment cycle, specifically reducing treatment from five to three days.
"The study as a whole is of great importance as, in addition to broadening the potential area of use and thereby increasing the future market for ANXV, we can get answers to questions that arise in our ongoing partnership discussions," Haegerstrand noted.
The company's long-term goal involves entering a license agreement to enable continued ANXV development, with opportunities enhanced by the expanded clinical portfolio across multiple ophthalmology indications.
Financial Position and Development Pipeline
The Phase 2a study is financed through a rights issue of approximately SEK 50 million completed in March 2025. As of the second quarter, the company reported cash of SEK 46.3 million, sufficient to fund operations through the middle of the first quarter of 2026.
Beyond ophthalmology, Annexin is advancing preclinical studies in oncology and sickle cell anemia, positioning for future clinical studies in these additional therapeutic areas while maintaining focus on potential licensing partnerships in the ophthalmology space.
