MHRA Approves Phase 2a ANXV Study for DR and RVO

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted approval to initiate a Phase 2a proof-of-concept clinical study evaluating ANXV, a novel drug candidate developed by Annexin Pharmaceuticals, for the treatment of diabetic retinopathy (DR). The study will also include patients with newly diagnosed retinal vein occlusion (RVO), following the completion of a previous Phase 2a study in the RVO population.

Exploring Shorter Treatment Cycles in Retinal Vascular Disease

The primary objective of the trial is to assess the efficacy and safety of a shorter treatment cycle compared to previous studies. Initial patient dosing is expected to begin within the coming month, and preliminary data is anticipated approximately three months later.

The open-label Phase 2a study will be conducted at The Retina Clinic in London, United Kingdom, under the leadership of Professor Paulo-Eduardo Stanga, who will serve as the principal investigator. The study's design allows for parallel evaluation of both indications, DR and RVO, using an adaptive methodology to optimize efficiency and resource use.

Study Design and Evaluation Parameters

Participants will receive a 5-day course of ANXV treatment, followed by a 30-day observation period involving detailed testing. All patients will be monitored for an additional 90 days for longer-term safety and efficacy signals.

Clinical endpoints for the study will include evaluations of safety and tolerability, as well as potential signals of treatment effect. Additional assessments will measure best corrected visual acuity (BCVA), the extent of retinal damage and swelling, the need for anti-VEGF injections, and results from functional tests along with detailed analyses of blood flow and vascular changes.

The trial is designed without a placebo or active comparator arm and will initially enroll six patients, three with diabetic retinopathy and three with newly diagnosed RVO. The results from the first month will guide treatment decisions for the subsequent participants across both indications.

Study Objectives and Broader Development Potential

Anders Haegerstrand, CEO of Annexin Pharmaceuticals, commented on the MHRA’s approval and the broader implications of the study:

"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. 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."

Preparing for Phase 2b and Future Collaborations

The adaptive and resource-efficient design of this Phase 2a study supports Annexin Pharmaceuticals’ ongoing strategy to advance ANXV toward a larger Phase 2b trial, which is expected to be conducted independently or in collaboration with an industrial partner.