Annexin Pharmaceuticals Reports Promising Early Results from Phase 2a Study of ANXV in Diabetic Retinopathy and Retinal Vein Occlusion
核心洞察
Annexin Pharmaceuticals (搜索) announced favorable findings from the first two patients in its Phase 2a NEXUS study evaluating ANXV for diabetic retinopathy (搜索) and retinal vein occlusion (搜索), with no safety concerns identified after one month of treatment.
The diabetic retinopathy (搜索) patient showed significant improvements in retinal perfusion, capillary density, and reduced macular swelling, while the retinal vein occlusion (搜索) patient demonstrated rapid perfusion improvements similar to the unaffected eye.
ANXV represents a potential first-in-class biologic targeting the underlying vascular occlusion and loss of retinal perfusion, addressing unmet medical needs in diseases affecting over 100 million people globally with diabetic retinopathy (搜索) alone.
Annexin Pharmaceuticals (搜索) AB has reported encouraging early results from its ongoing Phase 2a proof-of-concept study evaluating ANXV, a recombinant human Annexin A5 protein, in patients with diabetic retinopathy (搜索) (DR) and retinal vein occlusion (搜索) (RVO). The first two patients treated in the NEXUS study showed no safety-related findings and demonstrated signals of therapeutic effect one month after treatment.
Clinical Results Show Promise Across Both Indications
The diabetic retinopathy (搜索) patient, who presented with moderate retinal swelling and minimal visual impairment, experienced multiple functional and anatomical improvements thirty days after ANXV treatment. Significant improvements in retinal perfusion and retinal capillary density were observed using OCT Angiography (OCTA). The patient also showed enhanced retinal response to light and improved electrical conduction of sensory input, as measured by microperimetry and electroretinogram respectively.
Additional improvements included decreased macular swelling (intraretinal fluid) and improved vascular anatomy with fewer microaneurysms and hemorrhages. According to the study investigators, these improvements are considered very unlikely to be spontaneous. Visual acuity and the degree of diabetic retinopathy (搜索) remained stable at Day 30.
The second patient, diagnosed with central RVO and presenting with retinal swelling but minimal visual impairment, demonstrated rapid improvements in retinal perfusion similar to the unaffected eye. While retinal swelling persisted to some degree, requiring an anti-VEGF (搜索) injection on Day 30, the perfusion improvements align with previous observations from Annexin's concluded US Phase 2a study in RVO.
Expert Commentary Highlights Differentiated Mechanism
"The findings in the first two patients are very encouraging. In RVO the results are in line what had been observed previously. Importantly, the study includes for the first-time patients with DR," said Professor Paulo-Eduardo Stanga at The Retina Clinic London and Principal Investigator of the study. "In addition to the absence of ANXV related toxicity, the rapid improvements in multiple highly relevant parameters that we observe a month after ANXV treatment in these patients are very promising. We can see increased retinal capillary density and increased retinal vascular perfusion in both of these vision threatening conditions."
Anna Frostegård, Chief Scientific and Medical Officer at Annexin Pharmaceuticals (搜索), emphasized the significance of the diabetic retinopathy (搜索) findings: "The first ever patient with DR treated with ANXV has responded with both functional and anatomical improvements which aligns with the mechanism of action and our expectations. To the best of our knowledge such findings are not expected in response to for example anti-VEGF (搜索) or cortisone."
Addressing Unmet Medical Need
Current treatments for both RVO and DR target macular edema and retinal neovascularization but fail to address the underlying vascular occlusion or loss of retinal vascular perfusion that leads to retinal ischemia. ANXV represents a potential first-in-class approach targeting the actual cause of these diseases.
Diabetic retinopathy (搜索) affects over 100 million people globally and is one of the leading causes of vision loss and blindness in people with diabetes. The disease occurs when high blood sugar levels damage small retinal blood vessels, leading to leakage, oxygen deprivation, and formation of fragile new blood vessels. Current treatments including anti-VEGF (搜索) injections, laser treatment, and surgery are often costly, require repeated interventions, and do not always provide sufficient efficacy.
RVO affects an estimated 16 to 28 million people worldwide. The vascular disease blocks blood flow in retinal veins, often leading to severe visual impairment or blindness requiring long-term treatment. Standard care consists of monthly eye injections to treat macular swelling but has no effect on the underlying vascular blockage.
Study Design and Future Plans
The adaptive Phase 2a study is being conducted at The Retina Clinic London and initially plans to include three patients with diabetic retinopathy (搜索) and three patients with newly diagnosed RVO. Patients receive ANXV treatment for five days and undergo detailed follow-up testing for 30 days, with less intensive monitoring for an additional 90 days.
The study evaluates safety, tolerability, and effect signals through standardized visual acuity tests, assessment of diabetes-related retinal damage, retinal swelling measurements, and objective functional tests analyzing blood flow and vascular changes. If results prove promising, the company intends to increase patient numbers in DR while exploring shortened three-day treatment duration in RVO, with up to twelve patients total planned for initial inclusion.
Anders Haegerstrand, CEO of Annexin Pharmaceuticals (搜索), noted the commercial implications: "While still in a single DR patient, these findings in an additional medically important and commercially attractive ophthalmic indication provides further strength to our ANXV program. It provides strong motivation to move forward forcefully to develop ANXV towards the market."
The company expects to treat two additional diabetic retinopathy (搜索) patients during the current quarter while working to increase RVO patient recruitment. Results are anticipated to strengthen interest from potential licensing partners and support continued development financing as the company prepares for a Phase 2b study expected to be conducted with or by an industrial partner.