Exonate Ltd.

CAMBRIDGE, England--(BUSINESS WIRE)--Exonate Ltd., a biotechnology company developing novel, non-invasive, small-molecule therapeutics for patients with retinal vascular diseases, today announced plans to initiate a Phase IIb clinical trial for its lead candidate EXN407. The CLEAR-DE (Clinical Evaluation of a New Eye Drop for Alleviating Retinopathy in Diabetic Eye Disease) trial will further evaluate the clinical efficacy, optimal dosing and safety profile of EXN407 in patients with non-proliferative diabetic retinopathy (NPDR). The progression to a Phase IIb clinical study follows encouraging results from Exonate’s Phase Ib/IIa study in March 2024. In the study, lead candidate EXN407 met its primary safety and tolerability endpoints, with no drug-related serious adverse events, and high patient compliance. Importantly, exploratory efficacy signals showed a reduction in vascular leakage, a key pathological driver in diabetic retinopathy. The Phase IIb trial to investigate the efficacy of EXN407 in NPDR patients is planned to begin in early 2026, with 140 patients currently enrolled across multiple sites in Australia, the Middle East and China. Diabetic retinopathy (DR) is a leading cause of vision loss globally. An estimated one-third of the nearly 500 million people living with diabetes are affected by NPDR, the early stage of diabetic eye disease, which can lead to more severe, vision-threatening complications. Current therapeutic approaches involve monthly intraocular injections, initiated only after symptoms appear. This limits opportunities for early therapeutic benefit, and due to the invasive nature and frequency, is often deferred until irreversible damage has occurred. EXN407 is a first-in-class, twice-daily topical formulation of a selective SRPK1 inhibitor – a minimally-invasive approach with potential to become the first effective eye-drop therapy for NPDR and diabetic macular oedema (DME). Designed to modulate expression of vascular endothelial growth factor (VEGF) via alternative mRNA splicing, EXN407 selectively inhibits the pro-angiogenic isoforms that drive disease progression, causing abnormal and leaky blood vessel formation in the retina. “The Phase Ib/IIa data demonstrated the clear potential of EXN407 as a non-invasive treatment for diabetic eye disease,” said Dr. Catherine Beech, Chief Executive Officer of Exonate. “This therapy could transform the treatment landscape for early-stage disease by providing clinical benefit while avoiding the burden of injections, representing a significant advancement for patients and physicians alike. We’re excited to progress to the CLEAR-DE Phase IIb trial and are actively seeking strategic partners to support its execution.” For more information about Exonate’s pipeline and clinical development plans, please visit: https://www.exonate.com/

Seven months after the FDA approved the reintroduction of Roche's eye implant Susvimo, the US agency has expanded its approved use to include treatment for diabetic macular edema, the primary cause of blindness related to diabetes. This marks the second approved indication for Susvimo, which was initially cleared in October 2021 as an alternative to frequent eye injections for wet age-related macular degeneration. LAS VEGAS, April 30, 2025 /PRNewswire/ -- Diabetic macular edema is a complication that develops from diabetic retinopathy, a widespread consequence of diabetes and a leading cause of irreversible vision loss in working-age adults globally. Diabetic retinopathy occurs due to long-term damage to the retina's small blood vessels, leading to fluid leakage and swelling—especially in the macula. As per DelveInsight's analysis, there were about 1.9 million prevalent cases of DME across the 7MM in 2023, with numbers expected to grow at a notable CAGR during the forecast period. The primary treatment for DME includes intravitreal injections, where the eye is numbed with drops before injecting medication directly into the vitreous humor. Common anti-VEGF drugs used include AVASTIN, EYLEA, and LUCENTIS. Additionally, corticosteroids are beneficial in cases of DME linked to inflammatory eye conditions and can be delivered via eye drops, oral tablets, or periocular injections to help reduce inflammation. The US FDA approved sustained-release corticosteroid implants for more serious or longer-lasting conditions, are OZURDEX, RETISERT, and ILUVIEN. Learn more about the diabetic macular edema treatment @ New Treatment for Diabetic Macular Edema EYLEA (Regeneron Pharmaceuticals) – Aflibercept is a recombinant fusion protein composed of the VEGF-binding regions derived from the extracellular domains of human VEGF receptors 1 and 2, linked to the Fc segment of human IgG1. It functions as a VEGF trap by binding to circulating vascular endothelial growth factors (VEGFs), thereby neutralizing VEGF-A, VEGF-B, and placental growth factor (PGF). This action inhibits the formation of new blood vessels in the tumor's choriocapillaris. In July 2014, the U.S. FDA approved EYLEA (aflibercept) Injection for treating diabetic macular edema (DME). Ranibizumab (Genentech) – Marketed as LUCENTIS, ranibizumab is a monoclonal antibody fragment (Fab) derived from the same original mouse antibody as bevacizumab. It is an anti-angiogenic therapy used to treat the "wet" form of age-related macular degeneration (AMD), a leading cause of vision loss in older adults. Ranibizumab has shown comparable effectiveness to bevacizumab. Developed by Genentech, it is marketed in the U.S. by Genentech and internationally by Novartis. The FDA approved ranibizumab for the treatment of DME in 2012. In February 2025, Roche announced that the FDA had approved Susvimo (ranibizumab injection) 100 mg/mL for treating diabetic macular edema (DME), a major cause of vision loss in adults with diabetes. This approval is based on positive one-year data from the phase III Pagoda trial, which demonstrated that Susvimo offered sustained improvements in vision for individuals with DME, with a safety profile consistent with what is already known for the drug. In the study, patients receiving Susvimo refills every six months experienced vision gains comparable to those receiving monthly intravitreal injections of 0.5 mg ranibizumab (9.6 letters versus 9.4 letters on an eye chart, equivalent to about two lines of vision improvement). Susvimo delivers a specially formulated version of ranibizumab continuously through the Port Delivery Platform, offering a less frequent dosing alternative compared to other treatments that may require monthly eye injections. The FDA initially approved Susvimo in 2021 for the treatment of neovascular age-related macular degeneration (nAMD). Regulatory reviews in other countries are currently underway. Learn more about the FDA-approved DME drugs @ Drugs for Diabetic Macular Edema Treatment Key players, such as Oculis, Rezolute, AsclepiX Therapeutics, and others, are evaluating their lead candidates in different stages of clinical development, respectively. They aim to investigate their products for the treatment of diabetic macular edema. The DME market has a promising outlook with the emerging therapies. Some of the drugs in the pipeline are OCS-01, RZ402, AXT107, and others. DME leads to significant vision loss in diabetics, affecting up to 7.9% of type 1 and 12.8% of type 2 diabetes patients. AbbVie, in collaboration with REGENEXBIO, is developing the gene therapy ABBV-RGX-314. This therapy targets wet age-related macular degeneration, diabetic retinopathy (DR), and other chronic retinal diseases, potentially offering a breakthrough in treating these conditions. Discover which therapies are expected to grab major DME market share @ Diabetic Macular Edema Market Report OCS-01 is a high-concentration dexamethasone eye drop developed for diabetic macular edema (DME), utilizing the proprietary Optireach solubilizing platform. This technology enhances drug solubility, allows for higher concentrations, prolongs ocular retention, and improves bioavailability in eye tissues, including the retina. Recently, in April 2025, Oculis announced the completion of patient enrollment for its Phase 3 DIAMOND-1 and DIAMOND-2 clinical trials evaluating OCS-01 eye drops for diabetic macular edema (DME). These pivotal registration studies aim to support global regulatory filings, including a New Drug Application (NDA) submission and potential approval by the U.S. FDA. Over 800 patients were enrolled across 119 sites in the U.S. and other countries. The swift completion of enrollment in the DIAMOND program—comprising two double-masked, randomized, multi-center Phase 3 trials—is a significant milestone. These studies are assessing the efficacy and safety of OCS-01 over a 52-week treatment period in patients with DME. Topline results are anticipated in Q2 2026, followed by an NDA submission. If approved, OCS-01 would be the first topical eye drop treatment for DME, offering a novel therapeutic option for early intervention and for patients who do not adequately respond to anti-VEGF therapies. RZ402 is an oral small-molecule drug that selectively inhibits plasma kallikrein (PK) for the long-term treatment of DME. By blocking kallikrein activation, RZ402 aims to prevent bradykinin-driven vascular leakage and inflammation. Phase II results revealed a significant reduction in central subfield thickness (CST) across all dosing levels, with improvements of up to ~50 microns compared to placebo. The treatment was well tolerated and showed a favorable safety profile. AXT107 targets two validated mechanisms for treating retinal vascular diseases: it inhibits the pro-angiogenic receptor VEGFR2 and activates the vessel-stabilizing Tie2 receptor. These effects are mediated through its interaction with integrins αvβ3 and α5β1. AXT107 is formulated as a microparticulate suspension designed for intraocular injection and is currently undergoing Phase I/II clinical trials, according to the company's development pipeline. Discover more about drugs for DME in development @ Diabetic Macular Edema Clinical Trials The anticipated launch of these emerging therapies for DME are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the DME market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for DME is expected to grow from USD 3 billion in 2023, with a significant CAGR by 2034. Owing to the higher patient pool and higher diabetic macular edema treatment cost, according to the estimates, the United States had the highest market size in DME, i.e., ~60% of the total market size of DME in the 7MM, in 2023, followed by Germany and Japan. DelveInsight's analysis forecasts market growth due to the introduction of emerging therapies, expecting a rise in market size during the study period (2020–2034). The anticipated increase in market size is driven by advancements in treatment options, greater healthcare access, and a rising prevalence of the condition, which together foster higher demand for innovative and effective therapies. DelveInsight's latest published market report, titled as Diabetic Macular Edema Market Insight, Epidemiology, and Market Forecast – 2034 , will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the DME country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The DME market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Prevalent Cases of DME Total Diagnosed Prevalent Cases of DME Gender-specific Diagnosed Prevalent Cases of DME Age-Specific Diagnosed Prevalent Cases of DME Subgroup-specific Diagnosed Prevalent Cases of DME The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM DME market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this DME market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the DME market. Also, stay abreast of the mitigating factors to improve your market position in the DME therapeutic space. Related Reports Diabetic Macular Edema Epidemiology Forecast Diabetic Macular Edema Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted diabetic macular edema epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Diabetic Macular Edema Pipeline Diabetic Macular Edema Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key diabetic macular edema companies, including Mylan Pharmaceuticals, Kodiak Sciences, Celltrion, Exonate, Opthea, AsclepiX Therapeutics, Allgenesis Biotherapeutics, Ascentage Pharma, Rezolute, Ocuphire Pharma, Oxurion, MingSight Pharmaceuticals, Adverum Biotechnologies, among others. Diabetic Retinopathy Market Diabetic Retinopathy Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key diabetic retinopathy companies, including Genentech, Inc., Regeneron Pharmaceuticals, Roche, Opthea Limited, Regenxbio, Kodiak Sciences Inc, Ocuphire Pharma, Eisai Co Ltd, Apexian Pharmaceuticals, Oculis, among others. Diabetic Retinopathy Pipeline Diabetic Retinopathy Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key diabetic retinopathy companies, including Kodiak Sciences, Novartis, Regenxbio Inc., OcuTerra Therapeutics, Ocular Therapeutix, Bayer, RemeGen, Roche, Ocuphire Pharma, Adverum Biotechnologies, Boehringer Ingelheim, Palatin Technologies, Valo Health, EyePoint Pharmaceuticals, Kubota Vision, MingSight Pharmaceuticals, Oxurion, Aerie Pharmaceuticals, AsclepiX Therapeutics, Ocugen, Ashvattha Therapeutics, Stealth BioTherapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

EXN407 is the first topical SRPK1 inhibitor to demonstrate both safety and signals of biological response as monotherapy for these indications Data generated fully support continued clinical development of EXN407 into the CLEAR-DM Phase IIb study Full Phase Ib/IIa data selected for presentation at ARVO 2024 CAMBRIDGE, England & NOTTINGHAM, England--(BUSINESS WIRE)-- Exonate Ltd., a biotechnology company developing novel, non-invasive, small-molecule therapeutics for patients with retinal vascular diseases, today announced the data from a successful Phase Ib/IIa trial for lead candidate EXN407. These data demonstrate the safety and tolerability of EXN407, as well as clear indications of biological activity, positioning it well for further development as the first topical treatment for retinal vascular diseases such as diabetic retinopathy and diabetic macular oedema. Exonate is now planning to progress EXN407 to the CLEAR-DM (Clinical Evaluation of a New Eyedrop for Alleviating Retinopathy in Diabetic Macular Oedema) Phase IIb clinical trial. EXN407 is a twice-daily formulation, comprised of a small molecule SRPK1 inhibitor. The eye drop formula exploits SRPK1 involvement in the alternative splicing of vascular endothelial growth factor (VEGF), a protein heavily involved in the regulation of blood vessel growth. Through inhibition of SRPK1, EXN407 can selectively target pro-angiogenic isoforms of VEGF that lead to vascular retinal disease progression via aberrant growth of leaky blood vessels within the eye. The mild NPDR/DME (NCT04565756) clinical study assessed the safety, tolerability and signals of biological response to EXN407 monotherapy in a double-masked, placebo-controlled Phase Ib/IIa dose-ranging clinical trial in treatment-naïve patients with mild/moderate non-proliferative diabetic retinopathy (NPDR) and mild diabetic macular oedema. The independent Dose Escalation Committee characterised EXN407 as safe and well-tolerated, with 100% of patients completing the study without requiring anti-VEGF rescue, and no major or serious adverse events reported relating to EXN407. Moreover, EXN407 exhibited high levels of tolerability, with drop comfort scores similar to placebo and artificial tears. In addition to the primary safety and tolerability endpoints, the study concluded that there were promising signals of biological response from EXN407, demonstrating sustained decreases in macular thickness, relative to the placebo group and comparable to previously reported anti-VEGF injections. The trial further noted that EXN407 treatment led to a significant decrease in vascular leakage (60% of EXN407-treated patients relative to 20% placebo) and that EXN407 inhibited further increases to vascular leakage (10% of EXN407-treated patients relative to 50% placebo). “The Phase Ib/IIa data demonstrate the clear potential of EXN407 as a non-invasive treatment for these devastating, sight-threatening conditions, and the favourable safety pro biological activity of EXN407 support its continued clinical development in retinal vascular diseases,” said Catherine Beech, chief executive officer of Exonate. “The results suggest that topical ocular EXN407 may provide clinical benefit and substantially reduce the injection burden for patients with diabetic eye disease. We look forward to engaging with strategic partners to support the CLEAR-DM phase IIb trial, which has been designed to fully demonstrate the clinical benefits of EXN407 in NPDR/DME.” Full results of the Phase Ib/IIa will be presented at the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) in May 2024: To find out more information about Exonate’s therapeutic pipeline for diabetic complications and other indications, as well as our clinical programmes, please visit: View source version on businesswire.com: Contacts Katie Odgaard Tel: +44 (0)7787 502 947 Email: katie.odgaard@zymecommunications.com Source: Exonate View this news release online at: