AsciepiX Therapeutics Inc. has announced the dosing of a Phase 1/2a clinical trial for diabetic macular edema (DME) using an intravitreal “self-forming gel depot peptide”. The Phase 1/2a “CONGO” clinical trial is aimed to evaluate the safety and bioactivity of “AXT107” among patients with DME. The experimental treatment for AXT107 uses a “cryptic peptide” within collagen IV to activate “naturally existing, homeostatic mechanisms of angiogenesis”, the objective being to use a stable peptide inhibiting VEGFR2 (vascular endothelial growth factor) and activates Tie2 (angiopoietin-1 receptor) for the treatment of retinal vascular diseases. The sponsor has suggested that the dosing may only require a once yearly treatment, compared to the current regimen of 1-3 monthly anti-VEGF treatments. Subject to clinical trial results, the approach may be applicable to the treatment of DME, wet AMD or RVO. According to the company, an estimated 7.7 million patients in the US have diabetic retinopathy, and of those, there may be an estimate of 750,000 that have DME.
AsclepiX uses a computational biology platform to rationally design novel peptide moieties to interfere vascular homeostasis, focused on retinal disease and oncology. A number of these peptides may provide long half-life compounds that can generate a “unique intravitreal self-assembling gel depot” using a formulation that could significantly reduce the burden of dosing and treatment. Using AXT107, the Tie2 activation may complement the effects of anti-VEGF action, is capable to improve vision gains and reduce vascular leakage and the suppression of inflammation. According to the sponsor of the study, AXT107 has shown “superiority to, and greater durability, than Eylea in animal models and has demonstrated safety in animal and toxicology studies for 15 months”.
Following the announcement of the dosing on AXT107, Dr. Steven Altschuler, MD, Chairman, AsclepiX Therapeutics, Inc., stated that, “dosing the first patient with AXT107 shortly after the acceptance of its IND is a significant milestone for AsclepiX, as it marks the first clinical stage product and trial for our company”. In addition, in December 2020, the FDA cleared the Investigational New Drug application (IND) for AXT107 for “the treatment of retinal diseases including DME, neovascular age-related macular degeneration (nAMD) and macular edema following retinal vein occlusion (RVO)”. Further, the company’s Chief Medical Officer, Theresa Heah, MD, MBA, suggested that patients may benefit to achieve sustained vision gains and extended duration of action for the treatment, “thereby reducing patient treatment burden”.