AsclepiX Therapeutics Completes Enrollment in nAMD DISCOVER Trial

In December 2023, AsclepiX Therapeutics, Inc., a clinical-stage biopharmaceutical company, launched the DISCOVER Phase 1/2a trial to evaluate the safety and bioactivity of AXT107 (gersizangitide) in patients with neovascular age-related macular degeneration (nAMD). This open-label, single ascending dose study is designed to assess three different dose strengths of AXT107 administered via suprachoroidal injection over a nine-month period. The trial aims to assess the safety, tolerability, and biological activity of AXT107, with preliminary results expected by the second quarter of 2025.

AsclepiX Therapeutics, leveraging computational biology developed at Johns Hopkins University, completed the enrollment of 15 patients in the DISCOVER trial by May 2024. The trial involves three dose levels of AXT107: 125 µg, 250 µg, and 500 µg, with three, three, and nine patients in each group, respectively. The study's primary objectives focus on evaluating the safety and tolerability of AXT107, while secondary endpoints include assessing efficacy through central subfield thickness (CST) and best-corrected visual acuity (BCVA).

To date, no significant safety concerns have been reported following the single injection of AXT107. Robert J. Dempsey, Chief Executive Officer of AsclepiX Therapeutics, expressed gratitude toward the dedicated team and principal investigators who facilitated the rapid enrollment of the trial. Drs. David Almeida, William Bridges, Sabin Dang, and David Lally were specifically acknowledged for their essential roles in achieving this milestone.

AXT107 is an integrin-regulating peptide with a unique mechanism of action that targets neovascularization, vascular permeability, and vascular inflammation. It operates by inhibiting pro-angiogenic vascular endothelial growth factor receptor 2 (VEGFR2) and activating the vessel-stabilizing receptor tyrosine kinase (Tie2), pathways validated for treating retinal vascular diseases. AXT107 interacts with integrin αvβ3 and integrin α5β1, making it effective against these conditions.

Formulated as a microparticulate suspension suitable for suprachoroidal injection, AXT107 is designed to maintain sustained biological activity far exceeding the current standard of care. This novel formulation allows for a potentially extended duration of action, which could significantly reduce the frequency of treatments required for patients.

The effects of AXT107 on the Tie2 pathway complement its anti-VEGF action, offering the potential for improved vision gains, reduced vascular permeability, and suppressed inflammation. Given its low aqueous solubility, AXT107, administered intraocularly, promises substantial durability, which could lessen the treatment burden for patients compared to existing therapies.

AsclepiX Therapeutics, founded by researchers from Johns Hopkins University School of Medicine, utilizes computational biology to identify potent peptide regulators of vascular homeostasis. The company’s lead candidate, AXT107, represents a promising advancement in the treatment of retinal diseases.