Yearly Dosing of AXT107 May Have Promise for Retinal Diseases


Animal models suggest the investigative treatment is safe and tolerable in treating retinal vascular diseases.

retinal disease

Findings from an animal model study support the safety and tolerability of AXT107, a synthetic 20-mer peptide inhibitor of VEGF receptor 2 and activator of Tie2 intended to treat retinal vascular diseases.

This data was presented at the annual Association for Research in Vision and Ophthalmology (ARVO) Virtual Meeting.

A team led by Niranjan Pandey, AsclepiX Therapeutics, Baltimore, evaluated different doses of intravenous AXT107 (100 µg, 250 µg, 500 µg, and 1000 µg) in both rabbits and minipigs. The team studied GLP safety over the course of 9 months. Ocular tissue distribution was assessed in the rabbits and minipigs over 15 and 9 months, respectively.

Overall, they found the treatment to be well-tolerated following a single dose. There were no observed increases in intraocular pressure, no inflammation, as well as no changes in ERG through 9 months.

Close observation showed AXR107 to form a compact gel from its initial liquid state in the vitreous. The size of this gel was noted to be dependent on dose, and it remained below the visual axis over the course of the observation period.

Using mass spectrometry, Pandey and team were able to quantify the AXR107 gel, noting that it decreased over the study period in both species.

“AXT107 was found at efficacious levels on the retina and choroid-RPE and at low levels in the bulk vitreous, and below levels of quantitation (BLQ) in the aqueous humor and other ocular tissues,” they wrote. Levels of the gel were also BLQ in the plasma over the course of the study period.

Furthermore, the levels of AXR107 in the retina and choroid were comparable at each studied dose. And finally, they reported that the half-life of the gel was 180.

“These studies suggest that AXT107 is safe for intravitreal injection and support potentially once a year dosing in the clinic,” they concluded.

Earlier this year, AsclepiX Therapeutics announced that the first patient had been treated with the investigative treatment as part of its Phase 1/2a CONGO clinical trial. The trial investigators are currently assessing its safety and bioacitivity in patients with diabetic macular edema (DME).

In December 2020, the US Food and Drug Administration (FDA) had cleared the company’s Investigational New Drug application (IND) for AXT107 for treatment of diseases like DME, neovascular age-related macular degeneration (nAMD) and macular edema following retinal vein occlusion (RVO).

“With AXT107, patients may benefit from achieving sustained vision gains and extended duration of action, thereby reducing patient treatment burden,” said Theresa Heah, MD, MBA, Chief Medical Officer and Executive Vice President, Operations, AsclepiX Therapeutics, in a statement.

“We are excited to partner with leading retinal specialists to advance the AXT107 trials, and appreciate their dedication to continue to treat patients and participate in trials despite the ongoing pandemic.”

The study, “AXT107 an Inhibitor of Neovascularization, Vascular Leakage, and Inflammation, Is Well-Tolerated and Could Potentially Be Dosed Once a Year to Treat Retinal Vascular Diseases,” was presented at ARVO 2021.

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