Abstract
Purpose :
Currently there is no approved treatment for retinal vein occlusion (RVO), either acute onset or chronic. Management of RVO is limited mainly to aVEGFs that target macular oedema secondary to RVO. There is an important need to improve the blood flow to the retina, especially in an acute RVO setting. It would potentially reduce the risk of blindness and provide other benefits for patients with RVO. Thus, there is a high unmet medical need for a safe and effective treatment for patients with an acute RVO event.
ANXV, a novel biologic drug candidate, is currently in Phase 2 as a potential first-line treatment for patients recently diagnosed with retinal vein occlusion (RVO). ANXV is expected to act focally at the site of occlusion as an anti-adherent, anti-thrombotic, anti-inflammatory, membrane repair and anti-apoptotic agent by targeting phosphatidylserine (PS). Thereby limiting the retinal area of non-perfusion (RANP) and providing short and long-term benefits for RVO patients beyond that of the current standard of care.
A Phase 1 First in Human (FIH) trial with ANXV had been completed in healthy male volunteers aged between 18 and 59 years.
Methods :
Phase 1 FIH trial with ANXV was designed as an adaptive, randomized, double-masked, placebo-controlled, ascending single and multiple dose study to evaluate the safety, tolerability and pharmacokinetics of ANXV administered as an intravenous (IV) infusion in subjects. The subjects received placebo (n=14) or ANXV (n=32) in a range of 0.25 mg-2.0 mg as single or multiple doses once daily for five consecutive days. Each dose was administered as a 50 mL IV infusion over 1 hour.
Results :
Based on the PK data, all subjects administered ANXV were exposed. No Serious Adverse Events (SAEs) were reported, and most treatment emergent adverse events (TEAEs) were of mild intensity. No TEAEs resulted in any action taken to the use of investigational medicinal product, study interruption, or withdrawal from the study. No deaths were reported in the study.
Conclusions :
Based on the assessment of TEAEs, safety laboratory data, as well as all other safety and exploratory/pharmacodynamic parameters, single IV doses of 0.25 to 2.0 mg ANXV and 5 consecutive days of IV dosing with 0.5 to 2 mg/day ANXV were considered safe and well-tolerated in healthy male volunteers aged between 18 and 59 years.
This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.