BioAge Labs announced the expansion of its BGE-102 development program into ophthalmology. The company plans to initiate a proof-of-concept (POC) clinical study evaluating BGE-102 in patients with diabetic macular edema (DME).

BGE-102 is a potent, structurally novel, orally administered small-molecule inhibitor of the NLRP3 inflammasome designed to achieve therapeutically relevant retinal exposure. Activation of the NLRP3 inflammasome is a central pathological feature across multiple retinal diseases. In DME, hyperglycemia-driven NLRP3 activation leads to vascular leakage, retinal damage, and progressive vision loss. DME will serve as the initial ophthalmology indication for BGE-102, with the potential to expand into additional NLRP3-driven retinal diseases, including geographic atrophy.

“The efficacy observed with injectable IL-6 inhibitors in retinal disease validates targeting the inflammatory cascade in the eye,” said Kristen Fortney, PhD, CEO and co-founder of BioAge. “NLRP3 sits at the apex of this cascade, and BGE-102 offers the potential to deliver broader anti-inflammatory benefit in an oral formulation. This approach could meaningfully reduce treatment burden for patients with serious, sight-threatening conditions who currently require frequent intravitreal injections. In our ongoing phase 1 trial, BGE-102 has already demonstrated the potential for best-in-class reductions in inflammatory markers of cardiovascular risk, our primary development focus, with a phase 2a readout anticipated in the second half of 2026. Together, these features position BGE-102 as a potential ‘pipeline in a pill’ across cardiovascular, CNS, and ocular diseases driven by NLRP3-mediated inflammation.”

In a preclinical model of DME, oral administration of BGE-102 demonstrated dose-dependent preservation of retinal vascular integrity, achieving near-complete protection from vascular leakage and up to 90% preservation of microvascular structure. 

Planned Proof-of-Concept Trial in DME

BioAge plans to initiate a randomized, controlled phase 1b/2a POC trial in patients with DME in mid-2026. The study will include three treatment arms to evaluate BGE-102 as a monotherapy and in combination with a VEGF inhibitor. The primary objective of the trial is to demonstrate ocular target engagement and pharmacodynamic activity to support future development in inflammatory retinal diseases.

The primary endpoint will be percent change in intraocular IL-6 levels. Secondary and exploratory endpoints will include best-corrected visual acuity (BCVA), central subfield thickness (CST), and intraocular and plasma biomarkers. Topline results from the study are anticipated in mid-2027.