BioAge Labs, Inc. (Nasdaq: BIOA) (“BioAge” or the “Company”), a clinical-stage biopharmaceutical company developing therapeutic product candidates for cardiometabolic diseases by targeting the biology of human aging, today announced that the first participant has been dosed in QUELL-DME, a randomized, controlled Phase 2 trial evaluating once-daily oral BGE-102 in adults with diabetic macular edema (DME). The dosing of the first participant marks the official start of clinical testing for BGE-102 in this indication and represents an important step forward for BioAge’s pipeline. The approximately 180-participant study will evaluate BGE-102 both as monotherapy and in combination with standard-of-care anti-VEGF therapy, with change in best-corrected visual acuity at week 12 as the primary endpoint. BGE-102 is a structurally novel, once-daily oral NLRP3 inhibitor. If successful, the trial could support the development of a convenient oral treatment option for a condition that currently relies on repeated eye injections.
The Rationale Behind Targeting NLRP3 in DME
“NLRP3 is a compelling target in DME because it sits upstream of the inflammation that drives vascular leakage and vision loss. The disease affects about one million people in the United States and is a leading cause of vision loss in working-age adults. Standard care still requires repeated injections into the eye; many patients delay starting treatment, miss doses, or never achieve adequate disease control,” said Kristen Fortney, Ph.D., CEO and co-founder of BioAge. “QUELL-DME asks a straightforward question: can once-daily oral BGE-102 improve vision? Dosing the first participant marks an important milestone for the program. The trial’s results will also guide development in other NLRP3-driven retinal diseases, including geographic atrophy.”
The rationale for targeting NLRP3 is rooted in the biology of inflammation. NLRP3 is an inflammasome, a protein complex that plays a central role in the body’s inflammatory response. When NLRP3 is activated, it triggers the release of pro-inflammatory cytokines such as IL-1Ī² and IL-18, which can drive vascular leakage and tissue damage. In DME, this inflammation contributes to the swelling of the macula, the part of the retina responsible for sharp central vision. By inhibiting NLRP3, BGE-102 aims to reduce inflammation at its source, potentially preventing the vascular leakage that leads to vision loss.
The Burden of DME and the Need for New Options
Diabetic macular edema is a complication of diabetic retinopathy and affects approximately one million people in the United States. It is a leading cause of vision loss in working-age adults, which makes it a significant public health concern. The condition can interfere with daily activities such as reading, driving, and recognizing faces, and it can have a profound impact on quality of life. Current standard care involves repeated injections of anti-VEGF therapy directly into the eye, a procedure that is effective but burdensome for patients. Many patients delay starting treatment, miss doses, or never achieve adequate disease control because of the inconvenience, discomfort, or anxiety associated with intravitreal injections. An oral therapy that could be taken once daily would potentially address these challenges by offering a more convenient and less invasive treatment option.
QUELL-DME Trial Design
QUELL-DME is a randomized, controlled, three-arm Phase 2 trial evaluating oral BGE-102 in patients with diabetic macular edema, both as monotherapy and as an add-on to standard-of-care anti-VEGF intravitreal therapy. The three-arm design allows researchers to compare BGE-102 alone against the current standard of care and against a combination of the two, providing valuable data on whether the oral therapy can stand on its own or whether it is best used alongside existing treatments. The primary endpoint is change in best-corrected visual acuity at week 12, which is a direct measure of how well patients can see. Secondary endpoints are likely to include measures of retinal thickness, safety, and tolerability, although the company has not disclosed the full details of the trial design.
The results of QUELL-DME will also guide development in other NLRP3-driven retinal diseases, including geographic atrophy. Geographic atrophy is an advanced form of age-related macular degeneration that leads to progressive loss of retinal tissue and vision. Like DME, geographic atrophy involves inflammation and vascular dysfunction, and NLRP3 inhibition could potentially play a role in treating the condition. If BGE-102 demonstrates efficacy and safety in DME, it could provide a foundation for expanding the program into additional retinal diseases, increasing the potential value of the asset.
For BioAge, the dosing of the first participant in QUELL-DME is a significant milestone that moves the company closer to potentially offering a new treatment option for patients with DME. The trial will take time to complete, and the results are uncertain, but the start of dosing represents tangible progress in the company’s efforts to develop therapies for cardiometabolic diseases by targeting the biology of human aging.