Empire Forum 2026: Ambati on K8 phase 2 results in geographic atrophy

Geographic atrophy (GA) is a multifactorial disease, and approaches that target a single pathway, such as complement, "have been generally unsuccessful or barely successful," according to Jayakrishna Ambati, MD, president of Inflammasome Therapeutics, director of the Center for Advanced Vision Science, and DuPont Guerry III Professor of Ophthalmology at the University of Virginia (Charlottesville). At The Empire Forum: Retina Pipeline and Markets, held November 5, 2026, in New York, New York, Ambati spoke with Modern Retina about the phase 2 results for kamuvudine-8 (K8; Inflammasome Therapeutics) in GA that he presented at the meeting.

How does inflammasome inhibition differ from targeting complement?

In GA, multiple factors, including toxins and stressors, cause retinal pigment epithelium (RPE) cells and photoreceptors to die, Ambati explained. Rather than targeting a single pathway, K8 blocks a final common pathway that addresses complement activation as well as other factors upstream of RPE and photoreceptor cell death. "It's very fortuitous, really, that inflammasome activation is the final common pathway for multiple GA stressors," he said.

What did the phase 2 trial show?

In the multicenter US phase 2 trial (NCT06164587), the lead 0.7-mg dose reduced disease progression by 54% over 6 months, Ambati said. In a piecewise model, the reduction was 43% in the first 3 months and 70% in the second 3 months. "We see acceleration of effect once the drug actually has time to work," he said. The trial enrolled 30 participants with bilateral GA at 9 centers; the worse-seeing eye received K8 and the fellow eye served as the control, and the 54% reduction reached statistical significance (P = .016).¹

Functionally, eyes with non-subfoveal lesions, where visual acuity can still be preserved or improved, showed a 4-letter visual acuity benefit (adjusted, 4.0 letters; P = .004).¹ Ambati said the results were expected, following 10 years of preclinical studies in multiple animal models.

K8 was also engineered for convenience, he noted. It is given quarterly, requires no cold-chain storage, and comes in a preloaded injector, features he said could expand reach to patients in satellite offices and reduce capital burden. The injection volume is 1 microliter or less, compared with the 100-microliter injections of the 2 approved GA therapies, which he credited for the absence of intraocular pressure issues. Together, these characteristics could expand the treatment population and improve treatment persistence, he said.

Beyond GA, inflammasomes are involved in many diseases, including diabetic macular edema, Ambati said. A parallel molecule, kamuvudine-9 (K9), has shown neuroprotective and neuro-restorative effects, completely reversing vision loss and paralysis in models of multiple sclerosis. Inflammasome Therapeutics has "strong hopes for this platform approach of K8 and K9 in treating not just geographic atrophy, but many neurodegenerative diseases as well," he said.

Reference
1. Inflammasome Therapeutics reports positive six-month phase 2 results for K8 in geographic atrophy. News release. Inflammasome Therapeutics. July 17, 2026. Accessed November 5, 2026. https://www.businesswire.com/news/home/20260717057639/en