NEWTON, MASS. 17 July 2026
The 0.7 mg cohort showed a 54% lower mean rate of geographic atrophy (GA) growth (slope) than control eyes; a prespecified extrafoveal analysis showed an adjusted 4.0-letter mean BCVA advantage for K8 versus control – both statistically significant.
Inflammasome Therapeutics today announced positive Phase 2 results for its investigational drug K8 in GA, with no safety signals of concern identified over six months. Furthermore, K8 was associated with a significantly slower disease progression and visual-acuity advantage. Geographic atrophy affects an estimated eight million people worldwide, including around 1.5 million in the US and 2.5 million in Europe.
In the 0.7 mg cohort, the mean rate of geographic atrophy growth was 54% lower than in the pooled group of control eyes over six months (two-sided p=0.016, FDA-preferred slope analysis). A prespecified analysis of eyes with extrafoveal lesions also showed a covariate-adjusted 4.0 ETDRS letter mean advantage for the entire K8-treated group in best-corrected visual acuity compared with all control eyes over six months (two-sided p=0.004).
For historical context, over the first six months, the two FDA-approved geographic atrophy treatments reduced the mean rate of growth by approximately 13–14% compared with controls and neither reported a visual-acuity benefit in their Phase 3 trials.[1]
All three K8 dose cohorts in this trial slowed mean rate of geographic atrophy growth compared with the pooled control group over six months to a substantially greater extent than these previous benchmarks.
K8 (kamuvudine-8), an investigational drug with a unique mechanism of dual inflammasome inhibition, is delivered through a bioerodible sustained-release intravitreal implant. The study evaluated administration once every three months.
The findings are being presented by Professor Jayakrishna Ambati, MD, of the University of Virginia, at the American Society of Retina Specialists Annual Meeting in Montreal.
Commenting on the results, Professor Jayakrishna Ambati, MD — Director of the Center for Advanced Vision Science and DuPont Guerry, III Professor of Ophthalmology at the University of Virginia, and founder of Inflammasome Therapeutics — noted that the combination of reduced lesion growth and a visual-acuity signal together suggests K8 could be affecting both retinal-cell survival and function: "Clearing the high bar of 50% in slowing lesion growth and preserving or improving visual function suggests that K8 not only stops retinal cells from dying but also improves the function of distressed cells by reducing inflammation."
He continued, “The statistical significance of K8’s lesion growth reduction fulfills the promise of earlier studies which predicted that a drug with strong efficacy could demonstrate a statistically significant effect in GA with 30 patients.[2] The dual benefit of K8 on structure and function should now be confirmed in Phase 3 studies.”
Professor Anat Loewenstein said the combination of reduced lesion growth and a visual-acuity signal was particularly encouraging given the continuing unmet need in geographic atrophy:
“In my opinion, we have a real chance for a breakthrough here. We all know there is a huge unmet need in the management of geographic atrophy, which remains the main reason for visual acuity loss in macular degeneration. Since we have a signal here for a treatment resulting in both a lower lesion growth rate and a visual-acuity benefit, this reflects the direction the retina community has been seeking. I firmly believe these results warrant rigorous confirmation in Phase 3,” said Professor Anat Loewenstein, MD, MHA, Professor of Ophthalmology and Vice President at the Tel Aviv Medical Center, Sidney Fox Chair of Ophthalmology at the Gray Faculty of Medical and Health Sciences, Tel Aviv University, and a member of the Retina Scientific Advisory Board of Inflammasome Therapeutics.
Professor Frank Holz was highly encouraged by the results and supported rapid advancement to Phase 3 studies:
“These K8 Phase 2 results are spectacular and extremely encouraging. I look forward to the Phase 3 trials starting as soon as possible so that these findings may be confirmed and a more efficacious treatment for GA can be developed”, said Professor Frank Holz, MD, PhD, Professor and Chairman of the Department of Ophthalmology at the University of Bonn, Germany.
Study findings
The primary endpoint was analyzed using the FDA-preferred linear mixed-effects slope model accounting for treatment, baseline lesion area, time, interactions between these factors, and inter-eye correlation.
Visual function was assessed in a prespecified analysis of eyes with extrafoveal lesions because they have the greatest potential for vision preservation or improvement. The reported mean 4.0-ETDRS letter difference in best-corrected visual acuity between the treated and control groups over the six-month analysis period was adjusted for lesion growth rate, lesion focality, and low-luminance deficit, covariates that influence vision loss in geographic atrophy.
Study design
The multicenter US study enrolled 30 participants with bilateral geographic atrophy across nine clinical centers, representing 60 eyes.
The worse-seeing eye received a bioerodible K8 implant, while the fellow eye remained untreated. Three doses were evaluated: 0.3 mg, 0.7 mg and 1.05 mg. Participants received K8 at baseline and a repeat injection at Month 3.
Each dose cohort was compared with the pooled control group. The statistical model accounted for baseline lesion area, treatment dose, time, interactions between these terms, as well as the correlation between eyes from the same participant. GA imaging was assessed by masked readers at an independent reading center.
Safety findings
No safety signals of concern were identified across the 30-patient study through Month six.
There were no drug-related serious adverse events and no dose-limiting toxicities. Importantly, there were no events of endophthalmitis, intraocular inflammation, neovascular age-related macular degeneration, retinal vasculitis or optic neuropathy.
Administration and product profile
K8 was administered using a 24-gauge in-office injector. The bioerodible implant is designed to provide sustained drug delivery over three months.
The product requires no refrigeration or cold-chain storage and is supplied in a preloaded injector, avoiding the need to withdraw the product from a vial before administration.
The study regimen involved administration at baseline and Month 3, supporting further evaluation of a potential once-quarterly dosing schedule.
Planned Phase 3 development
Inflammasome Therapeutics plans to initiate a global Phase 3 pivotal program to evaluate K8 in geographic atrophy.
Dr. Charles C. Wykoff said the findings provided a strong rationale for evaluating K8 in a larger pivotal program:
“These initial data in a clean, fellow-eye controlled trial appear quite promising. If the anatomic benefit is replicated in Phase 3, that would be highly differentiated. Stable or improved vision vs loss in the control eyes is notable, and repeat dosing every 3-4 months would also be a welcomed advance. Overall, these substantive findings support moving efficiently into a pivotal program,” said Dr. Charles C. Wykoff, MD, PhD, Retina Specialist and Director of Research, Retina Consultants of Texas, a member of the Inflammasome Therapeutics scientific advisory board and a planned investigator in the Phase 3 program.
The company will now seek regulatory input as it prepares the planned global Phase 3 program.
Dr. Babur Lateef, Chairman of the Board of Directors of Inflammasome Therapeutics, said the company would now focus on regulatory engagement and advancing K8 into Phase 3 development: “We are thrilled to see, perhaps for the first time, an investigational therapy demonstrate a statistically significant reduction in the rate of GA progression and a statistically significant benefit in visual function within six months.
We look forward to engaging with regulatory agencies as we move forward with our global Phase 3 pivotal program. We are grateful to the patients, physicians, and their staff for their eager participation in the K8 clinical program along with the employees of Inflammasome Therapeutics for their deep dedication in achieving this important milestone.”
K8 remains investigational and has not been approved by the US Food and Drug Administration or any other regulatory authority. Its safety and efficacy have not been established.
[1]FDA Center for Drug Evaluation and Research. Application Number: 217171Orig1s000. Statistical Review. October 24, 2022 and FDA Center for Drug Evaluation and Research. Application Number: 217225Orig1s000. Clinical Review. July 31, 2023[2] Sunness JS, et al. Ophthalmology 2007;114:271–7 and Yehoshua Z, et al. Ophthalmology 2011;118:679–86
[2] Sunness JS, et al. Ophthalmology 2007;114:271–7 and Yehoshua Z, et al. Ophthalmology 2011;118:679–86