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. Over six months, K8 was associated with significantly slower disease progression and a visual-acuity advantage, with no safety signals of concern. GA 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 GA growth was 54% lower than in the pooled control group over six months (two-sided p=0.016, FDA-preferred slope analysis). A prespecified analysis of eyes with extrafoveal lesions showed a covariate-adjusted 4.0-ETDRS-letter mean advantage in best-corrected visual acuity (BCVA) for the K8-treated group versus all control eyes over six months (two-sided p=0.004).

For context, over the first six months the two FDA-approved GA treatments reduced the mean rate of growth by approximately 13–14% versus controls, and neither reported a visual-acuity benefit in its Phase 3 trials.¹ All three K8 dose cohorts slowed mean GA growth against the pooled control group substantially more than those benchmarks. Cross-trial comparisons are not based on head-to-head studies.

K8 (kamuvudine-8) is an investigational dual inflammasome inhibitor delivered as a bioerodible sustained-release intravitreal implant, dosed 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.

A signal on both structure and function

The combination of reduced lesion growth and a visual-acuity signal together suggests K8 could be affecting both retinal-cell survival and function.

Professor Ambati — 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 — said: “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. 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 patients². The dual benefit of K8 on structure and function should now be confirmed in Phase 3 studies.”

Professor Anat Loewenstein, MD, MHA, Professor of Ophthalmology and Vice President at Tel Aviv Medical Center, said: “We have a real chance for a breakthrough here. The unmet need in GA — still the main cause of visual-acuity loss in macular degeneration — is huge. A signal for both a lower lesion-growth rate and a visual-acuity benefit reflects the direction the retina community has been seeking. These results warrant rigorous confirmation in Phase 3.”

Professor Frank Holz, MD, PhD, Professor and Chairman of the Department of Ophthalmology at the University of Bonn, Germany, said: “These K8 Phase 2 results are spectacular and extremely encouraging. I look forward to the Phase 3 trials starting as soon as possible.”

Study design and findings

The multicenter US study enrolled 30 participants with bilateral GA 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 — administered at baseline and repeated at Month 3.

The primary endpoint, mean rate of GA growth, was analyzed using the FDA-preferred linear mixed-effects slope model, with each dose cohort compared against the pooled control group. Imaging was assessed by masked readers at an independent reading center.

Visual function was assessed in a prespecified analysis of extrafoveal-lesion eyes, which have the greatest potential for vision preservation. The 4.0-letter BCVA difference was adjusted for lesion growth rate, lesion focality and low-luminance deficit.

Safety

No safety signals of concern were identified through Month 6. There were no drug-related serious adverse events and no dose-limiting toxicities, and no events of endophthalmitis, intraocular inflammation, neovascular age-related macular degeneration, retinal vasculitis or optic neuropathy.

Product profile

K8 was administered using a 24-gauge in-office injector. The implant delivers the drug over three months, requires no cold-chain storage, and is supplied preloaded, avoiding vial withdrawal before administration.

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, MD, PhD, Retina Specialist and Director of Research at Retina Consultants of Texas, said: “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 be a welcomed advance. Overall, these substantive findings support moving efficiently into a pivotal program.”

Dr. Babur Lateef, Chairman of the Board of Directors, said: “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.”

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