Inflammasome Therapeutics is eyeing a Phase III study for its geographic atrophy (GA) therapy, kamuvudine-8 (K8), after the drug significantly slowed disease progression in a mid-stage study.
During the Phase II trial (NCT06164587), which enrolled 30 patients with GA in both eyes, patients received either 0.3mg, 0.7mg or 1.05mg of K8 at zero and three months via a bioerodible intravitreal implant. The primary endpoint measured the average rate of GA growth in treated patients versus a pooled control group.
Go deeper with GlobalData
Discover B2B Marketing That Performs
Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.
Within the 0.7mg patient cohort, the drug triggered a statistically significant 54% reduction in GA growth versus the pooled control group over a six-month treatment period – highlighting the treatment’s potential to stave off disease progression.
As well as meeting its primary endpoint, K8 also signposted its potential to improve the sharpness and clarity of vision in patients with lesions outside of the fovea, as treated individuals experienced a significant 4.0-point improvement in a key measure of visual acuity at the six-month mark versus all the control eyes.
Patients with GA also tolerated K8 well, with no one experiencing any treatment-linked serious adverse events (SAE) or dose-limiting toxicities following treatment in this Phase II study.
Inflammasome, a Massachusetts-based biotech, designed K8 as a dual inflammasome inhibitor, which they propose can offer disease-modifying benefits in GA by blocking the downstream release of pro-inflammatory cytokines responsible for both cell death and tissue degeneration – two factors that lead to the worsening of the condition.
Next steps for K8
Following K8’s mid-stage success, Inflammasome is planning to begin a Phase III development programme for the drug in GA.
According to Dr Charles Wykoff, retina specialist and director of research at Retina Consultants of Texas, this is a logical next step, as the initial data appears promising. “If the anatomic benefit is replicated in Phase III, that would be highly differentiated.”
“Stable or improved vision versus loss in the control eyes is notable, and repeat dosing every three to four months would be a welcome advance,” Wykoff added.
Inflammasome looks ahead to Phase III as several other GA drugs progress down the late-stage clinical pipeline – including Ocugen’s one-time gene therapy, OCU410, which the biotech plans to progress to pivotal development in Q3 2026.
Another potential player in this space is Belite Bio, which is developing a retinol-binding protein 4 (RBP4) inhibitor, tinlarebant, in both GA and rare, inherited central vision loss-causing condition, Stargardt disease.
Currently, US regulators have only approved Apellis Pharmaceuticals’ Syfovre (pegcetacoplan) and Astellas Pharma’s Izervay (avacincaptad pegol) for the treatment of GA. In Europe, neither Syfovre nor Izervay is available to patients, as regulators declined to approve them due to their perceived unfavourable benefit-risk profile.
Despite the US availability of two treatments for GA, patients with the condition still face significant unmet needs, as neither drug has been shown to improve visual acuity or restore lost eyesight. If approved, K8 could become the first therapy on the GA market to offer such benefits, provided they translate through to a Phase III study.
According to a recent report from GlobalData, parent company of Clinical Trials Arena, the age-related macular degeneration (AMD) market – under which GA falls – is estimated to grow from $7.8bn to $20.5bn between 2024 and 2034. The segment’s growth will be, in part, driven by the availability of GA therapies.
