Critical Path Institute (C-Path) announced October 13, 2022 that its Transplant Therapeutic Consortium (TTC), of which Veloxis Pharmaceuticals is a member, received a draft qualification opinion for the iBox Scoring System through the European Medicines Agency's (EMA) qualification of novel methodologies for drug development. This significant qualification means that the iBox Scoring System, which supports the evaluation of novel immunosuppressive therapy (IST) applications with EMA, will soon be available to be used as a secondary efficacy endpoint in kidney transplant clinical trials.
The draft qualification opinion is going through a public consultation stage through Nov. 17, 2022. After the public consultation period, the iBox Scoring System will represent the first qualified endpoint for any transplant indication and, importantly, the scoring system – with or without biopsy – can be used to demonstrate the superiority of a new IST compared to the standard of care (SOC) at six months up to 24 months post-transplant in pivotal or exploratory drug therapeutic studies.
The iBox Scoring System is a composite biomarker that utilizes multiple clinically relevant patient features to fully reflect the heterogeneity of graft failure, including measures of renal function (eGFR and proteinuria) and immunologic response to the graft (donor-specific antibody), with or without direct assessment of allograft health through histopathology (Banff lesion scores). The TTC, in collaboration with the Paris Transplant Group, has been key in adapting the iBox Scoring System from a tool in patient-level decision-making to application as an efficacy endpoint in clinical trials.
Improving the long-term outcomes in kidney transplant recipients is the ultimate goal and, as such, the qualification of the iBox Scoring System has been TTC's primary initiative since its inception in 2017. This important accomplishment is the first step to enhance development of innovative therapies in kidney transplant and was made possible by the international transplant community's ongoing collaboration, data sharing, expertise and dedication.
"Veloxis is proud to be a part of the Transplant Therapeutics Consortium and the incredible work to achieve draft qualification opinion status for the iBox Scoring System," says Mark Hensley, CEO of Veloxis.
The TTC is also working on its next regulatory milestone – the regulatory endorsement of the iBox Scoring System as a reasonably likely surrogate endpoint by the U.S. Food and Drug Administration (FDA), to whom the qualification plan has been submitted and is under review by the agency.