C-Path's Transplant Therapeutics Consortium receives EMA Draft Qualification Opinion for the iBox Scoring System

CARY, N.C., U.S.A. (October 14, 2022) – 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.

About Veloxis Pharmaceuticals

Veloxis Pharmaceuticals, Inc, an Asahi Kasei company, is a fully integrated specialty pharmaceutical company committed to improving the lives of transplant patients. Headquartered in Cary, North Carolina, USA, Veloxis is focused on the global development and commercialization of medications utilized by transplant patients and by patients with serious related diseases.

About Asahi Kasei

The Asahi Kasei Group contributes to life and living for people around the world. Since its foundation in 1922 with ammonia and cellulose fiber business, Asahi Kasei has consistently grown through the proactive transformation of its business portfolio to meet the evolving needs of every age. With more than 40,000 employees around the world, the company contributes to sustainable society by providing solutions to the world's challenges through its three business sectors of Material, Homes, and Healthcare. Its healthcare operations include devices and systems for acute critical care, dialysis, therapeutic apheresis, transfusion, and manufacture of biotherapeutics, as well as pharmaceuticals and diagnostic reagents.

About Transplant Therapeutics Consortium

The Transplant Therapeutics Consortium (TTC) was launched in April 2017 and co-founded by the American Society of Transplantation (AST) and the American Society of Transplant Surgeons (ASTS). TTC brings together pharmaceutical companies, diagnostic companies, academic and nonprofit partners working toward a common goal of moving the field forward toward drug development solutions in transplantation. TTC is managed and supported by the Critical Path Institute (C-Path).

About Critical Path Institute

Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, C-Path in Europe is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations. For more information, visit c-path.org. 3

Critical Path Institute is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) and is 54.2% funded by the FDA/HHS, totaling $13,239,950, and 45.8% funded by non-government source(s), totaling $11,196,634. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government.