From an Idea to a Registrational Pathway for Islet Transplantation
This milestone represents the culmination of a remarkable scientific and translational journey that began nearly a decade ago.
At NobleCon in 2018, Dr. Camillo Ricordi, as a member of Noble Capital Markets’ Scientific Advisory Board, was asked to review emerging life-science companies and technologies to help assess their scientific and investment potential. One of those companies was Anelixis Therapeutics, led by Dr. Steven Perrin. Noble subsequently documented that encounter and Ricordi’s review of Anelixis as the beginning of the relationship between Ricordi and Perrin.
Ricordi immediately recognized the potential importance of Anelixis’ next-generation, re-engineered anti-CD40L antibody, then known as AT-1501, particularly for transplantation. The approach had special significance for the Diabetes Research Institute: Drs. Norma Kenyon, Camillo Ricordi and their collaborators at the University of Miami Diabetes Research Institute (UM-DRI) had spent more than two decades investigating CD40/CD40L blockade as a strategy to protect transplanted islets while reducing dependence on calcineurin inhibitors such as tacrolimus.
Ricordi became an enthusiastic advocate for advancing the Anelixis technology and exploring its application to transplantation. The program subsequently attracted the resources necessary to advance into clinical development. In 2020, Anelixis was acquired by Novus Therapeutics, which became Eledon Pharmaceuticals; the company acquired the intellectual property underlying AT-1501, subsequently named tegoprubart.
The scientific rationale for bringing this new generation of anti-CD40L therapy back to transplantation was strengthened by extensive preclinical work. In nonhuman-primate islet and kidney transplantation, AT-1501 demonstrated the ability to promote allograft survival and function, building on work supported by the Diabetes Research Institute Foundation, NIH and Anelixis/Eledon.
The next challenge was translating that promise into patients.
Ricordi subsequently helped catalyze support for a first clinical pilot of tegoprubart in human islet transplantation, ultimately bringing together Breakthrough T1D, The Cure Alliance, the University of Chicago and the Diabetes Research Institute. The investigator-initiated clinical trial was led by Dr. Piotr Witkowski at the University of Chicago, in collaboration with Ricordi and the Cell Transplant Center at UM-DRI. Breakthrough T1D began funding the program in 2023, with initial additional support from The Cure Alliance and the Diabetes Research Institute.
The results have exceeded our initial expectations. All 12 patients transplanted in the initial study achieved insulin independence, with no reported graft-rejection episodes or de novo donor-specific HLA antibodies. These findings provide compelling early clinical evidence that islet transplantation can achieve insulin independence using a tegoprubart-based, calcineurin-inhibitor-free regimen rather than conventional tacrolimus-based immunosuppression.
And now we have reached another historic milestone.
On September 3, 2026, Eledon Pharmaceuticals announced the submission to the FDA of an IND for an Eledon-sponsored, registrational study of tegoprubart in islet cell transplantation for patients with Type 1 diabetes.
For the islet-transplant field, this represents an unprecedented step forward on the path toward making islet transplantation a broadly accessible functional cure for Type 1 diabetes.
We are profoundly grateful to Eledon Pharmaceuticals for this remarkable demonstration of commitment to the field and to patients with T1D. By assuming responsibility for this next stage of clinical development and pursuing a registrational pathway, Eledon is helping move islet transplantation from highly successful but still limited academic programs toward the possibility of broader regulatory approval and patient access.
We are equally grateful to the Diabetes Research Institute Foundation, Breakthrough T1D and The Cure Alliance for believing in this approach and supporting the work required to bring it to patients. The decades of research by Drs. Norma Kenyon, Camillo Ricordi and their collaborators at UM-DRI helped establish the scientific and preclinical foundation that made this clinical translation possible.
And we congratulate Dr. Piotr Witkowski for his extraordinary leadership of the University of Chicago clinical program and for translating years of collaborative scientific work into remarkable results in patients. His leadership, together with the collaboration between the University of Chicago and UM-DRI, has helped create the clinical foundation upon which Eledon can now pursue a registrational pathway for tegoprubart in islet transplantation.
The next frontier
There is still important work ahead. Tegoprubart remains investigational, and the newly submitted registrational program must establish the evidence necessary for FDA approval. But for the first time, we can see a credible path from decades of academic research in CD40L blockade, through a redesigned antibody and an extraordinarily encouraging clinical pilot, to a formal registrational development program for islet transplantation in Type 1 diabetes.
We also hope that the United States will modernize its regulatory framework for deceased-donor islets so that these minimally manipulated human islets can ultimately be regulated in a manner more analogous to transplanted organs, as occurs under different regulatory frameworks in several other countries. Such reform could allow U.S. academic transplant centers to regain competitiveness, expand patient access, and accelerate innovation.
And this is only one step on a longer journey. The next quantum leap will be to combine increasingly effective cell-replacement therapies with strategies capable of further minimizing – and ultimately eliminating – the need for chronic systemic immunosuppression.
Anelixis -> AT-1501 -> Eledon -> tegoprubart -> successful human islet transplantation -> a registrational IND.
WE DID IT.
And, more importantly, we now have an opportunity to transform what is possible for people living with Type 1 diabetes.
Selected public sources
- Noble Capital Markets / GlobeNewswire (December 10, 2019): NobleCon investor conference announcement documenting Ricordi’s review of Anelixis and the origin of the Ricordi-Perrin relationship.
- Eledon Pharmaceuticals corporate filings: 2020 Anelixis acquisition by Novus Therapeutics and subsequent development of AT-1501/tegoprubart.
- Peer-reviewed preclinical literature on AT-1501/tegoprubart in nonhuman-primate islet and kidney transplantation.
- Breakthrough T1D: reports on support for the tegoprubart islet transplantation clinical program.
- Eledon Pharmaceuticals: clinical updates from the investigator-initiated islet transplantation trial.
- Eledon Pharmaceuticals (September 3, 2026): announcement of IND submission for an Eledon-sponsored registrational study of tegoprubart in islet cell transplantation for Type 1 diabetes.