Shares of Eledon Pharmaceuticals, Inc. (ELDN) are down over 50% at $1.91 on Friday, after the company reported mixed results from its Phase 2 BESTOW trial.
BESTOW is a Phase 2 study of the company's drug candidate Tegoprubart in participants undergoing kidney transplantation. Tegoprubart aims to prevent the immune system from attacking the new kidney, potentially improving the chances of a successful transplant.
The study's primary efficacy endpoint was the change in estimated glomerular filtration rate (eGFR) at 12 months post-transplant. Secondary endpoints included biopsy-proven acute rejection (BPAR), patient and graft survival, composite efficacy failure, iBox score, donor-specific antibodies (DSAs), delayed graft function (DGF), and new-onset diabetes after transplantation (NODAT).
According to the trial results, the eGFR for the Tegoprubart treatment arm was 69 mL/min/1.73 m² at 12 months vs. 66 mL/min/1.73 m² for Astellas' Tacrolimus, considered the mainstay of immunosuppressant regimens following solid organ transplantation, which was not statistically significant.
The efficacy failure composite endpoint, comprising death, graft loss and biopsy-proven acute rejection, is the approval endpoint currently recognized by the U.S. Food and Drug Administration.
Efficacy failure composite endpoint was 22% in the Tegoprubart group vs. 17% in the Tacrolimus group, demonstrating non-inferiority for Tegoprubart vs. Tacrolimus, using a 20% non-inferiority margin.
Although the primary endpoint did not reach statistical significance, the company plans to advance Tegoprubart into Phase 3, noting that the eGFR level observed in the phase 2 trial is the highest mean eGFR level reported to date in kidney transplant clinical trials evaluating rejection prevention.
The company believes these results, if replicated in a Phase 3 study, would be sufficient to support Tegoprubart's approvability.
https://www.nasdaq.com/articles/eledon-advance-kidney-transplant-rejection-drug-phase-3-despite-phase-2-miss-key-goal
