Eledon Pharmaceuticals (ELDN) investor relations material

Program overview and unmet need

• Tegoprubart is being developed as an alternative to calcineurin inhibitors (CNIs) for kidney transplant rejection, aiming to address the nephrotoxicity, hypertension, and diabetes risks associated with tacrolimus, the current standard of care since 1994.

• The goal is to improve patient quality of life and extend organ survival, as current kidney transplants last 10–15 years and many patients require multiple transplants.

• Tegoprubart is hypothesized to reduce adverse events and better protect transplanted organs immunologically.

Clinical trial design and endpoints

• Two ongoing studies: phase 1B (open-label, safety/tolerability) and phase 2 (superiority design, head-to-head with tacrolimus, 120 patients, primary endpoint eGFR at 12 months).

• Both studies use similar induction regimens, with maintenance therapy replacing tacrolimus with tegoprubart.

• The phase 2 study is powered to detect an 8–9 point difference in eGFR between arms.

• FDA is considering iBOX, a composite endpoint, as a new approval standard, with non-inferiority as baseline and potential for superiority labeling.

Key results and comparative data

• Phase 1B showed 12-month eGFR of 68 for tegoprubart, compared to low 50s for tacrolimus in historical data.

• iBOX scores for tegoprubart were -4.1 (on-treatment), outperforming historical CNI (-2.9) and BALADA sub-studies (-3.5 to -3.2); a 0.4 difference in iBOX is considered clinically meaningful.

• Acute rejection rates were 18% at 12 months, similar to BALADA sub-studies and higher than standard of care, but rejections were treatable and did not correlate with worse long-term outcomes.

• Safety profile in phase 1B was favorable, with no graft loss, delayed graft function, deaths, drug-induced tremors, or sepsis; viral infections were managed as per standard protocols.