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 prevention, aiming to replace tacrolimus due to its nephrotoxicity, hypertension, and diabetes risks.

• Current standard of care has remained unchanged for 30 years, with significant adverse events impacting patient quality of life and organ longevity.

• Improving long-term graft survival and reducing side effects are key goals, as many patients require multiple transplants over their lifetime.

Clinical trial design and endpoints

• Phase I-B and II studies are ongoing, both aiming to replace tacrolimus in maintenance immunosuppression.

• Phase I-B is open-label, focusing on safety and tolerability, while phase II is a head-to-head superiority trial against tacrolimus, powered to detect an 8–9 point eGFR difference at 12 months.

• Key endpoints include eGFR, graft and patient survival, rejection rates, and iBOX composite scores, with eGFR most heavily weighted.

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

Key results and safety profile

• Phase I-B showed 12-month eGFR of 68 (n=32), outperforming historical tacrolimus data (low 50s).

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

• Acute rejection rates were 18%, similar to BALAD sub-studies and higher than standard of care, but rejections were treatable and did not impact long-term outcomes.

• Safety profile showed absence of typical tacrolimus side effects (no graft loss, delayed function, deaths, tremors, or sepsis); viral infections were managed as per standard protocols.