Eledon Pharmaceuticals (ELDN) investor relations material

Key data and clinical progress

• Over 100 human post-transplant patients have received tegoprubart, with data spanning kidney, islet cell, heart, and kidney xenotransplantation.

• Phase 2 BESTOW study in kidney transplantation met non-inferiority on survival, organ survival, and rejection, with a notably improved safety profile versus tacrolimus.

• Islet cell transplantation data showed all six initial patients became insulin-independent, with no severe hypoglycemic events.

• Orphan Drug Designation was granted for liver transplantation, with plans to start a liver IST by year-end.

• Long-term data updates from phase 1b and 2 studies are expected in June or September, with ongoing regulatory discussions for phase 3 trial design.

Safety, efficacy, and trial design insights

• Tegoprubart demonstrated a strong safety advantage over tacrolimus, with lower rates of tremors, new-onset diabetes, bacteremia, sepsis, hypertension, and heart failure.

• Numerical but not statistical superiority was seen in kidney function (eGFR), attributed to uneven distribution of high-quality donor organs between study arms.

• Future phase 3 trials will stratify for organ quality to ensure balanced comparison.

• Non-inferiority on efficacy with better safety is expected to drive pharmacoeconomic benefits, including reduced inpatient dialysis days and improved quality of life.

• FDA discussions will focus on a one-year triple endpoint, with iBox as a potential secondary endpoint and a minimum of 300 patients per arm.

Market opportunity and strategic positioning

• Initial commercial focus is on the 27,000 annual U.S. kidney transplant recipients, with future plans for a switch label requiring a separate study.

• Tegoprubart aims to replicate tacrolimus’s market capture over cyclosporine by offering non-inferior efficacy and superior safety.

• Pricing strategy leverages high transplant and dialysis costs, with room for premium pricing based on clinical and economic benefits.

• Belatacept is not seen as a primary competitor due to its limited adoption and safety concerns; tegoprubart is positioned against tacrolimus.

• Strategic collaborations are being pursued in islet cell and xenotransplantation, aiming to be the immunomodulator of choice for both deceased donor and stem cell-derived islets.