Eledon Pharmaceuticals (ELDN) Leerink’s Global Healthcare Conference 2025 summary

Key clinical program updates

• Anticipates major data readouts in 2024, including Phase 1b renal transplant data in August and top-line Phase 2 BESTOW results in Q4.

• Phase 1b study shows mean eGFR in high 60s, outperforming standard tacrolimus regimens.

• BESTOW Phase 2 is a global, 120-patient, head-to-head superiority trial against tacrolimus, powered to detect a nine-point eGFR difference.

• Islet cell transplant study at University of Chicago shows two of three patients off exogenous insulin, with normalized HbA1c.

• Xenotransplant collaborations with eGenesis and United Therapeutics are advancing, with more clinical activity expected in 2024.

Mechanistic and safety insights

• Tegoprubart targets CD40 ligand, offering transient, T-cell-specific immunosuppression, unlike CD40 receptor approaches.

• Blocking CD40 ligand has shown superior efficacy in preventing transplant rejection across species.

• Safety profile across studies and the drug class remains favorable, with no increase in opportunistic infections or malignancy.

• Some dropouts in Phase 1b due to unrelated events or protocol limitations, but most patients remain on study or in extension.

Regulatory and future development plans

• Phase 3 study will be designed post-BESTOW readout, likely expanding on the current design and global reach.

• Awaiting FDA guidance on iBox as a potential endpoint, which could enable superiority designs and more efficient trials.

• iBox requires robust data collection across eight parameters, including eGFR and histology.