Eledon Pharmaceuticals, Inc. Q4 FY2021 earnings call

• David-Alexandre Gros highlighted 2022 plans to explore tegoprubart's potential in up to four open-label clinical trials across neurodegeneration (ALS), transplantation (kidney and islet cell), and autoimmunity (IgA nephropathy).
• Completed enrollment in Phase 2a ALS study, on track to report top-line data in Q2 2022.
• Received regulatory clearance for Phase 1b kidney transplant trial in UK, working on enrolling patients; nonhuman primate kidney study showed tegoprubart prevented rejection.
• Received IND clearance for islet cell transplant Phase 2a trial; regulatory clearance in Australia, NZ, Malaysia for IgA nephropathy trial.
• Steve Perrin detailed ALS program progress, renal transplant Phase 1b trial plans, islet cell transplant focus on type 1 diabetes, and IgA nephropathy trial details.

For the three months ended December 31, 2021, the net loss was $8.8 million or $0.59 per share compared to a net loss of $5.9 million or $2.13 per share in the same period of 2020. Research and development expenses were $6.2 million in Q4 2021 compared to $3 million in Q4 2020, an increase of $3.2 million. For the full year ended December 31, 2021, the net loss was $34.5 million or $2.33 per share compared to a net loss of $22.8 million or $15.72 per share in 2020. R&D expenses were $23.7 million in 2021 compared to $6.1 million in 2020, an increase of $17.6 million. G&A expenses were $13.1 million in 2021 compared to $10.1 million in 2020, an increase of $3 million. Cash and cash equivalents ended 2021 at approximately $84.8 million, expected to fund operations into 2024.

• Expect top-line data from Phase 2a ALS study in Q2 2022.
• Plan to enroll first patients in Phase 1b kidney transplant trial and open sites for IgA nephropathy trial.
• Nonhuman primate kidney study data ongoing; islet cell and IgA nephropathy trial data expected later in 2021.
• Cash position allows funding operations into 2024 to complete planned trials.

None explicitly detailed in the transcript beyond general forward-looking statement disclaimers regarding potential differences between forecasts and actual outcomes due to factors beyond control.

Q: In ALS, are pro-inflammatory biomarkers elevated in all patients or just rapid progressors, and has correlation with clinical outcomes been evaluated before?

A: Levels of pro-inflammatory markers in about 80% of ALS patients, not statistically meaningfully associated with progression rate; prior studies with therapeutic intervention on biomarkers not done before.

Q: For kidney and islet cell transplantation, how many patients' worth of data is needed to share results?

A: For IgAN, 24 weeks of data; for kidney and islet cell, data even as short as 90 days can be meaningful, with a handful of patients in kidney trial and low single digits in islet cell.

Q: Remaining steps to top-line data for ALS?

A: Completed dosing and follow-up, now finalizing data entry, cleaning, and generating final tables; biosamples still need processing, but no more sample collection; intend to present at scientific forum after top-line.

Q: Nonhuman primate data for renal transplant relative to other published CD40 renal transplant data?

A: Preliminary observations show tegoprubart prevented rejection in all four animals, consistent with historical anti-CD40 ligand antibodies and islet cell transplant model; study ongoing, data to be fully analyzed next quarter.

Q: Next steps with FDA for renal transplant in US and biomarkers in ALS?

A: Finish nonhuman primate study next quarter, get ALS data, then discuss next steps with FDA; in ALS, looking for knockdown of pro-inflammatory markers as primary biomarker objective, with neurofilament light chain as exploratory endpoint.

Q: Progress with islet cell transplant program?

A: Not yet enrolled patients; second US site expected to open mid-year, allowing enrollment and quick data generation within 90 days once patients are enrolled.