Monopar Presents Phase 3 Data Showing Greater Neurologic Benefit with ALXN1840 vs SoC in Wilson Disease Patients

New analyses from the FoCus trial demonstrate ALXN1840 provided greater neurologic improvement and less worsening than standard of care.

Apr. 20, 2026 at 4:34am

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A ghostly, translucent X-ray image revealing the intricate molecular structure of the ALXN1840 compound, conceptually illustrating its potential to better manage copper levels and neurological symptoms in Wilson's disease.The novel copper-sequestering mechanism of ALXN1840 could offer improved neurologic outcomes for Wilson disease patients compared to standard treatments.Wilmette Today

Monopar Therapeutics announced new analyses from the Phase 3 FoCus trial of ALXN1840 (tiomolibdate choline, TMC) showing greater neurologic benefit versus standard of care (SoC) in Wilson disease patients with neurologic symptoms at baseline. The data will be presented at the American Academy of Neurology (AAN) Annual Meeting 2026.

Why it matters

These findings highlight the potential of ALXN1840 to meaningfully change the treatment landscape for Wilson disease patients with neurologic symptoms by delivering both improved clinical outcomes and a lower likelihood of neurologic deterioration compared to standard of care.

The details

In the FoCus trial, analysis of patients with neurologic symptoms at baseline (TMC: n=77; SoC: n=35) showed treatment with ALXN1840 resulted in higher rates of neurologic improvement and lower rates of worsening. Clinically meaningful neurologic worsening at Week 48 was observed in 25% of SoC patients vs 9% of ALXN1840 patients (p=0.038). Clinically meaningful neurologic improvement at Week 48 was observed in 45% of ALXN1840 patients vs 32% on SoC. The durable neurologic benefit in the ALXN1840 group continued to increase during long-term follow-up.

  • The data will be presented at the American Academy of Neurology (AAN) Annual Meeting 2026, taking place April 18-22, 2026.
  • Monopar plans to submit a New Drug Application (NDA) for ALXN1840 to the FDA in mid-2026.

The players

ALXN1840

A novel first-in-class Albumin Tripartite Complex (ATC) activator under investigation for the treatment of Wilson disease. ALXN1840 rapidly mobilizes and tightly sequesters excess copper, suppressing its redox reactivity, limiting oxidative damage, and blocking transport across the blood-brain barrier.

Monopar Therapeutics Inc.

A clinical-stage biopharmaceutical company developing ALXN1840 for Wilson disease and other radiopharmaceutical programs.

Dr. Peter Hedera

Department of Neurology, University of Louisville School of Medicine, who will present the FoCus trial results at AAN 2026.

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What they’re saying

“These data highlight the potential of ALXN1840 to meaningfully change the treatment landscape for Wilson disease patients with neurologic symptoms by delivering both improved clinical outcomes and a lower likelihood of neurologic deterioration compared to standard of care.”

— Dr. Peter Hedera, Department of Neurology, University of Louisville School of Medicine

What’s next

Monopar plans to submit a New Drug Application (NDA) for ALXN1840 to the FDA in mid-2026 based on these positive findings.

The takeaway

The durable neurologic benefit and favorable safety profile of ALXN1840 demonstrated in the Phase 3 FoCus trial could make it a promising new treatment option for Wilson disease patients with neurologic symptoms, addressing a critical unmet need in this patient population.