WILMETTE, Ill., April 19, 2026

Monopar Therapeutics announced new Phase 3 clinical data demonstrating greater neurologic benefit of ALXN1840 compared to standard of care (SoC) in patients with Wilson Disease presenting neurologic symptoms. The findings, presented at the American Academy of Neurology Annual Meeting 2026, highlight the therapy’s potential to address a critical unmet need in neurologic Wilson disease, where treatment options remain limited and disease progression can be severe if not effectively managed.

Phase 3 Data Show Superior Neurologic Outcomes

Results from the randomized Phase 3 FoCus trial showed that ALXN1840 delivered higher rates of neurologic improvement and lower rates of disease worsening compared to SoC through Week 48. Clinically meaningful neurologic worsening occurred in 25% of SoC patients versus only 9% in the ALXN1840 group, while improvement rates were 45% for ALXN1840 compared to 32% with SoC.

Additionally, global clinical assessments demonstrated significantly better outcomes, with CGI-S improvement reaching 61% vs 17% and CGI-I improvement at 47% vs 19%, reinforcing the therapy’s strong efficacy profile. These results indicate that ALXN1840 not only improves symptoms but also reduces the risk of neurologic deterioration, a key challenge in managing Wilson disease.

Durable Benefit and Strong Safety Profile

Advertisement

cGxPJobs Banner

Long-term data further showed that neurologic benefits with ALXN1840 were durable and sustained over multiple years, with continued improvement observed during extended treatment follow-up. Importantly, the therapy demonstrated a favorable safety and tolerability profile across Phase 2 and Phase 3 studies involving 266 patients, with drug-related serious adverse events reported in only 4.9% of patients and neurologic serious events in less than 1%. No treatment-related deaths were observed, supporting its potential as a safe long-term treatment option for both treatment-naïve and previously treated patients.

Novel Mechanism Targets Copper Toxicity

ALXN1840 (tiomolibdate choline) is a first-in-class Albumin Tripartite Complex (ATC) activator designed to rapidly mobilize and sequester excess copper, addressing the underlying cause of Wilson disease. By increasing fecal copper excretion and limiting oxidative damage, the therapy helps restore copper balance and prevent further neurologic damage. This targeted mechanism of action differentiates ALXN1840 from conventional therapies and supports its potential to transform the treatment landscape for Wilson disease patients with neurologic involvement.

With plans to advance toward a New Drug Application (NDA) submission to the U.S. FDA in mid-2026, Monopar aims to bring ALXN1840 closer to regulatory approval. The Phase 3 data presented at AAN 2026 reinforce its position as a promising next-generation therapy capable of delivering meaningful clinical benefits and improving long-term outcomes in this rare genetic disorder.

Source: Monopar Therapeutics press release