WILMETTE, Ill., June 26, 2026
Monopar Therapeutics announced new analyses from its Phase 3 FoCus clinical trial demonstrating that ALXN1840 (tiomolibdate choline) delivered greater neurologic improvement and superior overall clinical benefit compared with standard of care (SoC) in patients with Wilson disease. The data were presented at the 12th Congress of the European Academy of Neurology (EAN 2026) in Geneva, Switzerland, following previously reported results showing the investigational therapy successfully met its primary endpoint of superior copper mobilization. The latest findings further strengthen the clinical profile of ALXN1840 as the company prepares its planned New Drug Application (NDA) submission to the U.S. FDA in mid-2026.
Phase 3 Results Demonstrate Significant Neurologic Improvement
The new analysis focused on 207 Wilson disease patients with neurologic symptoms at baseline enrolled in the randomized Phase 3 FoCus trial. Patients treated with ALXN1840 experienced statistically significant neurologic improvement on the physician-assessed Unified Wilson Disease Rating Scale (UWDRS Part III) over the 48-week study period (p=0.006), while patients receiving standard therapy did not show significant improvement. Investigators also reported significantly greater global clinical improvement using the Clinical Global Impressions–Improvement (CGI-I) scale at Week 48 (p<0.001). In addition, a larger proportion of patients receiving ALXN1840 achieved meaningful neurologic recovery across multiple improvement thresholds. The investigational therapy also demonstrated similar or better outcomes than standard treatments across psychiatric and liver-related clinical measures.
Favorable Safety Profile Supports Regulatory Submission
Across Phase 2 and Phase 3 clinical studies, ALXN1840 has demonstrated a well-established safety profile in 266 patients, representing 645 patient-years of clinical experience, with a median treatment duration of 2.58 years and some patients treated for more than eight years. Drug-related serious adverse events occurred in 4.9% of patients, while neurologic serious adverse events remained below 1%, and no treatment-related deaths were reported. According to study investigator Dr. Aurélia Poujois, meaningful neurologic improvement remains difficult to achieve with existing Wilson disease therapies, making the sustained improvements observed with ALXN1840 particularly encouraging. These results support Monopar’s strategy to advance the therapy toward regulatory review.
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ALXN1840 Advances Toward Potential New Treatment Option
Wilson disease is a rare inherited disorder caused by mutations in the ATP7B gene, leading to dangerous copper accumulation in the liver, brain, and other organs that can become life-threatening without treatment. ALXN1840 is a first-in-class albumin tripartite complex (ATC) activator designed to rapidly bind excess copper, reduce oxidative damage, and prevent copper transport into the brain while improving copper elimination. Alongside its late-stage Wilson disease program, Monopar Therapeutics continues advancing radiopharmaceutical candidates for cancer imaging and treatment. With strong Phase 3 efficacy, favorable long-term safety, and planned FDA NDA submission in mid-2026, ALXN1840 could become an important new treatment option for patients living with Wilson disease.
Source: Monopar Therapeutics press release
