MNPR · Monopar Therapeutics Inc.

Monopar Therapeutics presented compelling new analyses from the Phase 3 FoCus trial at the American Academy of Neurology Annual Meeting on April 19, 2026, demonstrating ALXN1840 (tiomilibdate choline)'s superior neurologic outcomes in Wilson disease patients with baseline neurologic symptoms compared to standard of care. In the randomized trial subset (TMC n=77; SoC n=35), ALXN1840 significantly reduced clinically meaningful worsening to 9% at Week 48 versus 25% on SoC (p=0.038), while achieving higher improvement rates of 45% versus 32%. CGI-S and CGI-I improvements were markedly better with ALXN1840 (61% vs 17%, p=0.008; 47% vs 19%, p=0.003), addressing a critical unmet need in neurologic Wilson disease management. Benefits were durable, increasing over long-term follow-up up to approximately 3 years, and consistent across treatment-naïve and experienced patients. The drug's favorable safety profile, with drug-related SAEs in only 4.9% of 266 patients (neurologic SAEs <1%) and no treatment-related deaths, supports its advancement. These findings bolster Monopar's planned NDA submission to the FDA in mid-2026, positioning ALXN1840 as a potential first-in-class treatment that could transform outcomes for this rare genetic disorder affecting copper metabolism.