Monopar’s ALXN1840 Cuts Neurologic Worsening to 9% vs 25% at Week 48
Monopar reported Phase 3 FoCus trial results showing ALXN1840 reduced neurologic worsening to 9% versus 25% (p=0.038) and increased improvement rates to 45% versus 32% at Week 48 in Wilson disease patients. Benefits grew over three years with 4.9% SAEs (<1% neurologic, no deaths), underpinning a planned mid-2026 NDA submission.
1. Phase 3 FoCus Trial Outcomes
The randomized Phase 3 FoCus trial enrolled 112 Wilson disease patients with neurologic symptoms (ALXN1840: 77; standard of care: 35), showing ALXN1840 reduced clinically meaningful neurologic worsening to 9% versus 25% at Week 48 (p=0.038) and demonstrated higher improvement rates.
2. Neurologic Improvement and Worsening Metrics
At Week 48, clinically meaningful neurologic improvement occurred in 45% of ALXN1840 patients versus 32% on standard of care. Clinician CGI-S scores improved in 61% versus 17% (p=0.008) and CGI-I improvements were 47% versus 19% (p=0.003).
3. Long-Term Efficacy and Safety Profile
Neurologic benefit continued to increase during long-term follow-up, sustained over approximately three years. ALXN1840 exhibited a favorable safety profile with drug-related serious adverse events in 4.9% of patients, neurologic events under 1% and no treatment-related deaths.
4. Regulatory Path Forward
These positive outcomes support advancement of ALXN1840 toward a planned New Drug Application submission to the U.S. Food and Drug Administration in mid-2026.