ALXN1840 (WTX101) - (FoCus)

June 30, 2026: Monopar Therapeutics Inc. (NASDAQ: MNPR) announced the FDA granted Rare Pediatric Disease (RPD) designation to ALXN1840 (tiomolibdate choline, TMC), its late-stage candidate for Wilson disease. The RPD designation could make Monopar eligible, upon NDA approval, to receive a pediatric Priority Review Voucher (PRV) that can be used for priority review of a subsequent marketing application or sold/transferred.

The release also cites clinical support for ALXN1840: in a Phase 3 pivotal trial, it met the primary endpoint by showing rapid and sustained copper mobilization significantly greater than standard of care over 48 weeks in both previously treated and untreated patients. Monopar reports durable clinical improvement and a favorable safety/tolerability profile across 645 patient-years of follow-up in 266 patients. ALXN1840 is an oral, once-daily tablet.

Monopar also notes radiopharmaceutical programs: MNPR-101-Zr (Phase 1) for imaging advanced cancers, MNPR-101-Lu (Phase 1a) and MNPR-101-Ac (late preclinical) for treating advanced cancers.