Drugs / TIOMOLIBDATE CHOLINE

Regulatory milestones approvals, filings & regulatory actions · 1 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Filed US (FDA) — Wilson disease 2026-07-22

“today announced that it has initiated the rolling submission of a New Drug Application (“NDA”) to the U.S. Food and Drug Administration (“FDA”) for ALXN1840” monopartx.com ↗

“the rolling NDA submssion, that Monopar has initiated for ALXN1840” monopartx.com ↗

Trials 19

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 19 trials
Phase 1 NCT04610580 Jan → Mar 2021 — Alexion Pharmaceuticals, Inc. Completed No outcome recorded
Phase 1 NCT04526210 Oct 2020 → May 2021 Wilson disease Alexion Pharmaceuticals, Inc. Completed No outcome recorded
Phase 1 NCT04560816 Jul 2020 → Mar 2021 — Alexion Pharmaceuticals, Inc. Completed No outcome recorded
Phase 1 NCT04526197 Jul → Nov 2020 Wilson disease Alexion Pharmaceuticals, Inc. Completed No outcome recorded
Phase 1 NCT04594252 Jul → Nov 2020 — Alexion Pharmaceuticals, Inc. Completed No outcome recorded
Phase 1 NCT05303324 Jul → Oct 2019 — Alexion Pharmaceuticals, Inc. Completed No outcome recorded
Phase 1 NCT05641311 Feb → May 2019 Wilson disease Alexion Pharmaceuticals, Inc. Completed No outcome recorded
Phase 1 NCT05319912 Apr → May 2014 — Alexion Pharmaceuticals, Inc. Completed No outcome recorded
Phase 1 NCT05319899 Jan → Mar 2014 — Alexion Pharmaceuticals, Inc. Completed No outcome recorded
Phase 1/21 trial
Phase 1/2 NCT00352742 Jun 2006 → Sep 2008 plasma cell myeloma Attenuon Terminated No outcome recorded
Phase 26 trials
Phase 2 NCT04422431 Dec 2020 → May 2022 Wilson disease Alexion Pharmaceuticals, Inc. Completed No outcome recorded
Phase 2 NCT04573309 Sep 2020 → Jun 2022 Wilson disease Alexion Pharmaceuticals, Inc. Completed No outcome recorded
Phase 2 NCT02273596 Nov 2014 → Oct 2016 Wilson disease Alexion Pharmaceuticals, Inc. Completed No outcome recorded
Phase 2 NCT00674557 Jun 2008 → Mar 2009 breast cancer Cancer Research UK Terminated No outcome recorded
Phase 2 NCT00405574 Nov 2006 → Jun 2008 prostate cancer Attenuon Unknown No outcome recorded
Phase 2 NCT00383851 Sep 2006 → Sep 2008 melanoma Attenuon Unknown No outcome recorded
Phase 32 trials · 1 met primary
Phase 3 NCT05047523 Sep 2021 → Jun 2023 Wilson disease Alexion Pharmaceuticals, Inc. Terminated No outcome recorded Stop: Business
Phase 3 NCT03403205 Feb 2018 → Jun 2023 Wilson disease Alexion Pharmaceuticals, Inc. Terminated Met primary Stop: Business
Phase not stated1 trial
— NCT05686564 — Wilson disease Alexion Pharmaceuticals, Inc. No longer available No outcome recorded

News releases announcing trial results or a regulatory action · 12

DateIssuerRelease
2026-07-22 Monopar Therapeutics Regulatory Monopar Initiates Rolling NDA Submission for ALXN1840 in Wilson Disease monopartx.com ↗
Monopar has submitted the first completed sections of the NDA.
2026-06-30 Monopar Therapeutics Regulatory Monopar Therapeutics Receives FDA Rare Pediatric Disease Designation for ALXN1840 for the Treatment of Wilson Disease monopartx.com ↗
2026-06-26 Monopar Therapeutics Results Monopar Presents New Analyses of Phase 3 FoCus Data at EAN 2026 Showing Greater Neurologic and Global Clinical Benefit with ALXN1840 Versus Standard of Care in Wilson Disease monopartx.com ↗
ALXN1840 also demonstrated similar or better outcomes compared to SoC across a range of psychiatric and hepatic measures during the 48-week study.
2026-06-01 Monopar Therapeutics Results Monopar Presents Phase 2 ALXN1840 Data Demonstrating Liver Disease Stabilization and Neurologic Improvement in Treatment-Experienced Wilson Disease Patients at EASL 2026 monopartx.com ↗
ALXN1840 (tiomolibdate choline) Stabilizes Liver Disease and Improves Neurological Symptoms as well as Quality of Life in Treatment-Experienced Wilson Disease Patients
2026-05-19 Monopar Therapeutics Results Monopar Announces Publication of Phase 2 Study Demonstrating ALXN1840 Significantly Improves Copper Balance in Patients with Wilson Disease monopartx.com ↗
2026-04-19 Monopar Therapeutics Results Monopar Presents Phase 3 Data Showing Greater Neurologic Benefit with ALXN1840 vs SoC in Wilson Disease Patients with Neurologic Symptoms at AAN 2026 monopartx.com ↗
Monopar Therapeutics Inc. (“Monopar” or the “Company”) (Nasdaq: MNPR), a clinical-stage biopharmaceutical company developing innovative treatments for patients with unmet medical needs, announced new analyses from the randomized controlled Phase 3 FoCus trial of ALXN1840 (tiomolibdate choline, TMC) showing greater neurologic benefit versus standard of care (SoC) in Wilson disease patients with neurologic symptoms at baseline.
2025-11-09 Monopar Therapeutics Results Monopar Presents New Data and Analyses on Rapidly Improved Copper Balance in Wilson Disease Patients on Tiomolybdate Choline at AASLD - The Liver Meeting® 2025 monopartx.com ↗
The mean daily copper balance among patients treated with ALXN1840 in the study (n=8) was significantly lower - indicating improvement - compared with their pre-treatment baseline.
2025-09-23 Monopar Therapeutics Results Monopar Announces Journal of Hepatology Publishes Physicians’ Letter to the Editor Demonstrating ALXN1840 Rapidly Improved Copper Balance in Wilson Disease Patients monopartx.com ↗
By controlling for the other routes of copper loss by comparing pre- and post-ALXN1840 treatment, the analysis demonstrates that ALXN1840 statistically significantly improved copper balance (increases copper excretion) over the duration of the study.
2025-09-14 Monopar Therapeutics Results Monopar to Present New Long-Term Neurological Efficacy and Safety Data for ALXN1840 in Wilson Disease at the 150th American Neurological Association Annual Meeting monopartx.com ↗
The new data presented at ANA highlight the long-term neurological benefit of ALXN1840, and follow the recent presentation of long-term hepatic and systemic efficacy and safety data at the European Association for the Study of the Liver (EASL) International Liver Congress 2025.
2025-05-07 Monopar Therapeutics Results Monopar Presents ALXN1840 Late-Breaker Data at EASL 2025 monopartx.com ↗
Sustained improvements from baseline in the Unified Wilson Disease Rating Scale (“UWDRS”) Part II (patient-reported symptoms) and Part III (clinician-assessed symptoms);
2022-06-23 AstraZeneca Results ALXN1840 shows rapid and sustained improvement in copper mobilisation from tissues, potentially closing treatment gaps for Wilson disease community astrazeneca.com ↗
In the trial, people taking ALXN1840 experienced rapid copper mobilisation, with a response at four weeks and sustained through 48 weeks.
2021-08-26 AstraZeneca Results ALXN1840 FoCus Phase III trial in Wilson disease met primary endpoint demonstrating improvement in copper mobilisation from tissues astrazeneca.com ↗
Positive high-level results from the FoCus Phase III trial in Wilson disease showed ALXN1840 met the primary endpoint with a statistically significant improvement in daily mean copper mobilisation from tissues, demonstrating superiority compared with standard-of-care (SoC) treatments.

All press releases naming this drug 14 releases

DateIssuerRelease

Evidence & citations 11 cited values

Every value below carries the sentence it was read from. 20 sources stand behind the page.

FieldValueCited text
Known as TIOMOLIBDATE CHOLINE ChEMBL registry synonym — accepted as the source's own label CHEMBL4594259 ↗
Known as ALXN1840 ClinicalTrials.gov intervention name — accepted as the source's own label NCT05319899 ↗
12

NCT05686564 ↗

NCT05641311 ↗

NCT04560816 ↗

NCT04526210 ↗

NCT04610580 ↗

NCT03403205 ↗

NCT05319912 ↗

NCT05047523 ↗

NCT04594252 ↗

NCT04573309 ↗

Show 2 more sources

Known as ATN-224 ClinicalTrials.gov intervention name — accepted as the source's own label NCT00352742 ↗
3

NCT00674557 ↗

NCT00383851 ↗

NCT00405574 ↗

Known as Bis-choline tetrathiomolybdate “Bis-choline tetrathiomolybdate (WTX101) is an oral first-in-class copper-protein-binding molecule that targets hepatic intracellular copper and reduces plasma...” PMID 28988934 ↗ Oct 2017
1

NCT04422431 ↗

Known as Choline tetrathiomolybdate ChEMBL registry synonym — accepted as the source's own label CHEMBL4594259 ↗
Known as formerly WTX101 “formerly WTX101” NCT04594252 ↗
1

“formerly WTX101” NCT04573309 ↗

Known as WTX-101 ChEMBL registry synonym — accepted as the source's own label CHEMBL4594259 ↗
Action Inhibit “SOD1 inhibitor ATN-224” NCT00674557 ↗
Modality Small molecule “ATN-224 is a small molecule that has been shown in pre-clinical studies to be antiangiogenic.” NCT00352742 ↗
1

CHEMBL4594259 ↗

Route Oral “orally active” NCT00383851 ↗
Target SOD1 “SOD1 inhibitor ATN-224” NCT00674557 ↗