Tetrathiomolybdate
- Generic Name
- Tetrathiomolybdate
- Drug Type
- Small Molecule
- Chemical Formula
- MoS4
- CAS Number
- 16330-92-0
- Unique Ingredient Identifier
- 91U3TGV99T
- Background
Tetrathiomolybdate is an oral, small-molecule, anticopper agent that is highly specific for lowering the levels of free copper in serum. COPREXA has completed pivotal clinical trials for the treatment of neurologic Wilson's disease. It is also developed for fibrotic disorders based upon the rationale that the fibrotic disease process is dependent upon the availability of free copper in the body.
- Indication
Investigated for use/treatment in liver disease and pulmonary fibrosis.
ALXN1840 Shows Promising Long-Term Efficacy and Safety for Wilson Disease at EASL 2025
• Monopar Therapeutics presented late-breaker data at EASL 2025 showing sustained clinical improvements in Wilson disease patients treated with ALXN1840 (tiomolybdate choline) over a median treatment duration of 2.63 years.
• The pooled analysis from multiple clinical trials (n=255) demonstrated improvements in patient-reported symptoms, copper mobilization, and clinical assessments, with fewer than 5% of patients experiencing drug-related serious adverse events.
• Patients reported higher convenience and effectiveness with ALXN1840 compared to standard of care, suggesting the drug candidate could provide meaningful benefits for the management of this rare genetic condition.
Monopar Therapeutics Advances Wilson Disease and Radiopharmaceutical Programs
• Monopar Therapeutics in-licensed ALXN-1840 from Alexion for Wilson disease, planning regulatory discussions with the FDA focusing on patients with severe symptoms.
• Phase 1 clinical trials for MNPR-101-Lu in advanced solid cancers and MNPR-101-Zr for tumor imaging are actively enrolling patients and showing promising early data.
• A registered public offering generated $17.7 million, extending operational runway into the first half of 2026 to support clinical programs and R&D.
• Monopar filed a provisional patent for new radiopharmaceutical compounds and linkers, aiming to expand its pipeline with high-value cancer targets.
Sangamo Therapeutics' Fabry Disease Candidate ST-920 Gains Accelerated Approval Pathway
• Sangamo Therapeutics' shares surged after the FDA agreed to a regulatory pathway for accelerated approval of isaralgagene civaparvovec (ST-920) for Fabry disease.
• The FDA will consider data from the Phase I/II STAAR trial, using the rate of decline in eGFR at 52 weeks as the primary basis for approval.
• Sangamo plans to submit a BLA in the second half of 2025, three years ahead of previous estimates, potentially bringing the treatment to patients sooner.
• Septerna, focusing on GPCR therapies, raised $288 million in an upsized IPO, highlighting renewed interest in biotech IPOs.
Monopar Acquires Late-Stage Wilson Disease Drug Candidate ALXN-1840 from Alexion, AstraZeneca Rare Disease
• Monopar Therapeutics has acquired ALXN-1840, a late-stage drug candidate for Wilson disease, from Alexion, AstraZeneca Rare Disease, taking over global development and commercialization.
• ALXN-1840, also known as bis-choline tetrathiomolybdate, is an investigational oral medicine designed to selectively bind and remove copper from the body and has shown promise in clinical trials.
• The Phase 3 FoCus trial demonstrated that ALXN-1840 achieved three-times greater copper mobilization compared to standard-of-care, with rapid response observed at four weeks and sustained through 48 weeks.
• Monopar's acquisition is driven by the continued unmet medical need in Wilson disease and testimonials from clinical trial patients who reported benefit from ALXN-1840.
Monopar Acquires Late-Stage Wilson Disease Drug Candidate ALXN-1840 from Alexion
• Monopar Therapeutics has obtained an exclusive worldwide license from Alexion for ALXN-1840, a Wilson disease drug candidate that has completed Phase 3 trials.
• The agreement includes an upfront payment of cash and Monopar equity, along with future tiered royalties and milestone payments for Alexion.
• ALXN-1840 demonstrated significant copper mobilization in Phase 3 trials, with generally mild to moderate adverse events and no neurological worsening observed.
• Monopar plans to oversee all future global development and commercialization of ALXN-1840, building on substantial clinical data already generated by Alexion.
Drug Development Updates
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