Monopar Acquires Late-Stage Wilson Disease Drug Candidate ALXN-1840 from Alexion, AstraZeneca Rare Disease

Key Insights

• 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 Therapeutics Inc. has announced an agreement with Alexion, AstraZeneca Rare Disease, granting Monopar an exclusive worldwide license to ALXN-1840 (bis-choline tetrathiomolybdate), a late-stage drug candidate for Wilson disease. Alexion previously advanced ALXN-1840 through a Phase 3 clinical trial that met its primary endpoint. Monopar will now be responsible for all future global development and commercialization activities. The acquisition aims to address the significant unmet medical needs in Wilson disease, a rare genetic disorder affecting approximately one in 30,000 live births in the US.

ALXN-1840: A Novel Approach to Copper Mobilization

ALXN-1840 is an investigational once-daily, oral medicine designed to selectively and tightly bind and remove copper from the body’s tissues and blood. The drug has been granted Orphan Drug Designation in both the United States and the European Union.

Phase 3 FoCus Trial Results

The pivotal Phase 3 FoCus trial was a randomized, rater-blinded, multi-center clinical trial evaluating the efficacy and safety of ALXN-1840 versus standard-of-care (SoC) in Wilson disease patients aged 12 years and older. The trial enrolled 214 patients and met its primary endpoint, demonstrating three-times greater copper mobilization from tissues compared to the SoC arm (Least Square Mean Difference [LSM Diff] 2.18 μmol/L; p< 0.0001). Patients taking ALXN-1840 experienced rapid copper mobilization, with a response observed at four weeks and sustained through the 48-week study period. The most frequently reported adverse event in the ALXN-1840 treatment group was a reversible increase in transaminase levels.

Monopar's Strategic Move

According to Monopar, the decision to acquire ALXN-1840 was influenced by the continued high level of unmet medical need in Wilson disease and positive testimonials from clinical trial patients. The company believes that the substantial clinical data package generated by Alexion will further the understanding of Wilson disease and benefit the patient community.

Under the terms of the license agreement, Monopar will pay Alexion an upfront payment in the form of cash and equity. Future payments are based on tiered royalties on net sales and pre-determined regulatory and sales milestones. Monopar's COO, Andrew Cittadine, also noted the strategic alignment with AstraZeneca's presence in the radiopharma field, an area of continued growth for Monopar.