Monopar and Alexion, AstraZeneca Rare Disease, Partner on Late-Stage Wilson Disease Drug

Monopar Therapeutics Inc., a biotechnology company listed on Nasdaq under the symbol MNPR, has entered into an agreement with Alexion, a division of AstraZeneca Rare Disease. This agreement grants Monopar an exclusive global license to ALXN-1840 (bis-choline tetrathiomolybdate), a promising drug candidate for treating Wilson disease that has successfully completed a Phase 3 clinical trial with positive results. Monopar will now oversee all further development and global commercialization.

Dr. Chandler D. Robinson, Co-Founder and CEO of Monopar, has a well-established history with tetrathiomolybdate. He conducted early research on the drug, leading to its development into ALXN-1840, with findings published in Science. Dr. Robinson has also actively engaged with the Wilson disease community, delivering a keynote address at the Wilson Disease Association’s 2013 Annual Conference. However, in 2023, Alexion decided to discontinue the ALXN-1840 program for Wilson disease following a review of Phase II mechanistic trial results and subsequent discussions with regulatory bodies.

Chris Starr, PhD, Co-Founder and Executive Chair of Monopar, noted that after Alexion's announcement in 2023, many stakeholders, including Wilson disease patients and members of the Wilson Disease Association, encouraged Monopar to acquire the rights to ALXN-1840. The positive feedback from clinical trial patients who benefited from the drug was a significant factor in Monopar's decision to pursue this opportunity. Dr. Starr brings extensive experience to this endeavor, having co-founded BioMarin and Raptor Pharma, both of which specialized in orphan drugs.

Dr. Robinson emphasized the comprehensive clinical data collected by Alexion on ALXN-1840, including a completed pivotal Phase 3 clinical trial, which enhances the understanding of Wilson disease and could benefit the affected community.

According to the license agreement, Monopar will make an upfront payment to Alexion in cash and equity. Future payments will be based on tiered royalties from net sales and pre-agreed regulatory and sales milestones.

Andrew Cittadine, Chief Operating Officer of Monopar, expressed excitement about partnering with Alexion and AstraZeneca. He highlighted AstraZeneca's significant presence in the radiopharma field, which aligns with Monopar's recent positive clinical data on their novel radiopharma program.

Wilson disease is a rare genetic disorder characterized by the body's inability to eliminate excess copper effectively. This leads to toxic copper accumulation in organs such as the liver and brain, causing significant damage over time. Symptoms vary widely and can include liver disease, psychiatric issues, and neurological problems, such as personality changes, tremors, and difficulties with movement, swallowing, or speech. In severe cases, the damage may be irreversible.

ALXN-1840 is an investigational oral medication taken once daily for Wilson disease. The drug is designed to bind and remove copper from the body selectively and effectively. It has received Orphan Drug Designation in the United States and orphan designation in the European Union for treating Wilson disease.

The pivotal Phase 3 FoCus trial was a randomized, multi-center study aimed at evaluating the efficacy and safety of ALXN-1840 compared to standard-of-care (SoC) treatments in patients aged 12 and older. The primary endpoint measured copper mobilization over 48 weeks. The trial involved 214 patients distributed into two cohorts based on their prior treatment status. The trial successfully met its primary endpoint, demonstrating that ALXN-1840 achieved three times greater copper mobilization from tissues compared to SoC.

Monopar Therapeutics is advancing ALXN-1840 for Wilson disease and has several radiopharma programs targeting advanced cancers at various stages of development, including MNPR-101-Zr, MNPR-101-Lu, and MNPR-101-Ac225.