Monopar Therapeutics Inc. (Nasdaq: MNPR) has announced an agreement with Alexion, AstraZeneca Rare Disease, securing an exclusive worldwide license for ALXN-1840 (bis-choline tetrathiomolybdate), a late-stage drug candidate for Wilson disease. Alexion had previously advanced ALXN-1840 through a Phase 3 clinical trial that met its primary endpoint before terminating the program in 2023. Monopar will now assume responsibility for all future global development and commercialization activities. This strategic move significantly enriches Monopar's pipeline with a promising asset for a rare disease with substantial unmet medical needs.
Agreement Terms and Strategic Rationale
Under the terms of the license agreement, Monopar will make an upfront payment to Alexion comprising both cash and equity in Monopar. Additionally, Alexion is eligible to receive future payments based on tiered royalties on net sales, as well as pre-determined regulatory and sales milestones.
Chris Starr, PhD, Co-Founder and Executive Chair of Monopar, noted the circumstances leading to the acquisition: “Upon the 2023 announcement, Chandler, due to his long history with the program and the continued high level of unmet medical need, was contacted by Wilson disease patients, executives and board members of the Wilson Disease Association, as well as physicians regarding the potential for Monopar to obtain rights to ALXN-1840...we decided that this was an opportunity Monopar needed to pursue”.
ALXN-1840 Clinical Data and Efficacy
ALXN-1840 is an investigational once-daily, oral medicine designed to selectively bind and remove excess copper from the body's tissues and blood. The pivotal Phase 3 FoCus trial was a randomized, rater-blinded, multi-center study evaluating ALXN-1840 versus standard-of-care (SoC) in Wilson disease patients aged 12 years and older.
The trial 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). This included patients who had been previously treated for an average of 10 years. Patients taking ALXN-1840 experienced rapid copper mobilization, with a response observed at four weeks and sustained through the 48-week study duration. The most frequently reported adverse event in the ALXN-1840 treatment group was a reversible increase in transaminase levels.
Wilson Disease: Unmet Needs and Market Opportunity
Wilson disease is a rare and progressive genetic condition affecting approximately 1 in 30,000 live births in the US, in which the body's pathway for removing excess copper is compromised, leading to toxic copper accumulation in the liver, brain, and other organs. This can result in a wide range of symptoms, including liver disease, psychiatric disturbances, and neurological issues such as tremors and difficulty walking. Current treatments often have significant side effects or limited efficacy, highlighting the need for improved therapeutic options.
Chandler D. Robinson, MD, Co-Founder and Chief Executive Officer of Monopar, emphasized the potential benefit to the Wilson disease community, stating, “Alexion has generated a substantial clinical data package on ALXN-1840, including a completed Pivotal Phase 3 clinical trial. The medical data gathered from Alexion’s clinical trials furthers our understanding of Wilson disease and stands to benefit this community.”
