Monopar Therapeutics is recovering a drug from the scrap heap of AstraZeneca’s rare disease pipeline. It has licensed ALXN-1840, a candidate for the treatment of Wilson disease that was previously considered promising enough that Alexion, now an AZ subsidiary, paid $855 million to acquire it six years ago.
There’s no mystery to Monopar’s interest in ALXN-1840. Its co-founder and CEO, Chandler Robinson, M.D., helped discover the drug two decades ago as an undergraduate at Northwestern University.
Monopar will pay AZ an undisclosed sum upfront, which includes equity in the company. Monopar also will be on the hook for tiered royalties based on sales and regulatory milestones. The company did not immediately respond to a request for more information on the deal.
In 2021, AstraZeneca scored in a phase 3 trial as ALXN-1840 outperformed standard-of-care treatments for Wilson’s disease—which included trientine, penicillamine and zinc, or a combination of the medicines. The study achieved its primary endpoint as ALXN-1840 cleared copper from tissues three times better than the other medicines.
The study set ALXN-1840 up for approval, but two much smaller phase 2 mechanistic trials of the candidate subsequently came up short, convincing AZ to discontinue development of the drug last year. The studies indicated that copper was being redistributed in the body instead of being eliminated.
But Robinson still sees potential that his 10-year-old, Wilmette, Illinois-based company can unlock.
“Alexion has generated a substantial clinical data package on ALXN-1840, including a completed pivotal phase 3 clinical trial,” Robinson said in a release. “The medical data gathered from Alexion’s clinical trials furthers our understanding of Wilson disease and stands to benefit this community.”
According to Monopar’s co-founder and executive chair Chris Starr, Ph.D., following AZ’s announcement to ground ALXN-1840, patients, physicians and board members of the Wilson Disease Association urged the company to revive development of the treatment.
“Due in no small measure to the testimonials Chandler received from clinical trial patients who reported benefit while on the drug for years, we decided that this was an opportunity Monopar needed to pursue,” Starr said.
Wilson disease is rare autosomal recessive disorder caused by a mutation in the ATP7B gene. It disrupts the body’s ability to metabolize copper and leads to liver and brain damage, eventually resulting in death at an early age if not treated. Patients with Wilson disease suffer from the loss of function in a copper-binding protein. That leads to the accumulation of copper in the body and, ultimately, to liver disease.
Findings Robinson made while a student at Northwestern—which were published in the academic journal Science—led to the development of ALXN-1840. In 2010, Robinson became CEO of Tactic Pharmaceuticals, which developed the drug, then dubbed decuprate. In 2011, Tactic gained orphan drug designation for the compound.
A year later, Wilson Therapeutics—a company focused solely on finding treatments for the disorder—bought decuprate. In 2018, Alexion bought out the company to acquire the drug.
In its second-quarter earnings report, Monopar—which also is developing radiopharmaceutical drugs—revealed cash, cash equivalents and short-term investments of $7.1 million, with the company projecting its runway to extend to the end of August of next year.