Monopar Targets Mid-2026 FDA Submission for Wilson Disease Drug

WILMETTE, Ill. – March 27, 2026 – Monopar Therapeutics has set a clear timeline for its lead drug candidate, ALXN1840, announcing plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in mid-2026 for the treatment of Wilson disease. The announcement came as the clinical-stage biopharmaceutical company reported its fourth-quarter and full-year 2025 financial results, revealing a robust cash position and a strategic pivot toward commercial readiness.

This move marks a significant milestone for Monopar and offers a potential new therapeutic option for patients with Wilson disease, a rare and debilitating genetic disorder. The company’s progress follows a year of positive clinical data presentations and constructive engagement with regulators, positioning ALXN1840 as a key asset in its pipeline.

“2025 was a productive year for Monopar, marked by multiple ALXN1840 data presentations, an important publication, a strengthened balance sheet and continued progress toward a planned New Drug Application submission for ALXN1840 in Wilson disease,” said Chandler Robinson, MD, Chief Executive Officer of Monopar.

A New Horizon for Wilson Disease Treatment

Wilson disease is a rare genetic condition where impaired copper elimination leads to toxic accumulation in vital organs, primarily the liver and brain. This can result in severe hepatic, neurological, and psychiatric symptoms. While current standard-of-care treatments, such as chelating agents and zinc salts, exist, significant unmet needs persist, including challenges with side effects and treatment adherence.

The urgency for new options was highlighted during an Externally Led Patient-Focused Drug Development (EL-PFDD) meeting in January 2026, where patients and their families described the profound burden of the disease to the FDA.

Monopar's ALXN1840 aims to address this need with a novel mechanism of action. The drug, an investigational once-daily oral medicine, functions as an albumin tripartite complex (ATC) activator, which allows it to selectively bind and mobilize excess copper. This targeted de-coppering approach differentiates it from existing therapies.

The company has backed its regulatory plans with a wealth of clinical evidence presented throughout 2025. At the European Association for the Study of the Liver (EASL) 2025 congress, Monopar presented pooled long-term data from 255 patients showing a favorable safety profile and sustained efficacy over a median treatment duration of 2.63 years. Later, at the American Neurological Association (ANA) 2025 annual meeting, data demonstrating long-term neurological benefits earned an “Abstract of Distinction” honor. This analysis showed statistically significant neurological and psychiatric improvements sustained over six years, with some patients who switched from standard care experiencing a reversal of symptoms.

Further reinforcing the drug's profile, a publication in the Journal of Hepatology reported statistically significant improvements in copper balance, driven by increased fecal copper excretion. Upcoming presentations at EASL 2026 and the American Academy of Neurology (AAN) 2026 Annual Meeting are expected to provide further evidence of ALXN1840's ability to stabilize liver disease and improve neurological symptoms.

Building a Commercial Powerhouse

Signaling a strategic shift from pure research and development to commercial preparation, Monopar recently appointed Susan Rodriguez as its first Chief Commercial and Strategy Officer. This move underscores the company's confidence in ALXN1840's path to market.

Rodriguez brings over three decades of biopharmaceutical leadership, with a notable track record in launching rare disease therapies. Her experience includes key commercial roles at Ardelyx, Avadel Pharmaceuticals, and Tolmar Pharmaceuticals. Her appointment is pivotal as Monopar begins to build the commercial infrastructure necessary to navigate the complex landscape of rare disease drug launches, which involves intricate market access, specialized patient support, and targeted physician education.

“We also recently strengthened our leadership team with the addition of Susan Rodriguez as Chief Commercial and Strategy Officer as we prepare for the potential launch of ALXN1840,” CEO Chandler Robinson stated in the company's release. This strategic hire is a clear indicator that Monopar is actively laying the groundwork for a successful product launch, should the FDA grant approval.

Fortified Finances and a Clear Runway

Monopar’s ambitious clinical and commercial plans are supported by a significantly strengthened financial position. As of December 31, 2025, the company reported cash, cash equivalents, and short-term investments of $140.4 million. This financial runway, which Monopar expects to fund operations through at least the end of 2027, was bolstered by an underwritten public offering in 2025 that generated approximately $91.9 million in gross proceeds.

The company's financials for 2025 also reflect its strategic focus. The net loss for the year was $13.7 million, a decrease from $15.6 million in 2024. This was primarily driven by lower Research & Development (R&D) expenses, which fell to $9.9 million for the year from $13.0 million in 2024. The reduction is largely attributable to the absence of one-time costs associated with the in-licensing of ALXN1840 from Alexion in 2024. Conversely, General & Administrative (G&A) expenses more than doubled to $6.8 million in 2025, reflecting increased personnel costs, board compensation, and legal fees associated with patent protection and building out the corporate structure for its next phase of growth.

Beyond Wilson Disease: A Diversified Pipeline

While ALXN1840 is its most advanced asset, Monopar is also cultivating a diversified pipeline in oncology through its MNPR-101 radiopharmaceutical platform. This platform targets the urokinase plasminogen activator receptor (uPAR), a protein overexpressed on several aggressive cancers, including pancreatic, colorectal, and triple-negative breast cancer.

The platform includes three distinct programs. MNPR-101-Zr is a zirconium-89 based imaging agent currently in Phase 1 clinical trials in Australia, designed to visualize uPAR-expressing tumors. The therapeutic candidates include MNPR-101-Lu (lutetium-177), which has received IND clearance to begin a Phase 1 trial in the US, and MNPR-101-Ac (actinium-225), a highly potent agent in late-stage preclinical development.

This dual focus on a late-stage rare disease asset and an innovative, early-stage oncology platform provides Monopar with multiple avenues for future growth and mitigates the risk associated with a single-product pipeline. As the company advances toward its pivotal NDA submission for ALXN1840, its radiopharmaceutical programs continue to progress, promising future developments in the challenging field of cancer treatment.