Monopar starts rolling NDA for Wilson disease drug ALXN1840, reports Q2 2026 results
The company said it began a rolling FDA submission for ALXN1840 in July, backed by Rare Pediatric Disease designation and new Phase 3 data, with cash of $134.3 million.
Monopar Therapeutics said that on July 22, 2026, it initiated a rolling submission of a New Drug Application to the FDA for ALXN18401. The company noted that the FDA authorized the rolling process, letting completed sections of the application be submitted and reviewed while remaining sections are finalized1. Monopar said it expects to complete the NDA submission over the next few months1.
The FDA also granted ALXN1840 Rare Pediatric Disease designation on June 30, 2026, according to the release, a designation given to therapies for serious or life-threatening diseases primarily affecting children from birth to 18, which could make Monopar eligible for a pediatric Priority Review Voucher at NDA approval, transferable or sellable to another sponsor1.
On clinical data, Monopar presented new Phase 3 FoCus trial analyses at the European Academy of Neurology meeting on June 28, 2026. The poster, titled "Greater clinical benefit with tiomolibdate choline versus standard-of-care in neurologic Wilson disease patients in the Phase 3 FoCus Trial," reported that patients with neurologic symptoms at baseline showed significant neurologic improvement over time and greater global clinical improvement compared with standard-of-care therapy1. Separate Phase 2 data presented at the EASL Congress on May 29, 2026 showed that in a heavily pre-treated Wilson disease population, ALXN1840 stabilized liver disease and produced clinically meaningful improvements in neurologic symptoms and quality of life, as the company described it1.
On leadership, the release said Susan Rodriguez, who became Chief Commercial and Strategy Officer in March 2026, is overseeing preparation for a possible commercial launch1.
Financially, Monopar reported cash, cash equivalents and investments of $134.3 million as of June 30, 20261, and said this funding is expected to support operations through at least December 31, 2027, covering regulatory and potential commercial work on ALXN1840, continued MNPR-101 development, and internal R&D1. Net loss for the quarter was $5.3 million, or $0.62 per share, versus $2.5 million, or $0.35 per share, in the same quarter of 20251.
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