Eton Pharmaceuticals Reports Positive Pivotal Study Results for ET-600

DEER PARK, Ill., March 14, 2025 - Eton Pharmaceuticals, Inc., a forward-thinking pharmaceutical company specializing in treatments for rare diseases, has announced promising outcomes from its bioequivalence study of ET-600. This unique oral solution, using desmopressin, is being developed for managing central diabetes insipidus. The study involved 75 human participants, confirming that ET-600's pharmacokinetic profile matches closely with the FDA-approved reference product containing the same active ingredient.

The study was meticulously structured, involving healthy adult participants who were randomly assigned to receive either the test drug or the reference drug. It followed an open-label, balanced, randomized, single-dose, three-treatment, three-sequence, three-period, three-way crossover design. The success of this trial is a significant milestone for Eton, as the company is planning to file a New Drug Application (NDA) with the U.S. FDA for ET-600 by April 2025. Pending approval, ET-600 would become the only FDA-approved oral liquid formulation of desmopressin available in the market.

Sean Brynjelsen, CEO of Eton Pharmaceuticals, expressed enthusiasm about ET-600 moving forward in the development pipeline, closer to patient availability. He emphasized the importance of their relationships within the pediatric endocrinology sector, highlighting the necessity for medications like desmopressin that allow precise, adjustable dosing crucial for pediatric patients. Brynjelsen mentioned that Eton is preparing for the NDA submission and has initiated pre-launch activities to gear up for a potential market release in early 2026.

ET-600 is an innovative formulation designed for central diabetes insipidus treatment. The formulation has secured a patent extending to 2044, with another patent application currently under review by the United States Patent and Trademark Office. Central diabetes insipidus is a rare condition affecting approximately 3,000 pediatric patients across the United States.

Eton Pharmaceuticals is a pioneer in the pharmaceutical industry, concentrating on the development and commercialization of therapies for rare diseases. Presently, the company markets seven rare disease products, including INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. Besides ET-600, Eton has four additional product candidates in advanced stages of development: ET-400, Amglidia®, and ZENEO® hydrocortisone autoinjector.

This advancement with ET-600 underscores Eton's commitment to addressing unmet medical needs in rare disease communities, particularly for conditions that require specialized treatment approaches. As the company progresses with its regulatory plans, it continues to solidify its position as a leader in rare disease therapeutics, poised to bring innovative solutions to patients who need them most.