Novel composition-of-matter patent supports formulation innovation as development advances and patient access expand
CHICAGO — April 9, 2026 —Emalex Biosciences today announced that the U.S. Patent and Trademark Office issued a patent covering an orally disintegrating tablet (ODT) formulation of ecopipam, an investigational D1 receptor antagonist in development for the treatment of Tourette syndrome.
The milestone comes as Emalex’s Expanded Access Program for ecopipam is enrolling up to 200 patients across the United States, reflecting continued progress across the company’s development and access initiatives.
“Developing alternative dosage forms is a long-term effort that matters because how a medicine is delivered can be as important to patients as the medicine itself,” said Emalex Biosciences CEO Eric Messner. “This issued patent reflects our formulation approach supporting the ODT program, while our Expanded Access Program underscores our commitment to patients.”
Emalex plans to evaluate the ODT formulation through a development pathway that includes process development, scale-up, cGMP manufacturing, stability, and a first-in-human pharmacokinetic study, targeted for late 2026 or early 2027. Planned clinical work will assess the performance of low-dose ODT formulations compared with the immediate-release tablet, along with standard tolerability and palatability assessments.
The Expanded Access Program will enroll approximately 200 patients at sites across the United States, providing a pathway for eligible patients who have exhausted approved treatment options to access ecopipam outside of clinical trials. Physicians may request ecopipam for patients who have been treated with an FDA-approved therapy for Tourette syndrome, including aripiprazole, haloperidol, pimozide or another D2 receptor antagonist, and who have experienced treatment failure, tolerability, safety concerns or lack access to approved medications.
Tourette syndrome is a chronic, childhood-onset neurodevelopmental disorder characterized by motor and vocal tics and is associated with significant morbidity that can disrupt school, work and social functioning. Despite available treatments, many patients continue to experience inadequate symptom control or tolerability challenges.
Participation in the Expanded Access Program requires physician oversight, FDA authorization, and Institutional Review Board approval. Enrollment is limited, and eligibility is determined on a case-by-case basis. Patients and clinicians with questions can contact Emalex Biosciences at medicalaffairs@emalex.com.
About Ecopipam
Ecopipam is a first-in-class investigational compound being studied as a potential treatment for certain central nervous system disorders. It blocks dopamine activity at the D1 receptor. Dopamine receptors are grouped into two families based on genetic structure: D1, which includes D1 and D5 subtypes, and D2, which includes D2, D3, and D4 subtypes. D1 receptor supersensitivity may contribute to the repetitive and compulsive behaviors associated with Tourette syndrome. Currently approved therapies act at D2 receptors. Ecopipam has been generally well tolerated in clinical trials conducted to date and has received Orphan Drug and Fast Track designation from the FDA for the treatment of pediatric patients with Tourette syndrome. Adverse events reported in clinical trials have primarily affected the central nervous system and have included headache, fatigue, somnolence, insomnia, restlessness, anxiety, depression and, rarely, suicidal ideation.
About Emalex Biosciences
Emalex Biosciences was created by Paragon Biosciences to develop new treatments for central nervous system disorders. The company is advancing a new class of therapy for patients with Tourette syndrome and other conditions with limited treatment options. Learn more at emalexbiosciences.com.
About Paragon Biosciences
Paragon Biosciences, founded by Jeff Aronin, creates, builds and funds innovative biology-based companies. Its portfolio companies advance scientific breakthroughs aimed at addressing significant unmet medical needs. Learn more at www.paragonbiosci.com.
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