Emalex Receives U.S. Patent for Orally Disintegrating Ecopipam Formulation

Novel composition-of-matter patent supports formulation innovation as development advances and patient access expands

CHICAGO--(BUSINESS WIRE)--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 [e-mail].

About Ecopipam

Ecopipam is a first-in-class investigational compound that is being studied as a potential treatment for certain central nervous system (CNS) disorders. It blocks the actions of the neurotransmitter dopamine at the D1 receptor. Dopamine is a neurotransmitter in the central nervous system, and its receptors have been classified into two “families” based on their genetic structure: “D1” (including subtypes D1 and D5) and “D2” (including subtypes D2, D3, and D4). D1 receptor super-sensitivity may be a mechanism for the repetitive and compulsive behaviors associated with Tourette syndrome. Currently approved therapies for the treatment of Tourette syndrome act at D2 receptors. Ecopipam has been shown to be 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 affecting primarily the central nervous system have been reported in clinical trials conducted to date, including 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 https://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 https://www.paragonbiosci.com.