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Noema Pharma Announces Recruitment of First Patient in Orpheus Phase 2b Study of PDE10A Inhibitor gemlapodect in Adults with Childhood Onset Fluency Disorder

Friday, August 05, 2022

Noema Pharma, a clinical-stage biotech company targeting debilitating central nervous system (CNS) indications, today announced the dosing of the first patient in the Orpheus Phase 2b study of Noema’s PDE10A inhibitor gemlapodect (NOE-105) in Childhood Onset Fluency Disorder (COFD—commonly known as stuttering).

Orpheus is a multi-center, 11-week prospective, placebo-controlled study to evaluate the safety and efficacy of gemlapodect as monotherapy in adults suffering from COFD. The study is expected to report at the end of 2023.

"Recruiting the first participant is an important step in the clinical investigation of gemlapodect in this indication. I am very pleased with the progress we are making in the Orpheus study with significant interest from people struggling from the condition to participate in the study," said Dr Gerald Maguire, Principal Investigator of the Orpheus Study. "Our main goal is to evaluate the safety and efficacy of gemlapodect as a monotherapy for COFD in adults. People who stutter are looking for solutions that would enable them to achieve their full potential, both personally and professionally. In the absence of any FDA approved therapies for this condition that affects over one percent of our population, persons who stutter are eagerly awaiting an effective, safe, and proven therapy."

“The World Stuttering Network is committed to helping people who stutter at different stages of their lives”, said Tom Scharstein, president of the World Stuttering Network. “In their early adulthood, people who stutter face significant challenges in their social and professional functioning, specifically as they are in search of employment and expanding their social network beyond their childhood friends. Providing them with help to overcome these challenges would enable them to reach their optimal potential."

"Treatment options for Childhood Onset Fluency Disorder are severely lacking, with currently no FDA-approved therapy," said George Garibaldi, MD, President and Global Head of Research and Development at Noema Pharma. "The Orpheus study presents an exciting opportunity to investigate the potential of a novel therapeutic candidate in this common but often overlooked condition, which can have devastating effects in the personal and professional lives on those who suffer from it. The study also marks the expansion of Noema Pharma's clinical pipeline and complements Noema’s continued commitment to our mission of developing therapies for underserved conditions of the central nervous system."

Childhood onset fluency disorder (COFD), also known as stuttering, is a neurodevelopmental disorder most often exhibited in patients at an early age, affecting ~5% of children aged six and older. While most children grow out of their stuttering by their adolescent years, approximately 20% continue to be affected by the condition into adulthood, leading to significant disability at school, work, and in their social life. While no medication has been specifically approved for this disorder, patients often rely heavily on off-label use of antipsychotics, which provide some clinical benefit but are associated with significant long-lasting motor and metabolic side effects, including insulin resistance and type 2 diabetes.

Gemlapodect is an inhibitor of PDE10A, an enzyme highly expressed in medium spiny neurons of the mammalian striatum, which modulates dopamine D2 receptor signaling and does not appear to be associated with the metabolic abnormalities that are typically observed with most second-generation antipsychotics.

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