Omeros' OMS824 Huntington's disease programme receives US FDA fast track designation

The US Food and Drug Administration (FDA) has granted Fast Track designation to Omeros Corporation's phosphodiesterase 10 (PDE10) inhibitor, OMS824 for the treatment of cognitive impairment in patients with Huntington's disease. OMS824 selectively inhibits PDE10, an enzyme expressed in areas of the brain linked to a wide range of diseases that affect cognition, including Huntington's disease and schizophrenia.

Omeros has conducted successful clinical trials assessing the safety, tolerability, pharmacokinetics and target engagement of a wide range of doses of OMS824 in its phase I programme. Positive data from the company's OMS824 phase IIa schizophrenia trial were recently announced, and Omeros expects to begin enrolling patients this quarter in its phase II trial evaluating OMS824 for Huntington's disease.

FDA's Fast Track programme facilitates the development of drugs intended to treat serious or life-threatening conditions and that have the potential to address unmet medical needs. A drug programme with Fast Track status is afforded greater access to the FDA for the purpose of expediting the drug's development, review and potential approval. Many drugs that receive Fast Track designation are also considered appropriate to receive Priority Review, and their respective New Drug Applications (NDAs) may be accepted by the FDA as a "rolling submission" in which portions of an NDA are reviewed before the complete application is submitted. Priority Review and rolling submission can each provide further acceleration of FDA's approval process.

"FDA's Fast Track designation of OMS824 for Huntington's disease reflects the unmet need associated with cognitive impairment in Huntington's patients and recognizes the drug's potential to treat this condition," stated Gregory A Demopulos, MD, chairman and chief executive officer of Omeros. "Together with the orphan drug status previously awarded OMS824 by the FDA for Huntington's, we have the opportunity to streamline the development of this promising compound. We look forward to enrolling patients in our OMS824 phase II clinical trial for Huntington's disease within the next few weeks."

PDE10 is an enzyme that is expressed in areas of the brain linked to diseases that affect cognition and psychomotor functions, including Huntington's disease and schizophrenia. Huntington's disease is a hereditary neurodegenerative disorder that leads to movement, cognition, and behavioral abnormalities and premature death. Schizophrenia is a group of severe brain disorders characterized by an abnormal interpretation of reality, which can manifest as delusions, hallucinations, and/or disordered thinking and behavior. Cognitive dysfunction is responsible for substantial disability in both of these diseases and is not meaningfully improved by current medications.

Omeros' proprietary compound OMS824, currently in phase II clinical programmes, inhibits PDE10 and is being developed for the treatment of cognitive disorders. In addition to potential benefits on cognition, OMS824 could also improve the motor and psychiatric abnormalities in Huntington's disease as well as the positive (e.g., hallucinations) and negative (e.g., flat affect) symptoms of schizophrenia. Omeros has been awarded both Orphan Drug and Fast Track Designations by the US FDA to evaluate OMS824 in Huntington's disease, and a Fast Track application to the FDA for the evaluation of OMS824 in schizophrenia is currently under review.

Omeros is a clinical-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics targeting inflammation, coagulopathies and disorders of the central nervous system.