The US Food and Drug Administration (FDA) has cleared Omeros Corporation's Investigational New Drug Application (IND) to evaluate OMS824 in Huntington's disease.

OMS824 selectively inhibits phosphodiesterase 10 (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. OMS824 has shown promising results in animal models directly relevant to Huntington's disease and, as previously announced, OMS824 was well tolerated and exhibited favourable pharmacokinetic properties in a phase 1 clinical programme.
Omeros plans to advance OMS824 into phase 2 clinical trials for Huntington's disease next quarter and for schizophrenia later this year, the OMS824 IND for use in patients with schizophrenia having already been cleared by the FDA.

Omeros also announced that it has requested Orphan Drug Designation from the FDA for OMS824 in the treatment of Huntington's disease. Orphan-designated drugs are eligible for incentives such as a faster approval process and additional market exclusivity. This designation is granted to drugs that are expected to provide significant therapeutic advantage over existing treatments and that target conditions affecting 200,000 or fewer US patients annually. Huntington's disease is estimated to affect approximately 31,000 US patients annually, and the only FDA-approved treatment for the disease is tetrabenazine, which is indicated for Huntington's-related movement disorders. Omeros recently announced that it has requested Fast Track designation for OMS824 in the treatment for Huntington's disease. Fast Track designation is reserved for drugs being developed to treat life-threatening conditions with the potential to address unmet medical needs and typically provides priority review status.

"Cognitive impairment is a hallmark of Huntington's disease and schizophrenia, and there currently are no drugs that improve cognition in either disorder," stated Gregory A. Demopulos, chairman and chief executive officer of Omeros. "In Huntington's disease, OMS824 also has the potential to improve motor control and to reduce the rate of neurodegeneration, slowing disease progression. We are pleased that the FDA has now cleared the way for us to evaluate OMS824 in both patients with Huntington's disease and schizophrenia, and we look forward to initiating enrollment in our phase 2 clinical trials."

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 behavioural abnormalities and premature death. Cognitive dysfunction is responsible for substantial disability in these diseases and is not improved by current medications. Omeros' proprietary compound OMS824 inhibits PDE10 and is being developed for the treatment of cognitive disorders. In addition to potential benefits on cognition, OMS824 could also improve psychiatric manifestations, such as the positive (e.g., hallucinations) and negative (e.g., flat affect) symptoms of schizophrenia.

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