Omeros’ Investigational New Drug Application for OMS906 Cleared by FDA
-- Paroxysmal Nocturnal Hemoglobinuria Slated as Initial Indication --
FDA’s clearance of the IND allows the initiation of the clinical program for OMS906. A Phase 1, placebo-controlled, double-blind, single-ascending-dose and multiple-ascending-dose study will evaluate the safety, tolerability, pharmacodynamics and pharmacokinetics of OMS906 administered subcutaneously and intravenously to healthy subjects. Enrollment is planned to begin in early September. The trial will provide additional information regarding the anticipated OMS906 dosing regimen in patients, which is targeted for once monthly subcutaneous administration. Following adequate Phase 1 data collection and analysis, the initial Phase 2 clinical trial is planned in patients with life-threatening paroxysmal nocturnal hemoglobinuria (PNH), a rare disease characterized by red blood cell destruction, blood clots and impaired bone marrow function.
“FDA’s decision clears the way for us to begin clinical trials with OMS906,” stated
MASP-3 is responsible for the conversion of pro-factor D to factor D. It is believed to be the premier target in the alternative pathway with the lowest native circulating level and low relative clearance compared to the other alternative pathway proteins. Also, unlike C5 and C3 blockers, MASP-3 inhibition leaves intact the lytic arm of the classical pathway, important for fighting infection. Omeros has created a broad intellectual property position directed to the use of MASP-3 inhibitors, including OMS906, for the treatment of a wide range of disorders related to dysfunction or excessive activation of the complement system’s alternative pathway. As part of its complement franchise, Omeros also broadly controls the inhibition of MASP-2, the effector enzyme of the lectin pathway of complement. A rolling biologics license application (BLA) for Omeros’ lead MASP-2 inhibitor narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) is targeted for completion this quarter. Narsoplimab is also in Phase 3 clinical programs for immunoglobulin A (IgA) nephropathy and atypical hemolytic uremic syndrome and is being advanced to treat critically ill patients with COVID-19.
OMS906 is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-3 (MASP-3), the key activator of the complement system’s alternative pathway. The complement system plays a central role in inflammation and becomes activated as a result of tissue damage or microbial infection. Responsible for the conversion of pro-factor D to factor D, MASP-3 is believed to be the premier target in the alternative pathway – it has the lowest native circulating level and low relative clearance compared to the other alternative pathway proteins and, unlike C5 and C3 blockers, MASP-3 inhibition leaves intact the lytic arm of the classical pathway, important for fighting infection. MASP-3 inhibitors are thought to have preventive or therapeutic effects across a broad range of diseases including paroxysmal nocturnal hemoglobinuria (PNH), hemolytic uremic syndrome (HUS), atypical HUS, traumatic brain injury, arthritis, wet age-related macular degeneration, ischemia-reperfusion injury, transplant-related complications and other immune-related disorders. Through its growing intellectual property position, Omeros controls the use of MASP-3 inhibitors across a wide range of alternative pathway-related diseases and disorders.
Omeros is a commercial-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market and orphan indications targeting inflammation, complement-mediated diseases, disorders of the central nervous system and immune-related diseases, including cancers. In addition to its commercial product OMIDRIA (phenylephrine and ketorolac intraocular solution) 1%/0.3%, Omeros has multiple late-stage clinical development programs focused on complement-mediated disorders, including COVID-19, and substance abuse. A rolling biologics license application for narsoplimab, the company’s lead MASP-2 inhibitor, in hematopoietic stem cell transplant-associated thrombotic microangiopathy is being completed for submission to the
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