Omeros Corporation (OMER) recently announced that the company has filed an Application for Orphan Drug Designation with the US Food and Drug Administration (:FDA) for its candidate OMS721.
OMS721 is the leading human monoclonal antibody for Omeros’ mannan-binding lectin-associated serine protease-2, or MASP-2 program.
The filing comes close on the heels of the positive preclinical data announced by the company on OMS721 in a model of thrombotic microangiopathy (TMA) earlier in the month.
We note that TMA is a disorder in the microcirculation of the body's organs, in particular the kidney and brain.
Omeros plans to initiate clinical trials for OMS721 initially for use in the treatment of atypical hemolytic uremic syndrome (aHUS), which is a rare and dangerous form of TMA.
Omeros plans to file for the same designation in Europe as well. We note that Alexion’s (ALXN) Soliris is also indicated for aHUS.
Meanwhile, in another positive development, Omeros announced positive results from the multiple-ascending-dose (:MAD) section of the company’s phase I study on OMS824 in Mar 2013. We note that OMS824 is the lead compound in Omeros' phosphodiesterase 10 (PDE10) program.
In Dec 2012, Omeros reported promising data from the single-ascending-dose (:SAD) study portion of a phase I study on OMS824. Based on the positive data published by the company on OMS824 in Dec 2012 and Mar 2013, it now plans to advance the candidate into phase II development.
Omeros is developing the candidate as a PDE10 inhibitor, which is an enzyme expressed in areas of the brain linked to a wide range of diseases, including Huntington's disease and schizophrenia, that affect cognition.
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