1 May 2018 - - The US Food and Drug Administration has granted breakthrough therapy designation to Seattle, Washington-based small-molecule and protein therapeutics specialist Omeros Corp. (NASDAQ: OMER) for OMS721 for the treatment of patients with high-risk hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), specifically those patients who have persistent TMA despite modification of immunosuppressive therapy, the company said.
OMS721 is Omeros' lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system.
Breakthrough therapy designation was granted based on data from Omeros' Phase 2 clinical trial evaluating OMS721 in patients with high-risk HSCT-TMA. Phase 3 clinical programmes are in progress for OMS721 in atypical hemolytic uremic syndrome, in immunoglobulin A nephropathy and in HSCT-TMA.
Two Phase 2 trials are ongoing.
Omeros has established a compassionate-use program for OMS721, which is active in both the US and Europe.
Omeros is committed to discovering, developing and commercializing both small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, coagulopathies and disorders of the central nervous system.
It has multiple Phase 3 and Phase 2 clinical-stage development programs focused on complement-associated thrombotic microangiopathies, complement-mediated glomerulonephropathies, Huntington's disease and cognitive impairment, and addictive and compulsive disorders.