Omeros announced that the FDA has granted breakthrough therapy designation to OMS721 for the treatment of Immunoglobulin A (IgA) nephropathy. 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 IgA nephropathy and other kidney diseases. The data were recently presented at the 54th European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) Congress in Madrid. Proteinuria is an important marker for disease progression in patients with IgA nephropathy, and improvement in proteinuria is associated with improved clinical outcomes. The clinical trial data show unprecedented improvement in proteinuria following only 12 weeks of OMS721 treatment, with a 77-percent mean reduction in urine albumin-to-creatinine ratios (P = 0.026) and a 73-percent mean reduction in 24-hour urine protein levels (P = 0.013). In response, many physicians attending ERA-EDTA in Madrid and representing centers across Europe, the US and Asia that manage large numbers of IgA nephropathy patients asked to participate in Omeros’ planned phase 3 clinical trial. These physicians have been added to the ongoing clinical site evaluation for the phase 3 clinical program.
FDA’s breakthrough therapy designation enables expedited development and review of a drug candidate for the treatment of a serious or life-threatening disease. Preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over existing therapies is required. Benefits of breakthrough therapy designation include the eligibility for priority review of the application and rolling submission of portions of the application. FDA personnel, including senior management, provide guidance to the company to determine the most efficient route to approval. OMS721 is the only drug candidate in development for the treatment of IgA nephropathy that has been granted breakthrough therapy designation by FDA.
“We are pleased that FDA has granted breakthrough designation to OMS721 for IgA nephropathy and appreciate the agency’s recognition of the potential importance of OMS721 in the treatment of this disease,” Gregory A. Demopulos, MD, chairman and chief executive officer of Omeros, said in a company news release. “OMS721 appears to be helping IgA nephropathy patients with a rapidity and magnitude not previously seen with any other therapy, and we look forward to working closely with the FDA to accelerate its development.”
There is no approved treatment for IgA nephropathy. The most common primary glomerulopathy globally, it accounts for up to 10 percent of all dialysis patients. In the US alone, an estimated 120,000 to 180,000 patients have this disease. Approximately 40 percent of IgA nephropathy patients develop end-stage renal disease, a life-threatening condition, within 20 to 30 years following diagnosis.
OMS721 is also being evaluated in a phase 3 clinical program for atypical hemolytic uremic syndrome and in a phase 2 clinical program for hematopoietic stem cell transplant-associated thrombotic microangiopathy.