FDA Clears IND for PNH Treatment OMS906

On August 31, 2020, biopharmaceutical company Omeros Corporation announced that it will soon move forward with clinical trials for OMS906. This decision follows clearance of an Investigational New Drug Application (IND) by the FDA. Currently, OMS906 is designed to treat patients with severe paroxysmal nocturnal hemoglobinuria (PNH). However, the first clinical trials will be used to test safety, efficacy, tolerability, and both pharmacodynamics and pharmacokinetics in health volunteers. OMS906 can be administered either intravenously or subcutaneously.


So what is OMS906? The therapy is described as a human monoclonal antibody which targets mannan-binding lectin-associated serine protease-3 (MASP-3). That’s quite a mouthful, so let’s unpack it. According to the National Cancer Institute (NCI), a human (or humanized) monoclonal antibody is:

A type of antibody made in the laboratory by combining a human antibody with a small part of a mouse or rat monoclonal antibody. The mouse or rat part of the antibody binds to the target antigen, and the human part makes it less likely to be destroyed by the body’s immune system.

In this case, the antibody binds to MASP-3. This activates an alternative pathway in the complement system. On a physiological level, the complement system regulates inflammation. When MASP-3 malfunctions, it can cause an immune system overreaction. In fact, in patients with PNH, the immune system attacks stem cells. By inhibiting MASP-3, OMS906 allows an immune response while also managing the excess activation. In addition to PNH, MASP-3 inhibitors can potentially help patients with atypical HUS, traumatic brain injury, arthritis, wet macular degeneration, and more.

Omeros Corporation begins enrolling clinical trial participants in September. Should the trial be successful, the organization will launch a Phase 2 clinical trial for patients with PNH.

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal nocturnal hemoglobinuria (PNH) is an inherited hematopoietic stem cell disorder. Caused by PIGA gene mutations, PNH affects red and white blood cells, as well as platelets. The genetic mutation stimulates the production of PNH cells. These cells survive when the immune system attacks stem cells. As a result, platelets are abnormal and red blood cells are destroyed. Generally, PNH is diagnosed between ages 35-40. An estimated 30% of cases resulted from aplastic anemia treatment. On average, the survival rate is 10 years following diagnosis.

Symptoms include:

  • Chest and abdominal pain
  • Dark or bloody urine
  • Kidney disease
  • Sexual dysfunction
  • Fatigue
  • Blood clots
  • Easy bruising and bleeding
  • Shortness of breath / difficulty breathing
  • Headache
  • Red blood cell destruction
  • High heart rate
  • Difficulty swallowing

Learn more about PNH.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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