paroxysmal Nocturnal hemoglobinuria (PNH)

Our Committment to patients with PNH

paroxysmal Nocturnal Hemoglobinuria 

PNH is an orphan disease characterized by persistent anemia and continued hemolysis despite currently-approved therapies. In this rare and life-threatening blood disorder the immune system attacks unprotected red blood cells resulting in significant disease outcomes including cellular destruction and long-term anemia. Red Blood Cells without a protective coating (CD55/59 proteins) are recognized as foreign and are removed by the immune system.

We understand the disease process and therefore have developed products which will correct this destruction without attenuating simultaneously the positive aspects of immune surveillance against true invading antigens. Our antibodies target only the part of immune system (the alternative pathway) that are exacerbated in the disease and are therefore an improvement over those that are currently being marketed.

NovelMed has developed a pipeline of therapeutic antibody candidates for treating PNH, aHUS, and several others where chronic hemolysis is important in the disease pathogenesis. Our preclinical studies provide strong support for the development of our lead drug candidates with a high level of safety and efficacy. Our drug is also expected to have a greater delivery convenience due to its unique mechanism of action.

Currently marketed drugs for PNH occupy less than one-half of the total market. Furthermore, current drugs for this disease carry Black Box Warnings and can only control intravascular hemolysis, and leaves extravascular hemolysis present in most treated patients. Based on our laboratory findings and associated pre-clinical data, our drug could have a transformational effect with better efficacy, safety and potency. Based on the mechanism of action, our drug is expected to be free of black-box warnings.

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