In numerous rare diseases, the complement alternative pathway (CAP) is activated by artificial surface, body's own proteins and cells, bacteria and viral surfaces. In these settings, dysregulation of CAP activity induces inflammation and cellular destruction which is further exacerbated to cause chronic rare diseases. NovelMed’s strategy to selectively target the CAP has led to the development of several First-in-class antibody-based drugs that have shown remarkable results in disease models.
Whereas the CAP is known to play a significant role in pathogenesis of numerous rare diseases, it is well accepted that the complement classical pathway (CCP) must remain intact to protect the patients from an unwanted bacterial and viral attacks and therefore CCP plays a pivotal role in host defense against infection. Whereas both CAP and CCP are intertwined and generally considered inseparable. Through our innovation, we have redefined the science that separates the two well accepated pathways CAP and CCP. This First-in-class strategy is unique and has been used for the selection of the clinical candidates suitable for the rare diseases which are complement mediated. The CCP is important for patient's well being and therefore must be preserved during drug treatments. Our drugs selectively target the CAP which is the only pathway dysregulated in many rare diseases.
NM8074 is the first protease inhibitor inhibitor discovered to block the activity of the two powerful proteases of the CAP. The molecule has no effect on the activity of the two proteases of the CCP. Therefore, NM8074, a First-in-class molecule, is a selective blocker of the CAP and CAP associated inflammatory responses and cellular damages associated with rare disease. The drug binds Factor B and; a) neutralizes the activity of both C3 and C5 CAP convertases, b) blocks the production of C3b responsible for extravascular hemolysis in PNH and kidney failure in C3G patients, and c) formation and deposition of the killer MAC (C5b-9). The two powerful properties among many others makes the drug viable for the treatment of many rare-diseases where CAP plays an important role in rare disease pathogenesis.
Our approach is to block the production of multiple inflammatory mediators with a single product (anti-complement antibody). We believe that blockade of multiple inflammatory mediators via a singular mechanism is the key to developing multiple-targeted therapies from a single site of action. Such a desirable approach makes us unique in the inflammatory space from the mechanistic standpoint.