The complement Alternative Pathway (AP) is activated in numerous rare diseases related to hematological, renal, neurological, inflammatory and ocular disorders. Our intellectual property addresses these diseases, and with time we plan to target additional diseases where our drug candidates are expected to demonstrate therapeutic efficacy.
Our high affinity and highly potent antibodies selectively block the AP without affecting the classical pathway (CP). The CP remains fully functional and its functions, such as opsonization and bacterial clearance, remain intact.
Numerous molecules of C3a, C3b, C5a, C5b and MAC are produced via AP in a disease state. Our lead drug candidates block the formation of each one of these molecules, via AP, to benefit in a variety of chronic diseases.
Our laboratory model systems have proven that our clinical candidates would effectively treat complement-mediated diseases. Our current therapeutic antibody landscape is unique and tailored to treat rare diseases that continue to have an unmet need despite treatment. Our drug candidates selectively block the AP and leave the classical pathway intact for host defense, which is critical to keep patients free of infections during treatment for chronic conditions.
Two lead candidates (NM8074 and NM5072) have completed phase I trial in healthy volunteers. Multiple Phase II trials have been registered at www.clinicaltrials.gov. A Phase II clinical trial in naive PNH patients is currently in progress. We continue to file for regulatory approval for various other clinical indications listed below.
We lead the knowhow in the Alternative Pathway demonstrated by our issued patent portfolio. Most of the companies in this space, including Apellis, Novartis, and Alexion, have followed our lead in their respective programs.
Our preclinical research was supported by significant funding from the National Institute of Health. Our Clinical program was funded by licensing to a large pharma. Our clinical results are validated by the FDA, evidenced by their approval of IND for multiple indications and orphan disease designation of our lead antibodies.
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