Developing Oral Treatment for Rare Autoimmune Diseases with Ben Zimmer Priovant Therapeutics

Ben Zimmer, CEO of Priovant Therapeutics, focuses on developing therapies for rare autoimmune diseases specifically dermatomyositis, which affects the skin, muscles, and organs, and non-infectious uveitis, a severe ocular inflammatory condition. While these conditions are symptomatically different, mechanistically, they have features in common related to the underlying pathology of the diseases. The drug in development is an oral once-daily therapy that addresses the inflammatory conditions and cytokines driving the pathology. Ben explains, "During COVID, there was a lot of talk about the cytokine storm and these are molecules involved in immune cell signaling. So, they're basically ways that different types of immune cells signal to each other to do different things. There's a large variety of different cytokines and some autoimmune diseases. There are only maybe one or two cytokines or a small number that are driving the pathology of the disease."   "Both dermatomyositis and non-infectious uveitis are highly inflammatory conditions with a lot of different pathogenic cytokines, a lot of different inflammatory axes involved. And so, one of the neat things about our drug, brepocitinib, is that it works on a set category of enzymes called JAKs, and then there are four different types of JAKs, JAK1, JAK2, JAK3, and you might guess JAK4, but it's actually called TYK2. And our drug inhibits TYK2 and JAK1. And by doing that, it suppresses the signaling of a large number of different cytokines." "Uveitis and dermatomyositis have a number of overlapping pathogenic cytokines involved, as well as some different cytokines, but they are both suppressed through the inhibition of TYK2 and JAK1, which our drug does. So, our thesis is to find these highly inflammatory indications, highly inflammatory diseases with high morbidity, which can be expressed in many different organ systems." #PriovantTherapeutics #RareDisease #OrphanIndication #Uveitis #NIU #Dermatomyositis #JAK1 #TYK2 priovanttx.com  Download the transcript here