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We are a late-stage biopharmaceutical company focused on developing novel therapies for immune-mediated diseases. Our proprietary Tailored Covalency® platform enables us to design and develop reversible covalent and irreversible covalent, small molecule inhibitors with potencies and selectivities that we believe will rival injectable biologics, but with the convenience of an oral or topical therapy. Our overall portfolio includes rilzabrutinib, PRN2246/SAR442168, PRN473 Topical, PRN1371 and our oral immunoproteasome inhibitor program. We retain full, worldwide rights to rilzabrutinib, PRN473 Topical, PRN1371 and our oral immunoproteasome inhibitor program, and have an ongoing collaboration with Sanofi for PRN2246/SAR442168. The company has prioritized the portfolio and our current clinical pipeline includes rilzabrutinib (formerly known as PRN1008), PRN473 Topical, and PRN2246/SAR442168. Our lead candidate, rilzabrutinib, a wholly owned Bruton Tyrosine Kinase (BTK) inhibitor, is in a Phase 3 trial for the treatment of pemphigus (pemphigus vulgaris (PV) and pemphigus foliaceus (PF)), and in a Phase 1/2 trial for the treatment of Immune Thrombocytopenia (ITP). In addition, we anticipate initiating a Phase 2 trial of rilzabrutinib for the treatment of IgG4-Related Disease (RD). The following chart summarizes the status of the drug candidates in our current pipeline: We have developed a BTK inhibitor franchise that encompasses two differentiated oral drug candidates, rilzabrutinib and PRN2246/SAR442168, and one topical drug candidate, PRN473 Topical. Our most advanced drug candidate, rilzabrutinib, is designed to form a reversible covalent bond with the BTK enzyme. This bond confers enhanced selectivity and potential durable potency, which we believe will enable long-term, systemic treatment of chronic immunological disorders. The second drug candidate in our BTK franchise is PRN2246/SAR442168, an irreversible covalent BTK inhibitor that we designed to cross the blood-brain barrier and modulate immune cell function in the brain for the treatment of multiple sclerosis, or MS, and potentially other central nervous system, or CNS, diseases. The third drug candidate in our BTK franchise is PRN473 Topical, a topical reversible covalent BTK inhibitor that we designed for immune-mediated diseases that could benefit from localized application to the skin with low to no systemic exposure.
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