A First-in-class Topical Immunoregulatory Therapeutic for Psoriasis

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A First-in-class Topical Immunoregulatory Therapeutic for Psoriasis BioTherapeutics Inc (BTI) is an emerging biotech company that synergistically combines the power of advanced computational modeling with translational experimentation to accelerate the development of novel product candidates for precision immunology. An unmet clinical need for safer, more effective drugs for plaque psoriasis remains as current therapies have limited efficacy and adverse side effects. The Technology & Product. Psoriasis (PsO) is a global health concern that affects 125 million people worldwide. PsO is presented as scaly and erythematous skin plaques in the scalp, elbows, knees, and lower back, associated with severe itchiness and persistent rashes that have a significant detrimental impact on quality of life. Despite advancements in treatments, an unmet clinical need remains for the development of novel therapeutics against plaque psoriasis. This SBIR application aims to advance the development of a novel first- in-class therapeutic that is being evaluated for the treatment of multiple autoimmune diseases as a well-tolerated, topical therapeutic for the treatment of plaque psoriasis. The Specific Aims for the SBIR Phase I application are to: 1. Evaluate the permeability and long-term stability of the novel topical drug product formulation. We will further characterize the topical formulation developed by in vitro permeation testing and long- term stability in preparation for the initiation of the clinical studies. 2. Compare the therapeutic efficacy of our product candidate to current approved PsO therapeutics in a mouse model of psoriasis. We will use the imiquimod mouse model of psoriasis to evaluate disease activity, histological lesions, plus local and systemic inflammation. 3. Determine the translational potential in human primary cells. We will use blood samples from psoriasis patients to investigate the new candidate’s functional ability to induce immunoregulatory changes in patients. Expected successful outcomes: i) >10% permeation by IVPT; ii) >40% decrease in composite score on day 7 compared to other drugs; and iii) >40% decrease in Th17 differentiation in human primary cells. Our long-term goal is to develop safer, more effective first-in-class immunoregulatory therapeutics for PsO with a novel mechanism of action that address an unmet clinical need for a market exceeding $12 billion and growing 5% annually.