Biotherapeutics, Inc. — Department of Health and Human Services SBIR Phase I: NIAMS

Description

Development of Novel Topical Therapeutics for Psoriasis BioTherapeutics Inc (BTI) is an emerging biotech company that synergistically combines the power ofadvanced computational modeling with translational experimentation to accelerate the development of novelproduct candidates for precision medicine and health. An unmet clinical need for safer, more effective drugsfor psoriasis remains as current therapies have limited efficacy and adverse side effects. Psoriasis afflicts over 7 million people in the United States and 15 million worldwide. Current therapies forpsoriasis have limited long-term efficacy but significant adverse side effects. Three-fourths of psoriasis patientsare managed with topical therapies, yet many of these therapies remain corticosteroids, which struggle to offer sustained remission and have been implicated in decrease responsiveness to treatment in a variety of autoimmune diseases. Meanwhile, roughly half of patients on oral or systemic psoriasis treatments willdiscontinue therapy due to loss of response or concern over side effects. Therefore, there is an unmet clinicalneed for the development of more effective, safer, topical therapeutics for the treatment of psoriasis. This SBIRPhase I application aims to develop a topical, first-in-class therapeutic for the treatment of psoriasis through anovel mechanism of action at the crosstalk between keratinocytes and immune cells.The Specific Aims for the SBIR Phase I application are to: (1) Characterize the therapeutic efficacy of our new therapeutic in a mouse model of psoriasis to determinethe effects on keratinocytes and immune cells through histological, metabolic and flow cytometry analysis. (2) Develop a topical drug product formulation through generation of five or more prototype formulations andtesting permeation in vitro and therapeutic efficacy in a mouse model of psoriasis. (3) Evaluate the translational efficacy in human primary cells from psoriasis patients through assessment ofcytokine profile and functional assays.Expected successful outcomes: i) 50% reduction in composite score on day 7 with at least one dose of treatment in vivo, ii) rt10% permeation by IVPT in our lead topical prototype, and iii) ≥ 40% decrease in IL-17 and IFNγ production in primary T cells treated with our novel therapeutic. The SBIR Phase II will perform IND-enabling safety and toxicology studies on the finalized topical formulation of our compund, characterize the efficacy of combination therapies of our topical new therapeutic with currently approved systemic agents for psoriasis, and initiate Phase 1b clinical testing in psoriasis patients. Commercial Application: This SBIR will develop a first-in-class topical therapeutic for psoriasis for addressing an unmet clinical need of 15 million patients, and a global market exceeding $12 billion annually.