A First-in-class Topical Immunoregulatory Therapeutic for Psoriasis

一流的牛皮癣局部免疫调节疗法

基本信息

批准号：
10820331
负责人：
Nuria Tubau Juni
金额：
$ 29.53万
依托单位：
BIOTHERAPEUTICS, INC.
依托单位国家：
美国
项目类别：
财政年份：
2023
资助国家：
美国
起止时间：
2023-09-20 至 2025-03-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10820331
关键词：
Acceleration Activities of Daily Living Address Adrenal Cortex Hormones Advanced Development Affect Anti-Inflammatory Agents Antibodies Atopic Dermatitis Autoimmune Diseases Back Benign Betamethasone Binding Biological Assay Biological Availability Biological Products Biological Response Modifier Therapy Biopsy Biopsy Specimen Biotechnology Blood specimen CD4 Positive T Lymphocytes Cells Chronic Disease Chronic small plaque psoriasis Clinical Clinical Research Computer Models Cream Crohn&apos s disease Data Development Disease Disease remission Dose Dose Limiting Elbow Eosinophilic Esophagitis Epithelial Cells Exanthema Formulation Goals High Pressure Liquid Chromatography Histologic Human IL17 gene Imiquimod Immune Immune system Immunology Immunosuppressive Agents In Vitro Inflammation Mediators Inflammatory Inflammatory Response Interleukin-6 Journals Knee Knowledge Lead Lesion Ligase Marketing Modeling Oral Outcome Pathway interactions Patients Permeability Persons Pharmaceutical Chemistry Pharmaceutical Preparations Phase Preparation Proliferating Psoriasis Public Health Quality of life Regulatory T-Lymphocyte Safety Scalp structure Skin Skin Plaques Small Business Innovation Research Grant Technology Testing Therapeutic Topical Corticosteroids Toxic effect Translations Treatment Efficacy Ulcerative Colitis Validation commercial application experimental study global health immunoregulation inhibitor interleukin-23 keratinocyte lanthionine mouse model novel novel drug class novel therapeutics pharmacologic phase 3 study programs public health relevance research clinical testing response side effect success systemic inflammatory response translational potential

项目摘要

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.

牛皮癣的第一类局部免疫调节治疗 Biotherapeutics Inc（BTI）是一家新兴的生物技术公司通过翻译实验的高级计算建模，以加速新型产品候选者的精确免疫学。未满足的临床需求对牙菌斑的更安全，更有效的药物牛皮癣仍然是当前疗法的有效性和不良副作用。技术和产品。牛皮癣（PSO）是全球健康问题，影响1.25亿人全世界。 PSO在头皮，肘部，膝盖和较低背面，与严重的瘙痒和持续性皮疹有关，对质量产生重大不利影响生活。尽管治疗方面取得了进步，但仍未满足的临床需求为开发新颖针对斑块牛皮癣的治疗剂。该SBIR应用程序旨在推动新颖的首先发展正在评估用于治疗多种自身免疫性疾病的课堂疗法作为一种耐受性良好，用于治疗斑块牛皮癣的局部治疗方法。 SBIR I期应用程序的具体目的是： 1。评估新型局部药物产品配方的渗透性和长期稳定性。我们将进一步表征通过体外渗透测试开发的局部公式和长期为启动临床研究准备的术语稳定性。 2。将我们的产品候选者的治疗效率与当前批准的PSO治疗进行比较在牛皮癣的小鼠模型中。我们将使用牛皮癣的咪喹莫德小鼠模型来评估疾病活性，组织学病变，以及局部和全身性炎症。 3。确定人类原代细胞中的翻译潜力。我们将使用来自牛皮癣患者调查新候选者诱导免疫调节的功能能力患者的变化。预期的成功结果：i）> IVPT渗透10％； ii）>第7天的复合分数下降40％与其他药物相比； iii）>> 40％的人类原代细胞中Th17分化的降低。我们的长期目标是为PSO开发更安全，更有效的第一类免疫调节疗法一种新型的行动机制，该机制解决了超过120亿美元的市场的未满足临床需求并增长每年5％。