A First-in-class Topical Immunoregulatory Therapeutic for Psoriasis

一流的牛皮癣局部免疫调节疗法

• 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
• 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' 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

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.

治疗银屑病的一流局部免疫调节治疗药物 BioTherapeutics Inc（BTI）是一家新兴的生物技术公司，它协同结合了 先进的计算建模与平移实验，以加速开发新的 用于精确免疫学的候选产品。对更安全、更有效的斑块药物的未满足的临床需求 牛皮癣仍然是因为目前的治疗方法具有有限的功效和不良副作用。 技术与产品。银屑病（PsO）是一个全球性的健康问题，影响着1.25亿人 国际吧PsO表现为头皮、肘部、膝盖和下肢的鳞状和扁平状皮肤斑块， 背部，伴有严重瘙痒和持续性皮疹，对质量有重大不利影响 生命尽管在治疗方面取得了进展，但对于开发新的抗肿瘤药物仍然存在未满足的临床需求。 治疗斑块状银屑病。这项SBIR应用旨在推动一种新型的第一代- 正在评估用于治疗多种自身免疫性疾病的同类治疗药物， 用于治疗斑块状银屑病的局部治疗剂。 SBIR第一阶段应用的具体目标是： 1.评价新型外用制剂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和长期- 为启动临床研究做准备的长期稳定性。 2.将我们的候选产品的治疗效果与目前批准的PsO治疗方法进行比较 在牛皮癣的小鼠模型中。我们将使用咪喹莫特银屑病小鼠模型来评估疾病 活动，组织学病变，加上局部和全身炎症。 3.测定人原代细胞中的翻译潜能。我们将使用来自 银屑病患者，以调查新候选人的功能能力，诱导免疫调节 患者的变化。 预期成功结局：i）IVPT渗透>10%; ii）第7天综合评分降低>40% iii）人原代细胞中Th 17分化降低>40%。 我们的长期目标是开发更安全，更有效的一流免疫调节治疗PsO， 一种新的作用机制，可解决超过120亿美元且不断增长的市场未满足的临床需求 每年5%。