Anti-scar peptide for cleft lip repair

用于唇裂修复的抗疤痕肽

• 批准号: 9474430
• 负责人: Chia Soo
• 金额: $ 96.81万
• 依托单位: SCARLESS LABORATORIES, INC.
• 依托单位国家: 美国
• 财政年份: 2017
• 资助国家: 美国
• 起止时间: 2017-09-11 至 2020-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9474430
• 关键词: Address; Adrenal Cortex Hormones; Adverse effects; Animal Model; Animals; Appearance; Area; Atrophic condition of skin; Award; Biological Assay; Birth; Bone Growth; Bone Tissue; Botox; Burn injury; Businesses; Capital; Cell Culture Techniques; Chemicals; Chemistry; Child; Cicatrix; Cleft Lip; Clinical; Clinical Research; Complication; Congenital Abnormality; Cosmetic surgery; Cosmetics; Cutaneous; Cyclic GMP; Deformity; Dermal; Dermis; Development; Dose; Drug Evaluation; Drug Kinetics; Ensure; Esthetics; Evaluation Research; Extracellular Matrix; Family suidae; Funding; Goals; Growth; Head; Human; Hypertrophic Cicatrix; Impaired wound healing; Injectable; Intellectual Property; Investigational Drugs; Investigational New Drug Application; Jaw; Laboratories; Lip structure; Live Birth; Maintenance; Marketing; Mechanics; Methods; Minority; Mission; Modeling; National Institute of Dental and Craniofacial Research; Operative Surgical Procedures; Outcome; Pathologic; Patients; Peptides; Pharmaceutical Preparations; Phase; Populations at Risk; Prevention strategy; Process; Production; Property; Proteins; Proteoglycan; Quality of life; Readiness; Regimen; Research Design; Research Institute; Resources; Risk; Safety; Second Look Surgery; Secure; Skin; Skin wound; Small Business Innovation Research Grant; Tensile Strength; Therapeutic; Toxic effect; Toxicology; Trauma; United States Food and Drug Administration; United States National Institutes of Health; Validation; Visual; base; cell motility; cleft lip and palate; clinical development; commercialization; congenital anomaly; craniofacial; design; drug development; effective therapy; fetal; fibromodulin; healing; improved; improved outcome; infancy; innovation; loss of function; meetings; migration; novel; patient population; programs; protein aminoacid sequence; psychologic; public health relevance; repaired; safety study; soft tissue; tool development; wound

PROJECT SUMMARY/ABSTRACT Normal skin scarring and pathological hypertrophic scarring (HS) are common complications of surgery. HS is more prevalent in non-Caucasian patients (up to 47%), may require multiple surgical revisions, and can cause severe functional, aesthetic, and psychological difficulties. HS is a common post- surgical outcome for cleft lip and palate (CLP), the most common craniofacial congenital anomaly present at birth. Our goal is to develop our fibromodulin (FMOD)-based peptide, F06-C40, to promote healing and reduce scar formation in mechanically approximated dermis. Our proposed broad indication in cutaneous scar reduction will address an unmet need in a large population of patients, thus making it very commercially attractive to potential partners and investors. This, in turn will enable us to meet the needs of patients in less commercially viable areas such as CLP which reflects the mission of the National Institute of Craniofacial and Dental Research Institute (NIDCR). This PAR-16-027 Commercialization Readiness Pilot (CRP):SB1 proposal directly continues the Fast- Track SBIR Phase II award R44DE024692, `Anti-scar peptide for cleft repair', and is intended to accelerate the Investigational New Drug (IND) submission for first-in-human F06-C40 trials. Based on FDA guidance, Scarless Laboratories (SL) has designed the phase 1/2 clinical study and plans to submit the IND application and begin phase 1/2 studies before the end of 2017 (year 1). To expedite and derisk critical technical, regulatory/clinical, and business milestone activities that could impact or delay F06-C40 commercialization, we proposed the following CRP:SB1 AIMs: AIM 1 will support CMC technical development and minimize technical risks by ensuring a robust ensuring a robust process development optimization program for F06-C40 DS production and an analytical development and validation plan for F06-C40 DP; AIM 2 will expedite clinical development and minimize regulatory risks by incorporating quantifiable drug development tools to ensure efficient development with optimal clinical study design and develop clinical methods to assess safety, efficacy and related pharmacokinetics in our clinical program to meet FDA requirements; AIM 3 will support the conduct of extended toxicology studies including animal pharmacokinetics to support longer dosing of patients in clinical studies, a greater breadth of indications and populations at risk with unmet needs; and AIM 4 is intended to fully integrate SL's intellectual property, market focus, and business strategies to maximize valuation. If funded, we have also secured commitments for $2.5M non-SBIR Phase III `matching' funds from investors to finish phase 1/2 clinical studies. Our long-term goal is to improve outcomes for all patients with skin wounds due to surgery, burns, or other trauma.

项目摘要/摘要 正常的皮肤疤痕和病理肥厚疤痕（HS）是手术的常见并发症。 HS是 非高加索患者（多达47％）中更普遍，可能需要多次手术修订，并可能导致 严重的功能，美学和心理困难。 HS是唇裂的常见后手术结果 和口感（CLP），这是出生时最常见的颅面先天性异常。我们的目标是发展 我们的纤维瘤蛋白（FMOD）基于F06-C40的肽，以促进愈合并减少疤痕形成 机械近似真皮。我们拟议的皮肤疤痕减少的广泛迹象将解决 大量患者的需求未满足，因此对潜在伴侣的商业吸引力非常有吸引力 和投资者。反过来，这将使我们能够满足不那么可行的地区患者的需求，例如 作为反映国家颅面和牙科研究所的使命的CLP （NIDCR）。 该PAR-16-027商业化准备飞行员（CRP）：SB1提案直接继续快速 Track SBIR II期奖R44DE024692，“抗裂隙肽进行裂口维修”，旨在加速 首先人类F06-C40试验的研究新药（IND）提交。基于FDA指导， 无疤面实验室（SL）设计了1/2阶段的临床研究，并计划提交IND申请 并在2017年底之前开始1/2阶段的研究。为了加快和挑剔的技术， 监管/临床和商业里程碑活动可能会影响或延迟F06-C40商业化，我们 提出以下CRP：SB1 AIMS：AIM 1将支持CMC技术开发并最大程度地降低技术 通过确保强大的F06-C40 DS的强大过程开发优化程序来实现风险 F06-C40 DP的生产以及分析开发和验证计划； AIM 2将加速临床 通过合并可量化的药物开发工具来开发和最大程度地降低监管风险 通过最佳临床研究设计和开发临床方法来评估安全性，功效 以及我们的临床计划中相关的药代动力学，以满足FDA要求； AIM 3将支持 进行扩展的毒理学研究，包括动物药代动力学，以支持更长的患者给药 在临床研究中，面临未满足需求的风险的适应症和人群的广度；目标4是 旨在完全整合SL的知识产权，市场重点和业务策略，以最大化 估值。如果资助，我们还获得了250万美元的非SBIR III阶段“匹配”资金的承诺 投资者完成1/2阶段临床研究。我们的长期目标是改善所有患者 由于手术，烧伤或其他创伤引起的皮肤伤口。