Anti-scar peptide for cleft lip repair

用于唇裂修复的抗疤痕肽

基本信息

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
项目类别：
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

项目摘要

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.

项目概要/摘要正常皮肤疤痕和病理性肥厚性疤痕（HS）是手术常见的并发症。 HS是在非白种人患者中更为普遍（高达 47%），可能需要多次手术修复，并可能导致严重的功能、审美和心理困难。 HS 是唇裂术后常见的结果和腭（CLP），出生时最常见的颅面先天性异常。我们的目标是发展我们基于纤维调节蛋白 (FMOD) 的肽 F06-C40，可促进愈合并减少疤痕形成机械逼近真皮。我们提出的皮肤疤痕减少的广泛适应症将解决大量患者的需求未得到满足，因此对潜在合作伙伴极具商业吸引力和投资者。这反过来将使我们能够满足商业可行性较差地区的患者需求，例如作为 CLP，体现了国家颅面和牙科研究所的使命 (NIDCR)。此 PAR-16-027 商业化准备试点 (CRP):SB1 提案直接延续了 Fast- 跟踪 SBIR 二期奖项 R44DE024692，“用于唇裂修复的抗疤痕肽”，旨在加速首次人体 F06-C40 试验的研究性新药 (IND) 提交。根据 FDA 指导， Scarless Laboratories (SL)已设计1/2期临床研究并计划提交IND申请并在 2017 年底（第一年）之前开始 1/2 期研究。为了加快关键技术并消除风险，可能影响或延迟 F06-C40 商业化的监管/临床和业务里程碑活动，我们提出以下 CRP:SB1 AIM：AIM 1 将支持 CMC 技术开发并最大限度地减少技术通过确保稳健的风险确保稳健的 F06-C40 DS 工艺开发优化计划 F06-C40 DP 的生产以及分析开发和验证计划； AIM 2将加速临床通过结合可量化的药物开发工具来确保开发并最大程度地降低监管风险通过最佳临床研究设计进行高效开发并开发评估安全性、有效性的临床方法以及我们临床计划中的相关药代动力学，以满足 FDA 的要求； AIM 3 将支持进行广泛的毒理学研究，包括动物药代动力学，以支持患者更长的给药时间在临床研究中，更广泛的适应症和面临需求未满足风险的人群；目标 4 是旨在充分整合 SL 的知识产权、市场焦点和业务战略，以最大化估价。如果获得资助，我们还获得了来自以下机构的 250 万美元非 SBIR 第三阶段“匹配”资金的承诺：投资者完成1/2期临床研究。我们的长期目标是改善所有患有此病的患者的治疗结果由于手术、烧伤或其他外伤造成的皮肤伤口。