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.

项目总结/摘要 正常皮肤瘢痕和病理性增生性瘢痕是外科手术常见的并发症。HS是 在非白人患者中更常见（高达47%），可能需要多次手术翻修，并可能导致 严重的功能、美学和心理困难。唇裂术后HS是一种常见的并发症 和腭（CLP），最常见的颅面先天性异常，目前在出生时。我们的目标是开发 我们的纤维调节蛋白（FMOD）为基础的肽，F06-C40，以促进愈合和减少疤痕形成， 机械接近的真皮。我们提出的皮肤瘢痕减少的广泛适应症将解决 大量患者的未满足需求，因此对潜在合作伙伴极具商业吸引力 和投资者这反过来又使我们能够满足那些在商业上不太可行的地区的病人的需要， 作为CLP，它反映了国家颅面和牙科研究所的使命 （NIDCR）。 本PAR-16-027商业化准备试点（CRP）：SB 1提案直接延续了快速- 跟踪SBIR第二阶段奖R44 DE 024692，“用于裂缝修复的抗疤痕肽”，旨在加速 首次人体F06-C40试验的研究性新药（IND）提交。根据FDA指南， 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1年）前开始1/2期研究。为了加快和降低关键技术的风险， 可能影响或延迟F06-C40商业化的监管/临床和商业里程碑活动，我们 提出了以下CRP：SB 1 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F06-C40 DS的稳健工艺开发优化计划， F06-C40 DP的生产和分析开发及验证计划; AIM 2将加快临床 通过整合可量化的药物开发工具， 通过最佳临床研究设计进行有效开发，并开发临床方法以评估安全性、有效性和安全性， 和相关的药代动力学，以满足FDA的要求; AIM 3将支持 进行扩展毒理学研究，包括动物药代动力学，以支持患者更长时间给药 在临床研究中，适应症和未满足需求的风险人群的范围更广; AIM 4是 旨在充分整合SL的知识产权、市场重点和业务战略， 估值如果获得资助，我们还获得了来自以下方面的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者的结局， 由于手术、烧伤或其他创伤造成的皮肤创伤。