Anti-scar peptide for cleft lip repair

项目摘要/摘要 正常皮肤瘢痕和病理性增生性瘢痕(HS)是手术常见的并发症。HS IS 在非高加索患者中更为常见(高达47%)，可能需要多次手术矫正，并可能导致 严重的功能、审美和心理困难。HS是唇裂手术后常见的结局 和腭部(CLP)，这是出生时最常见的头面部先天性异常。我们的目标是发展 我们基于纤维调素(FMOD)的多肽F06-C40，可促进伤口愈合并减少瘢痕形成 机械地接近真皮。我们建议的皮肤疤痕减少的广泛适应症将解决 在大量患者中未得到满足的需求，从而使其对潜在合作伙伴具有商业吸引力 和投资者。这反过来将使我们能够满足商业上不太可行的领域的患者的需求，例如 作为中电，体现了国家颅面牙科研究所的使命 (NIDCR)。 此PAR-16-027商业化准备试点(CRP)：SB1提案直接延续快速- Track SBIR第二阶段奖R44DE024692，用于唇裂修复的抗疤痕多肽，旨在加速 研究新药(IND)提交人类首个F06-C40试验。根据FDA的指导， 无疤痕实验室(SL)已经设计了1/2期临床研究，并计划提交IND申请 并在2017年底(第一年)之前开始第1/2阶段研究。为了加快和降低关键技术的风险， 可能影响或推迟F06-C40商业化的监管/临床和业务里程碑活动，我们 提出了以下合作伙伴关系：SB1目标：AIM 1将支持CMC技术开发并将技术需求降至最低 通过确保F06-C40 DS的稳健确保稳健的流程开发优化计划来实现风险 F06-C40 DP的生产和分析开发和验证计划；AIM 2将加快临床 通过纳入可量化的药物开发工具来确保开发和最大限度地减少监管风险 通过最优的临床研究设计和开发临床方法来评估安全性、有效性的高效开发 和相关的药代动力学，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，以支持患者更长剂量的服药 在临床研究中，更广泛的适应症和面临未得到满足需求的风险人群；目标4是 旨在充分整合SL的知识产权、市场重点和业务战略，以最大限度地 估值。如果获得资金，我们还从以下方面获得了250万美元的非SBIR第三阶段匹配资金的承诺 投资者将完成1/2期临床研究。我们的长期目标是改善所有患有 因手术、烧伤或其他创伤而造成的皮肤创伤。

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.