Anti-scar peptide for cleft lip repair

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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.