Idiopathic pulmonary fibrosis (IPF) is a progressive interstitial lung disease with a median survivalof only 3 - 5 years from diagnosis. Although two FDA-approved drugs, pirfenidone and nintedanib,may slow the rate of decline of lung function in some IPF patients, neither drug significantly altersthe course of this lethal disease. There is a great need for new drugs with greater efficacy andless toxicity for the treatment of patients with IPF. This STTR application addresses the unmetneed for new therapeutic approaches to IPF that would be more effective and less toxic thancurrent treatments.Small peptides are widely involved in multiple cellular events and play very important roles invarious cell functions. Interest in peptides as potential drug candidates remains high. Withadvances in such fields as chemical synthesis and peptide formulation, peptide drugs - especiallyshort synthetic and long-acting peptides - are quickly increasing in the global market. Theadvantages of small peptides as drugs include their high biological activity, high specificity, andlow toxicity.FibroBiologics, LLC proposes to develop the novel peptide M10 as an efficacious antifibrotictherapeutic agent, with a lead indication for the treatment of patients who suffer from IPF. InSpecific Aim 1, we will determine antifibrotic activity of M10 in primary lung fibroblasts isolatedfrom IPF patients and evaluate an inhibitory effect of M10 on fibrogenic characteristics of IPF lungfibroblasts. In Specific Aim 2, we will define the efficacious dosing of M10 in two different animalmodels of pulmonary fibrosis: bleomycin-induced therapeutic mouse model and FSP-drivenTβR1CA mouse model. The successful completion of these two specific aims will provideimportant information about the feasibility of developing M10 as a novel IPF therapeutic and willjustify further studies focusing on gaining FDA clearance, scaling production, and a human clinicaltrial.
Public Health Relevance Statement: Project Narrative
In this Phase I STTR, FibroBiologics, LLC will determine antifibrotic activity of a novel antifibrotic
peptide M10 in primary lung fibroblasts isolated from IPF patients and prove its feasibility in two
different animal models of pulmonary fibrosis: bleomycin-induced and FSP-driven TβR1CA
mouse models of pulmonary fibrosis. The impact of the proposed work on public health will be
significant, as it will contribute to the development of an effective strategy for the treatment
of IPF. Moreover, the M10-based therapeutics developed in our study might be effective in
other pathological conditions and diseases characterized by excessive scarring and fibrosis.
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