Catabasis Pharmaceuticals, Inc. announced the publication of preclinical data on CAT-5571, a novel activator of autophagy and potential oral treatment for cystic fibrosis (CF). The preclinical data demonstrate an increase in CF transmembrane conductance regulator (CFTR) activity and trafficking which are detailed in an article titled Fatty Acid Cysteamine Conjugates as Novel and Potent Autophagy Activators that Enhance the Correction of Misfolded F508del-CFTR in the Journal of Medicinal Chemistry. The publication describes the synthesis and biology of the CAT-5000 series of molecules developed using the proprietary Catabasis SMART linker drug discovery platform. The data demonstrate that CAT-5571 is a novel autophagy activator which, in combination with the current standard of care therapy, increased the cell surface expression and function of CFTR in bronchial epithelial cells isolated from multiple CF patients with the F508del mutation. When CAT-5571 was used in combination with lumacaftor and ivacaftor, it significantly enhanced the effects on the cells of the standard of care combination, both in the amount of the more mature C-band form of the CFTR protein with complex glycosylation, and in the amount of the CFTR protein reaching the cell surface. Importantly, CAT-5571 significantly enhanced the lumacaftor/ivacaftor-mediated chloride current increase in cultured primary homozygous F508del human bronchial epithelial cells. Catabasis expects to initiate a Phase 1 clinical trial with CAT-5571 for the potential treatment of CF in Fourth Quarter 2017 or First Quarter 2018. CAT-5571 is a novel molecule comprising cysteamine covalently conjugated to docosahexaenoic acid (DHA) using the company’s SMART linker drug discovery platform to enhance the intracellular activity of the bioactive components. CAT-5571 allows sustained intracellular delivery of the two bioactive components leading to activation of autophagy through two different pathways. Autophagy is a process that maintains cellular homeostasis and host defense mechanisms, and is known to be impaired in CF. Company have found that the level of autophagy activation achieved with CAT-5571 cannot be replicated by administering the bioactive components either individually or in combination, even at much higher concentrations.