Targeting kidney fibrosis by a novel myofibroblast specific small molecule inhibitor

Introduction

Chronic kidney disease (CKD) is a progressive disease that is prevalent in 10% of the developed world and develops as a result of diabetes mellitus, hypertension, glomerulonephritis, or acute kidney injury. Over time, CKD can progress to end-stage renal disease (ESRD); a condition that can only be treated through hemodialysis or kidney transplantation, posing a severe medical, societal, and economic challenge.

Pathological Mechanism

Fibrosis is recognized as a key pathological mechanism in CKD, responsible for slowly destroying the tissue architecture of the kidney. Current therapies for CKD alleviate symptoms of the disease by controlling hypertension and hyperglycemia. However, while fibrosis is a promising therapeutic target, no antifibrotic therapy for the kidney has reached the clinic. Hence, there is an urgent need for disease-modifying and curative treatments that can halt fibrosis.

Project Overview

In FibroTarg, we will assess the technical and commercial feasibility of Fibrolisine, a first-of-its-kind anti-fibrotic drug candidate, for the treatment of CKD. Our preliminary data has confirmed a unique expression profile of our therapeutic target and reduced fibrosis onset in in vivo knockout models without side effects after kidney injury.

Expected Outcomes

Hence, Fibrolisine is expected to target the process of fibrogenesis and halt the progression to ESRD in early-stage CKD patients. Through the activities in the FibroTarg project, we aim to:

1. Demonstrate the antifibrotic potential of Fibrolisine in advanced human kidney fibrosis organoid models.
2. Assess its efficacy in in vivo models of kidney injury.
3. Investigate the commercial potential of Fibrolisine by analyzing the commercial landscape and developing an initial commercialization strategy.