Phase II clinical trial for new IPF drug FG-3019

(dailyRx News) Patients with idiopathic pulmonary fibrosis have not had many treatment options to improve their odds of survival. Results of clinical trials due in 2014 may change that. A review of current treatment and the status of clinical trials for idiopathic pulmonary fibrosis (IPF) was recently published.
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The approval of pirfenidone in Europe has been encouraging and results of three other important clinical trials separately testing the triple therapy of prednisolone, azathioprine and n-acetylcysteine; nintedanib and pirfenidone are due in 2014. Preliminary results with these drugs have shown potential for some of these agents to help slow the progression of IPF and give patients a better quality of life.
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When data from three clinical trials using pirfenidone were grouped together and analyzed, the data showed that treatment with pirfenidone reduced the risk of disease progression by 30 percent compared to placebo. A new clinical trial using pirfenidone, named ASCEND is underway and is due to report results in 2014.

"We believe that the ASCEND trial of pirfenidone for idiopathic pulmonary fibrosis will be successful when the data is top lined Q2 2014," Summer Street's Bart Classen says, in a bullish note on shares of InterMune (ITMN +0.3%) which he says could rise another 20% in Q1.

The researchers showed that removing a gene in the myofibroblasts that makes a specific subset of proteins called alpha v integrins blocks the ability of these cells to trigger activation of TGF beta. Furthermore, they were able to replicate the effect of the gene deletion by treatment with a small molecule compound, thus opening the door to a potential new therapy for patients.

“This is the first foray into targeting not just a single integrin, but rather several integrins that appear to be working in concert to promote fibrosis,” said David Griggs, Ph.D., Director of Biology at Saint Louis University’s Center for World Health and Medicine and an author of the paper.

“We have developed small molecular compounds that selectively inhibit these integrins, which suppress TGF beta protein, and these have been effective in animal models of lung and liver fibrosis.”

The small molecule was not only able to prevent fibrosis; it made fibrosis less severe even when the treatment was started after fibrosis had begun, Griggs added.