Idiopathic Pulmonary Fibrosis and an Attempt at Crossing the Valley of Death

In my prior blog entry, I promised to reveal what keeps me awake at night. Well, several nights ago, I rocketed up from my sleep. "What's the matter this time?" asked my wife. "There's a chance I may have helped some people," I answered.

Idiopathic pulmonary fibrosis (IPF) is a devastating disease characterized by scarring of the lungs, leading to respiratory failure. Despite an improved understanding of IPF's molecular mechanisms, the prognosis is poor, with a median survival of some three years from the onset of symptoms. Other than lung transplantation, there is no known effective treatment.

Estimated to affect five million people worldwide and 128,000 persons in the US, IPF has no known cause. These numbers are small relative to the 200,000 persons diagnosed with lung cancer in the US each year, but this is of little comfort if you contract IPF. Peter Benchley, the author of Jaws, died of IPF in 2006. A member of my family also died from the disease.

Several years ago, I read about a tiny Israeli biotech company named Compugen, which models disease processes at the molecular level and aims to predict drug candidates in silico, i.e. by computer. Although larger entities have failed at what Compugen is attempting, Compugen has persisted in its lonely efforts and believes it is well along the road to success.

In February 2007, Compugen, created a platform for the predictive discovery of novel G-protein coupled receptors ("GPCRs"), and in the initial run of this platform, eight novel GPCR ligands were found. Given that some 40% of all drugs modulate GPCRs and the relative high probability of newly discovered GPCR peptide ligands ultimately becoming drugs, Compugen's announcement elicited significant recognition from the scientific community.

In March 2009, Compugen announced that one of these newly discovered ligands, CGEN-25009, was able to activate the Relaxin receptor, and studies conducted by Professor Daniele Bani, an expert in the field of relaxin and fibrotic diseases from the University of Florence, demonstrated that administration of CGEN-25009 led to robust reduction of the fibrotic tissue in the lungs of mice induced with IPF.

Now you might think that such news would cause Big Pharma to come running. Not so. Pulmonary fibrosis claims fewer victims than other diseases. Also, notwithstanding the fact that the pipelines of several Big Pharma companies have gone dry, the focus of some of these companies remains short-term.

In brief, CGEN-25009 needed to be escorted across the "valley of death," the period when promising drug candidates are at too early a stage to attract capital for continued development from Big Pharma.

In 2009, I wanted to do something that mattered. Specifically, I wanted to move CGEN-25009 ahead. I called Compugen, and was fortunate enough to be hired as an external consultant.

Next, a cold call to the Pulmonary Fibrosis Foundation in Chicago, which was sufficiently intrigued to consult, inter alia, with the University of Pittsburgh's Simmons Center for Interstitial Lung Diseases.

Last week it was announced in a press release that the Pulmonary Fibrosis Foundation will provide a grant to the Simmons Center to further establish the anti-fibrotic properties of CGEN-25009 in multiple animal models of fibrosis, to elucidate the mechanism of CGEN-25009's anti-fibrotic effects, and to prioritize potential biomarkers for the study of CGEN-25009 in patients with IPF (see: http://www.cgen.com/Content.aspx?Page=press_releases&NewsId=569).

It didn't happen overnight, and my persistent pestering intended to advance this project post-haste nearly rendered me persona non grata. In addition, there can be no assurances that CGEN-25009 will ever become a marketed drug; however, there is certainly hope, and on a personal level, I am excited by this new model for cooperation between academia and industry.

[I am a Compugen shareholder, and in 2009 I began work as an external consultant to Compugen. The opinions expressed herein are mine and are based on publicly available information. This blog entry has not been authorized or approved by Compugen, the Pulmonary Fibrosis Foundation or the University of Pittsburgh, and this is not a recommendation to buy or sell Compugen shares.]