One of the big dreams in the pharmaceutical business is to develop a once-daily oral pill that can relieve symptoms of autoimmune diseases. These are the kind of energy-sapping, pain-inflicting, or disfiguring illnesses people get when the body’s immune system goes awry and attacks healthy tissue like it’s a foreign virus.
No one has come close to doing this with a safe and effective pill, but one little-known biotech company plugging away for years in an office park in Mountain View, CA, ChemoCentryx, has recently produced some eye-opening results. If ChemoCentryx can nail its next big trial, it could be a couple years away from delivering a potent new option for patients with Crohn’s disease, an autoimmune disease of the intestines.
“This is the most exciting phase in our development since I started the company 13 years ago,” says ChemoCentryx CEO Tom Schall.
ChemoCentryx has kept a low profile since its founding 1997. The company has scooped up more than $330 million in financing during those years from partnerships, government grants, and venture capitalists and private equity investors. The vision—backed by HBM BioVentures, Alta Partners, OrbiMed Advisors, Jennison Associates, and GlaxoSmithKline among others—is to create small-molecule oral pills that interact with novel protein targets called chemokines and chemokine receptors. By limiting the activity of the chemokine system, the idea is that ChemoCentryx can disrupt a vital process that leads to autoimmunity, without shutting down essential immune defense functions that protect people from infections.
The prize at stake here is among the biggest in the pharmaceutical business. Estimates are that between 14 and 24 million people in the U.S. have autoimmune diseases. Fortunes have been made for Amgen, Johnson & Johnson, and Abbott Laboratories on the back of one-hit wonder drugs for such conditions, including rheumatoid arthritis and psoriasis. Total sales of rheumatoid arthritis drugs alone are estimated to grow from $7 billion in 2007 to $17 billion by 2017, according to Datamonitor. And there are an estimated 80 different autoimmune diseases to tackle, although not all are going to be that lucrative.
Plenty of people argued in the late ’90s and early 2000s that the original biotech drugs for autoimmune diseases would flop because patients wouldn’t want to take injectables for a chronic condition. Plenty of companies have tried, unsuccessfully, to create conventional small-molecule drugs that can be made into more convenient oral pills for autoimmunity, figuring that whoever does it, will take over the market.
I’ve covered a few startups in other parts of the country going after such treatments, notably Plymouth, MI-based Lycera and San Diego-based CalciMedica. Both of those companies, however, are at much earlier stages, on the cusp of entering clinical trials.
ChemoCentryx is going about its plan a little differently, essentially to try to carve out a niche as “chemokines-R-us.” Schall, a Stanford-trained cancer biologist with experience at Genentech and Schering-Plough, told me all about this with the kind of enthusiasm I’d expect from a graduate student or postdoc. “I’ve been living and breathing this stuff essentially my entire adult life,” he says.
“This stuff” is the science of chemokines—signaling molecules that control how immune cells move in and out of inflamed tissues—and the receptors on immune system cells that chemokines bind to. The key to ChemoCentryx’s approach, as in many fields of drug development, is to tap deep understanding of these targets in order to craft drugs that act on them specifically, while causing minimal collateral damage in the form of side effects.
Pretty much all of the Big Pharma companies, to varying degrees, have tried or are trying to make drugs like this against chemokine-system targets. Pfizer has
