Published: Sept. 3, 2002

A spin-off company formed by several University of Colorado faculty to research and treat heart failure has been selected as one of the top 100 companies that will shape the world's future by the Red Herring Magazine.

Myogen Inc. was founded in 1996 by an inter-campus group that also launched the CU Cardiovascular Institute. Founding scientists of Myogen include Professor Leslie Leinwand, chair of CU-Boulder's department of molecular, cellular and developmental biology and Dr. Michael Bristow, head of the division of cardiology at the CU Health Sciences Center in Denver, who both co-direct the CU Cardiovascular Institute.

Other founding scientists include HSC Professor Benjamin Perryman and Professor Eric Olson of the University of Texas Southwestern Medical Center in Dallas.

Myogen is a privately held biopharmaceutical company dedicated to discovering, developing and commercializing drugs for the treatment of heart failure and related cardiovascular diseases. "We know there is a huge market for heart-failure drugs because most companies are wary of taking on the risk of research and development costs that can run into hundreds of millions of dollars and take years," Leinwand said.

Dr. Bristow, who developed Myogen's primary strategy of in-licensing existing drugs and the drug development of "proprietary targets," said the Myogen-CU connection is a valuable one.Ìý "The university gains a direct route of commercializing its intellectual properties and also receives research dollars, while the company gains access to discovery at various stages and can greatly extend its research and development reach."

Spawned at CU's Fitzsimmons facility in Aurora, Myogen moved to Westminster in 1998. At that time, J. William Freytag, an experienced executive manager in healthcare industry research, development and planning was hired as Myogen's president and CEO.

"University spin-off companies are generally a positive venture, and I think ours is a win-win situation for both the University of Colorado and Myogen," said Dr. Bristow, also Chief Scientific and Medical Officer and director of Myogen.Ìý

Myogen's strategy of licensing products developed by other companies and conducting additional research and development on new drugs caught the eye of private investors early, said Leinwand. She noted that many of Myogen's 47 employees have extensive industry experience, and Myogen has sponsored several cardiovascular research projects at the two CU campuses.

"Our original investors perceive this to be a low-risk, high-return venture," said Dr. Bristow. "We are not a one-trick pony. We have two drugs in late-stage clinical trials now being held around the world, another in early clinical trials that appears to be more effective than a drug recently approved by the Federal Drug Administration, and multiple early-phase pharmaceuticals that show promise.

"We essentially have four shots on goal rather than one, and any one of these could be a blockbuster," he said. "Typical biotech companies have just one shot."

Perryman, director of molecular cardiology at HSC, said Myogen is an unusual company. "We are not like a lot of start-ups," he said.Ìý

"We raised $90 million in venture capital from an all-star group of investors, and we already have income from a drug we are marketing in Europe," said Perryman. "Spin-off companies play an important role in industry, especially companies like Myogen that are far enough along to have minimized their financial risk."

Myogen currently is marketing a drug called Perfan I.V., an intravenous form of enoximone, in Europe. An oral form of enoximone -- an agent that increases the force of contraction of the heart in heart-failure sufferers by improving heart function -- is currently in pivotal Phase III clinical trials in North America and Europe.

Another drug known as ambrisentan -- part of a new class of compounds called endothelin receptor antagonists that show promise in treating conditions like heart failure, chronic renal failure and pulmonary arterial hypertension -- is in Phase II clinical trials by Myogen. The company also is engaged in several promising research programs targeting the discovery and development of drugs that could halt or reverse the progression of heart failure.

Leinwand and her CU-Boulder research team have been studying transgenic mice that carry gene mutations for a heart disease shown to be the leading cause of sudden death in young athletes. Known as hypertrophic cardiomyopathy, it is a genetic disease marked by a thickening of the heart muscle that can obstruct blood flow in heart ventricles, lessening their ability to pump blood into arteries.

"Most of our capital goes into purchasing drugs and running clinical trials, which can be extremely expensive," said Leinwand. "Companies often provide the technology and equipment, while universities can provide research innovation."

Founded in 1993, Red Herring magazine was designed for wealthy, technology-savvy senior executives who use technology to drive company efficiency, according to the Red Herring Corp. More than 20 percent of the 33,000 subscribers are millionaires.

The magazine, in its annual "Red Herring 100" report published in June 2002 and subtitled "The Companies That Will Shape the Future," selected the top 50 private and top 50 public companies whose products, services and business models define innovation. Of the 50 private companies, six biotechnology firms, including Myogen, were selected.

In the article, the Red Herring authors wrote of Myogen: "It's rare to find an upstart with three drugs in late-stage clinical trials for unserved areas of heart disease. Or to find one that has a management team with 20 years of collaborative history. Heart disease is the top killer in the United States, yet the market is still wide open. We like this firm's chances."

So does Leinwand, who said the cost of treating chronic heart failure in the United States exceeds $30 billion annually.Ìý

"Heart failure is a long-term result of cardiac injury that can last years and result in poor quality of life involving extensive hospitalization," she said. "The market for effective drugs to treat heart failure is huge, so if a company develops and markets the right drug, the payoff would be phenomenal in terms of finances and improved quality of life."