Christopher Reinhard will tell you there is nothing unusual about the 20-plus years he’s spent getting an experimental heart therapy to late-stage clinical trials.
“Very rarely will you get a short story on development of a drug,” said Reinhard (above), who is a principal investor and the CEO of San Diego-based Angionetics. Two decades “is about what you would expect” for a new drug-making method, Reinhard said.
That may be true, but it doesn’t begin to convey the tortuous path that Reinhard has followed to get Angionetics where it is today. The biotech is starting a phase 3 trial in the next few months that seeks to enroll some 320 patients with myocardial ischemia—when clogged coronary arteries reduce the flow of oxygen-rich blood to the heart.
To treat the disease, Angionetics isn’t testing some new type of cholesterol-lowering drug, or a stent to help open clogged arteries.
Rather, it’s attempting a risky and less-proven method—gene therapy, in which new genetic instructions are transported into the body to help produce a specific protein. Gene therapies have been in development for decades, but are only now starting to come of age thanks to a variety of technological advances. Two therapies are approved in Europe, from UniQure (NASDAQ: QURE) and GlaxoSmithKline, both for ultra rare immune and metabolic diseases. Spark Therapeutics (NASDAQ: ONCE) this year is expected to file the first ever U.S. approval application for a gene therapy, a treatment for a form of childhood blindness.
Angionetics’ gene therapy, Ad5FGF-4 (Generx), is intended to stimulate the growth of new blood vessels in the heart. A catheter inserted through the groin delivers the genes—carried within modified viruses—into heart cells, where they are supposed to produce a protein, fibroblast growth factor-4, that helps grow new blood vessels.
The hope is to ease chest pain and relieve the effects of clogged coronary arteries by stimulating the growth of new blood vessels in areas in the heart where there is insufficient blood flow. “We’re just taking the heart and trying to enhance its ability to grow more blood vessels,” Reinhard said.
While gene therapies are more advanced than ever, and several experimental treatments aimed at heart disease and heart failure are being tested, none have yet succeeded. A heart failure gene therapy from San Diego’s Celladon, for instance, failed in 2015.
Still, the potential prize is substantial. Of the estimated 16.5 million Americans with coronary heart disease, Angionetics’ Reinhard said about half experience heart-related chest pain.
The current standard of care offers two principal methods of treatment. The first course of therapy is usually to prescribe drugs like nitrates that temporarily dilates blood vessels to … Next Page »