Regado Biosciences Kicks Off Big Clinical Trial For Heart Drug

9/17/13Follow @benthefidler

Regado Biosciences (NASDAQ: RGDO) will now have the chance to prove that the two-pronged anticoagulant it pitched at its IPO roadshow is all it’s cracked up to be.

The Basking Ridge, NJ-based biotech has officially enrolled the first patient in a massive late-stage clinical trial for its lead drug, REG-1. Regado expects to enroll a total of 13,200 patients at 500 sites around the world and complete the trial in 2015. The study has huge implications for Regado’s future: the company is pouring as much as $150 million into the effort and doing so without the help of a deep-pocketed pharmaceutical partner. But Regado hopes that the study will show that REG-1 is better at preventing strokes and other so-called ischemic events in patients undergoing an angioplasty than The Medicines Co.’s bivalirudin (Angiomax), and in doing so, prove the gamble was worth the risk.

REG-1 is a two-drug system that includes an anticoagulant called pegnivacogin and anivamersen, an RNA aptamer—a string of RNA letters—that binds to pegnivacogin and blocks its activity. The idea is that by carefully timing injections of each drug a doctor can dial the clot-busting effect up or down in real time during a coronary procedure like an angioplasty. This fine control, Regado says, should make those procedures safer than they otherwise would be. Too much anticoagulation can lead to dangerous bleeding, and none of the other injectable anticoagulants used in such procedures—such as bivalirudin and heparin—have a control agent that can modify their effects.

Regado has to prove this on a large scale, of course, and that’s what the late-stage trial is all about. Patients undergoing an angioplasty will randomly get either REG-1, or bivalirudin. Regado hopes to show that REG-1 leads to fewer ischemic events—strokes, non-fatal myocardial infarctions, and deaths, for example—than bivalirudin, and is just as safe to use.

Investors were hesitant to buy into the plan during Regado’s IPO. The company initially set a range of between $14 and $16 per share, but ultimately priced the offering at $4 per share—raising just $47 million and marking a departure from the recent run of big biotech IPO successes. Its stock closed at $5 per share on the Nasdaq on its first day of trading, and has hovered around the $4 to $5 per share mark ever since.

Even so, investor sentiment shifted a bit yesterday as sell-side analysts initiated coverage of Regado. Canaccord Genuity’s Ritu Baral, for example, set a $14-per-share target for Regado’s stock, predicted the trial will be successful, and estimated peak worldwide sales of REG-1 will be about $2.5 billion per year.

“[REG-1] could improve outcomes across a large number of CV procedures,” Baral wrote in a note to investors.

Regado’s shares jumped about 40 percent and closed at $7.63 apiece on Monday. Now it has to show that that optimism is founded. Regado plans to report its first interim analysis of the trial in the second quarter of 2014, when it expects to have enrolled about 1,000 patients.

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com Follow @benthefidler

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