Gil Van Bokkelen, CEO of stem cell company Athersys (NASDAQ:ATHX), is typically one to speak in cautious and measured tones, but he made a brash statement in the company’s most recent quarterly earnings call.
Van Bokkelen said Cleveland-based Athersys’ MultiStem stem cell therapy for ischemic stroke “could easily be one of the biggest blockbusters that the industry has ever seen.”
He also shared some eye-popping numbers: “Even if you make some pretty modest assumptions about what the therapy would be priced at, then that leads you pretty quickly to a picture or a scenario where you could see that this market opportunity could be $15 billion to $20 billion a year easily.”
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So were Van Bokkelen’s bold pronouncements a legitimate assessment of MultiStem’s outlook, or just empty puffery from a small biotech on a shoe-string budget badly in need of corporate partners?
Steve Brozak, president of New Jersey-based research firm WBB Securities, leans toward the former.
“I would say that he is quite right,” Brozak said. “This type of basic building-block technology is so powerful that Gil wasn’t using hyperbole when he said that.”
Athersys’ MultiStem technology is an off-the-shelf stem cell treatment derived from the bone marrow of adults or other nonembryonic sources. The technology has shown promise in reducing inflammation, protecting damaged tissue and forming new blood vessels for several indications, including stroke.
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Brozak praised the science behind MultiStem as “very powerful,” and noted that the technology “has the ability to reset the immune system.” (Brozak said WBB doesn’t own any Athersys shares, but has acted as a financial adviser to the company.)
Late last year, Athersys began enrollment in a phase 2 stroke trial designed to enroll about 140 patients. That means that even with the most optimistic projections, Athersys’ stroke therapy is unlikely to reach the market until 2015 or so.
Regardless of what you think of Van Bokkelen’s bold statements, he made a pretty compelling case for MultiStem’s potential benefit to stroke patients.
Each year about 2 million people in the U.S., Europe and Japan suffer an ischemic stroke, according to Van Bokkelen. The problem is that only about 10 percent of ischemic stroke victims are treated with the best medication for them, a clot-busting drug called tPA. That’s because tPA must be administered within three to four hours of a stroke to be effective, and most patients either don’t get to the doctor in time or aren’t diagnosed in time.
“The risk is that you really can’t administer tPA to those patients after that three- to four-hour window because you could cause bleeding into the brain, which could actually make things worse, or it could even result in the death of the patient,” Van Bokkelen said.
MultiStem’s big advantage is that its “window of intervention” is much wider that that of tPA, Van Bokkelen said. In the phase 2 trial, MultiStem is being administered intravenously one to two days after the stroke has occurred. Preclinical studies have suggested that MultiStem can be administered up to a week after stroke and still be effective, Van Bokkelen said.
“I would characterize the prime market opportunity or clinical opportunity, if you will, as being the segment of patients that have the most significant deficits following a stroke,” Van Bokkelen said. “So, if you just say, look, we’re only really focused here on trying to treat the bottom 50 percent, if you will, of patients that have suffered a stroke, you’re still talking about a million patients a year.”
It’s the promise of MultiStem’s technology, plus the size of the ischemic stroke market that has Van Bokkelen thumping his chest about the technology’s potential — and has a veteran analyst like Brozak buying what Van Bokkelen is saying.
