Thanks in part to a SBIR Fast Track grant worth up to $2.8 million, stem cell company Athersys (Nasdaq: ATHX) is gearing up to advance its third MultiStem program into Phase 2 clinical trials.
Cleveland-based Athersys develops an off-the-shelf biologic product made from adult stem cells that it’s applying to disease indications in inflammation, neurology and cardiovascular disease.
With the grant from the National Heart, Lung and Blood Institute, the company will initiate a study of MultiStem in patients who have suffered a heart attack, or acute myocardial infarction, early next year.
Already the company has two Phase 2 trials in progress, although one has been held up by enrollment challenges. Athersys is working with Pfizer on a trial of MultiStem in treatment-resistant ulcerative colitis patients. Last time we checked in with the company, enrollment for the study had been pushed back from late 2012 to early summer 2013. Now Chairman and CEO Gil Van Bokkelen said Pfizer reports that enrollment should wrap up in October or November, with data expected in early 2014.
Van Bokkelin attributed these delays to the timing of the study and the recruiting landscape. “There are a number of different, more traditional pharmaceutical or even biologic approaches that are being developed that we’re competing with for enrollment,” he said.
A planned decrease in revenues from that collaboration led to a sizable drop in the company’s overall Q2 revenue, which was down to $.6 million from $2.7 million in the same period last year – and a net loss of $5.9 million over the quarter. The company is still stocked with $18.9 million in cash.
One analyst asked Van Bokkelen for his reaction to a big news item in regenerative medicine this week — Osiris Therapeutics’ (NASDAQ:OSIR) stock more than doubling after the company announced what Van Bokkelen called pretty compelling data” on a wound-healing compound.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
“I am thrilled for Osiris […] I think it’s a step forward for them and a step forward for the field,” Van Bokkelen said, adding that the more examples of regenerative medicines having an impact, the better it’s going to be for companies that are well-positioned to be leaders in the field.
“I also feel very confident […] about the strength of our approach and our product platform, and from a competitive position we think we have a clear path to establishing best-in-class product opportunities in multiple different areas,” he said, proceeding to imply that the company has done early studies in other disease areas like wound healing that it hasn’t yet talked about.
In addition to its regenerative medicine program, Athersys is also working on a new class of compounds that target the 5HT2c receptor in the brain. It’s been on the prowl for a partner to work with on developing these drugs for treatment of obesity and schizophrenia.
“We are doing this very methodically and working diligently to make sure we are picking the right potential partners and constructing our alliances in the most effective way,” Van Bokkelin said. He admitted that ideally the partnership would already be secured by now, but said the company is more committed to doing it right than doing it fast.
Athersys stock has dipped 24 percent since April.
