Stem cell therapy developer Athersys (NASDAQ:ATHX) plans to begin the first clinical study of its MultiStem technology in stroke patients by the end of the year.
The planned double-blind, placebo-controlled study could involve as many as 150 patients, CEO Gil Van Bokkelen said in a conference call with analysts and investors after announcing the Cleveland-based company’s first-quarter financial results.
Finally beginning a clinical study of stroke patients is significant for Athersys because stroke is likely the most promising application of MultiStem, an off-the-shelf stem cell treatment derived from the bone marrow of adults or other nonembryonic sources.
Stroke holds so much potential for Athersys simply due to the size of the market — more than 700,000 patients suffer from ischemic stroke each year in the United States.
MultiStem has shown promise in studies of animals with surgery-induced strokes. The stem cell-based technology is capable of reducing inflammation, protecting damaged tissue and forming new blood vessels.
Athersys also plans to begin a phase 2 study of heart attack patients later in the year. The company recently completed an “end-of-phase 1” meeting with the FDA regarding its clinical studies involving heart attack patients, Van Bokkelen said. Athersys is collaborating with Angiotech Pharmaceuticals on the heart attack application of MultiStem.
Athersys’ first quarter revenue grew 76 percent to $3 million, boosted by milestone and license payments from development partners such as RTI Biologics, which Athersys is working with to develop orthopedic implants based on MultiStem.
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.
However, the company’s quarterly loss widened to $3.9 million from $2.6 million in the year-ago quarter. That was driven in part by an increase in research and development expenses.
At the end of the quarter, Athersys’ cash and cash equivalents stood at $25.3 million. That number was boosted by a direct offering earlier in the quarter that netted the company nearly $12 million.
