Stem cell developer Athersys (NASDAQ:ATHX) has received a U.S patent that covers the use of nonembryonic, multipotent stem cells in the treatment of graft-versus-host disease.
Graft-versus-host disease (GvHD) often occurs after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient’s body. The condition is often associated with treatment for leukemia and other blood diseases.
The patent pertains to Athersys’ MultiStem technology, 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.
“The patent provides the company with protection in this high-value area through 2028 and lays the groundwork for additional protection in related areas of treating immune system dysfunction,” said chief operating officer B.J. Lehmann in a statement.
Earlier this year, Athersys reported positive results for a phase 1 trial investigating MultiStem to treat GvHD. The company plans to interact with federal regulators to determine the next clinical steps for its GvHD treatment, which is expected to be a phase 2/3 study, according to the statement.
In 2010, Athersys was granted orphan drug designation for the treatment of GvHD by the U.S. Food and Drug Administration, which is intended to aid the development of drugs for rare diseases and conditions.
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.
Potential benefits to Athersys of the designation include funding for clinical studies, study-design assistance, tax incentives and seven years of market exclusivity for the product upon regulatory approval.
