Neuregulins: GGF2In 2002, Acorda acquired an exclusive worldwide license from CeNeS Pharmaceuticals (now part of Paion AG) to its neuregulins intellectual property and technology related to neuregulins - a class of naturally occurring protein growth factors that have multiple effects on the nervous and cardiovascular systems, and potential therapeutic applications in both.
The most clinically advanced of these agents, Glial Growth Factor 2 (GGF2), is a member of the neuregulin family of growth factors related to epidermal growth factor. The neuregulins bind to erbB receptors, which translate the growth factor signal to the cell and cause changes in cell growth, protein production and gene expression.
These changes have been shown to enhance neuroprotection and repair of nerve tissues, which have led to promising results in a range of preclinical models of disease and injury to both the CNS and peripheral nervous system (PNS):
Published studies have shown that GGF2 can stimulate remyelination in preclinical models of MS.
Acorda and its collaborators have demonstrated that neuregulins can protect cells of the substantia nigra in the brain and preserve motor function in preclinical models of Parkinson's disease.
Neuregulins also have been found to protect the brain and enhance recovery following both transient and permanent ischemia models of stroke.
In addition, neuregulins protect central and peripheral neurons from chemically induced death and dysfunction, such as following exposure to common chemotherapeutics.
Neuregulins also have been shown to protect heart muscle cells (cardiomyocytes) from a wide range of injuries both in vitro and in vivo. Neuregulins have shown the ability to restore cardiac function in preclinical models of heart failure caused by myocardial infarction, rapid pacing, and viral and chemically induced cardiomyopathies.
We plan to initiate a Phase 1 single-dose clinical trial of the neuregulin GGF2 in patients with heart failure based on an IND filed with the U.S. Food and Drug Administration (FDA) on March 19, 2010. We are beginning the clinical program with a cardiac indication because we believe that "proof of concept" in humans may be studied more efficiently in this indication. However, we are also continuing preclinical studies of potential neurology indications for GGF2 and other neuregulin growth factors.