Neuregulins are a class of naturally occurring protein growth factors that potentially have multiple effects on the nervous and cardiovascular systems. Acorda is developing its lead recombinant neuregulin candidate, Glial Growth Factor 2 (GGF2), in a number of nervous system and cardiac indications, including heart failure, stroke and peripheral nerve injury. The most advanced of these is a clinical-stage program in heart failure.
Acorda completed a Phase 1 clinical trial of GGF2 in heart failure patients in late 2012. Data from this trial, designed to study safety, tolerability and exploratory measures of efficacy in a single ascending dose of GGF2 in people with heart failure who were already on optimized regimens of currently available cardiovascular therapies, were presented at the American College of Cardiology 62nd Annual Scientific Session in March 2013.
Acorda has discussed the results from the initial GGF2 study with the U.S. Food and Drug Administration (FDA) and has reached agreement on the design of the next clinical study of GGF2 in heart failure, which is expected to begin in 2013.
Heart failure is a chronic, progressive condition in which the heart muscle is unable to pump enough blood to meet the body's need for oxygen. Heart failure is most commonly the result of damage to the heart, caused by coronary artery disease or from added stress to the heart from other health conditions, such as diabetes or high blood pressure.
GGF2 may offer a unique heart failure therapeutic option, as existing medications aim to compensate for the heart's diminished blood pumping ability, but do not directly restore heart muscle function. GGF2 acts at the level of the muscle cells, or cardiomyocytes, and is believed to improve the heart's ability to contract by restoring cardiomyocyte functions that are impaired in heart failure. Preclinical studies have found that GGF2 improves contractile function and may also protect the heart structure from acute and chronic stressors.
Stroke/Peripheral Nerve Damage
Neuregulins have also demonstrated a range of effects in neuroprotection and repair in preclinical models of disease and injury to both the central and peripheral nervous systems.
In preclinical studies, GGF2 has been shown to partially protect the brain from the consequences of stroke and enhance recovery of sensorimotor function with a wide treatment time window of several days. Preclinical studies have also shown improvements in function following damage to peripheral nerves. In a preclinical model that simulates nerve damage that occurs with surgical prostatectomy, GGF2 was shown to protect nerves and restore erectile function.