Acorda Therapeutics, Inc. (Nasdaq: ACOR) today announced that the first patient has been enrolled in the second clinical trial of Glial Growth Factor 2 (GGF2). This Phase 1b single-infusion trial in people with heart failure will assess tolerability of three dose levels of GGF2, and also includes several explorative measures of efficacy.
“The first clinical trial with GGF2 in people with heart failure provided initial data on tolerability and activity of a single infusion over a wide range of doses. That study also supported observations in preclinical models that GGF2 improved heart function as measured by ejection fraction,” said Anthony Caggiano, M.D. Ph.D., Vice President of Research and Development at Acorda. “This second trial will employ a narrower range of single infusion doses to assess safety, various measures of efficacy, including changes in ejection fraction and cardiovascular endurance, and whether there is evidence of interaction with other medications commonly used in heart failure.”
Additional details on the study design and site locations can be found at: http://clinicaltrials.gov/ct2/show/NCT01944683?term=ggf2&rank=1.
Results of the first Phase 1 GGF2 clinical trial were presented earlier this year at the American College of Cardiology 62 nd Annual Scientific Session. The trial design included seven cohorts of six participants each, four of whom received GGF2 and two of whom received placebo at each dose. In the study, a single infusion of GGF2 in patients with heart failure was generally well tolerated up to dose levels of 0.75 mg/kg. Among trial participants who received GGF2, the most commonly observed adverse events were headache, site injection reaction and gastrointestinal symptoms. A dose limiting toxicity (DLT) was identified in a single subject receiving the highest planned dose level of 1.5 mg/kg. This DLT was characterized by transient elevations in liver enzymes and bilirubin which met the Hy’s Law definition for drug induced liver injury. No liver enzyme elevations were seen below the 1.5 mg/kg dose in this study. Trial participants who received a single GGF2 infusion showed a consistent dose-dependent improvement in left ventricular ejection fraction at 28 and 90 days after the infusion as compared to participants who received a placebo infusion. These cardiodynamic results and the safety findings were reviewed with the U.S. Food and Drug Administration (FDA), and the current trial was designed to further define safe dose level ranges while exploring efficacy measures and potential drug interactions.GGF2, which is part of a family of proteins known as neuregulins, has been shown to be pharmacologically active in a number of preclinical models of cardiovascular and neurological conditions. GGF2 acts directly on heart muscle cells, or cardiomyocytes. GGF2 may offer a unique treatment strategy as it is believed to improve the heart's ability to contract by promoting the repair of damaged heart muscle and improving its contractile function.