Acorda Launches Second Trial of GGF2

Acorda Therapeutics has enrolled the first patient in its second 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 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, according to the company. The 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.

In the first 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.