GGF2 (Glial Growth Factor 2) is known to stimulate the growth and differentiation of a variety of cells including glial cells, the support cells of the nervous system. These glial cells form the myelin sheath that insulates nerve cells and are essential for their survival and proper functioning. In demyelinating diseases such as multiple sclerosis, the myelin sheath is damaged, leading to the degeneration of nerve cells.

In preclinical studies, PAION’s license partner Acorda Therapeutics, Inc. (Acorda) demonstrated that GGF2 can stimulate the cell growth necessary to protect and regenerate a damaged myelin sheath. GGF2 is the lead neuregulin in Acorda’s portfolio. Neuregulins have also shown the ability to restore cardiac function in preclinical models of heart failure caused by myocardial infarction, heart rhythm disorders and myocardial dysfunctions.

In 2013, Acorda announced positive results of the Phase I trial with GGF2. The study identified a maximum tolerated dose of GGF2 and the preliminary efficacy measures showed that GGF2 improves heart function. Acorda discussed the findings from the study with the FDA and reached agreement on the next clinical study of GGF2 in heart failure. This Phase Ib study primarily involves the continued investigation of the safety profile but also the efficacy of GGF2 across a range of doses. The start of the study was made public by Acorda in October 2013.

In June 2015, Acorda announced that they had stopped enrolment in the trial based on the occurrence of a case of hepatotoxicity (liver injury) meeting Hy’s Law criteria, based on blood test results. Acorda also received a notification of clinical hold from the FDA following the submission of this information. There was one Hy’s Law case reported in the previous Phase I study. In both cases the abnormal blood tests resolved within several days. The 22 patients who were dosed in the trial will complete the pre-planned one year of follow up. Outside of the hepatotoxicity case, the safety profile from this trial was consistent with the first Phase I trial, but efficacy data was inconclusive which Acorda believes was in part due to the very small number of patients in the trial. Acorda has ongoing analyses and non-clinical studies to investigate the biological basis for liver effects, and will need to meet with the FDA to review these and other data from the cimaglermin studies and to request that the program be removed from clinical hold.


Under the following page you will find more information on PAION's partnership with Acorda Therapeutics, Inc.