Santhera Starts Phase III Study with Raxone in DMD

Santhera Pharmaceuticals has announced that the first patient has been enrolled at the University of Kansas Medical Center (KUMC), Department of Neurology, Kansas (USA) in Santhera’s randomized, double-blind, placebo-controlled phase III (SIDEROS) trial. The trial will assess the efficacy of Raxone in slowing the rate of respiratory function decline in Duchenne muscular dystrophy (DMD) patients receiving concomitant glucocorticoids.

“We first observed the efficacy of Raxone in slowing the rate of respiratory function decline in DMD
patients in both glucocorticoid-using and non-using patients in the phase II DELPHI study,” commented Thomas Meier, PhD, CEO of Santhera.

“The successful Phase III DELOS trial which enrolled glucocorticoid non-using patients then confirmed a clinically relevant and statistically significant benefit of Raxone treatment on pulmonary function. The now initiated Phase III SIDEROS trial is designed to confirm the efficacy of Raxone in patients experiencing respiratory function decline that are currently taking glucocorticoids. If successful, this study will provide data that support use of Raxone in all DMD patients experiencing respiratory decline irrespective of their glucocorticoid use. The high level of interest from investigators and the patient community should allow us to recruit this study quickly.”

“We are hopeful that this phase III trial is the final step in the development program with Raxone in
DMD,” said Gunnar Buyse, MD, PhD, Professor of Child Neurology at the University Hospitals
Leuven (Belgium) and SIDEROS PI and Lead Investigator for Europe. “Following the exploratory
phase II program and the successful phase III DELOS trial, I am grateful that Santhera is committed
in exploring the full therapeutic potential of Raxone for patients with DMD.”

“Maintaining pulmonary function in patients with DMD has only recently become a prominent therapeutic objective in DMD, particularly in non-ambulatory patients,” added Oscar Henry Mayer, MD, Medical Director of the Pulmonary Function Testing Laboratory at the Children’s Hospital of Philadelphia and Lead Investigator for US. “A patient and caregiver survey conducted by Parent Project Muscular Dystrophy clearly demonstrated that the DMD community highly values treatment options for pulmonary complications.”